[House Hearing, 109 Congress]
[From the U.S. Government Publishing Office]
PATIENT SAFETY AND QUALITY ISSUES
IN END STAGE RENAL DISEASE TREATMENT
=======================================================================
HEARING
before the
COMMITTEE ON WAYS AND MEANS
U.S. HOUSE OF REPRESENTATIVES
ONE HUNDRED NINTH CONGRESS
SECOND SESSION
__________
DECEMBER 6, 2006
__________
Serial No. 109-87
__________
Printed for the use of the Committee on Ways and Means
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COMMITTEE ON WAYS AND MEANS
BILL THOMAS, California, Chairman
E. CLAY SHAW, JR., Florida CHARLES B. RANGEL, New York
NANCY L. JOHNSON, Connecticut FORTNEY PETE STARK, California
WALLY HERGER, California SANDER M. LEVIN, Michigan
JIM MCCRERY, Louisiana BENJAMIN L. CARDIN, Maryland
DAVE CAMP, Michigan JIM MCDERMOTT, Washington
JIM RAMSTAD, Minnesota JOHN LEWIS, Georgia
JIM NUSSLE, Iowa RICHARD E. NEAL, Massachusetts
SAM JOHNSON, Texas MICHAEL R. MCNULTY, New York
PHIL ENGLISH, Pennsylvania JOHN S. TANNER, Tennessee
J.D. HAYWORTH, Arizona XAVIER BECERRA, California
JERRY WELLER, Illinois LLOYD DOGGETT, Texas
KENNY C. HULSHOF, Missouri EARL POMEROY, North Dakota
RON LEWIS, Kentucky STEPHANIE TUBBS JONES, Ohio
KEVIN BRADY, Texas MIKE THOMPSON, California
THOMAS M. REYNOLDS, New York JOHN B. LARSON, Connecticut
PAUL RYAN, Wisconsin RAHM EMANUEL, Illinois
ERIC CANTOR, Virginia
JOHN LINDER, Georgia
BOB BEAUPREZ, Colorado
MELISSA A. HART, Pennsylvania
CHRIS CHOCOLA, Indiana
DEVIN NUNES, California
Allison H. Giles, Chief of Staff
Janice Mays, Minority Chief Counsel
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C O N T E N T S
__________
Page
Advisory of November 29, announcing the hearing.................. 2
WITNESSES
The Honorable David M. Walker, Comptroller General, U.S.
Government Accountability Office............................... 26
Leslie V. Norwalk, Acting Administrator, Centers for Medicare and
Medicaid Services.............................................. 45
______
Ajay K. Singh, Associate Professor of Medicine, Brigham and
Women's Hospital, Harvard Medical School, Boston, Massachusetts 12
Laura T. Pizzi, Research Associate Professor of Health Policy,
Department of Health Policy, Thomas Jefferson University,
Philadelphia, Pennsylvania..................................... 8
Dennis J. Cotter, President, Medical Technology and Practice
Patterns Institute, Inc., Bethesda, Maryland................... 18
SUBMISSIONS FOR THE RECORD
American Society of Pediatric Nephrology, Indianapolis, IN,
statement...................................................... 66
Amgen, statement................................................. 67
Carrancejie, Richard, Birmingham, AL, statement.................. 75
DaVita Patient Citizens, statement............................... 76
Henrich, William, Anemia Management, letter...................... 76
Ishak, Noshi, Central New Hampshire Kidney Center, Laconia, NH,
letter......................................................... 78
Kidney Care Partners, statement.................................. 81
National Kidney Foundation, Inc., New York, NY, statement........ 83
Renal Physicians Association, Rockville, MD, statement........... 85
Robinson, Kris, American Association of Kidney Patients, Tampa,
FL, statement.................................................. 86
Schatell, Dori, Medical Education Institute, Madison, WI,
statement...................................................... 88
Sweeney, Jim, Coalition for Dialysis Patient Choice, letter...... 90
Tate-Harris, Patricia, Association of Dialysis Advocates, Baton
Rouge, LA, statement........................................... 92
PATIENT SAFETY AND QUALITY ISSUES
IN END STAGE RENAL DISEASE TREATMENT
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WEDNESDAY, DECEMBER 6, 2006
U.S. House of Representatives,
Committee on Ways and Means,
Washington, DC.
The Committee met, pursuant to notice, at 10:45 a.m., in
Room 1100, Longworth House Office Building, Hon. William M.
Thomas (Chairman of the Committee) presiding.
[The advisory announcing the hearing follows:]
ADVISORY
FROM THE
COMMITTEE
ON WAYS
AND
MEANS
CONTACT: (202) 225-1721
FOR IMMEDIATE RELEASE
November 29, 2006
FC-27
Thomas Announces Hearing on
Patient Safety and Quality Issues in
End Stage Renal Disease Treatment
Congressman Bill Thomas (R-CA), Chairman of the Committee on Ways
and Means, today announced that the Committee will hold a hearing on
safety and quality for Medicare beneficiaries with End Stage Renal
Disease (ESRD). The hearing will take place on Wednesday, December 6,
2006, in the main Committee hearing room, 1100 Longworth House Office
Building, beginning at 10:30 a.m.
In view of the limited time available to hear witnesses, oral
testimony at this hearing will be from invited witnesses only.
Witnesses will include experts on Medicare payment and treatment of
beneficiaries with ESRD and government officials. 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:
In 1972, Medicare began to cover treatment for patients with kidney
failure, known as ESRD. Patients with kidney failure are typically
treated with dialysis and are prescribed medication to address anemia,
calcium and other deficiencies.
Between 1998 and 2003, ESRD treatment spending increased by almost
50 percent. In 2004, Medicare covered about 309,300 dialysis patients,
nearly 93 percent of all such patients in the United States. According
to U.S. Renal Data System (USRDS) and the Medicare Payment Advisory
Commission (MedPAC), Medicare spends about $64,000 per year for each
person on hemodialysis for all medical services.
In the last 10 years, mortality rates for ESRD patients have
declined except for patients that have been receiving therapy for 5 or
more years. During the same time period, however, hospitalizations for
infections and cardiovascular complications are up 20 and 10 percent,
respectively. To address these problems, the Centers for Medicare and
Medicaid Services (CMS) has taken steps to improve quality and safety
in ESRD facilities. For instance, in 2004, CMS developed a dialysis
facility comparison website that contains service and quality
information on all Medicare approved dialysis facilities.
However, significant problems remain with the quality of care for
patients that receive dialysis for kidney failure as well as the
payments for this population. Two recent studies have indicated two
specific concerns:
1. Patient safety. USRDS data show that 40 percent of patients in
the dialysis population that are being treated with an anemia drug have
a red blood cell count above the Food and Drug Administration (FDA)
recommended level. Moreover, half of the 40 percent have a level
associated with the higher risk of cardiovascular events and mortality,
according to a November 2006 study published in the New England Journal
of Medicine.
2. Inefficient and unnecessary Medicare spending. A recent study
from November 2006 in Dialysis and Transplantation found that the
population with a red blood cell count above industry guidelines also
has higher drug costs, specifically, $3,100 per patient per year more
just on the anemia drug.
In March 2006, the MedPAC reported that dialysis facilities
continue to lose money on the composite rate, which includes the costs
of nursing services, equipment and supplies. However, the losses are
partially recouped by Medicare payment for drugs at Average Sales Price
plus 6 percent. The Commission reported that the Medicare Modernization
Act of 2003 (P.L. 108-173) made Medicare's drug payments less
profitable in total, but also reported that the financial incentive to
use more drugs persist even under the revised payment policy.
In announcing the hearing, Chairman Thomas stated, ``While we have
made gains in improving the End Stage Renal Disease program, clearly we
need to continue to explore what more can be done to improve patient
safety and quality of care. Patient safety and efficient use of
taxpayer dollars are critical. We should examine the increased dosage
of these drugs and the possible detrimental health effects. I am also
concerned that Medicare has not been a prudent purchaser in this arena,
given its rapid growth in spending. ESRD providers do not receive an
annual update which is why a permanent solution that provides payment
stability is critical to end perverse incentives based on the
utilization of drugs.''
FOCUS OF THE HEARING:
In continuing the Committee's consideration of improving the
quality of health care in the Medicare program, the hearing will focus
on recent research on the Medicare payment for drugs used in treating
ESRD patients, the quality and safety of the treatment for ESRD
patients as well as oversight on the CMS operations related to ESRD.
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Chairman THOMAS. Good morning. First of all, I want to
thank our witnesses and the Members of the Committee. In an
attempt to make sure that we can utilize the time as usefully
as possible on a very important hearing--made more timely by
recent publications that were released in a GAO study--
notwithstanding the fact we are in a lame duck session in which
system negotiations are occurring between the House and the
Senate; therefore, we have asked the panel of specific experts
who have a body of written information that has been available,
and they will comment directly on that. The Chair would request
that Members limit any questioning if at all possible, so that
we could delve then relatively quickly to members of the
administration of the GAO, who have time constraints of their
own.
The Committee will hear from our distinguished panel of
witnesses, basically, whether Medicare is appropriately
safeguarding the packets and the integrity of the trust funds
because, recently, the scientific and even mainstream press
have pointed out a growing concern about unsafe and
questionable treatment for Medicare's coverage for kidney
failure, also known as End Stage Renal Disease.
We know that for more than 30 years Medicare has covered
treatments for patients with ESRD. Treatments usually consist
of dialysis, also with anemia, a lower number of red blood
cells, drugs. Medicare payments for these treatments have
increased rapidly by almost 50 percent between 1998 and 2003.
In fact, one of the drugs to treat ESRD has been identified as
the single largest expenditure in Medicare part B each year,
notwithstanding the small population that receives the drug.
More importantly, there has been longstanding safety concerns
about whether patients receiving treatment for ESRD are
actually being harmed by the perhaps high doses of anemia drugs
they are prescribed. According to the U.S. Renal Data System 40
percent of the dialysis patients treated for low red blood
cells with anemia drugs actually have a red blood cell count
above the FDA recommended levels. In fact, after the drugs,
beneficiaries have a level high enough to trigger serious
cardiovascular problems, some resulting in death. So, the
question is not only one of taxpayers' money being spent on a
monopoly drug; it is the question of what is reasonable and
appropriate from a health point of view.
We are also anxious to hear testimony from the Centers for
Medicare and Medicaid Services, CMS. In April of this year, the
Chair wrote to then administrator Mark McClellan asking several
pointed questions about why CMS had developed the policy to
deal with what we considered to a certain extent out-of-control
dosing of ESRD patients at a different level than the FDA
recommended and the labels on the drugs prescribed.
If this was the right policy, the Chair believes it should
have been easy to answer the letter. It took CMS 8 months,
until this week in fact, to respond. Then, again, in November,
not having that in a response, House Subcommittee Ranking
Member Stark and the Chairman wrote a letter to Acting
Administrator Norwalk--and the Committee appreciates her
ability to attend today--reiterating our concerns. Again, the
letter was not responded to until Monday night. Obviously,
today we are going to talk about the letter response but, more
importantly, the concerns that the letter reflected.
Now, after a number of months and having seen a significant
number of publications focusing on exactly those issues,
hopefully, we will be able to get some understanding of the
issue of the treatment for patients. Obviously, we are going to
solicit ideas for improving qualities for these beneficiaries,
and we are interested in hearing now the GAO's testimony
following the release yesterday of their report on Medicare
payment for ESRD services. We have a significant document, a
printed evidentiary record, that will be in front of us, and we
want to know where we are going to go from here.
So I am excited about this hearing. Obviously, this is the
beginning, notwithstanding the fact it is coming at the end of
this Congress; these questions will obviously carryover. I
appreciate the ongoing bipartisan working relationship that we
have had on this very important issue, and I will recognize the
gentleman from New York for any statement he may wish to make.
Mr. RANGEL. Thank you, Mr. Chairman. This meeting is
historic for a variety of reasons. One, because you visited
with us in the Democratic Caucus together and made it clear
that you and Peter Stark were in agreement on this subject
matter. For us, that is a gigantic step, and we wanted that to
be properly recorded.
The second thing----
Chairman THOMAS. Would the gentleman yield?
Mr. RANGEL. I would be pleased.
Chairman THOMAS. I am pleased you finally got it right.
Mr. RANGEL. The second thing is this probably will be the
last formal hearing that you will be chairing. I think the
audience should know that, notwithstanding what the record
would indicate, these Committee hearings, as it relates to your
relationship with me, that the audience should know that Bill
Thomas and I have never, but never, in the years that we have
served in the House of Representatives had an unpleasant
conversation outside of the Committee room.
I also would like to say that the Committee has agreed, and
I have agreed, to host the reception that was supposed to be a
surprise, but knowing how difficult it is in the last few days
of our work, that we hope that you will be available at 5:00
tomorrow when the Committee members and staff would like to
thank you for the dedication which you have given to the
Committee, the Congress, and the country.
Lastly, we would like to wish you a happy birthday. This is
your 65th birthday, and now I can see why you are concerned
about Social Security, as you become eligible for Medicare, and
I have personally had a special Social Security card made up in
connection with reform that Jim McCrery and I are going to be
working on, and I have signed this so that if you have any
problems at all, you can rest assured that this will be able to
get you the proper health care that you might need.
Chairman THOMAS. I want to thank the--this looks like the
$3 bill he gave me last year. The gentleman needs to know that,
unfortunately, I voted for the extension of age not nearly long
enough based on life expectancy so I don't get to use my Social
Security card until I am 65 years, 8 months. However, Medicare
kicks in immediately and hence the reason for this particular
hearing.
Mr. RANGEL. I would like to yield the substantive questions
to your colleague, Peter Stark.
Mr. STARK. Thank you.
Thanks, Bill. My wishes for a happy 65th birthday. I wish I
could remember what I did on mine.
But now that you are no longer just an observer, I want to
ensure you that we will do our best to make it a successful
program for you and all Americans.
I am pleased to call this hearing. It is--the ESRD policy
is unique in our country. Some may say it is our only form of
socialized medicine. Almost everybody who is involved in
dialysis is involved in the government finance program. One of
the problems that we have had is that we have been involved in
using these drugs which have cost us a couple billion dollars a
year, and many of us have maintained for a long time that we
should be getting a better deal. We are now faced with the
potential that we may actually, through policies of
reimbursement, be putting people at risk for danger to their
health, and that is something that I don't think we should
tolerate.
I wanted as many of you in the room to know I have a long
history on this issue, and it has come to my attention, Mr.
Chairman, that recently certain interests may be
misrepresenting my past positions, and I would just like to
submit for the record a copy of a letter I wrote to CMS in 1997
that has been circulated and a written response from my then
lead staff person Bill Vaughan who helped draft my response,
and I just add this to clarify the record if Mr. Chairman would
accept.
Chairman THOMAS. Without objection.
[The information follows:]
November 29, 2006
The Honorable Fortney H. (Pete) Stark
Ranking Member, Subcommittee on Health
Committee on Ways and Means
239 Cannon House Office Building
Dear Congressman Stark:
As a former staff member who served on the Ways and Means Health
Subcommittee between 1996 and the spring of 2001, your current staff
has asked me to elaborate on the letter you asked me to draft, and
which you signed, addressed to former Health Care Financing
Administrator Nancy-Ann DeParle dated December 8, 1997 relating to
Medicare coverage of EPO.
Apparently someone--who has not read the letter thoroughly--is
alleging that this letter indicates your support of higher dosages of
EPO. That is a complete misreading of your letter. Your letter was an
effort to encourage the removal of financial incentives that have long
distorted the administration of EPO--a distortion that has cost
taxpayers hundreds of millions--perhaps billions!--of dollars and which
we now find may have been hurting the health of hundreds of thousands
of patients. As you repeatedly stressed to me, your goal on the
Subcommittee has always been to encourage the best practice of
medicine, without financial influences to over--or under--treat
patients. This letter is part of that theme--a theme seen in your other
efforts, such as the physician referral laws (Stark I and II) and your
successful amendment to limit the amount that managed care physicians
can be financially placed at risk for under-treatment of patients.
Because historically there has been a spread between what Medicare
reimburses a dialysis center for a unit of EPO and what the company's
net selling cost of the product to the center is, centers have profited
by increasing their use of EPO. I once even saw a chart that a
salesperson for the company gave to dialysis clients showing how profit
would increase as dosage was increased!
Only by eliminating the profit incentive to administer higher and
higher doses can patients have the peace of mind that they are getting
an appropriate level of EPO. Ideally, in my opinion, centers would be
reimbursed for their net acquisition cost plus a dollar for
administration (since it is generally administered through an existing
line to the patient).
The VA, Kaiser, and most of Europe generally (but on a case by case
basis) administers EPO subcutaneously, which in most people results in
the more efficient uptake of the medicine and can save substantial
amounts of money because less EPO is necessary to achieve the same
hematocrits. I urge you to encourage such a policy, perhaps sharing the
savings with beneficiaries through reduced copayments as compensation
for the inconvenience of the injection. It would also be useful to ask
if the company has finally made a dose formulation that minimizes any
pain of injection.
The December 8, 1997 letter was written before I remember ever
seeing any studies (such as those excellent papers by Dennis Cotter,
et. al) raising safety concerns of over-dosage. It was written before
the recent important discussion of CMS dosage rates exceeding FDA
recommended dosages. In writing the letter, we were concerned that the
HCFA policy would cause centers to under-dose because of fear of non-
payment. Under-dosing for financial reasons is clearly as bad as over-
dosing for financial gain. Your letter was designed to deal with both
issues: allow upward dosing where a physician thought it was
appropriate, but take away almost all of the overpayment incentive that
was causing over-dosing for financial gain.
As a former staffer, who organized the very first Ways and Means
oversight hearings on the ESRD program in 1975, I have long felt that
the financial incentives in this program have been an abuse to the
taxpayer and to the best care of patients. I deeply regret that we did
not make more progress on this issue when I was one of your staff
members, and I wish you the best in finally achieving good health
policy at a reasonable cost to taxpayers.
Sincerely,
William Vaughan
P.S. Don't cap medical malpractice! Maybe it's time for someone to
get sued for the abuse of patients in this sector.
Mr. STARK. I look forward to hearing from our witnesses.
Chairman THOMAS. Any other member may put a written
statement in the record. Our witnesses have an extensive body
of studies and the rest, and my goal would be to have you
present in a very succinct way, in the time you have available,
the key points you might want to make based upon these recent
studies, which have obviously been very timely and focused us
on the concerns that we had, some general concerns that clearly
now have been evidenced by clinical studies as well. It seems
to me, given the three panelists, that we would start with Dr.
Pizzi for no other reason than the fact that you are in the
middle.
STATEMENT OF LAURA T. PIZZI, PharmD, MPH, RESEARCH ASSOCIATE
PROFESSOR OF HEALTH POLICY, DEPARTMENT OF HEALTH POLICY, THOMAS
JEFFERSON UNIVERSITY, PHILADELPHIA, PENNSYLVANIA
Dr. PIZZI. Chairman Thomas and distinguished Committee
members. My name is Dr. Laura Pizzi. I am research associate
professor of health policy at Jefferson Medical College in
Philadelphia. I am a pharmacist by training but for the past 10
years have worked as a researcher on issues related to the
costs and outcomes of pharmaceuticals and presently lead a
group of six researchers at Jefferson who are dedicated to this
topic. I hold the secondary appointment as adjunct assistant
professor in pharmaceutical business at the University of
Sciences in Philadelphia and am co-editor of the text entitled,
Economic Evaluation in U.S. Health Care, Principles and
Applications, which was released last year.
I am here today to discuss the results of a study that I
led at Jefferson which was published in the November 2006
journal, Dialysis and Transplantation. This study was conducted
by our team at Jefferson along with Dr. David Goldfarb, who is
a nephrologist at the New York Harbor Veterans Affairs Medical
Center in New York City, and Dr. Joseph Fuhr, who is a
professor of economics in Chester, Pennsylvania. The study was
funded by a grant from Watson: Laboratories. While we received
funding from Watson, our team formulated the research
objective, designed the study, and performed the analysis
independently. My testimony does not reflect the views of the
sponsor nor of Jefferson.
The objective of this study was to determine the extent to
which health care providers adhered to clinical practice
guidelines for the treatment of anemia in patients receiving
hemodialysis. The guidelines that we used were those published
by the Kidney Disease Outcomes Qualified Initiative, also known
as KDOQI, released in the year 2000 by the National Kidney
Foundation. We compared actual utilization in practice for the
anemia drugs, erythropoietin, or EPO, and intravenous iron to
the KDOQI 2000 guideline recommendations. Actual utilization
was obtained from the United States Renal Data Service annual
report for 2004.
The critical target for anemia in this population was a
hemoglobin of 11-12 mg/dL. To reach this hemoglobin level,
patients need to have their iron stores replenished with
intravenous iron, and they also need to receive EPO to
stimulate red blood cell production. The guideline calls for an
initial EPO of 120-180 units per kg per week, which we assumed
remained the dose during the study period. We calculated
recommended dosages based on the average weight of an adult
receiving hemodialysis, 159.5 pounds.
For iron, we used recommended doses from KDOQI. We then
examined actual utilization per the United States Renal Data
Service Data and compared it to what was recommended by the
KDOQI 2000 guidelines. Our findings indicated that there was
significant overuse of EPO and slight underuse of intravenous
iron. Although we were not surprised to see that the providers
were not strictly adhering to the guideline, we were quite
surprised by the extent to which EPO use in practice deviated
from KDOQI recommendations.
Next, we converted the difference in utilization to dollars
based on 2005 Medicare reimbursement rates. We estimate that
CMS could have reduced expenditures for these drugs by 36
percent if dialysis facilities adhered to the guidelines. If
CMS spends $2 billion per year on EPO, it is reasonable to say
that several hundred million dollars could have been saved if
the providers followed the guidelines.
Chairman THOMAS. Dr. Pizzi, you are down to about a minute,
and I would prefer that you move to your recommendations and
conclusions, because we have your written testimony, and it
will be made a part of the record.
Dr. PIZZI. I believe the best way to address the matter of
EPO overuse is to reward dialysis providers who achieve an
appropriate hemoglobin target--whether the target is based on
KDOQI recommendations, product labeling, expert opinion, or a
combination of these sources. Once the target has been agreed
upon, CMS may wish to consider a pay-for-performance
reimbursement policy centered upon that hemoglobin target as
follows: Lower the reimbursement rate for EPO such that it is
cost neutral to the facilities and reward facilities with a
higher reimbursement rate for the dialysis session for patients
whose hemoglobin is in the target range.
In conclusion, the results of our study indicate that EPO
use far exceeded what was recommended in the KDOQI guideline
during the study period. Despite changes in the guideline as
well as the Medicare reimbursement policy, I believe EPO is
still being used in excess today.
I thank you very much for your consideration and hope that
a pay-for-performance-based reimbursement policy will be
evaluated as a means to ensure sufficient treatment for this
vulnerable population.
Thank you very much.
[The prepared statement of Dr. Pizzi follows:]
Statement of Laura T. Pizzi, Research Associate Professor of Health
Policy, Department of Health Policy, Thomas Jefferson University,
Philadelphia, Pennsylvania
Chairman Thomas and distinguished Committee Members, my name is Dr.
Laura Pizzi and I am a Research Associate Professor of Health Policy at
Jefferson Medical College in Philadelphia. I am a pharmacist by
training, but for the past 10 years have worked as a researcher on
issues related to pharmaceutical cost and outcomes and presently lead a
group of 6 researchers at Jefferson who are dedicated to this topic. I
hold a secondary appointment as Adjunct Assistant Professor of
Pharmaceutical Business at the University of the Sciences in
Philadelphia and am co-editor of the text entitled ``Economic
Evaluation in U.S. Healthcare: Principles and Applications'' which was
released last year.
I am here today to discuss the results of a study that I led at
Jefferson, which was published in the November 2006 issue of the
journal Dialysis and Transplantation.\1\ This study was conducted by
our team at Jefferson, along with Dr. David Goldfarb who is a
nephrologist at New York Harbor Department of Veterans Affairs Medical
Center in New York City and Dr. Joseph Fuhr who is a professor of
economics at Widener University in Chester, Pennsylvania.
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\1\ Pizzi LT, Patel NM, Maio VM, Goldfarb DS, Michael B, Fuhr JP,
and Goldfarb NI. Economic Implications of Non-adherence to Treatment
Recommendations for Hemodialysis Patients with Anemia. Dialysis and
Transplantation 2006;1-7.
---------------------------------------------------------------------------
The study was funded by a grant from Watson Laboratories in
Morristown NJ. While we received funding from Watson, our team
formulated the research objective, designed the study, and performed
the analysis independently. My testimony does not reflect the views of
the sponsor nor of Jefferson.
Summary of the Study
The objective of this study was to determine the extent to which
healthcare providers adhere to clinical practice guidelines for the
treatment of anemia in patients receiving hemodialysis. The guidelines
that we used were those published by the Kidney Disease Outcomes
Quality Initiative (KDOQI), released in the year 2000 by the National
Kidney Foundation.\2\
---------------------------------------------------------------------------
\2\ National Kidney Foundation. KDOQI Clinical Practice Guidelines
for Anemia of Chronic Kidney Disease 2000. American Journal of Kidney
Disease 2000; 37:S182-S238 (suppl 1).
---------------------------------------------------------------------------
We compared actual utilization in practice for the anemia drugs,
erythropoietin or ``EPO'' and intravenous iron to the KDOQI 2000
guideline recommendations. Actual utilization was obtained from the
United States Renal Data Service (USRDS) Annual Report for 2004, which
includes 431,284 active patients.\3\ This data source captures patient
and facility records from the CMS End Stage Renal Disease (ESRD)
Program's Management and Medical Information System, an Annual Facility
Survey, and data related to services delivered via Medicare, including
treatments administered to ESRD patients, patient outcomes, and costs.
The report is updated annually.
---------------------------------------------------------------------------
\3\ USRDS Annual Data Report 2004. Available at: http://
www.usrds.org/adr_2004.htm (Accessed 27 Nov 2006)
---------------------------------------------------------------------------
From USRDS, we obtained the total number of Medicare beneficiaries
who received hemodialysis, which was 372,643. Approximately 96%
received EPO at least once during a 3 month period.
The clinical target for treatment of anemia in this population, per
the KDOQI 2000 recommendations, was a hemoglobin level of 11-12 mg/dL.
To reach this hemoglobin level, patients need to have their iron stores
replenished with intravenous iron, and they also need to receive EPO,
which stimulates red blood cell production and thereby works to correct
the anemia. The recommended target for iron stores was a serum ferritin
level of at least 100ng/mL.
The guideline called for an initial EPO dose of 120-180 units per
kg per week, which we assume remained the dose during the study period.
We calculated the recommended dosage of EPO based on a 72.5 kg adult,
which is the average weight of hemodialysis patients reported by USRDS.
For iron, the recommended dose for adults was 100-125mg given
intravenously at every hemodialysis session for 8-10 doses followed by
a maintenance dose of 25-125mg per week upon reaching the target
ferritin level.
We then examined actual utilization, per USRDS 2004, and compared
it to what was recommended by the KDOQI 2000 guidelines. Our findings
indicated that there was significant over use of EPO and slight under
use of intravenous iron. Although we were not surprised to see that
providers were not strictly adhering to the guideline, we were quite
surprised by the extent to which EPO use in practice deviated from
KDOQI recommendations.
Next, we converted the difference in utilization, which was actual
versus recommended practice, to dollars based on 2005 Medicare
reimbursement rates for EPO and iron. We estimate that CMS could have
reduced expenditures for these drugs by 36% if dialysis facilities
adhered to the guidelines. If CMS spends $2 billion per year on EPO, it
is reasonable to say that several hundred million dollars could have
been saved on the drug if providers followed the guidelines.
Recent Data
If we were to repeat our study today using the same clinical target
but newer data from the 2006 USRDS Annual Report, our findings
regarding EPO over use would hold, because the mean EPO dose according
to this latest report is similar to what we used in our study.
Specifically, the mean monthly EPO dose that we used in our study was
76,473 units per month, and data from 2006 USRDS shows a mean monthly
EPO dose ranging from approximately 72,000-81,000 units per month in
calendar year 2005.\4\
---------------------------------------------------------------------------
\4\ USRDS Annual Data Report 2006. Table 5.37. Available at: http:/
/www.usrds.org/atlas.htm (Accessed 29 Nov 2006)
---------------------------------------------------------------------------
In addition to the costs resulting from EPO overuse, safety
concerns have emerged about maintaining hemoglobin levels above 13.5mg/
dL, as we know from Dr. Singh's testimony on the findings from the
Correction of Hemoglobin and Outcomes in Renal Insufficiency (CHOIR)
trial.\5\ In response to this study, the FDA issued an Alert on
November 16, 2006, which states that the target hemoglobin for EPO
should not exceed 12 g/dL.\6\ Although the KDOQI guidelines were
recently updated in 2006 and now recommend a hemoglobin 11g/dL while
not routinely maintaining the level 13g/dL, the upper threshold of
13g/dL was established prior to publication of the CHOIR. As a result
of these developments, the National Kidney Foundation announced last
week that it will convene an expert panel to assess EPO use.\7\
---------------------------------------------------------------------------
\5\ Singh AK, Szczech L, Tang KL, Barnhart H, Sapp S, Wolfson M,
and Reddan D, for the Correction of Hemoglobin and Outcomes in Renal
Insufficiency (CHOIR) Investigators. Correction of anemia with epoetin
alfa in chronic kidney disease. New England Journal of Medicine 2006;
355:2085-98.
\6\ Information for Healthcare Professionals: Erythropoiesis
Stimulating Agents (ESA) [Aranesp (darbepoetin), Epogen (epoetin alfa),
and Procrit (epoetin alfa)]. FDA ALERT 11/16/2006. Available at: http:/
/www.fda.gov/cder/drug/InfoSheets/HCP/RHE_HCP.pdf (Accessed 27 Nov
2006)
\7\ Berenson A. Treatment of anemia questioned. New York Times,
November 30, 2006.
---------------------------------------------------------------------------
Hence, though our study focused on the economic impact of non-
adherence to the guidelines, very recent data and concerns have emerged
about the safety impact of maintaining a hemoglobin level exceeding
12g/dL. These recent events, coupled with the results from our study,
provide evidence that it is time to more aggressively manage EPO use in
dialysis facilities.
Recommendations
In the time since our study was completed, CMS did change the
reimbursement policy for EPO. The revised payment policy required the
dose to be reduced by 25% when the hemoglobin exceeded 13g/dL.\8\, \9\
Providers who failed to reduce the EPO dose by 25% received a payment
reduced by 25%, unless the higher dose was approved through an appeals
process. This policy change marked a step towards more efficient
treatment, however in my opinion, it will not sufficiently stimulate
renal dialysis facilities to achieve the clinical target.
---------------------------------------------------------------------------
\8\ Claims Monitoring Policy: Erythropoietin/Darbopoietin Alfa
Usage for Beneficiaries with End Stage Renal Disease. Center for
Medicare and Medicaid Services, 2005. Available at: www.cms.hhs.gov/
coverage/8b5.pdf Accessed 12/02/05.
\9\ Levy R. The new CMS monitoring policy for anemia drug
reimbursement: Implications for providers. Dialysis and Transplantation
2006; 35(2): 88-90.
---------------------------------------------------------------------------
I believe that the best way to address the matter of EPO over use
is to reward dialysis providers who achieve an appropriate hemoglobin
target--whether that target is based on the KDOQI recommendations,
product labeling, expert opinion, or a combination of those sources.
Clearly, a target hemoglobin of 11-12mg/dL is appropriate, but there is
uncertainty about whether the window should be expanded to include
hemoglobin levels between 12 and 13mg/dL. I trust that Dr. Singh's
research along with the recommendations from the National Kidney
Foundation's expert panel will help to inform this matter.
Once the target has been agreed upon, CMS may wish to consider a
pay-for-performance reimbursement policy centered upon that hemoglobin
target as follows:
1. Lower the reimbursement for EPO such that it is cost-neutral to
the facility
2. Reward facilities with a higher reimbursement rate for the
dialysis session (composite rate) for patients' whose hemoglobin is in
the target range
CMS might also consider further boosting the composite rate for
patients who are given subcutaneous EPO (as opposed to intravenous
EPO), because administering the drug subcutaneously has been shown to
require significantly lower dosages.\10\, \11\, \12\, \13\
---------------------------------------------------------------------------
\10\ Hynes D, Stroupe KT, Kaufman JS, Reda DJ, Peterman A, Browning
MM, Huo Z, and Sorbara D. Adherence to guidelines for ESRD anemia
management. American Journal of Kidney Diseases 2006;47(3):455-6.
\11\ Thamer M, Zhang Y, Kaufman J, Stefanik K, Cotter DJ. Factors
influencing route of administration for epoetin treatment among
hemodialysis patients in the United States. American Journal of Kidney
Diseases 2006;48(1): 77-87
\12\ Besarab A. Optimizing Anemia Management with Subcutaneous
Administration of Epoetin. Nephrology Dialysis Transplantation 20(6):
vi10-vi16, 2005.
\13\ Besarab A. Reyes CM. Hornberger J. Meta-analysis of
Subcutaneous versus Intravenous Epoetin in Maintenance Treatment of
Anemia in Hemodialysis Patients. American Journal of Kidney Diseases
2002; 40(3):439-46.
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It is important to keep in mind, however, that pay-for-performance
measures do not necessarily reduce costs, and an increase in the base
composite rate may be necessary to maintain the supply of dialysis
facilities. In other words, some amount of monies that would have
otherwise been spent on EPO would be re-allocated to dialysis
facilities through the composite rate. However, implementation of the
policy would stimulate dialysis facilities to use EPO more efficiently
and would reduce their reliance on revenues from the product.
Conclusion
In conclusion, the results of our study indicate that EPO use far
exceeded what was recommended in the KDOQI guideline during the study
period. Despite changes in the guideline as well as the Medicare
reimbursement policy, I believe that EPO is still used in excess today,
primarily because dialysis facilities do not have a financial incentive
to manage its use. Thank you very much for your consideration and I
hope that a pay-for-performance based reimbursement policy for EPO will
be evaluated as a means of ensuring the safe and efficient treatment of
anemia in this vulnerable population.
Chairman THOMAS. Thank you. I apologize moving you to that,
but the statement that you have made is based upon data that is
well known and published.
Dr. Singh.
