[House Hearing, 113 Congress]
[From the U.S. Government Publishing Office]






21ST CENTURY CURES: EXAMINING BARRIERS TO ONGOING EVIDENCE DEVELOPMENT 
                                  AND 
                             COMMUNICATION

=======================================================================

                                HEARING

                               BEFORE THE

                         SUBCOMMITTEE ON HEALTH

                                 OF THE

                    COMMITTEE ON ENERGY AND COMMERCE
                        HOUSE OF REPRESENTATIVES

                    ONE HUNDRED THIRTEENTH CONGRESS

                             SECOND SESSION

                               __________

                             JULY 22, 2014

                               __________

                           Serial No. 113-163

[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]

      Printed for the use of the Committee on Energy and Commerce
                      energycommerce.house.gov
                                   ______

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                    COMMITTEE ON ENERGY AND COMMERCE

                          FRED UPTON, Michigan
                                 Chairman
RALPH M. HALL, Texas                 HENRY A. WAXMAN, California
JOE BARTON, Texas                      Ranking Member
  Chairman Emeritus                  JOHN D. DINGELL, Michigan
ED WHITFIELD, Kentucky                 Chairman Emeritus
JOHN SHIMKUS, Illinois               FRANK PALLONE, Jr., New Jersey
JOSEPH R. PITTS, Pennsylvania        BOBBY L. RUSH, Illinois
GREG WALDEN, Oregon                  ANNA G. ESHOO, California
LEE TERRY, Nebraska                  ELIOT L. ENGEL, New York
MIKE ROGERS, Michigan                GENE GREEN, Texas
TIM MURPHY, Pennsylvania             DIANA DeGETTE, Colorado
MICHAEL C. BURGESS, Texas            LOIS CAPPS, California
MARSHA BLACKBURN, Tennessee          MICHAEL F. DOYLE, Pennsylvania
  Vice Chairman                      JANICE D. SCHAKOWSKY, Illinois
PHIL GINGREY, Georgia                JIM MATHESON, Utah
STEVE SCALISE, Louisiana             G.K. BUTTERFIELD, North Carolina
ROBERT E. LATTA, Ohio                JOHN BARROW, Georgia
CATHY McMORRIS RODGERS, Washington   DORIS O. MATSUI, California
GREGG HARPER, Mississippi            DONNA M. CHRISTENSEN, Virgin 
LEONARD LANCE, New Jersey                Islands
BILL CASSIDY, Louisiana              KATHY CASTOR, Florida
BRETT GUTHRIE, Kentucky              JOHN P. SARBANES, Maryland
PETE OLSON, Texas                    JERRY McNERNEY, California
DAVID B. McKINLEY, West Virginia     BRUCE L. BRALEY, Iowa
CORY GARDNER, Colorado               PETER WELCH, Vermont
MIKE POMPEO, Kansas                  BEN RAY LUJAN, New Mexico
ADAM KINZINGER, Illinois             PAUL TONKO, New York
H. MORGAN GRIFFITH, Virginia         JOHN A. YARMUTH, Kentucky
GUS M. BILIRAKIS, Florida
BILL JOHNSON, Missouri
BILLY LONG, Missouri
RENEE L. ELLMERS, North Carolina

                         Subcommittee on Health

                     JOSEPH R. PITTS, Pennsylvania
                                 Chairman
MICHAEL C. BURGESS, Texas            FRANK PALLONE, Jr., New Jersey
  Vice Chairman                        Ranking Member
ED WHITFIELD, Kentucky               JOHN D. DINGELL, Michigan
JOHN SHIMKUS, Illinois               ELIOT L. ENGEL, New York
MIKE ROGERS, Michigan                LOIS CAPPS, California
TIM MURPHY, Pennsylvania             JANICE D. SCHAKOWSKY, Illinois
MARSHA BLACKBURN, Tennessee          JIM MATHESON, Utah
PHIL GINGREY, Georgia                GENE GREEN, Texas
CATHY McMORRIS RODGERS, Washington   G.K. BUTTERFIELD, North Carolina
LEONARD LANCE, New Jersey            JOHN BARROW, Georgia
BILL CASSIDY, Louisiana              DONNA M. CHRISTENSEN, Virgin 
BRETT GUTHRIE, Kentucky                  Islands
H. MORGAN GRIFFITH, Virginia         KATHY CASTOR, Florida
GUS M. BILIRAKIS, Florida            JOHN P. SARBANES, Maryland
RENEE L. ELLMERS, North Carolina     HENRY A. WAXMAN, California (ex 
JOE BARTON, Texas                        officio)
FRED UPTON, Michigan (ex officio)
 
 
 
 
 
 
 
 
 
 
 
