[House Hearing, 110 Congress]
[From the U.S. Government Publishing Office]
HEARING ON STRATEGIES TO INCREASE
INFORMATION ON COMPARATIVE
CLINICAL EFFECTIVENESS
=======================================================================
HEARING
before the
SUBCOMMITTEE ON HEALTH
of the
COMMITTEE ON WAYS AND MEANS
U.S. HOUSE OF REPRESENTATIVES
ONE HUNDRED TENTH CONGRESS
FIRST SESSION
__________
JUNE 12, 2007
__________
Serial No. 110-46
__________
Printed for the use of the Committee on Ways and Means
U.S. GOVERNMENT PRINTING OFFICE
45-994 PDF WASHINGTON DC: 2009
---------------------------------------------------------------------
For Sale by the Superintendent of Documents, U.S. Government Printing Office
Internet: bookstore.gpo.gov Phone: toll free (866) 512-1800; (202) 512�091800
Fax: (202) 512�092104 Mail: Stop IDCC, Washington, DC 20402�090001
COMMITTEE ON WAYS AND MEANS
CHARLES B. RANGEL, New York, Chairman
FORTNEY PETE STARK, California JIM MCCRERY, Louisiana
SANDER M. LEVIN, Michigan WALLY HERGER, California
JIM MCDERMOTT, Washington DAVE CAMP, Michigan
JOHN LEWIS, Georgia JIM RAMSTAD, Minnesota
RICHARD E. NEAL, Massachusetts SAM JOHNSON, Texas
MICHAEL R. MCNULTY, New York PHIL ENGLISH, Pennsylvania
JOHN S. TANNER, Tennessee JERRY WELLER, Illinois
XAVIER BECERRA, California KENNY HULSHOF, Missouri
LLOYD DOGGETT, Texas RON LEWIS, Kentucky
EARL POMEROY, North Dakota KEVIN BRADY, Texas
STEPHANIE TUBBS JONES, Ohio THOMAS M. REYNOLDS, New York
MIKE THOMPSON, California PAUL RYAN, Wisconsin
JOHN B. LARSON, Connecticut ERIC CANTOR, Virginia
RAHM EMANUEL, Illinois JOHN LINDER, Georgia
EARL BLUMENAUER, Oregon DEVIN NUNES, California
RON KIND, Wisconsin PAT TIBERI, Ohio
BILL PASCRELL, JR., New Jersey JON PORTER, Nevada
SHELLEY BERKLEY, Nevada
JOSEPH CROWLEY, New York
CHRIS VAN HOLLEN, Maryland
KENDRICK MEEK, Florida
ALLYSON Y. SCHWARTZ, Pennsylvania
ARTUR DAVIS, Alabama
Janice Mays, Chief Counsel and Staff Director
Brett Loper, Minority Staff Director
______
SUBCOMMITTEE ON HEALTH
FORTNEY PETE STARK, California, Chairman
LLOYD DOGGETT, Texas DAVE CAMP, Michigan
MIKE THOMPSON, California SAM JOHNSON, Texas
RAHM EMANUEL, Illinois JIM RAMSTAD, Minnesota
XAVIER BECERRA, California PHIL ENGLISH, Pennsylvania
EARL POMEROY, North Dakota KENNY HULSHOF, Missouri
STEPHANIE TUBBS JONES, Ohio
RON KIND, Wisconsin
Pursuant to clause 2(e)(4) of Rule XI of the Rules of the House, public
hearing records of the Committee on Ways and Means are also, published
in electronic form. The printed hearing record remains the official
version. Because electronic submissions are used to prepare both
printed and electronic versions of the hearing record, the process of
converting between various electronic formats may introduce
unintentional errors or omissions. Such occurrences are inherent in the
current publication process and should diminish as the process is
further refined.
C O N T E N T S
__________
Page
Advisory of June 12, 2007, announcing the hearing................ 2
WITNESSES
The Honorable Thomas Allen, a Representative in Congress from the
State of Maine................................................. 6
______
Carolyn M. Clancy, M.D., Director, Agency for Healthcare Research
and Quality, Rockville, Maryland............................... 15
Peter R. Orszag, Ph.D., Director, Congressional Budget Office.... 21
Mark Miller, Ph.D, Executive Director, Medicare Payment Advisory
Commission..................................................... 43
______
Gail Wilensky, Ph.D., Senior Fellow, Project Hope, Bethesda,
Maryland....................................................... 79
David Dale, M.D., President, American College of Physicians...... 83
Gail Shearer, Director, Health Policy Analysis, Consumers Union.. 89
Susan Hearn, Ph.D., Senior Project Manager in Environment Health
and Safety, Dow Chemical Co., Midland, Michigan................ 95
Steve M. Teutsch, M.D., MPH, Executive Director, Outcomes
Research and Management, Office of Scientific and External
Affairs, Merck and Co.,Inc., West Point, Pennsylvania.......... 98
SUBMISSIONS FOR THE RECORD
Advanced Medical Technology Association, statement............... 116
Coalition for Health Services Research, statement................ 119
Epilepsy Foundation, Letter...................................... 124
Mental Heath America, statement.................................. 126
National Alliance on Mental Illness, Arlington, Virginia,
statement...................................................... 130
Pharmaceutical Research and Manufactures of America, statement... 132
Society of General Internal Medicine, statement.................. 134
HEARING ON MEDICARE PAYMENT ADVISORY
COMMISSION'S ANNUAL MARCH REPORT
----------
TUESDAY, JUNE 12, 2007
U.S. House of Representatives,
Committee on Ways and Means,
Subcommittee on Health,
Washington, DC.
The Subcommittee met, pursuant to notice, at 10:05 a.m., in
Room 1102, Longworth House Office Building, The Honorable
Fortney Pete Stark (Chairman of the Subcommittee) presiding.
[The advisory announcing the hearing follows:]
ADVISORY
FROM THE
COMMITTEE
ON WAYS
AND
MEANS
SUBCOMMITTEE ON HEALTH
CONTACT: (202) 225-3943
FOR IMMEDIATE RELEASE
June 12, 2007
HL-13
Stark Announces a Hearing on
Strategies to Increase Information on
Comparative Clinical Effectiveness
House Ways and Means Health Subcommittee Chairman Pete Stark (D-CA)
announced today that the Subcommittee on Health will hold a hearing on
strategies to increase research and information on comparative clinical
effectiveness. The hearing will take place at 10:00 a.m. on Tuesday,
June 12, 2007, in room 1100, Longworth House Office Building.
In view of the limited time available to hear witnesses, oral
testimony at this hearing will be from the invited witness only.
However, any individual or organization not scheduled for an oral
appearance may submit a written statement for consideration by the
Committee and for inclusion in the printed record of the hearing.
BACKGROUND:
Rising health care costs and a lack of evidence justifying extreme
variations in the provision of medical services have led many to call
for a Federal effort to substantially increase information on the
relative effectiveness of health care services. Health policy experts
across the political spectrum advocate that such information is a
sorely needed public good, and that greater investment in comparative
effectiveness research is critical to assuring high-quality care and
reducing unnecessary spending.
Various authorities both within and outside government have called
for national investment in comparative effectiveness information, and
have identified issues and options to help Congress determine optimal
financing and governance for this activity. Providing Medicare with
better information about the relative strengths and weaknesses of
various products, procedures and services will help public and private
payers equitably and efficiently manage rising health care costs.
On announcing this hearing, Chairman Stark said: ``As Medicare's
steward, Congress needs to ensure that Medicare resources are being
used effectively to provide high quality care and achieve the best
possible patient outcomes. Getting reliable, unbiased comparative
information is our best shot at reducing health spending while
improving care and preserving access. This hearing will identify ways
we can get the information we need to achieve this important goal.''
FOCUS OF THE HEARING:
The hearing will review the benefits of comparative effectiveness
research, and opportunities for the Federal Government to expand the
availability and value of such research.
DETAILS FOR SUBMISSION OF WRITTEN COMMENTS:
Please Note: Any person(s) and/or organization(s) wishing to submit
for the hearing record must follow the appropriate link on the hearing
page of the Committee website and complete the informational forms.
From the Committee homepage, http://waysandmeans.house.gov, select
``110th Congress'' from the menu entitled, ``Committee Hearings''
(http://waysandmeans.house.gov/Hearings.asp?congress=18). Select the
hearing for which you would like to submit, and click on the link
entitled, ``Click here to provide a submission for the record.'' Once
you have followed the online instructions, completing all informational
forms and clicking ``submit'' on the final page, an email will be sent
to the address which you supply confirming your interest in providing a
submission for the record. You MUST REPLY to the email and ATTACH your
submission as a Word or WordPerfect document, in compliance with the
formatting requirements listed below, by close of business June 26,
2007. Finally, please note that due to the change in House mail policy,
the U.S. Capitol Police will refuse sealed-package deliveries to all
House Office Buildings. For questions, or if you encounter technical
problems, please call (202) 225-1721.
FORMATTING REQUIREMENTS:
The Committee relies on electronic submissions for printing the
official hearing record. As always, submissions will be included in the
record according to the discretion of the Committee. The Committee will
not alter the content of your submission, but we reserve the right to
format it according to our guidelines. Any submission provided to the
Committee by a witness, any supplementary materials submitted for the
printed record, and any written comments in response to a request for
written comments must conform to the guidelines listed below. Any
submission or supplementary item not in compliance with these
guidelines will not be printed, but will be maintained in the Committee
files for review and use by the Committee.
1. All submissions and supplementary materials must be provided in
Word or WordPerfect format and MUST NOT exceed a total of 10 pages,
including attachments. Witnesses and submitters are advised that the
Committee relies on electronic submissions for printing the official
hearing record.
2. Copies of whole documents submitted as exhibit material will not
be accepted for printing. Instead, exhibit material should be
referenced and quoted or paraphrased. All exhibit material not meeting
these specifications will be maintained in the Committee files for
review and use by the Committee.
3. All submissions must include a list of all clients, persons,
and/or organizations on whose behalf the witness appears. A
supplemental sheet must accompany each submission listing the name,
company, address, and telephone and fax numbers of each witness.
Note: All Committee advisories and news releases are available on
the World Wide Web at http://waysandmeans.house.gov.
The Committee seeks to make its facilities accessible to persons
with disabilities. If you are in need of special accommodations, please
call 202-225-1721 or 202-226-3411 TTD/TTY in advance of the event (four
business days notice is requested). Questions with regard to special
accommodation needs in general (including availability of Committee
materials in alternative formats) may be directed to the Committee as
noted above.
Chairman STARK. The Subcommittee will begin. Today is
another in our series of hearings on how to improve Medicare,
and we'll focus on long-term solutions to increase and assure
the value of health care expenditures. This we hope to deal
with the issue of comparative clinical effectiveness. That
means comparing the relative value of different clinical
treatments, drugs, devices, tests, procedures, bandages, pills,
anything else you want to take, and trying to get a comparative
ranking.
Too often, physicians and patients have trouble
understanding when a new product or test or procedure will be
most helpful and how to choose among existing courses of
treatment. Given the absence of information on comparative
effectiveness, it's hardly surprising that GAO and MedPAC find
dramatic variation in the use of medical services across
regions, from different providers, by different specialties.
Even worse, researchers find that the areas with the highest
use of some services aren't necessarily linked to higher
quality or better outcomes. To the contrary, beneficiaries may
be put at greater risk when they're subjected to more and more
complicated tests and treatments.
As Medicare's Board of Directors, Congress should ensure
that Medicare resources are used effectively and efficiently to
provide high-quality care and achieve the best possible
outcomes. Getting reliable, unbiased comparative information is
our best chance at controlling health care spending while
improving care and access. Even if Mr. Orszag won't give us
savings immediately for our efforts, we can identify ways that
we can get the information we need to achieve this goal and lay
the groundwork for a more efficient and effective system.
Health policy experts across the political spectrum
advocate that comparative information is sorely needed for the
public good. They argue that greater investment in comparative
effectiveness research is critical to assuring high-quality
care and reducing unnecessary expenditures. Better information
about the relative strengths and weaknesses of various
products, procedures and services will help physicians and
patients make wise decisions and will help public service and
private payers equitably manage rising health care costs.
Many countries have already made major investments to
provide this information to physicians, patients, policymakers,
and it's high time we do the same. Many of my colleagues urge
that we should pay for performance. We already do that. The
providers perform and we pay. It's just that we pay the same
whether the service is done on the right people at the right
time or the wrong people at the wrong time. We really have to
know what the effective and appropriate services are before we
can know how to reward the care that achieves the best
outcomes.
Various authorities both within and outside government have
called for a substantial national investment in comparative
effectiveness information and have identified issues and
options to help us determine the optimal financing and
governance for this activity. My personal preference is to move
toward a system that's accountable yet independent and free
from both industry and political influence. I want to repeat
that. Free from both the industry and political influence. Both
clinicians and patients need to be confident that the work that
will be done is in the best interests of the patient. To me,
that points to a government-led effort.We are fortunate to have
with us today some of the leading experts as well as
representatives of the prominent stakeholders. I am
particularly pleased to welcome our first witness, but prior to
that and prior to my introduction of him, I'd like to yield to
Mr. Camp for any remarks he'd care to make.
Mr. CAMP. Well, thank you, Chairman Stark. The U.S. health
care system lacks the kinds of comparative information that
would allow consumers to make informed health care choices and
providers to prescribe the best course of care. While we have
agencies like the FDA to determine if drugs and devices are
safe, we have very little information that compares the actual
effectiveness of drug devices and medical procedures.
Frequently, physicians lack the information that would allow
them to compare what treatments work best for particular types
of patients. This type of data could help health care providers
identify best practices, ultimately leading to improved
clinical outcomes.
Comparative effectiveness data will also be necessary if we
are to ever more to a more consumer-focused model of delivering
health care, where individuals are able to make more choices
about the care they receive. This information also has the
ability to help reduce expenditures under the major Federal
health programs by helping to eliminate unnecessary procedures,
leading to a significant savings for taxpayers.
At the same time, we cannot allow comparative effectiveness
to become another hurdle that slows patients' access to new
therapies. The current health care bureaucracies, especially
those in the Medicare and Medicaid programs, are often unable
to keep up with the dynamic and changing nature of health care.
Government agencies should not be required to use or to rely
solely on comparative effectiveness data to set reimbursements
or make coverage decisions.
Comparative effectiveness can be a building block that
leads to better health outcomes, but only if it's done in a
transparent and timely way. This will require a process that
encourages independent research, which also recognizes that not
all therapeutic choices will be appropriate for all patients.
Patient protections must be in place to make certain that
unique patients are not harmed in the name of cost saving
initiatives. Put simply, comparative effectiveness should be
viewed as a tool to influence decisions. It should not,
however, be used to limit patient access to the best course of
treatment.
I look forward to hearing the testimony of the witnesses
today, and hope that we can work together on legislation that
will provide providers and patients with the information that
can improve the quality of their health care.
Thank you, Mr. Chairman. I yield back the balance of my
time.
Chairman STARK. You're welcome. Mr. Camp, I'd like to
associate myself with your remarks. I think our, at least my
interest in this effort, is to get us the information. I don't
want to predetermine how we would use that information, but
right nowand I've said this often to other people. We just went
through an allergy season that was one of the worst, my
pediatrician tells us, in 14 years. But you saw six or eight or
ten antihistamines in every popular magazine, but nobody really
had good records of which one was the best to use for your
kids.
As I'd say to the people in the audience, when the men in
the audience get prostate cancer, you won't know what is the
best treatment. You'll know that you'll live through the
initial treatment whether you do chemo or radiation or surgery.
We just don't have figures that are reliable that will tell you
what happens to you 10 years out. The same with the ladies in
the audience who may unfortunately get breast cancer. You won't
have the kinds of statistics we need for you to predict with
your physician what's the best course for you to follow. I hope
that whatever comes out of the work we do on this Committee
will help providers, beneficiaries, the taxpayers and everybody
to understand that better.
To that end, I'm pleased to welcome our first witness,
Congressman Tom Allen from Maine, who has been a leader in the
House on the issue of comparative effectiveness and has
recently submitted legislation to address this problem. The
subsequent witnesses I'm sure will enlighten us on whatever
their strategy is to improve health care in the United States,
and I look forward to their testimony.
Tom, would you like to enlighten us in any manner you'd
care to? If you have a prepared statement, it will appear in
the record in its entirety without objection.
STATEMENT OF THE HONORABLE TOM ALLEN, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF MAINE
Mr. ALLEN. Thank you, Mr. Chairman. Thank you for holding
this hearing, and I want to thank you and Ranking Member Camp
for inviting me to testify today. I would ask my entire
statement to be submitted.
Chairman STARK. Without objection.
Mr. ALLEN. I will talk from that statement. We have a huge
gap in our health care system today: the absence of
independent, evidence-based information about the comparative
effectiveness of prescription drugs, medical devices and other
treatments that deal with the same illness or condition.
Better information on how various treatments compare to one
another will enable doctors and their patients to make informed
decisions about whether new or high-priced drugs, devices and
other medical treatments do or do not provide better clinical
outcomes. The health care marketplace will be greatly enhanced
by independent information more widely disseminated.
We've seen remarkable innovations in health care in recent
years, but the aggregate health care costs have grown
considerably faster than the overall economy. The United States
spends more than any other nation on health care, but we rank
37th in the world in health outcomes. Nearly 47 million
Americans are uninsured, and millions more are underinsured.
On May 7th of this year, I introduced H.R. 2184, the
Enhanced Health Care Value for All Act, with Representative Jo
Ann Emerson, who has been with me on this issue for the
lastwell, this is the third Congress. The bill extends the
success of the Agency for Healthcare Research and Quality's
Effective Health Care Program and provides more funding for on
the comparative effectiveness of health care services,
including prescription drugs, medical devices, procedures, and
other treatments, and can include clinical trials as well as
surveys of existing literature.
Better clinical information on health care products and
service is a public good. Therefore, the legislation creates a
public-private funding mechanism to pool Federal resources with
funds from health insurance plans and large employers with
self-insured plans.
The investment in comparative effectiveness studies
provides a sound, bipartisan approach to a fundamental
challenge: how to ensure that we get the best value for our
health care dollar. This initiative has gained the support of a
very broad group of stakeholders representing patients, medical
professionals, health services researchers and health care
purchasers.
My bill expands on Section 1013 of the Medicare
Prescription Drug Improvement and Modernization Act of 2003.
That provision authorized AHRQ to carry out systematic reviews
of existing research on the clinical comparative effectiveness
and safety of prescription drugs and other treatments. Section
1013 was based on legislation that Representative Emerson and I
introduced in the 108th Congress, and H.R. 2184, our current
bill, expands on the small initial investment in comparative
effectiveness, $15 million appropriated by Congress in the last
two fiscal years.
Leaders at CMS, MedPAC, the Institute of Medicine and CBO
have begun to explore this issue in greater detail. Economists
Uwe Reinhardt, Stuart Altman and former CMS Administrator Gail
Wilensky have put forth bold visions on where increased
investment in comparative effectiveness could take us.
To have the best medical outcome, patients need the right
care at the right time. Without good information on comparative
effectiveness, we can't be confident that we are using products
in the optimal way. The FDA approval process, as Mr. Camp
indicated, does not give us this data, because it tests only
for safety and for effectiveness compared to a placebo, not
effectiveness compared to other drugs or devicesother drugs in
that casethat treat the same illness or condition.
In closing, I want to make four critical points:
Number one. More effective treatments mean better health
outcomes and reduce side effects.
Two. Overall comparative effectiveness data doesn't negate
the need for individualized care. this is not, as some critics
argue, a path to one-size-fits-all medicine.
Three. Broadening comparative effectiveness studies will
provide better information about differential impacts on
subpopulations and the interaction of various treatments in
patients with multiple illnesses. In other words, broader
information will be very helpful.
Fourth. High quality comparative effectiveness studies
shouldn't threaten innovation. Instead, they will clarify the
many reasons individuals can be exceptions to ``average''
outcomes and will facilitate transition to a system of
affordable, personal health care.
I thank you very much for having me here today and look
forward to working with you on this legislation.
[The prepared statement of Mr. Allen follows:]
Prepared Statement of The Honorable Thomas Allen,
a Representative in Congress from the State of Maine
Mr. Chairman and Ranking Member Camp, thank you for inviting me to
testify today.
We have a huge gap in our health care system: the absence of
independent, evidence-based information about the comparative
effectiveness of prescription drugs, medical devices, and other
treatments that treat the same illness or condition.
Having better information on how various treatments compare to one
another will enable doctors and their patients to make informed
decisions about whether new or high priced drugs, devices, and other
medical treatments do or do not provide better clinical outcomes. The
health care marketplace will be greatly enhanced by independent
information more widely disseminated.
We have seen remarkable innovations in health care in recent years.
New discoveries in medicine, medical devices and treatments have
improved the quality of life and extended the average life expectancy
of Americans.
Yet aggregate health care costs have grown considerably faster than
the overall economy, contributing to double digit inflation for health
care services. By 2016, U.S. health care spending is expected to almost
double to $4.1 trillion and account for 20 percent of every dollar
spent.
The U.S. spends the most per capita of any nation on health care,
yet it ranks behind most industrialized nations on major health
outcomes. Nearly 47 million Americans are uninsured, and millions more
are underinsured.
On May 7, 2007 I introduced H.R. 2184, the Enhanced Health Care
Value for All Act, with Representative Jo Ann Emerson. The bill builds
on the success of the Agency for Health Care Research and Quality's
``Effective Health Care Program'' and provides increased funding to
finance new research on the comparative effectiveness of health care
services (including prescription drugs, medical devices, procedures,
and other treatments), which may include clinical trials.
Recognizing that better clinical information on health care
products and services is a public good, the legislation creates a
public-private funding mechanism which will pool federal resources with
funds from health insurance plans and large employers with self-insured
plans.
Investment in comparative effectiveness studies provides a sound,
bipartisan approach to a fundamental challenge: how to ensure that we
get the best value for our health care dollar. This initiative has
gained the support of a broad group of stakeholders representing
patients, medical professionals, health services researchers and health
care purchasers.
My bill expands on Section 1013 of the Medicare Prescription Drug,
Improvement, and Modernization Act of 2003. This provision provided
authority for the Agency for Health Care Research and Quality to carry
out systematic reviews of existing research on the clinical comparative
effectiveness and safety of prescription drugs and other treatments.
Sec. 1013 was based on legislation that Representative Emerson and I
introduced in the 108th Congress (H.R. 2356) which gained broad
bipartisan support.
The Enhanced Health Care Value for All Act expands on the small
initial investment in this research of $15 million appropriated by
Congress in Fiscal Years 2005, 2006 and 2007.
Leaders at the Centers for Medicare and Medicaid Services, Medicare
Payment Advisory Commission, and Institute of Medicine have also begun
to explore this issue in greater detail. Economists Uwe Reinhardt,
Stuart Altman and former Health Care Financing Administration (now CMS)
Administrator Gail Wilensky have put forth bold visions on where
increased investment in comparative effectiveness could take us.
There is widespread agreement that to have the best medical
outcome, patients need the right care at the right time. If we don't
have good information on comparative effectiveness we can't have strong
confidence we are using products in the optimal way. The FDA approval
process does not give us this data. For example, it approves drugs
after testing for safety and effectiveness compared to a placebo, not
for comparative effectiveness at treating a particular illness.
In closing, I would like to make four critical points about
comparative effectiveness studies:
1. More effective treatments mean better health outcomes and
reduced side effects.
2. Overall comparative effectiveness data doesn't negate the
need for individualized care. This is not, as some critics
argue, a path to ``one size fits all medicine.''
3. Broadening comparative effectiveness studies will provide
better information about differential impacts on sub-
populations and the interaction of various treatments in
patients with multiple illnesses.
4. High quality comparative effectiveness studies shouldn't
threaten innovation; instead they will clarify the many reasons
individuals can be exceptions to ``average'' outcomes, and will
facilitate transition to a system of affordable, personalized
health care.
Thank you for inviting me to testify today. I look forward to
working with you to move toward a common goal: improving value in
health care spending and ensuring that patients have access to the
treatments that are right for them.
Chairman STARK. Tom, thank you. If I understand your bill
correctly, the question of who gets to plead their product or
service at this court of effectiveness, that the entry key
would be an FDA approval, for example.
Mr. ALLEN. Yes.
Chairman STARK. So, first you'd have to go through the
process of proving that it was safe and as opposed, say, to a
placebo, it did something.
Mr. ALLEN. Right.
Chairman STARK. That you just weren't bringing a placebo to
the table.
Mr. ALLEN. That's correct. I don't think it's right to slow
down the process of getting approval at the FDA level, because
I think that the AHRQ ought to be able to decide the
appropriate time to conduct research on a particular group of
drugs or a particular group of devices or treatments.
Chairman STARK. Would the stakeholders haveor the
manufacturers, the providers, the physicians, the
pharmaceutical companies, whomeverwould they have input into
selecting theor setting the agenda for this panel? In other
words, there's certainly going to be more people lined up
wanting an effectiveness test, then there will be a time lag.
Howhave you given any thought to how that would be fairly
determined?
Mr. ALLEN. Well, what we've done is we create in this
legislation, we expand the authority for the comparative
effectiveness advisory board, and we want to make sure that the
selection of the members of that board takes out people with
financial interests in stakeholders, whether they be
manufacturers of devices or manufacturers of prescription
drugs.
Then for each individual study, there is a clinical
advisory panel. We want the same transparency, the same process
there to make sure that the people with a financial interest in
the outcome of the study are not choosing the people who will
oversee it.
Chairman STARK. So, to pick an example, MedPAC. Would that
meet the standards that you have in mind? The way it's
formulated and the panel is picked?
Mr. ALLEN. Yes. We're looking for panels that have some
degree of political oversight, because all of this run
ultimately through the Secretary of Health and Human Services,
but is as independent as possible and as transparent, and fully
transparent, so that conflicts of financial interests are
weeded out. So, you'll really have the independent, evidence-
based product at the end of the day that we need to do this
research effectively.
Chairman STARK. Now I hesitate to bring this up, but I know
you're talking about buy in effect an all payer system, which
makes sense. But can you give us a ballpark idea of what we're
talking about in costs? I know you have it coming out ofthe
money coming out of the trust fund. The question, could we use
appropriated funds? I don't know. But what kind of agive me a,
to the closest billion dollars or so what are we going to spend
on this?
Mr. ALLEN. Well, as I understand it, ultimately, that's up
to this Committee and the Congress as a whole. But the
legislation, we think the appropriate scale of investment os $3
billion over 5 years, and that one-third of that, $1 billion,
should be public money and should come from the Medicare trust
fund, limited to a maximum of $200 million a year.
The other we have support from the insurers and from large
employers with self-insured plans, and they have bought into
the concept of funding the other two-thirds. Because, remember,
this information is going to be of great help to insurers and
to large companies with self-insured plans. They're going to
use it their own way. What we're simply trying to do is get
good, evidence-based information out there for the public.
Chairman STARK. So, what you're suggesting is that for the
public, the beneficiaries at large, the public, the government
should come up with that money out of revenues of one sort or
another? The other two-thirds would be contributed by the
providers and/or the manufacturers, people with a commercial
interest at stake?
Mr. ALLEN. Well, by the insurance plans and by large
employers. We are not suggesting that the pharmaceutical
industry contribute to that fund. We are saying those who use
the information in their own way. This kind of research is
being done now by individual insurance companies, but because
that information is not widely shared, it's notit doesn't have
the impact that it could.
Chairman STARK. Thank you. Mr. Camp?
Mr. CAMP. Well, thank you, Mr. Chairman. Mr. Allen, thank
you for your testimony. Obviously, we often lament the fact
that we don't have good enough data or information even for
policy decisions, much less so that individuals and physicians
can make the appropriate decisions on the kind of care, as the
Chairman mentioned earlier in this hearing.
But, obviously, I think there's a broad agreement that this
kind of information, comparative effectiveness research, could
help informed decision making in the health care area.
But as you look at other nations that do this, and they're
further along than we are, there's a concern that insurance
companies, maybe even CMS in America, could use this
information to limit access to certain treatments, because
someone may not fit the ``average'' stereotype. I realize there
could be subgroups that might sort of mitigate that. But does
your legislation have any specific safeguards to assure that
access to patient-centered treatments is still available?
Mr. ALLEN. I would put it this way. The legislation is
designed simply to deal with access to information and make
that information available. We would obviously be open to
suggestions along the lines that you are making here today. But
the legislation itself is very clear. In your opening, you
raised the concern about whether the information would inform
decisions or drive decisions. It is not the intention of this
legislation to drive decisions. But we're obviously open to
suggestions to clarify the point that you're making today.
Mr. CAMP. Why did you choose the comptroller general and
not have this be a part of AHRQ? Any particular reason?
Mr. ALLEN. In terms of selecting the panels?
Mr. CAMP. Yes. Sort of the authority or the chain of
command of this group would report to the comptroller general.
I just was wondering your thought process behind that choice
and not some other model.
Mr. ALLEN. What we are trying to do is get a mix of some
political oversight and some independence from politics. We are
trying to get something like the MedPAC board. I mean, that's
the goal. It's a bit of a difficult situation, because though
we call for a study of completely independent funded centers,
research centers, we think that probably, probably that leaves
you never can be quite sure that you've got either the
transparency and maybe the risk of being infiltrated by parties
with interests in that kind of system.
We think we've got the right balance, but that's why we
were doing it. Trying to get a balance with some control and
the right mix of control and independence.
Mr. CAMP. All right. Thank you. Thank you, Mr. Chairman.
Chairman STARK. Mr. Doggett?
Mr. DOGGETT. Well, thank you very much for your testimony.
I don't know of another Member of Congress who has given more
thought than you have to different ways to improve our health
care system, and I think this is one important aspect of that.
As far as the funding, Chairman Stark. explored that with
you a little bit. I know that, for example, on NIH, that's all
done through appropriated funds. Should we be considering an
alternative that focuses on appropriated funds to pay for this
important public purpose?
Mr. ALLEN. I would say that we believe, I believe, that an
all payer system better reflects the sharing of the benefits.
That you can try to pay for this entirely through appropriated
funds, but then it becomes a heavier lift in terms of getting
comparative effectiveness research on the scale that I believe
it's needed.
In fact, you alreadywe already have buy-in from insurance
plans and some large employers who believe and understand that
it will help both improve the quality of their plans and their
management of their plans, and drive down their costs. So, the
benefits are both public and private. There are savings to the
public systems and there are savings to the private systems,
and that's why we argue an all payer plan makes the most sense.
Mr. DOGGETT. Thank you very much for your important
recommendation.
Chairman STARK. Thank you. Mr. Ramstad, would you like to
inquire?
Mr. RAMSTAD. Thank you, Mr. Chairman. Tom, good to see you
as always.
Mr. ALLEN. Thank you.
Mr. RAMSTAD. I appreciate your kicking off this dialog.
Like many concepts, I think comparative effectiveness sounds
good in theory. Who can argue with comparison and
effectiveness, comparative effectiveness? But I do have
concerns about its implementation, just to follow up on Mr.
Camp's line of questioning.
My question is this. With respect to your legislation, I'm
concerned about the power that the single comparative
effectiveness entity or authority might have to determine
what's covered by insurance, again, to follow up on Mr. Camp's
questioning.
Let me ask you specifically, would this advisory board,
this comparative effectiveness advisory board, as provided in
your legislation, make specific recommendations about which
procedures, which devices, which drugs Medicare and private
plans should cover or not cover?
Mr. ALLEN. My understanding of the legislation the way it's
meant to work is the answer is no. The answer is, as I
understand it, simply the studies will be done. They will be
available on a public website for those who want the
information to review, and to use in making their own
decisions. This legislation does not try to drive the
decisionmaking process of other bodies.
Mr. DOGGETT. So, it wouldn't preempt current law with
respect to that decisionmaking process?
Mr. ALLEN. That is correct.
Mr. DOGGETT. It wouldn't say we recommend that Device A
should be reimbursed and Device B should not?
Mr. ALLEN. That is correct.
Mr. DOGGETT. Okay. That's all I have. Thank you again, Tom.
Mr. Chairman, I yield back.
Chairman STARK. Thank you. Mr. Camp, we have some full
Committee Members who are not on the Subcommittee. Could they
thank you. Then I'd call one of our distinguished Members who
is here, Mr. Becerra. Would you like to inquire?
Mr. BECERRA. Mr. Chairman, I just wanted to acknowledge and
welcome our colleague from Maine, Mr. Allen, for being here
with us. I applaud him on his effort, but I will yield to my
other colleagues.
Chairman STARK. Ms. Schwartz, would you like to?
Ms. SCHWARTZ. Thank you. I might hold my questions for the
next panel, but I do think the interest in expanding our
information about effectiveness and comparing that
effectiveness is potentially very, very helpful to providers
making that decision. I do share the concern I think of the
other side. Mr. Camp mentioned about this information not being
used in a way to limit access, particularly when it's a
specific device or treatment is necessary for a particular
person.
So, it's interesting information I think partly how we
expand and how we get that information out there, but then how
do we actually make sure it's used in the best way possible to
help our providers provide the best care for the consumers in
this country.
Chairman STARK. Mr. McDermott?
Mr. MCDERMOTT. Thank you, Mr. Chairman. Tom, in answer to
your question, or Congressman Allen, in answer to your
questions to Mr. Camp and Mr. Ramstad, this is basically a
toothless tiger. It will gather up information and have
information on a website but have no impact, no intended
impact. Is that correct?