STATEMENT OF AJAY K. SINGH, MBBS, MRCP (UK), MBA, CLINICAL
CHIEF, RENAL DIVISION, DIRECTOR, DIALYSIS SERVICES, BRIGHAM AND
WOMEN'S HOSPITAL, HARVARD MEDICAL SCHOOL
Dr. SINGH. Chairman Thomas and distinguished Committee
members. My name is Dr. Ajay Singh. I am the clinical chief of
the Renal Division and director of dialysis Services and a
physician at the Brigham and Women's Hospital and an associate
professor of medicine at Harvard Medical School. I practice
medicine, teach physicians and students, and conduct patient-
oriented research. I am the first author and one of the
principal investigators of the CHOIR study. My written remarks
will be submitted as part of the record, and I wish to just
focus on the top conclusions from the CHOIR study.
In the CHOIR study, we tested whether targeting a
hemoglobin of 13.5 grams per deciliter versus a hemoglobin of
11.3 grams per deciliter in patients with chronic kidney
disease not on dialysis was associated with a survival benefit
and lower cardiovascular complications. To our surprise,
patients who were randomized with the higher hemoglobin group
had an excess risk of 34 percent with respect to death and
cardiovascular complications compared to those patients
randomized to the lower hemoglobin group.
Of note, we also found there were 52 deaths in the higher
hemoglobin group versus 36 deaths in the lower hemoglobin
group, a hazard ratio of 1.48 or a 48 percent higher risk. We
also found a higher risk for hospitalization for heart failure
to 41 percent higher risk. We did not find any incremental
improvement in quality of life for three different parameters
of quality of life that we tested, and we also found that for
cardiovascular risk adverse events, there were more adverse
events in the higher hemoglobin group versus the lower.
Therefore, the conclusion was there was both increased risk and
no substantive incremental quality of life benefit in raising
the hemoglobin among patients with chronic kidney disease not
on dialysis.
Now, some have argued that while this study looked at
patients with chronic kidney disease not on dialysis and this
data should not apply to patients on dialysis, I would
respectfully disagree with that. Both the National Kidney
Foundation guidelines, the European Best Practice guidelines of
anemia as well as the FDA label have aggregated patients not on
dialysis with kidney disease with those on dialysis and have
framed guidelines with respect to the higher hemoglobin level.
Until we get further data, I would argue that we should default
in the direction of patient safety by aiming for hemoglobin
levels no higher than 12 grams per deciliter, and our
recommendation of the paper was 11-12 grams a deciliter of
hemoglobin.
The other aspect of this was the issue of what one should
do with the rising number of proportional patients on dialysis
that have hemoglobin levels beyond 12 grams per deciliter. As
the Committee will note as part of the record information,
there are a number--there are a number of dialysis providers
who have patients who have hemoglobin levels above this 12-
grams-per-deciliter-range. Based on this study and some other
controlled studies, I would suggest and agree with the notion
that we should adopt a bundling for epoetin because it confers
with the potential of benefit without incentives, financial
incentives, to use higher levels of epoetin or aim for higher
hemoglobin levels. The only caveat I would suggest, there would
be some form of risk assessment in the patient population so
that providers are not disincentivized to treat sicker
patients.
So, in conclusion, our study as well as studies that have
been published prior to this suggest increased risk in raising
the hemoglobin level beyond 12 grams. I believe that this will
further reinforce the FDA label, which is clear, and a new
alert was published recently. I believe that this
recommendation should apply to both dialysis patients as well
as pre-dialysis patients until we have more evidence from
studies that hopefully will be funded in the future. I believe
that the best recourse to try and prevent hemoglobin levels
rising in both the dialysis population is to take the approach
of bundling of services so that there is--we remove the
financial incentives to use larger doses of EPO and aim for
higher hemoglobin levels.
[The prepared statement of Dr. Singh follows:]
Statement of Ajay K. Singh, Associate Professor of Medicine, Brigham
and Women's Hospital, Harvard Medical School, Boston, Massachusetts
I am Dr. Ajay K. Singh, Clinical Chief, Renal Division and
Director, Dialysis Services and a physician at the Brigham and Women's
Hospital and an Associate Professor of Medicine at Harvard Medical
School. I practice medicine, teach physicians and students, and conduct
patient-oriented research. I am the first author and one of the
Principal Investigators of the CHOIR study. This study examined the
effect of normalizing the hemoglobin with epoetin alfa in patients with
chronic kidney disease not receiving dialysis. My comments today solely
reflect my own views. I plan to discuss 3 issues:
(1) The importance of treating anemia in kidney disease patients,
(2) the optimal hemoglobin in patients with kidney disease, and (3) My
support for bundling of epoetin and other injectibles into the dialysis
composite rate to remove incentives for over-treatment.
1. Anemia of Chronic Kidney Disease
Anemia is highly prevalent among patients with chronic kidney
disease (CKD). By the time patients develop advanced kidney disease
over 60% are anemic and need treatment with an erythropoiesis
stimulating agent (ESA). On dialysis, over 95% of patients require an
ESA. Erythropoetin is the most costly drug for CMS/Medicare--accounting
for over $2 billion.
There is a good body of evidence that supports anemia treatment--
fewer transfusions and improved quality of life, upto a hemoglobin of
10 to 11 grams per deciliter. The recommended strategy for treatment of
anemia is based on guidelines disseminated by the National Kidney
Foundation and the FDA label for epoetin and darbepoetin.
2. The Optimal hemoglobin in patients with kidney disease, based on
current evidence, should be no higher than 12 grams per
deciliter, conforming to the FDA label.
Several randomized controlled studies, both in dialysis and in
predialysis patients, demonstrate at best only modest benefit in
quality of life and increased risk of cardiovascular complications and
death in patients treated to a hemoglobin level that exceeds 12 grams
per deciliter.
It is important to note:
a.) Studies that have shown benefit for cardiovascular outcomes or
survival are retrospective and observational in design. There is broad
consensus that even the best designed and conducted retrospective
observational studies are inferior to randomized controlled studies.
b.) Randomized controlled studies that have looked at patients with
kidney disease, whether on dialysis or not, i.e., in the aggregate,
have demonstrated increased risk.
The normal hematocrit study--increased risk for clotting
of dialysis access, and risk of death or heart attack
The Canada-Europe study--increased risk of stroke
The CHOIR study: a hazard ratio of 1.34 (or a 34% higher
risk) of death and cardiovascular complications.
The higher rate of composite events was explained by a higher rate
of death (48% higher risk, P=0.07) and heart failure hospitalization
(41% higher risk, P=0.07). While quality of life showed improvement
from baseline values in both groups it was similar between the two
groups. However, more patients in the high hemoglobin group experienced
least one serious adverse event compared to the low hemoglobin group.
The CREATE study: an absolute increase in cardiovascular
events and in the time to dialysis.
Whether one looks at studies focused narrowly on the dialysis
population or on predialysis patients, signals for increased risk are
evident with only very modest benefit in quality of life.The National
Kidney Foundation (NKF) Kidney Disease Quality Initiative (KDOQI), the
European Best Practice Guidelines for Anemia and the FDA have all
considered both dialysis and pre-dialysis patients together. The data
on the optimal hemoglobin level has been considered in the aggregate
and applied to both populations.
Collectively, these studies demonstrated risk with normalizing the
hemoglobin in patients with kidney disease on dialysis. The results
reinforce the FDA label for epoetin of not recommending hemoglobin
levels of greater than 12 grams per deciliter in patients with kidney
disease. A final point, it is reassuring that the FDA is empowered with
evaluating the efficacy and safety of drugs in the United States. The
primacy of the FDA in regulating epoetin therapy in the United States
should be maintained.
Hemoglobin levels among Dialysis Patients in the United States
Despite the FDA label, the United States Renal Data System (USRDS)
a large federally funded registry of patients on dialysis, in its 2006
report indicates that more than 40% of dialysis patients have a
hemoglobin level greater than 12 g/dL. Over 20% have hemoglobin levels
above 13 g/dL.
The explanations provided for this include the inability to target
a narrow range of hemoglobin because of a phenomenon termed hemoglobin
cycling and that patients have excursions in hemoglobin levels beyond
the 12 grams per deciliter range for only a very brief period of time.
However, achieving the FDA recommended range is achievable by some
dialysis chains. Only 30% of patients dialyzed at Davita facilities
have hemoglobin levels of less than 12 grams per deciliter, whereas
over 80% of DCI patients are able to maintain their hemoglobin level at
less than 12 grams per deciliter. As well, USRDS data suggests that
excursions over 12 g/dL may occur for 3 or more months. The strategy of
targeting patients using higher epoetin doses to a higher hemoglobin
with these transient excursions could be harmful.
The use of subcutaneous epoetin has been clearly shown to result in
the use of approximately 1/3rd less epoetin yet only a small minority
of dialysis facilities use the subcutaneous route for epoetin
administration.
Despite CMS reimbursement changes, or because of them, data
suggests that the proportion of patients outside of the FDA label
appears to be increasing--some have termed the reason for this as being
driven by ``perverse incentives''.
3. Bundling of injectibles, including epoetin, offers several benefits
and ought to be adopted.
The bundling of injectible drugs into the reimbursement of the
dialysis procedure, i.e., into the composite rate offers several
benefits and should be adopted.
a.) It removes incentives for over-treatment--aiming for higher
hemoglobin levels using higher and higher doses of epoetin.
b.) It will likely reduce the escalating costs for injectible
drugs, particularly epoetin, in the treatment of dialysis patients.
c.) It will encourage the use of subcutaneous administration of
epoetin--a practice widely used in Europe, Canada, and in our own VA
system. This should facilitate lower doses of epoetin in the treatment
of anemia.
Summary:
I recommend that the importance of following the FDA label for
epoetin in the treatment of anemia of kidney disease should be
followed.
a.) The hemoglobin target should not be greater than 12 grams per
deciliter as iterated in the FDA label.
b.) Medicare should modify its reimbursement policy to comply with
the FDA label. Adopting a bundled reimbursement schedule will likely
remove the incentive for higher epoetin use and should increase
subcutaneous administration of epoetin.
The Target Hemoglobin in Patients with Chronic Kidney Disease
Introduction
Anemia is highly prevalent among patients with chronic kidney
disease (CKD) (1). Treatment of CKD anemia with erythropoietin has been
shown to enhance quality of life (2,3), however, evidence supporting a
benefit of anemia correction in improving cardiovascular morbidity and
mortality has been limited and based largely on observational studies
and smaller interventional trials (4-6). These studies have
demonstrated an association of high hemoglobin (>12.0 g/dL) with a
lower rate of cardiovascular morbidity and death (5,6). However, as
others have pointed out elsewhere, observational studies have
limitations (7,8). Primarily, observational designs are unable to
easily adjust for the effect of confounding. Indeed, Parfrey has
recently pointed out that survivor bias could be an important reason
for explaining the discordant findings between observational studies
and randomized controlled trials in anemia (7). Related to this, Cotter
and colleagues have also presented data suggesting that the hemoglobin
level per se may not be a valid surrogate outcome in assessing the true
effect of anemia correction in kidney disease patients (8). A further
problem with the published studies has been that assessment of quality
life may be limited by the open label design of some studies or have
used quality of life instruments that have not been adequately
validated in kidney disease patients (9). The purpose of this article
is to critically appraise these studies in an attempt to arrive at some
conclusions about the optimal target hemoglobin in CKD patients.
Randomized Studies show No Benefit of a Higher Hemoglobin level
Several randomized controlled studies have been published thus far
(10-15). With the exception of two studies, the Normal Hematocrit Study
(10) and the Canada-Europe Study (11), observations have been limited
by the sample size used or premature discontinuation of the study. The
Normal Hematocrit study was a randomized controlled study of
hemodialysis patients with established heart disease comparing a
hematocrit target of 42% to 30%. The study was stopped by the Data
Safety Monitoring Board because of a higher rate of vascular thrombosis
in the patients randomized to the higher hematocrit group. However, the
patients in the higher hematocrit group also had a higher, although not
statistically significantly higher, rate of non-fatal myocardial
infarction (MI) and death. Several explanations were entertained to
explain these findings. These included: the possibility of very high
hematocrit's in the higher hemoglobin group resulted in
hemoconcentration and therefore thrombosis, greater use of iron, and a
lower dialysis dose. The Canada-Europe study also randomized
hemodialysis patients to a higher versus lower hemoglobin (hemoglobin
values of 13.0 versus 11.0 g/dL, respectively). However, in contrast to
the Normal Hematocrit Study, Parfrey et al selected patients that were
not at high risk of cardiovascular disease by excluding patients with
symptomatic heart disease as well as those with left ventricular
dilatation at baseline. Moreover they enrolled incident dialysis
patients. While they did not evaluate hard endpoints such as death, or
myocardial infarction, or stroke, they did evaluate changes in cardiac
geometry (left ventricular volume index (LVVI) and left ventricular
mass index (LVMI). Moreover, they assessed heart failure and quality of
life. No significant benefit in either of the cardiac structural or
functional parameters was observed in the high versus low hemoglobin
groups. However, a statistically significantly higher rate of
cerebrovascular accident in the higher hemoglobin group was observed on
secondary analysis. Quality of life did show an important difference in
the high versus lower hemoglobin group with respect to the Vitality
score, which was improved over time in patients randomized to the
higher hemoglobin. In this regard, the Canada-Europe results were
concordant with the Normal Hematocrit Study that also showed
improvement in specific quality of life domains in the high versus low
hematocrit groups.
Hemoglobin Variability Necessitates a Broader Hemoglobin Range
An important problem with setting hemoglobin targets has become
apparent from recent studies that have evaluated the variability of
hemoglobin levels in patients on epoetin treatment in the dialysis
setting. Three studies collectively suggest that it is difficult to
maintain the hemoglobin level in the 11 to 12 g/dL range (16-18). In
the study by Fishbane et al >90% of dialysis patients studied
experienced hemoglobin cycling (16). These investigators reported that
the mean number of hemoglobin excursions was 3.1 1.1 per
patient/year. The mean amplitude per hemoglobin excursion was 2.51
0.89 g/dL, and the mean duration of hemoglobin excursions
was 10.3 5.1 weeks. Indeed, the NKF Work Group has
``rejected, identifying a target hemoglobin level bounded by narrow
upper and lower values (e.g., 11.0 to 12.0 g/dL) (15). Such a target
affords neither clarity nor simplicity, is possible to achieve in only
a minority of patients, discourages flexibility in managing individual
patients, and likely promotes cycling of hemoglobin results greater
than and less than the target.''
Recent New Data from Randomized Controlled Trials
The recent publication of the CHOIR and CREATE studies informs the
debate regarding the target hemoglobin level in CKD patients (19,20).
CHOIR was an open-label,randomized trial that studied 1432 patients
with CKD: 715 patients randomized to receive epoetin alfa targeted to
achieve a hemoglobin of 13.5 g/dL, and 717 were randomized to receive
epoetin alfa targeted to achieve a hemoglobin of 11.3 g/dL (19). The
median study duration was 16 months. The primary end point was a
composite of death, myocardial infarction, congestive heart failure
(CHF) hospitalization (excluding hospitalization during which renal
replacement therapy occurred), and stroke. Two-hundred-twenty-two
composite events occurred: 125 events among the high hemoglobin group
and 97 events among the low hemoglobin group P=0.03, hazard ratio of
1.34; with 95 percent confidence interval of 1.03 and 1.74. The higher
rate of composite events was explained largely by a higher rate of
death (48% higher risk, P=0.07) or CHF hospitalization (41%, P=0.07).
Although neither death nor CHF hospitalization were statistically
significantly higher in the higher versus lower hemoglobin group, the
study was not powered for this purpose. While quality of life showed
improvement from baseline values in both groups and were similar
between the two groups. However, more subjects in the high hemoglobin
group experienced least one serious adverse event compared to the low
hemoglobin group. The Cardiovascular Risk Reduction by Early Anemia
Treatment with Epoetin beta (CREATE) study enrolled approximately 600
patients. Subjects were randomized to an early anemia correction or a
late anemia correction group (20). The early anemia correction group
received epoetin beta therapy immediately for a target hemoglobin 13-15
g/dL. The late anemia correction group did not receive treatment until
their hemoglobin is >10.5 g/dL; their target hemoglobin was 10.5-11.5
g/dL. The study showed that ``complete correction'' was not associated
with a statistically significantly higher rate of the first
cardiovascular event (58 events in the high hemoglobin group versus 47
events in the low hemoglobin group; hazard ratio of 0.78, 95%
confidence interval, 0.53 to 1.14; P=0.20). However, left ventricular
mass index remained stable in both groups but dialysis was required in
more patients in the higher versus lowed hemoglobin group (127 vs. 111,
p=0.03). On the other hand, unlike CHOIR, in CREATE a quality of life
benefit, at least in year 1 of the study, was observed for the higher
versus lower hemoglobin group.
Therefore, both studies showed either risk or no benefit in aiming
to completely correct the hemoglobin in CKD patients, not receiving
dialysis. The CHOIR study was larger and showed a statistically
significant difference for the primary endpoint, whereas the CREATE
study was much smaller and showed a trend for increased risk but did
not reveal statistically significant differences for the primary
endpoint. It is important to note that, unlike the Normal Hematocrit or
Canada-Europe studies, both CHOIR and CREATE evaluated pre-dialysis CKD
patients and so the results may not be generalizable to the dialysis
community. However, both the Normal Hematocrit and the Canada-
Europestudies in dialysis patients also demonstrated either no benefit
or increased risk. Collectively, this data strongly suggests that the
most prudent course is to partially correct the hemoglobin in all
chronic kidney disease patients, whether on dialysis or not, until more
data is available in future studies.
References
1. Astor BC, Muntner P, Levin A, Eustace JA, Coresh J. Association
of kidney function with anemia: the Third National Health and Nutrition
Examination Survey (1988-1994). Arch Intern Med. 2002 Jun
24;162(12):1401-8.
2. Jones M, Ibels L, Schenkel B, Zagari M. Impact of epoetin alfa
on clinical end points in patients with chronic renal failure: a meta-
analysis. Kidney Int. 2004 Mar;65(3):757-67.
3. Ross SD, Fahrbach K, Frame D, Scheye R, Connelly JE, Glaspy J.
The effect of anemia treatment on selected health-related quality-of-
life domains: a systematic review. Clin Ther. 2003 Jun;25(6):1786-805.
4. Levin A. Anemia and left ventricular hypertrophy in chronic
kidney disease populations: a review of the current state of knowledge.
Kidney Int Suppl. 2002 May;(80):35-8.
5. Collins AJ, Li S, St Peter W, Ebben J, Roberts T, Ma JZ, Manning
W. Death, hospitalization, and economic associations among incident
hemodialysis patients with hematocrit values of 36 to 39%. J Am Soc
Nephrol. 2001 Nov;12(11):2465-73.
6. Xue JL, St Peter WL, Ebben JP, Everson SE, Collins AJ. Anemia
treatment in the pre-ESRD period and associated mortality in elderly
patients. Am J Kidney Dis. 2002 Dec;40(6):1153-61.
7. Parfrey PS. Target hemoglobin level for EPO therapy in CKD. Am J
Kidney Dis. 2006 Jan;47(1):171-3.
8. Dennis J. Cotter, Kevin Stefanika, Yi Zhang, Mae Thamer, Daniel
Scharfstein, James Kaufman: Hematocrit was not validated as a surrogate
end point for survival among epoetin-treated hemodialysis patients
Journal of Clinical Epidemiology 57 (2004) 1086--1095
9. Strippoli GF, Manno C, Schena FP, Craig JC.Paoletti E, Cannella
G. Haemoglobin and haematocrit targets for the anaemia of chronic renal
disease. Cochrane Database Syst Rev. 2003;(1):CD003967.
10. Besarab A, Bolton WK, Browne JK, Egrie JC, Nissenson AR,
Okamoto DM, Schwab SJ, Goodkin DA. The effects of normal as compared
with low hematocrit values in patients with cardiac disease who are
receiving hemodialysis and epoetin. N Engl J Med. 1998 Aug
27;339(9):584-90.
11. Parfrey PS, Foley RN, Wittreich BH, Sullivan DJ, Zagari MJ,
Frei D. Double-blind comparison of full and partial anemia correction
in incident hemodialysis patients without symptomatic heart disease. J
Am Soc Nephrol. 2005 Jul;16(7):2180-9. Epub 2005 May 18.
12. Foley RN, Parfrey PS, Morgan J, Barre PE, Campbell P, Cartier
P, Coyle D, Fine A, Handa P, Kingma I, Lau CY, Levin A, Mendelssohn D,
Muirhead N, Murphy B, Plante RK, Posen G, Wells GA. Effect of
hemoglobin levels in hemodialysis patients with asymptomatic
cardiomyopathy. Kidney Int. 2000 Sep;58(3):1325-35.
13. Roger SD, McMahon LP, Clarkson A, Disney A, Harris D, Hawley C,
Healy H, Kerr P, Lynn K, Parnham A, Pascoe R, Voss D, Walker R, Levin
A. Effects of early and late intervention with epoetin alpha on left
ventricular mass among patients with chronic kidney disease (stage 3 or
4): results of a randomized clinical trial. J Am Soc Nephrol. 2004
Jan;15(1):148-56.
14. Levin A, Djurdjev O, Thompson C, Barrett B, Ethier J, Carlisle
E, Barre P, Magner P, Muirhead N, Tobe S, Tam P, Wadgymar JA, Kappel J,
Holland D, Pichette V, Shoker A, Soltys G, Verrelli M, Singer J.
Canadian randomized trial of hemoglobin maintenance to prevent or delay
left ventricular mass growth in patients with CKD. Am J Kidney Dis.
2005 Nov;46(5):799-811.
15. McMahon LP, Roger SD, Levin A; Slimheart Investigators Group.
Development, prevention, and potential reversal of left ventricular
hypertrophy in chronic kidney disease. J Am Soc Nephrol. 2004
Jun;15(6):1640-7.
16. Fishemoglobinane S, Berns JS: Hemoglobin cycling in
hemodialysis patients treated with recombinant human erythropoietin.
Kidney Int 68:1337-1343, 2005
17. Lacson E Jr, Ofsthun N, Lazarus JM: Effect of variability in
anemia management on hemoglobin outcomes in ESRD. Am J Kidney Dis
41:111-124, 2003
18. Berns JS, Elzein H, Lynn RI, Fishemoglobinane S, Meisels IS,
Deoreo PB: Hemoglobin variability in epoetin-treated hemodialysis
patients. Kidney Int 64:1514-1521, 2003
19. Singh AK, Szczech L, Tang KL, Barnhart H, Sapp S, Wolfson M,
Reddan D: Correction of Anemia with epoetin alfa in chronic kidney
disease. New Engl J Med 2006; 355: 2085-98
20. Drueke TB, Locatelli F, Clyne N, Eckhardt KU, Macdougall IC,
Tsakiris D, Burger HU, Scherhag A. Normalization of hemoglobin levels
in Chronic Kidney Disease and Anemia. New Engl J Med 2006; 355: 2071-84
Chairman THOMAS. Thank you very much, Dr. Singh.
Mr. Cotter.
STATEMENT OF DENNIS J. COTTER, PRESIDENT, MEDICAL TECHNOLOGY
AND PRACTICE PATTERNS INSTITUTE,
BETHESDA, MARYLAND
Mr. COTTER. Chairman Thomas, Congressman Rangel,
Congressman Stark, distinguished members. Good morning. I am
Dennis Cotter, President of the Medical Technology and Practice
Patterns Institute. I appreciate the opportunity today to talk
about patient safety and quality issues. We have studied
clinical outcomes of ESRD patients for more than 10 years. For
almost two decades great controversy has surrounded the anemia
treatment management goal; that is the target hematocrit.
During this time, they have increased hematocrit from 33
percent to 37.5 percent; most recently to 39 percent and
higher. Were these charges warranted? The answer to this
question became clear when results of new clinical trials
joined with earlier clinical trials demonstrated that patients
targeted to higher hematocrit levels have increased mortality
and many other adverse side effects. Through the current rules
which endorse expanding EPO reimbursement to allow hematocrit
to be targeted to any level, CMS has implemented a policy that
can be harmful to its beneficiaries and will cost hundreds of
millions of dollars in additional expenditures.
For some patients, it takes a small amount of EPO to
elevate EPO hematocrit. For others, it takes a large amount.
Clinical trials have shown that those targeted to high
hematocrits and high EPO doses have higher mortality rates to
those targeted to low hematocrit and low EPO doses. Because the
population is made up of both EPO responders and EPO non-
responders, the question remains whether patients who
experience higher mortality rates were predominantly EPO
responders or non-responders.
It is unlikely that industry sponsored research will answer
this important question. Answering this question is the subject
of our ongoing NIH funded research that addresses the concern
that EPO therapy itself might contribute to harmful outcomes.
Current CMS policy and industry sponsored clinical practice
guidelines support both high target hematocrit and high EPO
doses, assuming that high hematocrits improve outcomes, an
assumption that is contrary to clinical trial. To date, no
normal assessment of the appropriate dosing level has been
conducted nor has a payment policy been implemented to
encourage optimal dosing. Removal of a profit incentive by
adding EPO to the composite rate should reduce over utilization
and would also encourage research to determine optimal dosing.
CMS policy appears to be heavily weighted on both the opinion
and the notion that hematocrit variability is the overriding
problem. As a result, this has encouraged EPO over-utilization,
driving higher Medicare payments. Given the new policy, which
opens up the upper limit of the target hematocrit, it is
anticipated that providers will respond to the financial
incentive with even more aggressive use of EPO.
Our recommendations: Adhere to the FDA label until further
studies clarify the causal link between EPO, hemocrit and
patient outcomes. Following FDA dose titration recommendations
should be sufficient to maintain hematocrits within the 30-36
percent hematocrit range, deemed to be the safest range for all
patients.
Treatment guidelines and reimbursement policies must put
restrictions on the level of EPO dose, if necessary.
Further studies are needed to study patients that are
hyporesponsive to high EPO doses. Regarding EPO and for all
future drug evaluations, avoid the over reliance on
observational studies, often industry sponsored as opposed to
rigorously controlled randomized clinical trials. It is
imperative that EPO coverage decisions adhere to established
hierarchy of evidence that focuses primarily on RCTs and
systematic reviews.
Finally, promote research which is independently funded
rather than industry sponsored for the development of treatment
guidelines and payment policies.
Thank you for your consideration of our concerns.
[The prepared statement of Mr. Cotter follows:]
Statement of Dennis J. Cotter, President, Medical Technology and
Practice Patterns Institute, Inc., Bethesda, Maryland
Chairman Thomas, Congressman Stark, distinguished Committee
members, good morning. I am Dennis J. Cotter, President of the Medical
Technology and Practice Patterns Institute. I appreciate the
opportunity to talk about patient safety and quality issues. We have
studied clinical outcomes of ESRD patients for more than 10 years.
For almost two decades, great controversy has surrounded the anemia
management treatment goal, that is, the target hematocrit. During this
time, CMS has increased hematocrit targets, from 33% to 37.5%, and most
recently, to 39% and higher. Were these changes warranted? The answer
to that question became clear when results of new clinical trials,
joined with earlier trial results, demonstrated that patients, targeted
to higher hematocrit levels, have increased mortality and many other
adverse side effects. Through the current rules which endorse expanding
EPO reimbursement to allow hematocrit to be targeted to any level, CMS
tacitly has implemented a policy that can be harmful to its
beneficiaries and will cost hundreds of millions of dollars in
additional expenditures.
THE SCIENCE
For some patients, it takes a small amount of EPO to elevate the
hematocrit (EPO responders) and, for others, its takes a large amount
(EPO non-responders). Clinical trials have shown that those targeted to
high hematocrits and high EPO doses have higher mortality rates than
those targeted to low hematocrits and low EPO doses. Because the
population is made up of both EPO responders and EPO non-responders,
the question remains whether patients who experienced higher mortality
rates were predominately EPO responders or EPO non-responders. It is
unlikely that industry-sponsored research will answer this important
question. Answering this question is the subject of our on-going NIH
funded research which addresses the concern that EPO therapy, itself,
might contribute to harmful outcomes. Current CMS policy and industry-
sponsored clinical practice guidelines support both high target
hematocrit and high EPO doses, assuming that high hematocrits improve
outcomes, an assumption that is contrary to clinical trial results. To
date, no formal assessment of the appropriate dosing levels has been
conducted, nor has a payment policy been implemented to encourage
optimal dosing. Removing the profit incentive, by adding EPO to the
composite rate should reduce over-utilization and would also encourage
research to determine optimal dosing.
THE POLICY
CMS policy appears to be heavily weighted both on opinion and on
the notion that hematocrit variability is the over-riding problem. As a
result, over the years this policy has encouraged EPO over-utilization,
driving higher Medicare payments. Given the new policy, which opens the
upper limit of the target hematocrit, it is anticipated that providers
will respond to the new financial incentive with even more aggressive
use of EPO.
WHERE WE GO FROM HERE
Our recommendations are the following:
Adhere to the FDA-approved label until further studies
clarify the causal link among EPO, hematocrit, and patient outcome.
Following FDA dose titration recommendations should be sufficient to
maintain hematocrits within the 30-36% hematocrit range, deemed to be
the safest range for all patients.
Treatment guidelines and reimbursement policies must put
restrictions on the level of EPO dose, if necessary. Further studies
are needed of patients who are hypo-responsive to high EPO doses.
Regarding EPO, and for all future drug evaluations, avoid
over-reliance on observational studies, often industry-sponsored, as
opposed to rigorously controlled randomized clinical trials. It is
imperative that the EPO coverage decisions adhere to established
hierarchy of evidence that focuses primarily on RCTs and systematic
reviews.
Promote research which is independently funded, rather
than industry-sponsored, for the development of treatment guidelines
and payment policies
Thank you for your consideration of our concerns.
Chairman THOMAS. Thank you all very much.
I will ask a couple of questions and urge my colleagues to
focus their responses as well. History is history, but we now
have some fairly clear evidence. Can you think of a worse
system to treat patients with End Stage Renal Disease than
having facilities offering the service not getting updates or
cost-of-living adjustments but in fact a fixed dollar payment
for years and having a drug which is a significant assistance
in a monopoly situation with no competitive pricing structure
being available and, in fact, encouraged in terms of increased
uses. Now we see clearly beyond what most people are now
beginning to believe would be appropriate standards. What would
we add to that to make it worse than the structure we now have?
Is there anything we are missing in terms of policy that could
make sure that we don't endanger these people even more?
Mr. COTTER. This is a recipe for disaster. That is why we
are here today. EPO dosing is a--it is done under the notion
that the drug does no harm. Because of that notion, there is a
very aggressive attitude toward using high doses of EPO to
continue.
Chairman THOMAS. That, my understanding, is the way it is
administered, in terms of not allowing any new or inventive
approaches, for example subcutaneous, in terms of advantages.
There is no ability to continue to move toward better practices
within the structure that we have established, i.e., there is
no incentive and I don't think anyone should focus on the
people who are running these services and who are doing the
best job they can. It is the structure under which they are
operating these services that don't allow them to move into
those. Is that an appropriate statement?
Mr. COTTER. This is viewed as an income stream. If it was
viewed by providers as a cost, then the incentive would be to
provide optimum dosing as does the VA. The VA doses using the
subcutaneous route, and that, within the VA, EPO treatment is
viewed as a cost. So, if you change the incentives around, you
will motivate providers to become much more efficient.
Chairman THOMAS. Dr. Singh, you made a statement about your
study about comparisons with people who are not in end stage
renal but obviously chronic kidney disease and other patients.
Notwithstanding the exclusivity of drugs used in each of these
areas, i.e. monopoly, did you find any difference, any
significant difference that would require a maintenance of
drugs dealt with in a different way for those populations, or
was there sufficient commonality from your study that we should
look at that was more of a combined group and therefore
possibly have an opportunity to deal with what is now a
monopoly in the End Stage Renal Disease of administering of
drugs?
Dr. SINGH. I agree with you. I think the study that we
published as well as other studies do not provide sufficient
evidence at the present time to distinguish treatment in one
population versus the other. In other words, at this moment I
think, like the approach taken by the FDA as well as by the
National Kidney Foundation, it seems reasonable to consider
these populations and studies on these populations in the
aggregate and nothing--there has been no convincing evidence
that has supported the idea of separating these populations
out. There is no reason in my mind to think that the dialysis
population will benefit from higher doses of EPO or from higher
hemoglobin levels beyond what we have found in our studies. In
fact two other randomized controlled studies support the notion
of increased risk in the dialysis population.
Chairman THOMAS. Why was the CHOIR trial terminated?
Dr. SINGH. It was terminated because the Data Safety
Monitoring Board saw evidence for increased risk for adverse
risk in the higher hemoglobin group, and they found that there
was going to be no likelihood of showing any benefit, and
therefore, the DSMB terminated the study. We followed that
recommendation.
Chairman THOMAS. The policies that were currently
advocating under CMS are which end of the spectrum vis-a-vis
the CHOIR trial? At the high end or the low end?
Dr. SINGH. The Medicare policy recommends there be a 25-
percent reduction in the epoetin dose when the hemoglobin level
hits 13 grams or hematocrit at 39 percent what's remarkable is
that as recent, as the same week as the CHOIR study was
published, one of the largest dialysis providers circulated a
guideline for protocol for hemoglobin management which
recommended only a 10-percent reduction in epoetin dose when
the hemoglobin level reaches 13 grams and a 25-percent
reduction when it reaches 14 grams or higher, so, clearly, even
beyond what Medicare recommends and certainly well beyond what
the CHOIR data suggests, which is considered safe. So, you are
absolutely right. There is--not only does this study suggest
increased risk beyond 13, beyond actually 12.5 because that was
the achieved hemoglobin level. But Medicaid guidelines are at
13, where there is a reduction and dialysis providers are even
flaunting that and going for higher levels still.
Chairman THOMAS. It is in large part because of the payment
system and the structure we are dealing with and some
incentives of the structure.
Can you give us--is there agreement between the three of
you and more than the decade long study and others that would
give us one, two, three fairly simplified steps that we could
take that could at least get us significantly in a different
direction? Can you give it to me in just a few terms?
Obviously, you have advocated bundling. Are there any other
suggestions?
Dr. SINGH. I would recommend they should be--risk
adjustment of the population has been recommended by CMS
because these are complicated sick dialysis patients and we do
not want to actually disincentivize the treatments of patients
who are sick.
Chairman THOMAS. I understand that. Have you read the GAO
study?
Dr. SINGH. Yes.