 
                             C O N T E N T S

                              ----------                              
                                                                   Page
Hon. Joseph R. Pitts, a Representative in Congress from the 
  Commonwealth of Pennsylvania, opening statement................     1
    Prepared statement...........................................     2
Hon. Henry A. Waxman, a Representative in Congress from the State 
  of California, opening statement...............................     5
Hon. Fred Upton, a Representative in Congress from the State of 
  Michigan, prepared statement...................................    83
Hon. Frank Pallone, Jr., a Representative in Congress from the 
  State of New Jersey, prepared statement........................    83

                               Witnesses

Josh Rising, Director, Medical Devices, The Pew Charitable Trusts     6
    Prepared statement...........................................     8
Louis Jacques, Senior Vice President and Chief Clinical Oficer, 
  ADVI...........................................................    13
    Prepared statement...........................................    15
    Answers to submitted questions...............................    87
Michael A. Mussallem, Chairman and CEO, Edwards Lifesciences.....    21
    Prepared statement...........................................    23
    Answers to submitted questions...............................    91
Gregory Schimizzi, Co-Founder, Carolina Arthritis Associates.....    36
    Prepared statement...........................................    38
    Answers to submitted questions...............................   100
Mary Grealy, President, Healthcare Leadership Council............    53
    Prepared statement \1\.......................................    55
    Answers to submitted questions...............................   106

                           Submitted Material

Letter of July 22, 2014, from the Lupus Foundation of America to 
  the subcommittee, submitted by Mr. Pitts.......................    85

----------
\1\ The attachments to Ms. Grealy's testimony are available at 
  http://docs.house.gov/meetings/IF/IF14/20140722/102524/HHRG-
  113-IF14-Wstate-GrealyM-20140722.pdf.

 
21ST CENTURY CURES: EXAMINING BARRIERS TO ONGOING EVIDENCE DEVELOPMENT 
                           AND COMMUNICATION

                              ----------                              


                         TUESDAY, JULY 22, 2014

                  House of Representatives,
                            Subcommittee on Health,
                          Committee on Energy and Commerce,
                                                    Washington, DC.
    The subcommittee met, pursuant to call at 3:00 p.m., in 
room 2322, Rayburn House Office Building. Hon. Joseph R. Pitts 
(chairman of the subcommittee) presiding.
    Present: Representatives Pitts, Burgess, Shimkus, 
Blackburn, Lance, Bilirakis, Ellmers, Pallone, Green, Barrow, 
DeGette, and Waxman (ex officio).
    Staff: Leighton Brown, Press Assistant; Noelle Clemente, 
Press Secretary; Sydne Harwick, Legislative Clerk; Robert 
Horne, Professional Staff Member, Health; Carly McWilliams, 
Professional Staff Member, Health; Chris Sarley, Policy 
Coordinator, Environment & Economy; Heidi Stirrup, Health 
Policy Coordinator; Jessica Wilkerson, Legislative Clerk; Ziky 
Ababiya, Staff Assistant; Eric Flamm, FDA Detailee; Eddie 
Garcia, Professional Staff Member; Karen Nelson, Deputy 
Committee Staff Director for Health.

OPENING STATEMENT OF HON. JOSEPH R. PITTS, A REPRESENTATIVE IN 
         CONGRESS FROM THE COMMONWEALTH OF PENNSYLVANIA

    Mr. Pitts. The subcommittee will come to order. The chair 
will recognize himself for an opening statement.
    In this, the sixth hearing of our 21st Century Cures 
Initiative, we are examining continued evidence development and 
communication of information regarding treatments and cures in 
the real world setting. Discovery of the risks and benefits of 
drug or treatment does not end with FDA approval or clearance. 
It is often just the beginning of learning about different uses 
for drugs and devices, for different indications, conditions, 
and populations. Treatment in the real world also brings out 
additional information on safety and efficacy, and ensuring 
that this knowledge is shared widely among providers, patients, 
and researchers is critical.
    As a result, the ability of patients, physicians, and 
developers to communicate effectively is so important for the 
future of cures in this country. Unfortunately, many of the 
witnesses and participants we have had before us since the 
Cures Initiative began have raised concerns regarding barriers 
to communication and evidence development. This hearing is a 
direct result of the feedback we have received from patient 
groups and other interested parties.
    As today's witnesses will discuss, efforts to limit off 
label use among the provider community, limitations on 
communication found under HIPAA, and the Physician's Sunshine 
Act are just a few of the barriers to 21st century cures that 
have been raised with us over the past few months. It is my 
hope that this hearing allows the members an opportunity to 
consider those potential barriers and the role they play in our 
healthcare system.
    With that thought in mind, I would like to thank all of our 
witnesses for being here today, and I will yield the balance of 
my time to Dr. Burgess, vice chairman of the subcommittee.
    [The prepared statement of Mr. Pitts follows:]