Mr. ALLEN. I think that sound, evidenced-based information
on complicated topics that are widely available to providers
and the public has an impact; that it drives decisions. I'll
give you an example where it might have been effective in the
past. Celebrex and Vioxx, two drugs to treat osteoarthritis,
were advertised at the level, you know, several hundred
millions of dollars went into advertising those drugs. They
were best used, according to the research, for those people
who, when taking ibuprofen, had gastrointestinal troubles.
But they were marketed through Dorothy Hamill and others to
a much broader to the entire population as if the entire
population had something to gain. Simple comparative
effectiveness studies on Celebrex, Vioxx and ibuprofen, to give
just one example, might have driven a very different, number
one, marketing strategy, but also a very different strategy by
those who were prescribing those particular the two
prescription drugs.
I would add, specifically in the case of those drugs, if we
had had broader information, surveys of existing literature,
ongoing comparative effectiveness studies that were
independent, the problems with Vioxx, for example, might have
been discovered considerably sooner. That's why I don't believe
that there's a need at this moment. I think we need to create
the system and provide the information, and that information
will drive decisionmaking in a helpful way.
Mr. MCDERMOTT. The reason I asked the question is I guess
if you've been here a while you sort of see stuff and you see
it happen and you wonder about it. The quality control agency
was a very effective agency in the past. Once they did a study
on back surgery. They suggested that back surgery in many
instances was not useful at all. They immediately were attacked
by those folks who were the beneficiaries of that kind of
surgery, not the patients, but the professionals involved in
it. Their study was discredited, and there was a great big
hooha because they were about to use this in Medicare in terms
of making decisions about whether or not they were going to
give it.
Now I wonder about why more information. I know insurance
companies. I've dealt enough with them, when we did the effort
with Mrs. Clinton back in 1993. Insurance companies have panels
where they decide what they're going to put out and what
they're going to pay for, what they're going to use stem cell
transplants for, or what they're going to use bone marrow
transplants for, or what illnesses they're going towhat are
they going to use and pay for. They don't share that
information at all. You can't find out from Blue Cross/Blue
Shield who makes those technical decisions.
I guess I have some problem with creating another set of
evidence-based medicine evidence, but not using it to drive
decisionmaking in terms of how we control cost.
It seems to me that we're just saying let the market do
whatever it wants to do, and anybody who can come up with a new
device comes in and somehow gets it approved and away it goes,
there has to be some way you look at the evidence as to whether
this device is actually better than this device, or this
medicine better than this medicine. Otherwise, there is no end
of the cost in health care. That's what we've got today. That's
why we've got the prices going out of sight.I don't did you
just figure you didn't want to use it yet until you had the
information gathered? Or do you think it's not a good idea to
have the government in the position of trying to protect the
patient's cost?
Mr. ALLEN. I would say that we believe that by itself, all
by itself, bettera widerwider availability of better
information, evidenced-based information that's generated by
independent studies, not by the manufacturers of the device or
the drug, and that serves everyone, that's available to
everyone, it's available to the public and people running the
public bodies. It's available to doctors and hospitals and
other health care providers.
That information is going to, we believe, just by being out
there and being of the kind of information of which there isn't
enough now, we think that is going to drive better health care
decisions both in terms of quality and in terms of cost.
Now, I mean this is, you know, my crystal ball only works
some days and not today. But I think all by itself, having
significantly more and better information on comparative
effectiveness, is going to drive better decision making and
save cost at the same time.
Mr. MCDERMOTT. I hope you're right.
Mr. ALLEN. I hope I am, too. Mr. Chairman?
Chairman STARK. Go ahead.
Mr. ALLEN. Can I just clarify one?
Chairman STARK. Sure.
Mr. ALLEN. When Mr. Camp was questioning me, I want to
clarify some of the things I said right then. The comparative
effectiveness advisory board reports ultimately to the
Secretary of Health and Human Services and to Congress, not to
the comptroller general. The comptroller general's only role is
to oversee the advisory board's selection process, as it does
for MedPAC. It's an attempt to get and you may have, I didn't
say this, and you may have understood this. But it's the
attempt to make that selection process of the advisory board
independent of political considerations.
Chairman STARK. Thank you. I want to thank you for your
efforts and your testimony and assure you that as soon as one
of my colleagues plagiarizes your good bill and introduces it
under their own name, it will probably scoot right through this
Committee.
[Laughter.]
Chairman STARK. You'll be left out there wondering what
happened.
Mr. ALLEN. Mr. Chairman, you can't be serious.
Chairman STARK. Oh, ho ho. But thank you very much, and we
appreciate your contribution to our venture here in learning
about this.
Mr. ALLEN. Thank you.
Chairman STARK. You're going to be followed by a panel of
government experts in this case; Dr. Carolyn Clancy, who is the
Director of the Agency for Healthcare Research and Quality, and
Dr. Peter Orszag, who is the Director of the Congressional
Budget Office.
Dr. Mark Miller, who advises us frequently and often and
most helpfully on issues before us in the Medicare arena.
After you are seated, get a chance to get settled, we will
have you proceed in the manner in which I called you.Without
objection, your full prepared testimony will appear in the
record and you can enlighten us in any manner in which you are
comfortable.
Dr. Clancy, would you like to lead off?
STATEMENT OF CAROLYN M. CLANCY, M.D., DIRECTOR, AGENCY FOR
HEALTHCARE RESEARCH AND QUALITY, ROCKVILLE, MARYLAND
Dr. CLANCY. Thank you. Good morning, Mr. Chairman and
Members of the Subcommittee. I am very pleased to testify
before you on the exciting issue of comparative effectiveness.
I ask that my written testimony and samples of AHRQ's
comparative effectiveness reports and other materials be made
part of the record.
I am thrilled about the growing interest in and attention
to enhancing the role of comparative effectiveness research in
our health care system. I am particularly pleased to be able to
tell you about the important work that my agency, the Agency
for Healthcare Research and Quality, is doing in the area of
comparative effectiveness.
AHRQ's mission is to improve the quality, safety,
efficiency, and effectiveness of health care for all Americans.
Effectiveness in our mission includes comparative
effectiveness: What is the right innovation or treatment for
the right patient at the right time?
Comparative effectiveness research is a means to an end.
Our mission is fulfilled when health care decisionmakers,
including patients, clinicians, purchasers, and policy-makers,
use up-to-date evidence-based information about their treatment
options to make informed health care decisions. Since they are
making these decisions every day, we have a sense of urgency
about this work.
As a doctor and as AHRQ's director, I am very pleased that
Congress recognized the programs and infrastructure the AHRQ
has established for providing the health care system with
scientific evidence when it authorized us to do comparative
effectiveness research. This infrastructure enabled AHRQ to
establish our Effective Health Care program and begin work
rapidly without having to reinvent the wheel or create a new
bureaucracy.
In addition, our reputation as an unbiased and trusted
source of information has moved the health care system to adopt
and use the findings to improve the quality, safety, and
effectiveness of health care.
An important hallmark of the Effective Health Care program
is transparency in all aspects of the process. Priorities for
the program are set after receiving broad public input through
Federal Register notices, public listening sessions, and other
means.
We have been very fortunate to have developed very
cooperative relationships with a broad range of stakeholders,
and we actively seek out comments and input from as many as
possible.
The public and all interested stakeholders have the
opportunity to comment on the initial research priorities,
framing of the specific questions, and draft reports. We also
extensively focus test and seek input from consumers on
materials that we develop specifically for them based on our
comparative effectiveness reports.
In addition to the open invitation to comment,
manufacturers are notified when a study is begun that affects
their products, and are invited to submit relevant studies and
data. Input from manufacturers can raise questions and
concerns. We have worked with our researchers to develop a
process to minimize conflicts so we can create public/private
partnerships with confidence.
As AHRQ has implemented the Effective Health Care program,
we have five significant observations to share. First is
priority-setting. It is important to set clear priorities that
meet the needs of all the stakeholders in health care.
Therefore, end users and stakeholders must continuously provide
input through an open and transparent process.
Second is framing the research questions. Research must
follow how clinicians and patients make health care decisions
every day. After much deliberation, we decided that our
research should focus on conditions rather than interventions.
At the end of the day, that is how health care decisions are
made. It is also very important to recognize the importance of
updating findings frequently to incorporate new evidence that
may change or modify the conclusions about what works best and
for whom.
The third relates to balancing benefits and harms.
Comparative effectiveness research, by definition, must provide
information on benefits and harms of a particular medication or
intervention. Evaluating the balance of harms and benefits is a
critical component of informed decisionmaking.
Few interventions are risk-free, and for many chronic
conditions the therapeutic goal is management of symptoms and
disease state rather than cure. Oftentimes the decision comes
with some assumption of harm by both patient and clinician, but
with the understanding that the benefits are worth that risk.
Fourth, research is a means, not an end. The ultimate goal
of our research efforts is to develop timely, relevant
information for decisionmaking. This requires us to go well
beyond the products of traditional research, namely, scholarly
articles, and translate findings in language and formats that
are appropriate for different audiences. Our goal is that
valid, reliable, and useful information be there when and where
people need it.
Fifth, trust is incredibly important. We view it as a
process, not a structure. As Gail Wilensky has said,
comparative effectiveness research can be risky business, and
there may be winners and losers. While we have learned that
there are often no clear winners and losers, but differing
risks and benefits between different interventions, it is still
important that there be a level playingfield among all
stakeholders. For that reason, our program has adopted a policy
of transparency and inclusion.
In addition, it is clear that the program's success to date
is dependent on effective collaboration with scientists from
industry as well as academia. We are working very carefully to
minimize conflicts in the analysis and development of
knowledge, while taking advantage of the collective knowledge
of a variety of different researchers through peer review and
methodological work.
The question of trust also extends to the integral role
that patients plan in research. Although government and the
private sector pay for research, patients assume the risks and
benefits of enrolling in clinical trials and other studies. For
that reason, it is critically important to recognize that these
findings need to be carefully translated so that patients can
receive the benefits of this knowledge in making their
decisions.
We all need to learn from the knowledge gained in research.
But for patients, it can be a matter of life and death. Mr.
Chairman, at some point we are all consumers of health care, so
we can all recognize the importance of having unbiased,
trustworthy information to inform our decisions.
AHRQ's Effective Health Care program is a model for how
this vision can be achieved. It is a transparent, participatory
approach that is driven by the needs of users and which
encourages broad engagement of stakeholders to explore and
mitigate any controversies, and to expand opportunities for the
rapid diffusion of findings of this research.
In short, the Effective Health Care program represents a
foundation on which a large investment in comparative
effectiveness can be built. Thank you very much, and I would be
pleased to answer any questions.
[The prepared statement of Dr. Clancy follows:]
Prepared Statement of Carolyn M. Clancy, M.D., Director,
Agency for Healthcare Research and Quality, Rockville, Maryland
Good morning, Mr. Chairman and Members of the Subcommittee. I am
Dr. Carolyn Clancy, the Director of the Agency for Healthcare Research
and Quality (AHRQ), an agency of the U.S. Department of Health and
Human Services (HHS). I am very pleased to testify before you on the
exciting issue of comparative effectiveness. I am thrilled about the
growing interest in, and attention to, enhancing the role of
comparative effectiveness research in our health care system. And I am
particularly pleased to be able to tell you about AHRQ's important
efforts in this area of research.
This is a very interesting time in the history of health and
medicine. Our investments in biomedical research have resulted in many
new diagnostic and therapeutic options. Clinicians and patients can
often now choose among an expanded array of choices for treating
hypertension, heart failure, HIV, mental illness, and other chronic
illnesses, and unprecedented innovations in diagnosis and prediction
bring us closer to a vision of personalized health care than ever.
We also are beginning to reap the benefits from the advances in
health information technology (health IT) that can bring this
information immediately to clinicians, patients, and others when and
where they need it. Health IT also is enhancing our research capacity
and our ability to diffuse breakthroughs quickly and efficiently
throughout the health care system. Health IT can make research a
natural by-product of delivering health care.
While this brave new world of health care presents wonderful
opportunities, it also creates challenges. Chief among them is how to
evaluate these innovations and determine which represent added value,
which offer minimal enhancements to current choices, which fail to
reach their potential, and which work for some patients and not for
others. The need to develop better evidence about the benefits and
risks of alternative choices is imperative.
The mission of AHRQ is to improve the quality, safety, efficiency,
and effectiveness of health care for all Americans. Effectiveness sits
squarely in our mission--what is the right treatment for the right
patient at the right time.
Comparative effectiveness research is a means to an end. Our
mission is fulfilled when health care decision makers including
patients, clinicians, purchasers, and policymakers--use up-to-date,
evidence-based information about their treatment options to make
informed health care decisions.This goal was the inspiration for the
creation of AHRQ by those Members of Congress who grasped the power of
information to improve the health care system and the health of
Americans.
Effective Health Care Program
AHRQ was granted authority under Section 1013 of the Medicare
Prescription Drug, Improvement, and Modernization Act of 2003 (MMA) to
conduct and support evidence syntheses and research on topics of
highest priority to Medicare, Medicaid, and the State Children's Health
Insurance Program (SCHIP).
I would like to thank Congress for its recognition of the programs
and infrastructure that AHRQ has established for providing the health
care system with the scientific evidence that it needs to provide safe,
high quality effective health care. AHRQ's Evidence-based Practice
Centers (EPCs), Centers for Education and Research on Therapeutics, and
other research programs have become a trusted, unbiased national source
of information on health care diagnostics and treatments
These programs are an integral part of the AHRQ's Effective Health
Care program, which was created under the authority of Section 1013.
AHRQ was able to establish the Effective Health Care program and begin
work very quickly because of our solid, existing research enterprise.
The Secretary of HHS, Michael Leavitt, has established priorities
for research conducted in the Effective Health Care Program by
establishing high priority conditions that have a major impact for the
Medicare, Medicaid, and SCHIP programs. To be effective, comparative
effectiveness research must be relevant to its users. Decision makers
are often faced with situations for which multiple different treatments
are relevant at different times. For example, should a fifty-five-year-
old woman with a scan showing greatly decreased bone density take
drugs, increase Vitamin D and calcium intake, focus on weight-bearing
exercise, or watchfully wait? We know that drugs are effective, but
there is limited information on their long-term effects. Some women
will develop kidney stones after increased calcium; current evidence
does not allow precise formulation of an effective exercise
prescription; and many women will never experience a fracture.
The key to success for this research is that it provides evidence
that informs the choices confronting clinicians and patients and, where
possible, should closely align with the sequence of decisions they
face. As MMA Section 1013 directs, we also need to ensure that findings
are frequently revisited, so they remain relevant and up-to-date. New
evidence, such as a genetic test that identifies people at increased
risk of untoward outcomes, affects comparative effectiveness and should
be incorporated into these reviews at the appropriate times.
Under the statute, the Secretary of HHS is required to establish
priorities, informed by a transparent priority-setting process that
includes all stakeholders. Priorities for the Effective Health Care
Program therefore are set after receiving broad public input through
Federal Register notices, public listening sessions, and other means.
There was much discussion within HHS about how to approach these
priorities. During our discussion of research on diseases or
conditions, for example, we debated specific questions about treating
diabetes and heart disease, and whether our research should center on
particular medications and interventions, such as stents or proton pump
inhibitors.
We decided to take a disease- and condition-based approach because,
at the end of the day, that is how health care decisions are made. A
patient comes to the health care system with a condition or disease,
and all decisions, including how best to treat it, follow.
In December 2004, based on input from stakeholders, the Secretary
of HHS identified 10 priority conditions--all of special significance
to the Medicare program--to be the first addressed by the Effective
Health Care Program. These conditions are:
Arthritis and nontraumatic joint disorders
Cancer
Chronic obstructive pulmonary disease/asthma
Dementia, including Alzheimer's disease
Depression and other mood disorders
Diabetes mellitus
Ischemic heart disease
Peptic ulcer/dyspepsia
Pneumonia
Stroke, including control of hypertension
AHRQ's Effective Health Care Program comprises three parts. The
first capitalizes on effectiveness research conducted by AHRQ's
existing 13 EPCs, which were created in 1997. The EPCs develop
comparative effectiveness reviews which focus on treatments for the
priority conditions. These reports synthesize currently available
scientific evidence, including both published and unpublished studies,
comparing treatments, including drugs, to determine relative benefits
and risks, and wherever possible, measure these outcomes for
subpopulation groups. In addition, the EPCs identify major gaps in the
existing knowledge base.
To help fill these gaps, AHRQ established the second part of the
Effective Health Care program, called the DEcIDE network (Developing
Evidence to Inform Decisions about Effectiveness), which will focus on
conducting rapid-cycle research to address specific issues that do not
necessitate larger, more time-consuming randomized clinical trials. The
DEcIDE network consists of 13 research centers that have access to
databases that contain clinical information for more than 50 million
patients but do not identify them individually.
The third part of the Effective Health Care program is the John M.
Eisenberg Clinical Decisions and Communications Science Center, based
at the Oregon Health and Science University's Department of Medicine.
The Eisenberg Center was established to ensure that the findings of our
comparative effectiveness research are translated into formats that are
understandable for all potential users. The center--named the John M.
Eisenberg Center in honor of AHRQ's late director--assists in ensuring
that effectiveness research leads to real-world quality improvements by
translating complex scientific findings into understandable language
for different audiences. The Center will help assure that reports are
presented in formats that make them useful to a wide range of audiences
and also will develop tools that encourage and empower consumers to
make informed health care decisions.
An important hallmark of the Effective Health Care program is
transparency in all aspects of the process. The transparency begins
with the open process for setting research priorities, described
earlier. The public and all interested stakeholders also have the
opportunity to comment on the framing of specific research questions,
as well as commenting on draft reports. In addition to the open
invitation to comment, manufacturers are notified when a study is begun
on one of their products and are invited to submit relevant studies and
data.
Draft research questions and reports are posted on AHRQ's Effective
Health Care Program Web site (www.effectivehealthcare.ahrq.gov), and
the Web site has a listserv that automatically notifies interested
parties when draft questions or draft reports are posted.
Comparative Effectiveness Reviews
To date, AHRQ has released seven comparative effectiveness reviews.
These reviews can be found on the Effective Health Care Program Web
site, discussed above. They are:
Gastroesophageal Reflux Disease (GERD)
For management of gastroesophageal reflux disease,
medications called proton pump inhibitors can be as effective
as surgery in relieving the symptoms and improving quality of
life.
Breast Cancer Diagnosis
Among women who receive an abnormal mammography findings or
physical exams, four common noninvasive tests (magnetic
resonance imaging, ultrasonography, positron emission
tomography scanning, and scintimammography) are not accurate
enough to routinely replace biopsies.
Managing Anemia In Cancer Patients
Among cancer patients undergoing chemotherapy or radiation,
there is no clinically significant difference between epoetin
and darbepoetin in the management of anemia. The drugs show no
clinically significant difference in improving hemoglobin
concentration and reducing the need for transfusion.
Osteoarthritis Drugs
Non-steroidal anti-inflammatory drugs (NSAIDs) and COX-2
inhibitors present similar increased risks of heart attacks
while offering about the same level of pain relief for patients
with osteoarthritis. The exception is naproxen, which presents
a lower risk of heart attack for some patients than other
NSAIDs or COX-2 inhibitors.
Renal Artery Stenosis
Increasing numbers of patients with narrowed kidney arteries
are undergoing vessel-widening angioplasty and placement of a
tubular stent, but evidence does not show a clear advantage of
that treatment over prescription drug therapy.
Off-Label Use Of Atypical Antipsychotics
Some newer antipsychotic medications approved to treat
schizophrenia and bipolar disorder are being prescribed for
depression, dementia, and other psychiatric disorders without
strong evidence that such off-label uses are effective.
Research is urgently needed for new treatments of dementia
patients with severe agitation.
Second-Generation Antidepressants
Today's most commonly prescribed antidepressants are
similarly effective to first-generation antidepressants and
provide relief to about six in 10 patients, but current
evidence is insufficient for clinicians to predict which
medications will work best for individual patients. Six in 10
patients experience at least one side effect, ranging from
nausea to sexual dysfunction.
In January 2007, AHRQ released the first summary guide for
consumers and clinicians derived from a comparative effectiveness
report by the Eisenberg Center. The consumer report, titled Choosing
Pain Medicine For Osteoarthriti s, translates the information from the
comparative effectiveness report on osteoarthritis drugs into language
that will help consumers choose among their treatment options. The
companion guide, Choosing Non-Opioid Analgesics for Osteoarthritis,
further synthesizes the evidence into a resource that can help
clinicians work with their patients to make informed decisions about
treatments for osteoarthritis.
AHRQ has a series of upcoming reports that deal with critically
important issues facing the health care system. They include:
Medications for type 2 diabetes
ACEIs (Angiotensin-converting enzyme Inhibitors) vs.
ARBs (angiotensin II receptor antagonists) for high blood
pressure
Surgery vs. stents coronary artery disease
Medications and other treatments (e.g., diet,
exercise) for low bone density
Health Information Technology
I would like to mention briefly the role of health IT, which will
make it easier for researchers to gather information for their research
and for users of research findings to get information in real time when
they need it. The health care system's growing investments in health IT
provide us with an unprecedented opportunity for redefining the
possibilities of observational studies, accelerating and targeting the
uptake of relevant information, and providing feedback to the
biomedical enterprise itself.
Health IT will make it possible for research to answer the pressing
questions facing the health care system more quickly and efficiently.
In the future, health IT will provide us with the vehicle for
transforming our health services research enterprise so that we can
evaluate the effectiveness of interventions and treatments in real time
as a byproduct of providing care.
AHRQ's Fiscal Year 2008 budget request includes $15 million for a
personalized health care initiative that will begin the infrastructure
for a federated system of databases that can help answer critical
comparative effectiveness questions. This system would enable
researchers to match treatments and outcomes, and in that way learn
from the nation's day-to-day medical practice and improve safety and
effectiveness of medical treatments.
Health IT also will greatly improve the ability to diffuse evidence
and information more quickly throughout the health care system. For
example, clinical decision support tools will make it possible to
deliver relevant information to clinicians and patients, at the point
of decision making. Most commonly envisioned as a pop-up reminder on a
screen, clinical decision support should include information
communicated directly to patients and caregivers at home--by phone,
computer, or by other means.
Conclusion
As AHRQ has implemented the Effective Health Care program, we have
some significant observations:
Priority setting: It is important to set clear priorities
that meet the needs of all of the stakeholders in the health
care system. Therefore, end users and stakeholders must
continuously provide input through an open and transparent
process.
Framing the research questions: Research must track closely
with how clinicians and patients make health care decisions
every day. The Secretary's decision to use a disease- and
condition-based approach to priorities embodies this
perspective. It is also very important to recognize the
importance of revising findings frequently to incorporate new
evidence that may change the conclusions of what works best and
for whom.
Balancing benefits and harms: Comparative effectiveness
research must provide information on benefits and harms of a
particular medication or intervention. Evaluating the balance
of harms and benefits is a critical component of informed
decision making. Few interventions are risk free, and for many
chronic conditions the therapeutic goal is management of
symptoms and disease state rather than cure. Often times, the
decision comes with some assumption of harm--by both patient
and clinician--but with the understanding that the benefits are
worth that risk.
Research is a means, not an end: The ultimate goal of our
research efforts is the development of timely, relevant
information for decision making. This requires us to go beyond
the products of traditional research, namely scholarly
articles, and translate findings into language and formats that
are appropriate for different audiences. Creating evidence and
information that is not useful and accessible, or that does not
take advantage of the latest communication technologies and
vehicles, is a missed opportunity.
Trust as a process, not a structure: As has been stated,
comparative effectiveness research can be a risky business, and
there are winners and losers. Therefore, it is important that
there be a level playing field among stakeholders. AHRQ's
Effective Health Care program has adopted a policy of
transparency and inclusion. Manufacturers are notified when a
study is begun, are invited to submit relevant studies and
data, and have the opportunity--along with any other interested
party--to comment on the framing of the specific research
questions as well as draft reports. In addition, it is clear
that the program's success is dependent on effective
collaboration with scientists from industry as well as
academia. At the same time, we ensure that the authors of the
comparative effectiveness reports are free of conflict to make
sure that the results are not perceived as being biased in any
way.
The question of trust also extends to the integral role that
patients play in research. Although government and the private sector
pay for research, patients assume the risks and benefits of enrolling
in clinical trials and other studies. A question that is the subject of
debate is whether study findings can ethically be kept secret from
other researchers and patients themselves. We all need to learn from
the knowledge gained in research, but it can be a matter of life and
death for patients. We must move to an atmosphere where it is
unacceptable to hold back research findings that may have an impact on
the care that patients receive.
In conclusion, the U.S. health care system is poised to take
advantage of advances in science and health information and
communications technology in ways that have previously only seemed like
something out of science fiction.
The need for valid, reliable, and accessible information on the
comparative benefits and potential harms of treatment options has
gained an urgency due to recent policies to promote the adoption of
interoperable health IT, continued expansion of diagnostic and
treatment options, increased consumer interest in health and health
care decisions, and broad interest in improving value.
AHRQ's Effective Health Care program is a model for how this vision
can be achieved: A transparent, participatory approach that is driven
by the needs of users and encourages broad engagement of stakeholders
to mitigate any expected controversies and to expand opportunities for
diffusion of findings of comparative effectiveness research. The
Effective Health Care Program represents a foundation in which a larger
investment in comparative effectiveness can be built.
Thank you very much and I would be pleased to answer any questions.
Chairman STARK. Thank you, Doctor.
Dr. Orszag?
STATEMENT OF PETER R. ORSZAG, Ph.D., DIRECTOR, CONGRESSIONAL
BUDGET OFFICE
Mr. ORSZAG. Thank you, Mr. Chairman and Members of the
Committee. My oral testimony this morning will make three basic
points.
First, the central fiscal challenge facing the United
States is the growth of health care costs, not aging, despite
what many media portrayals would suggest. This chart
illustrates the point. Over the past four decades, costs per
beneficiary in Medicare and Medicaid have grown 2.5 percentage
points faster than income per capita. If that rate of growth
continued, you wind up on the top line. Medicare and Medicaid
would grow from 4\1/2\ percent of the economy to 20 percent of
the economy by 2050.
The bottom dotted line shows you what happens if that
excess cost differential were zero, and isolates the effect of
aging on the programs. I think you can see that where you wind
up on that bottom line is higher than where you start. So,
there is some effect of aging, but that difference is much
smaller than the difference between the bottom dotted line and
the top line in 2050.
In other words, the central long-term fiscal challenge
facing the United States is how rapidly health care costs grow
compared to income per capita, not the aging of the population
or the coming retirement of the baby boomers.
These rising health care costs, by the way, also represent
a challenge not only for the Federal Government, but for
private payors. Indeed, costs per beneficiary in the public
programs have tracked costs per beneficiary in the rest of the
health sector over long periods of time.
My second point is that a substantial opportunity exists to
constrain health care costs, both in the public programs and in
the rest of the health system, without adverse health
consequences. Perhaps the most compelling evidence in favor of
this observation is the substantial geographic variation in
costs per beneficiary across parts of the United States that
cannot be explained by the underlying riskiness of the
patientand I present the chart here, with the darker areas
being higher spending regionsand which do not translate into
higher life expectancy or measured improvements in other health
statistics in the higher spending regions.
Furthermore, hard evidence is often unavailable about which
treatments work best for which patients or whether the added
benefits of more expensive but more effective care are worth
the cost. The variation in treatments across the United States
is often greatest for those types of care for which evidence
about relative effectiveness is lacking.
Various Federal efforts to conduct this kind of comparative
effectiveness have been undertaken in the past and some
continue today, but on a scale that is significantly smaller
than most observers believe is warranted.
If policy-makers want to expand Federal efforts to study
comparative effectiveness, the effort could be organized and
funded in various different ways. My written testimony
describes some of these options, and a forthcoming CBO report
that has been requested by the Senate Finance and Senate Budget
Committees will go into even more detailed analysis of them.
However the effort is organized, having more health records
available in electronic form would facilitate the use of
existing data for research, which could create new
opportunities for examining what works and what doesn't in a
rigorous way.
Finally, comparative effectiveness research holds the
potential to reduce health care spending significantly over the
long term without having adverse effects on health. To effect
medical treatment and reduce health spending in this way, the
results of comparative effectiveness research would have to be
used in ways that changed the behavior of doctors, other
professionals, and patients.
For example, the higher value care identified by
comparative effectiveness research could be promoted in the
health system through financial incentives, the payments that
doctors receive, or the cost-sharing that patients face. Making
substantial changes in payment policies or coverage under the
Medicare program to reflect information about comparative
effectiveness would almost certainly require legislation.
I must also note that getting to the point where additional
research on comparative effectiveness could have a noticeable
impact on health care spending would take time. In addition to
the time required to get the new activities underway and up to
scale, a lag would exist before the results were generated,
particularly if they depended on new clinical trials.
As a result of these lags and other implementation lags, it
is relatively unlikely that there would be any significant net
reduction in costs over the next decade or so. But despite all
these caveats, it is so rare as CBO director that I get to have
the ability to say that anything holds the potential to reduce
costs over the long term that I want to repeat that final
observation and repeat my basic conclusions, which are:
The United States is on an unsustainable fiscal course
largely because of projected health care costs. There is a
substantial amount of variation in those costs that cannot be
explained by the underlying riskiness of the patients and that
does not translate into better health outcomes for the
population. Additional research on comparative effectiveness,
if combined with incentives to implement the results of that
research, hold substantial potential to reduce health care
costs over the long term without impairing, and perhaps even
improving, the health of Americans.
Thank you very much, Mr. Chairman.
[The prepared statement of Mr. Orszag follows:]
[GRAPHIC] [TIFF OMITTED] T5994A.000
[GRAPHIC] [TIFF OMITTED] T5994A.001
[GRAPHIC] [TIFF OMITTED] T5994A.002
[GRAPHIC] [TIFF OMITTED] T5994A.003
[GRAPHIC] [TIFF OMITTED] T5994A.004
[GRAPHIC] [TIFF OMITTED] T5994A.005
[GRAPHIC] [TIFF OMITTED] T5994A.006
[GRAPHIC] [TIFF OMITTED] T5994A.007
[GRAPHIC] [TIFF OMITTED] T5994A.008
[GRAPHIC] [TIFF OMITTED] T5994A.009
[GRAPHIC] [TIFF OMITTED] T5994A.010
[GRAPHIC] [TIFF OMITTED] T5994A.011
[GRAPHIC] [TIFF OMITTED] T5994A.012
[GRAPHIC] [TIFF OMITTED] T5994A.013
[GRAPHIC] [TIFF OMITTED] T5994A.014
[GRAPHIC] [TIFF OMITTED] T5994A.015
[GRAPHIC] [TIFF OMITTED] T5994A.016
[GRAPHIC] [TIFF OMITTED] T5994A.017
[GRAPHIC] [TIFF OMITTED] T5994A.018
[GRAPHIC] [TIFF OMITTED] T5994A.019
Chairman STARK. Thank you very much.
Dr. Miller?
STATEMENT OF MARK MILLER, Ph.D., EXECUTIVE DIRECTOR, MEDICARE
PAYMENT ADVISORY COMMISSION
Dr. MILLER. Chairman Stark, Ranking Member Camp, and
distinguished Members of the Subcommittee, much of MedPAC's
work is devoted to improving the efficiency of the Medicare
Program either through reducing unnecessary costs or improving
the quality of care for the dollars that we do spend.
The Commission is acutely aware of the long-run
sustainability problems facing the Medicare Program. In
addition to what Peter has said, I would add that the Part A
trust fund currently looks like it will be exhausted in 2019.
At historical rates of taxation, Medicare will consume 24
percent of personal and corporate income tax by 2030. The rate
of change in part B premiums and copayments is faster than
beneficiaries' incomes, making Medicare more and more
unaffordable.
Medicare needs a broad range of policy changes to gain
control of spending without sacrificing quality. One of the
changes is to better understand what works in health care and
what does not work. Comparative effectiveness analysis
evaluates the relative effectiveness of drugs, devices,
therapies, and procedures. The outcomes of this analysis can be
evaluated in terms of clinical outcomes, such as mortality and
morbidity; functional outcomes, like quality of life and
patient satisfaction; and economic outcomes, such as cost-
effectiveness.
The private sector is unlikely to produce this type of
information on the scale that is needed because it is costly
and what it has produced can be used by anyone, including their
competitors. Moreover, private payors fear litigation on being
the first to act on comparative effectiveness information. We
also point out that there is recent research in JAMA and other
clinical journals that indicate that the results of studies
sometimes are influenced by the source of funding.
There is a lot of positive work being done by Federal
agencies, AHRQ, NIH, CMS, and VA. Here the Commission, however,
has two concerns. No Federal agency has its mission and budget
devoted solely to the production of competitive effectiveness
information, and consequently, the information will not be
produced on a sufficient scale.
Second, none of these agencies are organized and funded in
a way that allows them to be truly independent. For the
Medicare Program, competitive effectiveness information could
be used to differentiate payment among providers to encourage
the pursuit of evidence-based medicine. It could be used to
avoid higher cost, expensive services when there is no clinical
evidence that they are better than existing treatments. Other
researchers have discussed the idea of requiring manufacturers
to enter risk-sharing relationships where payments are rebated
to a payor if a product does not perform as expected.