Chairman THOMAS. One of the concerns that we have that we
will be presented shortly is we have made recommendations; we
have demanded certain aspects. The argument has been that we
have not been able to develop them.
Thank you very much.
Mr. Rangel.
Mr. RANGEL. Thank you so much for sharing your views with
us.
Could you tell us, why in the world would the CMS have a
policy that differs from with the GAO, the FDA, the National
Kidney Foundation, the National Institutes of Health, the
providers of health care, as to why they would want to
encourage through policy the overuse of a drug that places
peoples' lives at risk and even death and is more costly to the
Federal Government? Now just try to think of any reason why any
agency or department of the Federal Government would want to do
this.
Mr. COTTER. Yes, I think, from my understanding of this, we
wrote a policy analysis of this and published in the Health
Affairs Journal, is that there is a notion that the policy must
impress hematocrit variability. However, that is driven by
aggressive dosing so it is a self-fulfilling prophecy. If you
raise the hematocrit target, you encourage more aggressive
dosing which in turn raises more variability. That is the only
argument that CMS has had that they have claimed that the
policy is based on. They have claimed that there is no science
to support these high levels. It is only this issue that is
non-scientific of hematocrit variability.
Dr. PIZZI. Mr. Cotter used the word notion. The notion has
been in the dialysis community for several years that the
higher the hemocrit, the better the outcome. Even the National
Kidney Foundation updated their guideline this year to increase
the upper hemoglobin limit to 13 without founded evidence to
support that. Now the results of the trials testing these
levels doses are starting to come back, and, in fact, we have
found out that 13 is too high. CMS, as well as the National
Kidney Foundation, I believe and I agree with Mr. Cotter and
Dr. Singh that it is time to revert back to the label, which is
a hemoglobin of 10 to 12.
Dr. SINGH. I think we have limited presentation at the
present time as to why this occurs and whether it is related to
factors such as high dosing or frequent measurement of
hemoglobin. Nevertheless, we certainly should seek increased
risk of hemoglobin levels that are beyond the current label of
the drug and my advice would be that we take greater pains and
I think CMS takes greater pains to ensure that the population
of dialysis patients that this represents a vulnerable
population of hemoglobin levels that are below that level until
we get more data, until more studies are published to indicate
this is a--that the alternative is a reasonable strategy.
Mr. RANGEL. Thank you. Mr. Chairman, I want to congratulate
you for the order of the witnesses, to have the experts testify
early. Whether we can establish this as some kind of a policy,
that makes it easier for us.
Chairman THOMAS. I appreciate the gentleman. I am doing
this for pure convenience of members in a very extreme time,
and I don't intend to make any kind of a precedent. I am
walking out the door in a few days, and you can deal with it on
your own time.
The gentlewoman from Connecticut wish to you inquire.
Ms. JOHNSON OF CONNECTICUT. How easy is it to achieve
stability in a patient to--how big an issue is this hematocrit
variability? I gather from you that we really don't know enough
to actually keep a patient stable at 12, or we would be doing
it.
Mr. COTTER. We have done a study of this because we wanted
to prepare something for this hearing. It is simply that if you
maintain a prudent dosing strategy as recommended in the FDA
labeling, you start off in small doses and you tritiate up
until the patient responds to the drug, you do not have this
variability. What providers are doing now, they are starting
with very high doses beyond the FDA label and these patients,
some of them overshoot. Some don't. Remember, I said we have
responders and non-responders to EPO therapy. So, for those
both responders and non-responders, they get the same dose.
Ms. JOHNSON OF CONNECTICUT. Thank you. So, the variability
issue can be addressed by titrating up, and if you do that,
then there is no difference in frailty or nature of the patient
as to how variable, how much their swings and their hemocrit
levels as you dose.
Mr. COTTER. That is why you want to titrate up.
Ms. JOHNSON OF CONNECTICUT. Once you get there, are there
still those swings?
Mr. COTTER. There could be swings, sure. But the FDA label
says that the target should be 30 to 26, so that allows the
swings.
Ms. JOHNSON OF CONNECTICUT. So, you think the swing being
allowed to go up to 13 is too big a swing?
Mr. COTTER. Absolutely.
Ms. JOHNSON OF CONNECTICUT. How effective has the CMS new
payment policy adopted in April been in at least bringing down
dosing above 13?
Mr. COTTER. Well, according to the GAO report, I will let
them tell you about that, but it looks like dosing is migrating
up slightly right now, but they are reporting an average dose.
That means there are some patients on the high end of that
average are getting very high doses.
Ms. JOHNSON OF CONNECTICUT. On the bell curve, their new
payment policy penalizes high doses of patients that are at 12/
13, and my understanding is, we will hear more from the next
panel, but that has brought down the outliers. Is that your
understanding?
Mr. COTTER. I would like to hear CMS defend that because,
in October of this year, they virtually annihilated that
restriction. It is basically not there any longer.
Ms. JOHNSON OF CONNECTICUT. The 25 percent payment cut?
Mr. COTTER. I don't know how a contractor could implement
that direction that they are giving him, and by the way, the
target is not even 39 percent. It could go higher. It can go to
42. It can go to 50. There is no higher bound.
Ms. JOHNSON OF CONNECTICUT. But there are payment cuts----
Mr. COTTER. The only payments cut is that, if a monthly
dose goes over 500,000 units per or more, which is 5 to 10
times above the FDA label; it is bizarre.
Chairman THOMAS. Thank you.
Mr. Stark, do you wish to inquire.
Mr. STARK. I want to thank the panel for all of the work
they have done in this area. I have a couple of, I guess, less
technical questions.
But my understanding is that if we--I can't pronounce
subcutaneous, but I am going to ask Dr. Pizzi, if we inject the
drug, then we might be able to use about 30 percent less; is
that correct?
Dr. PIZZI. That is true. There are numerous studies that
indicate that. That is one of the standard ways to introduce
savings because the drug essentially acts as a depot.
Mr. STARK. Could the makers of the drug make it more
comfortable for us who don't like getting stuck?
Dr. PIZZI. The key is that to make it more comfortable, the
clinics have a multi-dose vial which has a benzyl-alcohol in
it, and that reduces some of the stinging. There are other
techniques that can be used to reduce discomfort, too.
Mr. STARK. I am all for that.
Then the other question is, would it make any sense, Dr.
Singh, if you know, in paying for the application of this drug
as it is now used in the dialysis system, why we should pay
more than a dollar per application, not counting the cost of
the drug? It is just dumped in the system, I gather, and takes
no particular--nothing more than adding it to the mix. Is that
a fair layman's description of how you would add this if you
are not injecting it?
Dr. SINGH. I think that is, sir. It is a little bit more
complicated than just adding it. I think that there is a
significant--there is a significant amount of work that goes in
to the administration in a safe manner to prevent----
Mr. STARK. Even when it is done as----
Dr. SINGH. Even when it is done as part of it. So, I don't
think I would advocate the idea that we just add it in a sort
of routine manner. However, I would just add that the notion
that we can give it subcutaneously is not a bad idea. After
all, our veterans in this country received this drug
subcutaneously largely. People in Europe and people in Canada.
So, I do agree that it is--there is some discomfort giving it
subcutaneously, but it is certainly good enough for some of our
finest people.
Mr. STARK. We are talking about saving 600 million bucks a
year, which is not chump change.
Final question, Dr. Singh. With all of this recent
information about overdosing things, how have you changed both
your own practice, and at Brigham Young, how are you changing
what you teach your residents or interns?
Dr. SINGH. I am the medical director of a dialysis unit in
Boston. We have instituted a new protocol to ensure that the
patients' hemoglobin levels stay below the--to the degree we
can help it below 12 grams per deciliter. The USRDS shows that
chain in which I practice has 80 percent of their patients that
are below 12 grams per deciliter in contradistinction to other
dialysis providers who have higher proportional patients.
We have also instituted modifications for our health care
system partners, health care in Boston, to try to ensure that
we follow the FDA label. Certainly teaching people out there
both at the American Society of Nephrology and elsewhere, I
certainly advocate the notion that we should follow the FDA
level and stay below 12 grams per deciliter.
Mr. STARK. There is always the danger of underdosing. We
certainly don't want to have payment practices that would
encourage that. But in the general practice in dialysis today,
how would you rank the danger of overdosing as a--or
underdosing? Is there as much danger in underdosing as
overdosing?
Dr. SINGH. I think the biggest problem currently is the
fact that patients are coming to dialysis still who have never
ever been treated with epoetin and should be. So, certainly, we
could increase the awareness with the population with
increasing treatment well before they start dialysis. However,
I do not believe that underdosing will be as much of an issue
as much as overdosing, especially since the studies now show
continued increased risk.
Chairman THOMAS. Did I hear you say, Dr. Singh, given the
current payment system and the structure of the monopoly drug,
that you are following a policy which reduces the income to
those dialysis centers that you are involved with?
Dr. SINGH. We have taken the approach that we will follow
what is appropriate from a clinical standpoint, and if that
means that we get paid less, that is appropriate. But we want
to make sure that we treat patients to a level of hemoglobin--
--
Chairman THOMAS. I understand that, but what you have done
is made a conscious decision based upon your knowledge not to
follow a system which you could easily follow. Wouldn't it make
a whole lot of sense to change the system so you don't have to
make that decision?
Dr. SINGH. Yes, sir. I agree with you.
Chairman THOMAS. Any additional questions?
Mr. CAMP. I don't have a question. I want to associate with
your remarks.
Mr. LEWIS OF GEORGIA. Mr. Singh, I understand that 30
percent of dialysis patients are African-American, but yet we
are only 8 to 12 percent of the total population. These numbers
are not improving, and we are not seeing any improvement in
keeping patients off of dialysis. What do you need from
Congress to help prevent patients from going on dialysis? In
the minority community, what needs to be done to address this
disparity.
Dr. SINGH. Thank you, sir. Recent data from the--published
from the United States Renal Data System from the NIH suggest
there may be a leveling off of the incident rate of patients
starting dialysis. But the--but your point is well taken. We
still have an unsatisfactory number of people starting dialysis
in the United States, and it is over-representative of
minorities, particularly African-Americans. We also have no
change in the mortality or no significant change in the
mortality of patients on dialysis.
If you ask me, what we need to do is to increase the
greater funding to the NIH so that independent research can be
performed by investigators in the United States to understand
what factors increased the risk of progression of patients,
particularly African-Americans, to dialysis; what are the
factors that account for this very high risk of cardiovascular
disease among dialysis patients; as well as studies on anemia,
to try and truly understand this issue of hemoglobin cycling
and why there is this excess risk should be funded by Congress.
I think we need--we certainly need more money funded through
the appropriate channels through the NIH to fund more studies.
But I certainly agree with you that African-Americans
represent a disproportionate amount of people on dialysis, and
the mortality of these people have not changed significantly
over the past decades. So, we do need help, and we need help in
terms of funding in the research community to gain a great
understanding and develop better strategies.
Chairman THOMAS. Thank the gentleman. We are calling it End
Stage Renal Disease, and what we really need to put emphasis on
is prevention and education so they never reach the end stage
along with the additional study that you are making, and
frankly, that doesn't take an NIH study. That takes talking
about diet, lifestyle and the rest.
Thank you very much for your research.
Again, it is quite amazing that, all of a sudden, in a
couple of months, significant research is coming out focusing
on this issue. I assume that there is going to be continued
examination of this. Obviously, we need all the help we can get
in terms of not only understanding the application of these
drugs that are literally miracle drugs but the manner in which
we provide it to people who provide the service and in fact the
taxpayers pay for it. Thank you very much.
I would now ask the second panel to please come before us.
The Chair is conscious of the time restraints on very busy
people.
The Chair is pleased to have, once again, the Honorable
David M. Walker, and I would like to welcome Leslie Norwalk,
who is the new Acting Administrator for the Centers for
Medicare and Medicaid Services. Thank you very much for
attending.
Dr. Walker, obviously, you have just concluded the study.
Again, interestingly, all of this is coming together at the
same time, and we have it available if anyone hasn't seen a
copy of it. I do think it is very useful, and as is customary,
any testimony that you have written will be made a part of the
record, and you can address us in any way you see fit in terms
of what we now have before us in the GAO study.
STATEMENT OF THE HONORABLE DAVID M. WALKER, COMPTROLLER
GENERAL, U.S. GOVERNMENT ACCOUNTABILITY OFFICE
Mr. WALKER. Thank you, Mr. Chairman. I appreciate the
opportunity to be here today to participate in the hearing on
Medicare patients with End Stage Renal Disease. Let me also
note, happy birthday and all of the best to you in retirement
from the Congress.
As you know, from a broader perspective, the level and
growth of Medicare spending combined with the over $30
trillion-plus amount of unfunded obligations for Medicare
serves as evidence that the current program is unsustainable in
its present form. Furthermore, Medicare's sheer size and
complexity make it vulnerable to improper payments and
inefficient payment systems. Over the years, the GAO has worked
with this Committee to try to address these challenges. CMS has
taken a number of related efforts, and some progress clearly
has been made, but as most GAO reports note, more remains to be
done.
With regard to End Stage Renal Disease drug reimbursements,
the GAO report which you held up--and I have a copy as well
that you requested--points to ways to improve the efficiency of
Medicare's payments in connection with the End Stage Renal
Disease program. Over the years, we have observed that bundled
payments tend to be more efficient than paying for services one
at a time. Bundled payments cover a range of services delivered
to the patient. As such, they give providers an incentive to
furnish only those services that patients truly need because
providers can not prosper by providing extra services.
Today's hearing focuses on End Stage Renal Disease paid
under part B of Medicare. But one has to look at the whole part
of part B to see the inefficiencies inherent in paying for
services one at a time. Spending for physician services and
other part B services over the past several years has been
growing at an alarming rate. It is essential that Congress find
ways to restructure payments to institute necessary
efficiencies and control spending growth while maintaining
quality and assuring patient safety.
Our report on End Stage Renal Disease drug payments
observes that the current method of setting drug payment rates
is an improvement over the previous system. However, it does
not provide appropriate incentives. It does not control the
incentive to over-utilize such drugs. The system in place pays
providers the manufacturers' average sales price for the drug,
plus 6 percent. Any system that provides for cost-plus payments
provides an incentive for related parties to provide
unnecessary care and extra services. Indeed, this is one of the
reasons that the Congress changed Medicare's inpatient hospital
payment to a bundle payment system in the mid-eighties.
We also observed in our report that Medicare's End Stage
Renal Disease drug payment is dominated by a single drug,
Epogen, which for several years has been Medicare part B's
highest spending drug, approximately $2 billion in 2005. We
also expressed concerns that there are currently no direct
competitor drugs in the End Stage Renal Disease market. The
lack of effective price competition could be having
considerable adverse effects on Medicare's overall spending.
Furthermore, the lack of significant efforts to verify the
accuracy of the average sales price for drugs that are
separately billable under part B is also a matter of concern,
and that goes beyond End Stage Renal Disease.
Finally, returning to the bundling theme. We observed that,
currently, congressionally mandated research on creating a
bundled system for End Stage Renal Disease services, including
drugs, has been delayed. The research being conducted by CMS
would, among other things, ensure that providers are
appropriately compensated for variations of complexity in
patients' treatment. While this is important, we do not believe
it is necessary or desirable to delay movement to a bundled
rate for End Stage Renal Disease services any longer than
absolutely necessary. For this reason, we have suggested that
Congress consider mandating the establishment of a bundle
payment system for all ESRD services, including drugs, as soon
as possible.
Mr. Chairman, this concludes my prepared remarks. I look
forward to responding to your questions.
Thank you.
[The prepared statement of Mr. Walker follows:]
Statement of The Honorable David M. Walker, Comptroller General, U.S.
Government Accountability Office
Mr. Chairman and Members of the Committee:
I am pleased to be here to discuss highlights from our report
entitled End-Stage Renal Disease: Bundling Medicare's Payment for Drugs
with Payment for All ESRD Services Would Promote Efficiency and
Clinical Flexibility.\1\ The report examines Medicare payments for
certain drugs provided to patients with end-stage renal disease (ESRD),
a condition of permanent kidney failure.\2\
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\1\ GAO, End-Stage Renal Disease: Bundling Medicare's Payment for
Drugs with Payment for All Services Would Promote Efficiency and
Clinical Flexibility, GAO-07-77 (Washington, D.C.: Nov. 13, 2006).
\2\ These drugs are covered under Medicare Part B, the part of
Medicare that covers a broad range of medical services, including
physician, laboratory, and hospital outpatient services and durable
medical equipment. Part B-covered drugs are typically administered by a
physician or other medical professional rather than by patients
themselves. In contrast, drugs covered under the new prescription drug
benefit, known as Part D, are generally self-administered by patients.
---------------------------------------------------------------------------
Through Medicare's ESRD benefit, patients receive a treatment known
as dialysis, which removes excess fluids and toxins from the
bloodstream. Patients also receive items and services related to their
dialysis treatments, including drugs to treat conditions resulting from
the loss of kidney function, such as anemia and low blood calcium. The
Centers for Medicare & Medicaid Services (CMS), the agency that
administers the Medicare program, divides ESRD items and services into
two groups for payment purposes. In the first group are dialysis and
associated routine services--such as nursing, supplies, equipment, and
certain laboratory tests. These items and services are paid for under a
composite rate--that is, one rate for a defined set of services. Paying
under a composite rate is a common form of Medicare payment, also known
as bundling. In the second group are primarily injectable drugs and
certain laboratory tests that were either not routine or not available
in 1983 when Medicare implemented the ESRD composite rate. These items
and services are paid for separately on a per-service basis and are
referred to as ``separately billable.''
Over time, Medicare's composite rate, which was not automatically
adjusted for inflation, covered progressively less of the costs to
provide routine dialysis services, while program payments for the
separately billable drugs generally exceeded providers' costs to obtain
these drugs. As a result, dialysis facilities relied on Medicare's
generous payments for separately billable drugs to subsidize the
composite rate payments that had remained nearly flat for two decades.
In addition, the use of the separately billable drugs by facilities
became routine, and program payments for these drugs grew
substantially. In 2005, program spending for the separately billable
drugs accounted for about $2.9 billion. Medicare's payment for these
separately billable drugs is the focus of my remarks today. My remarks
are based on the information included in our aforementioned report.
Background
Since the Medicare Prescription Drug, Improvement, and
Modernization Act of 2003 (MMA) was passed,\3\ how separately billable
drugs are paid for has changed--from payment based on each drug's
average wholesale price (AWP),\4\ to payment based on each drug's
average acquisition cost, to payment based on the manufacturer's
average sales price (ASP) for each drug. Specifically, beginning in
2006, payment for each drug is set at ASP + percent.
---------------------------------------------------------------------------
\3\ Pub. L. No. 108-173, 117 Stat. 2066.
\4\ Epogen, one of the separately billable drugs, was not paid
under the AWP method. The method Medicare used to pay for Epogen was an
amount set in statute for a single year--$10.00 per 1,000 units in
1994. CMS continued to pay this rate at its discretion until 2005.
---------------------------------------------------------------------------
In recent years, CMS has been exploring, as required by the
Congress, the creation of a bundled payment for all ESRD services,
including the drugs that facilities currently bill for separately. In
response to a mandate that CMS study the feasibility of creating a
bundled payment,\5\ the agency issued a study in 2003 concluding that
developing a bundled ESRD payment rate was feasible and that further
study of case-mix adjustment--that is, a mechanism to account for
differences in patients' use of resources--was needed. In the MMA, the
Congress required that CMS report on the design of a bundled
prospective payment system for ESRD services, including a case-mix
adjustment methodology, and conduct a 3-year demonstration to test the
design of a bundled ESRD payment system.\6\
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\5\ Medicare, Medicaid, and SCHIP Benefits Improvement and
Protection Act of 2000, Pub L. No. 106-554, app. F, Sec. 422(b),(c),
114 Stat. 2763A-463, 2763A-516-517.
\6\ Pub. L. No. 108-173, Sec. 623(e),(f), 117 Stat. 2066, 2315-
2317.
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New Payment Provisions Reduced Subsidy from Separately Billable Drugs
but Did not Eliminate Incentives to Ovceruse These Drugs
The effect of several legislative and regulatory changes since 2003
has been to raise the composite rate for dialysis services while
reducing Medicare's pre-2005 generous payments for separately billable
ESRD drugs. Under the first legislative change in 2005, Medicare
expenditures for certain of these drugs dropped 11.8 percent. Under the
current payment method, based on the ASP for each drug, Medicare's
payment rates have varied from quarter to quarter but have remained
relatively consistent with the lower 2005 payment rates.
The ASP-based rates are an improvement over the pre-MMA method, as
ASP is based on actual transactions. However, certain unknowns about
the composition of ASP and the ASP-based payment formula make it
difficult for CMS to determine whether the ASP-based payment rates are
no greater than necessary to achieve appropriate beneficiary access.
For one thing, CMS has no procedures for validating the accuracy of a
manufacturer's ASP, which is computed by the manufacturer. For another,
CMS has no empirical justification for the 6 percent add-on to ASP.
Regardless of how payment for these drugs is calculated, as long as
facilities receive a separate payment for each administration of each
drug and the payment exceeds the cost of acquiring the drug, an
incentive remains to use more of these drugs than necessary.
The ASP payment method is of particular concern with respect to
Epogen, which in 2005 accounted for $2 billion in Medicare payments and
is Medicare's highest Part B expenditure drug. Introduced in 1989,
Epogen--the brand name for epoetin alpha--was an expensive breakthrough
drug used to treat anemia in patients with ESRD. Most ESRD patients
receive injections of Epogen at nearly every dialysis treatment.
Preliminary data for 2006 suggest that Epogen use, which grew rapidly
in the years before the MMA provisions took effect, continues to grow,
although at a slower rate than previously. Epogen is the only product
available in the domestic ESRD market for anemia management. However,
the ASP method relies on market forces to achieve a favorable rate for
Medicare. When a product is available through only one manufacturer,
Medicare's ASP rate lacks the moderating influence of competition. The
lack of price competition may be financially insignificant for
noncompetitive products that are rarely used, but for Epogen, which is
pervasively and frequently used, the lack of price competition could be
having a considerable adverse effect on Medicare spending.
Bundled Payment System for ESRD Services, Including Injectable Drugs,
Would Promote Efficiency and Clinical Flexibility
Medicare's approach to paying for most services provided by health
care facilities is to pay for a group--or bundle--of services using a
prospectively set rate. For example, under prospective payment systems,
Medicare makes bundled payments for services provided by acute care
hospitals, skilled nursing facilities, home health agencies, and
inpatient rehabilitation facilities. In creating one payment bundle for
a group of associated items and services provided during an episode of
care, Medicare encourages providers to operate efficiently, as
providers retain the difference if Medicare's payment exceeds the costs
they incur to provide the services. Medicare's composite rate for
routine dialysis and related services was introduced in 1983 and was
the program's first bundled rate.
Experts contend that a bundled payment for all dialysis-related
services would have two principal advantages. First, it would encourage
facilities to provide services efficiently; in particular, under a
fixed, bundled rate for a defined episode of care,\7\ facilities would
no longer have an incentive to provide more ESRD drugs than clinically
necessary. Second, bundled payments would afford clinicians more
flexibility in decision making because incentives to prescribe a
particular drug or treatment are reduced. For example, providers might
be more willing to explore alternative methods of treatment and modes
of drug delivery if there were no financial benefit to providing more
services than necessary.
---------------------------------------------------------------------------
\7\ In the case of the composite rate, one dialysis session
constitutes an episode of care. Unlike the current composite rate
payment method, a newly designed payment bundle could define the
episode of care more broadly. For example, the new payment bundle could
cover dialysis and related items and services for 1 month.
---------------------------------------------------------------------------
In the MMA, the Congress required CMS to issue a report and conduct
a demonstration of a system that would bundle payment for ESRD
services, including drugs that are currently billed separately, under a
single rate. Any payment changes based on CMS's report or demonstration
would require legislation.\8\ Both the report, due in October 2005, and
demonstration, mandated to start in 2006, are delayed, and CMS
officials could not tell us when the report or results from the
demonstration would be available.
---------------------------------------------------------------------------
\8\ The MMA specified that drugs billed separately at the time the
legislation was enacted continue to be billed separately and not
bundled in the composite rate. MMA sec. 623 (d)(1), 1881 (b)(13)(B),
117 Stat. 2314-15 (to be codified at 42 U.S.C. 1395rr(b)(13)(B)).
---------------------------------------------------------------------------
In light of these circumstances, we have asked the Congress to
consider establishing a bundled payment for all ESRD services as soon
as possible. In our view, Medicare could realize greater system
efficiency if all drugs and services were bundled under a single
payment. A bundled payment would encourage facilities to use drugs more
prudently, as they would have no financial incentive to use more than
necessary and could retain the difference between Medicare's payment
and their costs. To account for facilities' increased or decreased
costs over time, a periodic reexamination of the bundled rate may be
necessary. This would ensure that facilities would be paid
appropriately and that Medicare could realize the benefit of any cost
reductions.
Mr. Chairman, this concludes my prepared statement. I will be happy
to answer any questions you or the other Committee Members may have.
Chairman THOMAS. Thank you.
Prior to going to Ms. Norwalk, because I know you have an
important meeting to go to, if it is okay, and we will
determine the length of the questioning perhaps like to just
draw you out a bit in terms of your statement prior to moving
to the CMS testimony.
As you know, I am very loathe to have Congress attempt to
legislate the ways in which services are offered, but I want to
understand in your underscore in your GAO in page 6 that in
2003 the Medicare Modernization Act required CMS to design a
system that would no longer pay for each injectable ESRD drug
in a separate rate. You then go on in the same paragraph to
conclude CMS report is designing a report for bundled ESRD
payment was due in September 2005. However, as of November of
2006, CMS officials could not tell us when the report would be
issued. The demonstration testing the feasibility of a bundled
rate mandated to start in July 2006 is also delayed.
If in fact this is what CMS has been doing, what is it that
we are going to do as a Congress to put together a package
which would answer those issues? You said perhaps it isn't as
complicated as other others are talking about. What we heard
from the previous panel is, there are drugs, and my
understanding is they are identical, notwithstanding the fact
they are used in separate purposes. The separate purposes are
far closer together, if we can believe Dr. Singh's testimony,
than we would have thought and that could possibly create a
competitive model rather than a monopoly. Notwithstanding--
since 2003--CMS can't put together a bundling package and
present it to us based upon our requirements in MMA, what is it
that you would be suggesting that we might do? That is what I
am looking for.
Mr. WALKER. What I am suggesting is, there appears to be
agreement that it makes sense to move to a bundled approval for
payment for these drugs. And----
Chairman THOMAS. I believe the Congress certainly believes
that.
Mr. WALKER. I believe that CMS believes that as well, and I
think it is important that it is the Congress's will to say
that while CMS needs to do demonstration work and while I think
additional consideration needs to be given to drugs that are
essentially identical or similar to Epogen that are not used in
End Stage Renal Disease but are used for other purposes, I
think it is important that the Congress's will be noted to say
we expect you to move to bundled services by X date and you
have to decide what X date is.
Chairman THOMAS. But we did, in essence, in 2003, while
leaving it to the professional competence of CMS to move
forward with that.
Mr. WALKER. My understanding is that you required something
be done and a report be issued by a date certain rather than
necessarily move to the bundled services by a date certain.
Chairman THOMAS. Okay. I think, possibly given the
commonality of our position here, a more insistent direction
with the specific date could produce something, but I will go
back to the recent attempt to get letters answered and the
dateline in terms of answering simple letters. So, to a certain
extent, I appreciate the requirement to put a firm date, but I
will tell you, I have no assurance that any firm date will be
met.[11:45 a.m.]
Chairman THOMAS. If they cannot answer letters, I doubt if
they are going to give us a program. That is my concern.
Therefore, I also want to focus on how we can create a degree
of competition and how we can create an opportunity to allow
dialysis facilities to utilize various methods of infusion,
injection, subcutaneous, others that are not absolutely
dictated to by a policy that controls price and circumstances;
and that perhaps is an area we might be able to move with, as
well as bundling.
Mr. WALKER. Well, as you know, there is another drug
company that is proposing to bring a drug to market that
potentially would be used for End-Stage Renal Disease. There is
litigation that is currently pending with regard to that. That
was obviously beyond the scope of our study, but it is a fact,
and so that is one possibility for competition.
Another thing that you touched on, Mr. Chairman, which I
think is appropriate, is there are at least two drugs that are
used, one of which is identical to Epogen and one of which is
similar but not identical to Epogen. They are not used for End-
Stage Renal Disease, but they are used for chronic kidney
disease, and one would think that you could look at some of the
pricing arrangements and other types of activities going on
there. You are not requiring them to be used for End-Stage
Renal Disease, but there is information there that I think
would be relevant in determining, in effect, what should be
paid for these drugs.
Chairman THOMAS. Thank you.
The gentleman from New York.
Mr. RANGEL. I have no questions. I just would like to thank
you, Mr. Walker, for the dedicated service that you have
provided over the years for the Congress and the country.
Chairman THOMAS. The gentlewoman from Connecticut, the
Chairman of the House Subcommittee.
Ms. JOHNSON OF CONNECTICUT. Mr. Walker, did you look at the
policy that CMS adopted in April?
Mr. WALKER. I apologize, Mrs. Johnson. My staff tells me
that we looked primarily at the payment methodology. We looked
at the policy, but we did not look at anything in depth other
than the payment methodology. That is the really the only
thing.
Ms. JOHNSON OF CONNECTICUT. Did you also look at why the
demonstration has been slow to come together and the issue of
risk adjusting in the demonstration? Dr. Singh did indicate
that he thought risk adjustment was important. My understanding
is that risk adjustment in a demo setting is an easy--I could
be misinformed about that. I have not had time to do an in-
depth development of my knowledge based on this issue. But I
need to know why the demo has been hard to come together. While
I will ask CMS that later on, since you are here and you gave
this report I want to know, did you look at that?
Mr. WALKER. It is clear that the delay has in large part
been due to considerations of looking at risk adjustment. We
are not saying that that should not be done. We are not saying
that you should discontinue the demonstration project either.
We think it is important that that be done.
The bottom line point is this: It seems clear that Congress
intended our work supports, and it is my understanding that CMS
agrees, that we need to move to a bundled payment system as
quickly as possible. I think the administrator is in a better
position to answer why it has been delayed and when she thinks
it is going to be completed. But for the interests of the
patients and the interests of the taxpayers, I think we need to
move to a bundled payment system as soon as possible.
Ms. JOHNSON OF CONNECTICUT. Thank you.
My concern is that the express purpose of the demonstration
project was to demonstrate a bundled payment solution; and if
risk adjustment is difficult, that will affect patients. So, we
want a system that not only pays appropriately but makes the
drugs available for treatment appropriately. If risk adjustment
is the problem, why would we want to implement a bundled
payment without having the ability to risk adjust it?
So, I do not differ on the goal at all. I am just a little
mystified. I want to learn more about how we are going to
achieve that goal, and I want to be sure that the knowledge
base is firm before we make a national change in our National
payment system. That is my only concern.
Chairman THOMAS. Will the gentlewoman yield in terms of
that concern?
Ms. JOHNSON OF CONNECTICUT. Certainly.
Chairman THOMAS. Notwithstanding the obvious difficulty,
given the time lag, does it make any sense at all in focusing
on this that we could at least begin to nail down a couple of
specifics that does not involve the universal concerns that are
being discussed?
Number one, at what point do we begin to focus on the
question of the percentage dosage? When you have got an FDA
rate, you have got a label recommendation, you have got a
manufacturer putting out a warning label, you have got studies
that produce it, and you have CMS continuing to increase the
percentage, at least that could be reconciled.
Secondly, if you have other drugs that could possibly
provide or a structure in which when you do not have--and a lot
of times what we have done is, when you do have a monopoly, you
can create Government as a surrogate competitor and create a
price which would control the amount, rather than leaving it to
open market and incentives which are designed to require people
to continue to use larger amounts.
Within the current structure, you have at least several, I
would think, abilities to adjust payment arrangement
competition and come to some agreement at least on the
conservative do-less-harm side about the dosage question.
Could not we at least do those while they are trying to
complement risk factors and other arrangements?
Mr. WALKER. We are saying similar things, Mr. Chairman,
just in different ways.
The other thing I would suggest is, it seems to me that
there is also a possibility to continue the demonstration
project, move to some type of a bundled payment system with the
consideration that there might be some adjustment in payment at
some future date depending upon the results. But I would leave
that to the CMS administrator.
Ms. JOHNSON OF CONNECTICUT. Reclaiming my time. Let me also
put one other thing on your agenda, because I was not aware of
that until this hearing. If you can titrate up to a stable
dosage or if you can deliver this treatment more cheaply
through injection, why are we not looking at those things, too?
Because our bundled payment ought to take into account the
lower cost of a different delivery system. Did you look at
those issues?
Mr. WALKER. We did. One of the things that is included in
our report is our point about the need for clinical flexibility
to consider alternative means to be able to achieve the desired
result, which includes what you said, Mrs. Johnson.
Ms. JOHNSON OF CONNECTICUT. We certainly do want that
clinical flexibility if the patient is going to be served. That
goes to the bundling issues and the risk.
Chairman THOMAS. Yes, obviously, if in fact another portion
of the Government is using the subcutaneous method, you would
at least think that that could be an option that you would look
at, given the cost deferential.
The gentleman from California.
Mr. STARK. General Walker, thank you for this report.
You indicate that CMS has no procedure for validating the
accuracy of the manufacturer's average sale price, which the
manufacturer computes. I mean, they say, here is our average
sales price, and we have no way to verify that, as I gather.
So, if I said what you are suggesting is that Amgen tells
CMS what the average sales price is for Epo and then they go
out and sell it at whatever price they want to sell it, perhaps
there are deep discounts to volume purchasers.
Do we know? Is that transparent to you? Do you know, are
there big discounts? Do we have any idea how this pricing
system works?
Mr. WALKER. Mr. Stark, we know what the statute says. The
administrator may want to address what, if any, concerns they
have about what they can do, given the statutory language. But
what we can tell you is this, is we do not think there is
adequate transparency and we don't think there is adequate work
being done to verify the average sales price.
I reflect back--I am a Ronald Reagan, George Herbert Walker
Bush and Bill Clinton Presidential appointee. We can all
remember President Reagan saying: Trust but verify. That
applies to drug prices, too.
Mr. STARK. Thank you very much.