               Prepared statement of Hon. Joseph R. Pitts

    The Subcommittee will come to order.
    The Chair will recognize himself for an opening statement.
    In this, the fourth hearing of our 21st Century Cures 
Initiative, we are examining continued evidence development and 
communication of information regarding treatments and cures in 
the real world setting.
    Discovery of the risks and benefits of a drug or treatment 
does not end with FDA approval or clearance. It is often just 
the beginning of learning about different uses for drugs and 
devices, for different indications, conditions, and 
populations.
    Treatment in the real world also brings out additional 
information on safety and efficacy, and ensuring that this 
knowledge is shared widely among providers, patients, and 
researchers is critical.
    In such ways, the ability of patients, physicians, and 
developers to communicate effectively is so important for the 
future of cures in this country. Unfortunately, many of the 
witnesses and participants we have had before us since the 
cures initiative began have raised concern regarding barriers 
to communication and evidence development.
    As our witnesses will discuss, efforts to limit off-label 
use among the provider community, limitations on communication 
found under HIPAA, and the Physician Sunshine Act are just a 
few of the barriers to 21st Century Cures that have been raised 
with us over the past few months. It is my hope that this 
hearing allows the members an opportunity to consider these 
potential barriers and the role they play in our health care 
system.
    The importance of today's hearing and the reason for our 
calling it is really a direct result of the feedback we have 
received from patient groups and other interested parties.
    With that though in mind, I would like to thank all of our 
witnesses for being here today, and I yield the balance of my 
time to Rep. ----------------------------------.