The Commission has examined this issue over the last 3
years, and in our forthcoming June report, we recommend that
the Congress charge an independent entity to sponsor
comparative effectiveness research and disseminate it to
patients, providers, and payors.
To be clear, this organization would not be involved in
coverage and payment policy decisions. It would generate
information only, information to be used by patients and
providers in making clinical decisions, and by payors to
determine coverage and payment.
The Commission has discussed the characteristics of this
entity. It should be independently governed and have a stable
funding source. I will return to those two points. It should
have transparent processes in terms of the agenda-setting, the
research results, and the methods used. It should seek input
from all stakeholders, establish consistent research methods,
and establish ethical standards for the conduct of this
research. As I mentioned, it should disseminate the
information.
Returning to governance, the Commission has concerns that
any Federal agency is truly independent when it comes to
financing and disseminating unpopular studies and results. The
Commission, however, is also equally concerned that a wholly
private organization would not be transparent and objective
enough to assure credibility. The Commission favors a public/
private governance structure that would resolve these concerns
and broadly represent patients, providers, and payors in the
two sectors.
On financing, and public financing specifically, the
Commission discussed a continuum of options, from the
appropriations process to mandatory funding streams. The
Commission favors a stable funding source, and on balance that
probably means a mandatory source would be more stable while
retaining congressional oversight.
Similarly, the Commission discussed a continuum of private
financing mechanisms, from voluntary donations to taxes. Again,
the Commission favors a more stable funding stream, and some
researchers have suggested, for example, proposing a small tax
on health insurance premiums to fund this effort.
In closing, the Commission was also concerned that this
endeavor not entail creating or recreating existing resources.
There is an available infrastructure in the Federal agencies,
the states, and the private sector. Based on new authority,
AHRQ has created research networks and networks to disseminate
study results. One could envision an entity with a public/
private governance structure which sets an agenda, sponsors
research that is conducted by both private organizations and
Federal agencies, and then disseminates the information to
patients, payors, and providers.
I look forward to your questions.
[The prepared statement of Mr. Miller follows:]
[GRAPHIC] [TIFF OMITTED] T5994A.020
[GRAPHIC] [TIFF OMITTED] T5994A.021
[GRAPHIC] [TIFF OMITTED] T5994A.022
[GRAPHIC] [TIFF OMITTED] T5994A.023
[GRAPHIC] [TIFF OMITTED] T5994A.024
[GRAPHIC] [TIFF OMITTED] T5994A.025
[GRAPHIC] [TIFF OMITTED] T5994A.026
[GRAPHIC] [TIFF OMITTED] T5994A.027
[GRAPHIC] [TIFF OMITTED] T5994A.028
[GRAPHIC] [TIFF OMITTED] T5994A.029
[GRAPHIC] [TIFF OMITTED] T5994A.030
[GRAPHIC] [TIFF OMITTED] T5994A.031
[GRAPHIC] [TIFF OMITTED] T5994A.032
[GRAPHIC] [TIFF OMITTED] T5994A.033
[GRAPHIC] [TIFF OMITTED] T5994A.034
[GRAPHIC] [TIFF OMITTED] T5994A.035
[GRAPHIC] [TIFF OMITTED] T5994A.036
[GRAPHIC] [TIFF OMITTED] T5994A.037
Chairman STARK. Well, I would start right there. All of you
have touched on this issue of who would be in charge or what
kind of a bureaucracy or agency would handle this work. Mark, I
direct this to you: Does there exist in our world today an
agency, whether it studies defense or space or is there an
agency that would meet the standards that you suggest?
Dr. MILLER. The Commission discussed a couple of models
that one could use. The notion of like a Federal Reserve Board,
where you have a board that is appointed and operates with some
degree of independence. There are arrangements in the Federal
agencies where you have combinations of the Federal agency and
a not-for-profit corporation, which can take Federal and
private dollars to execute agendas.
Chairman STARK. Such as?
Dr. MILLER. There is a list in our testimony. But these
things can be things like a Jet Propulsion Laboratory
associated with NASA, things I am not necessarily expert in.
But there is a list of them in our testimony.
The concern there would be in a situation like that whether
the Federal agency would be truly able to operate
independently. I think that is what drove the Commission in the
direction of considering a public and private governance
structure that would be able to set the agenda and direct the
research. There are a couple of models out there that people
could work with.
Chairman STARK. Dr. Clancy, your agency has been suggested
as a repository or operator of all this. What do you think we
should do? Do you want us to put it in AHRQ and cut you loose
from HHS, or what would you suggest?
Dr. CLANCY. Well, first let me say that the Department and
Secretary Leavitt have been very supportive of this work, and
he believes that this is quite critical to a focus on getting
better value in health care, which I think you and your
colleagues share a strong interest in as well.
We believe that doing this research well and rapidly and
bringing the kind of information to patients and clinicians
that they need today is, by definition, a team sport, and that
we have the teams and infrastructure in place to make the most
of an investment as rapidly as possible. If one starts all over
again, it is a little bit like that movie, ``We Are Marshall.''
Right? You can get there, but it is going to take some time to
build a new infrastructure and so forth.
More important, I think that we are very proud of the
relationships that we have built with a variety of
stakeholders. Even some industries that were worried about work
that we were doing came around when we were done to say thank
you. We were treated fairly. This was transparent. We had a
say. To the extent that we had information to share that could
also be shared broadly, you were there to help us, and so
forth.
So, I think that we have begun to set a track record here
and that that is an important consideration.
Chairman STARK. But it would be my understanding that the
Secretary, whoever that might be, could sequester the results
of your reports at any time. For example, if Tommy Thompson
were back in that seat and you came out with something
criticizing Swiss cheese, there is no way that report would
reach the public under any circumstances, I think, the way you
are structured. Is that not correct?
Dr. CLANCY. No. Actually, for this program, that is not
correct, in part because we have set it up to be transparent
from beginning to end, including posting the draft reports. In
fact, those draft reports are often covered by electronic
newsletters and so forth with people voicing their concerns or
issues or other particulars they want to raise. We think that
is great because
Chairman STARK. What you are suggesting is AHRQ now can
operate without any political influence on the results of its
work?
Dr. CLANCY. That has been our record with this program,
yes.
Chairman STARK. That is good to know.
Dr. Orszag, just one question. I am afraid I know the
answer. But it is often troublesome to us, good and bad, I
guess, that we can't get scored for savings where we are not
required by law to spend the money. For example, everybody in
this room would probably agree that for every dollar we spent
on early childhood preventive medicine, we would save $5 over
the next 10 years of that child's life. But the $5 we save you
won't score us as a savings because we are not required to
spend it.
Is there any thought, any hope, that weI guess you would
call it more dynamic scoring, but that would turn the whole
budget process on its head. But is there any way out of that
dilemma, where the prospective, I guess, social savings to the
country as a whole would help us?
You mentioned, and you bring it out: We spend five times as
much on medical care today as we spend on the whole automobile
industry. Think about that, and look at all the traffic jams we
have. But we are spending five times as much in our gross
domestic product on medical care as we do on automobiles, which
is something to think about when you are commuting.
But what about is there any chance we could find a
different way to score some kinds of savings?
Mr. ORSZAG. Let me say things three things. The first is
and the preventive medicine example that you raised often has
very long-term payoffs in terms of cost savings. The budget
window, which is chosen by the Congress, not by CBO, has been
five to 10 years. So, that is one inherent limitation in this
process that in many cases, cost savings over very long periods
of time are just outside the window that you normally look at.
The second thing is that CBO is reexamining many issues to
see whether there are offsetting behavioral responses and other
things that do not get into dynamic scoring, which involves
macroeconomic responses. There are a variety of questions that
we are currently reexamining as new evidence comes to light. We
would welcome evidence that would help us in that effort.
The final thing is on dynamic analysis itself, CBO and the
Joint Committee on Taxation and other official entities have in
recent years started to do dynamic analysis on some legislative
proposals. We are reexamining how we do that also so that to
the extent that on the spending side of the budget there are
things that have high economic returns and evidence in that
favor, that could be incorporated at some point into the
process.
But we are currently just reexamining the evidence to build
the evidence base, and we are not at the point where we are
able to do that yet.
Chairman STARK. Thank you.
Mr. Camp?
Mr. CAMP. Thank you, Mr. Chairman.
Dr. Miller, do I understand that one of the models that you
are suggesting for this might be similar to the testimony we
are going to be hearing in a few minutes from Dr. Wilensky
about maybe having a federally funded research and development
center that is mainly funded by the government but attached to
AHRQ? Is that one of the examples I thought I heard you
describe?
Dr. MILLER. Yes. That is one of the models that is reviewed
in our report. The various pros and cons of that are talked
through. I would just say that in making that point, the
concern would be that the Federal agency that it is attached to
would have the latitude to disseminate research, set an agenda
and disseminate research, even if it was reaching conclusions
that are unpopular.
I think that is why the Commission was moving more toward
some governance structure that was more separate than a Federal
agency. But it is one ofyes, it is one of the models.
Mr. CAMP. You mean your concern is that the Federal agency
would disseminate information that is unpopular?
Dr. MILLER. It would be unable to disseminate such
information.
Mr. CAMP. Unable to because of political interference?
Dr. MILLER. Correct.
Mr. CAMP. Dr. Orszag, you mentioned that comparative
effectiveness with the right incentives could be a real help. I
notice in your written testimony you mention that there might
be the incentive might be to pay for additional costs of less
effective treatment, for example, the concern being that and I
would ask both you and Dr. Clancy to comment on this there are
certain chronic conditions that have unique circumstances that
respond to different treatments or different drug regimens.
There may not be one medication that fits every particular
situation. Just for example, there are 15 drugs to treat
patients with epilepsy, and two patients with epilepsy could
suffer the exact same seizures but they require different
medications based on their body composition or other factors.
How can we ensure that a system that has comparative
effectiveness ensures each unique patient has access to the
medication that is most effective?
Mr. ORSZAG. Well, and as my written testimony emphasizes,
one of the goals of comparative effectiveness research should
be to identify the sub-populations for which different
treatments or interventions are more or less effective so that
it is not such a blunt conclusion.
Incentive structures could then be tied off of that more
disaggregated, more nuanced data. I agree with you that a blunt
approach could not only be counterproductive, but backfire. One
of the goals of this effort, presumably, would be to get a
finer level of disaggregation about what works and what
doesn't.
Mr. CAMP. All right. Dr. Clancy, any comment?
Dr. CLANCY. Yes. Just to make the point that the question
you are raising in my mind reinforces exactly the importance of
this kind of research. You know, today the vast majority of
patients who are lucky enough to have insurance actually do
make differential decisions based on tiering of pharmaceutical
benefits and so forth.
Oftentimes they have to do that in the absence of
information, and their clinicians actually don't have bits of
information, but it is not organized in a way that helps them
sit down with a patient and say, these are the options that
would be best for you.
So, that is actually the vision we have of how this
information would work.
Mr. CAMP. Do you have any thoughts just on Dr. Miller's
comments that if this research were connected with a Federal
agency, they would be unable to release anything unpopular
givenhow do you square that with your other comments about the
transparency?
Dr. CLANCY. Well, the point about transparency is that you
have got broad engagement of many people in health care who
care a lot about the information. So, you will be hearing from
the American College of Physicians and others in the next
panel. If they care about it, they are going to be saying,
okay, so where is it, I mean, in the event that something that
has not been in our experience were to occur. That is actually
very, very helpful.
Not only that, they become a very helpful and supportive
dissemination partner because at the end of the day, I am an
internist. Many internists would actually prefer to get
information that is coming from the College of Physicians than
from any government agency. I don't take that personally; it is
just because the College brings a certain level of credibility
to it.
So, we see that engagement throughout the process as quite
critical to the success of this work.
Mr. CAMP. All right. Thank you. Thank you, Mr. Chairman.
Chairman STARK. Thank you. Mr. Doggett?
Mr. DOGGETT. Thank you for your testimony.
Dr. Clancy, if I might inquire if you about a specific that
may have some implications for other kinds of services, and
that is imaging, for which Medicare has seen such an increase
in cost.
While comparative effectiveness information is certainly
useful in looking at imaging, I hear concerns voiced by
radiologists that this greatly increases the administrative
burden and that it encourages insurance companies to require
precertification and other types of utilization management
restrictions to limit imaging services.
How do we ensure that the comparative effectiveness
information is used to improve quality without weighing down
the health care provider with greater administrative burdens?
Dr. CLANCY. First, let me say that I am very pleased that
we have had a number of collaborative opportunities and ongoing
relationships with the College of Radiology and others. In many
ways, that college kind of gets the work we do more than some
others, which is a good thing.
I don't think you can entirely guard against it. But I do
think if the information is transparent both in terms of what
are the facts and what do we not know, then people have good
grounds to ask questions and to push back if there are policies
that are actually getting in the way of what is good patient
care.
Mr. DOGGETT. I know that you have done one report on the
effectiveness of noninvasive diagnostic tests for breast
abnormalities. Do you have plans to investigate the comparative
effectiveness for any other conditions where imaging is used as
a diagnostic tool?
Dr. CLANCY. I don't know, but I will be happy to provide
that answer in writing. I don't have a good list with me.
Mr. DOGGETT. Sure. Should comparative effectiveness data
also be used to develop imaging certification standards that
would assure that the provider is properly trained to provide
the imaging scan, has suitable equipment, and has the training
to read the scan effectively?
Dr. CLANCY. We think that is very important in terms of
getting at dimensions of improving quality of care. But I would
say that most people would say that is not a part of
comparative effectiveness per se.
Mr. DOGGETT. Thank you very much.
Dr. Orszag, you talked about the regional differences that
exist. How do you take the comparative effectiveness
information and disseminate it in a way to reduce those
regional variations?
Mr. ORSZAG. Well, the first step is to develop the
information because a lot of that variation is arising in
situations where there is no evidence on what works and what
doesn't. Therefore, doctor norms in different parts of the
country take hold which are not based on scientific evidence
and therefore don't translate into improvements in life
expectancy or other metrics of health quality even though they
cost more. So, the first thing is the provision of the
information.
As I think most of the written testimony emphasized,
whatever entity is designed to do this or expanded to do this
kind of research would have to pay a lot of attention to the
dissemination of the information to health professionals.
Having a more developed HIT backbone, health information
technology backbone, could facilitate that.
Then finally, to the extent that the information is
ultimately incorporated into financial incentives for providers
or for patients, that is a very direct way of signaling
information.
Mr. DOGGETT. I was interested also in the graph that you
had because it is so dramatic. Do you have it broken down into
a percentage as to how muchif you assume that health care costs
to Medicare continue rising at the current rate, how much is
attributed to just an increase in the population of aged
beneficiaries and how much of it is related to rising cost?
Mr. ORSZAG. Yes. In fact, on that first chart, the bottom
dotted line is precisely what you identified, the demographic
effect. So, there are more beneficiaries, and they are getting
older, and that drives up cost. But that cost increase is
pretty modest, and if I added Social Security to the curve, it
would be a little bit more.
It is much, much smaller than how much is driven by the
rate at which health care costs grow compared to income per
capita. I think we have woefully under-invested in options that
could help bend that curve, which is the central long-term
fiscal challenge facing the country.
Mr. DOGGETT. Thank you all.
Chairman STARK. Mr. Ramstad?
Mr. RAMSTAD. Thank you, Mr. Chairman. I want to thank all
three of you true experts for your testimony.
Dr. Orszag, I have a question. My overriding concern here,
and it is really quite compelling, I believe, is that this new
standard or model, the comparative effectiveness analysis, call
it what you will, that it could end up denying patients
lifesaving medical technology, appropriate medical technology.
I have seen the empirical data, and they all suggest, at
least the studies I have seen, that medical technology saves
dollars in the aggregate rather than costing dollars. Some
policy-makers don't understand that, I realize. But anyway,
that is not my question, but I think it needs to be taken into
account.
My question is this: How would we ensure under this new
paradigm, if you willhow would we ensure that complex study
results, such as evaluation of a surgical procedure versus a
medical therapy, be properly conducted and analyzed? What would
be the mechanism to ensure such a quality study?
Mr. ORSZAG. Well, a few comments. First, I think the
institutional design of the entity or entities that were
charged with conducting this kind of analysis would have to
include standards for how the research would be conducted.
So, for example, that the researchers not have financial
ties to the companies that might be producing certain things;
that the statistical techniques usedand I want to pause on that
for a second because I do think if we are going to
significantly expand this kind of research, it is not likely
that we will be able to rely solely on randomized trials.
So, the expanded use of statistical analysis of health
records, basically, will be necessary. Having a dramatically
expanded system of electronic health records would facilitate
the kind of rigorous studies that could provide detailed
analysis of sub-populations in a way that we currently only
have a limited ability to do.
Mr. RAMSTAD. So, you think it would be better able to
consider highly nuanced situations across sub-populations?
Mr. ORSZAG. With an expanded electronic health record
backbone, there would be a much greater ability to study sub-
populations, yes.
Mr. RAMSTAD. Dr. Clancy, I see you shaking your head
affirmatively. Would you care to comment?
Dr. CLANCY. Yes. I would just like to reinforce that. Our
2008 budget request includes a request for $15 million, with
which we will be launching a partnership with private sector
health care organizations that have made the investments in
electronic health records so that we can actually, in effect,
work with them to create a distributed network both to do the
kind of work that Dr. Orszag just described, but also to make
sure that those organizations can use the findings as rapidly
as possible, which is, I think, a point here that I don't want
us ever to lose.
Dr. MILLER. If I could just----
Mr. RAMSTAD. Please, Dr. Miller.
Dr. MILLER [continuing]. I mean, one other part of the
structure is to be sure that the results are open to public
review and comment, much like you have in an academic process,
so that other researchers and other analysts and other parties
can comment on the work.
Mr. RAMSTAD. Of course, there would be a mechanism for the
dissemination to all concerned parties, patients, as well as
providers, practitioners.
Let me ask you a final question, if I may, Dr. Miller. One
of the things I have learned over the years is the
effectiveness of a medical device often depends on the skill of
the physician using the device. Certainly physicians have to
learn how to best use the device, determine which patients are
the appropriate candidates for treatment, and so forth.
How can we make sure that the comparative effectiveness
device is assessed in the context of physicians developing the
skills to use that particular device?
Dr. MILLER. Well, I think a lot of this research tries to
see how the given intervention works in a real world setting.
So, part of the research can actually address the skills that
are needed toif it happens to be a device or a particular
procedure. Some of that can be built into the study itself. The
idea here is real world use of the intervention.
Mr. RAMSTAD. So, we are not going to determine comparative
effectiveness before doctors develop the skills to use whatever
device?
Dr. MILLER. When you have a controlled trial, you are
actually doing the procedure or testing the drug itself. Some
of what you would need would come out of that process.
Mr. RAMSTAD. Well, thank you. My time is up. I appreciate
your responses.
Thank you, Mr. Chairman.
Chairman STARK. Mr. Pomeroy, would you like to inquire?
Mr. POMEROY. I sure would, Mr. Chairman. Thank you.
I want to especially congratulate Dr. Orszag for his
testimony. He couldn't have laid it out more clearly. We are
heading to a financial train wreck. Health spending is the
driver, the largest single driver of this fiscal train wreck
ahead, and that if you look at it, we are seeing money pour out
of the Treasury in differing ways across the country, ways that
don't seem to be getting us anything in terms of health care
return.
So, getting to the bottom of that one, you just couldn't
have laid that out more clearly for us. I wouldn't think there
would be any bipartisan disagreement across this panel. We have
got to get to the bottom of that. Data and the analysis of it,
capturing outcome data, procedural data, trying to get our
hands around it, is a way to do it.
We have been talking about this, however, since I was
insurance commissioner. I remember this coming up in the late
eighties, early 19nineties. I thought, ah-hah, this is really
going to advance the practice of medicine in this country. Man,
we have just gotten almost nowhere. I am a littlewell, anyway,
we have to get it right away.
Now, I have heard from some associations that essentially
the procedure for data collection and outcome analysis launched
by legislation we passed is not built on a collaborative basis
at all and doesn't haveit is not going to work very well. Dr.
Clancy, can you reflect on those concerns?
Dr. CLANCY. I am not sure which legislation that you are
referring to. I am pleased with the collaborations that we have
had to date, but I would be happy to follow up on specific
concerns that you have been hearing.
Mr. POMEROY. Did Committee on Ways and Means toward the
second half of last year do something about data?
Dr. CLANCY. You may be talking about physicians reporting
on quality.
Mr. POMEROY. Yes.
Dr. CLANCY. Yes.
Mr. POMEROY. That would be an essential component of this
effort, wouldn't it?
Dr. CLANCY. No. It is a little bit separate. Where
physicians might be involved
Mr. POMEROY. But equality data will illustrate what you are
paying for that is providing value versus what you are paying
for that is not providing value in terms of expanded health
outcomes. Correct?
Dr. CLANCY. What the quality data is looking at right now
are those areas, just a few, where we are pretty sure what the
right thing to do is. For example, diabetics should have a
certain type of test done, surgeons should be doing something
to minimize the occurrence of infections after surgery, and so
forth. It is a very small subset of samples.
I think where many physicians are feeling frustrated right
now, particularly those in small practices, is that it feels
like a burden and not much value added. That is a concern we
take very seriously. It is a bit peripheral to our topic of
conversation today.
At the same time that they are not really loving that
quality reporting, a number have been coming to us saying, we
want to create our own registries. Some have done that. The
Society of Thoracic Surgeons is probably the best example.
But the orthopedists, the College of Surgeons, and many
others, the bariatric surgeons, all want to collect information
so that they can get at some of the questions that your
colleague was asking Dr. Orszag about, about physician skill,
about potential harms, and how people do over time. We are very
much looking forward to working with them on that.
Mr. POMEROY. It strikes me that as clear as this is
conceptually, getting into it is quite difficult, thenwho works
it up, what is measured, how you measure it. Dr. Miller, I do
see the quality tying directly into this whole effort at trying
to evaluate what is unnecessary to pay for and trying to get at
disparate practice patterns across the country, with an eye on
cost savings.
What is MedPAC's response to what passed late last year
relative to quality reporting?
Dr. MILLER. I think again we are talking about two slightly
different issues. But just toI do think I understand where you
are going. Just to address your issue, one tack that we took
when we were talking through the collection of quality data for
physician services is we think that a lot of information can be
collected from the claim stream.
So, for example, if a physician ought to be, for a
diabetic, ordering certain types of tests or having eye exams
or foot exams, that type of thing, some of that information,
whether it occurred because they are billing for it, can be
collected through the claim stream without a significant burden
on the physician themselves.
Now, I want to be clear. This is not to say that we don't
think there may be information that should come from the
physician themselves. For example, we talked about the notion
of physician offices reporting on their functionality. Do they
have the ability to do a patient register? Do they do those
types of things? But there is a lot of information that can be
collected from the claim stream without putting a burden on the
physician.
Now, just one last point. The connection here is if we have
comparative effectiveness information through analysis,
clinical trials, that type of thing, that can tell you which
measures you might want to be collecting from physicians.
Mr. POMEROY. Thank you. Thanks, Mr. Chairman.
Chairman STARK. Mr. Becerra.
Mr. BECERRA. Thank you, Mr. Chairman. Thank you to the
three of you for your testimony.
Dr. Clancy, I may make a mistake here, and forgive me if I
do. But I believe you know my wife fairly well, Dr. Carolina
Reyes.
Dr. CLANCY. I certainly do.
Mr. BECERRA. I know that if she knew that you were here
today, she would want me to pass along a hello because I know
she always speaks so very highly of you.
Dr. CLANCY. Thank you. Likewise.
Mr. BECERRA. Thank you. Dr. Miller, MedPAC and others have
said we need to do more when it comes to figuring out how to
best compare different services, devices, and all the rest to
cut back on the costs of health care. MedPAC is probably going
to issue a report that says that we need to do more on
comparative effectiveness to get better results. Right?
Dr. MILLER. On Friday.
Mr. BECERRA. Okay. Dr. Orszag, I think you have talked
about, as Mr. Pomeroy said, this looming crisis in our
budgeting and how health care is such a big part of it. I think
most people would agree that if we do a good job with this
comparative effectiveness, we are going to save some money.
But I don't believe that you are prepared or we are
prepared to see, coming out of those who do the analysis of how
this affects dollars, you are going to come out and say, this
scores well and has a big savings for us, at least not at this
stage.
Dr. ORSZAG. What I would say is this holds substantial
potential to reduce costs over the long term if it is
implemented aggressively. Savings over the next decade is a lot
harder because you have to get this thing up and running, you
have got to do the studies, and then you have got to get it
implemented.
So, if you are looking at bending the curve over the long
term, there might be a material effect. If you are looking for
cost savings over the next 10 years, that is a much harder
thing.
Mr. BECERRA. If we had a bill that would provide a chunk of
money so that Dr. Clancy could do more research, how would you
score it?
Dr. MILLER. Over the next 10 years or over
Mr. BECERRA. Ten years is our horizon.
Dr. ORSZAG. Yes. Over the next 10 years, again, I would not
be expecting any significant cost savings over that period.
Mr. BECERRA. So, Dr. Clancy, now I come to you. I believe
you have about $15 million or so to try to do some of this
research. You were authorized to get up to 50 million, but you
got 15 through the appropriations process, far less than what
you were authorized to get, far less than you probably needed
to be authorized to get, but you have 15 million.
If we were to go legislation in this PAY-GO world where we
have to pay for everything that we propose that is new
spending, we are going to have a tough time figuring out how to
get you the dollars you need to do the research we need so we
can start saving the money that everyone acknowledges that we
can gain from this.
So, it seems like we are in this awful dilemma, this Catch-
22, where we know that there are savings. We know long term
they will be there. But for our purposes, we can't score them
as savings, and as a result you get these meager allocations of
money through the appropriations process, which never leaves
you enough time and resources to do the research that will
prove what we say we know.
So, I am wondering if you can help us out of this quicksand
and tell us, how can we persuade our colleagues that we must do
much more than just provide $15 million in research dollars so
you can do the work to prove the effectiveness of this?
Dr. CLANCY. Well, first of all, that is a great question.
Thank you very much. The MMA, Section 1013, I think only had a
limit for the first year. You are right that it took the
appropriations another year to catch up.
I do think that we have heard considerable private sector
interest in being part of a serious public/private
collaboration, and I think that is a good thing, with all the
caveats about minimizing conflicts and so forth. I think that
may be part of the answer.
Ultimately, I think the answer is going to be in the return
on investments. I think that we are beginning to make a
downpayment now. We will actually let a contract in the next
few weeks to see the extent to which we can take advantage of
all the investments that organizations have made in electronic
health records to create network where we can learn much
faster.
Beyond that, if Dr. Orszag can't help you out, I have to
say I would defer to his expertise.
Mr. BECERRA. You mentioned the private sector funding.
Obviously, the folks in the private sector have a massive
interest in this as well. My concern is, as we said before, we
need these firewalls to make sure that the influence doesn't
drive us in the wrong direction.
You believe that we could create those firewalls, that the
tendencies wouldn't be to try to direct the research in ways
that benefit those from the private sector who are providing
the resources?
Dr. CLANCY. I think with this public/private partnership,
we will have a terrific opportunity to begin to test that. We
are going to start off with issues that no single health care
system is big enough to address on its own. We are actually
going to be examining the impact of breakthrough treatments,
sometimes referred to as personalized health care, to find out
how rapidly those treatments are diffused. Do they have the
impact that is expected that we see in the laboratory? What
happens to them when they are used off-label, and so forth.
I think that might be one framework to begin. I think the
concern is this private sector interests are coming up to say,
I like this study, and I will contribute here but not over
here. I think a robust framework that addresses the issues you
raised would need to mitigate that concern.
Mr. BECERRA. Thank you. Thank you, Mr. Chairman.
Chairman STARK. Mr. Kind, would you like to inquire?
Mr. KIND. Great. Thank you, Mr. Chairman. I want to thank
our guests here today, too, for your testimony and your help
and your recommendations and guidance on this issue.
We in Wisconsin have been very fortunate and quite
delighted with the whole collaboration for health care quality
that has been assembled between our providers. It is a
volunteer basis, but they are establishing standards, reporting
requirements, transparency. It appears to be really paying
great dividends now, especially in light of a recent report
issued by the Federal Agency for Healthcare Research and
Quality ranking Wisconsin hospitals number one in this
endeavor. We appreciate that recognition.
But of course, Wisconsin too is one of the lower reimbursed
states in the nation when it comes to the Federal programs and
Medicare reimbursement rates. So, there is a great interest and
drive for outcomes-based or performance--based measures and
standards which will drive these reimbursements.
But to me, it seems the key is making sure that we have got
a totally integrated health information technology system out
there. Governor Doyle just announced a statewide project for
all of our providers. But how we get there and how soon we get
there is going to be crucial.
As far as establishing the standards, the measurements, the
effectiveness, which would then drive reimbursements and best
practices throughout the country, my question is: Can we get
there without mandating it, without the threat of no Medicare
reimbursements unless you have HIT fully in place?
But if we can do it through an incentive basis, what is the
best incentives to provide? Because this is expensive, and
right now there is very little financial incentive for some
providers to do it. A lot of providers are, but there is
proprietary interest being built up now with the systems that
they are using.
The question is, can we integrate that across the bod? What
type of incentives should we be looking at to help drive the
whole HIT movement throughout the country so we can establish
these standards and measurements and start doing some real
comparison across the board?
So, we be looking at accelerated depreciation for these
hospitals who are implementing these systems, or to the
providers of these HIT systems through the Tax Code? Should
there be grants offered to hospitals for training purposes for
implementing HIT technology? Should we be looking at grants for
lean or Six Sigma programs with our health care providers, too,
to go after the low-hanging fruit? Dr. Miller?
Dr. MILLER. Yes. The Commission looked at this issue a
couple of years ago. It didn't rule out things like the grants
and trying to put money on the table to bring people together
in a community. But I think the feeling of the Commission was
the first and strongest signal, and I think there has been some
reference to this elsewhere to start to build it into the
payment system.
For example, does a physician's office have the capability
to have patient registries? Does it have prompts to say that
for my diabetics, I need to do this next test? You wouldn't
say, I am paying you to purchase this software, but it would
say, your payments will be increased if you have this
capability, and then let the market work in behind it to say
what is the best way to get that capability.
Then you change the return on investment ratio that right
now, a physician's office will look at it and say, I have a lot
of expenditures but I am not sure what I am getting back. If
you can change that ratio, you can change the incentive. Then
we made recommendations for hospitals and managed care plans,
et cetera.
The point was that the first signal should be through the
payment system to say, if you have these capabilities or these
measures, your payments will increase from----
Mr. KIND. This would be on a temporary basis, I assume.
Otherwise we are paying more money to try to make providers
more efficient and more outcomes-oriented.
Dr. MILLER [continuing]. No. The Commission's view was that
paying for these kinds of outcomes should be something that
would be an ongoing basis so that we don't payand I think this
was a statement made early onwe don't pay the same to each
provider. We pay more to the providers who have better quality
outcomes, greater capabilities to track their patients. This
would be on an ongoing basis.
Then they did talk about some of those other things that
you talked about. But I think their feeling was, first let's
get Medicare to drive this signal pretty hard through its
payment system.
Mr. KIND. All right. Dr. Clancy?
Dr. CLANCY. Yes. First let me just say we work closely with
the Wisconsin Collaborative for Healthcare Quality, and they
are terrific. So, I just wanted to let you know that.
Mr. KIND. Good.
Dr. CLANCY. Second, we have been supporting a grants
program of close to $200 for the past several years, evaluating
the impact of selected applications of health information
technology on improving quality and safety, with a special
focus on those providers in rural and underserved areas. We
also support these health information exchange projects,
similar to what Governor Doyle would like in six states right
at the moment.
So, we have a resource center that I think can give a lot
of lessons to providers. Having said that, I think we do keep
coming up against the issue of what is the incentive to adopt
and what is going to make it worthwhile. It is an issue we are
pushing on very hard right now in the Department.
As we speak, the American health information community is
meeting today. You are likely to see a demonstration coming out
of CMS in the near future. There are a number of demonstrations
ongoing now that I think get right at this incentives issue.
The issue I am working on very specifically, and we
recently heard from the collaborative, which was great, is how
can we make sure that certified electronic health records in
the very near future include the functionality to report on
quality.
Right now, providers who have made that investment have
sometimes been disappointed after they made a big investment
only to find out that there is no way to just hit the F7 key;
for example, up go the quality measures. But that problem will
get solved over the next year or so, and it will include the
reminders. We don't want to get better at driving by figuring
out how to drive faster through the rear view mirror. We need
to do the right thing to begin with.
Mr. KIND. Great. Thank you. Thank you, Mr. Chairman.
Chairman STARK. Mr. English, would you inquire?
Mr. ENGLISH. Thank you, Mr. Chairman, and thank you for the
opportunity.
I was intrigued by one of Mr. Ramstad's remarks to Dr.
Orszag, and I guess I would like to reframe it as a question to
Dr. Clancy. Specifically, how will you update the comparative
effect of this measure to be reasonably sure that you are not
dampening the innovation in patient treatment and medical
devices that is occurring today?