Chairman THOMAS. We have been discussing bundling in terms
of the Government's ability to create a structure which would
limit controlling. It is my understanding that the private
sector has had an ability to create bundling in terms of the
purchase of drugs that are used. Is that--did you examine that
at all, the way in which the provider, the manufacturer of the
drugs is bundling the use of particular drugs as an incentive?
Mr. WALKER. We did not look at that in the context of this
report.
Chairman THOMAS. Are you aware of it?
Mr. WALKER. Somewhat, yes.
Chairman THOMAS. We will ask that question.
Mr. WALKER. There is unbundling that occurs, too, in order
to affect pricing.
Chairman THOMAS. Absolutely. So, bundling is on both sides.
Any additional questions? Thank you very much, and I
apologize for causing you some problem in trying to get across
the bridge.
Mr. WALKER. Thank you, Mr. Chairman.
Ms. TUBBS JONES. Mr. Chairman, I want to introduce
something for the record. Mr. Chairman, thank you very much for
the opportunity.
Mr. Walker, I want to introduce for the record two articles
by a constituent of mine by the name of Dr. Wish. Dr. Wish is a
physician at University Hospital in Cleveland; and, in that
capacity, he is a Professor of Medicine and the Medical
Director of Hemodialysis Services at University Hospital.
Among other things, Dr. Wish is the President of the End-
Stage Renal Disease Network, and he is on the CMS Advisory
Board, and he asked me to specifically introduce into the
record two of his articles: One of them, The Economic Realities
of Erythropoiesis-Stimulating Agent Therapy in Kidney Disease,
and another one, an editorial, Can Evidence Drive the
Development of a Sound National Epo Reimbursement Policy for
the United States?
I am assuming you know Dr. Wish and his prominence in this
area. I thought as long as we were discussing these issues it
would be important that we have some information from his
background into the record.
Mr. Chairman, I thank you very much for the opportunity.
Chairman THOMAS. Thank you very much.
Thank you, Mr. Walker.
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Chairman THOMAS. Let me say at the outset, any written
testimony you have will be made part of the record. Ms.
Norwalk, notwithstanding the fact that you are sitting here now
as someone who is newly arrived in the position, filling a slot
from someone who had been the head of the FDA and was a medical
doctor, we are completely aware of the fact that these policies
and decisions were ongoing and that for us to ask you with the
expectation or belief that you might be able to respond in
depth, not saying that as you spend some time in that position
you would not be able to but that at the onset and given the
timing that we have, I want to encourage you, if we move into a
questioning session, to feel perfectly comfortable asking any
person who is part of the CMS support structure to identify
themselves and come to the table so that you do not have to
turn around and ask them the question and have them provide you
the answer. Because that is a perfectly legitimate and proper
way to operate, given how long you have been on the ground even
in an acting capacity.
Ms. NORWALK. Thank you, Mr. Chairman.
Chairman THOMAS. It is not personal, and it is not directed
toward you. You just happen to have currently moved very
briefly into a position in which we expect you to know
everything about everything, and that is not fair.
So, with that, the time is yours.
STATEMENT OF LESLIE V. NORWALK, ESQ., ACTING ADMINISTRATOR,
CENTERS FOR MEDICARE AND MEDICAID SERVICES
Ms. NORWALK. Thank you, Chairman Thomas, distinguished
Members of the Committee. Thank you for the opportunity to
appear before you today.
Mr. Chairman, I also would like to wish you a happy
birthday. As the Acting Administrator of CMS, this has special
meaning in my heart, given that you are now an official
Medicare beneficiary, entitled to all of the benefits that you
helped create and improve during your service in Congress.
You asked me to be here today to discuss a very important
issue, safety and quality in the treatment of patients with
End-Stage Renal Disease, or ESRD. Roughly 400,000 Americans
suffer from ESRD and are entitled to Medicare coverage on the
basis of that diagnosis, regardless of age or disability.
The ESRD population has grown steadily in recent years.
Although better management of diabetes and hypertension could
help stem the growth, initiatives to promote efficient, high-
quality ESRD care are integral to CMS's overall agenda and
value-based payment reforms.
This administration has demonstrated a strong commitment to
promoting quality across the board. CMS launched the Medicare
Quality Initiative in 2001, promoting greater accountability in
consumer choice through unprecedented public disclosure of
provider performance on a range of quality measures.
To date, CMS has implemented initiatives focused on nursing
homes, home health agencies, hospitals and dialysis facilities.
We announced the ESRD Quality Initiative in 2004 to stimulate
and support improving quality of dialysis care. For these and
other efforts CMS was recognized earlier this year by the
American Association of Kidney Patients for dedication to
improving lives of kidney patients and strong leadership in
health care for all Americans.
Projects such as Dialysis Facility Compare on the Web site
and the Fistula First Breakthrough Initiative are just two
examples of the many steps CMS has taken to promote high-
quality care for people with ESRD.
The Fistula First Breakthrough Initiative aims to increase
the use of fistulas in hemodialysis treatment of patients with
ESRD. Fistulas are considered to be the gold standard for
establishing access to a patient's circulatory system as is
required during hemodialysis.
Fistulas last longer, require less rework and repair and
are often associated with lower rates of infection,
hospitalization and death. Simply put, appropriate use of
fistulas optimizes patient care, including a possible reduction
in Epo dosing.
CMS also contracts with the End-Stage Renal Disease
Networks across the country to help monitor and improve the
quality of care for ESRD patients. The ESRD Networks are
similar to the QIOs, which work with hospitals, physicians and
other Medicare providers to promote quality and best practices.
Our networks focus on quality treatment, quality
improvement and promoting transparency in renal dialysis
treatment. Their efforts are complemented by our survey and
certification process for ESRD facilities, which enforces
compliance with regulations prescribing minimum standards for
Medicare-approved dialysis facilities with respect to quality,
patient safety and access to Medicare benefits.
I know that many of you share my strong interest in a more
rational payment system for ESRD care, requiring a CMS report
to Congress and demonstration under the Medicare Modernization
Act of 2003 to advance a fully bundled ESRD prospective payment
system. As you know, the GAO recently reported that such a
payment structure would promote quality, safety and savings in
the ESRD benefit; and I want to be clear that I completely
agree.
While the MMA-required report is now overdue, I want to
assure you that CMS is fully committed to completing the
analytical groundwork relevant to a fully bundled payment
system. CMS completed an initial round of research in mid-2006,
but developing a payment model that adequately captured
variation of dosage of Epo was a key, but difficult, area of
this research. CMS felt that the approach examined in the
initial round too closely linked the payment to actual drug
utilization and so began a new phase of research to address
this and other areas of our new payment structure.
Our current research, which is nearing completion, focuses
on predictors used in the current basic system augmented by
other adjusters such as comorbid conditions and other patient
characteristics. I expect to receive the research findings from
our contractor early next year and hope to detail those
findings in our official report to you by next summer.
With the benefit of those findings and recent work of the
GAO, MEDPAC and others, I look forward to working with you to
develop an effective bundling proposal. I believe that payment
reform is critical to improving ESRD patient care.
Finally, I have heard recently from you, Mr. Chairman, and
from you, Mr. Stark, regarding a monitoring policy that we have
in place to encourage appropriate use of anti-anemia agents in
the ESRD population. Anemia can be severe and debilitating in
ESRD patients if left untreated, but, fortunately, drug
compounds with Epo in this context can largely alleviate the
symptoms.
Anemia's severity is monitored by a patient's hematocrit,
the proportion of red blood cells in whole blood. Scientific
evidence and, indeed, FDA labeling for Epo indicate that
patient hematocrit should be maintained between 33 and 36
percent for optimal results.
In a recent letter, you noted that CMS's hematocrit
monitoring policy does not reduce provider payment for Epo
until a patient's hematocrit reaches 39 percent. While this is
accurate, we believe it is important to keep in mind the
distinction between regulating the safety and effectiveness of
a particular drug versus determining the amount a provider
should be paid for administering that drug to a patient.
Our provider and contractor manuals specifically require
providers to target a hematocrit range of 30 to 36 percent
consistent with the FDA label. Moreover, we require that
patient records reflect the clinical reason for dose changes
and hematocrit levels outside of the 30 to 36 percent range.
Medicare contractors currently may review medical records
to ensure appropriate dose reductions are applied and
maintained and hematological target ranges are maintained.
Hematocrit levels can change unexpectedly for a multitude of
reasons. Our instructions to carriers about reviewing claims
takes this into account.
Our monitoring policy for carriers is not establishing a
therapeutic hematocrit target, which we believe is a clinical
judgment appropriately left to a treating physician. Our
monitoring threshold for carriers is slightly above the FDA
label to avoid penalizing providers that make appropriate dose
reductions in response to unexpected increases in their
patients' hematocrit levels. Rather, 39 percent is a marker of
the point at which a Medicare carrier must reduce payment to a
provider because the reported hematocrit was not maintained at
a level consistent with the FDA label. CMS developed this
policy after considering the body of available scientific
evidence as well as public comments received in response to our
proposed policy issued in 2004.
We are, of course, very interested in recent research
findings regarding Epo use in patients with chronic kidney
disease. Following publication, a study of this nature
typically is subject to international scrutiny and examination.
Experts will review the study design, methodology results and
conclusions. In fact, it would be very useful for CMS to have
access to the raw data behind the CHOIR and other similar
studies so we may take them into account as we continue to
review our policies.
We look forward to further research developments on this
issue. Like Congress, we are concerned about overuse and
improper use of any drug we cover. We are always reassessing
our policies to see if we can strengthen our programs to ensure
the best possible patient outcomes.
I would be happy to answer any questions you may have.
[The prepared statement of Ms. Norwalk follows:]
Statement of Leslie V. Norwalk, Acting Administrator, Centers for
Medicare and Medicaid Services
Chairman Thomas, Representative Rangel, thank you for the
opportunity to appear before you today regarding important safety and
quality issues in the treatment of Medicare patients with kidney
failure, or End Stage Renal Disease (ESRD). Roughly 400,000 Americans
suffer from ESRD and require either kidney dialysis or transplantation
to live. ESRD is Medicare's only disease-specific program; it entitles
people of all ages to Medicare coverage on the basis of their ESRD
diagnosis. The number of individuals covered under Medicare by virtue
of their ESRD diagnosis continues to grow steadily. Estimates suggest
that as many as 20 million Americans currently are afflicted by some
stage of Chronic Kidney Disease (CKD). Many will progress to ESRD and
the need for some form of renal replacement therapy unless new ways of
treating CKD are found.
The Centers for Medicare and Medicaid Services (CMS) believes that
in general, treatment decisions for ESRD patients are best left to the
clinical judgment of treating physicians. The CMS is charged with
determining appropriate coverage and payment for services to Medicare
beneficiaries. In recent years, CMS has worked hard to ensure its
coverage and payment policies promote high quality care, which is in
the best interests of the beneficiaries we serve as well as the long-
term financial health of the Medicare program.
Quality and safety initiatives have been at the center of the
Administration's health care agenda for more than five years. We have
made significant strides in promoting greater transparency in the
health care industry, giving Medicare beneficiaries and all consumers
unprecedented access to information that supports meaningful choices.
Whether considering dialysis facilities, hospital services, skilled
nursing providers or prescription drug benefits, people with Medicare
can find the information they need to identify the best quality and
value among available options. The CMS has devoted significant
resources to ESRD quality and patient safety issues, with a
comprehensive Quality Roadmap, the ESRD Quality Initiative, and ongoing
research to explore ESRD payment reforms, among other efforts.
The Congress also has been an important partner in these
achievements. Efforts such as the Care Management for High Cost
Beneficiaries Demonstration create a platform for research to improve
quality care and reduce the costs of caring for fee-for-service
beneficiaries with one or more chronic diseases, who generally incur
high Medicare costs. CMS has selected six sites under the
demonstration, including one in New York state that focuses on
beneficiaries with chronic kidney disease. Programs under the
demonstration are testing ways to increase adherence to evidence-based
care, reduce unnecessary hospital stays and emergency room visits, and
help participants avoid costly and debilitating complications.
The CMS Quality Roadmap & Medicare's ESRD Quality Agenda
In 2005, CMS issued the ``CMS Quality Roadmap,'' to promote the
right care for every person, every time. The Quality Roadmap builds on
the Institute of Medicine's six aims for healthcare: Patient-centered;
Safe; Accessible; Effective; Efficient; and Equitable.
The CMS Quality Roadmap presents five strategies to achieve its
vision:
Partnering and collaborating with other healthcare
stakeholders;
Collecting and publicly reporting data that measures the
quality, efficiency and cost of healthcare;
Striving to reform healthcare reimbursement systems to
promote quality and efficiency, while avoiding unnecessary costs and
complications;
Promoting the use and availability of clinical
information for providers and Medicare beneficiaries, particularly
through the adoption of health information technology, to assist them
in providing and receiving high-quality and efficient care; and,
Promoting the use of evidence-based healthcare
information, in clinical, coverage, and payment systems, ensuring that
the latest treatments, medical devices and services are available to
clinicians and their patients, while avoiding inappropriate or wasteful
use of those treatments.
Significant work and leadership in clinical quality initiatives
also preceded the adoption of the CMS Quality Roadmap. In 2001, the
Administration launched the Medicare Quality Initiative in pursuit of
quality health care through accountability and public disclosure not
just for Medicare patients, but for all Americans. Following the
implementation of specific initiatives focused on nursing homes, home
health, and hospitals, CMS announced the ESRD Quality Initiative in
2004.
Specific objectives of the ESRD Quality Initiative, which focuses
on dialysis facilities, reflect an array of goals to stimulate and
support improvement in the quality of dialysis care:
Refining and standardizing dialysis care measures, ESRD
data definitions, and data transmission to support the needs of
Medicare's ESRD program;
Empowering patients and consumers by providing access to
facility service and quality information;
Providing quality improvement support to dialysis
providers;
Assuring compliance with conditions of coverage; and,
Building strategic partnerships with patients, providers,
professionals, and other stakeholders.
While all efforts under the ESRD Quality Initiative are
significant, the Fistula First Breakthrough Initiative is particularly
noteworthy. Under the initiative, facilities submit data to Medicare
contractors charged with quality review of dialysis facilities (``ESRD
Network Organizations'') to facilitate a more coordinated approach to
care. The initiative has led to a significant increase in the use of AV
Fistulas in treating dialysis patients--a measure associated with
considerable reductions in avoidable hospitalization and death for ESRD
beneficiaries.
The ESRD Quality Initiative also supports the annual collection of
Clinical Performance Measures (CPMs) for a random sample of dialysis
patients nationwide. With these measures, CMS can identify and track
opportunities for improvement in areas such as the adequacy of
hemodialysis and peritoneal dialysis, anemia management, and vascular
access management. The Quality Initiative also includes the Dialysis
Facility Compare resource on www.medicare.gov, which contains quality
information for all Medicare approved dialysis facilities in the United
States. Patients and consumers are able to search and compare
facilities on this site and choose a dialysis facility that best meets
their needs.
In addition to various efforts under the ESRD Quality Initiative,
CMS partners with states to conduct regulation and enforcement
activities to ensure that dialysis facilities comply with federal
safety and quality standards. Under this survey and certification
program, CMS establishes standards for safe and effective operation of
dialysis facilities; develops guidelines and procedures; provides
training for surveyors; and coordinates state activities. Currently,
dialysis facilities are surveyed roughly every 36 months. State survey
agencies also will investigate specific complaints on an as needed
basis, outside of the regular survey cycle.
Finally, nearly all patients with ESRD suffer from debilitating
anemia. Much of this anemia can be managed through drug therapy. CMS
has had a quality initiative for years to encourage appropriate
management of anemia in ESRD patients, including an active monitoring
policy for patients being treated with erythropoietin.
All of these examples demonstrate a commitment by the
Administration and CMS to ensuring and improving high quality care for
the ESRD population. We have made significant strides over the last 5
years, and will continue to work to increase the availability of
consistent, standardized core data elements that promote greater
transparency and better care outcomes for ESRD patients.
Anti-Anemia Agents Used in ESRD Patients
Two prescription drugs commonly are used for anemia management in
patients with ESRD who are dialyzed in renal facilities: epoetin alfa
(Epogen') and darbepoetin alfa (Aranesp'). These
products rely on erythropoietin to help control anemia. To promote
appropriate usage, CMS has in place a monitoring policy that considers
both hematocrit levels and erythropoietin dosage levels.\1\
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\1\ Anemia severity is monitored by measuring the hematocrit with a
simple blood test that reveals the proportion of red blood cells in
whole blood. The hematocrit result is expressed as a percentage.
Alternatively, the hemoglobin concentration in whole blood may be used
to monitor anemia. The numeric value of the hematocrit is generally
three times the value of the hemoglobin measured simultaneously, though
they are expressed using different units. Thus, for example, a
hematocrit of 30 percent corresponds to a hemoglobin concentration of
10 g/dl.
---------------------------------------------------------------------------
Current kidney disease clinical guidelines, determined through
national consensus processes by multiple ESRD experts and stakeholders,
call for maintaining the hematocrit level of patients receiving
erythropoietin within a narrow target range of 33-36 percent. Because
many factors such as nutritional status, infection, and bleeding may
cause the hematocrit to fluctuate, it is not easy to manage patients to
this narrow range. Some patients might be above (or below) the target
in one month, for example, but below (or above) it in others. If one
superimposes frequent and significant changes in doses of anemia
management drugs on these existing fluctuations, patient hematocrit
fluctuations can become even more variable and difficult to interpret
and manage, particularly within the narrow target range of 33-36
percent.
Promoting Appropriate Payment through Hematocrit Monitoring
ESRD treatment facilities submit claims to CMS monthly for
erythropoietin, which is billed separately from other dialysis
services. The claim form includes fields where the facility must report
the beneficiary's hematocrit test result. Commonly, a dialysis
patient's hematocrit level is tested many times during a month.
CMS is committed to establishing and maintaining policies in all
areas of the Medicare program that protect beneficiaries, promote
efficient and appropriate use of medical interventions, and enable
providers to render excellent care. The newly revised CMS monitoring
policy for erythropoietin used in ESRD patients instructs providers on
how to submit claims, and instructs CMS contractors on how to
adjudicate the claim. Under the policy, Medicare expects a 25 percent
reduction in the dosage of erythropoietin for patients whose hematocrit
exceeds 39.0. If the dosage is not reduced, payment is made for the
drugs as if the reduction occurred.
The new monitoring policy is not a national coverage determination,
and thus it is not a determination of the reasonableness and necessity
of using an anti-anemia agent to maintain hematocrit levels above 36
percent. The monitoring policy clearly articulates that providers
should adhere to the FDA label instructions for erythropoietin, i.e,
seeking to achieve a hematocrit of 30-36 percent. The instruction to
carriers to initiate monitoring when the hematocrit reaches 39 percent
is not a new hematocrit range policy, but instead establishes a marker
of the point at which payment must be reduced because the reported
hematocrit was not maintained at levels consistent with FDA labeling.
The value 39 percent is not a therapeutic hematocrit target, which
CMS believes is appropriately left to a treating physicians' clinical
judgment. Rather, it is the target to initiate reduction in payment, a
function appropriate to the mission of CMS. To be clear, it recognizes
the difficulty in the clinical setting of maintaining the hematocrit in
the narrow clinical guideline range of 33-36 percent, and therefore
does not immediately cut off payment for a single hematocrit value that
fluctuates above this narrow range. However, it does set in motion a
policy that will reduce reimbursement if the hematocrit level remains
above 39 and the provider does not reduce erythropoietin dosage as FDA
labeling and national clinical guidelines indicate.\2\
---------------------------------------------------------------------------
\2\ The FDA labeling for Epogen' and Aranesp'
notes that as the hematocrit approaches a reading of 36, the dose of
the drug should be reduced by 25 percent.
---------------------------------------------------------------------------
A provider submitting a claim for erythropoietin in an ESRD patient
with a hematocrit above 39 may inform CMS that a dose reduction has
occurred, despite the continued high hematocrit, using a modifier on
the claim form. If the provider has not reduced the dose or informed
CMS that a dose reduction has occurred, however, Medicare's payment
systems will apply a 25 percent reduction in payment. The provider is
given appropriate notice of the payment reduction and may appeal the
determination.
Promoting Ptient Savety through Hematocrit Monitoring
Consistent with the approach taken to advance all of its quality
and transparency initiatives, CMS worked closely with the ESRD
community and other stakeholders in developing the revised hematocrit
monitoring policy. CMS announced its intent to develop the new policy
in fall 2003, along with a solicitation of scientific literature from
the industry. In the interest of promoting quality and efficiency in
the care of ESRD patients, CMS was determined to develop a permanent,
evidence-based policy for erythropoietin payment and hematocrit
monitoring.
Scientific literature submitted to CMS demonstrated that patients
with hematocrit levels within the target range had better health
outcomes than those with hematocrits below the target level. The data
also demonstrated that there is considerable natural variability in
individual patient hematocrit levels, making it difficult to
consistently maintain a hematocrit within the narrow range of 33-36
percent.
After analyzing the literature CMS developed a proposed policy,
published in July 2004. The CMS reviewed available scientific evidence
along with a large volume of public comments from the stakeholder
community in developing the final policy. The final policy issued in
November 2005 reflected a careful balance to ensure proper patient care
while allowing appropriate payment for services rendered by treating
physicians. In attempting to implement this policy, CMS became aware
that there were process issues in collecting the claims-based
information necessary to adjudicate these claims and, after working
with stakeholders and CMS contractors, a revised erythropoietin
monitoring policy was issued in April, 2006.
Appropriate interpretation of the evidence for erythropoietin
treatment of anemia in the ESRD population is disputed within the
stakeholder community. Several reasons for this dispute are not readily
amenable to correction by CMS. In addition, many clinical trials have
methodological restrictions that limit the degree to which their
findings can be generalized among the Medicare population. Other
published reports of clinical trials do not necessarily present all of
the available data due to limitations of space and other factors. It is
possible that some of the outstanding questions could be addressed in
part by analyzing collected but unpublished data.
CMS believes that, in general, medical decisions are best made by
the treating physician. The human physiologic response to
erythropoietin is not immediate, and the effect of a given dosage on
the hematocrit or hemoglobin of a given individual can vary widely.
This variation also is reflected in the wide and unpredictable
variation in the dosage needed to achieve and maintain hematocrit
within the target range, although other factors also contribute to
variation in dosage. Current accepted medical practice may also include
the use of drugs for indications that are not covered by an FDA label,
but that are supported by clinical evidence in peer reviewed medical
literature. Medicare may provide coverage for off-label uses of drugs
and biologics when those items are considered reasonable and necessary.
Mainstream press has recently focused on two trials published in
the New England Journal of Medicine (NEJM) regarding erythropoietin use
in chronic kidney disease (CKD) patients. However, the study
populations for these trials do not necessarily reflect the Medicare
ESRD patient population. Both studies addressthe optimal target level
for hemoglobin in CKD patients who do not yet need dialysis. It is
possible, if not probable, that many of the study subjects were not
Medicare beneficiaries because they were too young to qualify for
Medicare and were not disabled. Only patients with ESRD, who require
dialysis or transplant, are eligible for Medicare regardless of age or
disability; other patients with CKD are not Medicare beneficiaries
(unless their age or a disability qualifies them). This distinction is
important.
Anemia management for patients with ESRD cannot be assumed to be
the same for patients, often younger, with CKD (who do not yet require
dialysis). The NEJM study authors did not generalize their findings to
the ESRD population. Patients receiving dialysis are exposed to
clinical situations that patients with CKD not requiring dialysis are
not exposed to, including: artificial kidney membrane exposure; large
fluid shifts during dialysis; anti-coagulation received while on
dialysis; different medications or other treatments. Finally, the NEJM
studies looked at patients who were intentionally maintained at high
hematocrit levels (the clinical study, research goal), as opposed to
the typical ESRD patient who may fluctuate periodically above a
hematocrit of 39 percent, but is not maintained at that level (the
clinical practice situation).
In spite of the NEJM studies' focus on patients with CKD, not ESRD,
CMS considers the findings significant. Any scientific study published
in a peer-reviewed journal such as the NEJM will be subject to
international scrutiny and examination. Experts review the study
design, methodology, results and conclusions. CMS will be participating
in that scrutiny, which may include the need to design and implement
further randomized clinical trials.
CMS is committed to establishing and maintaining policies in all
areas of the Medicare program that protect beneficiaries, promote
efficient and appropriate use of medical interventions, and enable
providers to render excellent care. In the case of ESRD, and
specifically the monitoring policy for anti-anemia therapies, CMS is
exploring a number of approaches to collecting additional data. The
current policy was developed after carefully analyzing and weighing a
significant body of data and clinical evidence from a variety of
sources; additionally, the policy was reviewed and reassessed 6 months
after its initial publication. CMS is just now beginning to obtain
sufficient claims data to attempt to assess whether the monitoring
policy is achieving its stated goals: encouraging providers to try to
maintain hematocrits in the range consistent with FDA labeling and
national clinical guidelines, while not paying for unjustified dosages
that maintain patients outside that range. Additional data sources will
allow CMS to continue this pattern of vigilant, ongoing assessment of
the monitoring policy. Further data also could support the possibility
of an alternative CMS policy for anemia management and treatment.
The current monitoring policy relies on data submitted on the
claims form. This effort could be expanded; in fact, CMS already is
pursuing a number of enhancements. Currently, claims data do not
provide either the route of administration for erythropoietin or the
size of individual doses. CMS is implementing changes that will
introduce a 100 unit code to capture dosing information with greater
precision and, in conjunction with line item billing, will permit
tracking of individually prescribed doses versus an aggregate monthly
total for facilities.
In addition, CMS is implementing requirements to include the route
of administration on claims for erythropoietin administered to ESRD
beneficiaries (not chronic kidney disease patients). Existing CMS
survey data suggest that subcutaneous administration is employed in
only 7 percent of hemodialysis patients, differs by geographic location
(more likely in the Midwest and West), and differs by dialysis facility
ownership. Inasmuch as studies have suggested that subcutaneous may be
a preferred route of administration, potentially requiring lower levels
of erythropoietin to achieve the desired therapeutic effect, data of
this nature is critical to continuous evaluation of the hematocrit
monitoring policy.
Using the information currently collected, CMS also is able to
quantify monthly utilization of erythropoietin, though the accuracy of
these data is limited to what providers report on the claims--typically
including quantities of the drug that have been opened but not
necessarily provided to any patient (referred to as wastage). These and
other limitations result in current claims data providing only a
limited picture of erythropoietin utilization and anemia management.
Additional data would be helpful.
One possible approach is to collect data--such as the dosage of
erythropoietin actually administered or additional hemoglobin /
hematocrit measurements--through clinical trials. Such an approach is a
challenge to implement, however. The CMS' authority to condition
Medicare coverage on participation in clinical trials and collection of
data is could be constrained by the Health Insurance Portability and
Accountability Act, the Privacy Act, and other concerns.
Another approach might be to create registries of data submitted by
hospitals and other facilities. Such registries could be a robust data
collection mechanism, pursuing elements beyond what can be collected on
the claim form. Before such an approach could be adopted, however, CMS
must assess potential restrictions to requiring hospitals and
facilities to report information to a registry. Provider burden also
would be an important consideration.
CMS could consider requiring additional Clinical Performance
Measures through the existing Quality Initiative. The CPM project
collects clinical information on dialysis patients in order to measure
and track quality of care received by patients in dialysis facilities.
However, CPMs currently are collected on just a 5-percent sample of
dialysis patients nationwide. It will take a number of years before
CPMs can be collected more broadly--ideally for all dialysis patients--
due to limitations in facilities' and with CMS' own data collection
systems.
Bundled Payment
In addition to significant quality efforts, CMS is committed to
efficient and appropriate payment for all Medicare providers. In the
context of ESRD care, many have urged a shift from the current model of
paying independently for dialysis treatments and separately billable
drugs, to a system of bundled payment. CMS is generally supportive of
such reforms, and has devoted resources to research and development of
a system that encourages high quality and efficient care through
mechanisms such as value-based purchasing.
The CMS believes that a bundled payment system should promote
efficiency and clinical flexibility for ESRD facilities. The system
should guard against incentives to under-treat patients or to ``cherry-
pick'' patients in order to maximize facility profits. Accomplishing
these goals will require (1) research to support the development of an
adequate case mix adjustment for a fully bundled system, and (2)
mechanisms to ensure beneficiary protections and promote quality care.
The CMS has made significant accomplishments towards implementing a
basic case mix adjusted composite rate system, as required by the
Medicare Modernization Act of 2003 (MMA). Following the MMA's
enactment, CMS funded research activities to develop new case-mix
adjustments, which were implemented in April 2005. Since then, CMS has
pursued several research approaches that could be used in a
demonstration of a bundled PPS for ESRD facilities.
At this point, CMS is continuing its research on approaches that
achieve our goals related to quality and payment accuracy. Development
of a payment model that addresses the substantial variation in the
dosage of erythropoietin has been a key area of this research. We
continue to devote a considerable amount of time and resources to
developing an appropriate ESRD payment system, including further
research on targeted case-mix adjusters and quality incentives. We
expect to detail the results of this work in the report to Congress
required by section 623 of the MMA and move forward with a
demonstration to further test these approaches, as the law requires. We
expect these efforts, coupled with prior research, will provide a well-
informed basis for comprehensive ESRD payment reform in the future.
Conclusion
This Administration has made significant strides in promoting and
ensuring quality care for ESRD patients. From the CMS Quality Roadmap
and efforts under the ESRD Quality Initiative like Fistula First, to
selecting a Chronic Kidney Disease--focused site under the Care
Management for High Cost Beneficiaries Demonstration, to ongoing
research in support of comprehensive ESRD payment reform, CMS is
helping to improve quality and efficiency in the care of ESRD patients.
The significant strides made over the last 5 years have laid important
groundwork for further improvement. The CMS will continue to build on
these efforts, and looks forward to further work with the Congress and
the ESRD community to achieve our common goals.
Ms. NORWALK. Before we start, if I might ask my colleague,
the Chief Medical Officer of CMS, who is a nephrologist, Barry
Straube, to sit with me, that would be great.
Chairman THOMAS. Welcome, Dr. Straube.
You indicated--and all of us, obviously, have been
interested in this focus at the continuing increase percentage,
now to 39; and your argument, as I heard it, was that this was
done in an attempt to provide a payment regulation structure.
Because, obviously, a physician should be the one to determine
that level.
I guess the difficulty I have in responding to that is
that, notwithstanding the FDA label position, the manufacturer
itself responding to concerns about that, the studies that we
now have, which certainly any scientific method needs to be
duplicated, followed up, examined, CMS continues to increase
the percentage; and the argument for it, as I hear from you, is
that it is a payment construction.
Of course, we understand your need to create systems that
assist you in making payments. In fact, the whole discussion of
bundling is to try to change the payment system. I guess, based
upon the studies that we have seen, which certainly have to be
validated and duplicated, this is something that probably
should not be looked at as a payment question, absent pretty
clear evidence of what is happening on overdosage, which if you
had kept the policy at, say, the FDA label which it was at one
time of 33 percent we would not be having those consequences.
So, it is difficult for me to listen to a justification for
a level driven by a payment policy, when all of the evidence I
have heard is focusing on the medical. I understand you are not
supposed to make the medical decision. But if that is the case,
why would you then go ahead and change the FDA labeling? For
payment purposes?
Ms. NORWALK. I think there are a couple of different points
I would make, Mr. Chairman.
The first is that the change in monitoring policy over this
past year was critically important because our prior policy was
not doing an effective job in reducing the dosage of Epo. At
37.5 and a 3-month rolling average, it was phenomenally
difficult for our carriers to implement and actually go after
those providers who were actually overdosing on Epo. It was
difficult for the providers to follow that rolling average.
So, we felt it important, in order to keep the dosage down,
to readjust our policies so that carriers could follow as well
as the providers; and I can--I will talk in a second about how
exactly that policy works.
But I would like to point out a couple of things in the FDA
label, because I think it is important for this discussion as
we focus on the clinical need. Of course, understanding that
this is something that we have asked providers to follow, there
are a number of things.
One, the label talks about the idea that sufficient time
should be allowed to determine a patient's responsiveness to a
dosage of Epo before adjusting the dose. In fact, the label
talks about an interval of 2 to 6 weeks that may occur between
the time of dose adjustment and a significant change in
hematocrit levels. That is important because our payment
systems are on a monthly basis.
Since it may take 2 to 6 weeks--in fact, at the longer
weeks, 6 weeks--for that to adjust, we did not want to have a
payment system that penalized a provider who was actually doing
the right thing, because the patient, for whatever reason, has
his own physiological change to the drug which had not yet gone
into effect.
Moreover, the label talks about dose adjustment as saying
that if the hematocrit level exceeds 40 percent, the dose of
Epo should be withheld until the hematocrit falls to 36
percent. So, one thing that the label does do in terms of a
number is not actually 39, which is our level for payment
changes, but is in fact 40 before the dose would be withheld.
Chairman THOMAS. I understand that. That gets back, I
guess, to an earlier question that still perplexes a number of
us.
In dealing with the payment concerns in allowing for an
adequate leeway for the medical decisions, was there any
discussion at all about trying to go public and push the idea
that perhaps no update for this particular area of Medicare
services, as opposed to skilled nursing, as opposed to any of
the others, would be one of the fundamental changes that you
could make and that income from drug usage which is used to try
to augment the fact that there is no update could easily be
changed, which would be payment questions--instead of dealing
with the dosage on payment questions, which continues the
perverse aspect of a monopoly drug being overused to help
compensate for the cost structure in what you are monitoring
says is important for payment purposes?
Ms. NORWALK. I think----
Chairman THOMAS. In other words, you created a trough--not
you, CMS--and CMS is staying in this trough in trying to
control the payment structure. When all you had to do is take a
step back and say they are not getting an update. They should
be updated regularly like everyone else, and it should not be a
monopoly drug.
For example, I would be interested in asking a question
along this line, and that is that what we did with the part B
drugs in MMA was to require a single rate based on sales across
all settings. So my question then, going to this issue, would
be: Why does Medicare and the beneficiaries pay more for Epogen
Alpha per 1,000 units to treat anemia in a dialysis center,
compared to the same 1,000 units administered to cancer
patients in the physician office setting?
Clearly, part of Dr. Singh's and other testimony is that
there is a whole lot more of an analogous relationship than it
appears to be and that that goes to the payment question again
on a monopoly structure. Why are you paying two different rates
for similar usages?