If a plan, for example, limits access or bases coverage on
formulas derived from comparative effectiveness studies, how
does the plan avoid penalizing doctors who are on the cutting
edge and who are trying new surgical techniques or new
interventions to help their patient?
Dr. CLANCY. Thank you. That is a concern that many people
have. We are committed to updating our reports to reflect the
latest changes in science. We recognize in particular that for
devices, this is a particularly unique challenge because they
are constantly being updated.
This is an area where clinical trials are unlikely to be
the answer to that particular question. In fact, we think it is
very consistent with the kind of observational studies we are
funding right now through a network that we call DEcIDE, and
would be very interested in additional input on this question.
Because as these devices get better and as we learn more
and can collect information about which patients benefit and
under what circumstances, we will all be better off. Again, we
think that transparency throughout the process, from setting
the agenda to framing the questions to reviewing the reports
and so forth, is the greatest protection against information
being used in a way that is contrary to patients' interests.
Mr. ENGLISH. Doctor, I think that is a good answer. I guess
my concern would continue to be a process one, that simply how
can you as a government entity stay on top of this in realtime
and stay on top of current practice? But that is something that
I will monitor and welcome the opportunity to engage with you
on.
Dr. Miller, right now clearly there are significant gaps
between what we know based on current science and evidence and
what actually gets implemented into everyday clinical practice.
In your view, shouldn't we be doing more research to get at the
best approaches to closing some of those gaps?
Dr. MILLER. If I understand the question, the answer is
yes. That is a lot of what we were talking about today and will
be discussed in the report that is going to come out on Friday.
But maybe I didn't quite understand.
Can I say one thing about?----
Mr. ENGLISH. Go ahead.
Dr. MILLER [continuing]. You asked about the stifling of
innovation.
Mr. ENGLISH. Yes.
Dr. MILLER. I think one thing that all of us should keep in
mind is that innovation investment is occurring now, and it is
doing that on the basis of a very fragmented information basis,
and in some cases information that is not completely unbiased.
I think one of our arguments here is that if we want a
market to work well, we shouldn't be afraid of complete
information. I think your concern about being sure that we are
on top of it is well taken. But right now the information is
very fragmented and incomplete, but driving lots of investment
and lots of dollars.
Mr. ENGLISH. Well said. I will yield back the balance of my
time.
Chairman STARK. Thank you. Ms. Schwartz, would you like to
inquire?
Ms. SCHWARTZ. Thank you, Mr. Chairman. Thank you for the
opportunity.
I guess I should say that there are two particular reasons
why I am here, and I think I am looking at Dr. Clancy because
my understanding is that you do a pretty good job of what you
do. Having one of the evidence-based practice centers13 of them
having one of them in my district, and I have visited with them
and asked people in the community or providers and payors, and
tremendous respect for the information that comes out of ECRI,
which is in Plymouth Meeting, Pennsylvania. I think they do a
good job, and your shop as well.
So, I think you have already answered the question that has
been asked, which seems like a reasonable one, is why create a
new system to do this when we already have one that is really a
public/private partnership in a way already? Because there are
these eleven, in some cases private, in my case, I assume they
all are evidence-based research institutes that actually do
this work on your behalf and helps you do all the work that you
do.
Is that not true, that there is a mechanism in place now,
and as was pointed out before, if you actually just do more of
what you are doing, again with the right priorities, that you
could have more of an effect in controlling some of the cost,
or at least providing information to the providers and to the
usually hospitals and doctors to know that they are using the
most effective treatments and devices?
Dr. CLANCY. The vast majority of the investments we make in
this program are indeed to private sector entities. Some are at
academic medical centers. Some, like ECRI, are freestanding
independent institutes or like Blue Cross Blue Shield Tech
Association and so forth.
So, yes, I would say that this is very much a public/
private partnership. Thank you for your comments on ECRI. They
are also very, very interested in this issue and making
contributions.
Ms. SCHWARTZ. I know that they would be happy to do more if
they the funding to do that. I hope to be a part of this.
Do you have any evidence yet about when you do do a
comparative study and get that information out, that you have
even anecdotal information coming back, sayingfrom hospitals or
physician practices that they have changed their behavior
because of information you have put out?
Because that is the point, I think, that Dr. Orszag made,
is that we need to have some sense that it is being used in the
real world of health care. Someone is taking that action,
saying I used to use this treatment because I always did, and I
am not up on the evidence but your information actually made a
difference. Or, in fact, I was buying the cheapest thing out
there, and your information has actually helped me buy
something else because it is more effective.
Dr. CLANCY. We have information that a variety of
organizations have been very, very enthused about this
information. Much of it is anecdotal and more like case
studies. We work very closely with the College of Physicians
that you will be hearing from, from a variety of consumer
groups. Consumer Reports takes advantage of information that is
produced by another one of the evidence-based practice centers,
and so forth.
I think it would probably be more helpful if I could get
you a thoughtful written response to that question.
Ms. SCHWARTZ. I think it would be helpful to the Committee,
in particular as we move forward, to make sure that it is being
used, and it is being used in a timely fashion and, of course,
kept up to date.
My other than question may actually be for Dr. Orszag more.
That is: You suggested that because of the regional
differences, that the I assume doctors and hospitals are not
using evidence-based information as they make their decisions.
What do you think they are making their decisions on?
Mr. ORSZAG. A lot of the variation seems to occur where
there simply is no evidence. So, I will give you an example.
You fracture your hip. You go in for surgery. The variation in
surgery costs for hip fractures is very small across the United
States.
For the follow-up costs of that hip fracture surgery, there
is no evidence on whether you should go see your doctor four
times a month or twice a month. Should you get an MRI or not?
Should you do physical therapy or not? There is just no
evidence.
For the follow-up costs of the hip fracture surgery, there
is a lot of variation. I think what happens is in some parts of
the country, the doctors have been trained in a particular way
or believe that a certain thing works without any scientific
evidence behind that. That is what happens.
Ms. SCHWARTZ. So, the standard of practice is just
different, in a way?
Mr. ORSZAG. The doctor norms differ. That is why you see
this variation even within relatively small geographic areas,
where different doctor groups are practicing in different ways.
Ms. SCHWARTZ. Do you think cost comes into this, that
peopleare you suggesting that even the decision about what
device you might buy, that cost comes into that as much as
information about how effective it is? Is it cost? Is it
availability? Is it who comes to the hospital and sells it?
That is what I was wondering about, too. Is it based on just
access to the information?
Mr. ORSZAG. Our payment----
Ms. SCHWARTZ. Which then speaks to how do we get at the
good information broadly enough so that that actually is
available in helping to make these conditions?
Mr. ORSZAG [continuing]. Our payment system seems to
accommodate or facilitate these variations in doctor norms
because especially within Medicare, we basically provide, we
pay for, whatever doctors order, to a first approximation. So,
if in one area doctors practice in a certain way, they get paid
for that, and if in another area they practice in a more cost-
effective way, they get paidthe payments reflect that.
So, the payment system is playing a role, and it is
accommodating this variation in doctor norms.
Ms. SCHWARTZ. All right. Dr. Clancy, did you want to
comment on that at all?
Dr. CLANCY. I think the availability of facilities and
providers in a particular area often has a lot to do with, say,
the follow-up costs or other examples of variation. So, for
example, if there are few physical therapies in an area,
doctors are going to order less of it, as compared with a
community where there are a lot of terrific physical
therapists, and so forth.
When you look at different providers and how they are
distributed across the country, it kind of makes you scratch
your head. But that clearly, I think, does have an impact on
norms as well.
Ms. SCHWARTZ. All right. Thank you for your indulgence, Mr.
Chairman.
Chairman STARK. Thank you. Do any of the Members have any
other burning issues? Before I thank the panel for your
patience and your efforts in helping us wind our way through
this process. Thank you very much.
Our next panel will be led of by Dr. Gail Wilensky, who is
a Senior Fellow at Project Hope, formerly a director of CMS.
Dr. David Dale, who is president of the American College of
Physicians.
Ms. Gail Shearer, who is the Director of Health Policy
Analysis for Consumers Union.
Dr. Susan Hearn, who is the Senior Project Manager in
Environmental Health and Safety at the Dow Chemical Company of
Midland, Michigan.
Dr. Steve Teutsch, who is the Executive Director of
Outcomes Research and Management for the Office of Scientific
and External Affairs at Merck and Company of West Point,
Pennsylvania.
Welcome the panelists. Without objection, your testimony
will appear in the record in its entirety. We would ask you to
summarize for us or add to it in any way that you are
comfortable.
Gail, do you want to lead off? You have to turn your mike
on.
STATEMENT OF GAIL WILENSKY, Ph.D., SENIOR FELLOW, PROJECT HOPE,
BETHESDA, MARYLAND
Ms. WILENSKY. I should know that by now. Thank you, Mr.
Chairman and Members of the Subcommittee. It is a pleasure to
be here. The comments I am going to be making reflect my
training as an economist, my experience at HCFA and MedPAC, and
also my membership on a Committee established by Academy Health
to look at the issue of placement, structure, and financing of
comparative effectiveness research. But my comments here
reflect my personal views and not those of these organizations.
I have included in my testimony an article that was
published online in Health Affairs late last year that lays out
my thinking on some of the fundamental issues regarding
placement financing and functions. This has been a very topical
subject, and the comments today reflect some of the evolution
in my thinking since then.
The rationale for such a center, I think, has been stated
well. We have an unsustainable rate of spending growth, and we
have that in a world where there is clear and persistent
indications of problems with both patient safety and with
quality.
To be sure, better information will not by itself be enough
to moderate spendingyou have heard that from several people
beforeand maybe not even enough to alter practice behavior.
Changing the incentive structure that faces patients and
clinicians, using comparative clinical effectiveness
information along with cost data to set reimbursement rates,
and a whole myriad of other changes will be needed. the other
hand, without better information on what works when, for whom,
provided under what circumstances, it is hard to imagine how
the U.S. will learn how to spend smarter.
I have been advocating for a center that would have an
information function rather than a decisionmaking function as
it would pertain both to coverage decisions and reimbursement
decisions, although I primarily see the information in such a
center as informing better clinical decisionmaking and helping
in the design of smarter decisions regarding reimbursement, as
opposed to setting any additional criteria for coverage. These
are different and fundamental roles compared to some of the
centers that exist in other parts of the world.
I believe that the function for such a center should be to
fund new research, synthesize existing research, and make sure
that information is available about what is likely to result
from using different treatment options for different subgroups
in the population.
We need to be functioning on medical conditions rather than
specific interventions or therapeutics; that is, cardiovascular
disease or orthopedic surgery, and not a particular device. We
need to be sure medical procedures are being included, and not
just look at a particular therapeutic or medical device.
We need to recognize that technologies are rarely always
effective or never effective, and that the role for a center is
to inform decisionmaking about the likelihood that a favorable
outcome will occur. We need to recognize that it is likely that
the outcomes will differ for different subgroups in the
population. As was asked in the previous panel, we need to
understand that this is a dynamic process and not a once done,
finished forever process.
I believe that the characteristics of the data help
determine where the center might best lie. The data needs to be
regarded as objective, credible, transparent, protected from
the political process and also from the interest of affected
parties. It needs to be timely and understandable.
To me, having looked at the various choices, I believe the
best combination is a federally funded research and development
center like the Lawrence Livermore Labs attached to AHRQ, close
but not too close to government, enough to give it a little
protection in terms of both the view of the private sector and
the academic and other communities.
It is one that would be sponsored by an executive branch
agency, AHRQ, and make sure that it has a strong tie to AHRQ. I
think it is important that the center have both intramural,
that is, in-house research and extramural, that is, contract
research capability.
The governance also needs to make sure that it can stand
the test of credibility, objectivity, and transparency;
staggered-year appointments by the Executive Branch, maybe
subject to Senate confirmation, so that no one administration
has too much control, with specialized scientific boards.
In terms of funding, I think you can make an argument for
direct appropriation. But given the realism of the difficulties
that might present, I think you could also make an argument for
a contribution from the Medicare trust fund and also one that
has an assessment on privately covered lives because the payors
will be the major users.
Let me summarize, then. I believe such a center would be an
information center and not a decisionmaking center, providing
credible information to clinicians, patients, and payors to use
to make better decisions. I think it would have many important
purposes, including the development of a reimbursement system
in which copayments could be tiered to what makes the most
sense clinically and economically, informed by credible,
objective, transparent data.
Different payors can and should make different decisions.
If Medicare is to be able to make use of this kind of
information, it will need to have additional authority in
setting reimbursement rates according to what makes sense for
various subgroups of the population. I believe the agency would
be one of the many changes that needs to occur if we are ever
to learn how to spend smarter on Medicare. Thank you.
[The prepared statement of Ms. Wilensky follows:]
Prepared Statement of Gail Wilensky, Ph.D.,
Senior Fellow, Project Hope, Bethesda, Maryland
Mr. Chairman and Members of the Subcommittee: Thank you for
inviting me here to testify on strategies to increase information on
comparative clinical effectiveness. My name is Gail Wilensky. I am
currently a senior fellow at Project HOPE, an international health
foundation that works to make health care available to people around
the globe. I have previously directed the Medicare and Medicaid
programs as the Administrator of the Health Care Financing
Administration and also chaired the Medicare Payment Advisory
Commission. The views I am presenting here reflect my training as an
economist, my experience at HCFA and MedPac and also my membership on a
committee established in by AcademyHealth (the professional society for
health services research) that considered the placement, structure and
financing of comparative effectiveness research. My testimony today,
however, reflects my personal views and not necessarily the views of
Project HOPE, Academy Health or any other organization.
I am here today to discuss how to develop information on
comparative clinical effectiveness (CCE) through the creation of a new
Center for Comparative Clinical
Effectiveness. My testimony includes an article I wrote that was
published on-line in Health Affairs last November which lays out my
thinking on the fundamental choices regarding the placement, financing
and functions of such a center. As a result of the many conversations
that I have had about CCE with potential stakeholders, funders and
supporters or opponents, my thinking has evolved since the original
article. My current views are reflected in the following statement.
Rationale:
In a period when there is little consensus about how to reform
American health care, there seems to be a developing consensus on the
need for better information on comparative clinical effectiveness.
Driving this interest is the recognition that the current rate of
spending growth in health care (a long term average 2\1/2\% annual
growth rate in health care faster than the economy) is simply not
sustainable and that even with this spending growth, there are clear
and persistent indications of problems with patient safety and with
quality.
To be sure, better information will not by itself be enough to
moderate spending and maybe not even enough to alter practice behavior.
Changing the incentive structure that faces patients and clinicians,
using comparative clinical effectiveness information along with cost
data to set reimbursement rates and a whole myriad of other changes
will also be needed. On the other hand, without better information on
what works when, for whom, and provided under what circumstances, it is
hard to imagine how the U.S. will be able to develop strategies that
will allow the country to learn to spend ``smarter'' and without this,
it is hard to imagine how we will lower the longer-term ``excess''
spending growth rate.
Role of the Center:
The interest in comparative clinical effectiveness information is
neither new nor is it limited to the U.S. Other countries, however,
have tended to focus their analyses primarily on pharmaceuticals and
devices and their assessments tend to be an important or required
element in coverage or reimbursement decisions for their national
health systems.
I am advocating for a Center for Comparative Clinical Effectiveness
that would have a different focus and serve an information function
rather than a decision-making function--both as it may pertain to
coverage or reimbursement decisions. Further, I am assuming that the
information would primarily inform better clinical decision-making and
help in the design of smarter decisions regarding reimbursement as
opposed to setting new requirements for coverage. These are fundamental
and critical differences in roles.
The purpose of the Center on CCE is to fund new research,
synthesize existing research, disseminate and otherwise make available
what is known about the likely clinical results of using different
treatment options for different subgroups of the population. The focus
therefore is on medical conditions rather than on specific
interventions or therapeutics and needs to include medical procedures
rather than only be limited to pharmaceuticals and devices. It also
recognizes that technologies are rarely always effective or never
effective (assuming that some time of approval process is required such
as the FDA) and that the role of the center is to help inform various
decision-makers about the probability that a favorable outcome will
occur. Thus, comparative clinical effectiveness not only provides
information that is comparative across various interventions but also
recognizes that the outcomes may differ substantially for various
subgroups of the population. Because of the nature of the discovery
process and incremental changes that occur over time, it is important
to recognize that investment in CCE needs to be thought of as a dynamic
process and not once-done, finished forever.
Placement of the Center:
Over the past several months, there has already been a lot of
discussion about where such a center should be placed and what kind of
data should be included. In thinking about these issues, it is
important to think about the characteristics that the information
itself must possess if it is to serve the function envisioned for such
a center. The most important are for the data to be regarded as
objective, credible, and transparent--protected from both the political
process as well as the interests of affected parties. The information
should also be timely, span the full range of data available and be
understandable to the various parties who want to make use of the data
but the most important characteristics are those associated with
``trust''. Without that, the center won't be able to serve its
fundamental reason for existing.
Some have argued the merits of keeping the Center directly within
government, with many choosing to house it in the Agency for Health,
Research and Quality, AHRQ, the place where the Medicare Modernization
Act directed a limited amount of comparative clinical effectiveness
analysis to occur. Others have argued the merits of keeping it outside
of a direct involvement with government. While any placement will have
its advantages and disadvantages, on balance the one that is most
appealing to me is the use of a Federally Funded Research and
Development Center, FFRDC, which is attached to AHRQ. These are
entities that are primarily funded by government (minimum of 70%) and
are sponsored by an executive-branch agency, which monitors its use of
funds. There are several that have been around for many years. The
Lawrence Livermore Labs is one of the larger, better known FFRDC's.
This model best reflects the dictum of ``close . . . but not too close
to government'' and also assures a close linkage with AHRQ, the lead
agency for health services research which needs importantly to continue
in that role. I also think the Center would be most effective if it had
both intramural (in-house research) and extramural (contract research)
functions as do both AHRQ and the NIH. The in-house researchers provide
an important element of expertise and hands-on experience but my
assumption is that much of the work would be contracted out to
universities, free-standing research groups, etc.
Governance:
The governance of such a center is almost as important as its
placement. Again, the key concepts are credibility, objectivity and
transparency. This means a governing body that is reflective of all the
major stakeholders, with staggered year appointments by the executive
branch (and maybe subject to Senate confirmation) so that no one
administration has too much control. Specialized scientific advisory
boards would presumably be created for advice on particular comparative
effectiveness studies, particularly those involving new research.
Funding:
Like any new entity, a Center for Comparative Effectiveness would
require several years to reach a ``steady-state'' which I have assumed
would be several billions of dollars. Because information is clearly a
``public good'' as the economist uses the term, my preferred funding
would be by direct appropriation, as is the funding for the NIH. That,
however, may not be a realistic strategy. Another option is to combine
funding sources that include monies from direct appropriations, a
contribution from the Medicare trust fund and a small assessment on all
privately covered lives. Although all will benefit from the
availability of such information, thus the rationale for a direct
appropriation, the payers will be especially advantaged by having this
information available.
The Role of Costs:
The most controversial issue to date has been whether or not to
include cost-effectiveness or cost-benefit analysis directly in a
Center for Comparative Clinical Effectiveness. While I firmly believe
the data made available by the Center should be used by payers in doing
cost-effectiveness and cost benefit analyses and that funding to CMS
should be made explicitly for this purpose, along with the ability of
the agency to use such elements in their reimbursement decisions, I
believe it is best to keep these functions housed separately. Payers
would be wise to have their
C/E and C/B analyses subject to the same criteria of credibility and
transparency that are so critical to the acceptance of comparative
clinical effectiveness information. This will be key to their
acceptance and credibility although my expectation is that different
payers would use the information differently in designing their
reimbursement policies.
My rationale for the separation is two fold. One reason is
technical. The concepts and decisions involved with C/E and C/B
analysis are more controversial and subject to dispute: where in the
life cycle is the technology and how much does that affect costs, whose
costs are being measured--Medicare, small purchasers, large purchasers,
etc, what functions are or are not absorbed by the purchaser, i.e. is
the purchaser wholesale or retail, etc. In part because of these
technical issues but also because of the more controversial nature of
the implications of cost analyses, including the perceived threat that
could result from these analyses, I believe combining the inclusion of
cost analyses, particularly early on, will increase the political
vulnerability of a center for comparative political effectiveness and
since such information is the most elemental building block to learning
how to spend smarter, it needs to be protected.
Finally, to reiterate, the Center for Comparative Clinical
Effectiveness would be an information center, not a decision-making
center, providing credible information for clinicians, patients and
payers to use to make better decisions. Such information would have
many important purposes including the development of a reimbursement
system in which co-payments could be tiered to what makes the most
sense clinically and economically, informed by credible, objective
transparent data. Patients and clinicians that want more or want to
choose differently should be able to do so but should need to pay more
for their choices. Medicare does not currently have such authority in
setting reimbursement rates and granting the agency this authority
would be one of the many changes that would need to occur in learning
to spend smarter under Medicare.
Chairman STARK. Thank you.
Dr. Dale?
STATEMENT OF DAVID DALE, M.D., PRESIDENT,
AMERICAN COLLEGE OF PHYSICIANS
Dr. DALE. Thank you, Chairman Stark, Ranking Member Camp,
and Members of the Subcommittee for this opportunity for the
American College of Physicians to testify on comparative
clinical effectiveness. I am David Dale, president of the
College and professor of medicine at the University of
Washington. I will highlight our positions on this issue and
refer the Members to the written testimony previously
submitted.
The College strongly supports congressional efforts to
provide Medicare and all stakeholders with improved access to
information about the relative strengths and weaknesses of
various clinical products, procedures, services, based on the
best available evidence from clinical effectiveness research.
From the perspective of a practicing physician, the
increased availability of sound effectiveness data has direct
clinical usefulness. For example, I regularly advise men about
the diagnosis and treatment of prostate cancer and many other
problems in my general internal medicine practice.
When a patient, a close friend, recently asked me for
advice, I tried my best to give him a complete and unbiased
comparison of the risks and benefits of various treatment
strategies. But there is really very little comparative data
available.
Similarly, women with breast cancer are currently treated
with a wide range of therapies. We know relatively little about
their comparative effectiveness, particularly the long-term
effectiveness and the adverse effects of these therapies. But
we could know much more through gathering data from current
clinical practice if we had the mechanisms in place to do so.
The United States does not currently have a systematic
means of producing the information to compare the relative
effectiveness of drugs, durable equipment, therapies, and
procedures. This is in marked contrast to the organized
activities in a number of other countries, including Canada,
Great Britain, Germany, and Australia.
The College recommends that the Congress take efforts,
including allocation of secure and sustained funding, to
support a trusted entity that systematically develops evidence
on the relatively effectiveness of health care services.
The College believes that this trusted entity should be an
unbiased and independent organization; have transparent
procedures with strong stakeholder involvement, prioritized to
ensure the evidence produced has the greatest impact; present
its findings promptly in a way that is accessible and
comprehensible to all stakeholders.
The entity in the United States that currently best matches
this list of characteristics is the Agency for Healthcare
Research and Quality, AHRQ. The College commends the efforts of
AHRQ, and has recently urged Congress to increase its level of
funding in a joint letter signed by the American Medical
Association and over 80 other medical organizations.
The College believes that the greatest value of comparative
effectiveness data is to help answer the question of what works
best and for whom it works best, given the clinical conditions
of the patient and the patient's preferences. We believe that
the primary use of this information right now is for patient-
centered care and counseling.
Better information will enable physicians and empower
patients to engage in well-informed shared decisionmaking.
Shared decisionmaking is a key and essential element for
improving care through the patient-centered medical home, a
model of care now supported by provider groups representing
over 330,000 primary care providers.
The College is aware of suggestions concerning the
potential use of comparative effectiveness data by Medicare and
other payors to redesign their health benefits based on
reimbursement or patient cost-sharing of comparative evidence
on effectiveness. The College recognizes the potential savings
obtained through this approach, but we recommend that Congress
proceed cautiously. Experience and evidence are required to
determine the impact of such research on the quality of care
and patient satisfaction before it is integrated into the
payment process.
Finally, the College asks Congress to recognize the value
of health information technology. Better health information at
the level of the practicing physician will facilitate the
collection, reporting, and aggregation of clinical data to
support evidence-based research on a wide range of important
clinical problems.
The pathway for development of comparative efficacy data is
through implementation of interoperable health information
technology throughout our health care system. Thank you.
[The prepared statement of Dr. Dale follows:]
Prepared Statement of David Dale, M.D., President,
American College of Physicians
I am David C. Dale, MD, FACP, President of the American College of
Physicians and professor of medicine at the University of Washington.
The 123,000 internal medicine physicians and medical student members of
the American College of Physicians congratulate Chairman Stark and the
Members of the House Ways and Means Subcommittee on Health for
convening today's hearing on ``Strategies to Increase Research and
Information on Comparative Clinical Effectiveness.'' The College
strongly supports Congressional efforts to provide Medicare and all
stakeholders within the healthcare community with improved access to
information about the relative strengths and weaknesses of various
clinical products, procedures and services based on the best available
evidence of clinical effectiveness.
The Members of this Subcommittee are well aware of the significant
problems that characterize our current healthcare system:
the unsustainable growth in healthcare costs that
affect both payers and beneficiaries; \1\
---------------------------------------------------------------------------
\1\ Kaiser Family Foundation. Trends and indicators in the changing
healthcare market place. 2006. Accessed at http://www.kff.org/
insurance/7031/index.cfm on May 9, 2007.
---------------------------------------------------------------------------
the presence of significant quality gaps particularly
when compared to other industrialized nations that spend much
less on healthcare; \2\
---------------------------------------------------------------------------
\2\ Anderson G, Hussey PS. Comparing Health System Performance in
OECD Countries: Cross-National Comparisons Can Determine Whether
Additional Health Care Spending Results in Better Outcomes. Health
Affairs. May/June 2001;20(3):219-32.
---------------------------------------------------------------------------
the presence of significant variation in healthcare
costs throughout this country without any evidence that
increased costs result in improved care.\3\
---------------------------------------------------------------------------
\3\ Fisher, E., Wennberg, D., et al., The Implications of Regional
Variations in Medicare Spending: Part 2, Health Outcomes and
Satisfaction with Care, Annals of Internal Medicine 2003; 138:288-98.
As stewards of the Medicare Trust Fund and the largest payer of
healthcare services in the country, it is Congress' responsibility to
address these problems and help ensure that our taxpayer funds are
being used effectively to provide high quality care and achieve the
best possible patient outcomes. The increased production and
availability to payers, providers and beneficiaries of methodologically
sound information from a trusted source on the effectiveness of
alternative treatments would be a good step towards improving the value
obtained from healthcare dollars spent.
The Public Need for Comparative Clinical Effectiveness Research
From the perspective of the practicing physician, the increased
availability of sound comparative effectiveness data has direct
clinical usefulness. Each day in the privacy of the examination room,
patients are treated for conditions that have multiple treatment
options. Here we are talking about treating a common condition like
intermittent heartburn, to the more serious chronic conditions of high
blood pressure or diabetes, to the more immediate life and death issues
of to having to choose the best approach to treat diagnosed breast or
prostate cancer. The availability of valid, comparative effectiveness
data supplemented by the physician's clinical experience and
professional knowledge, helps ensure that an effective treatment choice
is made--one that meets the unique needs and preferences of the
patient.
The College has a long history of supporting evidenced based
practice, and since 1981 has been developing evidenced-based clinical
treatment guidelines through its Clinical Efficacy Assessment Program.
In fact, I was part of the original panel of experts of this program
and am currently Editor-in-Chief of ``ACP Medicine,'' a continually
updated, evidence-based reference of internal medicine published by the
College. My own patient care experiences, as well as the College's
experience in producing evidence-based analyses, supports the need for
an objective, evidence-based and refereed source of information from a
``trusted entity'' to compare the effectiveness of alternative
healthcare services.
The United States currently does not have a systemic means of
producing comparative information on the relative effectiveness of
drugs, durable equipment, therapies and procedures. The limited amount
of comparative effectiveness data that is produced is done piece-meal,
with little or no prioritization relative to the benefits it would
provide to individual patients and the general population, little
coordination or harmonization of clinical efficacy efforts, and uneven
methodological standards for evaluating clinical efficacy and reporting
the results to clinicians and patients. Often, evaluations are made on
a ``single therapy'' basis without comparing such therapies to
alternative treatments. The Federal Drug Administration assesses the
safety and effectiveness of drugs, and to a less extent medical
equipment, but the research it considers generally compares performance
to no treatment (placebo) conditions, rather than to alternative
products already in the market place. The National Institutes of Health
(NIH) is this country's largest sponsor of clinical trials that compare
alternative treatments, but funds for these studies represent only a
small amount of their budget. The Agency for Healthcare Research and
Quality (AHRQ) through Section 1031 of the Medicare Modernization Act
(MMA) was authorized by Congress in 2003 to conduct and support
research with a focus on outcomes, comparative clinical effectiveness,
and appropriateness of pharmaceuticals, devices, and health care
services. I will discuss more about this effort later in my testimony.
Private sector entities including pharmaceutical companies,
pharmaceutical benefit managers, health plans and large provider groups
also produce some comparative effectiveness data, but the details of
these studies are often not transparent, access to this data is limited
due to its proprietary nature, and there is evidence questioning the
objectivity of some of these findings.\4\
---------------------------------------------------------------------------
\4\ Medical Payment Advisory Committee. Presentation by Nancy Ray
on Comparative Effectiveness. April 12, 2007 Public Meeting. Access at
http://www.medpac.gov/public_meetings/transcripts/
0407_allcombined_transcript.pdf on May 9, 2007.
---------------------------------------------------------------------------
This hodge-podge of comparative effectiveness efforts is in marked
contrast to the activities conducted in a number of other countries,
including Canada, Great Britain, Germany an Australia. Perhaps most
recognized of these efforts is the National Institute for Health and
Clinical Excellence (NICE) program in Great Britain,\5\ which serves as
a model of a coordinated, prioritized comparative effectiveness program
designed to promote trust in its finding through transparency in its
proceedings and strong stakeholder involvement at all levels of the
process.
---------------------------------------------------------------------------
\5\ Pearson D, and Rawlins, M. Quality, innovation and value of
money. JAMA, Nov. 2005;294(20):2618-2622.
---------------------------------------------------------------------------
The College recommends that the Congress take efforts, including
allocation of secure and sustained funding, to develop or support a
trust entity that systematically develops evidence on the relative
effectiveness of various alternative heathcare services.
While the College currently has no formal position on the structure
of this entity (i.e. public, private or public-private), it believes
that this entity should have the following characteristics:
it should be an unbiased independent entity protected
from both governmental and private sector influence to
encourage trust in its findings.
its proceedings should be transparent.
it should involve stakeholders, including payers,
providers and beneficiaries, at all levels of the evidence
development process.
it should have a prioritization process, informed by
input from the stakeholder groups, that ensures that the
comparative effective evidence developed will have the greatest
impact in improving the quality and efficiency of care
provided.
it should support the development of all levels of
evidence including formal review and synthesis of evidence
already available in the clinical literature and the initiation
of new research in priority areas where such evidence does not
already exist.
it should have established processes that ensures
that the comparative effectiveness findings developed are
accessible in a comprehensive form to all stake holders and
reported in a manner that is useful for clinicians and
patients.
The entity that currently best matches this list of characteristics
is the AHRQ. Through its Effective Health Initiative, this agency has
established itself as a trusted source of comparative effectiveness
data. Since its recent implementation, it has produced seven
comparative effectiveness research reviews, it is in the process of
developing at least six others and has initiated at least 14 new
research projects. It has also made a substantial effort to ensure that
their findings are accessible to consumers, providers and policy makers
in a meaningful form. The College commends the efforts of the AHRQ and
has recently urged Congress to increase its level of funding in a joint
letter signed by the American Medical Association and over 80 other
medical organizations.\6\
---------------------------------------------------------------------------
\6\ Joint letter on SGR legislative options sent to key
Congressional Committee staff delivered on May 17, 2007.
---------------------------------------------------------------------------
If AHRQ is to be the ``trusted entity'' to conduct effectiveness
research, then it needs to be assured of sufficient and sustained
funding to support its activities and be protected from the normal
political influences that arise through the annual appropriations
process. If Congress chooses to create a new entity rather than
facilitate increased funding of the AHRQ to advance the development of
cost effectiveness evidence, it should use lessons learned from AHRQ in
developing this new entity and assure that the new entity is funded in
a way that will protect it from political influences that may arise
through appropriations.
Use of Comparative Effectiveness in Benefit Design Decisions
The College is also aware of suggestions concerning the potential
use of this data by Medicare \7\ (and other payers) to redesign their
healthcare benefits by basing reimbursement and/or patient cost-sharing
on the comparative evidence developed by the proposed entity. For
example, those procedures that prove generally more effective could
receive higher reimbursement and/or require a lower beneficiary co-
payment. The College, although recognizing potential savings obtainable
through this approach, recommends that Congress walk down the path of
using comparative effectiveness data in the Medicare benefit design
slowly and cautiously. It will take time for clinicians and patients to
develop trust and have confidence in the evidenced produced from any
new comparative effectiveness evidence producing entity. In addition,
procedures will need to be developed to ensure that the unique needs of
each patient can be recognized, and that clinical decisions are based
upon what is best for this patient, rather than the economic incentives
promoted by the benefit design.
---------------------------------------------------------------------------
\7\ Medical Payment Advisory Committee. Presentation by Gail
Wilensky and Marilyn Moon on Comparative Effectiveness. April 12, 2007
Public Meeting. Access at http://www.medpac.gov/public_meetings/
transcripts/0407_allcombined_transcript.pdf on May 9, 2007.
---------------------------------------------------------------------------
The appropriateness of including ``cost effectiveness'' as an
explicit element in comparative effectiveness research is complex and
controversial. Cost means different things to different people:
aggregate costs to a payers of services (Medicare), the economy
(societal costs), the individual (in the form of out of pocket
expenses, health care premiums, or individual tax payments to support
public programs), or clinicians (whose professional value system often
puts primacy of the individual patient's needs and preferences over
societal costs) are very different from each other and will result in
different value judgments. How the relative costs of a treatment and
procedure should be weighted against the evidence of clinical
effectiveness will involve value judgments that need to be made in an
open, transparent, and methodologically sound basis that takes into
account the different values that each stakeholder brings to the table.
For these reasons, the College suggests that federally-funded
comparative effectiveness research should, at least in its early
stages, focus on relative clinical efficacy rather than cost-
effectiveness. At the same time, however, we support further discussion
of how cost-effectiveness comparisons might be introduced into the
evaluation process at a later stage and used, at least in part, to
influence benefit design by Medicare and other programs.
Comparative Clinical Effectiveness Research and Shared Decision-Making
The greatest initial value of developed comparative effectiveness
data at this time is to help answer the question of what works best for
whom and the use of this information in providing effective patient-
centered treatment. Comparative effectiveness research from a trusted
entity will enable physicians and patients to engage in informed and
shared decision-making on the most desired and effective treatment
alternatives for that individual patient. Such shared decision-making
is a key element of the Patient-Centered Medical Home (PCMH). This care
model--supported by the 330,000 primary care physicians represented by
the American Academy of Family Physicians, the American Academy of
Pediatrics, the American Osteopathic Association and the American
College of Physicians and a coalition of large employers and consumer
organizations--would ensure that treatment decisions informed by
comparative effective evidence will be delivered in a coordinated,
integrated manner. The model also emphasizes the importance of actively
making treatment decisions a shared process between the patient and
their personal physician. Research using an active shared decision
making process, using available comparative effectiveness evidence,
indicates it has the potential to reduce unwarranted variations in
treatment among providers, increase patient accuracy in expected
treatment outcomes, and provide patients with greater comfort in the
treatment choice made.\8\
---------------------------------------------------------------------------
\8\ O'Connor, A. et al. Modifying unwarranted variations in health
care: Shared dicision making using patient decision aids. Health
Affairs Web Exclusive, October 7, 2004. Accessed at
http://content.healthaffairs.org/cgi/reprint/
hlthaff.var.63v1?maxtoshow=&HITS=10&hits=10&RE
SULTFORMAT=&author1=%27Connor&andorexactfulltext=and&searchid=1&FIRSTIND
EX=0&
resourcetype=HWCIT on May 7, 2007.
---------------------------------------------------------------------------
Finally, the College urges the Subcommittee to report legislation
to create Medicare payment incentives for physicians to acquire and use
health information technology (HIT) in their practices as a means of
facilitating the collection and reporting of clinical data on
effectiveness and facilitating evidence-based clinical decision support
and shared decision-making at the point of care. The availability of
clinical decision support technology at the site of care will make
evidence-based comparative research readily available to physicians and
their patients to support shared clinical decision-making between the
physician and the patient. The College specifically supports H.R. 1952,
the National Health Information Incentives Act of 2007, introduced by
Representatives Charles Gonzalez (D-TX) and Phil Gingrey, MD (R-GA) to
provide financial incentives to physicians through Medicare to adopt
and purchase HIT.
Summary and Conclusion
In summary, the College strongly supports Congressional efforts to
provide Medicare and all stakeholders within the healthcare community
with improved access to information about the relative strengths and
weaknesses of various clinical products, procedures and services.
Towards this goal, the College recommends that the Congress take
efforts, including the allocation of secure and sustained funding, to
create or support a trusted entity that systematically develops
evidence on the relative effectiveness of various alternative
healthcare services. That entity should have the following
characteristics:
it should be an unbiased independent entity protected
from both governmental and private sector influence to
encourage trust in its findings.
its proceedings should be transparent.
it should involve stakeholders, including payers,
providers and beneficiaries, at all levels of the evidence
development process.
it should have a prioritization process, informed by
input from the stakeholder groups, that ensures that the
comparative effective evidence developed will have the greatest
impact in improving the quality and efficiency of care
provided.
it should support the development of all levels of
evidence including formal review and synthesis of evidence
already available in the clinical literature and the initiation
of new research in priority areas where such evidence does not
already exist.
it should have established processes that ensures
that the comparative effectiveness findings developed are
accessible in a comprehensive form to all stake holders.
The Congress should give consideration to continuing to
support the work of the Agency for HealthCare Research and Quality as
the ``trusted entity'' for comparative effectiveness research, with
secure and sustained funding that is not subject to the political
pressures often associated with the annual appropriations process.
The College believes that the greatest value of developed
comparative effectiveness data at this time is to help clinicians and
patients answer the question of what works best for each patient and
for clinicians to partner with patients in an informed and shared
decision-making process when considering alternative treatment options,
a key element of the Patient-Centered Medical Home.
The College recognizes the potential savings obtainable
through comparative effectiveness research, but recommends that
Congress walk down the path of using comparative effectiveness data in
the Medicare benefit design deliberatively so that more experience is
gained first in the impact of such research and its credibility with
clinicians and patients. As confidence and trust in the process
increases, steps could then be taken by Congress to create a method for
incorporating such comparative effectiveness research into benefit
design issues.
Congress should recognize that inclusion of ``cost
effectiveness'' as an element of the comparative evaluation process
will introduce complex and controversial issues of how individual
patients, purchasers, clinicians, and society assign a relative value
to clinical effectiveness and cost. Such value judgments need to be
made in an open, transparent, and methodologically sound basis that
takes into account the different value systems that each stakeholder
brings to the table. For these reasons, the College suggests that
federally-funded comparative effectiveness research should, at least in
its early stages, focus on relative clinical efficacy rather than cost-
effectiveness. At the same time, however, we support further discussion
of how cost-effectiveness comparisons might be introduced into the
evaluation process at a later stage and used, at least in part, to
influence benefit design by Medicare and other programs.
The College asks Congress to recognize the value that a
more systemized approach to developing comparative effectiveness
evidence can be leveraged through:
The establishment of mechanisms to facilitate the
implementation of health information technology (HIT)
throughout the system
The implementation of the Patient-Centered Medical Home
(PCMH) care model.
Chairman STARK. Thank you.
Ms. Shearer?
STATEMENT OF GAIL SHEARER, DIRECTOR,
HEALTH POLICY ANALYSIS, CONSUMERS UNION
Ms. SHEARER. Mr. Chairman, Members of the Subcommittee,
thank you very much for the invitation to testify today on
strategies to increase research on the comparative clinical
effectiveness of medical treatments. We believe that
legislation on this issue is the single most important
investment you can make to moderate runaway health care costs
and pave the way for a health care system that better meets the
needs of all Americans.
Getting better value for our health care dollars is an
essential building block for health reform, regardless of
stripe of reform. In so-called consumer-centric models that
embrace health savings accounts, consumers need to know where
their dollars are most effective.
In a system of predominately employer-based health
coverage, employers have a strong financial incentive to learn
how to achieve the best health outcomes at the lowest cost. In
a universal health care system, whether through an individual
mandate, public/private expansions of coverage, or even a
Medicare-for-all type of model, the key to success will be
getting the maximum value from each premium or tax dollar
spent.
This is why we believe so strongly in the need for a
Marshall Plan-like commitment, to transform our knowledge base
about the comparative effectiveness of medical treatments to
fill the gaps in clinical research. Armed with this knowledge,
providers and consumers could identify and choose the best
treatment options, and payors could fine-tune benefit packages
and modify cost-sharing amounts to encourage the most cost-
effective care.
Today I want to concentrate on one public education program
that we have developed at Consumers Union that uses the best
available scientific evidence to help consumers, with their
health care professionals, choose the most effective, safest,
and affordable drugs. I highlight this work because it provides
a tangible example of the potential that expanded government-
funded comparative effectiveness research holds for improving
health care quality and lowering health care costs.
Consumer Reports' best buy drugs is based on systematic
reviews, unbiased medical reviews of the clinical research on
drug comparative effectiveness, safety, and side effects
conducted by the Drug Effectiveness Review Project. DERP is a
preeminent example of the pioneering lead that the States have
taken with financial support from AHRQ's funding of evidence-
based practice centers in the world of evidence-based medicine,
specifically, the research that is necessary to enable states
and other health care payors to shape benefit policy based on
unbiased scientific evidence.
Our best buy drugs program translates DERP's complex
medical reports into consumer-friendly reports that provide
information the public needs to understand the comparative
effectiveness, safety, side effects, and costs of drug options.
One of the reports is attached to my testimony today. It is
on proton pump inhibitors, which are anti-heartburn medicines.
We found that there is not a large difference between the
effectiveness of many drugs in this category. The big story,
though, is that there is a tenfold difference between the
monthly price of a heavily advertised purple pill, Nexium, and
the over-the-counter alternative, Prilosec OTC, that the best
science shows it is equally effective and safe for almost all
of us.
A month's supply of Nexium costs about $193, which a
month's supply of Prilosec OTC costs about $19 to $26. Having
said this, I want to stress that we always urge consumers to
consult with their doctors, and we urge all health plans to
have effective and easy-to-use exceptions policies for the
small amount of people who may not respond as well to one of
the best buy drugs.
In most categories that we have studied, and we have
studied 17 now, we have found that by switching from a high-
priced, typically highly advertised brand drug to a best buy
drug alternative, consumers can typically save between $1,000
and $2,000 a year. Helping consumers and health plans
substitute effective, safe, lower-cost medicines for the newest
heavily advertised alternative can save the nation billions of
dollars without sacrificing quality.
In fact, better information about comparative effectiveness
can help us as a nation improve health care quality by
educating consumers, physicians, pharmacists, and policy-makers
about the comparative effectiveness of alternative treatments.
Educating the public about low-cost, effective alternatives
can help make health care treatments and prescription drugs
more affordable. This in turn will increase the number of
patients who can get the treatment or drug that they need, and
ultimately improve health outcomes.
Consumers Union believes that it is appropriate for the
government to fully fund and sponsor this important research,
which should be available to all and constitutes the epitome of
a public good. The benefits will be shared by every one of us
who uses health care in this country.
Mr. Chairman, Members of the Subcommittee, the rapidly
rising cost of health care threatens the budgets of Federal and
state governments, and ultimately of health care consumers. We
believe that establishing a new program with sufficient
reliable funding to assess the comparative effectiveness of
alternative treatments is a necessary part of the solution to
this growing problem.
Enacting this legislation is probably the most important
thing that you can do in this Congress to save lives and money,
and to build a foundation for further reforms of our health
care system. Thank you.
[The prepared statement of Ms. Shearer follows:]
Prepared Statement of Gail Shearer, Director,
Health Policy Analysis, Consumers Union
Mr. Chairman, Members of the Committee:
Thank you for the invitation to testify on strategies to increase
information on comparative clinical effectiveness and H.R. 2184, The
Enhanced Health Care Value for All Act. We believe that this
legislation is the single most important investment you can make to
moderate run-away health care costs and pave the way for a health care
system that better meets the needs of all Americans.
Consumers Union \1\ is the independent, non-profit publisher of
Consumer Reports, with circulation of about 7 million (Consumer Reports
plus ConsumerReports.org subscribers). We regularly poll our readership
and the public about key consumer issues, and the high cost of health
care consistently ranks among their top concerns.
---------------------------------------------------------------------------
\1\ Consumers Union is a nonprofit membership organization
chartered in 1936 under the laws of the state of New York to provide
consumers with information, education and counsel about goods,
services, health and personal finance, and to initiate and cooperate
with individual and group efforts to maintain and enhance the quality
of life for consumers. Consumers Union's income is solely derived from
the sale of Consumer Reports, its other publications and from
noncommercial contributions, grants and fees. In addition to reports on
Consumers Union's own product testing, Consumer Reports and
consumerreports.org with more than 7 million paid circulation,
regularly carries articles on health, product safety, marketplace
economics and legislative, judicial and regulatory actions which affect
consumer welfare. Consumers Union's publications carry no advertising
and receive no commercial support.
---------------------------------------------------------------------------
I would like to commend the Committee for holding this hearing.
Health care costs currently consume about 16 percent of our gross
domestic product. This percent is projected to continue to grow at a
rate substantially higher than general inflation. It is imperative that
as a nation we learn how to get better value from every health care
dollar that we spend. We must not continue to pay $5 for a pill when
there is an equally effective and safe pill that is available for 50
cents.
Getting better value for our health care dollars is an essential
building block for health reform, regardless of the ``stripe'' of
reform. In so-called ``consumer centric'' models that embrace health
savings accounts, consumers need to know where there dollars is most
effective. In a system of predominantly employer-based health coverage,
employers have a strong financial incentive to learn how to achieve the
best health outcomes at the lowest cost. In a universal health care
system (whether through individual mandate, public/private expansions
of coverage, or even a Medicare-for-all model), the key to success will
be in getting the maximum value from each premium or tax dollar spent.
This is why we believe so strongly in the need for a Marshall Plan-
like commitment to transform our knowledge base about the comparative
effectiveness of medical treatments to fill the gaps in the clinical
research. Armed with this knowledge, providers and consumers could
identify and choose the best treatment options, and payers could fine-
tune benefit packages and modify cost-sharing amounts to encourage the
most cost-effective care. A commitment to funding and increased
reliance on evidence-based, unbiased clinical research and synthesis of
existing research should be a leading-edge building block for health
care reform. What does this mean for coverage? Some possible examples:
There might be zero co-payments for diabetic care and
other medical treatments that are clearly proven and necessary
to improve health outcomes. (Some insurers are already
experimenting with this approach to provide early care that
greatly improves health outcomes.)
There might be zero co-payments for certain generic
drugs, and much steeper co-payments for the equivalent, higher-
priced brand name drug where appropriate.
Our health care work on behalf of consumers is varied. Our
advocates work to improve quality (e.g., our Stop Hospital Infections
campaign to require public reporting of hospital acquired infection
rates), to improve safety (e.g., our Prescription for Change campaign
to reform drug safety laws), and improve affordability (e.g., our work
since 1936 in support of affordable health care for all). Consumer
Reports, Consumer Reports on Health, Consumer Reports MedicalGuide.org,
and consumerreports.org provide comprehensive information about a range
of health insurance products, health conditions, and treatments. In
addition, Jim Guest, CU's President and CEO, serves as a member of the
Institute of Medicine's Roundtable on Evidence-Based Medicine, which
has been convened to ``transform the way evidence on clinical
effectiveness is generated and used to improve health and health
care.'' \2\
---------------------------------------------------------------------------
\2\ Charter and Vision Statement, Institute of Medicine Roundtable
on Evidence-Based Medicine, http://www.iom.edu/CMS/28312/RT-EBM/
33544.aspx. See also: Institute of Medicine. 2007. Learning What Works
Best: The Nation's Need for Evidence on Comparative Effectiveness in
Health Care. http://www.iom.edu/ebm-effectiveness.
---------------------------------------------------------------------------
But today I want to concentrate on one consumer program that we
have developed that uses the best available scientific evidence to help
consumers, with their health-care professional, choose the most
effective, safest, and affordable drugs. I highlight this work, because
it provides a tangible example of the potential that expanded
government-funded comparative effectiveness research holds for both
improving health care quality and lowering health care costs. Consumer
Reports Best Buy Drugs \3\ is based on systematic reviews--unbiased
medical reviews of the clinical research on drug comparative
effectiveness, safety, and side effects--conducted by the Drug
Effectiveness Review Project (DERP). DERP, based at the Center for
Evidence Based Policy at Oregon Health and Science University,
coordinates the preparation of the careful (and heavily peer-reviewed)
reports that are written by various Evidence-based Practice Centers
which receive support from the federal Agency for Healthcare Research
and Quality (AHRQ). DERP is a preeminent example of the pioneering lead
that the states have taken--with financial support through AHRQ's
funding of Evidence-based Practice Centers--in the world of evidence-
based medicine, specifically the research that is necessary to enable
states and other health care payers to shape benefit policy based on
unbiased scientific evidence.
---------------------------------------------------------------------------
\3\ Reports can be downloaded for free at www.CRBestBuyDrugs.org.
---------------------------------------------------------------------------
Our Best Buy Drugs program translates DERP's complex medical
reports into consumer-friendly reports that provide information the
public needs to understand the comparative effectiveness, safety, side
effects, and cost of drug options.\4\ Thanks to grants from a private
philanthropy, The Engelberg Foundation, and the National Library of
Medicine, Consumers Union is able to provide this information free-of-
charge to the public. This is important, since one of our key target
audiences is low-income consumers who use multiple drugs--the very
people who are most in need of unbiased, accurate information.
---------------------------------------------------------------------------
\4\ For a more detailed description, see Steven D. Findlay,
``Bringing The DERP to Consumers: `Consumer Reports Best Buy Drugs',''
Health Affairs--Web Exclusive, June 6, 2006.
---------------------------------------------------------------------------
Attached to my testimony is a description of Consumer Reports Best
Buy Drugs and a sample of a 2-page summary for one class of drugs,
proton pump inhibitors (anti-heartburn medicines). As you can see from
the table, there is not a large difference between the effectiveness of
the drugs in this category. The big story, though, is that there is a
ten-fold difference between the monthly price of the heavily advertised
``purple pill'' Nexium and an over-the-counter alternative (Prilosec
OTC) that the best science shows is equally effective and safe for
almost all of us. A month's supply of Nexium costs about $193, while a
month's supply of Prilosec OTC costs $19 to $26. Having said this, I
want to stress that we always urge consumers to consult with their
doctors, and we urge all health plans to have effective and easy-to-use
exceptions policies for the small amount of people who may not respond
as well to one of the Best Buy Drugs.
We have completed reports for 17 classes of drugs that millions of
consumers need today, including: statins to lower cholesterol, the
high-blood pressure and heart disease medicines (ACE inhibitors,
calcium channel blockers, beta blockers), anti-depressants,
antihistamines, menopause drugs, attention deficit and hyperactivity
disorder (ADHD), insomnia, and many more. We are working in six states
(Arizona, California, Georgia, Maryland, Minnesota, and Pennsylvania)
on pilot outreach projects to bring this information to as many diverse
populations as we can. We are working with doctors, pharmacists, senior
networks, libraries, state officials, health insurance companies,
PBMs--basically any entity that shares the goal of improving health
care quality while lowering health care costs. Under our free,
information-sharing agreement with Medco Health Solutions, for example,
we are exposing millions of consumers to Consumer Reports Best Buy
Drugs information when they click through to our drug reports via
Medco's Internet tool, My Rx Choices, which helps members identify
effective, lower-cost drug options. We are working with Tarascon, a PDA
software provider, to provide summaries of our reports on physicians'
PDAs to help them at the point of prescribing.
Helping consumers--and health plans--substitute effective, safe,
lower-cost medicines for the newest, heavily-advertised alternative can
save the nation billions of dollars without sacrificing quality. In
fact, better information about comparative effectiveness can help us as
a nation improve health care quality by educating consumers,
physicians, pharmacists, and policy makers about the comparative
effectiveness of alternative treatments. Educating the public about
low-cost, effective alternatives can help make health care treatments
and prescription drugs more affordable, and this in turn will increase
the number of patients who can get the treatment or drug that they need
and ultimately improve health outcomes. The stakes are huge. The table
below shows estimates of annual savings for select drug categories for
consumers switching from a high priced brand to a Best Buy Drug:
------------------------------------------------------------------------
Potential
Individual
Category of Drug Annual
Savings
------------------------------------------------------------------------
Statins (to lower cholesterol) $1,300
------------------------------------------------------------------------
Proton Pump Inhibitor (for acid reflux, ulcers, heartburn) $1,740
------------------------------------------------------------------------
NSAIDs (arthritis and pain) $2,200
------------------------------------------------------------------------
Anti-depressants $1,200
------------------------------------------------------------------------
Beta Blockers (high blood pressure) $1,900
------------------------------------------------------------------------
We have estimated that Medicare beneficiaries who select Best Buy
Drugs in five leading drug categories, when selecting their Medicare
Part D plan, can save up to $5,000 a year.\5\ Those switching in just
one drug category can typically save more than enough to cover the cost
of their Part D premium. Needless to say, on an aggregate level, the
potential savings to the nation's health care payers (both taxpayers
and private payers) can be counted in the billions of dollars. And the
key to realizing these savings is the basic scientific research, the
clinical studies that compare drugs' effectiveness, and the systematic
reviews that allow for an unbiased assessment of all clinical research
that has been done.
---------------------------------------------------------------------------
\5\ ``Medicare Prescription Drug Benefit: Beneficiaries Can Lower
Out-of-Pocket Costs While Getting Safe and Effective Drugs,'' Consumers
Union, March 2, 2006.
---------------------------------------------------------------------------
The inclusion of Section 1013 in the Medicare Modernization Act
represented a turning point in health care in the United States, and we
thank you for that section. By way of background, it is important to
keep in mind the short history of the highly successful comparative
effectiveness program. Congressman Allen, Congresswoman Emerson and
this Committee were instrumental in getting this non-controversial but
pioneering provision into the Medicare Modernization Act.
We are very pleased with the work done to date to implement Section
1013. AHRQ has already released to the public reports \6\ that provide
consumer-friendly, unbiased reports about the comparative effectiveness
of various options for a number of conditions. However, this work has
been funded at a low level ($15 million/year), far less than even the
level of funding authorized by the legislation. The expectation to date
has been that Section 1013 as implemented by AHRQ would fund systematic
reviews rather than actual new clinical trials that assess the
comparative effectiveness of treatment options. In order to fund new
comparative clinical trials, a significant commitment of resources and
leadership will be needed in order to carry out research that will fill
the gaps in our knowledge about comparative effectiveness of existing
and future medical treatments. It is important to keep in mind that
comparative clinical trials are expensive, and can cost between $40
million and $150 million.\7\
---------------------------------------------------------------------------
\6\ They have produced consumer guides on pain for osteoarthritis,
and a report on gastroesophageal reflux disease (GERD). Other reports
(mostly for medical professions) are on cancer, diabetes, the digestive
system, heart and blood vessels, brain and nerve conditions, and mental
health. The reports are available to the public at http://
effectivehealthcare.ahrq.gov.
\7\ ``The Clinical Antipsychotic Trials of Intervention
Effectiveness (CATIE) cost $42.6 million; the Antihypertensive and
Lipid-lowering Treatment to Prevent Heart Attacks trial (ALLHAT) cost
$125 million; the Study of Tamoxifen and Raloxifene trial (STAR) cost
$118 million.'' Footnote 14, page 7-6, The Future of Drug Safety,
Institute of Medicine of the National Academies, September 22, 2006.
---------------------------------------------------------------------------
Consumers Union has endorsed legislation by Representatives Allen
and Emerson to create a $3 billion comparative effectiveness trust
fund, by diverting a fraction of each health penny into the fund.\8\
The fund would be used by AHRQ, with the advice of a new advisory
panel, to fund the needed research. The bill insures the principles
that any research must be independent, scientifically based,
transparent and public, and include input from all stakeholders, and
cover the full spectrum of health care treatments.
---------------------------------------------------------------------------
\8\ The amount proposed, $3 billion over five years, represents
about.24 percent of projected funding over five years of about $12.3
trillion.
---------------------------------------------------------------------------
Consumers Union believes that it is appropriate for the government
to fully fund and sponsor this important research, which should be
available to all and constitutes the epitome of a public good. The
benefits will be shared by every one of us who use health care in this
country.
With regard to governance of any new comparative effectiveness
research effort, we urge you to make sure that the research is
conducted independent of all the groups whose products and procedures
it compares. Far too many clinical trials and research papers funded by
product sponsors have been found to be distorted and biased. Even the
nation's finest medical journals have found themselves duped by bad
data and distorted results.\9\ We agree with the principles that have
been developed by the Alliance for Better Health Care, a broad
coalition of consumer, labor, health plans, research organizations and
employers, that works in support of research on comparative clinical
effectiveness and dissemination of the research.\10\ We commend AHRQ
for the superb job it has done to date implementing Section 1013, and
we would urge you to build on the work AHRQ has done by expanding the
model of systematic reviews it has tremendous success carrying out. We
urge you to give careful thought, and draw on the advice of experts in
organizational structures, to assure that the structure to successfully
implement this large responsibility is solid and assures high quality,
transparent, independent research and analysis.
---------------------------------------------------------------------------
\9\ PLoS Medicine, May 2005, Vol. 2, Issue 5 e138, ``Medical
Journals are an Extension of the Marketing Arm of Pharmaceutical
Companies,'' by Richard Smith.
\10\ Key principles include the need for a significant and stable
investment, consideration of the full spectrum of health care
treatments, scientific integrity and independence, transparency in all
processes, stakeholder involvement, and accountability of all conduct.
See Letter from Alliance for Better Health Care to Congressman Allen
and Congresswoman Emerson, May 14, 2007. *** TO PREVENT BAD
BREAK AT BODONI DASH *** deg.
---------------------------------------------------------------------------
Mr. Chairman, Members of the Committee, the rapidly rising cost of
health care threatens the budgets of federal and state governments, and
ultimately, the health of consumers. We believe that establishing a new
program--with sufficient, reliable funding--to assess the comparative
effectiveness of alternative treatments is a necessary part of the
solution to this growing problem. A fully funded, thorough comparative-
effectiveness effort would be a wise and cost-effective investment. We
commend you for giving this legislation serious attention. Enacting
this legislation is probably the most important thing that you can do
in this Congress to save lives and money, and to build a foundation for
further reforms of our health care system.
Chairman STARK. Thank you.
Dr. Hearn?
STATEMENT OF SUSAN HEARN, Ph.D., SENIOR PROJECT MANAGER IN
ENVIRONMENTAL HEALTH AND SAFETY, DOW CHEMICAL COMPANY, MIDLAND,
MICHIGAN
Ms. HEARN. Mr. Chairman, Ranking Member Camp, and Members
of the Subcommittee, Dow thanks the Subcommittee for holding
this hearing to examine the benefits of expanding the
availability of comparative effectiveness research. We, along
with members of the broader business community, believe that
improvements in quality and effectiveness of health care are
absolutely essential in addressing the many challenges facing
health care.
Comprehensive, timely generation, and effective
dissemination of comparative effectiveness research is
paramount in making progress. Most unfortunately, this is one
of the most significant voids in information we face today.
Dow provides comprehensive health care benefits to over
110,000 employees, retirees, and their dependents in the United
States. In total, we spent over $300 million on health benefits
in the U.S. last year, which is equivalent to about 31 cents a
share or 25 percent of what we spend on research and
development.
When you factor in indirect hidden costs to productivity
from chronic diseases like obesity, the economic impact
multiplies by a factor of 2 or 3. Absenteeism and presenteeism
play a big role in pushing our costs up to $700 million a year,
or about 70 percent of our R&D budget.
For the money we spend and for the peace of mind of our
employees and their families, we want to ensure that we are
getting the best value in health care available. Today, that is
not the case.
Medical errors are the eighth leading cause of death in the
United States, costing billions of dollars a year, and
inappropriate care represents up to 30 percent of medical
expenses. Comparative effectiveness research may greatly
improve health care quality and patient outcomes, ensuring that
consumers receive the best care at the best value.
It is extremely important that patients and payors are
assured the delivery of quality evidence-based health care.
With adequate funding, comparative effectiveness research can
provide information to enable physicians to make better
decisions based on the scientific information elicited in
evaluating various treatment options.
Consumers too need comparative information to make better
informed choices about their health care. As consumers are
encouraged to get more involved in management of their health
care, and as consumer-driven health plans become more
prevalent, consumers will need to know which treatments are
more effective in treating their particular condition.
Currently there are relatively few clinical effectiveness
research studies conducted each year. An increase in funding
this research could pay off substantially by reducing
ineffective treatments, thus improving the quality of care,
improving health outcomes, and saving significant health care
dollars.
The Agency for Healthcare Research and Quality conducts
such research through their Effective Health Care program to
examine the effectiveness of alternative treatments, and this
research is producing good results and valuable information to
help improve outcomes. At the current funding levels, this
research, however, is just scratching the surface. We encourage
Congress to significantly increase the funding for comparative
effectiveness research.
This research has the potential to improve health care
delivery and ultimately benefit the health of all Americans by
reducing inappropriate and ineffective care. Employers like Dow
will ultimately benefit from the availability of high-quality
effective treatment and the potential cost savings that are
expected through a significant reduction in wasteful spending
on less effective, ineffective, or inappropriate care.
Our employees will benefit through improved health
outcomes, and the peace of mind knowing that they have good
information to help make better health care decisions for
themselves and their families. This will also benefit Dow by
reducing the stress and anxiety that can often accompany a
personal or family illness. This indirect contribution to our
productivity, having our employees at the top of their game
always, will help us achieve our vision of being the most
profitable, most respected, and largest chemical company in the
world.
At Dow we have a very clear health strategy which
emphasizes prevention, quality and effectiveness of care, and
health system management. We are strongly committed and very
engaged at the local and national levels, working with many
partners to drive improvements and achieve better health
outcomes as well as an improved economic picture.
However, we can only do so much without the critical
information provided by comparative effectiveness research. We
urge you to significantly increase funding for comparative
effectiveness research and continue to support this
tremendously important program, and expand the good work of the
Agency for Healthcare Research and Quality. Thank you.
[The prepared statement of Ms. Hearn follows:]
Prepared Statement of Susan Hearn, Ph.D.,
Senior Project Manager in Environment Health and Safety,
Dow Chemical Company, Midland, Michigan
Mr. Chairman and Members of the Subcommittee, I am Susan Hearn,
Dr.P.H., Senior Project Manager in Environment, Health and Safety for
The Dow Chemical Company. The Dow Chemical Company (Dow) thanks the
Subcommittee for holding this hearing to examine the benefits of
expanding the availability of comparative effectiveness research. We,
along with the broader business community, believe that improvements in
quality and effectiveness of health care are absolutely essential in
addressing the many challenges facing health care. Comprehensive,
timely generation and effective dissemination of Comparative
Effectiveness Research (CER) is paramount in making progress. Most
unfortunately, this is one of the most significant voids in information
we face today. In this light, funding for CER is viewed as a key
investment in both improving the health of people and reigning in
future health care costs. As you have heard from so many, we must as a
nation find solutions to the affordability of health care or we will
continue to be at risk in the global economy.
Dow provides comprehensive health care benefits to over 110,000
employees, retirees and their dependents in the United States. In
total, we spent over $300 million on health benefits in the U.S. last
year--which is equal to about 31 cents a share, or 25 percent of what
we spend on Research and Development.
When you factor in the indirect, hidden costs to productivity from
chronic diseases like obesity, the economic impact multiplies by a
factor of two to three. Absenteeism and presenteeism play a big role in
pushing our costs up to $700 million per year or about 70 percent of
our R&D budget. For the money we spend--and for the peace of mind of
our employees and their families--we want to ensure that we are getting
the best value in health care available . . . and today, that is not
the case.
Medical errors are the eighth leading cause of death in U.S.,
costing billions of dollars each year, and inappropriate care comprises
up to 30 percent of medical expenses.
Comparative effectiveness research (CER) may greatly improve health
care quality and patient outcomes, ensuring that consumers receive the
best care at the best value. It is extremely important that patients
and payers are assured the delivery of quality, evidence-based health
care. With adequate funding, comparative effectiveness research can
provide information to enable physicians to make better decisions based
on the scientific information elicited in evaluating various treatment
options. Consumers, too, need comparative information to make better
informed choices about their health care. As consumers are encouraged
to get more involved in management of their health care and as consumer
driven health plans become more prevalent, consumers will need to know
which treatments are more effective in treating their particular
condition.
Currently, under authority granted by Section 1013 of the Medicare
Modernization Act (MMA), the Agency for Healthcare Research and Quality
(AHRQ) conducts research to examine the comparative clinical
effectiveness and appropriateness of different treatments, and the ways
that those treatments can be provided in a more effective and efficient
manner. Since FY2005, AHRQ has received $15 million per year as funding
for CER. AHRQ has already released final reports on treatment options
for breast cancer, gastroesophogeal reflux disease (GERD), cancer-
related anemia, low-bone density, depression and other conditions.
The findings released by AHRQ have just begun to show the value of
CER for patients, providers, and health care payers. However, there is
much more that can be done and, under current levels of funding, AHRQ
is very limited in the types and numbers of studies it can conduct. To
remedy this, and to ensure this research contributes fully to improving
care and saving significant federal dollars, we request that Congress
act to increase its investment substantially.
As a member of the Alliance for Better Health Care (ABHC), Dow
supports the principles developed for prioritizing, conducting,
disseminating, and using CER.
CER has the potential to benefit the health of all
Americans and is a true public good.
Significant and stable investment is needed in CER--
in the development of research methods and researchers, the
design and conduct of studies, the scientific review of
research, and the dissemination and communication of results--
for it to reach its full potential.
The scope of CER should address the full spectrum of
health care treatments, including pharmaceuticals, devices,
medical and surgical procedures, and other interventions.