Ms. NORWALK. One of the things that the MMA does under
section 303(c) is require that single-source drugs be paid by
ASP plus 6. That is in the physician office setting.
The interesting piece here is that when we looked at the
OIG study--and I think the GAO has a table of this in its
report--that if you look at the OIG study, the OIG study
details what the acquisition costs were for Epo in 2005. The
ASP plus 6 percent is slightly lower, depending on the quarter,
relative to the acquisition cost.
For CMS to update--actually, we found this actually going
down. So, in terms of whether there was an update, I am not
sure that an update was necessary, that ASP plus 6 has actually
come in slightly lower than the OIG's.
Chairman THOMAS. First of all, that is an arbitrary
structure in dealing with a noncompetitive drug, for which I
thought was inadequate at the time we wrote the bill. That is
okay. My question is simply why, in two different settings, for
similar uses, identical drugs are paid at different prices?
Ms. NORWALK. Well, they are both paid at ASP plus 6
percent. In terms of why is really an issue between the
companies and their marketing side.
Chairman THOMAS. One arena is a competitive one and the
other is not. Would that be one of the reasons?
Ms. NORWALK. It may well be that ASP plus 6 in the non-ESRD
market is competitive and may reduce the rate. But at the end
of the day for this, to solve this problem, I agree with the
GAO and, clearly, members of this Committee who think that a
bundled payment, including everything, including currently
separately payable drugs, makes the most sense, provided that
we can get it right to provide the quality for beneficiaries
and appropriately risk adjusted.
Chairman THOMAS. Appreciate that position.
I do believe that I do want to communicate, notwithstanding
the fact that you are new on the job, to those who have been
dealing with this decisionmaking and who have been less than
timely in responding to letters that Congress has written that
you have had since 2003. You are moving forward on it. I
believe the Congress will move if you do not. I am urging that
you do.
Does the gentlewoman from Connecticut have any questions.
Ms. JOHNSON OF CONNECTICUT. Let me just inquire a little
bit more about the demonstration project that you are working
on and the apparent difficulty of establishing an appropriate
case mix adjuster. Could you talk about that a little bit?
Then, also, could you talk about the issues raised by the
preceding panel that implied that if you titrate up you can get
to stability--I do not want to paraphrase them too flippantly,
but it sounded like easily and reliably. If that is the case,
does Medicare reimbursement policy encourage titrating up and
does Medicare reimbursement policy take any position in regard
to what is apparently a more cost-effective delivery system
that is widely used of injection?
Ms. NORWALK. To your first question, Mrs. Johnson, in terms
of the demo, one of the things that we have been struggling
with internally with our report to Congress in putting this
together, we have had an expanded bundle demo where we are
working with members of a FACA Committee that represents
patient groups, clinicians and other stakeholders.
But we have struggled with the ability to predict resource
utilization, which in a sense gets to some of the other
questions that you are asking. Since that is the basis of a
prospective payment system, we have taken longer than we would
like to take. But given that 93 percent of this population is
covered by Medicare, it is so critical that we get it right.
For that reason and because so many of these patients are
disproportionately either African American, Hispanic or
American Indian, this is critical to be accurate, to get it as
close to right as possible before it is implemented.
It is also important because of the small size of some of
the facilities. There are a couple of very big chains here, but
not all of them are big. Consequently, we want to ensure that
we do not put the small companies out of business with our
payment policies.
I think that the FACA Committee will be meeting again early
next year; and, hopefully, we will be moving forward. As we get
the research back, we will be working on our demonstration
project at the same time we are writing our report to Congress
as to not waste any time with either of those so we can get
them done as quickly as possible to you.
In terms of titration--and one of the things I will ask, if
it is okay, my colleague, Dr. Straube, to speak on the more
clinical issues. But in terms of titration, generally one of
things that the label talks about is the variability among
patients. In the label it talks about one of the largest
clinical trials, that approximately 65 percent of the patients
required doses of 100 units or less to maintain their
hematocrit levels at approximately 35 percent, almost 10
percent of patients required a dose of 25 units or less, and
approximately 10 percent required a dose of more than 200 units
to maintain their hematocrit at this level. So, there is a
pretty significant amount of variability there.
You also asked about subcutaneous administration or--
something that I was talking about in my opening statement--
about the use of fistulas, and the fistula first policy. Very,
very important. We would like to see more patients have access
to this. We have been promoting that policy.
I would anticipate under a bundled payment system that we
would see more use of this use of fistulas rather than being as
a apart of the whole dialysis treatment. It may be better for
patients, and I would anticipate that it would also reduce the
amount of Epo that needs to be administered. So, that is why we
have been supporting it.
Finally, if I may, Mrs. Johnson, I think that--I do not
know if we have got copies of this, but one of things when we
are talking about the payment policy here that we in no way at
CMS would like patients to stay at a hematocrit level of 39.
That is not our policy. But what happens is these patients may
be at 39--the hematocrit level may be at 39 for between 2 and 6
weeks; and then they come down to a more reasonable range where
we like to see it because of dosing changes, for example.
Over time--this is something in the American Journal of
Kidney Disease--nearly 30 percent of patients in the first
quarter of the year 2000 were at a hematocrit level that was
over 12. But by the end of the year only 5 percent from that
initial group. So, the numbers actually decreased very
significantly. So, that, over time, the number of patients who
were persistently above 12 in hemoglobin levels had come down
significantly.
You simply have a great deal of variability from one
quarter to the next. That is something that we thought was
important that our policy take into account.
Ms. JOHNSON OF CONNECTICUT. That issue of variability that
you have just described, does that--in other words, if you are
aiming for--if your policy is 33 to 36 percent, any bundled--
any policy has to take into account that you can go above that
and below that and not we lower quality treatment. Am I
understanding you correctly?
Ms. NORWALK. That is exactly the point that we were
concerned about.
One of the things that initially this Committee brought to
CMS's or then HCFA's attention in the late nineties was in fact
underdosing and that we have a policy that does not promote
underdosing because we cut off payment too soon.
Likewise, I appreciate that there is a concern about
overdosing, both for clinical reasons and I want to be sure
that we are walking that very narrow line so that physicians
can follow the label and do what is best for the patients and
not be adversely impacted, that the patient not be adversely
impacted by our payment requirements, something that is
reasonable and necessary versus what might be safe and
efficacious as the FDA determines.
Dr. STRAUBE. Mrs. Johnson, I think--just to reiterate a few
of the points that Leslie made, and you have asked some
important questions here. I think when I look back
historically--and I have taken care of many patients with
eopetin myself as a nephrologist--the first focus back in the
late nineties was the fact that 42 percent of patients had a
hemoglobin of 11 grams per deciliter or greater. So, there was
a preponderance of patients who were very low, and research
studies at that time clearly showed an association with
increased morbidity and increased mortality for people under a
hemoglobin of 11.
So, the trend of everybody involved with this, including
Congress, recommended CMS at the time increase its hematocrit;
and the 90-day rolling average monitoring policy that was put
into effect then was to get people--and has gone from 42
percent to 82 percent in 2005 now----
Ms. JOHNSON OF CONNECTICUT. Eighty-two percent complying?
Dr. STRAUBE [continuing]. that are over 11 grams per
deciliter.
The slide that Leslie has distributed to the Committee is
very, very important. Because people who are correctly
reporting that--at any given point in time there may be 30 or
more percent of people who exceed the 12 grams per deciliter of
the FDA label.
If you look at those patients who consistently stay above
that label, as opposed to occasionally going above it, it does
come down to only 5 percent.
Ms. JOHNSON OF CONNECTICUT. In other words, you do not want
your payment policy to penalize some variation but just to
penalize staying at high levels?
Dr. STRAUBE. Correct. Those patients who consistently
exceed that level. Because most patients do have some
variability; some patients considerable variability. It is in
contrast to perhaps some of the comments in the first panel. I
believe it is very difficult to maintain patients particularly
in that narrow range of 33 to 36 percent hematocrit or 11 to 12
grams per deciliter hemoglobin.
Ms. JOHNSON OF CONNECTICUT. So, you are saying it is very
difficult to maintain them. Because I tried to bring that out.
How easy is stability? They basically all said it is easy.
Dr. STRAUBE. I think it is more easy in the chronic kidney
disease patients who have less factors going on compared to
dialysis patients. But that narrower range, 11 to 12, is
difficult; and some patients just naturally will go in and out
without actually doing anything.
When you are changing doses of erythropoietin, when you are
being exposed to an artificial kidney membrane in a dialysis
machine, when they have other illnesses going on with chronic
inflammation and acute inflammation affecting the possible dose
of Epogen, it is not easy.
Ms. JOHNSON OF CONNECTICUT. So, different categories of
patients may need to be looked at slightly differently, or at
least your payment system wants to be able to take into account
case mix?
Dr. STRAUBE. Yes, indeed.
Ms. JOHNSON OF CONNECTICUT. Thank you. Thank you for these
charts.
Mr. STARK. Thank you, Mr. Chairman.
Welcome to the Committee, Ms. Norwalk.
Ms. NORWALK. Thank you.
Mr. STARK. I just wanted to know, is there any--I do not
know what a valid clinical study would be, but is there a valid
clinical study--I will use that word--that provides clinical
justification for not reducing the dosage until it gets to 39?
Ms. NORWALK. I think our point----
Mr. STARK. Is there any study that would support that that
you know of?
Ms. NORWALK. To not reducing it?
Dr. STRAUBE. Mr. Stark, I do not believe for a general
population there probably is such a study. I would agree. I
think for individual patients there might be indications for
not reducing it.
Mr. STARK. But you do not know of a clinical study?
Dr. STRAUBE. Not in terms of a general population, no.
Mr. STARK. Ms. Norwalk, it is my understanding that CMS
created something called the Epo Monitoring Policy Group,
right?
Ms. NORWALK. Yes.
Mr. STARK. They were supposed to advise CMS on policies
dealing with Epo, right? Now what troubles me is that of the 24
members--I am looking at the list here of maybe 22--18 of them
disclosed financial associations with Amgen or Johnson &
Johnson. Would not that, in fact, indicate that there might be
some prejudice on the part of the people that you picked to
advise you on this policy?
Ms. NORWALK. Either that or they happen to know a fair
amount about the topic. At the end of the day----
Mr. STARK. I know they did. But let's talk about DaVita,
Incorporated and AAMA and Gambro Health Care. They are also
putting a lot of our taxpayers' money in their pockets. When
you got two-thirds of your supposedly independent advisors--if
I may use term loosely--on the take from the people that we are
paying $2 billion a year to, does not that raise the issue that
maybe you are not getting the straight skinny?
Ms. NORWALK. I think it is important--and one of the
reasons you have seen our monitoring policy, that it be put out
publicly, it is important to get comment from across the board.
Mr. STARK. Oh, come on.
Ms. NORWALK. Whether it is that group or whether there is
another group, and at the end of the day----
Mr. STARK. You and I are not doctors, but you are a very
good lawyer. You would tear these guys apart on the witness
stand in 2 seconds, if you found out how much money they were
getting from Amgen. Look, you got the American Kidney Fund.
Amgen funds their clinical fellowship program.
You have got DaVita--that is self-explanatory; the Kidney
Care Partners, a lobbying group for all of the dialysis guys;
the National Kidney Foundation; 19--almost $20 million in
corporate donations from the platinum friends, Amgen, DaVita.
Ms. NORWALK. It is clear that you--how you feel about how
much we pay them.
Mr. STARK. It is a cozy club, isn't it?
Ms. NORWALK. That is one of the reasons why it is so
important that our Chief Medical Officer is a nephrologist.
Barry is not allowed to be paid by any of these groups.
Mr. STARK. I understand. But he is getting advice from
these guys. It just seems to me that the time has come to
understand, you know, these guys are not Bechtel in Iraq, and
we should be getting reasonable and decent advice.
It just seems to me that it is difficult to support, when
you empanel this group, knowing that most of them, your
organization--I am sure you had nothing to do with selecting
them, but it does lead us to become somewhat suspicious that we
are not----
Now I might say there is probably no nephrologist in the
United States--I will give Dr. Singh a chance to get out of
this--that has not received something from Amgen, a golf ball
or a dinner or something over time. So, it might be very hard--
--
Ms. NORWALK. Absolutely.
Mr. STARK [continuing]. to find. Billy Bud would have been
a good three-act play if he had not jumped in the first act.
But, nonetheless, this does seem rather suspect, overloaded
with lobbyists and people who stand to benefit financially,
tremendously, just to be selected.
Chairman THOMAS. I do understand the normal reaction would
be dollars paid to entities as a link that might occur.
I think you also have to take into consideration the fact
that you are dealing with a monopoly drug; and, notwithstanding
it is a monopoly, the Government, unwilling to create a
surrogate monitor for those prices and the fact that their
business is focused solely on use of that drug and the ability
to put pressure on the availability, the cost or the linkage of
that drug, may have as much to do with the positions that
people take than the dollar amounts paid----
That is just the other side of the coin, on allowing a
monopoly not only in the private sector but in terms of those
in Government positions making decisions.
I thank you for yielding.
Mr. STARK. I hope we will have a chance to explore this
further in the months ahead; and we will have ask Chairman
Thomas if he will agree, on a pro bono basis, to continue
advising us for all of the work that he has done on this. Maybe
we can come to a conclusion which would have us, I think, and
what I think my principal concern is is that CMS is the only
group here that we have heard from today--and this is the
troubling part--that has not erred on the side of caution.
I do not buy--and you are the only one who suggests the
lower limits are a danger. Dr. Singh says it is much less of a
danger than going over. So, that I would hope that that could
be the principal change in policy as we try and revise these
payment standards.
Ms. NORWALK. Well, I would be interested in hearing Dr.
Straube talk a little bit about the underdosing of this, which
clearly was a problem once upon a time, as you wrote to us
about it. It may well be that the literature has changed since
then, but in terms of the side of caution I think that we are
cautious.
We do require that providers use the FDA label as a policy.
We tell our contractors that they can, in fact, perform medical
review at lower levels than 39. I think we are working with
what is reasonable and necessary with a patient population that
even the FDA label admits to having variability and want to
ensure that those patients who for whatever reason in a
particular month or 6-week period may find their hematocrit
level above 39 and the doctor does the right thing in reducing
the Epo dosage, not wanting to penalize that doctor.
On the flip side of that, it is not reasonable and
necessary to have a patient persistently at that hematocrit
level of 39; and, consequently, that is why we are taking the
payment reductions there. I just want to be clear that our
policy is: Follow the FDA label.
Now I appreciate we may have--this CHOIR study is a new
study. It is something that we would like to take into account,
like to look at the data there. In particular, I would be
interested in reviewing the data around the 11.3 population,
because they, too, are going to have a bell curve, where they
will have people on the bell curve that are above a certain
number--above 12, I would suspect--and perhaps maybe at any
point in time a number of patients that may be above 13 or 39,
and not wanting to penalize even in that study--when the target
was the appropriate target, not wanting to penalize physicians
because a patient physiology was such that for a particular
moment in time, a static moment, that patient was above 39.
Mr. STARK. Thank you.
Dr. STRAUBE. Mr. Stark, I think we do very seriously take
Dr. Singh's study, for instance, and are considering that.
As you know, when a scientific study is presented in the
peer review literature, it has to get first by the review board
of the journal of which it is published. But, subsequently, the
medical community has to look at that article and put it into
context, having scrutiny of the whole methodology behind it, as
well as the conclusions.
We are going to be participating with that. I have already
talked with Dr. Singh. We would like some more of his data. But
we should also have caution against jumping to one--to taking
action that might have perverse consequences if we jump too
soon.
The bell curve--there is evidence that the bell curve that
we have described in terms of what dosage patients require,
when you shift patients from the high end to the left, that is,
to lower hemoglobins, you drive other patients down below 11.
As we talked about earlier, it is just as dangerous, if not
more so, to have low hemoglobin and hemocratics. So, we do not
want to have unintended consequences from jumping to take
action from a study that has just come out and has not had full
scrutiny yet.
Just to end, there was a report in the last week, as an
example, reiterating some reports that have come out over the
years that, for instance, patients who were End-Stage Renal
Disease patients and are obese do better than non-obese
dialysis patients. If we were to respond to that and say, gee,
we ought to recommend obesity in all of our dialysis patients
because their outcomes are better, I think people would not
think that was a smart thing to do.
So, I think that we have to have some caution, although we
are very seriously looking at Dr. Singh's research.
Chairman THOMAS. Thank you.
Dr. Straube, your last statement was, as far as I am
concerned, absolutely insulting, coming from a doctor,
indicating an example of what you would not do, okay?
Number one, I think you need to seriously consider that
your payment policy is killing people, okay?
The gentleman from California introduced a letter in the
record from 1997 because there is a whisper campaign going on
utilizing data from statements from that long ago to try to
intimidate, which is a typical practice in this area.
I did not even bother to introduce my letter from 1998,
which is also being used. Because, frankly, there was a problem
then. It was too low. At some point somebody has got to
consider that perhaps it is now too high, okay?
Ms. Norwalk, for you to say it may well be that literature
has changed since that time, what do you think that whole first
panel consisted of?
Ms. NORWALK. That is my point. The point I have is that
both underdosing and overdosing is something that is important.
Chairman THOMAS. Absolutely. Now we are focusing on
overdosing, whereas letters from 1997 or 1998 were focusing on
underdosing. In between those two periods, we have turned a
monopoly into a multi-billion dollar proposition.
I will tell you, as far as the Chair is concerned and other
Members of the Committee who are not here--and I can assure you
that it is shared broadly by the Committee, perhaps not as
vocally as the Chairman would present it--it is absolutely
unconscionable for CMS to allow a monopoly drug to be bundled
with other drugs in an attempt to force usage; and that is what
is going on.
Ms. NORWALK. Well, I look forward to our report to Congress
coming to you and be able to come up with a bundled system
where payment--where we do not have separate payment.
Chairman THOMAS. I am not talking about that. I am talking
about current practice in which we pointed that out to CMS, and
I haven't gotten an adequate response. Why would you allow
anyone to take a monopoly product--monopoly by the decision of
policy, not by the uniqueness in the industry--to be bundled
with other drugs that are also necessary, to create an
artificial demand for a product in which they control the
absolute existence of the business? That is what is going on
right now.
Ms. NORWALK. So, your point is, the payment rate should be
the same in the ESRD facility as the ASP plus 6 is in other
payments?
Chairman THOMAS. No. My only point was, why would you take
a drug which is a monopoly and allow the industry, the person
who produces it, to bundle it with other products they are
using to influence how much they can use and when, based upon a
pure pricing policy that we have created? That is what we are
doing right now.
Ms. NORWALK. So, when you talk about the monopoly--are you
referring to the Neulasta and the bundling? Is that what your
concern is?
Chairman THOMAS. I am concerned with any monopoly drug
accompanied with others that are not in which there could be a
control of supply, utilizing the monopoly drug as the key.
Ms. NORWALK. I misunderstood your original question. I
apologize.
Chairman THOMAS. I am not trying to not to get into very
narrow, particular examples. I would have that same feeling
about any monopoly drug being allowed to be combined with
others in which you can control the market and the purchase of
other drugs, and CMS has not addressed that. We have pointed it
out, we have asked you to respond, and we haven't had an
addressing on that.
Ms. NORWALK. I think it is something that we will clearly
continue to review. It is something that I would imagine that
the Antitrust Division at Justice and the FTC should be
reviewing. I also imagine that it is something that--it may
have other implications for fraud and abuse.
Chairman THOMAS. I do not think we have to go through the
Justice Department when all you have to say is, if we have
created a monopoly by virtue of our decisions, you do not get
to do that. You do not need the Justice Department to make the
decision. Perhaps your lawyer side is genuflecting in that
directing. Your medical side, which you are now beginning to
learn, ought not to go in that direction, but simply say, if we
are creating an artificial monopoly for you, you ought not to
be able to do those sorts of things, or it won't be a monopoly.
In fact, I think it should have a surrogate price, anyway.
Ms. NORWALK. We will go ahead and take a look at that
particular issue and ensure, as we always do, that whatever our
policy is it does not have impacts to the patient that are in
any way, shape or form a negative, and appreciating, of course,
that we want to always want to watch the public fisc, as we are
required to do.
Chairman THOMAS. Where we are now is we have a payment
policy that perhaps is killing people; and we are using $2
billion, the highest price paid in a relatively narrow area for
the use of the drug through the payment policy, that may in
fact be doing that. That is exactly where we are today.
Ms. NORWALK. Well, I will not quibble with your last part
of the statement. I will look at that.
I would disagree that, respectfully, Mr. Chairman, I do not
believe our payment policy in any way, shape or form is out to
kill people. In fact, what it is intended to do is ensure that
the physician has the ability to monitor where the patient is
so that the physician can appropriately titrate the dose for
that patient, for that patient's best interest.
Chairman THOMAS. I put it in that phrase because I have
learned over the years--and this is the only way you can do
it--that somebody is actually going to pay attention to what I
say if I put it in that extreme position.
But just as we were not doing a medical service to those
patients when we were underdosing, the argument that somehow we
do not want to take away the opportunity on the upper end, with
the understanding, not just the doctor making a decision but a
perverse payment system in which the providers of the services
are not given any kind of an ongoing periodic update, and that
they have gone for literally decades without a payment, and
that the only way they make the margin of the income is on a
drug which is an artificial monopoly imposed by Government
itself.
When you begin building that kind of a structure, to argue
that you are not going to examine the upper level because you
want to leave it to the physicians is to ignore the entire
dynamic and let me say also ignore the kind of business
practices that have been carried out by these people in the
shadows for a number of years.
One of the reasons I want to compliment the people who are
now putting out the kinds of studies which you are going to
look at and are going to find they are good is the fact that
within a 3-month period we have turned completely around that
shadowy area in which various pressures have been carried on
for years in which they cannot now be done, because it is
clearly public, and that if that kind of behavior continues it
will be exposed more in the popular press.
But we now have a series of studies I expect you to look
at. You are new. It was 8 months that you were not there, that
you did not respond to the letters that we wrote. But I expect
to see where you are on your bundling package.
As the gentleman from California indicated, it will be pro
bono, but he means I am going to be able to participate. I do
not believe in life after death, but I do not believe
retirement is death, either.
We are going to continue to look at this area for two
fundamental reasons: I am very much concerned about the health
of these patients, and I am very much concerned about the
enormous dollar amount that will produce the kind of behavior
in both the manufacturers of this and the users of it if we do
not create an opportunity for medically appropriate alternate
patterns to live.
We have a monopoly on the structure, we have a monopoly on
the drug, we have a monopoly on what they are required to do
because of the payment system. We have got to free it up, even
if we free it up with an artificial competition structure; and,
in my opinion, we are a little overdue from the Medicare
Modernization Act of 2003.
You may not be able to get your risk adjustment right. You
can deal with something like the monopoly payment structure.
You can deal with something like--and I really applaud you on
the fistula first. As far as I am concerned, that ought to be
an incentive in the payment structure to allow those various
functions.
All of those can be put in place without waiting for a risk
assessment structure. Those are pure payment policies devoid of
any concern about the dosage structure and the rest. I do not
know why those are not done already.
Ms. NORWALK. We will take a look at that.
Chairman THOMAS. I appreciate every time you said, ``We
will take a look at that.'' What we are trying to tell you is
we directed you in MMA. Taking a look at it is not enough. We
expect behavior on a relatively short timeframe or I might be
able, in a pro bono way, to convince Congress that we perhaps
begin to move from a legislative point of view. That would not
be the desired choice, but it will be a choice if we do not
begin to see in the ancillary areas, not the core, changes.
Ms. NORWALK. As I noted, we hope to have the report to
Congress research to us where we will be building the demo and
the actual report to Congress itself in short order; and I hope
to have the report to the Committee by the summer.
Chairman THOMAS. Thank you very much.
With that, the Committee stands adjourned.
[Whereupon, at 12:45 p.m., the hearing was adjourned.]
[Questions submitted from Mr. Johnson of Texas and Mr.
Weller to Ms. Norwalk, and their responses follow:]
Question from Mr. Johnson of Texas to Ms. Norwalk
Question: As someone who is vigilant over taxpayer dollars, I
appreciate the GAO being here today, and the way CMS has taken steps to
check the economic incentives for the utilization of drugs in the ESRD
program. But while removing incentives that might encourage the over-
utilization of drugs may benefit the taxpayer, it's also very important
to focus on ways to improve the quality of care provided to
beneficiaries.
I have here a list of clinical studies [attached below] that
suggest that more frequent dialysis--which is often provided in one's
own home--may significantly reduce the need for Epogen and other costly
medications.
Earlier this year, MedPAC raised some issues with the payment
surrounding home dialysis in its March report to Congress. Is CMS
exploring other potential policy changes that support more frequent
dialysis, which may reduce the clinical need for Epogen while improving
the quality of life for the patient?
[The studies referred to by Mr. Johnson of Texas are being retained
in the Committee files.]
Answer: Yes, the Centers for Medicare & Medicaid Services (CMS) is
exploring other potential policy changes that support more frequent
dialysis. Currently, there are two clinical trials in frequent dialysis
sponsored by National Institutes of Diabetes and Digestive and Kidney
Disease (NIDDK) and CMS. The goal of these trials is to test the
clinical outcomes relative to daily hemodialysis (five or six times per
week) compared to conventional hemodialysis (three times per week). The
trials are expected to be completed in 2010 at the earliest. Data
collection has already begun, starting in 2005.
______
Question from Mr. Weller to Ms. Norwalk
Question: The Hearing Notice cited that ``Between 1998 and 2003,
ESRD treatment spending increased by almost 50 percent.'' Obviously, we
want and need to be good fiscal stewards of the Medicare Program. My
concern is that we're jumping to conclusions on over-utilization of
EPOGEN with no accounting for ESRD patient growth, how co-morbidities
affect the treatment (for instance, diabetes is a major contributing
factor to ESRD--does that affect spending), the increased frequencies
of those co-morbidities, and the fact that the ASP system has driven
down Medicare payments for drugs and biologics since 2003 (including
for EPOGEN). Further, the EMP is now in place to catch any doctors who
are dosing patients to maintain them above the target hemoglobin
levels. Don't you think we need to move slowly and be fully informed
prior to legislating on the ESRD program?
Answer: Yes, we agree that additional information on the
appropriate use of erythropoietin stimulating agents (ESAs) in the
treatment of anemia is needed to develop a more complete picture of its
effect on different patient populations. Precipitous action could
actually harm patients unless we are clear regarding the benefits to be
gained from policy changes in this area.
The recent FDA black box warning for erythropoietin and
darbepoietin clearly indicates that there is significant concern that
Medicare beneficiaries may be harmed by these drugs. This warning was
precipitated by a number of new clinical studies relevant to the use of
ESAs to treat anemia in cancer patients. In light of this recent
research and the FDA warning, we have taken immediate steps to address
patient safety concerns for the non-ESRD Medicare population, such as
opening a national coverage analysis on the use of ESAs for conditions
other than ESRD. In addition, we made sure that Medicare's local claims
processing contractors were aware of the FDA warning. We understand
that, following the FDA warning and the subsequent revision of the
compendium citation, most if not all local Medicare contractors have
reviewed their policies on the use of ESAs in beneficiaries whose
anemia is related to cancer
For Medicare beneficiaries with ESRD, we are working closely with
the FDA to better understand potential patient safety concerns
associated with the use of ESAs to treat this patient population. In
May, the FDA will be holding an advisory Committee meeting to discuss
the use of ESAs in cancer treatment. CMS is working with the FDA to
plan a similar advisory Committee meeting on the use of ESAs for
patients with chronic kidney disease and ESRD to be held later this
year. In addition, we have been discussing safety concerns regarding
the use of ESAs in ESRD patients with renal professional associations,
large dialysis organizations, academic medical centers, pharmaceutical
companies, and other interested parties to gather as much information
as possible. In the course of gathering this information, it has become
apparent that additional research is needed to address all of the
questions being raised about the use of ESAs for this patient
population. As a result, CMS has begun preliminary discussions with the
National Institutes of Health about the possibility of collaborating on
a large clinical trial of ESA effects in ESRD patients to assess these
patient safety issues. At the same time, CMS is reviewing its recently
improved ESA monitoring policy and is in the process of implementing a
requirement for the use of modifiers to identify the route of
administration of ESAs. All of these actions will help produce the
information that is needed to support more definitive conclusions in
this area.
______
Question: At the end of the day, aren't physicians responsible for
hemoglobin levels and the EPO doses?
Answer: Yes. Medicare policy for the ESRD setting is intended to
ensure that medical decisions are made by physicians, generally
adhering to national guidelines and expert recommendations, such as the
Kidney Dialysis Outcomes Quality Initiative (KDOQI) guidelines.
However, our payment policy takes the FDA label safety issues into
account.
______
Question: In a recent New England Journal of Medicine article there
was reference to patients being treated with Procrit in the CHOIR study
at an average hemoglobin level of 12.6 grams per deciliter. From the
testimony today, it is clear that CHOIR was a clinical trial and that
patients were being treated toward a target hemoglobin that exceeds the
FDA label for EPOGEN.
Another problem I'm having with details of this study is that these
were non-dialysis patients that were treated as part of a clinical
trial. Lastly, I understand that CHOIR study did not follow the gold
standard of a double blinded design. Therefore, I don't see how
findings from a clinical trial on non-dialysis patients can be linked
across to ESRD patients. If Medicare dialysis patients were being
maintained at this level, wouldn't these patients be flagged by the EMP
and their doses reduced per the new CMS policy?
Answer: The new Medicare policy was not in effect during the CHOIR
study. As mentioned earlier, the Medicare policy is intended to ensure
that medical decisions are made by physicians and is consistent with
the FDA label and current kidney disease industry guidelines to
maintain a target hemoglobin level in the range of 10 g/dl to 12 g/dl.
02___
[Submissions for the record follow:]
Statement of American Society of Pediatric Nephrology,
Indianapolis, Indiana
The American Society of Pediatric Nephrology (ASPN) appreciates
this opportunity to submit testimony for the record of the Committee on
Ways and Means hearing on ``Patient Safety and Quality Issues in End-
Stage Renal Disease Treatment.'' The ASPN is a professional society
composed of pediatric kidney specialists whose goal is to promote
optimal care for children with kidney disease and to disseminate
advances in the clinical practice and basic science of pediatric
nephrology. The ASPN currently has over 600 members, making it the
primary representative of the pediatric nephrology community in North
America.
Background
Anemia is a complication of kidney disease, known as Chronic Kidney
Disease (CKD) or kidney failure, and End Stage Renal Disease (ESRD) or
Stage V CKD. Patients with kidney failure suffer from anemia because
their kidneys do not produce a hormone (erythropoietin) that regulates
red blood cell production. Anemia directly affects a pediatric
patient's quality of life, including neurocognitive development, school
attendance, exercise capacity and family support,\1\ making proper
anemia management critical to a patient's well-being. One of the key
medications used to treat anemia in this population of patients is
recombinant human erythropoietin (rHuEPO), commonly referred to as EPO.
---------------------------------------------------------------------------
\1\ American Journal of Kidney Diseases, S90-S92 (May 2006).
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Doctors determine a patient's degree of anemia with simple blood
tests, measuring the hemoglobin level. The hemoglobin levels that
define anemia in children with kidney disease differ from those in
adults, as they depend on the age and gender of the patient. In the
case of those patients who are then treated with EPO, the existing
National Kidney Foundation Kidney Disease Outcomes Quality Initiative
(NKF KDOQI TM) or KDOQI TM opinion-based
guidelines recommend a target hemoglobin level of 11.0 to 13.0 g/dl,
for children up to 19 years of age. Treatment thresholds in anemia
management should always be individualized to the needs of the patient,
allowing a trained professional, in consultation with the patient, to
determine the optimal dosing of EPO.
In light of the recent studies published in the New England Journal
of Medicine, ASPN agrees that it is essential for the kidney care
community to continue examining all available data to ensure that
public policies reflect appropriate anemia management for all patients,
both children and adults, with kidney disease and kidney failure.
However, it is important to point out that no reliable scientific
studies have been published that examine optimal hemoglobin levels for
children with CKD. For this reason, the ASPN requests that Congress
commission a study through the National Institutes of Health to test
and evaluate optimal hemoglobin levels specific to children with kidney
disease. Armed with this scientific literature, the kidney community
and government can work to promote the safest practices with the
highest quality of care for children with this chronic disease. ASPN is
committed to working with clinical researchers to carry out such
scientific studies.
Children Are More Vulnerable Than Adults
It has been said that children are not little adults, and this is
especially pertinent in the treatment of children with both CKD and
ESRD. Proper EPO dosing must take the age of the patient into
consideration. Furthermore, and in contrast to the adult patient, the
developing minds and bodies of children with kidney disease places them
at a disproportionate risk in the event of inappropriate anemia
management. Poor statural growth, impaired nutrition and abnormal
cognitive development are all potential adverse outcomes of poor anemia
management that mandate prospective study.
Children Have Unique Treatment Needs
Once children are diagnosed with CKD or ESRD, it is critical that
the pediatric nephrologist be able to adequately target the proper
hemoglobin level for the patient. Due to their size and age, a child's
body will respond differently than adults in similar stages of CKD or
ESRD. Consequently, pediatric treatment needs are unique in several
ways:
Children need different dosages of erythropoietin than
adults--not only because they are smaller, but also because the way
their bodies metabolize the drug may be different than what occurs in
adults.
Children sustain unique developmental and psychological
responses to kidney disease and kidney failure. The identification and
optimal management of these disorders in children and their
relationship to anemia management requires professionals with expertise
in pediatric nephrology.
Most importantly, there are distinct differences in the
frequency and type of co-existing illnesses that characterize the adult
and pediatric CKD populations which may result in the optimal
hemoglobin targets for children and adults receiving EPO to be
different.
Conclusion
ASPN appreciates the opportunity to provide these comments to the
Committee. The kidney community is largely unified in communicating a
concern that actions taken by Congress and the Centers for Medicare and
Medicaid Services (CMS) to revise anemia management guidelines must be
based on all available scientific literature. The recent studies
published in the New England Journal of Medicine only address the adult
chronic kidney disease population. For this reason, it is imperative
that further anemia management studies be conducted in all CKD and ESRD
populations, including children, to ensure that revised government
policies reflect sound scientific evidence.
The Society remains dedicated to providing the highest standard of
care and ensuring patient safety for our nation's pediatric kidney
disease patients.