Scientific integrity and independence are paramount.
CER should be based on scientific evidence employing
an array of appropriate methods, such as randomized clinical
control trials, observational studies, meta-analyses, and
systematic technology assessment reviews.
The processes for identifying research priorities,
conducting research, validating the science, and disseminating
results should be transparent.
Any entity that commissions or conducts CER should
involve stakeholders in setting research priorities and
disseminating research.
Board governance should assure accountability in the
conduct and dissemination of CER.
Comparative effectiveness research has the potential to improve
health care delivery and ultimately benefit the health of all Americans
by reducing inappropriate and ineffective care. Employers like Dow will
ultimately benefit from the availability of high-quality, effective
treatment and the potential cost savings that are expected through a
significant reduction in wasteful spending on less effective,
ineffective or inappropriate care. Our employees will benefit through
improved health outcomes and the peace of mind knowing that they have
good information to help make better health care decisions for
themselves and their families. This will also benefit Dow by reducing
the stress and anxiety that can often accompany a personal or family
illness. This indirect contribution to our productivity--having our
employees at the top of their game always--will help us achieve our
vision of being the largest, most profitable, most respected chemical
company in the world.
At Dow we have a very clear health strategy which emphasizes
prevention, quality and effectiveness of care and health system
management. We are strongly committed and very engaged at the local and
national levels working with many partners to drive improvements and
achieve better health outcomes, as well as an improved economic
picture. However, we can only do so much without the critical
information provided by comparative and effectiveness research. Our
senior physician was a member of the Clinical Research Roundtable
chartered by the National Academies. This group, which concluded its
work in 2004, made strong calls for expanded effectiveness research and
clinical effectiveness research.
We urge you to significantly increase funding for comparative
effectiveness research and continue to support this tremendously
important program and expand the good work of AHRQ.
The research that would be produced by this effort will be
invaluable to doctors, other health professionals, and patients, as
they increasingly demand to know the benefits of various treatment
options for their conditions. It promises to significantly improve
quality and safety as we learn more about what medical interventions
work, how well they work, and which ones do not work.
Thank you for the opportunity to share our views.
Chairman STARK. Thank you.
Dr. Teutsch?
STATEMENT OF STEVEN M. TEUTSCH, M.D., MPH, EXECUTIVE DIRECTOR,
OUTCOMES RESEARCH AND MANAGEMENT, OFFICE OF EXTERNAL MEDICAL
AND SCIENTIFIC AFFAIRS, MERCK AND CO., INC., WEST POINT,
PENNSYLVANIA
Dr. TEUTSCH. Good afternoon, Mr. Chairman and Members of
the Subcommittee. I am Dr. Steven Teutsch, Executive Director
of the Outcomes Research Group in the Office of External
Medical and Scientific Affairs at Merck. Thank you for the
opportunity to discuss issues of comparative effectiveness in
health care.
Merck supports a role for comparative effectiveness
analysis. We understand the needs of payors, providers, and
patients for better information on what works and for whom, and
believe that comparative effectiveness is an important
mechanism for producing that information.
We are collaborating with America's health insurance plans
to develop a road map to provide guidance on how comparative
effectiveness can be incorporated into coverage decisions, and
have cosponsored a forum last fall with AHIP and Kaiser
Permanente on the same topic.
Companies such as Merck have extensive experience in the
methods for assessing effectiveness as well. There is a lot at
stake in a greater national effort to conduct systematic
comparative effectiveness of medical treatments. Consumers and
patients want continued access to new, possibly life-saving
medical interventions. Providers want the ability to practice
the best medicine for their patients and to keep up with the
latest information. Payors want rapid diffusion of
scientifically valid information about what works best, for
whom, and in what circumstances. Innovative suppliers like
Merck want continued incentives to develop and market those
treatments.
Finally, while comparative effectiveness analysis generally
produced analyses at the group or sub-population level,
patients and physicians want to find the best treatment for
each individual. It will be important to bring the individual
and population sciences and perspectives together to optimize
patient management.
The science of comparative effectiveness has come a
tremendous way in recent years, and there is a consensus about
many of the methods. There remain a number of legitimate
concerns about the scope of comparative effectiveness, as well
as some specific methods and policy considerations. Among those
are whether economic analyses should be included, and if so,
from whose perspective.
What level of scientific rigor is needed for each type of
decision? How do we assure transparency of the scientific
process? Which observational methods are sound, replicable, and
transparent? How best to proceed when the available evidence is
insufficient?
Despite the worries and reservations, I think it is fair to
say that there is an emerging consensus among various system
stakeholders on the shape and purpose of a larger national
effort to conduct comparative effectiveness. This emerging
consensus seems to be shaping up as follows.
Comparative effectiveness analysis should be guided by
input from a broad array of public and private stakeholders. It
should be applied to the full array of health care
interventions, including diagnostics, procedures, and devices,
as well as drugs.
Resources should target research for diseases or conditions
that impose a high clinical and economic burden on the health
care system and society, and where the information can lead to
improvements in health and efficiencies in the health care
system. The analyses should be scientifically sound, rigorous,
predictable, replicable, transparent, and fair.
New stable sources of funding are needed to generate
evidence, since much of what is needed does not exist. We also
need to further develop the methods, assure that the results
can be used by decisionmakers, and develop the human capital
necessary to perform the work.
Comparative effectiveness analysis should be conducted by
an entity independent of payors, including the government
payors, and industry. The results should inform clinical
guidelines for use by medical professionals, quality
improvement, as well as by payors for coverage and
reimbursement.
There are other points that Merck would like to emphasize.
Results should be used equally by payors and others when the
results are positive and when the results are negative. For
example, where the outcome of an evaluation is positive, payors
should commit to reasonable coverage and encourage appropriate
use.
As payors use comparative effectiveness analysis to help
assess the value of an intervention, they should consider the
short and long-term value to patients. The purpose should not
be to hinder access to new technologies, but to assure their
appropriate use. Comparative effectiveness information cannot
be static. It must remain current with the state or the
science, and thus there must be timely processes to incorporate
new evidence.
Merck supports actions that bring new resources to bear on
this work in this country. We believe that, properly
implemented, it has great potential to assure better
decisionmaking and improved clinical management. The
pharmaceutical industry has a great deal of experience in this
area, and we believe that we have a vision of a better future
based on an expanded role for evidence-based medicine in
general and comparative effectiveness in particular.
I thank you for your time, and would be pleased to answer
any questions.
[The prepared statement of Dr. Teutsch follows:]
[GRAPHIC] [TIFF OMITTED] T5994A.038
[GRAPHIC] [TIFF OMITTED] T5994A.039
[GRAPHIC] [TIFF OMITTED] T5994A.040
[GRAPHIC] [TIFF OMITTED] T5994A.041
[GRAPHIC] [TIFF OMITTED] T5994A.042
[GRAPHIC] [TIFF OMITTED] T5994A.043
[GRAPHIC] [TIFF OMITTED] T5994A.044
[GRAPHIC] [TIFF OMITTED] T5994A.045
[GRAPHIC] [TIFF OMITTED] T5994A.046
Chairman STARK. Well, thank you all. I guess, first of all,
Dr. Teutsch, you are a member in good standing of PhRMA? You
haven't been kicked out?
Dr. TEUTSCH. Yes. The company is definitely a member.
Chairman STARK. Dr. Hearn, you are a member in good
standing of the National Association of Manufacturers? You
haven't been thrown out?
Ms. HEARN. Right.
Chairman STARK. You are not going to lose your 501 status,
I gather. Everyone is in good shape here. The questions, it
seems to me, are that we should proceed with outcomes research
or effectiveness research. There is a question of who, where,
what. Gail gave us four choices in hers. But what entity does
this?
The question of who pays for it, and I think that we get
unanimity that everybody doesin some manner the taxpayers, and
in some manner stakeholdersmaybe the docs have to kick in
through the AMA; I don't knowbut that everybody pays some
portion of the cost of maintaining this, and that the world has
free accessand I guess I am asking this of any of you; I will
get aroundto the information. I can think of no reason other
than to protect some personal identities that this information
shouldn't be publicly available to researchers or companies or
consumers or anybody that wants to use it.
So, I guess the next question is: How do we get started?
How do I sell my colleagues on both sides of the aisle that we
should proceed with this?
Let me start with Gail. You suggested, I think, most
prominently as a model, if not the place to start, would be
AHRQ. Is that a fair assessment?
Ms. WILENSKY. I believe the better strategy is to have what
is called a federally funded research and development center
attached to AHRQ. So, it would have the close linkage to AHRQ
is very important. I would like just a little distance to try
to make sure the objectivity and credibility is conferred.
Chairman STARK. I guess I would just ask if there are any
of the witnesses who would have an objection to at least
starting down this road. You are familiar, Dr. Teutsch, with
some of the Federal bureaucracy and the health bureaucracy.
Dr. TEUTSCH. Yes. I spent 21 years at CDC, and I had the
pleasure of working closely with AHRQ on a lot of its evidence-
based development projects. They provide an incredible
leadership.
We believe that there really should be some public/private
partnership to make it work. Gail obviously has given us an
example of what such a model might look like. We believe that
there needs to be a structure that does that, but that there is
also a group of stakeholders, as part of the leadership, that
can guide the process to make sure it meets the needs of the
users and all those who have important stakes in that
information.
Chairman STARK. somebody suggested a Federal Reserve-like
entity. But I am just thinking, as legislators, for us to
create a Federal Reserve, the first problem would be finding a
suitable site on the Mall. We would be competing with every
former president for memorials there. That may be somewhat more
ambitious.
But I am not so sure that a Federal Reserve-like structure
attached to AHRQ isI guess it is one thing to say, how do we
start? It would be wasteful, as far as I am concerned, to spend
two or 3 years getting some kind of a commission to decide
where are we going to start this entity. My instinct would be
to say, start it with AHRQ and have some kind of a sunset three
or four years out to see how they are progressing, and then
legislators could make a change if it wasn't working.
Would there be anybody on the panel that would be
uncomfortable with that sort of a procedure?
Ms. WILENSKY. The only hesitation and the answer is no. But
as a temporary measure, if this grows to be the size that I
think it needs to be, which is a couple billion dollars when we
are spending $2 trillion on health care, that kind of a size
would totally overwhelm AHRQ. AHRQ would become the Center for
Comparative Clinical Effectiveness Analysis.
AHRQ is the only place where any health services research
gets done. There are so many other questions that once this is
started, I think it needs to be in a slightly removed place.
This is a discussion I have had with Carolyn Clancy on
several occasions, and my understanding is she is quite
comfortable with this not quite an AHRQ but directly linked to
AHRQ because you want to make sure that there as I a close
working relationship.
Chairman STARK. But using AHRQ as a model, with some
additions.
Ms. WILENSKY. Right.
Chairman STARK. Dr. Teutsch, you mentioned something about
the idea, and I am not so sure that we could sell that, as I
that there be some requirement that where there is a savings,
either an improvement in health or a cost savings to
stakeholders, that it be required.
I am afraid, if that is what I understood your testimony to
be, that we ought to have a mechanism for saying if something
appears as Consumers Union might show us, that it saves us, as
individuals, a lot of money.
I am not sure we could get much further than that, and I
would be troubled by saying we would do anything except require
that the information be put out there, and that the
stakeholders, whomever they may be, use it as they choose
without any mandates from us. I don't think politically that
wouldbut maybe I misunderstood what you were saying.
Dr. TEUTSCH. Well, we are certainly not talking about
mandates ensconced in legislation. But this is a partnership
for which we all have to contribute in order to reach that
common good of getting appropriate care to patients and getting
the value for our health care dollar. That is going to require
a commitment to a common goal on all parts so that we then
develop the kind of quality improvement and reimbursement all
parties align to try and deliver that value.
What we are talking about here is understanding where that
value will be and providing that information. But we believe
all stakeholders have to have a common commitment to that
common goal.
Chairman STARK. I like the idea of a commitment, like a
pledge of allegiance. But that is okay. But to memorialize it
in legislation would trouble me some because as good a job as
you may do, with something for my kids, if you don't get rid of
the bubble gum flavor, they ain't ever going to buy a Merck
product, I can tell you, regardless of what the commission
would say they should do. So, there are some levels at which
you may never get the consumers to march in lockstep.
Well, I guess I would just as soon give Dave Camp a chance
to try and defeat what seems to be a unanimous recommendation
here.
Mr. CAMP. No. I actually liked what I have been hearing.
Chairman STARK. Good.
Mr. CAMP. Thank you, Mr. Chairman. I think there is a lot
of bipartisan support on this entire concept in the way to move
forward. I think it has been a good hearing. So, thank you for
having it.
I notice, Dr. Wilensky, in your testimony you mentioned
that the political vulnerability of the center is probably the
most critical issue. Dr. Mark Miller earlier said if it is
connected to a Federal agency, they won't deliver any bad news,
this center.
I notice you have got a model where there is a tie to AHRQ,
but somewhat independent. How do you think we best avoid this
problem of the difficult issues? This will be very
controversial information, I think, at times; obviously, very
helpful at times as well.
Ms. WILENSKY. Right. There is no perfect placement. So, I
want people to understand that. Everything is involving a
tradeoff between credibility and independence, objectivity.
The removal of being exactly a part of AHRQ, although the
legislation could describe how the information is able to be
released to the publicI mean, that is something legislation
could do I was more concerned. I have spoken to a number of
groups, industry but academic health centers or individuals on
the right and left of the political spectrum. The sense I had
is having this function directly in government AHRQ, NIH, new
agency left a lot of people feeling very uncomfortable. It was
too close to government.
That was why, to me, having the FFRDC linked to AHRQ was
appealing because on the other hand, you want to make sure you
have accountability. I mean, there are a lot of ways to remove
it from government. You could put it attached to the Institute
of Medicine, which I think is another option. You could have it
ms freestanding.
But if there is going to be a lot of public money here, I
have heard from Members and congressional staff that there is
in having make sure that there is a real accountability. That
is why the notion of having it linked to AHRQ was so appealing
as the best tradeoff, close but not too close, to government.
That seemed to strike the bell, so that I have moved
forward in the last 6 months in my thinking of being more
specific that I believe that is the best tradeoff. But I would
certainly not object to other kinds of tradeoffs.
Mr. CAMP. Yes. Well, it is a good suggestion. You view a
board similar to a MedPAC board, with a variety----
Ms. WILENSKY. Absolutely.
Mr. CAMP [continuing]. Of members that oversee this
organization?
Ms. WILENSKY. You need to have all the stakeholders
governing what goes onindustry, academia, patient advocates.
The consumers have to be involved. If they are not there, they
are going to be lobbing grenades from the outside.
So, the need to be on the governing board as to how
decisionmakinghow priorities are set; making sure the
information is fair and credible. Practicing physicians in
addition to the academic medical Committee have to be
represented. Everybody is going to be affected by this.
Mr. CAMP. I appreciate that. I wondered if anyone would
like to comment. First of all, I want to thank you all for your
testimony. It is very helpful.
I notice, Ms. Shearer, this best buy drugs program really
looks at the presence of a single disease. Many people,
particularly seniors, have multiple chronic illnesses and take
multiple prescription drugs. It is really that dynamic that I
think we have to be concerned about as we move ahead. My
concern is if you look at the best buy for a particular drug,
it may not relate with the entire health regimen an individual
is on. Can you comment on that?
Ms. SHEARER. Yes. That is a very, very important point.
That really goes to the heart of why each of our reports says
that recommends that the patient talk with their doctor. Each
of the systematic reviews that form the basis of our analyses
do look at co-morbidities, and we do try to include that
information. If a person has various conditions, there may be a
different recommendation.
So, to the extent possible, we try to address that in our
reports. But we always say that this is not about consumers
picking their medicine, but they should talk with their health
care provider.
Mr. CAMP. Last, Dr. Wilensky, you are viewing this
organization has the bulk of its funding from government
sources, but that there would be a significant private sector
component to this as well. How do you see that?
Ms. WILENSKY. There are two ways to do that. The law allows
for up to 30 percent of the funding to come from the private
sector. It is frequent that these FFRDCs and there is a good
description, I think, in MedPAC, and also the Institute of
Medicine has a very good description of some existing FFRDCs.
So, you can look at what is out there. How long. How big.
It could be through voluntary contributions. We have some
very large foundations that are interested in this type of
work, the Robert Wood Foundation. But also, several of the
insurance companies when they have converted to for-profit
status have set aside. These large foundations, they are all
expressing an interest.
So, one possibility is to have that. Another is to have
just a direct assessment. Then you could decide, does that
really count for being private? Or if it is a direct
assessment, is that public? I don't know what the legal ruling
would be.
It is the flexibility, and particularly in the startup, but
even ultimately having some private sector dollars in there to
make people feel like they are directly a part of the process.
But of course, you need to make sure there are a lot of rules
in place so that you can't have that influence what goes on in
terms of the studies.
But of course, that happens with FDA all the time. PhRMA
companies do the trials or fund the trials. They just have to
be subject to auditable results. They have to follow the rules
or the results aren't acceptable to the FDA review process. So,
we think there are some models as to how that part of it could
work.
Mr. CAMP. All right. Thank you. Thank you, Mr. Chairman.
Chairman STARK. Mr. Johnson?
Mr. JOHNSON. Thank you, Mr. Chairman.
Dr. TEUTSCH. how do you ensure that comparative
effectiveness research is not misused in ways to discourage
medical advances, and how do we make sure that the research
supports the quest for an implementation of new breakthrough
therapies?
Dr. TEUTSCH. That would be a concern, of course, that a
manufacturer would be likely to have, too. But we believe in
the medications and the innovation that industry delivers. We
believe that by developing products that provide real value,
making sure that value is clearly understood, and then that we
have the processes to help translate that so that the right
patients get them at the right time, are all ways to assure
that that innovation is adequately rewarded.
So, we actually see this as very much part of the solution
to stimulate innovation, and even personalized health care, as
we go forward.
Mr. JOHNSON. So, the doctor combines it?
Dr. TEUTSCH. Well, doctors do need that information, and
Dr. Dale can talk about it. It is very difficult to keep up
with all the current information that is available out there.
You heard earlier that there is a wide variation in care. What
we want is not homogeneity of care. We want to have doctors
with the right information to deliver the best information to
patients.
When we talk about comparative effect issues, it doesn't
mean one size fits all. It means understanding the tradeoffs
among these different innovations to where they fit most
appropriately into care, and how we take advantage of the
diagnostic products and other kinds of information so doctors
can make better decisions.
This feeds into the systems of decision support that will
allow doctors to have the information that they need so that
they can have the information that is scientific, but then
weigh it, along with the uncertainties and the preferences and
the values that the patients, the communities, and the
profession holds to make better decisions.
Mr. JOHNSON. Thank you, sir. Thank you, Mr. Chairman.
Chairman STARK. Let me try one more, if I can try the
patience of the panel.
In trying to legislate, trying to create this $2 billion
giant whichI mean, I am trying to think of how you ease into
this and can establish a protocol. For example, Dr. Dale, I
don't think we could start right away with surgical procedures
because I think you have got to wait 5 years or so to find out,
not that you lived through the operation but how is your life
five years later, to really study the effectiveness.
That gets me to the point thatand I don't mean to pick, Dr.
Teutsch, on your industry, but we do have an awful lot of
information about pharmaceuticals already, starting from the
genesis at FDA. Zocor is the same Zocor in California as it is
in Michigan as it is in Maine. So, we are not talking about
differentthey may use it differently in different parts of the
country.
But what if we started with that areaadmittedly, physicians
would be involved because some physicians would administer the
pharmaceuticals differentlyand then proceed, maybe take up the
vices or physician practices, as we get the ability to collect
the information.
Would there be any down side in that, just as a way to
begin? Dr. Teutsch, could Merck live with that?
Dr. TEUTSCH. I think that that actually creates some
issues. The reason is, the pharmaceutical side is a but like
looking under the lamppost because we do have good information.
In fact, the real paucity of information tends to be on the
utility of diagnostic devices and procedures. That is where
some 90 percent of the cost is. So, it is going to be important
to look at those technologies and help drive the generation of
that information.
So, we do need to look broadly, and in fact not look where
the information is best, but actually try to figure out where
there is a paucity of information where decisions are sub-
optimal because of the lack thereof.
Chairman STARK. Dr. Dale?
Dr. DALE. Well, I will just comment. I think there is room
for good work in all those areas. For instance, in the follow-
up on surgical patients, having an adequate record system to
know what the long-term consequences are. For instance, in our
country now with the rampant use of obesity surgery, what
happens? For the next patient, how do you describe what is
likely to happen to that person?
So, I think there is room in surgery and in devices. But I
would also say in terms of the long-term use of drugs as well.
Many side effects are not recognized in the early phase of
clinical trials when it is clear that there is an effect. But
the long-term consequences of use need to be better studied.
Ms. WILENSKY. I think if you want to affect spending, you
really have to move away from just the drug focus. That is just
10 cents of the dollar. A study that was recently reported
Chairman STARK. I didn't imply that we wouldn't do it all.
I am just saying at some point you have got to start.
Ms. WILENSKY. But I think if you think about a staggered
research agenda, where you want to have some early wins because
you want to make sure to everyone it is clear why this is good
investment.
But looking at areas that either high cost or high volume,
we could look at a couple of the major DRGs as to where are we
spending our money, where there are significant differences
about how you could go at delivering the care. There are a lot
of dollars attached.
A recent study that was released that looked at the whole
issue of stenting versus angioplasty versus conservative
treatment of medicine versus bypass surgery indicated the kinds
of questions that are still out there. It is only the first
look at a broad-based set of questions in cardiovascular
disease.
But cardiovascular disease, orthopedic surgeryI mean, these
are areas where there is a lot of money and a lot of
uncertainty in variation trying to inform who actually gains.
Because the answer is almost always going to be for some people
these aggressive interventions are really important.
The question is, what about the other groups? What can we
say about how to help them that may result in a lot of reduced
spending because they don't need such aggressive treatment? It
is the binary all-or-nothing thinking that tends to get us in
trouble.
If it is really important for one small subgroup of the
population, does that mean it is useful for everybody that
might have something that looks related? That is the kind of
work that we have got to get started on or we will never be
able to get Peter Orszag's money scored.
Chairman STARK. Is there anyone else who would like to
comment before you go to lunch?
Ms. SHEARER. Well, I would just like to make one point. I
think that Mr. Orszag gave us a very cautious read on potential
savings. I think it is important for the Committee to keep in
mind that the drug effectiveness review product is already
yielding huge savings on behalf of states that use that
information for their Medicaid programs.
So, I think that there is tremendous potential. I think
that it could be realized sooner than he may have thought in
his cautious analysis.
Chairman STARK. Well, in the absence of any of the
witnesses that would like to add any more comment to the
record, I would ask Mr. Camp if we can keep the record open so
that Dr. Clancy and others could respond to written questions
by Members. You may find in the coming days that Members will
send some of you written requests, which we would hope you
might be willing to respond to for the record.
I want to thank you for your patience and your contribution
to this very much. Thank you very much. The hearing is
adjourned.
[Whereupon, at 12:44 p.m., the hearing was adjourned.]
[Submissions for the Record Follow:]
Statement of Advanced Medical Technology Association
We thank the Committee for holding this important hearing today on
strategies to increase information on comparative clinical
effectiveness.
AdvaMed is the largest medical technology trade association in the
world. AdvaMed member companies produce the medical devices, diagnostic
products and health information systems that are transforming health
care through earlier disease detection, less invasive procedures and
more effective treatments. Our members produce nearly 90 percent of the
health care technology purchased annually in the United States and more
than 50 percent purchased annually around the world. AdvaMed members
range from the largest to the smallest medical technology innovators
and companies. The medical technology industry directly employs
approximately 350,000 workers in the U.S.
The Important Role of Research to Guide Clinical Decision-Making
AdvaMed is strongly committed to the principles of evidence-based
medicine and we support comparative effectiveness research as a means
to improve clinical outcomes and promote access to quality of care.
Sound comparative effectiveness research can be used to assist patients
and physicians in medical decision-making by identifying the relative
advantages and disadvantages of alternative means to prevent, diagnose
and treat disease.
For any government-funded comparative effectiveness research
initiative, we believe that the following principles should be applied
to ensure that comparative effectiveness research is carried out
appropriately:
Patient-centered care and independent professional
medical judgment. Comparative effectiveness research should
inform medical decisions, not replace medical judgment with
national treatment formulas. Its objective should be to provide
better evidence for physicians and patients to use in making
individual clinical decisions for each patient's. unique
condition.
Protecting patient access. Comparative effectiveness
research typically analyzes which medical intervention, on
average, is usually more effective across a population. The
intervention that is ``generally best,'' however, may not be
best for each individual patient. A determination of what may
be more effective on average may not account for the age or sex
of a patient. It wouldn't consider what is most effective under
the presence of comorbidities or special patient care needs.
Therefore, the entity should neither make recommendations nor
decisions about coverage. In addition, comparative
effectiveness research should not be used by Medicare,
insurance companies, or other payers to deny coverage. Patients
should maintain access to treatment options based on their
needs and preferences, including the desire to receive an
intervention that may be determined to be on average less
effective, but may be less painful or provide for shorter
recovery times for the patient.
Setting priorities. The comparative effectiveness
research agenda must be prioritized and designed with pre-
stated objectives, research questions, and stakeholder input.
It should focus resources on areas that have major clinical
significance and will have the greatest return on investment.
The agenda development process must be open and include
practicing physicians, patients, manufacturers, and other
stakeholders.
Robust databases and analysis. It is critical that
effectiveness be evaluated over a period of time that is
appropriate for the specific intervention being evaluated.
Studies should be based on the time period over which all
relevant benefits and other factors accrue, not set
arbitrarily--at 30 or 60 days, or 1 year. In addition, any
database that is used to assess the effectiveness of an
intervention must include robust data on that particular
intervention.
Using Clinical Information to Improve the Quality of Care and
Efficiency of the Health Care System
AdvaMed strongly supports using clinical information
that exists or could be derived from comparative effectiveness
research to improve patient care and reduce waste and
unnecessary costs in the health care system. Improvements could
include:
Advances in the quality of care provided,
including the appropriate use of preventive, screening
and diagnostic services and reduction in medical/
medication errors;
Changes in clinical processes that eliminate
practices that are not beneficial, as determined by a
consensus of the peer-reviewed literature or by the
relevant medical specialty societies; and
Improvements in administrative or health care
delivery processes, such as through the use of
information technology or the reduction of unnecessary
emergency room use.
For example, for more than a decade medical researchers have known
that people on ventilators should generally have their heads elevated.
When the patients are lying down, bacteria can easily travel from the
stomach, up to the mouth and breathing tube, and ultimately into the
lungs, causing pneumonia. When people are propped up, gravity becomes
their ally, but hospitals have had a hard time translating this
scientific knowledge into better medical care. Patients frequently need
to be put on their backs, to be bathed or to receive treatment, and
once they are lying down, doctors and nurses, who are busy worrying
about dozens of other things, don't always remember to move the bed
back up. The solution is to set up a rule that every patient on a
ventilator had to be sitting up. Making common-sense practice pattern
changes such as these to reduce infections in the intensive care unit
do not involve cutting-edge science, but can make a big difference,
cutting the incidence of ventilator-associated pneumonia by more than
40 percent in some hospitals. It can be the difference between life and
death for some patients.
We think there are potentially great opportunities for both quality
improvement and cost savings in these types of health system changes.
As a result, we believe that a government-funded comparative
effectiveness research initiative should include this type of research
involving health system changes that affect the management and delivery
of health care items, services, and procedures.
Appropriate Uses of Comparative Effectiveness Research
Comparative effectiveness research should be used to inform medical
decisions, not replace medical judgment with national treatment
formulas. We recommend:
Studying clinical effectiveness only. Patients should
have access to the interventions that are best for them.
Consequently, comparative effectiveness research should study
clinical effectiveness only, with the goal to inform medical
decision-making. As a result, quality of care and overall
efficiency in the health care system should improve.
Recognizing the process of medical device innovation.
Medical device innovation is evolutionary, and the
effectiveness of a particular product is dependent on the
training, experience, and skill of health care professionals.
Since many devices are a component of a medical procedure,
introduction of a new product may require that physicians
develop new skills. For example, the introduction of
percutaneous transluminal cardiac angioplasty (PTCA) during the
1980s offered an alternative to cardiac bypass surgery but
physicians needed additional training in order to perform the
procedure. The existence of a ``learning curve'' can be an
important constraint on the speed of introduction of a new
medical procedure.
As physicians gain experience with the device, they may be better
able to identify patients who are suitable--or not--for the therapy.
They learn how to recognize subtle anatomical differences that
influence how best to perform the procedure. Experience also helps the
clinician learn to tailor after care for the needs of a particular
patient. A case in point is bariatric surgery for weight loss. Clinical
practice guidelines recommend a training program involving at least 10
open procedures or 25 laparoscopic procedures performed under the
supervision of a qualified proctor. The development of this training,
experience and skill can have a major impact on patient outcomes.
Furthermore, research that is conducted too early may quickly
become dated, and a snapshot of a particular device at a specific time
may incorrectly state its relative effectiveness. Accordingly, studies
on the comparative effectiveness of devices should consider the effect
of training and experience upon outcomes, should be applicable to the
current generation of technology, and should only be conducted when the
technology has an experience base and is widely available and mature.
Likewise, those using the studies should recognize these challenges and
limitations.
Transparency and stakeholder input. Comparative
effectiveness research must be developed and conducted in an
open and transparent fashion that incorporates stakeholder
input. This must include all aspects of research to enhance the
credibility of its conclusions, including the determination of
research priorities, the research methodology, and opportunity
to comment on the proposed findings through a formal peer
review process. Stakeholders should include patients,
physicians, hospitals, and experts from the medical device and
diagnostics industry. Governance of any public-private entity
should include representation of all stakeholders.
Defining quality and benefit appropriately.
Comparative effectiveness research should be both comprehensive
and tailored to the specific intervention being evaluated. For
example, because diagnostics are used to inform clinical
decision-making, such technologies should be evaluated based on
their impact on patient care management. Comparative
effectiveness research should consider the influence of health-
related quality of life (including disability reduction,
functional status, reduction in pain, and overall patient
satisfaction); work loss and productivity loss; patient
adherence; patient preferences and lifestyle choices; symptom
control; reduction in medical/medication errors and enhancement
of patient and healthcare worker safety; and estimated long-
term outcomes (which may result long after a clinical trial has
ended).
Supporting personalized medicine. For personalized
medicine to flourish, comparative effectiveness research
findings should be used as a reference, not a mandate, for
individual treatment decisions. As scientific advances in
technology continue, genomic and proteomic analysis, health
information technology, and other innovations in health care
have the potential to promote tailored treatment decisions for
each individual patient's unique needs, thereby saving patients
from unnecessary care and saving the health care system from
the expense of trial-and-error approaches to therapy.
Communication of findings and conclusions. Research
findings should be communicated in a fashion that clearly
acknowledges any limitations of the research and underlying
data. Armed with the knowledge of which conclusions can and
cannot be drawn, patients and physicians will be able to use
the research findings appropriately for individual diagnosis
and treatment situations. Details regarding the assumptions and
data sources should also be readily available. At the same
time, there should be a system for assuring that health
professionals making decisions are aware of the findings of
comparative effectiveness studies.
Congressional oversight. Any government funded
comparative effectiveness research initiative, whether
conducted through existing agencies or a newly formed
organization, should be subject to Congressional and executive
branch oversight.
Conclusion
Thank you again for holding this important hearing. As supporters
of evidence-based medicine, we look forward to working on this effort
to deliver the right treatment to the right patient at the right time.
We believe that comparative effectiveness research is a means to
improve clinical outcomes and promote access to quality care. It can be
used to assist patients and physicians in medical decision-making for
prevention, diagnosis and treatment.
However, comparative effectiveness research should be used to
inform medical decisions, not replace medical judgment with national
treatment formulas. It should enhance, not hinder independent
professional medical decision-making. Since comparative effectiveness
research looks at what is best on average for patients, protections
must be established to ensure patient access to treatments, drugs, and
devices that meet their individual needs.
Statement of Coalition for Health Services Research
The Coalition for Health Services Research (Coalition) is pleased
to offer this testimony for the record regarding the promise of
comparative effectiveness research. The Coalition's mission is to
support research that leads to accessible, affordable, high-quality
health care. As the advocacy arm of AcademyHealth, the Coalition
represents the interests of 3,800 researchers, scientists, and policy
experts, as well as 135 organizations that produce and use health
services research.
Health care in the United States has the potential to improve
people's health dramatically, but often falls short and costs too much.
Health services research is used throughout the health care field to
understand how to better finance the costs of care, measure and improve
the quality of care, and improve coverage and access to affordable
services. As an emerging science in the broader field of health
services research, comparative effective research--where
pharmaceuticals, medical devices and medical procedures used to treat
the same conditions are evaluated for their relative safety,
effectiveness, and cost--has great potential to improve health care
quality and patient outcomes while ensuring that consumers receive the
best care at the best value. When optimally funded, comparative
effectiveness research has the promise to inform health care decisions
that are:
Patient-specific, enabling doctors to make
individualized treatment decisions according to patient
characteristics (sex, age, and race/ethnicity).
Evidence-based, providing patients and practitioners
with the timely, scientific information they need to evaluate
which treatment options will help them achieve better outcomes.