Statement of Amgen
Mr. Chairman, Mr. Rangel and Members of the Committee,
Amgen is pleased to submit this testimony for the record of the
Committee on Ways & Means Hearing on patient safety and quality of care
for Medicare beneficiaries with End Stage Renal Disease (ESRD).
Amgen, one of the biotechnology industry's founders and pioneers,
delivers vital medicines to fight serious illness. Amgen scientists
have discovered and brought to market novel therapies that have helped
millions of patients. Today, with a robust pipeline of potential new
medicines, Amgen is investing billions of dollars in research and
development to bring promising new therapeutics to patients.
I. The Benefits of EPOGEN' (epoetin alfa) for Treating
Anemia in KIDNEY DISEASE
Chronic kidney disease is an increasingly recognized and important
public health issue, affecting approximately 20 million Americans. The
most advanced stage of chronic kidney disease is ESRD. Patients at this
stage have inadequate kidney function to rid the body of harmful
toxins, and it is fatal unless treated with dialysis, an artificial
means of filtering the blood. In 1972, Congress enacted legislation
ensuring that people with advanced kidney failure would be able to
receive dialysis and other potentially life-saving treatments under
Medicare.
ESRD patients are highly vulnerable, and there are more than
300,000 people with kidney failure being treated with dialysis in the
United States today. Approximately one-third are African-American, and
1 in 5 are Hispanic. The mean age of dialysis patients in the U.S. is
approximately 65. Dialysis patients typically carry a heavy burden of
other medical conditions, including high blood pressure, diabetes,
heart disease and anemia. Given these facts, it is not surprising that
the survival rate of dialysis patients is quite low. In fact,
approximately 1 in 5 dialysis patients die every year--a death rate as
high as that seen with many cancers. Indeed, patients who require
dialysis are very sick, and the health care professionals who care for
them are highly specialized in their understanding of how best to treat
this precarious patient population.
Over 90% of ESRD patients develop anemia, a serious health
condition that places patients at increased risk of hospitalization and
mortality.
The kidneys produce a hormone called erythropoietin, which signals
the body to make red blood cells in the bone marrow. Red blood cells
carry life-sustaining oxygen from the lungs to all the vital tissues in
the body. Without enough erythropoietin, patients develop anemia, or
low numbers of red blood cells. Anemia is measured by a lab test called
hemoglobin. Healthy people have hemoglobin levels in the 14-16 grams
per deciliter (g/dL) range. If untreated, dialysis patients have
hemoglobin levels that are much lower, often in the range of 8-10 g/dL
or lower.
Anemia affects approximately 9 out of every 10 dialysis
patients.\1\ Patients with anemia suffer from severe fatigue and
markedly reduced quality of life. Anemia increases the likelihood of
being hospitalized and using more health care resources.\2,3\ In
addition, dialysis patients with anemia are at risk for cardiovascular
events like heart attack or stroke. And dialysis patients with anemia
are more likely to die than those without anemia.\4\
---------------------------------------------------------------------------
\1\ USRDS 2006 Annual Data Report
\2\ Collins AJ. Li S. Ebben J. Ma JZ. Manning W. Hematocrit levels
and associated medicare expenditures. American Journal of Kidney
Diseases. 2000 36(2):282-293.
\3\ Collins AJ, Li S, St Peter W, Ebben J, Roberts T, Ma JZ,
Manning W. Death, hospitalization, and economic associations among
incident hemodialysis patients with hematocrit values of 36 to 39%. J
Am Soc Nephrol. 2001 Nov;12(11):2465-73.
\4\ Roberts TL, Foley RN, Weinhandl ED, Gilbertson DT, Collins AJ.
Anaemia and mortality in haemodialysis patients: interaction of
propensity score for predicted anaemia and actual haemoglobin levels.
Nephrol Dial Transplant. 2006 Jun;21(6):1652-62.
---------------------------------------------------------------------------
Before the advent of EPOGEN' more than a decade and a
half ago, physicians had few options for treating anemia in ESRD
patients, and had to rely on blood transfusions. Unfortunately, blood
transfusions put patients at risk for complications such as blood-borne
infections and iron overload. Blood transfusions also limit the chances
for patients to successfully receive kidney transplants.\5\
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\5\ Lietz K, Lao M, Paczek L, Gorski A, Gaciong Z. The impact of
pretransplant erythropoietin therapy on late outcomes of renal
transplantation. Ann Transplant. 2003;8(2):17-24.
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EPOGEN' has reduced the need for blood transfusions and
improved health-related quality of life in dialysis patients.
Amgen has pioneered the development of innovative medicines that
safely and effectively treat anemia. EPOGEN' was developed
by Amgen scientists using recombinant DNA technology, and has the same
biological effects as naturally occurring erythropoietin.
EPOGEN' was approved by the FDA in 1989 for the treatment of
anemia in patients on dialysis.
The availability of EPOGEN' as a medicine to treat
anemia has been one of the major breakthroughs in treatment for
dialysis patients. Patients who are treated with EPOGEN'
have a dramatic reduction in the need for red blood cell transfusions,
and their quality of life is markedly improved by reducing fatigue
symptoms, increasing energy level, improving physical function, and
improving sleep.\6,7\
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\6\ Eschbach JW, Abdulhadi MH, Browne JK, Delano BG, Downing MR,
Egrie JC, Evans RW, Friedman EA, Graber SE, Haley NR, et al.
Recombinant human erythropoietin in anemic patients with end-stage
renal disease. Results of a phase III multicenter clinical trial. Ann
Intern Med. 1989 Dec 15;111(12):992-1000.
\7\ Evans RW, Rader B, Manninen DL. The quality of life of
hemodialysis recipients treated with recombinant human erythropoietin.
Cooperative Multicenter EPO Clinical Trial Group. JAMA. 1990 Feb
9;263(6):825-30.
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II. Quality of care and Safety profile of EPOGEN' in esrd
In its hearing notice, the Committee appears to have specific
concerns about safety and quality of care with regard to anemia
treatment in ESRD. Amgen shares the Committee's concerns for patient
safety and quality of care and appreciates this opportunity to respond
and to correct misinformation that the Committee has received.
EPOGEN has enabled the safe and effective treatment of anemia in
patients with ESRD.
EPOGEN' has been shown to be safe and effective in
multiple clinical trials and has over a decade and a half of safety
monitoring of real world use in more than 1.5 million dialysis
patients. When used according to its FDA-approved label,
EPOGEN''s safety profile is well-established and widely
known. The most frequently reported adverse events are detailed in the
product's label, which accompanies every vial of the product that is
sold. Recent safety concerns have arisen from experiments which target
and maintain hemoglobin levels above those recommended by the FDA
(discussed in later section).
Quality of care for anemia treatment in dialysis patients is measured
by the percentage of patients whose hemoglobin level is
maintained > 11 g/dL.
While the FDA label directs clinicians to target a range of 10-12
g/dL, U.S. clinical practice guidelines reflect a review of the
totality of evidence\8\, including United States Renal Data System
(USRDS) data showing that dialysis patients who have hemoglobin levels
of 10-11 g/dL have an 18% increase in their risk of death and an 8%
increase in their risk of being hospitalized when compared to patients
with hemoglobin levels between 11-12 g/dL.\9\ At the recent annual
meeting of the American Society of Nephrology in San Diego last month,
Allen Nissenson, M.D., Professor of Medicine at UCLA reminded the
community that clinical practice guidelines state that ``. . .
Hemoglobin should be 11.0 g/dL or greater.'' He noted that this
evidence-based recommendation was the result of review of 22
randomized, controlled clinical trials and evaluated a number of key
clinical outcomes such as mortality, cardiovascular events,
hospitalization and quality of life.\10\
---------------------------------------------------------------------------
\8\ KDOQI; National Kidney Foundation. Clinical practice guidelines
and clinical practice recommendations for anemia in chronic kidney
disease in adults. Am J Kidney Dis. 2006 May;47(5 Suppl 3):S16-85.
\9\ Roberts TL, Foley RN, Weinhandl ED, Gilbertson DT, Collins AJ.
Anemia and mortality in haemodialysis patients: interaction of
propensity score for predicted anemia and actual hemoglobin levels.
Nephrol Dial Transplant. 2006 Jun;21(6):1652-62. KDOQI; National Kidney
Foundation. Clinical practice guidelines and clinical practice
recommendations for anemia in chronic kidney disease in adults. Am J
Kidney Dis. 2006 May;47(5 Suppl 3):S16-85.
\10\ Slides presented by Allen Nissenson, MD. Professor, David
Geffen School of Medicine at UCLA at the 36th Annual American Society
of Nephrology Meeting. San Diego, CA. November 2006.
---------------------------------------------------------------------------
It is well documented, in both domestic and international studies,
that maintaining patients with a hemoglobin of less than 11 g/dL is
associated with increased hospitalization, healthcare expenditure, and
mortality.\11,12,13\ This finding has been reflected in the National
Kidney Foundation (NKF) 2000 and 2006 K/DOQIT Guidelines. Moreover, CMS
has developed and implemented clinical performance measures (CPM),
which can be used to assess the quality of care in dialysis facilities
across the U.S. One CPM (or quality indicator) has been defined as the
percentage of patients with a hemoglobin level greater than 11 g/dL.
This same quality indicator has been employed in Europe through the
European Best Practices Guidelines (EBPGs) and in Australia through
Australian guidelines.\14,15\
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\11\ Wolfe RA, Hulbert-Shearon TE, Ashby VB, Mahadevan S, Port FK.
Improvements in dialysis patient mortality are associated with
improvements in urea reduction ratio and hematocrit, 1999 to 2002. Am J
Kidney Dis. 2005 Jan;45(1):127-35.
\12\ Locatelli F, Pisoni RL, Combe C, Bommer J, Andreucci VE, Piera
L, Greenwood R, Feldman HI, Port FK, Held PJ. Anemia in haemodialysis
patients of five European countries: association with morbidity and
mortality in the Dialysis Outcomes and Practice Patterns Study (DOPPS
Nephrol Dial Transplant. 2004 Jan;19(1):121-32.
\13\ Volkova N, Arab L. Evidence-based systematic literature review
of hemoglobin/hematocrit and all-cause mortality in dialysis patients.
Am J Kidney Dis. 2006 Jan;47(1):24-36.
\14\ Locatelli F, Aljama P, Barany P, Canaud B, Carrera F, Eckardt
KU, Horl WH, Macdougal IC, Macleod A, Wiecek A, Cameron S; European
Best Practice Guidelines Working Group. Revised European best practice
guidelines for the management of anaemia in patients with chronic renal
failure. Nephrol Dial Transplant. 2004 May;19 Suppl 2:ii1-47.
\15\ Roger S; Caring for Australians with Renal Impairment
(CARI).The CARI guidelines. Haematological targets. Iron. Nephrology
(Carlton). 2006 Apr;11 Suppl 1:S217-29.
---------------------------------------------------------------------------
In addition to the wealth of clinical data that supports
maintaining hemoglobin levels above 11 g/dL, there is strong evidence
that this is also cost-effective. Data from the USRDS demonstrate that
patients who achieve hemoglobin levels between 11-12 g/dL save the
Medicare system approximately $675 per member per month as compared
with patients who achieve hemoglobin levels between 10-11 g/dL.\16\
---------------------------------------------------------------------------
\16\ Collins AJ, Li S, St Peter W, Ebben J, Roberts T, Ma JZ,
Manning W. Death, hospitalization, and economic associations among
incident hemodialysis patients with hematocrit values of 36 to 39%. J
Am Soc Nephrol. 2001 Nov;12(11):2465-73.
---------------------------------------------------------------------------
Importantly, there have been tremendous improvements in the quality
of care for dialysis patients over the past decade. According to the
USRDS 2006 Annual Data Report and the CMS 2005 Annual Report for ESRD
Clinical Performance Measures Project, the percentage of patients with
hemoglobin < 11 g/dL has decreased from 84% in 1991 to 17% in 2004, a
remarkable achievement by the nephrology community for patients. In the
past, efforts to modify policy to control utilization of
EPOGEN' at the upper values of the hemoglobin range have
actually increased the number of patients with hemoglobins below 11 g/
dL. As a result, extreme caution must be exercised when considering any
policy changes that may affect this precarious population.
Hemoglobin levels fluctuate and must be measured repeatedly over time.
When targeting hemoglobin in the 10 to 12 g/dL range, hemoglobin
values will fluctuate. Because of the constantly changing environment
of a dialysis patient's body, the same individual will be more or less
anemic and will sometimes react more strongly to EPOGEN' and
sometimes less, thus having excursions above and below the target
hemoglobin range. For example, infections are known to lower hemoglobin
and erythropoietin responsiveness, while iron supplementation will help
increase both. Thus, the physician must monitor hemoglobin and adjust
the EPOGEN' dose as needed with the goal of keeping the
patient's hemoglobin level in the target range for as much time as
possible. These temporary hemoglobin fluctuations are universally
understood by practicing nephrologists, and have been described in
multiple publications using USRDS and other data.\17,18\
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\17\ Ebben JP, Gilbertson DT, Roberts TL, Foley RN, Collins AJ.
Hemoglobin Level Variability: Associations with Comorbidity,
Intercurrent Events, and Hospitalizations. Clin J Am Soc Nephrol 2006
1(6):1205-1210.
\18\ Lacson E Jr, Ofsthun N, Lazarus JM. Effect of variability in
anemia management on hemoglobin outcomes in ESRD. Am J Kidney Dis. 2003
Jan;41(1):111-24.
---------------------------------------------------------------------------
The USRDS data cited in the Committee hearing notice states that 40% of
dialysis patients have hemoglobins over the FDA approved label
of 12 g/dL. However, this USRDS estimate is at a single point,
or ``snapshot'', in time, which does not give an accurate
picture of anemia management.
Dr. Collins, who leads the analysis of USRDS data, describes the
percentage of patients at a single point in time that have a hemoglobin
level above 12 g/dL or above 13 g/dL. The recently reported figures
appear to have raised concerns that physicians may be targeting higher
hemoglobin levels than those recommended by the FDA or established
guidelines. However, because of the routine and expected fluctuations
in hemoglobin levels, it can be extremely misleading to draw any
conclusions from a single hemoglobin measure without considering how
physicians respond to the hemoglobin level.
Dr. Collins and colleagues state ``. . . hemoglobin levels in
almost 90% of patients seem to be in flux across the K/DOQI target
boundaries such that a cross-sectional assessment of anemia management
cannot give an accurate picture of anemia treatment.''
In 2005, Dr. Collins published a paper demonstrating that in the
vast majority of cases, providers responded promptly to temporary
elevations in the hemoglobin level by appropriately adjusting the dose
and bringing patients back into the target range.\19\
---------------------------------------------------------------------------
\19\ Collins AJ, Brenner RM, Ofman JJ, Chi EM, Stuccio-White N,
Krishnan M, Solid C, Ofsthun NJ, Lazarus JM. Epoetin alfa use in
patients with ESRD: an analysis of recent US prescribing patterns and
hemoglobin outcomes. Am J Kidney Dis. 2005 Sep;46(3):481-8.
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III. existing scientific evidence does not provide justification for
congress to introduce new legislation
Several recent studies have raised concerns regarding the benefit-
risk profile of EPOGEN'.
Recently published studies do not provide the definitive information
needed to guide policy decisions.
The first paper, by Zhang and Cotter et al, is an article that
analyzes medical claims data generated by Medicare in an attempt to
link greater EPOGEN' use with higher hematocrit levels and
increased mortality.\20\ Zhang and Cotter conclude that high
EPOGEN' doses are associated with an increased risk of
death. This analysis suffers from a distortion commonly referred to as
``confounding by indication'' bias. Confounding occurs when another
variable (the confounder) other than that being studied affects the
outcome and leads to a false conclusion. For example: Analysis of
Medicare data has shown that people who visit the doctor more often are
significantly more likely to die. Therefore, it could be concluded that
visiting doctors causes people to die! Of course, this is not the case.
In reality, sicker patients see the doctor more frequently and sicker
patients are more likely to die than those who are less ill. The same
is true for Epoetin utilization. Sicker patients typically require more
Epoetin to achieve the desired hemoglobin response, and sicker patents
are also more likely to die.
---------------------------------------------------------------------------
\20\ Zhang Y, Thamer M, Stefanik K, Kaufman J, Cotter DJ. Epoetin
requirements predict mortality in hemodialysis patients. Am J Kidney
Dis. 2004 Nov;44(5):866-76.
---------------------------------------------------------------------------
Mr. Cotter and colleagues acknowledge this important limitation in
their paper (p. 874): ``this study has 2 noteworthy limitations. First
and most important, when interpreting patient outcomes associated with
prescribed epoetin dose, treatment-by-indication bias may exaggerate
hazards and obscure benefits.''
A more recent analysis, presented at the American Society for
Nephrology, replicated the Cotter analysis but introduced more
comprehensive adjustments for `confounding' using appropriate methods.
This study found no association between mortality risk and
EPOGEN' dose.\21\
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\21\ Wang et al. Exploring Relative Mortality and Epoetin Alfa
(EPO) Dose Among Hemodialysis Patients. Poster presented at the 36th
Annual American Society of Nephrology Meeting, San Diego CA November
2006.
---------------------------------------------------------------------------
The New England Journal of Medicine (NEJM) recently released the
results of two studies, CHOIR (sponsored by Johnson and Johnson and
CREATE (sponsored by Roche).\22,23\ These studies examined the effects
of more aggressive anemia treatment, not consistent with the FDA
approved prescribing instructions, in kidney disease patients who were
not receiving dialysis.
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\22\ Drueke TB, Locatelli F, Clyne N, Eckardt KU, Macdougall IC,
Tsakiris D, Burger HU, Scherhag A; CREATE Investigators. Normalization
of hemoglobin level in patients with chronic kidney disease and anemia.
N Engl J Med. 2006 Nov 16;355(20):2071-84.
\23\ Singh AK, Szczech L, Tang KL, Barnhart H, Sapp S, Wolfson M,
Reddan D; CHOIR Investigators. Correction of anemia with epoetin alfa
in chronic kidney disease. N Engl J Med. 2006 Nov 16;355(20):2085-98.
---------------------------------------------------------------------------
The CHOIR study treated anemic patients with chronic kidney disease
with Procritr (Epoetin alfa), targeting a hemoglobin level of 13.5 g/dL
versus a control group targeting a hemoglobin of 11.3 g/dl. In the
CHOIR study, adverse events, including deaths, were greater in the
group with the targeted hemoglobin of 13.5 g/dL.
Amgen takes the recent results of these trials very seriously.
However, there are some limitations in the CHOIR study which make
drawing definitive conclusions challenging. The gold standard in
clinical research is the randomized, double-blind, placebo controlled
trial, and this was an ``open label'' study where both clinicians and
patients knew they were receiving Procritr, and the dose of the drug.
Importantly, there was an unusually high drop-out rate in this trial
(38% of patients), raising concerns about the ability to draw
conclusions from the data presented. Most importantly, this study was
not conducted in patients undergoing hemodialysis.
In a recently published article, the principle investigator for
CHOIR, Dr Ajay Singh stated: ``It is important to note that, unlike the
Normal Hematocrit or the Canada-Europe studies, both CHOIR and CREATE
evaluated pre-dialysis patients and so the results may not be
generalizable to the dialysis community.'' \24\
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\24\Singh AK. The target hemoglobin in patients with chronic kidney
disease. Nephrology News and Issues. 2006 Dec 20(13): p 29-30.
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Importantly, this was an experiment conducted to test practices
that are not consistent with the FDA label or the way that Amgen
promotes the use of EPOGEN'. The results of this study
reaffirm Amgen's commitment to using the FDA-approved prescribing
instructions to inform clinicians about how to use EPOGEN'.
In U.S. clinical practice today, there is little evidence that
clinicians purposefully maintain hemoglobin levels above the FDA-
approved target range. Thus, the risks associated with persistently
elevated hemoglobin levels seen in the CHOIR study and other
experiments should not be applied to the transient elevations in
hemoglobin levels described in dialysis patients treated under real
world conditions.
Amgen promotes EPOGEN according to the FDA label, and is
proactively educating the clinical community about a recent FDA
advisory for erythropoietic therapies.
In response to the CHOIR study, an FDA advisory was issued. Amgen
is actively working with the FDA (in cooperation with Johnson and
Johnson) to update the EPOGEN' product label with
information about the CHOIR results and to inform prescribers. Amgen is
proactively sending copies of the FDA advisory to all nephrologists,
and our sales team is hand-carrying the advisory into physicians'
offices. Amgen continues to recommend, as we always have, that
physicians use our products in accordance with their FDA-approved
labels. Although these studies in non-dialysis patients should not
readily be generalized to patients receiving hemodialysis, we believe
it is important for Amgen to help educate the clinical community about
new scientific information, even if it is not definitive.
CMS coverage policy for EPOGEN, as well as its claims
monitoring policy for EPOGEN, are consistent with
the FDA approved prescribing instructions.
In the announcement for this hearing, concerns about patient safety
and Medicare spending on anemia treatments in ESRD were raised.
Further, the November 15, 2006 letter to CMS indicates a belief that
current CMS policies for EPOGEN' establish ``reimbursement
incentives for providers to increase hemoglobin doses'' and that these
CMS policies are in conflict with FDA labeling.
Below, Amgen would like to address apparent confusion of the CMS
coverage policy for EPOGEN' and a claims monitoring policy
CMS employs to ensure it is paying for appropriate use. Since its
inception, the CMS coverage policy for EPOGEN' has been
consistent with the FDA approved label. The claims monitoring policy
(also known as Erythropoietin Monitoring Policy or EMP) explicitly
refers to the coverage policy for EPOGEN' as well as the FDA
label in the manual instructions.
``. . . While Medicare is not changing its coverage policy on
erythropoietin use to maintain a target hematocrit level between 30%
and 36%, we believe the variability in response to EPO warrants
postponing requiring monitoring until the hematocrit reaches higher
levels. For dates of services April 1, 2006 and later, CMS will not
require contractors to initiate monitoring until the hematocrit level
reaches 39.0 (or hemoglobin of 13.0). This does not preclude the
contractors from performing medical review at lower levels. The Food
and Drug Administration labeling for EPO notes that as the hematocrit
approaches a reading of 36, the dose of the drug should be reduced by
25%. . . . Providers are reminded that CMS expects that as hematocrit
approaches 36% (hemoglobin 12 g/dL), a dosage reduction occurs.''
Medicare Claims Processing Manual (CMS Pub. 100-04), ch. 8, 60.4
The CMS Erythropoietin Monitoring Policy (EMP) has evolved based on
extensive scientific deliberation. The current policy
represents an important improvement, focusing on how physicians
manage anemia, by monitoring whether physicians appropriately
adjust the EPOGEN dose in response to elevated
hemoglobin levels.
The EMP has evolved over time to account for the expected temporary
fluctuations of hematocrit levels in different patients. When
physicians target hematocrit levels of 30-36% (consistent with the FDA
approved label hemoglobin target 10-12 g/dL\25\), the majority of those
patients--even on a stable dose of EPOGEN'--will experience
temporary elevations above 12 g/dL, as discussed earlier.
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\25\ Hematocrit is a percentage of packed red blood cells in a
given volume of blood, and is approximately equal to 3X hemoglobin.
Thus, a hematocrit of 36 corresponds to a hemoglobin of 12 g/dL.
---------------------------------------------------------------------------
To account for these frequent temporary elevations, CMS has long
recognized the need for a monitoring threshold above 36%. In 1997, CMS
instituted monitoring for a 90-day rolling average hematocrit in excess
of 36.5% (in effect, hemoglobins that were persistently above 12 g/dL).
CMS revised this policy in 1998, implementing a 90-day rolling average
where monitoring would occur when the hematocrit exceeded 37.5%
(hemoglobin of 12.5 g/dL).
In April of 2006, after several years of extensive deliberation and
consultation with clinical experts, CMS implemented a revised
monitoring policy. Under the 2006 EMP, providers are still responsible
for achieving the target hemoglobin in the FDA label range, and CMS
expects that providers will follow the package insert and reduce the
dose as hemoglobin levels approach 12 g/dL. If providers fail to reduce
the dose of EPOGEN' when the hemoglobin exceeds 13 g/dL they
are subject to a payment reduction.
Comments by former CMS administrator Dr. Mark McClellan, and by the
CMS Chief Medical Officer Dr. Barry Straube, reiterate the importance
of developing an appropriate monitoring policy that does not have a
negative impact on the health outcomes of ESRD patients.
``Our Agency has worked to review the literature and consult with
experts in the field to develop a means of monitoring erythropoietin
usage without the risk of a negative impact on the healthcare outcomes
of this vulnerable population. We are pleased to have found a
reasonable means for monitoring erythropoietin dosages that are in line
with the FDA-approved labeling for these drugs.'' CMS Administrator
Mark McClellan, November 2005
``While we have to be concerned with patients who have hemoglobin
[HGB] over 13 . . . Everybody has found that when you treat a group of
patients, whatever the hemoglobin target is, if you lower that upper
target range, you shift the hemoglobin levels. If the doctor tries to
control everybody below 12, you will have other patients on the other
end of the bell curve, below 11. And there are multiple studies, done
in 1997 and 1998, that were associated with higher mortality rates,
higher hospital admission rates and much higher complications--
cardiovascular complications [when the levels dropped below 11].'' CMS
Chief Medical Officer Dr. Barry Straube, Inside CMS, November 28, 2006
Preliminary data indicate that under the new EMP, EPOGEN
doses and hemoglobin levels have slightly decreased.
Since the EMP went into effect April 1, 2006, Amgen analysis shows
the average dose of EPOGEN' used in dialysis has decreased 2
percent. The average hematocrit has also decreased slightly in the few
months since the new policy implementation, suggesting that the policy
is having its intended effect of enforcing appropriate utilization.
CMS's Dr. Barry Straube states that CMS claims data, as well as data
from dialysis clinic Fresenius, demonstrate that hemoglobin levels have
fallen since the implementation of the EMP. The impact of the EMP needs
to be assessed over a longer period of time, but again, short-term
analyses suggest that the policy is enforcing appropriate utilization.
IV. Medicare Expenditures for EPOGEN'
The Committee raised concerns about Medicare spending on
EPOGEN' in the announcement for this hearing.
Increased Medicare spending on EPOGEN reflects growth in
the ESRD population, and substantial improvements in the
quality of care.
Growth in Medicare spending on EPOGEN' results from
several factors. One factor is growth in the ESRD patient population,
approximately 3% per year. A major factor has been the tremendous
increase in effective anemia management, reflected by improved
achievement of the CMS clinical performance measure, reducing the
percentage of patients with hemoglobin below 11 g/dL. As previously
stated, this percentage has decreased from 84% in 1991 to only 17% in
2004.\26\ Finally, the patient profile has changed over time. Today,
more ESRD patients have other serious conditions which impact anemia
and anemia treatment. For example, from 1995 to 2004 diabetes as the
primary cause of renal failure has increased from 36% to 46%, and the
number of patients with cancer has gone up 30%.1 As expected, this
improvement in hemoglobin outcomes is correlated with increased
EPOGEN' utilization.
---------------------------------------------------------------------------
\26\ USRDS 2006 Annual Data Report, CMS' 2005 Annual Report ESRD
Clinical Performance Measures Project
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Importantly, increasing Medicare expenditures on EPOGEN'
are not the result of higher prices. In fact, CMS per unit expenditures
for EPOGEN' have decreased 14% in recent years, from the
statutory rate of $11 per 1,000 units that was in effect in the early
1990's to the current market-based ASP + 6% reimbursement of $9.446 per
1,000 units in Q4 2006.
The hearing announcement contained estimates of increased costs
associated with the purported overuse of EPOGEN.
These estimates are incorrect and based on a flawed study and
should not be used as the basis for policy-making. Policy based
on the model in this study could seriously jeopardize the
quality of care for ESRD patients.
The hearing announcement noted ``a recent study from November 2006
in Dialysis and Transplantation\27\ that found that the population with
a red blood cell count above industry guidelines also has higher drug
costs, specifically, $3,100 per patient per year more just on the
anemia drug.''
---------------------------------------------------------------------------
\27\ Pizzi LT, Patel NM, Maio VM, Goldfarb DS, Michael B, Fuhr JP,
Goldfarb NI. Economic implications of non-adherence to treatment
recommendations for hemodialysis patients with anemia. Dialysis and
Transplantation. 2006 Nov; 35(11):654-732.
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The economic model (the Pizzi model) used in the cited study, while
published, is seriously flawed. Specifically, the results depend on a
flawed assumption that individuals should start and remain on 43,500
units of EPOGEN' a month without dose adjustments in
response to hemoglobin levels. This assumption does not reflect K/
DOQITTM guidelines and is not consistent with the FDA
approved prescribing instructions for EPOGEN' nor with
published USRDS dosing patterns. Both the 2000 K/DOQITTM
guidelines and FDA label provide a starting range of EPOGEN'
that doctors should use the first time a patient is given
EPOGEN', then instructs doctors to adjust the dose of
EPOGEN' until hemoglobin is in the target range of 11-12 g/
dL. In most cases, this results in a higher maintenance dose than the
actual starting dose. The last time the average U.S. EPOGEN'
dose approached the level assumed in the Pizzi model was 1992-1993. At
that time, 84% of patients were below the recommended hemoglobin level
of 11 g/dL.
Adopting the dosing patterns portrayed in the Pizzi model could
seriously jeopardize patient quality of care and significantly increase
the percentage of patients with hemoglobin below 11 g/dL, increasing
the risk of hospitalization and death and raising overall healthcare
costs. The $3,100 per patient per year savings are not real, and
unlikely to be realized given what is known about the current ESRD
population. The Dialysis and Transplantation study should not form the
basis of any policy decision, as it relies on faulty assumptions, makes
projections based on off-label use of the product and is not consistent
with the best available evidence.
When rigorously analyzed, the available data show that there is no
systemic abuse of EPOGEN.
In the November 15, 2006 letter to CMS, Chairman Thomas and Ranking
Member Stark raised concerns about ``systemic abuse'' of
EPOGEN'. Analysis of cross-sectional or ``snapshot'' in time
analyses may give this appearance, but upon careful review of these and
other data, EPOGEN' use appears appropriate and there is no
evidence of systemic overuse.
Amgen has analyzed data from provider datasets that report
hemoglobin values and EPOGEN' dosing on a per treatment
basis, allowing for very granular analyses, even more granular than
USRDS data that collect monthly information. A recent analysis of
approximately 300,000 patients demonstrated that most physicians are
using EPOGEN' in a manner that is safe and consistent with
its FDA label. In fact, among patients who have ever recorded an
elevation of the hemoglobin over 12 g/dL, over 50% of those excursions
over 12 g/dL are managed back into the recommended target range within
one month, and over 85% of those excursions within three months. These
data support previous analyses19 which have demonstrated that patient
hemoglobin levels are being appropriately managed when assessed over
time using appropriate methods.
Moreover, the new EMP is fully aligned with the FDA label and will
reinforce appropriate utilization of EPOGEN' by financially
penalizing providers attempting to maintain patients at higher
hemoglobin levels. The EMP notes that CMS expects that providers will
follow the EPOGEN' label and reduce the dose as hemoglobin
levels approach 12 g/dL and then requires an EPOGEN' dose
reduction when a hemoglobin level exceeds 13 g/dL. If providers fail to
reduce the dose of EPOGEN', they are subject to a payment
reduction.
Congress should not implement a payment system bundling dialysis
services with separately billable injectable drugs (referred to
as ``bundled'' payment) until the MMA mandated demonstration
project is completed.
Amgen does not believe that Congress should consider moving to a
single bundled payment for drugs and dialysis services in dialysis
until the MMA mandated three-year CMS demonstration project is
completed. The goal of the CMS project is to determine how best to
include separately billable drugs in the dialysis composite payment.
A bundled payment system could be dangerous for patients, and end
up costing the federal government more money. This is true for several
reasons. First, unless bundling is accompanied by a robust
scientifically valid risk adjustment system and an agreed-upon set of
quality safeguards, it may result in perverse incentives to undertreat
patients. Moreover, as evidenced by the broad and deep opposition of
patient groups and medical providers, there are serious risks to rural
patients and those in dialysis centers in underserved urban areas.
There are two critical elements necessary for the dialysis
composite rate to be successful, and to assure that this vulnerable
patient population is not harmed. The first is a robust and valid case-
mix adjustment method--designing a system than can accurately predict
which patients are most costly, and then adequately reimburse for those
patients (a major goal of the CMS demonstration project). The second is
a set of robust quality measures to safeguard patients against under
treatment that may result from financial incentives that may limit
their access to vitally necessary medical care. Congress recognized
these requirements, and mandated the conduct of a demonstration project
before implementing a bundled dialysis composite rate.
ESRD patients represent a seriously vulnerable patient group, at
high risk of death. Even among ESRD patients, there are some who are
more gravely ill and require significantly greater health care
intervention. Unless Medicare appropriately reimburses for these
patients, even one or two such patients in a single dialysis center can
literally ``tip the scales'' and cause a dialysis center to lose money,
and even risk closure. Many believe that the risk is highest for the
small dialysis organizations that serve poor patients in rural areas.
Several models and real world examples have demonstrated this
challenge and also the significant risk that a poorly designed system
of bundled payment could have negative consequences for patient care.
In 1989 Medicare paid for EPOGEN' at a rate of
$40 for up to 10,000 units, a case rate. When CMS recognized that under
this policy EPOGEN' doses were about half of what was
needed, the policy was subsequently changed to pay per 1,000 units
administered rather than at the case rate. This is an example of how
fixed payments can result in undertreatment.
A Medicare managed care capitation demonstration for ESRD
resulted in higher costs than the fee for service comparison group. The
additional costs to the federal government total approximately $18.5
million across the three years of the demonstration.\28\
---------------------------------------------------------------------------
\28\ Summary report can be accessed at www.cms.hhs.gov/
DemoProjectsEvalRpts/downloads/ESRD_Managed_Care_Summary.pdf
An appropriate case-mix adjustment methodology has also been
difficult to develop. A 1994 study by RAND and UCLA developed a method
that was not shown to be adequate for dialysis patients.\29\ A 2000
report released by the American Society of Nephrology and the Renal
Physicians Association also attempted to create a case mix adjustment
system, but was also found to lack the needed predictive power.\30\
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\29\ Farley DO, Kallich JD, Carter GM, et al. Designing a
capitation payment plan for Medicare end stage renal disease services.
Santa Monica (CA), RAND. 1994.
\30\ Bouchery EE, Gaylin DS, Rubin RJ, M.D., Shapiro JR, Held PJ.
Lewin Group: Capitation Models for ESRD: Methodologies and Results.
Prepared for Renal Physicians Association and the American Society of
Nephrology. 2000.
---------------------------------------------------------------------------
``Capitation contracts put physicians at financial risk.accepting
global capitation for a small group of patients may entail significant
risk on the part of the capitated physician or health plan. It would be
necessary to spread risk over many patients in order to reduce the
financial risk faced by the physician or health plan to an acceptable
level.''
In summary, eliminating separate payment for dialysis drugs, if not
implemented thoughtfully, could lead to unintended consequences
including:
Poorer quality of care, as dialysis units may need to
make compromises to offset lower overall reimbursement.
Higher overall Medicare costs as a result of poor quality
dialysis care.
Threats to access for poor and rural patients treated in
small dialysis facilities. Small rural clinics may begin to avoid
sicker/costlier patients in order to control costs, or close as a
result of financial burden.
In conclusion, we want to thank the Committee for this opportunity
to submit written testimony. We are proud of EPOGEN''s long
history of safely and effectively treating anemia in ESRD and stand
with nurses, physicians, and other healthcare providers in supporting
the best possible care for highly vulnerable kidney disease patients.
Given the current state of evidence there does not appear to be
justification for the introduction of new legislation. We remain
concerned that legislation based on an insufficient analysis of
scientific data could lead to negative outcomes for patients and for
health care in the United States.
Statement of Richard Carrancejie, Birmingham, Alabama
Please, read the Birmingham news article on 11/18/06 on dialysis.
we as patients are threatened everyday. the conditions are awful. we
have roaches, untrained staff, the staff curse the elderly and black
patients. our Medicare money doesn't go for or medical care. we are
threatened by management and doctors with transfers or being taken off
dialysis during treatment. most of the patients are afraid to speak out
because they may be harmed. local officials do nothing because it
involves a major employer [U.A.B.]. even the president of the college
does not care. U.A.B. had to pay back millions last year to Medicare,
they also lost the transplant records of 10,000patient.tyis is a
nightmare. our state health dept. has not inspected since 1998. the
positive room [blood disease] has been used by regular patients. the
positive machines have been used all over the facility and on holidays.
the Katrina victims have used our facilities, we do not know their
fate, yet. it is a tragedy, a medical nightmare. we need help and
protection from congress and the justice department. help us. this
tragedy has gone on to long. individuals and company must be punished
and serve jail time for the death of so many patients. I have been on
dialysis for three years, I have witnessed all these events. i have
contacted the chairman of DaVita and the president of U.A.B., no reply.
stop the Medicare money, have the FBI. and Medicare investigate,
prosecute and make the companies and hospital repay the tax payers and
put the worst law breakers away for along time.
Statement of DaVita Patient Citizens
Introduction
As America's largest dialysis patient organization, we are proud to
represent over 20,000 pre-dialysis and dialysis patients and their
families. On a wide variety of issues, we seek to ensure that the
patients' point of view is heard and considered by policy makers so
that continued progress may be made in the quality of care and life for
patients with kidney disease. We appreciate this opportunity to submit
testimony to the House Ways and Means Committee's Hearing on Patient
Safety and Quality Issues in End Stage Renal Disease.
Quality of Life
Anemia is a serious, life-threatening problem affecting almost all
dialysis patients. It causes fatigue, weakness and increased risk of
hospitalization and death. In most cases, the administration of
synthetic replacements for the hormone erythropoietin can manage our
anemia and restore our energy. With appropriate anemia management, we
require less medical attention and hospitalization, and we are better
able to lead productive, quality lives.
The Experts on Quality of Care
Every dialysis patient's anemia situation is different. The
decision of how to manage our anemia should therefore be made by our
physicians in consultation with ourselves. Typical physician
prescribing practices allow for physicians to use package inserts as a
guideline for their prescriptions. Many of us enjoy higher quality
lives because our doctors prescribe the appropriate amount of
erythropoietin for each of us individually. This allows each of us to
participate in the activities of daily living.
Research Applicable to ESRD Patients is Needed
Of course, it is critical that we, as well as our physicians, be
informed of any increased risks associated with anemia management. The
studies cited in recent news articles focused on Chronic Kidney Disease
(CKD) patients in stages 1-4, who are not on dialysis. The studies did
not focus on patients with End Stage Renal Disease (stage 5)--patients
like us, who are on dialysis. We therefore look forward to clinical
studies of anemia management in the ESRD population to determine the
appropriate approach to anemia management for dialysis patients.
Community Cooperation Improves Quality of Care
Recognizing the importance of appropriate anemia management, we
joined with the kidney care community in asking CMS to revise the April
2006 monitoring policy on anemia management to better align with
physician prescribing methods and to take into consideration the
patient's quality of life. We believe that this revised monitoring
policy is a vast improvement over the April 2006 policy.
Conclusion
DaVita Patient Citizens greatly appreciates this opportunity to
comment on ESRD patient safety and quality issues. We ask that, before
proposing further changes to the CMS monitoring policy on anemia
management, you take into consideration all of the data and the
population to which it applies, as well as, the patients' perspective.
December 20, 2006
Dear Mr. Chairman and Members of the Committee,
The American Society of Nephrology (ASN) appreciates the
opportunity to provide a written statement for the record regarding the
issue of anemia management. We commend the Committee for its efforts to
learn more about anemia management for individual's with kidney disease
and kidney failure.
Through the Medicare program, the federal government has assumed
primary responsibility for dialysis patients. The landmark 1972
(Medicare) legislation ensures that dialysis care is provided to this
most vulnerable population. We continue to support innovative policy
initiatives that reward improvements in care and the attainment of
quality benchmarks based on scientific findings. Our Society and its
members are dedicated to providing the highest standard of care and
ensuring patient safety.
The ASN is a professional association with approximately 10,000
members. Of this membership, about 95% are physicians, with the
remaining members basic scientists with a primary interest in renal
disease. Virtually every licensed nephrologist in the United States is
a member of the ASN, with an additional 3,000 nephrologists from 82
other countries comprising the remainder of our membership. The Society
is focused on promulgating innovative research related to renal
disease, and on providing continuing medical education to physicians
and scientists dedicated to the improved understanding and treatment of
renal disease.
In light of the recent studies published in the New England Journal
of Medicine, ASN agrees that it is essential for the kidney care
community to continue examining all available scientific data to ensure
that public policies reflect appropriate anemia management for dialysis
patients. We also reaffirm our ongoing commitment to work with the
Congress and CMS to ensure that Medicare policy reflects the best
science and ensures the welfare of patients, the public interest, and
Medicare's stewardship of patients with kidney disease.
Anemia is a complication of kidney failure, also known as End-Stage
Renal Disease (ESRD) and is a consequence of kidney disease in patients
receiving dialysis. Patients with kidney failure suffer from anemia
because their kidneys do not produce a hormone (erythropoietin) that
regulates red blood cell production. Anemia has a profound
physiological effect on every organ system (including the brain) and
directly affects patients' quality of life. Anemic kidney disease
patients have more difficulty performing every day activities,
including maintaining employment. Physicians determine a patients'
degree of anemia with simple blood tests, measuring the hemoglobin or
hematocrit levels. A healthy man has a hemoglobin level of 15 (roughly
a hematocrit level of45 percent), with slightly lower values in healthy
women. Before effective treatment was available, a dialysis patient
would typically have severe anemia with a hemoglobin level lower than
11 (hematocrit level of lower than 33 percent). Prior to the
introduction of recombinant human erythropoietin (EPO) as a therapeutic
agent in 1989, anemia management in dialysis patients was dependent on
transfusions and other approaches which were largely ineffective.
ASN recognizes that the optimal target hemoglobin/hematocrit level
for patients with kidney failure may not be straightforward. During the
past 10 years, several observational studies have suggested that higher
hemoglobin levels are associated with a lower risk of hospitalization
and death, and higher levels of cognitive function.
However, recently two clinical trials published in the New England
Journal of Medicine (NEJM) question whether higher hemoglobin targets
are optimal for patients with kidney failure and have fostered a great
deal of discussion within the scientific community. The CHOIR study
indicated an association in kidney disease patients not yet on dialysis
(patients diagnosed with Stages III and IV kidney disease) between
higher hemoglobin levels and an increased risk for cardiovascular
morbidity and death. The CREATE study (which was also published in the
same November issue of the New England Journal of Medicine), in a
similar group of patients with chronic kidney disease not on dialysis,
found no significant difference in the combined incidence of severe
adverse events between the higher and lower hemoglobin groups, although
hypertensive episodes and headaches were more frequent in the former
group.
A key component to any critical review of the scientific research
data examining utilization of erythropoietin is patient variability in
clinical response. Research has indicated that patient comorbidities,
intercurrent events including hospitalization, and practice patterns
contribute to this variability, which is not unique to patients with
kidney disease. One recent study concluded that the variability in the
response of hemoglobin levels to etythropoetin treatment over time in
individual patients may account for moving 28 percent of all dialysis
patients above and below the target hemoglobin levels during a one-year
timeframe. Other studies support this finding as well. Because of this
variability in patient physiology, optimal anemia management often
requires a highly individualized approach to treatment.
ASN urges that Congress and CMS should take all available studies
into account when setting Medicare policy. For example, the recent CMS
EPO Monitoring Policy issued before the publication of the CHOIR and
CREATE studies recognizes the need for reimbursement policy to take
into account patient variability. When reviewing this policy, it is
important to understand that it is not a treatment guideline. Rather,
it is an auditing tool. Under the policy, if a patients' hemoglobin
reaches 13 and the dose is not reduced, then CMS will reduce the
payment 25 percent. It does not call for, nor recommend, that patients'
hemoglobin levels are maintained above 12 in accordance with the Food &
Drug Administration label.
Congress should examine all of the available scientific literature
before advising any policy changes. The recent trials should be
reviewed along with those that are already a part of the literature, as
well as the Food and Drug Administration package insert, to determine
the optimal policies to be based on safety and efficacy. Policy should
not be based upon the result of a single clinical trial. ASN is
committed to working with the Congress and CMS to ensure that Medicare
policy reflects the consensus of the scientific community.
ASN appreciates the opportunity to provide the Committee this
statement for the record. ASN cannot emphasis enough that this debate
on anemia management is about patient safety and quality of life,
quality care and policy based and grounded in scientific findings. It
is imperative that the scientific community and government work
together to promote the highest quality of patient care. We look
forward to working with the Committee as we continue to evaluate
clinical data.
Sincerely,
, M.D.
President
Central New Hampshire Kidney Center
Laconia, New Hampshire 03246
December 19, 2006
Chairman and Members of the Committee on Ways and Means
I am a Nephrologist taking care of patients with End Stage Renal
Disease for the last twenty years. I am the owner and medical director
of Central New Hampshire Kidney Center (CNHKC) serving Laconia and the
Lakes Region area of New Hampshire since 1990. As a physician, owner
and administrator I had to make all the decisions that maintain my
patient's safety, health and well being in addition to maintaining the
financial viability of my dialysis unit. My choice was clear; patients
and their needs always come first and have priority to any financial
interest. Whenever a patient needed a medication he gets it because he
needs it not because I will make few dollars off my patient. That was
reflected in my use of EPO and how I was able to use 30--40% less drug
than the national average. The following graph reflects the average
weekly dose of EPO units used at CNHKC compared to the USA National
average as reported by the USRDS.
[GRAPHIC] [TIFF OMITTED] 35773A.011
In 1998 following the early results of the VA study``Subcutaneous
Compared with Intravenous Epoetin in Patients Receiving Hemodialysis''
which was then published by the New England Journal of Medicine, August
27, 1998 issue. http://content .nejm.org/cgi/content/abstract/339/9/
578?ijkey=08d84b8e606283b32c14b90c4284b3e
ba947fa58&keytype2=tf_ipsecsha
I switched the method of administration of EPO from intravenous to
subcutaneous and I noticed that my usage has dropped by more than 20%.
Since then I continued to use this method of administration. Since 1998
I was using an average of 6000 units per week lower than the national
average. This is equivalent to $3120.00 savings per patient per year
for the Medicare and tax payers. By doing this I lost income that I
could have generated from billing Medicare additional on average of
$150,000.00 per year for the last 8 years. Also I lost rebates from
Amgen because I did not achieve their required volume increase!!!!
As a physician and administrator I would like to share with you my
experience and few important points that need to be addressed in this
forum:
1. Method of administration of EPO:
It is a puzzle for me that the subcutaneous method is not the
standard method up till now. It is the standard method throughout
Europe, Canada and the Veterans Administration. By the year 2002 about
70% of VA patients were switched to subcutaneous administration
according to a VA press release http://www1.va.gov/resdev/news/
press_releases/epoetin-022802.cfm
It is clear in the literature that approximately 20 to 30 percent
of patients who receive EPO intravenously for the anemia of chronic
renal failure may develop an elevation in diastolic pressure of 10 mmHg
or more. In comparison, the blood pressure is less likely to rise after
subcutaneous administration.
Also it is clear that the main advantage of subcutaneous EPO is its
longer half-life: 24 hours versus four to nine hours when given
intravenously.
So just by changing the method of administration this drug becomes
safer and more effective and as an added bonus we will save the tax
payers a lot of wasted money.
Why for all those years this is not the standard of care?? What's
going on??
Why Amgen is not promoting a safer efficient way for using its
drug??
Since August 27, 1998 an Amgen sponsored study was published in the
New England Journal of Medicine ``The Effects of Normal as Compared
with Low Hematocrit Values in Patients with Cardiac Disease Who
AreReceiving Hemodialysis and Epoetin'' http://content.nejm.org/cgi/
content/short/339/9/584, we learned from this study that higher
hematocrit can kill dialysis patients. Yes, since 1998 not just in 2006
we learned that higher hematocrit can be dangerous to our patients. Did
this prompt Amgen to ensure safety of its drug? On the contrary Amgen
and its consultants shoved the results of its own study under the rug
and promoted achieving higher hematocrit!!
Why a chronic kidney disease patient receiving the EPO
subcutaneously will get switched to intravenous once he starts on
dialysis? Is this better for the patient or better for the bottom line?
All of a sudden ``it hurts''!! Yes, the bottom line!!
Those issues needs to be addressed and investigated as for the last
eight years tax payers paid at least 20% more for an expensive drug and
patients where unnecessarily subjected to potential worsening of their
blood pressure, which is the major cause of morbidity and mortality in
those patients, and probably was a contributing factor to the early
death for some of those patients.
2. Rebates:
This is a total disgrace to the practice of medicine. It is
shameful to allow rebates for achieving larger volume for the use of a
drug. It is shameful that the physician is forced to increase the dose
of EPO for a patient who has hemoglobin of 10.8 or 10.9 so the center
can meet the rebate threshold yet he knows that it will not do the
patient any good. It is a disgrace that we submit patient's labs to a
drug manufacturer so we can get a rebate.
This should STOP. It should have never been allowed. This is a
shameful black spot on the practice of medicine.
3. Average Sale Price + 6%:
What a joke? It will never cut on the use of drugs; it will make
the fat cats fatter and it is not just killing patients it will also be
killing the small providers and the competition or what ever left of
it!! If the ASP of a drug is $1.00 this means that someone is paying
$0.90 and another is paying $1.10 so the large provider will make a
profit of about 18% and the small independent provider like me will
lose about 5%. I don't think this is fair and it will never achieve
what it is intended for. You have to level the field by using the
Actual Sale Price or compensate the small provider different than the
large provider. It is ridiculous that the small provider will end up
contributing to the bottom line of Amgen and the large providers!!!
That's why Fresenius made a deal with Amgen to be its sole provider of
anemia drugs for the next five years to guarantee a lower price. This
policy did nothing but gave Amgen the green light to keep increasing
its price and Amgen delivered!!
4. EPO PRICE and COMPOSITE RATE:
For each dialysis treatment I get paid an average of $130.00 while
Amgen gets paid an average of $70.00 considering the current national
average use per treatment. It is amazing that the price of this drug is
more than 50% of the composite rate and yet the government is willing
to pay??!!!. I have to provide 3-4 hours of nursing care, dietician,
social worker, maintain equipment, maintain building and grounds,
provide supplies, provide labs, insurance . . . etc. and yet on the
other hand Amgen can decide it's own price no matter how ridiculous it
is and increase it whenever it wants, no questions asked!!! The
government controls the price for health care, the hospitals, clinics
and physicians but when it comes to pharmaceuticals, they have a free
ride. With the current drugs payment system as a small provider I end
losing about 5% on EPO which means that I end up contributing to Amgen
almost $3 every dialysis treatment. I don't think this is fair, this is
nothing but ridiculous. It may be appropriate to let the free market
work between the provider and the supplier but when the payer fixes the
price on the providers that messes the whole picture, this economical
concept is not an economy of capitalism not even economy of communism
this is nothing but an economy of terrorism for the small provider. It
is nothing but the pill of death for small providers. If the government
wants to maintain healthy competition between providers then it will
have to fix the price of EPO or force Amgen to sell it at a declared
fixed price which is the same to every provider with no rebates or
gimmicks. Amgen is a major partner in providing care for the ESRD
program, Amgen had a free ride for many years and at this stage it
needs to step up to its responsibilities and stop its practice of greed
and back door gimmicks.
I hope by sharing my concerns and frustrations as a small provider
you will have a better vision and understanding to what at stakes here
as our main goal is providing the best care to our patients in the most
efficient way and at the highest standards medically and ethically. I
will be glad to answer any question.
Sincerely
Noshi Ishak, M.D.
CEO, Medical Director
Statement of Kidney Care Partners
The undersigned members of Kidney Care Partners (KCP) appreciate
the opportunity to provide written testimony to the Committee regarding
the intersection of anemia management and Medicare policy. We commend
the Committee for its efforts to learn more about anemia management for
individuals with kidney disease and kidney failure. Through the
Medicare program, the federal government has assumed responsibility for
the health and safety of dialysis patients. Therefore, it is
appropriate that the Committee examine the optimum care patients should
receive, including issues related to drug utilization.
KCP is a coalition of patient advocates, dialysis providers,
physicians, nurses, and manufacturers. Our mission, individually and
collectively, is to ensure: (1) chronic kidney disease patients receive
safe and optimal care; (2) chronic kidney disease patients are able to
live quality lives; (3) dialysis care is readily accessible to all
those in need; and (4) research and development leads to enhanced
therapies and innovative products.
Our members are dedicated to providing the highest standard of care
and ensuring patient safety. The Centers for Medicare and Medicaid
Services (CMS), the Government Accountability Office (GAO), the
Medicare Payment Advisory Commission, and other organization have
recognized the improvement of quality by the kidney care community
during the last ten years. We continue to support innovative policy
initiatives that reward improvements in care and the attainment of
quality benchmarks. As part of our efforts, KCP launched the Kidney
Care Quality Alliance, which has developed a starter set of quality-
related measures that could be used to evaluate and reward high quality
care in the kidney care community.
In light of the recent studies published in the New England Journal
of Medicine, KCP agrees that it is essential for the kidney care
community to continue examining all available data to ensure that
public policies reflect appropriate anemia management for patients with
kidney disease and kidney failure. We are committed to working with
clinical researchers to determine the appropriate hemoglobin levels for
these patients. We also reaffirm our ongoing commitment to work with
the Congress and CMS to ensure that Medicare policy reflects the best
science and ensures the welfare of patients, the public interest, and
Medicare's stewardship of patients with kidney disease.
Anemia is a complication of kidney disease, which is known as
Chronic Kidney Disease (CKD) and kidney failure, also known as End
Stage Renal Disease (ESRD or Stage V kidney disease). Patients with
kidney failure suffer from anemia because their kidneys do not produce
a hormone (erythropoietin) that regulates red blood cell production.
Anemia has a profound physiological effect \1\ on every organ system
(including the brain) and directly affects patients' quality of
life.\2\ Anemic kidney disease patients have more difficulty performing
activities of daily living and maintaining employment. They experience
lower vitality and may suffer from depression.\3\ Doctors determine a
patients' degree of anemia with simple blood tests, measuring the
hemoglobin or hematocrit levels. A healthy man has a hemoglobin level
of 15 (roughly a hematocrit level of 45 percent), with slightly lower
values in healthy women. Before effective treatment was available, a
dialysis patient would typically have severe anemia with a hemoglobin
level lower than 11 (hematocrit level of lower than 33 percent).
---------------------------------------------------------------------------
\1\ Morrell Michael Avram, et al., ``Hemoglobin Predicts Long-Term
Survival in Dialysis Patients: A 15-Year Single-Center Longitudinal
Study and a Correlation Trend between Prealbumin and Hemoglobin'' 87
Kidney Internat'l (Supp.) S6-S11, S9 (2003).
\2\ Allen R. Nissenson & Lawrence T. Goodnough, ``Anemia: Not Just
an Innocent Bystander?'' 163 Arch. Intern. Med. 1400 (June 23, 2003).
\3\ Hans Furuland et. al., ``A Randomized Controlled Trial of
Haemoglobin Normalization with Epoetin Alfa in Pre-Dialysis Patients''
18 Nephrol. Dial. Transplant 353-61 (2003);
---------------------------------------------------------------------------
There is a large and extensive peer-reviewed volume of literature
discussing what the optimal target hemoglobin/hematocrit level for
patients with kidney failure should be. For example during the past ten
years, several observational studies have suggested that higher
hemoglobin levels reduce the risk of hospitalization and death, while
increasing cognitive function.\4\ As one of these studies suggests,
these outcomes could result in lower costs to the Medicare program.
Specifically, it found that Medicare patients with hematocrit values of
36 to less than 39 cost the program significantly less than those
patients with hematocrit values of less than 30.\5\ Other prospective
clinical trials have not observed benefits with higher hematocrit
levels.\6\ The FDA label recommends maintaining patients at a
hemoglobin level of 10-12.
---------------------------------------------------------------------------
\4\ S. Li & A.J. Collins, ``Association of Hematocrit Value with
Cardiovascular Morbidity and Mortality in Incident Hemodialysis
Patients'' 65 Kidney Int. 626-33 (2004); A.J. Collins, et al. ``Death,
Hospitalization, and Economic Associations among Incident Hemodialysis
Patients with Hematocrit Levels of 36 to 39%'' 12 J. Am. Soc. Nephrol.
2465-73 (2001); A.J. Collins, et al., ``Hematocrit Levels and
Associated Medicare Expenditures'' 36 [ 282-93 (2000); F. Locatelli, et
al., ``Anemia in Haemodialysis Patients of Five European countries:
Association with Morbidity and Mortality in the Dialysis Outcomes and
Practice Patterns Study (DOPPS) 19 Nephrol. Dial. Transplant 121-32
(2004); N. Ofsthun et al., ``The Effects of Higher Hemoglobin Levels on
Mortality and Hospitalization in Hemodialysis Patients'' 63 Kidney Int.
1908-14 (2003); E.G. Lowrie et al., ``Medical Outcomes Study Short
Form-36: A Consistent and Powerful Predictor of Morbidity and Mortality
in Dialysis Patients'' 41 Am. J. Kidney Dis. 610-9 (2003).
\5\ Allan J. Collins et al. ``Death, Hospitalization, and Economic
Associations among Incident Hemodialysis Patients with Hematocrit
Values of 36 to 39%'' 12 J. Am. Soc. Nephrol. 2465-73 (2001).
\6\ A. Besarab et. al., ``The effects of normal as compared with
low hematocrit values in patients with cardiac disease who are
receiving hemodialysis and epoetin'' 339 N Engl J Med. 584-90 (1998).
---------------------------------------------------------------------------
Two recent studies published in the New England Journal of
Medicine, which have engendered substantial controversy and
discussion,demonstrate the continuing debate within the scientific
community. The CHOIR study \7\ indicated an association in kidney
disease patients not yet on dialysis (patients diagnosed with Stages
III and IV kidney disease) between higher hemoglobin levels and an
increased risk for cardiovascular morbidity and death. The CREATE study
\8\ (which was also published in the same November issue of the New
England Journal of Medicine), in a similar group of patients not yet on
dialysis, found no significant difference in the combined incidence of
severe adverse events between the higher and lower hemoglobin groups,
although hypertensive episodes and headaches were more frequent in the
former group.
---------------------------------------------------------------------------
\7\ Ajay K. Singh, et al., ``Correction of Anemia with Epoetin Alfa
in Chronic Kidney Disease'' 355 N Engl J Med 2085-98 (2006).
\8\ Tilman B. Drueke, et al., ``Normalization of Hemoglobin Level
in Patients with Chronic Kidney Disease and Anemia'' 355 N Engl J Med
2071-84 (2006).
---------------------------------------------------------------------------
Clinical studies have found that determining optimal hemoglobin
levels is also complicated by patient variability in their response to
the drug. Researchers believe patient comorbidities, intercurrent
events like hospitalization, and practice patterns contribute to this
variability, which is not unique to the kidney care community. One
recent study concluded that the variability in the response of
hemoglobin levels to epoetin treatment over time in individual patients
may account for moving 28 percent of all dialysis patients above and
below the target hemoglobin levels during a one-year timeframe.\9\
Other studies support this finding as well.\10\ Because of this
variability in patient physiology, optimal anemia management requires a
highly individualized approach to treatment.\11\
---------------------------------------------------------------------------
\9\ E Lacson et al., ``Effect of Variability in Anemia Management
on Hemoglobin Outcomes in ESRD'' 41 Am J. Kidney Dis. 111-24 (2003).
\10\ Norma J. Ofsthun, et al., ``The Impact of the Change in CMS
Billing Rules for Erythropoietin on Hemoglobin Outcomes in Dialysis
Patients'' To Be Presented at RRI International Dialysis Conference
(January 2007).
\11\ Norma Ofthun et al., ``The Effects of Higher Hemoglobin Levels
on Mortality and Hospitalization in Hemodialysis Patients'' 63 Kidney
Internat'l 1908-14, 1913 (2003).
---------------------------------------------------------------------------
Congress and CMS should take all available studies, as well as the
Food and Drug Administration (FDA) label, into account when setting
Medicare policy. For example, the recent CMS EPO Monitoring Policy,
issued before the publication of the CHOIR and CREATE studies
recognizes the need for reimbursement policy to take into account
patient variability. When reviewing this policy, it is important to
understand that it is not a treatment guideline. Rather, it is a
reimbursement auditing tool. Under the policy, if a patients'
hemoglobin reaches 13 and the dose is not reduced, then CMS will reduce
the payment 25 percent. It does not call for, nor recommend, that
patients' hemoglobin levels should be maintained above 12.
In addition to the EPO Monitoring Policy, Congress may also
consider anemia management studies when discussing reforms to the ESRD
payment system. If Congress is considering payment revisions that
incorporate any or all separately billable drugs or biologics into the
composite rate, it is vital that an appropriate case-mix adjuster be
developed that accounts for the variability in patient response to
medications and the lack of predictability. Currently, there are no
universally accepted case-mix adjustors for patients on dialysis that
address patient variability in drug utilization. In its attempts to
develop an ESRD bundle, CMS has recognized the difficulties of
accounting for this variability as well: ``Implementation of a revised
outpatient ESRD payment system without consideration of this patient
specific variability may compromise patient access to quality of
care.'' \12\ In addition, it is critically important that if a bundle
is adopted, Congress also provide an annual update mechanism that would
allow CMS to provide updates to the base rate. Currently, the Medicare
ESRD program is the only Medicare program without such an update
mechanism. These challenges must be met before such revisions are made.
---------------------------------------------------------------------------
\12\ Department of Health and Human Services, ``Report to Congress:
Toward a Bundled Outpatient Medicare ESRD Prospective Payment System''
22 (2003).
---------------------------------------------------------------------------
Congress should examine all of the literature before advising any
policy changes. The recent trials should be reviewed along with those
that are already a part of the literature, as well as the FDA package
insert. Policy should not be based upon the result of a single clinical
trial. KCP members are committed to continuing their work with experts
in the kidney care community to determine appropriate hemoglobin levels
for patients with kidney failure, as well as with the Congress and CMS
to ensure that Medicare policy reflects the consensus of the scientific
community.
KCP appreciates the opportunity to provide these comments to the
Committee. Patient safety and quality care are at the heart of this
discussion. It is imperative that the community and government promote
the safest practices with the highest quality of care. We look forward
to expanding upon our comments based upon today's discussion as well.
Abbott Laboratories
American Kidney Fund
American Nephrology Nurses' Association
American Regent, Inc.
American Renal Associates, Inc.
American Society of Nephrology
American Society of Pediatric Nephrology
Amgen
California Dialysis Council
Centers for Dialysis Care
DaVita, Inc.
DaVita Patient Citizens
Fresenius Medical Care North America
Genzyme
Medical Education Institute
Nabi Biopharmaceuticals
National Renal Administrators Association
Northwest Kidney Centers
Renal Advantage Inc.
Renal Physician's Association
Renal Support Network
Satellite Healthcare
U.S. Renal Care
Watson Pharma, Inc.
Statement of National Kidney Foundation, Inc., New York, New York
The National Kidney Foundation (NKF) is the nation's oldest and
largest voluntary health organization serving the needs of patients
with kidney disease (CKD) and the health care professionals who care
for them. Our role is challenging since chronic kidney disease (CKD)
patients often have multiple related diseases and complications
including cardiovascular disease, hypertension, dyslipidemia, anemia,
and bone and mineral metabolism problems. NKF appreciates the concern
of the Ways and Means Committee for the quality of care for and safety
of end stage renal disease patients. A similar dedication has driven
the NKF's clinical practice guideline development program, known as the
Kidney Disease Outcomes Quality Initiative (KDOQI).
Since its inception in 1995, NKF's KDOQI has transformed medical
practice, community and public awareness, healthy policy, and patient
outcomes. KDOQI's 12 evidence-based clinical practice guidelines have
shaped the way we look at kidney disease and how it is treated in the
United States and around the world. The KDOQI process has always relied
on a structured review of the evidence and the independence of the work
group assembled to review each topic. Updates to the original KDOQI
clinical practice guidelines for hemodialysis, peritoneal dialysis, and
vascular access were published as a supplement to the July 2006 edition
of the American Journal of Kidney Diseases. A new version of the KDOQI
anemia guidelines, the Clinical Practice Guidelines for Anemia of
Chronic Kidney Disease, was published as a supplement to the May 2006
issue of the American Journal of Kidney Diseases. The KDOQI anemia
guidelines were originally published in 1997, and updated in 2001. This
new guideline has been expanded to cover all stages of chronic kidney
disease (CKD). Anemia often negatively affects the quality of life for
patients with CKD. However, among all the potential complications of
CKD, anemia is perhaps the most responsive to treatment.
The National Kidney Foundation has finalized plans for a formal
review of new information that might have an impact on these recent
recommendations from KDOQI on anemia management. This confirms KDOQI's
announcement last month that it would continue its decade-long process
of timely review of new data relevant to published KDOQI Clinical
Practice Guidelines. The Co-Chairs of KDOQI have asked the anemia work
group to reconvene on February 3, 2007, to discuss the implications of
recently published studies and studies accepted for publication on
anemia.
The first step in this process will be a structured review of the
new evidence by the NKF Evidence Review Team headquartered at Tufts New
England Medical Center in Boston. This evidence will then be examined
by the work group to determine if it has a material impact on any
recommendations made in the KDOQI Clinical Practice Guidelines and
Clinical Practice Recommendations for Anemia in Chronic Kidney Disease
published in May, 2006. When the anemia work group and the evidence
center complete the analysis of the new studies for both safety and
efficacy, appropriate announcements or publications will be developed.
This same concern for patient safety and quality of care for
Medicare beneficiaries in the End Stage Renal Disease Program leads the
NKF to urge Congress and the Centers for Medicare and Medicaid Services
to analyze with caution the recommendations from the Government
Accountability Office for bundled payment for dialysis services. We
draw the Committee's attention to a Report that Secretary Thompson sent
to Congress in 2003. The title of the Report is: ``Toward a Bundled
Outpatient Medicare ESRD Prospective Payment System.'' On page 22 there
is the following statement: ``Implementation of a revised outpatient
ESRD payment system without consideration of this patient specific
variability may compromise patient access to quality care.'' On page
31, ``The changes in practice patterns resulting from a bundled ESRD
(Prospective Payment System) will require monitoring to determine
whether clinical outcomes improve or decline as a result of the
system's financial incentives.'' On page 33, ``Several of the K/DOQI
clinical practice guidelines provide measures and minimum values of
quality dialysis. Efforts to collect and evaluate such measures will be
essential in order to ensure that clinical outcomes do not decline as
facilities respond to the new financial incentives created by a bundled
(Prospective Payment System).''
The recommendation for a bundled payment for dialysis services
reflects a concern about the potential for over-utilization that exists
under the current reimbursement policy. Nevertheless, a bundled system
creates incentives for underutilization that could negatively affect
dialysis patient outcomes. This is of particular concern in that higher
doses of epoetin are required in the African American population, those
individuals that have vasculitis as a cause of their kidney failure and
cancers such as Multiple Myeloma (United States Renal Data System 2003
Annual Data Report). Also, patients with chronic infections and
dialysis catheters also require more epoetin secondary to resistance to
the medication. As far as anemia therapy is concerned, the effect of
Medicare's early payment policy suggests that the threat of
underutilization is real. Medicare initially provided a flat payment
for erythropoietin, without regard to dosage. Under that payment
system, dosage was low and there was little improvement in the anemia
experienced by dialysis patients. There was also evidence of racial
disparities that developed based on the responsiveness to treatment
noted above (United States Renal Data System 2003 Annual Data Report).
We are concerned that these areas need to be given careful
consideration.
There are additional concerns about a bundled payment system that
should be addressed. Many patients have bone and mineral metabolism
disorders that require treatment with active vitamin D analogs. A
bundled payment system could result in a substitution of oral vitamin D
for injected vitamin D preparations. Not only would this shift cost
from Medicare Part B to Medicare Part D, but the effect of vitamin D
therapy could be much more dependent upon patient compliance. According
to the United States Renal Data System 2006 Annual Data Report, only
65% of individuals with Employer Group Health Plan medication coverage
routinely took their medications. This area should also be given
careful consideration. The potential for undertreatment may also lead
to elevated parathyroid hormone levels which is associated with epoetin
hyporesponsiveness, thereby compromising the bundled amount to cover
this medication.