Value-driven, empowering patients to make informed
decisions in the face of rising health care costs and myriad
treatment options.
There are increasing examples that demonstrate how comparative
effectiveness research provides the scientific basis needed to make
better decisions when it comes to the care we give and receive:
The Agency for Healthcare Research and Quality (AHRQ)
found that episiotomies--a preemptory incision intended to
prevent pregnant women from tearing tissue during labor--has no
positive benefit, and probably results in more complications
and causes more pain than if no incision was made during
childbirth.i The report will save millions of women
from having to undergo this painful procedure, not to mention
the costs saved by eliminating the routine use of this
procedure.
---------------------------------------------------------------------------
\i\ Viswanathan, M., et. al. ``The Use of Episiotomy in Obstetric
Care: A Systemic Review,'' Agency for Healthcare Research and Quality
(May 2004). Available on the Web at www.ahrq.gov/downloads/pub/
evidence/pdf/episiotomy/episob.pdf.
---------------------------------------------------------------------------
Another AHRQ study found that drugs can be as
effective as surgery in management of gastroesophageal reflux
disease (GERD)--where stomach acid enters the esophagus,
causing heartburn and potential esophageal damage.ii
GERD is one of the most common health conditions among older
Americans and results in $10 billion annually in direct health
care costs. Knowing that, for the majority of patients, drugs
can be as effective as surgery in relieving the symptoms could
result in significant health care savings and improved quality
of life.
---------------------------------------------------------------------------
\ii\ Ip, S., et. al. ``Comparative Effectiveness of Management
Strategies for Gastroesophageal Reflux Disease,'' Agency for Healthcare
Research and Quality (Dec. 2005). Available on the Web at
www.effectivehealthcare.ahrq.gov/reports/final.cfm.
---------------------------------------------------------------------------
The National Institute of Mental Health (NIMH) found
that, within a class of antipsychotic drugs, the older, less
expensive drug (Perphenazine) was just as effective and caused
no worse side effects than the three newer, more expensive
drugs in treating patients with schizophrenia. One of the newer
drugs (Zyprexa) was slightly more effective in controlling
systems than the other drugs, but at the cost of serious side
effects.iii This study enables greater flexibility
in care and informs patients and providers about costs and
quality of care.
---------------------------------------------------------------------------
\iii\ Lieberman, J.A., et. al. ``Effectiveness of Antipsychotic
Drugs in Patients with Chronic Schizophrenia, '' New England Journal of
Medicine, Vol. 353, No. 12, pp.1209-1223 (Sept. 22, 2005). Available on
the Web at http://content.nejm.org/cgi.content/abstract/353/12/1209.
---------------------------------------------------------------------------
In a study of more than 2,200 patients funded mostly
by the Veterans' Administration, researchers found that those
who underwent non-emergency angioplasty--a procedure where a
tiny wire-mesh tube called a stent is placed in an artery to
hold it open--were no less likely to suffer a heart attack or
die than those who took only aspirin and other medicines to
lower blood pressure and cholesterol and prevent clots, along
with adopting lifestyle changes.iv The procedure,
often performed to relieve chest pain and to reduce the risk of
having or dying from a heart attack, costs about $50,000 and
has become one of the most common medical procedures in the
United States.
---------------------------------------------------------------------------
\iv\ Boden, W.E., et. al. ``Optimal Medical Therapy with or without
PCI for Stable Coronary Disease,'' New England Journal of Medicine,
Vol. 356, No. 15, pp. 1503-1516 (April 12, 2007). Available on the Web
at http://content.nejm.org/cgi/content/abstract/356/15/1503.
As these examples suggest, comparative effectiveness research can
contribute greatly to better health care at lower cost. It is a true
public good, providing a basis for improvements in our health care
system that benefit the general public. Americans overwhelmingly agree.
According to a 2005 Research!America survey, approximately 95 percent
of Americas agree that it is important to support research that focuses
on how well the health care system works and how it could work better,
and that health care services should be based on the best and most
recent research available.v
---------------------------------------------------------------------------
\v\ Woolley, M. and S. Propst. ``Public Attitudes and Perceptions
about Health-Related Research.'' Journal of the American Medical
Association, Vol. 294, No. 11, p. 1382 (Sept. 21, 2005).
---------------------------------------------------------------------------
Despite the promise of, and general support for, comparative
effectiveness research, this type of health services research by
definition often results in ``winners'' and ``losers,'' making the
entity that commissions this research vulnerable and susceptible to
attack. For example, if research based on post-marketing surveillance
finds that device ``A'' has better outcomes and fewer risks than drug
``B,'' one would expect the demand for device ``A'' to increase at the
expense of drug ``B.'' The manufacturer of drug ``B'' might then
attempt to leverage the political process to discredit the research
and, as has happened in the past, exert political pressure to
substantially reduce the funding for, or even abolish the entity
funding, the research.
Given the potentially controversial nature of comparativeness
effectiveness research findings, in September 2005 AcademyHealth issued
a report that provided guidance on the placement, structure, and
funding of comparative effectiveness research (see appendix
A).vi The AcademyHealth report recommended that comparative
effectiveness research be established either within AHRQ or through the
creation of a new entity that would, in varying degrees, be linked the
lead agency for health services research. As part of this
recommendation, the report identifies four structural options for the
placement of this critical research function. These options range from
fully embedding the comparative effectiveness function in an
established federal agency to placing it, along with all other health
services research, in a new, quasi-governmental organization (see also
Appendix B):
---------------------------------------------------------------------------
\vi\ Placement, Coordination, and Funding of Health Services
Research within the Federal Government, AcademyHealth (Sept. 2005).
Available on the Web at http://www.chsr.org/placementreport.pdf.
Option 1: AHRQ sponsors and conducts comparative
effectiveness studies with oversight and guidance from an
---------------------------------------------------------------------------
external board and panel of experts.
Option 2: AHRQ sponsors and conducts comparative
effectiveness studies with oversight and guidance from an
external board and panel of experts, and establishes a
Federally Funded Research and Development Center
(FFRDC).vii The FFRDC would undertake syntheses of
research commissioned by AHRQ and others for the purpose of
making comparative effectiveness findings.
---------------------------------------------------------------------------
\vii\ An FFRDC is a private, nonprofit organization that is
sponsored by an executive branch agency. The sponsoring agency
monitors, funds, and assumes responsibility for the overall activities
of the FFRDC. While FFRDCs are not subject to federal personnel rules,
the organizations are prohibited from competing for government
contracts to ensure their independence, objectivity, and freedom from
organizational conflicts of interest.
Option 3: With AHRQ remaining as currently
structured, create a new, separate quasi-governmental entity
---------------------------------------------------------------------------
for comparative effectiveness research.
Option 4: Reconstitute AHRQ as a quasi-governmental
entity, retaining most of its existing functions and adding
comparative effectiveness research.
AcademyHealth assessed these four options against five principles
designed to further guide policymakers' deliberations on comparative
effectiveness research (see also Appendix C).
Comparative effectiveness research is a subset of the
broader field of health services research, so increased
investments in comparative effectiveness research should not be
at the expense of investments in a robust health services
research portfolio.
Given the potentially controversial nature of
comparative effectiveness findings, this research must be based
on scientific evidence and be kept separate from funding and
coverage decisions.
As a subset of the field of health services research,
comparative effectiveness research must be closely linked to
AHRQ--as the lead agency for health services research--to
ensure that findings are consistent with the best available
research, methods, and data.
Since comparative effectiveness research as a public
good requires significant federal investment and has the
potential to affect the delivery and cost of health care for
all Americans, the entity commissioning or conducting this
research should be subject to congressional oversight.
Stakeholders should be involved in developing the
research agenda and ensuring the validity of the research
produced. Ensuring transparency in the prioritization, conduct,
and dissemination of research will promote public acceptance of
the research findings and strengthen support for the program's
mission.
The entity's overall funding and ability to recruit the expertise
needed are critical factors that should inform the choice among these
options--the best arrangement for a budget of $50 million might not be
the best if $5 billion were to be made available for this function. It
may also be desirable to have portions of this responsibility
undertaken by a combination of entities. Under such a scenario, the
lead agency for health services research might commission and undertake
the research studies, an affiliated entity might do the assessments
based on that research, and an independent quasi-governmental entity
might develop consensus studies on the methods and data to be used for
these studies and assessments.
Regardless of how this research program is structured and governed
in the future, AcademyHealth and its Coalition recognize that
comparative effectiveness research will require a significant
investment to realize its potential. For example, some experts suggest
that a robust comparative effectiveness program should be funded at a
level of $4--$6 billion annually to meet the U.S. health system's
demands. Comparatively, the Federal Government last year spent nearly
$32 billion on health research, of which only 5 percent--about $1.5
billion--was apportioned to health services research. The Federal
Government's comprehensive investment in comparative effectiveness
research across the various agencies conducting and funding this work
is unknown, as this information is not systematically collected.
However, we do know that AHRQ's comparative effectiveness program was
appropriated $15 million in fiscal 2007 (and $15 million in each of the
previous three fiscal years).viii,}ix Congress should
increase and expand the sources of funding for conducting and
coordinating a wide spectrum of comparative effectiveness research,
including systematic reviews of existing literature, analysis of
administrative data and clinical registries, and pragmatic,
prospective, head-to-head trials. Doing so would ultimately help
patients, providers, payers, and policymakers make rational choices
about new and existing health services, and assure that our investments
in basic and clinical research are integrated into health care
delivery. After all, increased spending on new medicines and equipment
is wasted if the system does not adopt these new treatments in a safe
and efficient manner.
---------------------------------------------------------------------------
\viii\ Catlin, A., et. al. ``National Health Spending in 2005: The
Slowdown Continues,'' Health Affairs, Vol. 26, No. 1, pp. 142-153
(Jan./Feb. 2007).
\ix\ Federal Funding for Health Services Research, Coalition for
Health Services Research (Dec. 2006). Available on the Web at http://
www.chsr.org/AHfundingreport1206.pdf.
---------------------------------------------------------------------------
In addition, we believe that increased investment in comparative
effectiveness research must be coupled with greater investment in the
research infrastructure--the data, methods, and researchers needed to
conduct this work and ultimately generate meaningful research and
knowledge. The field of health services research has experienced an
erosion of investment in its methods, data, and particularly its
researchers over the last several years. If left unchecked, these
declining investments could threaten the field's capacity to address
public and private sector research needs.
In conclusion, the best health care decisions are based on relevant
data and scientific evidence. Increased investment in comparative
effectiveness research and the health services research infrastructure
will show returns in improved quality, accessibility, and
affordability. At a time when America is spending over $2 trillion
annually on health care, we need research--now more than ever--to help
us spend our health care dollars more wisely.
The Coalition appreciates the opportunity to submit this testimony
for the record and looks forward to working with the Subcommittee as it
continues to assess options for structuring and funding a robust
comparative effectiveness research capability in the United States. If
you have questions or comments about this testimony, please contact
Emily Rowe, Director of Government Relations.
Appendix A: Committee on Placement, Funding, and Coordination of Health
Services Research within the Federal Government
(Affiliations at time of committee appointment)
Sheila Burke, Committee Chair, Deputy Secretary and Chief Operating
Officer, Smithsonian Institution
Jeanne Lambrew, Ph.D., Vice Chair, Associate Professor, Department of
Health Policy, George Washington University
David Abernethy, Senior Vice President, Operations, HIP Health Plans
Michael Chernew, Ph.D., Professor, Department of Health Management and
Policy, School of Public Health, University of Michigan
Jordan Cohen, M.D., President, Association of American Medical Colleges
Judith Feder, Ph.D., Dean of Public Policy, Georgetown University
Harold S. Luft, Ph.D., Caldwell B. Esselstyn Professor and Director,
Institute for Health Policy Studies, University of California, San
Francisco
Nicole Lurie, M.D., Senior Natural Scientist and Alcoa Chair, RAND
Corporation
Donald M. Steinwachs, Ph.D., Professor and Chair, Department of Health
Policy and Management, Bloomberg School of Public Health, Johns Hopkins
University
Gail Wilensky, Ph.D., Senior Fellow, Project HOPE
Appendix B: Four Options for the Placement of
Comparative Effectivenss Research
------------------------------------------------------------------------
------------------------------------------------------------- -------------
Option 1: AHRQ sponsors and conducts AHRQ would
comparative effectiveness studies remain the lead agency
with oversight and guidance from an for health services
external board and panel of research, supporting a
experts. broad health services
research agenda,
including comparative
effectiveness.
AHRQ would
establish an external
board to oversee the
development of the
comparative
effectiveness research
agenda and a panel of
experts to validate
the science used to
conduct comparative
effectiveness studies.
------------------------------------------------------------------------
Option 2: AHRQ sponsors and conducts AHRQ would
comparative effectiveness studies remain the lead agency
with oversight and guidance from an for health services
external board and panel of research, supporting a
experts, and establishes a broad health services
Federally Funded Research and research agenda,
Development Center (FFRDC). including comparative
effectiveness.
AHRQ would
establish an external
board to oversee the
development of the
comparative
effectiveness research
agenda and a panel of
experts to validate
the science used to
conduct comparative
effectiveness studies.
AHRQ would
also establish an
independent FFRDC with
the limited mission of
reviewing and
synthesizing
comparative
effectiveness
research.
------------------------------------------------------------------------
Option 3: AHRQ remains as currently AHRQ would
structured and a new separate quasi- remain the lead agency
government entity is established to for health services
fund and conduct comparative research, supporting a
effectiveness research. broad health services
research agenda, but
not comparative
effectiveness.
A new quasi-
governmental agency
would be established,
with both public and
private funding, to
conduct both
intramural and
extramural comparative
effectiveness studies.
------------------------------------------------------------------------
Option 4: AHRQ is reconstituted as a AHRQ
quasi-governmental agency retaining reconstituted as a new
most existing functions and adding quasi-governmental
comparative effectiveness research. entity would conduct
and fund health
services research,
including comparative
effectiveness.
Those AHRQ
functions that must be
performed by a
governmental entity,
such as the Medical
Expenditure Panel
Survey (MEPS), would
be transferred to
other existing HHS
agencies.
The new quasi-
governmental entity
could be supported by
public and private
funds.
------------------------------------------------------------------------
Appendix C: Five Principles to Guide Decisions for the Placement of
Comparative Effectiveness Research
------------------------------------------------------------------------
------------------------------------------------------------- -------------
Principle 1: Overall funding for the Recognizes
field of health services research that while comparative
should continue to support a broad effectiveness research
and comprehensive range of topics. is important, it is a
subset of the broader
field of health
services research.
Regardless of
where comparative
effectiveness research
is placed, this
principle stresses the
need to fund a broad
health services
research portfolio.
------------------------------------------------------------------------
Principle 2: Assessments should be Given the
based on scientific evidence and controversial nature
kept separate from funding and of comparativeness
coverage decisions. effectiveness
findings, this
principle stresses the
need for a structure
that ensures the
scientific integrity
of comparative
effectiveness
research.
This principle
stresses the need to
separate the entity
that funds and
conducts these studies
from the entity
directly responsible
for making coverage
decisions.
------------------------------------------------------------------------
Principle 3: Entity commissioning or Recognizes
conducting comparative that comparative
effectiveness research should effectiveness research
maintain close linkage to the lead is a subset of the
agency for health services broader field of
research. health services
research.
As such,
comparative
effectiveness research
must be closely linked
to the lead agency in
order to ensure that
findings are
consistent with the
best available
research, methods, and
data.
------------------------------------------------------------------------
Principle 4: Entity commissioning or Since
conducting comparative comparative
effectiveness research should be effectiveness research
subject to congressional oversight. has the potential to
affect the delivery
and cost of health
care for all
Americans, this
principle recognizes
that the Federal
Government is
responsible for
ensuring that
decisions about what
health services and
products should be
provided are based on
sound scientific
research.
Since this
research requires
substantial federal
funding (and would not
be funded adequately
by the private sector
alone), this principle
recognizes the need
for appropriate
congressional
oversight of public
funding to ensure
accountability.
------------------------------------------------------------------------
Principle 5: Entity commissioning or Given the
conducting comparative controversial nature
effectiveness research should of comparative
involve key stakeholders to assure effectiveness
transparency of the methods and research, this
process, promote public acceptance principle recognizes
of research findings, and support the importance of
for the entity's mission. involving key private
sector representatives
in developing the
research agenda and
ensuring the validity
of the research
produced, thereby
increasing public
support for the
research findings and
the entity's mission.
As such,
comparative
effectiveness research
must be funded in an
open process to ensure
that no one group is
perceived as
dominating the process
and/or skewing the
results.
------------------------------------------------------------------------
Epilepsy Foundation
Landover, Maryland 20785
July 9, 2007
Chairman Pete Stark
House Ways and Means Health Subcommittee
1135 Longworth House Office Building
Washington, DC 20515
Dear Chairman Stark,
On behalf of the Epilepsy Foundation and the more than 3 million
Americans living with epilepsy, I am pleased to submit the following
comments in response to the Health Subcommittees' June 12, 2007 hearing
on Strategies to Increase Information on Comparative Clinical
Effectiveness.
The Epilepsy Foundation applauds the Committee's intent to address
the issue of rising health care costs and the lack of evidence
justifying extreme variations in the provision of medical services. We
recognize many of the potential benefits to increasing, and making
available, information on the relative effectiveness of health care
services.
Yet we are also concerned that this information not be based solely
on the limited perspective of random clinical trials and that the
application of Comparative Clinical Effectiveness not be based upon, or
utilized, for cost containment benefits only.
Epilepsy is a chronic neurological condition characterized by
recurrent seizures. Individuals with epilepsy are at a two--to three-
fold increased risk of death, as well as an increased risk of cognitive
impairments, employment difficulties and personal isolation (due in
part to the social stigmas associated with this
condition).i,}ii,}iii,}iv,}v,}vi,}vii,}viii Each year,
approximately 200,000 individuals in the United States are newly-
diagnosed with epilepsy.ix Although initial onset can occur
at any age, epilepsy most commonly arises in either early childhood or
old age.x
---------------------------------------------------------------------------
\i\ Cockerell OC, Johnson AL, Sander JW, Shorvon SD. Prognosis of
Epilepsy: A Review and Further Analysis of the First Nine Years of the
British National General Practice Study of Epilepsy, a Prospective
Population-Based Study. Epilepsia. 1997; 38(1):31-46.
\ii\ Hauser WA, Annegers JF, Elveback LR. Mortality in patients
with epilepsy. Epilepsia. 1980;21:399-412.
\iii\ Olafsson E, Hauser WA, Gudmundsson G. Long-Term Survival of
People with Unprovoked Seizures: A Population Based Study. Epilepsia.
1998;39:89-92.
\iv\ Zielinski JJ. Epilepsy and Mortality Rate and Causes of Death.
Epilepsia. 1974;15:191-201.
\v\ Nilsson L, Tomson T, Farahmand BY, Diwan V, Persson PG Cause-
Specific Mortality in Epilepsy: A Cohort Study of More Than 9,000
Patients Once Hospitalized for Epilepsy. Epilepsia. 1997;38(10):1062-8.
\vi\ Motamedi G, Meador K. Epilepsy and cognition. Epilpesy Behav.
2003;4(Suppl. 2):25-38.
\vii\ Fisher RS, Vickrey BG, Gibson P, Hermann B, Penovich P,
Scherer A, Walker S. The impact of epilepsy from the patient's
perspective I. Descriptions and subjective perceptions. Epilepsy Res.
2000;41:39-51.
\viii\ Sillanpaa M, Jalava M, Kaleva O, Shinnar S. Longer-Term
Prognosis of Seizures with Onset in Childhood. N. Engl. J. Med.
1998;338:1715-22.
\ix\ Epilepsy Foundation. Epilepsy and Seizure Statistics.
Available at: http://www.epilepsyfoundation.org/about/. Accessed on
April 19, 2007.
\x\ Centers for Disease Control and Prevention. Living Well with
Epilepsy, Report of the 2003 National Conference on Public Health and
Epilepsy. Available at www.cdc.gov/Epilepsy/pdfs/living_well_2003.pdf.
Accessed on January 10, 2007.
---------------------------------------------------------------------------
Modern treatment of epilepsy relies primarily on the use of one or
more antiepileptic drugs (AEDs) with the goal of preventing
seizures.xi Other treatment options may include surgery,
special diet and/or the use of a device to stimulate the vagus nerve.
---------------------------------------------------------------------------
\xi\ Sander JW. The Use of Antiepileptic Drugs--Principles and
Practice. Epilepsia. 2004;45(Suppl. 6):28-34.
---------------------------------------------------------------------------
Complete prevention of seizures remains elusive in approximately
one in three patients with epilepsy. In these patients, the goal of
treatment is to minimize the frequency and intensity of the seizures
without unacceptable side effects from treatment. Epilepsy, and
treatment to control seizures, is not a one size fits all condition. To
manage patients with epilepsy effectively, patients need access to the
full range of treatment options and physicians must tailor treatment
for each patient to achieve maximum control of seizures while
minimizing adverse side effects.xii,}xiii,}xiv,}xv,}xvi
---------------------------------------------------------------------------
\xii\ EUCARE. European white paper on epilepsy: Pharmacological
Treatment. Epilepsia 2003;44(Suppl. 6):33-4.
\xiii\ Stokes T, Shaw EJ, Juarez-GA, Camosso-Stefinovic J, Baker R.
Clinical Guidelines and Evidence Review for the Epilepsies: diagnosis
and management in adults and children in primary and secondary care.
Royal College of General Practitioners, London England. 2004. Link
available at: http://www.nice.org.uk/guidance/CG20. Accessed on January
23, 2007.
\xiv\ Glauser T, Ben-Menachem E, Bourgeois B, Cnaan A, Chadwick D,
Guerreiro C, Kalviainen R, Mattson R, Perucca E, Tomson T. ILAE
Treatment Guidelines: Evidence-based Analysis of Antiepileptic Drug
Efficacy and Effectiveness as Initial Monotherapy for Epileptic
Seizures and Syndromes. Epilepsi. 2006;47(7);1094-1120.
\xv\ French JA, Kanner AM, Bautista J, Abou-Khalil B, Browne T,
Harden CL, Theodore WH, Bazil C, Stern J, Schachter SC, Bergen D, Hirtz
D, Montouris GD, Nespeca M, Gidal B, Marks Jr. WJ, Turk WR, Fischer JH.
Bourgeois B, Wilner A, Faught Jr. RE, Sachdeo RC, Beydoun A, Glauser
TA. Efficacy and tolerability of the new antiepileptic drugs I:
Treatment of new onset epilepsy: Report of the Therapeutics and
Technology Assessment Subcommittee and Quality Standards Subcommittee
of the American Academy of Neurology and the American Epilepsy Society.
Neurology. 2004;62:1252-60.
\xvi\ French JA, Kanner AM, Bautista J, Abou-Khalil B, Browne T,
Harden CL, Theodore WH, Bazil C, Stern J, Schachter SC, Bergen D, Hirtz
D, Montouris GD, Nespeca M, Gidal B, Marks Jr. WJ, Turk WR, Fischer JH.
Bourgeois B, Wilner A, Faught Jr. RE, Sachdeo RC, Beydoun A, Glauser
TA. Efficacy and tolerability of the new antiepileptic drugs II:
Treatment of refractory epilepsy: Report of the Therapeutics and
Technology Assessment Subcommittee and Quality Standards Subcommittee
of the American Academy of Neurology and the American Epilepsy Society.
Neurology. 2004;62:1261-73
---------------------------------------------------------------------------
As the discussion of Comparative Clinical Effectiveness moves
forward the Foundation encourages that, throughout the process, two key
principles be applied.
1. Evidence being considered and applied should be broad and inclusive.
While a comprehensive, and inclusive, approach to comparative
clinical effectiveness and, in turn, evidence based healthcare can
indeed lead to high quality healthcare and maximize patient outcomes, a
limited approach, we fear, would limit access and, in turn, not offer
people with epilepsy, and others living with chronic disorders, the
optimal care that is available.
To assure a comprehensive approach to the topic of comparative
clinical effectiveness the Epilepsy Foundation supports a model that
encompasses a broad definition and application of what is accepted as
``evidence''. The Foundation feels strongly that treatment selection be
based on a combination of scientific research, physician expertise and
experience, and the patient's preferred outcomes, preferences and
expectations.
We are fearful that a ``one-size-fits-all'' approach to treatment
can result from strict reliance on ``evidence'' solely from random
clinical trails and published studies that do not take into account
diverse populations, co-morbidities, and other real world situations.
12. The process should be transparent and include consumer/patient
involvement.
As decisions are made as to the structure, funding, and utilization
of a comparative effectiveness entity we encourage that consumers/
patients be considered a primary stakeholder and participant at every
level.
We anticipate transparency and public comment periods will be
included throughout the process and that patients/consumers will be
encouraged to provide input. The Foundation would like to see that
outreach to, and participation by, consumers/patients will encourage
and empower them to be involved in research design, review and
translation, dissemination, implementation and evaluation. Undoubtedly,
government, researchers, funders, industry and payors will all play a
critical role. Each entity should strive to include the patient
perspective and involvement in their work on this issue.
Recognizing the importance of the patient perspective in this on-
going discussion, in early 2006 the Epilepsy Foundation became a
founding member of the National Working Group on Evidence-Based Health
Care. This group, comprised of consumers, caregivers, practitioners and
researchers, is committed to promoting accurate and appropriate
evidence-based policies. Information about this group, and our work,
can be found at www.evidencebasedhealthcare.org.
Again, the Epilepsy Foundation is grateful for the opportunity to
provide these comments to the dialogue on comparative clinical
effectiveness and welcomes the opportunity to be a continuing resource
and participant in this evolving issue. For additional information, or
if we can be of further assistance, please feel free to contact me.
Sincerely,
Donna Meltzer
Senior Director of Government Relations
Statement of Mental Heath America
Mental Health America (formerly the National Mental Health
Association) is the country's leading nonprofit dedicated to helping
ALL people live mentally healthier lives. With our more than 320
affiliates nationwide, we represent a growing movement of Americans who
promote mental wellness for the health and well-being of the nation--
everyday and in times of crisis.
We applaud the Subcommittee on Health, Committee on Ways and Means
of U.S. House of Representatives for holding a hearing to increase
information on Comparative Clinical Effectiveness on June 12, 2007. We
are encouraged by the new proposals to expand Comparative Clinical
Effectiveness efforts and improve the overall quality and value of
health care delivery in our country.
Mental Health America has followed the current national healthcare
reform debate and witnessed the balancing act between access to safe
and effective health care, cost and quality. In this environment of
steadily rising costs, Mental Health America is concerned that the
evaluation of ``quality'' of care is being used as a means of
justifying cost-based decisions about the types and quantity of health
services available. At every level of the debate--federal, local,
public and private--the stakes are high for how this will impact access
to care, particularly for vulnerable Americans who are reliant on
public systems to get basic care for chronic health conditions, such as
mental illnesses. Mental Health America is particularly concerned with
how these trends will impact the Medicaid program as well as the public
mental health system in this country.
As these deliberations continue to unfold, Mental Health America
offers the following comments for the Committee's consideration:
Promoting patient/consumer inclusion: The current comparative
clinical effectiveness proposals seek to influence and reform
health care delivery in the best interest of the patient.
However, these efforts often lack a balanced representation of
the very stakeholders for whom these decision will be most
important, patients/consumers. Decision makers must recognize
the importance of including patients/consumers as partners at
every stage of comparative clinical effectiveness efforts
including: research design, review, evaluation and governing
bodies that make decisions about how to apply evidence in
practice and policy. The process to evaluate and develop
evidence-based interventions, treatments and policies should be
transparent and open to the public and include all stakeholders
in the decision making process. Consumers and families should
have active and meaningful roles on review, evaluation and
governing bodies that make decisions about how to apply
evidence in practice and policy. In addition, clear and
complete research findings should be communicated to consumers
and families, and tools and practices should be developed to
aid consumers in how to use such information in their dialogue
with caregivers.
Preserving access to individualized care: Scientific and
practical evidence can aid in determining what is the best
intervention to optimize an individual's care, but such
evidence alone cannot guarantee the right choice for every
individual. Therefore, it is important that reimbursement and
coverage policies reflect the need for individualized care and
maintain flexibility for clinicians and individuals to access a
range of treatments and services. In addition, it is important
to advocate for wider investment in practical clinical trials
and other research methods that generate evidence applicable to
real-world treatment settings.
Promoting quality health care first: The underlying
motivation of these initiatives is cost containment rather than
improving quality of care. Safety and optimal treatment for the
individual should be the overriding goal of any comparative
clinical effectiveness approach. Treatment costs are relevant
to the discussion but must be weighed in the system context--
total care costs for an individual across services and
settings--rather than as unit costs. In the context of clinical
decision making, cost should be evaluated by providers and
consumers after a careful weighing and discussion of benefits
and risks and a dialogue that emphasizes choice across a range
of therapeutic options. Mental Health America opposes processes
and policies that emanate solely from cost containment
objectives. Moreover, our organization opposes the misuse of
the concept of evidence or the findings from comparative
clinical effectiveness initiatives as a justification for
denial of coverage, reimbursement or access to care except in
areas where significant safety concerns are identified.
Identifying and addressing gaps in research: The boundaries
of scientific research are stretching and revealing new
understanding and options for treating many chronic illnesses,
including mental health conditions. Even as emerging science
gives us information about how and why mental illnesses affect
individuals, and about genetic biomarkers that may better guide
treatment choices, it also reveals the absence of universally
effective treatments and practices and the limitations to the
current body of scientific evidence in mental health. We
believe there are limitations of clinical research design--
particularly the ``gold standard'' of randomized controlled
trials--which do not effectively measure important outcomes,
such as quality of life, employment, relationships, and the
impact of side effects of different treatments. Realities of
the individual consumer (age, gender, ethnicity, co-occurring
disorders, and treatment goals and preferences) often are not
captured as part of this research. Mental Health America
supports the role of all levels of evidence--including
randomized clinical trials, quasi-experimental studies,
observational studies and expert consensus--in creating the
evidence base for an intervention or service.
Our abovementioned comments mirror those to which we have submitted
to Oregon's Drug Effectiveness Review Project and the Agency for
Healthcare Research and Quality's Effective Health Care program.
Attached are two documents that Mental Health America submitted to
Oregon's Drug Effectiveness Review Project which include comments to
their reports on atypical antipsychotics and second generation
antidepressants. These documents illustrate in further detail the
concerns we have in using comparative clinical effectiveness approaches
and caution state leaders regarding the limitations of applying this
information to public policy decision making.
We hope you take into consideration our views and thank you again
for this opportunity to comment.
__________
Attachment A
NMHA Comments on the
Draft Drug Class Review for Atypical Antipsychotic Drugs--Update 1
March 14, 2006
Thank you for the opportunity to respond to the draft drug class
review on pharmacologic treatments for atypical antipsychotic (AAP)
medications. We appreciate that you have continued to update and review
your conclusions. We commend DERP for its inclusion of the CATIE study
in this review and we are most pleased to see that you chose to include
information about limitations of the research and addressed some of our
previous concerns.
However, the review still highlights the desperate need for better
research on both the efficacy and the effectiveness of these
medications and points to the ongoing necessity for caution when states
and private companies develop pharmacy policies based on limited
information. On page 13, the review acknowledges that ``quality of the
evidence on effectiveness is a key component, but not the only
component, in making decisions about clinical policies.'' NMHA
continues to encourage the Oregon Center to communicate to its
participating agencies and on its website that the Drug Effectiveness
Review Project needs to be used within a larger context of policy
decision-making. Below NMHA has additional questions and comments about
the review.
Overall Inadequate Research
As with previous reviews of atypical antipsychotic medications,
this review found that the research is generally inadequate to draw
appropriate conclusions about which atypical antipsychotic medications
work for different individuals. Comments from the CATIE principle
investigator Dr. Jeffrey Lieberman reinforce this notion. During the
National Institutes of Mental Health (NIMH) briefing on September 26,
2005, Dr. Lieberman stated, ``The outcomes show that Schizophrenia
patient choices must be individualized. What works for one individual
may not work for another.''
Furthermore, the CATIE data is presently incomplete. Only Phase 1
data has been released. Further analysis and more detailed evaluation
of study findings are forthcoming. It is important to note that CATIE
study researchers and NIMH Director Dr. Thomas Insel have said that it
would be counterproductive for anyone to use this data to reduce access
and that it would be ``premature to change public policy on the basis
of this study.''
This lack of data makes it very difficult for policymakers,
researchers, and others to draw any conclusions about the comparative
effectiveness of these medications. We strongly urge DERP to clarify in
its review that a general lack of evidence about differences does not
mean that there are no differences between the medications.
On page 18, the DERP review reports that only 3 effectiveness
trials were identified and reviewed for comparative effectiveness and
short term adverse events of AAPs in patients with Schizophrenia. Yet
in the review, it states that the results of effectiveness studies are
more applicable to the ``average'' patient than results from highly
selected populations in efficacy studies. The review states, ``The
remainder of the direct evidence comes from efficacy trails, which
include narrowly defined patient populations, and are not conducted
within the context of a care system with the typical range of co-
interventions and/or co-morbidities, and a small number of studies with
observational designs. The generalizability of the findings of the
efficacy studies to broader groups of patients and setting is
limited.'' We are very concerned by this finding, especially because it
limits stakeholders' abilities to use the review for policymaking
decisions. We encourage DERP to make a call for more research within
the review and to offer reasons why this lack of data is important for
policymakers.