Similarly, we are concerned that a bundled payment system could
lead providers to revert to the use of blood transfusions to treat
anemia in dialysis patients who are not responsive to erythropoiesis
stimulating agents. Not only would such a change in practice patterns
expose this vulnerable patient population to unknown risks from the
nation's blood supply but it would also make it difficult to consider
these patients for kidney transplantation since the transfusions
introduce antibodies that complicate organ matching.
These are only a few examples of the concerns with bundling. It was
stated at the hearing that it would be possible to make retroactive
adjustments should the bundling formula prove to be problematic.
However, there will be no way to reverse the negative patient outcomes
that could result from adoption of a bundled reimbursement policy that
does not address these issues.
The National Kidney Foundation appreciates the opportunity to
submit this testimony. The Committee members should consider the
National Kidney Foundation as a resource while it continues to
deliberate these issues. Thank you!
Statement of the Renal Physicians Association, Rockville, Maryland
The Renal Physicians Association (RPA) is the professional
organization of nephrologists whose goals are to ensure optimal care
under the highest standards of medical practice for patients with renal
disease and related disorders. RPA acts as the national representative
for physicians engaged in the study and management of patients with
renal disease. RPA greatly appreciates the interest of Committee Chair
William Thomas and Ranking Member Charles Rangel in the issues
surrounding anemia management services provided to patients with kidney
disease and kidney failure. We welcome the opportunity to offer our
perspective on these complex issues. Our testimony will focus on the
use of clinical practice guidelines and best evidence in healthcare
delivery, the role of the nephrologist in the care of patients with
kidney disease and the importance of maintaining physician prescribing
autonomy, the issue of ESRD patient variability related to EPO dose,
and common misperceptions regarding anemia management and reimbursement
for these services.
Clinical Practice Guidelines and Physician Prescribing Autonomy
RPA believes that clinical practice guidelines in renal care, like
those in other medical disciplines, should be evaluated on the basis of
the strength of evidence, an assessment of harms and benefits, and
should benefit from robust physician and other multidisciplinary input
and review. Guidelines developed with these considerations in mind will
enhance the delivery of high quality patient care and help ensure
kidney patient safety. RPA also believes that the current body of
literature in the area of anemia management fulfills these criteria,
and forms a solid foundation for public policy making efforts such as
the Centers for Medicare and Medicaid Services (CMS) recently finalized
EPO Monitoring Policy (EMP). Further, it is our opinion that the CHOIR
and CREATE studies recently published in the New England Journal of
Medicine, once they have been subject to the full measure of robust
scientific review, will likely represent an important addition to this
already significant body of literature, and should be considered
thoughtfully and thoroughly by care providers and policymakers.
However, RPA also feels compelled to note that clinical practice
guidelines are in fact guidelines, not required protocols, and that the
most important determining factor in the care of the patient should be
the physician's clinical judgment considered in the context of the
physician-patient relationship. RPA believes that it is of paramount
importance to maintain the physician's autonomy and ability to exercise
clinical judgment in prescribing for the individual patient. Decisions
for the individual may and should be permitted to deviate from the norm
on the basis of individualized clinical evaluation and specific patient
needs. This is a fundamental and well-recognized clinical principle in
medicine, and it is mandatory that it be maintained and protected. RPA
believes the CMS' EPO Monitoring Policy accounts for such use of the
physician's clinical judgment.
Variability in ESRD Patient Hemoglobin Levels
RPA believes that in the recent discourse on national coverage of
EPO, the critical issue of variability of individual patient response
to EPO dose has been understated. As noted in RPA's previous
correspondence to CMS on EPO coverage policy development, attempts to
assess or quantify individual sensitivities (i.e. responsiveness) to
EPO at a narrow level have not been successful. Thus,there is no
single, predictable response to a given dose of EPO, a fact that
accounts for the wide range in individual responses to treatment. As a
result, in the aggregate it is physiologically not rational to tailor a
normal distribution of patient responses to a payment limit: such a
paradigm cannot be successful in delivering optimaltreatment with
sophisticated agents to complicated patients. Payment limits structured
in this fashion place emphasis on the wrong arm of therapy: emphasis
should be placed rather on reducing the number of patients with low
hematocrits/hemoglobins (>30%/10 gm/dL). At the same time, Medicare
coverage policy should strive to maintain levels in all patients <11
gm/dL, given the ample data disclosing the adverse short and long-term
effects to patients with persistent anemia. Simply put, overemphasis on
monitoring patients at the upper end of the range should not create
problems for patients at the lower end, and RPA believes that the
current CMS EPO Monitoring Policy strives to avoid such problems in the
broad Medicare ESRD beneficiary population.
Misperceptions Regarding EPO Requirement
Finally, RPA would also like to take this opportunity to dispel
some common misperceptions regarding reimbursement for erythropoietin.
There have been articles in both the mainstream and medical trade press
implying that nephrologists have a financial incentive to prescribe
higher doses of erythropoietin to ESRD patients. This is simply not
true. Nephrologists prescribe EPO based on their clinical judgment of
what will optimize the individual patient's hemoglobin level. Moreover,
it is the dialysis facility that receives reimbursement for EPO
prescribed to ESRD patients, not the nephrologist, and thus any
inference that the nephrologist will personally benefit from
prescribing higher doses of EPO, or any drug, to ESRD patients is
erroneous.
Conclusion
In conclusion, RPA supports the use of clinical practice guidelines
in the development of protocols enhancing the delivery of high quality
patient care but believes they must be considered in the context of the
physician's clinical judgment. RPA believes that physician prescribing
autonomy must be maintained, and that the variability in ESRD patient
hemoglobin levels must be accounted for in the development of national
coverage policy for EPO. Finally, the misperception that nephrologists
have a financial incentive to prescribe high doses of EPO to ESRD
patients is erroneous. Once again, RPA appreciates the opportunity to
provide our perspective on these issues to the Committee, and we make
ourselves available as a resource to the Committee in its future
efforts to ensure the best possible health outcomes and quality of life
for Medicare beneficiaries with ESRD.
Statement of Kris Robinson, American Association of Kidney Patients,
Tampa, Florida
The American Association of Kidney Patients (AAKP) (www.aakp.org),
founded in 1969, is the nation's only kidney patient-led and managed
education and advocacy organization for people with kidney disease.
AAKP serves over one million Americans annually who have either lost
kidney function (and live with dialysis or transplant) or have chronic
kidney disease (CKD). As you may know, the average life expectancy for
individuals following initiation of dialysis therapy is short, less
than 5 years. As patients ourselves, we realize the important need to
ensure quality of care and access for all dialysis and potential
dialysis patients.
AAKP was instrumental in the fight for the enactment of the
Medicare ESRD Program. In 1972, Shep Glazer, the Vice President of our
forerunner organization, testified before the House Ways and Means
Committee while being dialyzed. This effort was crowned with success in
1972 when Congress enacted the program that continues to provide
Medicare funding for dialysis and kidney transplantation.
AAKP appreciates the opportunity to provide written testimony to
the House Ways and Means Committee. We are available to assist the
Committee with needed information as it continues to review quality of
care issues for today's dialysis patients. AAKP's written testimony
will provide patients' views on safety and efficacy in healthcare and
provide insight into what patients need to ensure a high quality of
life and health.
AAKP commends the Committee for assessing and reviewing patient
safety and quality issues for care received by dialysis patients.
AAKP's mission has always been to help all kidney patients achieve the
best possible quality of life and longevity.
Regarding specific issues under review by the Committee, AAKP has
developed and distributed position papers on the following topics in
recent years: 1). Support for the continuation of the patient and
physician relationship in medical care; 2). Support for continuous
quality care and improvement and 3). Support for continuous safety
monitoring. The content of these positions is summarized below:
Patient/Physician Relationship--AAKP strongly believes the
principle that a physician and patient must be permitted to decide a
care plan best suited for that patient. Averages and other statistics
are fine for certain purposes, but medicine is fundamentally about the
treatment of a unique individual. In this light, we worry that any
legislation that mandates particular treatment options may impede the
doctor/patient decision-making relationship.
ESRD Continuous Quality Improvement--AAKP supports legislation to
provide data on outcomes and quality of care for kidney patents. We
worry that piecemeal approaches to improving quality may not offer the
best health outcomes for patients and is why we have continuously asked
Congress to establish a ``National Commission on Improved Kidney
Patient Outcomes.''
Safety Monitoring--AAKP supports legislation to ensure safety in
healthcare settings. We applaud Congress, the Centers for Medicare and
Medicaid Services (CMS) and the renal community as a whole for
developing programs to ensure safety for all patients. However, we wish
to encourage Congress to look at the major safety issues that impact
all patients.
With regard specifically to the administration of erythropoietin
(EPO) to patients, AAKP has previously addressed CMS with comments and
questions regarding dosing policies. Though recent clinical studies
such as CHOIR have demonstrated mortality in non-dialysis patients, we
have asked CMS ``Is there any clinical data that demonstrates that
dialysis patients--either nationally or regionally--are in fact
receiving more EPO than necessary to maintain an appropriate hematocrit
level, or that inappropriate EPO prescribing by physicians is the
driver for increased EPO spending?'' We are awaiting a response.
Furthermore, AAKP is also interested in how the issues currently
being discussed about EPO will be affected by the ``Medicare
Prescription Drug, Improvement, and Modernization Act of 2003'' (DIMA)
(P.L. 108-173). In particular, section 623 of DIMA instructs CMS to
implement effective January 1, 2005, a new ``basic case-mix adjusted
composite rate,'' which would, inter alia, transfer the dollar
difference (the ``spread'') between acquisition and Medicare payment
rates for separately billed drugs and biologicals (including
erythropoietin) to the per-session composite rate for dialysis
treatment. Even if there is some current law financial incentive for
overutilization of EPO, would not that incentive be eliminated by
section 623? We are concerned that section 623 has not occurred as
scheduled.
National Commission on Improved Kidney Patient Outcomes--AAKP
previously wrote to the House Ways and Means Committee calling for a
``National Commission on Improved Kidney Patient Outcomes.'' We believe
a global perspective--rather than a piecemeal approach--is needed to
improve quality and coordination of medical care for dialysis patients,
and perhaps even create savings to Medicare. Indeed, as payor for the
medical care of about 75 percent of all kidney patients receiving
dialysis in the United States, CMS bears a special responsibility to
ensure that dialysis patients not only receive quality medical care--
but that care is provided in a manner that maximizes positive outcomes.
We would note the November 21 report by the HHS Inspector General
calling for more collection of quality of care date in the ESRD
program. AAKP believes these issues will become even more urgent as the
nation's dialysis population is expected to grow three-fold over the
next decade.
Moreover, in the kidney community today, there is a vigorous debate
about the adequacy of medical care of dialysis patients, prompted by
apparently higher U.S. dialysis patient disability, morbidity, and
mortality in cross-national studies. Some have argued that it is a
``national disgrace that the death rate now solidly stays in the region
of 24% every year and has more than doubled over the last 30 years''
(Kjellstrand, CM, Blagg, CR, ``Differences in dialysis practice are the
main reasons for the high mortality rate in the United States compared
to Japan,'' Hemodial Int. 2003; 7(1): 70). Others believe that cross-
national comparisons are flawed for selection reasons (i.e., sicker,
older patients are denied dialysis in comparison countries) and that
the U.S. should take pride in the unique availability of dialysis here
(see, e.g., Friedman, EA, ``International comparisons of survival on
dialysis: Are they reliable?'' Hemodial Int. 2003; 7(1):59-66). In any
case, with the U.S. ranking last among industrialized countries in
mortality for kidney patients, there is a clear need to take a close
look at the adequacy of medical care for U.S. dialysis patients.
Charged with a comprehensive program review, the agenda for such a
National Commission might also include patient access to other
important renal replacement treatments, such as home dialysis and
transplantation; nephrologists' residency training; and reimbursement
of rural facilities. There are also many other opportunities to improve
care and reduce costs to Medicare, including slowing the progression to
ESRD among chronic kidney disease patients (CKD), better chronic
disease management, advances in new technology and biomedical
solutions, more transplantation, and improved patient education. AAKP
stands ready to assist the Committee on ways to implement such a
Commission.
AAKP commends the Committee for addressing the issues of quality of
care as currently delivered to the over 300,000 dialysis patients. We
appreciate the opportunity to provide input into your efforts and look
forward to working with you to provide continuous quality improvement
to all patients.
Statement of Dori Schatell, Medical Education Institute, Madison,
Wisconsin
The Medical Education Institute (MEI) is a non-profit foundation
dedicated to the mission of helping people with chronic diseases learn
to manage and improve their health. Since 1993, MEI efforts have
focused on improving longevity and quality of life for people with
chronic kidney disease (CKD) through health behavior research and
evidence-based patient and professional education materials. I
appreciate the opportunity to provide an additional viewpoint for the
Committee.
To begin, I am dismayed by the implication that the K/DOQI
Guidelines were influenced by industry participation. When the MEI
administered the first DOQI Guidelines in 1996, I was the ``writer''
for the Anemia Work Group, recording their deliberations. Amgen
requested and was denied permission to observe the proceedings of this
Work Group--they read the final Guidelines at the same time as the rest
of the renal community and had no role in the outcome. The clinicians
who spent many months of intensive hours reviewing hundreds of
scientific papers and developing recommendations were a dedicated,
conscientious group who knew they were making history. I am very
saddened to hear their labors and the subsequent results of groups that
updated the National Kidney Foundation K/DOQI Guidelines denigrated and
their ethics called into question. Industry support from Amgen and
others for the original DOQI Guidelines and subsequent K/DOQI
Guidelines has moved the practice of nephrology forward by helping to
establish key clinical benchmarks in a number of vital areas of
practice, including nutrition, bone disease, dialysis adequacy,
vascular access, etc. Where would patients be today if those Guidelines
had not been written? Who would have supported their development if
industry had not stepped up to the plate?
Second, much of the criticism of current CMS policy regarding
anemia treatment for people on dialysis is based on the recently-
published CHOIR study. Having reviewed the results, which concluded
that patients with stages 3-4 CKD had a 34% higher risk of adverse
outcomes if their hemoglobin levels were 13.5 g/dL, several aspects of
this paper were of sufficient concern to possibly call the conclusions
into question:
Study power: A power analysis revealed that 1,352
patients would need to be enrolled; data were reported for 1,432
patients on the basis of intent to treat. But 549 patients withdrew
from the study without having had a composite event. Did this bias the
findings?
Baseline differences between the high and low hemoglobin
groups: There were significant differences in baseline data for
cardiovascular history. Those in the high Hgb group had a significantly
higher rate of high blood pressure (p=0.03) and coronary artery bypass
graft (p=0.05) prior to the study. Did this influence the results?
Differences in baseline GFR: It is unclear whether the
time to renal replacement therapy (RRT) analysis accounted for baseline
variations in GFR. Clearly individuals with a GFR of 15 at baseline are
much closer to needing RRT than those whose GFR was 50.
No blood pressure changes: Despite worse cardiovascular
outcomes in the high hemoglobin group, there were no significant
changes in blood pressure in the high vs. low hemoglobin group. This
seems odd.
Lack of statistical significance: The CHOIR authors state
that there were, ``no significant differences between the two groups in
the four individual components of the primary composite end point
(hospitalization, MI, stroke, or death). . . . However, the hazard
ratios for death and hospitalization for CHF had strong trends toward a
higher risk in the high-hemoglobin group than in the low-hemoglobin
group.'' Also, the risk of heart attack (MI) with high hemoglobin
was.91 (less than 1.00); thus those with higher hemoglobin actually had
a lower risk of MI.
Non-standard measurement of quality of life: It is
unclear why three separate tools were used to assess quality of life
(QOL). Given the highly unusual finding of no QOL benefit to a higher
vs. a lower hemoglobin, one must wonder if patients were overwhelmed by
the sheer number of survey items (a total of 83 questions, many with
sub-questions). Multiple studies in CKD and dialysis patients have
shown that those with higher hemoglobin levels score significantly
higher in physical and mental functioning on the SF-36 and Kidney
Disease Quality of Life (KDQOL).\1\ \2\ And, in the dialysis
population, higher physical and mental functioning independently
predict lower rates of hospitalization and death.\3\ One of the tools
used in the CHOIR study (LASA) was developed for breast cancer and has
been used only twice before in kidney patients. Interestingly, in one
of those two studies, 1,557 non-randomized predialysis CKD patients
received r-HuEPO, and their hemoglobin levels rose from 9.1 g/dL to
11.6 g/dL in 16 weeks with significant improvement in all QOL
parameters.\4\ In the other analysis by some of the same researchers,
there was ``a positive and significant relationship between Hb levels
and QOL.''\5\
\1\ Perlman RL, Finkelstein FO, Liu L, Roys E, Kiser M, Eisele G,
Burrows-Hudson S, Messana JM, Levin N, Rajagopalan S, Port FK, Wolfe
RA, Saran R. Quality of life in chronic kidney disease (CKD): a cross-
sectional analysis in the Renal Research Institute-CKD study. Am J
Kidney Dis. 45(4):658-66, 2005.
\2\ Mapes DL, Bragg-Gresham JL, Bommer J, Fukuhara S, McKevitt P,
Wikstrom B, Lopes AA. Health-related quality of life in the Dialysis
Outcomes and Practice Patterns Study (DOPPS). Am J Kidney Dis. 2004
Nov;44(5 Suppl 2):54-60
\3\ Lowrie EG, Curtin RB, LePain N, Schatell D. Medical outcomes
study short form-36: a consistent and powerful predictor of morbidity
and mortality in dialysis patients. Am J Kidney Dis. 2003
Jun;41(6):1286-92.
\4\ Provenzano R, Garcia-Mayol L, Suchinda P, Von Hartitzsch B,
Woollen SB, Zabaneh R, Fink JC; POWER Study Group. Once-weekly epoetin
alfa for treating the anemia of chronic kidney disease. Clin Nephrol
61(6):392-405, 2004.
\5\ Lefebvre P, Vekeman F, Sarokhan B, Enny C, Provenzano R,
Cremieux PY. Relationship between hemoglobin level and quality of life
in anemic patients with chronic kidney disease receiving epoetin alfa.
Curr Med Res Opin. 22(10):1929-37, 2006.
In light of these concerns--and of the exactly contradictory
findings of the CREATE study in a similar population published in the
same issue of the New England Journal of Medicine--the MEI urges the
Committee to proceed with caution and consider all of the available
data.
Third, previous CMS policies related to use of EPO to correct
anemia in people on dialysis have had unintended consequences that have
harmed patients. Early reimbursement of EPO offered incentives to
undertreat patients when dialysis centers were paid $40 for up to
10,000 units and $30 additional for more than 10,000 units. The
Hematocrit Measurement Audit (HMA) policy, which stopped EPO
reimbursement to dialysis centers for patients whose hematocrit levels
rose above a rolling average of 36.5%--without a provision to permit
physicians to medically justify higher levels--led to lower average
hematocrit levels and patients complained of a ``roller coaster''
effect that was very debilitating. Please see the attached article the
MEI published in Nephrology News and Issues with patient interviews
that illustrate in these individuals' own voices how difficult it is to
care for children or grandchildren, do simple tasks around the home
(like vacuuming, hammering nails, or washing windows), hold down a
job--or even walk to the mailbox with inadequate anemia correction, and
how very much better patients feel at a higher vs. a lower hematocrit.
Anecdotally, patients report they feel every percent of difference in
their hematocrit or hemoglobin. The MEI is concerned that bundling EPO
with other drugs may, over time, lead to underutilization as dialysis
centers attempt to hold down costs to compensate for inflation--unless
an appropriate case mix adjuster is used and an annual update mechanism
is created, as was proposed in the Kidney Care Quality and Improvement
Act.
Fourth, each year, according to the United States Renal Data System
(USRDS) half of the more than 100,000 individuals who reach end-stage
and need dialysis or transplant to survive are under age 65, or
``working-age.'' Enabling working-age patients to keep their job
benefits:
Patients themselves--through improved social contacts,
higher income than disability would pay, and access to benefits that
may include an employer group health plan (EGHP)
Dialysis providers--by improving payer mix for dialysis
centers
Medicare and Social Security--by reducing the number of
ESRD patients who have Medicare as their primary health coverage and
the number collecting disability benefits.
More than 354,000 working age patients started dialysis from 1992-
2003. Of these, 102,104 were working 6 months prior. More than 71% of
these working patients did not receive any EPO to treat their anemia
prior to kidney failure, contributing significantly to reduction in
employment levels in more than 31,000 patients.\6\ In 1973, the
Medicare ESRD Program was funded based on the belief that people who
received treatment for kidney failure would be active, productive, tax-
paying citizens. To the extent that physicians and patients are
frightened that appropriate anemia treatment will harm them and
patients are undertreated as a result, the goal of keeping patients
working will become even more difficult to achieve.
---------------------------------------------------------------------------
\6\ Hofmann RM, Schatell D, Witten B, Muehrer R, Gagnon R, Becker
BN. Factors contributing to employment of working-age ESRD patients at
initiation of dialysis therapy. Publication pending.
---------------------------------------------------------------------------
Finally, if there is a trade-off to be made between length of life
and quality of life (with a lower versus a higher hemoglobin level),
only one person can legitimately make that choice: the person with
anemia. I hope the Committee will bear in mind that reimbursement
policies ultimately and dramatically affect the day-to-day lives and
futures of people with kidney failure.
Perhaps the Committee could consider looking at innovative ways to
reduce costs while improving patient outcomes. For example, why not
incentivize patients to receive their EPO doses subcutaneously, which
is more effective and less costly--but requires more needle sticks.
Waiving all or a portion of their Medicare Part B premiums ($93.50
monthly in 2007) for patients who accept subcutaneous dosing would
likely save considerably more than it would cost. (Incidentally,
concerns about pure red cell aplasia with subcutaneous dosing of ESR
products in Canada and Europe have now been attributed to the use of
uncoated rubber stoppers in the vials, a practice that has now been
stopped).\7\
---------------------------------------------------------------------------
\7\ Boven K, Knight J, Bader F, Rossert J, Eckardt K, Casadevall N.
Epoetin-associated pure red cell aplasia in patients with chronic
kidney disease: Solving the mystery. Nephrol Dial Transplant. 20 Suppl
3:iii33-40, 2005.
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Coalition for Dialysis Patient Choice
December 20, 2006
The Coalition for Dialysis Patient Choice attended the recent
Congressional hearing on Patient Safety and Quality Issues in End Stage
Renal Disease Treatment and appreciates the opportunity to provide
comments to the Committee regarding reimbursement policies for anemia
management. The Coalition is a non-profit organization formed by
companies and organizations dedicated to increasing the availability of
innovative, more physiologic dialysis therapies (more frequent and/or
longer duration) and reducing the barriers to home and self-care
dialysis.
Given the critical role the Federal government has assumed in the
care of dialysis patients, we commend the Committee's efforts to seek
optimum care in anemia management. As payment methodologies are
revised, our Coalition asks that the potential to increase patient
access to home and more frequent dialysis modalities also be
considered. Greater utilization of these modalities will assist in
addressing the particular anemia management questions raised by this
Committee, and also has the potential to lower total Medicare costs and
improve beneficiary outcomes.
Hemoglobin levels in the dialysis population and utilization of
pharmaceuticals to address anemia are without doubt influenced by
payment policy. The same ``perverse incentives'' that influence in-
center IV drug utilization also discourage home therapies. As stated in
the Government Accountability Office's (GAO) recent report titled
Bundling Medicare's Payment for Drugs with Payment for All ESRD
Services Would Promote Efficiency and Clinical Flexibility, ``Studies
have shown that daily hemodialysis--which some experts contend is
clinically preferable--reduced the need for Epogen in some ESRD
patients with anemia . . . [h]owever, Medicare coverage is limited to
three dialysis treatments a week.'' Evidence supports that home
dialysis, whether peritoneal dialysis (PD) or home hemodialysis (HHD),
is currently underutilized--despite the continued evidence reported
annually, using Medicare data, that the home setting leads to the
lowest total cost of care.
We support the GAO's recommendation for the rapid implementation of
the ``expanded bundle'' as soon as possible. If coupled with
appropriate safeguards and case-mix considerations to protect patients
from EPO underutilization, this method represents a very effective way
to reverse these perverse incentives and their negative unintended
consequences. We further favor the implementation of the ``expanded
bundle'' for all beneficiaries as soon as practicable.
However, neither patients nor Medicare will realize the greatest
potential benefit if revision in payment policy results only in more
efficient utilization of pharmaceuticals within today's most prevalent
treatment regimen--thrice weekly hemodialysis in the center, or
``conventional'' hemodialysis. A large, recent randomized study of
conventional dialysis (the NIH/NIDDK's HEMO study) showed that
modifications within this treatment regimen were unlikely to lead to
significant improvement in patient outcomes. To materially improve
patient outcomes and reduce Medicare costs, more significant
modifications are required to the way dialysis is delivered. Simply
put, significantly better dialysis is required for further improvement,
and more frequent/longer dialysis provides this opportunity.
The clinical evidence supporting more frequent and longer dialysis
modalities is compelling and growing. By closer approximation of the
24/7 workings of the naturally functioning kidney, more frequent/longer
dialysis leads to a number of potential patient benefits. Relevant to
this discussion, these therapies have been shown to improve anemia
management with lower pharmaceutical needs. In addition, however, these
therapies can also:
Dramatically improve blood pressure management with fewer
antihypertensive drug needs
Better manage patient bone disease and vascular
calcification
Improve patient nutrition
Improve patient rehabilitation/functional status.
The most natural setting for these therapies is in the home, where
the patient retains control of his or her schedule and care. Together,
these improvements have the potential to dramatically reduce Medicare
ESRD drug and hospitalization expenses currently incurred by
conventional dialysis patients (these expenses represent over 60% of
the current total annual cost of care), and can facilitate patients'
continued contributions as productive members of society.
We believe that the expanded bundle structure, with appropriate
performance measures and the implementation of ``shared savings''
concepts that have been discussed, can be instrumental in encouraging
therapies beyond today's conventional, thrice weekly in-center
dialysis.
In summary, the Coalition asks that the Committee consider the
anemia management questions more broadly, and to take a total cost of
care perspective when recommending changes. We ask that you explicitly
consider the potential to encourage appropriate utilization of home and
more frequent/longer dialysis therapies when making payment policy
modifications. By doing this, not only will Medicare encourage optimal
anemia management but, at the same time, optimize patient care costs
and outcomes.
Again, we appreciate the opportunity to provide input into this
important process. We stand ready to provide any additional information
that will assist the Committee and the community in this valuable work.
Sincerely,
Joseph E. Turk, Jr.
NxStage Medical
Founding Member
Rod Kenley
Aksys Ltd.
Founding Member
Jim Sweeney
Renal Solutions, Inc.
Founding Member
Dori Schatell
Medical Education Institute
Supporting Partner
Statement of Patricia Tate-Harris, Association of Dialysis Advocates,
Baton Rouge, Louisiana
The Association of Dialysis Advocates (ADA) is a grassroots
patient/family advocacy organization dedicated to ensuring quality,
safe care for dialysis patients. ADA is self-funded through personal
contributions of our members. We do not accept financial or other
contributions from the dialysis industry, pharmaceutical industry,
healthcare industry, or any government entity so that we maintain our
integrity and objectivity in addressing dialysis-related issues.
ADA is encouraged by the House of Representatives Ways and Means
Committee's hearing of December 6, 2006 re: the ESRD Program. We are
encouraged that our congressional representatives are holding Centers
for Medicare & Medicaid (CMS) accountable for the quality of care
provided dialysis patients and for the efficient use of taxpayer
dollars that fund care. We are encouraged that our representatives
expressed favor towards a ``patients come first'' policy and pointed to
the need for CMS policies to reflect such a policy.
ADA is most grateful for the contributions and support of the
Hearing panelists for bringing forth data related to the use of Epogen
and its detrimental effect on patients. When this medication is not
administered within established guidelines for the benefit of patients,
poor patient outcomes can follow. We are also thankful for the recent
media interest and coverage that brought information to dialysis
patients and taxpayers regarding the clinical and fiscal issues related
to use and overuse of Epogen.
The hearing of December 6, 2006 is but another beginning. ADA
patients and families are hopeful that the Ways and Means Committee
will pursue all issues related to the federal ESRD program so that
patients receive quality and safe care at a reasonable cost to
taxpayers. ADA has longed believed that attention to the use of Epogen
has been a necessity.
ADA's positions regarding specific issues covered during the
hearing follow:
EPOGEN
ADA strongly believes that an immediate congressional directive
should be given to CMS to incorporate into the federal ESRD Program the
recent FDA warning regarding Epogen.
ADA supports bundling of services and drugs, including Epogen.
Additionally, ADA supports the clinical individualization of treatment
to best meet the needs of the patient. It is essential, ADA believes,
that the current financial incentive to overuse Epogen be removed and
replaced with emphasis on patient safety. Lastly, ADA believes that the
bundling of services and drugs will discourage the overuse of Epogen
and focus greater attention upon the adequacy of iron stores--and at
lesser cost--for what should be a more efficacious use of Epogen.
ADA supports subcutaneous administration of Epogen. ADA believes
that clinical and cost perspectives, as demonstrated by the United
States Veterans' Administration Hospitals (as well as in Europe), are
supported by subcutaneous administration of Epogen. Further, ADA
believes that use of multi-dose vials of Epogen will address existing
provider and patient concerns regarding stinging during subcutaneous
administration.
Lastly, we request that Congress intervene to ensure that (1)
policies, payment and others, are based upon safe delivery of care to
patients by experts having no conflict of interest due to their
positions, affiliations or relationships within the dialysis industry
and/or pharmaceutical companies and that (2) every effort be made to
ensure that no one pharmaceutical company monopolizes any medication
required for quality treatment of chronic renal failure and/or ESRD
patients. This is a population that is escalating--the protection
afforded a monopoly is not in the best interest of patients or
taxpayers.
INCREASED FUNDING FOR PREVENTION AND SPECIAL INVESTIGATIVE STUDIES
ADA is supportive of the suggestion that NIH be appropriated
funding to research the factors that c ontribute to over-representation
of minorities--particularly African-Americans, Hispanics and Native
American Indians--among the kidney failure/dialysis population. We
further support a focus upon prevention efforts accompanied by funding
that ensures attainment of program goals.
Congress has established 18 ESRD networks to provide quality
assurance of the ESRD program. African-Americans comprise 12-13% of the
general population yet represents, according to ESRD Networks 2005
Annual Reports, at least 33% of the dialysis population in ten of the
eighteen ESRD Network regions. In seven of the ten ESRD Network regions
the African-American dialysis population is 40% or greater. Glaringly,
the African-American dialysis population in Network 5 (Maryland,
Virginia, Washington, DC, West Virginia) is 59%; in Network 6 (Georgia,
North Carolina, South Carolina) African-Americans comprise 67% of the
dialysis population; in Network 8 (Alabama, Mississippi, Tennessee)
62.3%; and Network 13 (Arkansas, Louisiana and Oklahoma) 52.5%. Such
over-representation is an event that cannot, and must not, be minimized
but rather calls for assertive and aggressive prevention and treatment
programs.
CMS' RESPONSIBILITY TO BENEFICIARIES
Among other things, CMS' responsibility to beneficiaries is three-
fold: (1) ensuring quality, safe delivery of care (2) ensuring
sufficient information is provided in order for beneficiaries to make
informed decisions, and (3) ensuring an effective systematicmechanism
through which to address patient concerns related to care.
Quality, Safe Delivery of Care
No policy related to quality, safe care is truly meaningful unless
it is actually incorporated intothe clinical performance measures and
ancillary services required by its respective patient population. All
stake-holders from patient-families to healthcare workers fully
recognize that it will be incumbent upon Congress to ensure that CMS,
ESRD Networks and the state survey agencies each carry out
responsibilities related to the oversight and enforcement of ESRD
Conditions for Coverage.
The recent exposure of a dialysis facility in Birmingham, Alabama
(Birmingham News, November 19, 2006, ``Patients feel they're
mistreated: Dialysis centers focus on profits, advocates say'') that
had not been inspected since 1998 simply demonstrates the laxity with
which CMS, the ESRD Network and state survey agency carried out their
statutory, contractual responsibilities. Equally revealing were (1)
HHS, Office of Inspector General report, Availability of Quality of
Care Data in the Medicare End Stage Renal Disease Program, November
2006 report (OEI-05-05-0030) that reflected the ESRD networks' lack of
necessary data to identify facilities ``with quality improvement
needs,'' and (2) HHS, Office of Inspector General Civil Monetary
Penalty report (November 2006) that reported a dialysis facility/
owner's agreement to pay $150,000 to resolve liability for submission
of Medicare claims although
``inadequate and/or worthless services had been rendered to
patients''--worthless services that the HHS OIG alleged ``may have
contributed to seven deaths . . .''
Patients' lives are at stake! It is unconscionable that CMS, ESRD
networks and state survey agencies have permitted such failures in
oversight while dialysis chains/facilities and pharmaceutical companies
nevertheless have experienced raging financial returns.
Making Informed Decisions
ADA supports the 2004 ESRD Initiative for Quality Care. However,
ADA also firmly believes that the initiative's original intention
regarding the Dialysis Facility Compare website and chart was to
``empower consumers with quality of care information to make more
informed decisions about their healthcare'' and such has not been met.
Patients and consumers still need ``to review and compare facilities
and choose a dialysis facility that best meets their needs.'' While
there is information posted regarding anemia, hemodialysis adequacy,
and patient survival, ADA believes the information is limited. Dialysis
Facility Compare does not provide other highly pertinent information
for ordinary patients and/or consumers to truly make informed
decisions. For instance, patients and consumers are greatly interested
in (a) staffing--including their education, skills knowledge and
training, and licensing and/or certification (b) inspection reports,
and (c) infections and infection rates. And, most unfortunately, the
information provided is not easily understood by ordinary patient-
consumers. Both the public disclosure and the simplification of the
information found on the Dialysis Facility Compare chart will support
informed decision-making while furthering patient education and safety.
CONCLUSION
We, at the Association of Dialysis Advocates, are encouraged and
confident that the Ways and Means Committee will be steadfast in its
efforts to ensure quality care to dialysis patients and the efficient
use of public funds. Similarly, we are encouraged and confident that
the Ways and Means Committee will work to ensure that CMS, ESRD
Networks, and State Survey Agencies carry forth their responsibilities
in the best interest of dialysis patients, their families, and the
public. ADA stands ready to participate and serve in deliberations
related to the delivery of care in dialysis environments.
Very truly yours,
Patricia Tate-Harris
President