In addition, on page 20, the DERP review states, ``There is very
limited evidence regarding AAPs used for the treatment of schizophrenia
in subgroup populations.'' We are very concerned about the lack of
research focused on differences in responses by people of different
genders, race and ethnicity. Again, we encourage DERP to use this
review to call for more research in this area and to look at other
sources of information that addresses this important issue.
Adverse Events
We noted the review did find that patients appeared to have
significant differences in tolerability of side effects and other
adverse events. This is a critically important area, and is believed to
be strongly linked with patients continuing with certain medications.
We strongly recommend highlighting this finding in the introduction as
well as conclusion of the report.
Finally, the information organized is one piece in a complex puzzle
of making healthcare decisions. As state policymakers face difficult
choices in an environment of increasing healthcare costs, we are
concerned that this information will become political cover for
establishing policies that will harm the health of vulnerable citizens.
We recommend that the following guidance be shared with participating
agencies and be featured prominently in the reviews and in your cover
letters:
Please note that the information organized in the
drug class reviews is an important part of making decisions
about the effectiveness of this class of medications, but it
should not be the sole source of making such decisions. The
Oregon Center recommends that treatment guidelines, clinical
experience, and consumer input be also included in any pharmacy
management programs.
The Oregon Center urges participating agencies to
work with consumer and provider groups to assess what
medications work best in clinical practice, and to ensure that
adequate choices are available in participating agency's
pharmacy programs. All pharmacy policies should be voluntary to
provide flexibility to the physician and the patient.
All pharmacy policies should contain several choices
of medications to accommodate different responses to
medications, including different adverse responses or differing
responses based on gender, race, or ethnicity.
__________
Attachment B
NMHA Comments on the Draft Drug Class Review for Second Generation
Antidepressants
July 19, 2006
On behalf of the National Mental Health Association (NMHA), thank
you for the opportunity to respond to your draft drug class review on
pharmacologic treatments for second generation antidepressants.
We applaud the Drug Effectiveness Review Project (DERP) for
acknowledging on page 5 that, ``The tremendous volume and large
variability in the quality of evidence to support use of these products
makes it difficult for clinicians and decision makers to make evidence-
based decisions.'' This acknowledgment supports our concern of the
limitations of the scope and power of existing scientific research.
Below NMHA has additional comments about the review.
Effectiveness studies are lacking
We note that the report comments on the limited number of studies
that review effectiveness of the compared medications. While we
recognize this general weakness in scientific research, we are
surprised that DERP then concludes that it can comment on the
comparative effectiveness of these medications. Based on the fact that
an inadequate sample of such studies is presented in the report, we
urge DERP to comment prominently that such conclusions cannot be drawn
based on the available evidence.
Furthermore, of particular concern is the dearth of research on
quality of life measures as stated on page 16, ``Quality of life and
functional capacity were rarely assessed, and if they were, they were
considered only as a second outcome.'' These measures greatly impact a
patient's satisfaction with and adherence to a particular medication
and should be weighed equally to measures of efficacy and symptom
reduction. By including these measures, patients, physicians, payers
and decision makers will receive a more accurate picture of how
effective or ineffective a particular medication is.
Limited data on subgroups
The conclusions that were drawn from this report are generalized to
subgroups which were not included in many of the studies that were
reviewed. For example on page 28, the review states, ``We did not find
any evidence that one group has a greater benefit from an individual
drug than another.'' Yet we noticed that very little research focused
on differences in responses by people of different genders or racial
and ethnic minorities and for people with co morbid health conditions.
Furthermore, the report acknowledges on page 17 that, ``Most
studies received a fair rating for internal validity. The
generalizability of the results was hard to determine and might often
be limited.''
While we appreciate DERP's recognition of this issue, these
conclusions affirm what we know exists in the scientific research
today. Given this lack of data, however, we call on DERP to prominently
note that conclusions about response to second generation
antidepressants among subgroups is ``inconclusive'' rather than the
current conclusion that evidence shows ``no differences.'' Again, this
clarification will highlight that the evidence base is insufficient to
allow conclusions to be drawn that emphasize the need for
individualized care choices.
The information about scientific evidence that DERP presents in
this report is one piece in a complex puzzle of informing and improving
healthcare decisions. While NMHA is committed to promoting high quality
mental health care that is informed by the best scientific evidence
available, we continue to be concerned at the local application of
reports similar to DERP's as a rationale for limiting choice of care to
one or two agents, or requiring that individual's fail on the preferred
(cheapest) medication before being allowed to choose from more
(possibly) therapeutically appropriate choices.
As DERP prepares the final report, we urge you to
feature prominently the following key messages:
Clarify that the information organized in this
report is one component in making decisions about the
effectiveness of second-generation antidepressants; it
should not be the sole source of making such decisions
on a clinical or policymaking level.
Urge private and public policymakers and payers
to work with consumer and provider groups to
incorporate scientific evidence with knowledge from
clinical practice and from patient viewpoints and
values.
Make prominent the message that evidence that no
significant differences exist within this class of medications
does NOT imply that:
All medications are identical
None of the medications are efficacious in
treating depression.
Call for more research in primary care
populations, for subgroups, and for effectiveness
research for treatment of depression that includes
measure of functionality and patient preference.
Clearly state that current evidence on second-
generation antidepressants does not provide clear
justification for policies that limit choice of
medication because existing evidence affirms that
different therapies are efficacious for different
individuals.
NMHA continues to encourage the Oregon Center to communicate with
its participating agencies and on its website that the DERP needs to be
used within a larger context of policy decision-making.
If you have any questions, please contact Jennifer Bright, Vice
President for state policy.
Statement of the National Alliance on Mental Illness, Arlington,
Virginia
Chairman Stark and Congressman Camp, on behalf of the National
Alliance on Mental Illness (NAMI) I am pleased to offer the following
statement on strategies for increasing information comparative clinical
effectiveness. As the nation's largest organization representing people
with serious mental illness and their families--210,000 members and
1,200 affiliates in all 50 states--I am pleased to offer our views on
this important issue.
NAMI feels strongly that comparative effectiveness research can
serve as an important source of information that contributes to both
medical care decision-making between patients and providers, as well as
decisions about coverage and reimbursement. In addition to such
research, expert clinical guideline development, review of patient
registries and existing claims and utilization data, health services
research, disease management strategies, and e-health initiatives all
have a role in supporting better real-time decisions with a focus on
the individual patient/consumer.
NAMI is a member of the National Working Group on Evidence-Based
Healthcare, a coalition of patient and chronic disease advocacy
organizations that is working to ensure that comparative effectiveness
research plays the most appropriate role in our health care system.
NAMI concurs that the focus of comparative effectiveness research must
be on health conditions and chronic illness broadly, rather than
narrowly focused on particular healthcare technology. This will allow a
more useful analysis of all available approaches and a comparison of
risks and benefits that will be most relevant and useful to the end
user--patients and their families. It is also important that updates of
such research must be frequent to ensure that new evidence is rapidly
disseminated to clinicians and patients/consumers.
NAMI would like to commend the Agency for Healthcare Research and
Quality (AHRQ) for its pursuit of transparent and inclusive processes
to prioritize, conduct and disseminate the findings from its systematic
reviews in the Effective Healthcare program. NAMI, along with many of
our colleagues in the National Working Group on Evidence-Based
Healthcare, have participated in public forums, offered comments on
priority setting, key questions for the reviews, and draft reviews and
participated in review of dissemination materials from the Eisenberg
Center. These interactions have been positive and we have seen efforts
to incorporate recommendations and concerns into final products.
It should be noted however that this positive experience with AHRQ
is in contrast to NAMI's experience with other organizations involved
in such work, such as the Drug Effectiveness Review Project (DERP) and
Consumers' Union. While the DERP's processes have recently improved,
the organization has been resistant to wider transparency and inclusive
processes that incorporates meaningful input from patient advocacy
groups. This has been coupled with an absence of focus on methods of
dissemination that promote dialogue with all stakeholders to ensure a
balanced consideration of issues related to implementation in policy
decision-making.
DERP reviews in turn have been used to inform the work of
Consumers' Union's Best Buy Drugs program, which seeks to inform
consumers--principally by their own definition consumers without health
coverage or with high pharmaceutical costs--about both comparative
clinical effectiveness and cost-effectiveness. The Consumers' Union
program too often portrays choices for consumers in an overly-
simplistic and potentially misleading way. While there are disclaimers
on the site that consumers should not discontinue medications and that
individual's response to medications will vary, the notion of
identifying ``best buys'' leads to a conclusion that there is a choice
that will fit the majority of the audience.
In fact, there is overwhelming evidence that in the case of
medications to treat mental illness, individual response to medications
will vary based on age, gender, race/ethnicity, health status, co-
occurring health conditions--details which are absent from the Best Buy
literature. Further, NAMI is extremely concerned about the lack of
inclusion of chronic disease and patient advocacy organizations in the
development of Best Buy products. Such exclusion--even omitting public
comment periods that could ensure relevance to their audience--is
misguided in an environment where all stakeholder opinions must given
consideration.
As with our colleagues in the National Working Group on Evidence-
Based Healthcare, NAMI offers no specific recommendation on where an
entity conducting comparative effectiveness research should be placed,
we do urge policymakers to avoid seeking to reinvent processes or
organizational entities. Instead, it is important to emphasize the need
for an inclusive and broadly-representative partnership between
government, private entities, providers, as well as patients and their
families.
NAMI also has concerns about any entity that places payor and
private-stakeholder entities in leadership roles without adequate
balance from clinical and chronic disease and patient representation.
It is critical that the voice of Americans living with chronic diseases
and their families have meaningful and varied representation on
government bodies, advisory groups and other mechanisms to assure a
diverse voice. This is in contrast to current bodies organized around
evidence-based medicine in which a select few organizations are
representing the global consumer perspective.
NAMI shares the views of the National Working Group on Evidence-
Based Healthcare Working Group in urging that cost-effectiveness NOT be
part of any review of comparative clinical effectiveness. The
consideration of cost factors can sometimes be important to inform
clinical and consumer decision-making. However, this analysis of cost-
effectiveness must be separate and subsequent to analysis of clinical
value. This is a necessary separation to emphasize the focus on quality
of healthcare rather than cost first. Cost-effectiveness analysis is
even more complex and controversial in terms of reflecting stakeholder
values, thus potentially further politicizing any conduct of
comparative effectiveness analysis.
NAMI agrees strongly that there is tremendous risk in comparative
effectiveness research being used as a blunt policy instrument for cost
control. It is troubling that groups as respected as the Congressional
Budget Office (CBO) have articulated the misguided notion that such
research can provide a simplistic and objective decision about the best
choices of therapeutic interventions for every individual. Restrictions
on patient and clinician choice of therapies in state Medicaid programs
and in Medicare Part D drug plans demonstrate the potential for
inappropriate application of comparative effectiveness research to
coverage decisions, particularly for high-cost chronic health
conditions. These approaches fail to recognize and support
individualized care decisions and imply ``population based'' solutions
to complex individual health conditions.
NAMI shares the view of the National Working Group on Evidence-
Based Healthcare that comparative effectiveness research is only as
good as the measures and populations that are included in the research
being compared and synthesized. Its applicability to a specific
individual or a particular circumstance may be significantly limited.
Within a cost containment framework, studies that demonstrate similar
average outcomes on a limited number of targets can provide a rationale
for limiting treatment choices. In reality, differences between people
in their response to treatment and multifaceted outcomes that often
accompany the management of chronic illnesses confound this logic.
NAMI is encouraged that you and your colleagues in Congress,
including Representative Tom Allen, are open to suggestions about how
to ensure that HR 1184 incorporates protections to ensure patients and
consumers have access to all options for care. We share his view that
comparative effectiveness research be an informant rather than a driver
of healthcare decisions. Full participation of patients and families in
every aspect of comparative effectiveness research is critically
important and consistent with the historical tenets of evidence-based
medicine that balance research, clinical expertise with patient
perspectives and preferences.
Thank you for the opportunity to share NAMI's views on this
important issue.
Statement of Pharmaceutical Research and Manufactures of America
The Pharmaceutical Research and Manufacturers of America (PhRMA)
appreciates the opportunity to comment on comparative effectiveness
research and its role in improving the health care patients receive.
PhRMA supports the development and use of high quality evidence,
including comparative effectiveness evidence, for health care decision-
making. Development of high quality evidence can support physicians'
and patients' treatment decisions, consumers' decisions about health
plans and benefit designs, and health plans' policy decisions.
Proposals for expanding government-supported comparative effectiveness
research should be structured to promote better patient health rather
than to deny or delay patients' access to beneficial care, as occurs in
Europe and Australia.
PhRMA has previously issued principles on evidence-based medicine
and health outcomes research (attached), which encompass comparative
effectiveness research. Consistent with these principles, programs for
government-supported research should:
Recognize and support the central role of the
physician and patient in treatment decision-making;
Provide information to support good decision-making;
government or quasi-governmental organizations supporting
comparative effectiveness research should not make coverage
recommendations or decisions;
Improve quality of patient care by identifying and
supporting approaches to making better use of the evidence we
already have about what works in health care;
Support research to close evidence gaps across the
health care system, including care management, health benefit
and delivery designs, and the full range of treatments;
Encourage pluralistic approaches that provide for
multiple organizations to generate and evaluate evidence in
patient-centered, clinically sensitive ways;
Reflect emerging use of genomic, health information
technology, and other advances (``personalized medicine'') to
tailor treatment decisions;
Draw on a full range of evidence, including evidence
on patient reported outcomes and consider both direct benefits
and broader indirect benefits that are important to society,
such as quality of life, patient functionality and economic
productivity;
Utilize open, transparent, patient-centered processes
for setting research priorities, conducting research, and
applying and communicating findings; and
Ensure effective, balanced communication of results,
including disclosure of the limitations of the findings.
PhRMA recognizes the value of expanding the amount of available
evidence for health care decision-making. We also believe research on
how to make better use of the evidence we already have about what works
in health care needs to be an equally prominent part of any new
effort's agenda. This latter type of work likely has the greatest
potential to improve both outcomes and efficiency. For instance:
A National Institutes of Health official has pointed
out that if all heart patients were treated according to
current guidelines, heart disease would no longer be the
nation's number one killer.\1\
---------------------------------------------------------------------------
\1\ Statement by Dr. Claude Lenfant, Director National Heart, Blood
and Lung Institute. Dateline NBC. 21 May 2001.
---------------------------------------------------------------------------
Physician organizations have pointed out that there
are treatments known to effectively control diabetes, yet many
patients do not receive them.\2\
---------------------------------------------------------------------------
\2\ Agency for Health Care Quality and Research. Closing the
Quality Gap: Diabetes Care Strategies. April 2004.
---------------------------------------------------------------------------
A 2007 study in the journal Health Affairs estimates
that if all patients with hypertension were treated to
guideline, 89,000 premature deaths and 420,000 hospitalizations
could be avoided annually--in addition to the 86,000 premature
deaths and 8333,000 hospitalizations for heart attack and
stroke already avoided.\3\
---------------------------------------------------------------------------
\3\ D. Cutler, et al. The Value Of Antihypertensive Drugs: A
Perspective On Medical Innovation Health Affairs, January/February
2007; 26(1): 97-110.
Research that determines how to close these and many other known
gaps between what we already know and the care patients receive is a
priority that needs to be included in any new health research
initiative.
As part of this integrated approach, programs for comparative
effectiveness research should include research to develop comparative
evidence on care management approaches and benefit designs that ensure
delivery of high quality care, rather than being limited to treatments
and services. This broad agenda is defined in current statute (Sec.
1013 of the Medicare Modernization Act) but has yet to be fully
implemented. The importance of pursuing this portion of the agenda is
evident in recent reports of forward-looking employers who have
achieved better health outcomes and savings by modifying their health
benefit designs in ways that promote access to treatment for several
chronic conditions, rather than by restricting access.\4\ Likewise,
academic studies have pointed to the importance of benefit design in
determining health outcomes and costs.\5\
---------------------------------------------------------------------------
\4\ Milt Freudenheim. To Save Later, Employers Offer Free Drugs
Now, The New York Times, 21 February 2007.
\5\ A. Chandra, Harvard University, et al., ``Patient Cost-Sharing,
Hospitalization Offsets, and the Design of Optimal Health Insurance for
the Elderly,'' NBER Working Paper Series, Working Paper 12972, March
2007.
---------------------------------------------------------------------------
In addition, as the Committee considers expanding the government's
role in comparative effectiveness research, it should ensure that the
research questions that are relevant to patients, physicians and other
health care providers are given a high priority. Having an open,
patient-centered process for setting research goals and allowing
patient and provider voices to be heard will ensure that comparative
research benefits patients and providers.
PhRMA and our member companies are engaged in a number of
activities to strengthen the field of comparative effectiveness
research and enhance the evidence base. However, we also believe more
can be done to strengthen the field of comparative effectiveness
research and strengthen our health care evidence base.
While supporting steps to expand evidence on comparative
effectiveness, we also recognize that this type of evidence can be
misapplied as a blunt cost control tool through ``one size fits all''
coverage or payment policies. Physician and patient experience, a
growing body of research, and the emerging science of molecular
medicine all show why one size almost never fits all in medicine.
Comparative effectiveness research should be used in ways that reflect
differences in patient response to treatment and differences in
individual clinical needs and preferences, and enable physicians to
tailor treatment for the individual based on best available evidence.
Some proposals for creating a new comparative effectiveness
research entity have pointed to the example of governments in other
developed countries that use comparative and cost-effectiveness
information. Experience with the use of comparative--and cost-
effectiveness evidence in these countries illustrates the way it can
lead to patient access restrictions on important medical advances. In
the United Kingdom and Australia, patients face very significant
restrictions on access to treatments based on the use of rigid
comparative- and cost-effectiveness standards to establish centralized
coverage policies. Patients who have diseases such as cancer,
Alzheimer's disease, diabetes, osteoporosis, blindness, and rare
diseases, have faced government-imposed access restrictions. These
access barriers illustrate one of the major challenges of centralized
government approaches to generating and using comparative and cost-
effectiveness research, and the strengths of more pluralistic
approaches.
The range of different proposals for government comparative
research illustrates some of the important, unresolved issues in this
area. Important issues to be addressed include clarifying the
definition and goals of comparative effectiveness research; defining
the scope of government-supported comparative effectiveness research;
establishing patient-centered approaches to research priority-setting
and communication; developing research methods to support high quality
comparative effectiveness research; and understanding the relationship
between comparative effectiveness research, personalized medicine, and
health information technology. *** TO PREVENT BAD BREAK AT
BODONI DASH *** deg.
PhRMA looks forward to working with policy makers to address these
issues and advance the field of comparative effectiveness research.
Statement of Society of General Internal Medicine
Mr. Chairman and Members of the Subcommittee:
Thank you, Mr. Chairman, for calling this hearing to address the
issues surrounding comparative clinical effectiveness for improving
healthcare and thereby the health of Americans. Your long record of
fighting for the best possible health care for the American people is
well-known and deeply appreciated by all of us who share your passion
for improving a system that everyone in America knows has great
strengths and very deep flaws.
We are testifying today on behalf of the Society of General
Internal Medicine (SGIM), an organization comprised of approximately
3,000 academic general internists throughout the United States. SGIM
exists to promote improved patient care, research, and education in
primary care and general internal medicine. Our members are specialists
in adult medicine, treating patients who often present with complex,
multiple diseases--some chronic, some acute--in a healthcare system
that sometimes works against the provision of the highest quality care.
As an organization, we are especially sensitive to the needs of
minority and underserved populations, who suffer the most from
inadequate access to quality care and the needless health consequences
resulting from leaving otherwise minor conditions untreated.
We understand that comparative clinical effectiveness research will
not cure all the ills of the health care system. However, we believe
that such the development of the methods, individuals, and capacity to
do such research and to translate its findings into practice will very
significantly contribute to improving the quality of health care in
this country. Moreover, the implementation of the results of such
research could ultimately result in reductions in the costs of care,
thereby allowing much needed improvements in access to care for
underserved Americans.
As you know, Mr. Chairman, the FY08 Budget Resolution includes a
provision authorizing the committees of jurisdiction in the House and
Senate to establish a deficit neutral trust fund for comparative
effectiveness research (CER) in healthcare. Because members of SGIM are
leaders in the fields of clinical and health services research that
form the basis for CER, because SGIM members are the leaders of the
AHRQ and NIH research training programs that will need to produce those
who will make such a national CER initiative possible, and because SGIM
members are on the front line of delivering primary care, we fully
expect they will play a major role in the development of this emerging
sphere of research and its translation into clinical practice.
As the Subcommittee prepares to address this critical issue, SGIM
would like to offer comments on a variety of aspects of the legislation
you are developing, including (a) the location of the research
infrastructure within the existing governmental health care structure,
(b) the methods to assure that the research priorities chosen as well
as the conduct and dissemination of the research that is undertaken are
held to the highest scientific standards, free of political or other
undue influence, (c) the sources of funding for this initiative, and
(d) the substantive areas that need to be addressed to maximize the
impact of this research.
No Need to Reinvent the Wheel
SGIM believes that the prioritization and governance of the conduct
of comparative clinical effectiveness research is fundamentally a
Federal Governmental responsibility. The federal responsibility for
assuring that the provision of healthcare throughout the United States
is of the highest quality should be unquestioned. No outside entity--
whether for profit or non-profit--can demonstrate the strength, the
independence, the commitment to all citizens' health, and the degree of
acceptance that the Federal Government can. To ``outsource'' such a
responsibility would call into question the seriousness of the
commitment and could create a series of undermining unintended
consequences.
Within the Federal Government, SGIM believes strongly that the
Agency for Healthcare Research and Quality (AHRQ) is the natural home
for any newly-developed CER program. Placing the program into an
existing governmental agency saves both time and money during the
initial--and crucial--implementation phase. CER is an important
component of translational research. AHRQ's role in the Department of
Health and Human Services (HHS) is facilitating the translation of
medical evidence into practice, which it accomplishes, in part, through
its Evidence-Based Practice Centers, as well as through collaborations
with the Food and Drug Administration (FDA) and the Centers for
Medicare and Medicaid Services (CMS). AHRQ's role in leading the
development and implementation of health information technology (HIT)
to support the translation of such efforts into improved access and to
further quality improvement and patient safety also would leverage its
being the home for CER. Understandably and importantly, the national
healthcare industry outside of HHS looks to AHRQ as the fair and
committed leader and partner to fulfilling this translational role.
In addition, because AHRQ's authorizing statute already contains
the mandate to address issues related to health disparities, locating
the CER program in that Agency assures these critical issues will be
addressed in any approved research and that they will be reported in
the peer-reviewed scientific journal articles that result from that
research.
Assuring an Objective and Independent Process
Ultimately, comparative clinical effectiveness is about the
provision of quality health care. But, we would be naive not to
acknowledge that there are financial implications for the government,
for private industry, for physicians, and for patients involved in
nearly every decision that would be made in this field. For this
reason, it is imperative that this research be undertaken under the
most transparent, ethical and objective conditions possible.
First, the decision about what research to do--and not do--must be
based on scientific standards that are widely recognized and accepted.
Priority setting by AHRQ must be a public process in which all
stakeholders--individuals and organizations--have the opportunity to
make their case for what they believe to be the appropriate priorities.
But the decisions on those priorities should be made by an objective
advisory committee of career experts from within the government and
nationally-recognized authorities outside the government with no
conflicts of interest that could raise questions concerning the
objectivity of the decisions made.
Second, the conduct of the research must be held to the highest
ethical standards. SGIM believes that this research is best conducted
at academic health centers and other similar institutions throughout
the United States. Specific grant awards should be chosen by well-
established practices by peer-review panels operating within the
confines of the priority-setting described above. Research and
researchers should be reviewed periodically by career AHRQ employees to
assure the adherence to the high ethical standards.
Finally, it is critically important that the results of this
objective research be made available and broadly disseminated to the
American people and to the health care community in a timely and
accurate manner. Therefore, concrete provisions should be included in
the legislation to assure that no future Congressional Committee, no
Executive Branch employee, and no Administration is able to delay or
prevent the publication or dissemination of the results of this
research.
Sources of Funding
As the Subcommittee delves into this initiative, it will be
obligated to consider the source from which the resources to make it a
reality will come. This question is, to an extent, a subset of the
issue described above, as the sources of funding used can have a
significant impact on the objectivity and independence of the project.
There is no question that Medicare Trust Fund money should and will
be an important contributor to this effort. Medicare (and for that
matter, Medicaid) stand to benefit greatly from the improvements in
clinical effectiveness that will result from this concentrated research
effort and it is reasonable that these funds will contribute to the
investment needed to launch this initiative.
However, it is also undeniable that the private health insurance
industry will also benefit from learning what medical procedures,
processes, and products have the highest clinical utility for patients.
Clearly, treating or curing people faster results in long-term cost
savings that will inure to the benefit of the insurance industry. It
follows that they should be active participants in making the
investments that lead to the needed research.
The Subcommittee is uniquely positioned to devise a formula that
will result in both the public sector and the private sector
contributing to this initiative. It is important, however, that any
formula used is broad-based and does not tie dollars invested to any
specific research. To do otherwise would generate questions related to
the independence of the research effort, specifically as it relates to
the funding. That would undercut the arms-length relationship that will
be crucial for establishing the program's integrity.
The Needs for a Successful Comparative Clinical Effectiveness Research
Initiative
Merely suggesting that there should be a stronger national effort
in comparative clinical research, that it should be handled according
to the highest scientific standards through AHRQ, and that it should be
funded broadly will not lead to a successful, high impact, and
sustained result. For the success of this potentially transforming
healthcare initiative, it is critical that the Subcommittee be specific
in identifying those issues that are currently not being addressed
through existing research mechanisms.
To do this, SGIM recommends that the subcommittee create a six-part
research program by statute to address the specific unmet needs of
comparative clinical effectiveness research as called for in the Budget
Resolution:
1. Capacity to Develop Reports Based on Current Knowledge
There is an almost unlimited amount of recent research,
funded by the National Institutes of Health (NIH), Centers for
Disease Control and Prevention (CDC), AHRQ, and others, that
has already been done that can provide the raw material to set
in motion quickly research that will add significantly to our
knowledge base of the comparative effectiveness of clinical
treatments.
SGIM recommends that AHRQ use the infrastructure established
by Section 1013 of the Medicare Modernization Act (MMA), but
utilize not less than $25 million in the first year (with
amounts growing each year after that) to expand the existing
capacity beyond the current limited research that has been
conducted as a result of the modest effort of the MMA. Such
expansion would provide the wide range of data needed to
leverage optimally current information to result in a
measurable improvement in care.
The goal of this effort would be the production of a minimum
of 80-100 reports yearly on a range of important healthcare
issues with decision support for implementation.
2. Capacity to Develop New Understanding of Effective Care Based on
Ongoing Care
While SGIM believes it is important to obtain the maximum
benefit from existing research, we also believe in developing
new understanding about effectiveness based on on-going care.
Specifically, we would use, in part, the existing
infrastructure of the Centers for Education and Research on
Therapeutics (CERTs) and the Developing Evidence to Inform
Decisions about Effectiveness (DeCIDE) Network for this
purpose.
At the same time, we recommend the creation of additional
capacity to develop new information on the effectiveness of
treatments through the use of existing and developing public
and private sector electronic medical records (EMR) systems.
This would not create new databases, but would leave control of
health records and data with the owners of the data--a
distributive model. With greater emphasis being placed on EMRs,
it is important that any CER program be positioned to capture
data on EMRs for maximum benefit.
The objective of this effort would be to create methods and
capacity to do research cheaper and faster (e.g., 6-18 month
projects instead of multi-year multi-million dollar research)
that takes advantage of the increased use of EMRs and HIT for
assessing the outcomes of new and existing interventions for
which full scale clinical trials are not necessary. It is
anticipated that not less than $50 million could be expended on
this effort in the first year, with the available funds growing
each year thereafter.
3. Accelerating the Translation of New Information into Practice
and
Routine Healthcare Delivery
As indicated above, SGIM does not believe it is necessary or
wise to reinvent the wheel in the implementation of this
effort. We believe it is possible to use the existing
infrastructure of networks including Accelerating Change and
Transformation in Organizations and Networks (ACTION), the
Primary Care Practice-based Research Networks (PBRNs), and
AHRQ's Innovations Clearinghouse. These networks would be ideal
for translating new information into practice and routine
healthcare delivery, something that currently is lacking in our
health care system.
The goal of this aspect of the program would be to expand the
number of AHRQ's ``learning collaboratives'' that link cutting
edge providers and health plans with those seeking to emulate
the national leaders in adoption of health information
technology, patient safety, and quality improvement. In
addition, it should be a stated objective of the legislation to
create specific implementation roadmaps for proven
interventions and to expand support for training and
implementation teams. Not less than $50 million should be set
aside in the first year to meet these important objectives,
with the amount growing each year thereafter.
4. Demonstrate Approaches that Improve Efficiency and Reduce Waste
Currently, AHRQ provides very limited support for
organizational redesign to improve efficiency by
comprehensively addressing the structure and management of
healthcare settings, as well as processes of care. SGIM
recommends that this initiative be increased to not less than
$40 million in initial year funding, with the available funding
levels growing in each subsequent year.
The goal of this program is to use demonstrated and proven
approaches for increasing the efficiency with which care is
organized and delivered rather than the obtrusive traditional
cost containment measures that often generate negative
unintended consequences. We expect that this will generate
patient and provider support for needed expansion of healthcare
coverage in the context of limited resources.
In addition, we expect that this will create support for such
models of improved efficiency being adopted by healthcare
systems, clinics, and academic health centers that otherwise
would be hesitant to risk redesign of their systems.
5. Increase Transparency on Value to Support Consumer Choice
AHRQ presently plays a number of key roles related to quality
and value, including serving as the chair of the Ambulatory
Quality Alliance (AQA) and the co-chair of the joint AQA-
Hospital Quality Alliance Steering Committee, a leading role
with the Quality Demonstration projects, and chair of the
Quality Interagency Coordination (QuIC) Task Force of all
Federal Departments and agencies with an interest in healthcare
quality. In addition, it operates many public-private
partnerships. Thus, it would be reasonable and practical for
AHRQ to undertake the development of systems specifically
designed to measure and report on healthcare performance. We
would anticipate a need for not less than $30 million in the
first year of this undertaking, with the funding growing
substantially in subsequent years.
The first goal is for AHRQ to establish valid and robust
methods to measure and report the quality of healthcare
performance and to support transparency, accountability, and
rewards that are fair and accurate, something that is currently
very much in question and undermining trust in, and ultimately,
the positive impact of, such efforts.
The second goal is to create sufficient capacity to assist in
the implementation of such methods, including the use of
voluntary, consensus-based standard-setting intended to
harmonize measures across Federal agencies and across the
entire healthcare industry.
6. Research and Research Training to Support Remaking Healthcare
SGIM believes that AHRQ can reasonably use not less than $75
million in the first year (with growing outyear funding) to
apply existing approaches to funding peer-reviewed,
investigator initiated research, research training, and career
development to expand greatly the ability to address the wide
and crucial needs for basic research in healthcare delivery,
the development of new methods, and demonstrations of
innovative approaches to improve healthcare, to train badly
needed new researchers in this area, and to establish, support
and retain early researchers' career development.
The very specific goal of this initiative is to establish the
capacity for new innovative research and a cadre of researchers
nationally that would be positioned to apply the innovations
developed by biomedical researchers to improve the efficiency
and effectiveness of healthcare delivery and, ultimately, to
improve the health of our citizens with maximal efficiency.
SGIM estimates that the total first year funding for the six
initiatives discussed above should not be less than $270
million. We also believe that the goal should be to grow this
funding to not less than $1.0 billion per year at the earliest
possible date. We would support the Subcommittee devising a
mechanism to assure that the funds available are not
artificially limited through binding statutory provisions that
prevent fully addressing the need. Given that 17 percent of the
Gross Domestic Product (GDP) is spent on health care, this is
an exceedingly modest proposal designed to elevate comparative
clinical effectiveness research closer to its appropriate level
within a well managed health care system.
Conclusion
This Congress has an enormous task, and an enormous opportunity,
before it in addressing the shortcomings of the healthcare system.
Forty-seven million Americans, including nine million children, are
struggling without health insurance. Hospitals and physicians are
subject to annual reduction proposals in Medicare reimbursement rates.
States are feeling the strain of a Medicaid system that often treats
the poorest people in the most expensive manner possible--in emergency
departments.
In the face of these seemingly intractable problems, comparative
clinical effectiveness is a research field whose time clearly has come.
It will not solve everything that is wrong with the healthcare system.
But, it is a strong, serious, positive step that this Congress and
Administration can take together to improve the quality and the
availability of healthcare for all Americans.
The Society for General Internal Medicine is a long-time advocate
for the funding of such research and training for such research, with
the ultimate objective of improving healthcare and health. Our mission
is to promote improved patient care, research, and education in primary
care--a mission that is core to every action and position we take. We
are pleased and proud to participate in the debate over this important
initiative and look forward to working closely with you, Mr. Chairman,
and the other Members of the Subcommittee in the days and months ahead
to help bring this proposal to fruition.
Thank you.