    Mr. Burgess. Thank you, Mr. Chairman. I too want to welcome 
our witnesses. I certainly look forward to hearing from them 
today.
    I appreciate the continued series of hearings on the 21st 
Century Cures Initiatives. Certainly looking forward today to 
exploring the role that healthcare providers, physicians, can 
have in increasing communications between patients, 
researchers, and those who innovate. Different uses for 
therapies are constantly being discovered through information 
highways, including social networks, patient advocacy groups, 
and physicians sharing information.
    There is no doubt that technology and the ability to 
communicate easily with people all around the world will change 
how we conduct research, how clinical trials are managed, and 
how the post-market surveillance of technologies is handled.
    We must recognize this fact and be open to rethinking the 
traditional means of how we have engaged with our patients. We 
must also rethink our expectations of the ease with which 
patients may engage with each other. The fact of the matter is 
if I get on a plane with my iPad, I have got the New England 
Journal, I have got the Journal of the American Medical 
Association, and I have got the most current Journal of 
Obstetrics and Gynecology with me. And it is simply a matter of 
opening it and reading while on the plane. The ability to keep 
up with rapidly-changing and evolving fields is unlike anything 
anyone has ever had in the past.
    So this is the world in which we live today, and we need to 
be open to realizing the benefits that can be drawn from this 
fact. And also recognize that while we are exchanging 
information, patient advocacy groups are likewise engaged.
    So we certainly look forward to a lively discussion with 
the panel today. Mr. Chairman, I will yield back the time.
    Mr. Pitts. Anyone on our side seek time? Vice chair, Ms. 
Blackburn?
    Mrs. Blackburn. Thank you so much, Mr. Chairman. And to our 
panel, I want to welcome each of you. There is so much that is 
going on in the field of healthcare informatics, and Dr. 
Burgess just touched a little bit on that, and also medical 
devices. We are going to hear from Edwards Life Sciences about 
a heart valve which was approved in 41 countries before it was 
approved here in the U.S.
    And this is something that is unacceptable when you look at 
the length of time that it takes to get these medical devices 
through the FDA's process. In Memphis, Tennessee, my home 
State, one in four jobs is dependent on medical devices. And 
when you look at what is happening in the Nashville area with 
healthcare informatics, you realize the importance and the 
increasing importance of that as an economic development sector 
to our State.
    I think it is imperative that we provide a 21st century 
regulatory framework for 21st century technology and a 
framework that is going to encourage innovation while providing 
safe, effective, and new therapies. And with that, I yield back 
my time.
    Mr. Pitts. The chair thanks the gentlelady. Now filling in 
for the ranking member, Mr. Pallone, Mr. Green of Texas, 5 
minutes for opening statement.
    Mr. Green. Thank you, Mr. Chairman. And thank you and the 
ranking member, who will be here shortly, on this continuing 
series of hearings on the 21st Century Cures. This is really 
what our Health Subcommittee should be about, how we can help. 
And following my colleague from Tennessee, although I did not 
know that many jobs in Memphis were for medical. I thought it 
was just barbecue or Graceland.
    Mrs. Blackburn. If the gentleman will yield----
    Mr. Green. Briefly.
    Mrs. Blackburn [continuing]. It is because of the barbecue 
that we need the medical----
    Mr. Green. Well, as you know, there is a difference between 
Tennessee and Texas barbecue. We like----
    Mrs. Blackburn. I would ask the gentleman to yield again on 
that. There would not be a Texas if there were not Tennessee--
--
    Mr. Green. Well, and I cannot disagree with that because, 
frankly, we got all the rebels from Tennessee and helped us win 
independence in Texas. But with that, I am going to yield the 
balance of my time to my colleague, Congressman DeGette from 
Colorado.
    Ms. DeGette. Thank goodness. Mr. Chairman, I really want to 
thank you for holding this next hearing in this series on the 
21st Century Cures. I have got to say I was around my district 
all weekend, and everybody I talked to from the Jefferson 
County Economic Development Team to the telephone town hall 
meeting I had last night, to the OFA people. Everybody was 
excited to hear about this bipartisan effort that we are 
having, and I am excited, too.
    Throughout all of the previous hearings and roundtables 
that we have had on all of these topics, we have already 
learned a tremendous amount about what role Congress should 
play in helping to further advance and accelerate treatment and 
cures.
    Today the witnesses will talk about examining barriers to 
ongoing evidence development and communication. The potential 
areas for discussion are far ranging, to say the least, but I 
am looking forward to hearing some specifics from the witnesses 
on the potential benefits of enhanced data collection and 
improved maintenance and secured sharing of data and 
information.
    These types of evidence development and communication can 
and do play essential roles in the drug and device development 
and approval processes, as well as in reimbursement 
determinations. For example, how can we take advantage of data 
and information to more effectively identify patients for 
clinical trials that are relevant to their individual disease 
or condition? How can we harness the data and information 
collected during clinical trials? What about information after 
the drug or device is introduced into the market? And how do we 
effectively utilize this information while maintaining a high 
standard of privacy protections?
    On the reimbursement side, how is Medicare's coverage with 
the evidence development process currently being used? And how 
can we improve these processes to be clear?
    Just to talk for a minute about some of the things that are 
going on in terms of evidence sharing and data, Mr. Burgess 
talked about taking his iPad on the airplane. And I just 
literally got off the airplane from Denver where I was reading 
this article from The New Yorker this week. Maybe some of you 
have seen it. It is about a family who has a child with a very, 
very, very, very rare genetic disorder: NGLY1. And they finally 
got it diagnosed, but they did not think anybody else had it 
until the dad, who is a computer professor at the University of 
Utah, wrote a blog which went viral, and everybody read about 
it.
    And the upshot is that they have now identified patients 
with this genetic disorder around the world. They have all met. 
They have put together a research consortium. They have people 
doing research and writing a paper to be published in a 
scientific journal. And they are on their way to try to figure 
out what they can do about this very, very rare defect.
    These patients did this on their own because they were 
sophisticated parents. So what I would like to know is what can 
we do to harness this in a much more systemic way so that these 
types of communications can occur effortlessly both within the 
United States and with our colleagues around the world. So all 
of these are important questions.
    I really look forward to hearing the testimony today and to 
learning about these topics. Thank you very much, and I yield 
back.
    Mr. Pitts. The chair thanks the gentlelady, and now 
recognizes the ranking member of the full committee, Mr. 
Waxman, 5 minutes for opening statement.

OPENING STATEMENT OF HON. HENRY A. WAXMAN, A REPRESENTATIVE IN 
             CONGRESS FROM THE STATE OF CALIFORNIA

    Mr. Waxman. Thank you very much, Mr. Chairman. Today we 
have an opportunity to learn more about several issues that 
were raised at our previous meetings on the 21st Century Cures 
Initiative. From the first roundtable discussion that kicked 
off the initiative, we heard that FDA and NIH are leaders in 
driving and using advances in molecular medicine and digital 
technology to help get new cures to patients more quickly. They 
have also made great strides in improving and streamlining 
procedures for conducting clinical trials and in reviewing 
innovative new drugs and medical devices.
    However, we also heard about impediments that stand in the 
way of researchers and companies making full use of these 
advances. While patient registries can facilitate enrollment in 
clinical trials and help researchers find new research avenues 
to pursue, many believe more could be done to encourage their 
development and use.
    Electronic health care records can help physicians and 
sponsors identify patients for clinical trials and evaluate the 
effects of drugs already on the market, but privacy concerns 
are limiting their use. And although FDA has shown an 
increasing willingness to accept data from smaller clinical 
trials, the more limited data generated to support FDA approval 
may not be adequate for coverage decisions by Medicare or 
private insurers. I look forward to hearing more about these 
barriers and what can be done to address them.
    We should remember, though, that we have a review and 
approval system that is already working quite well. It has led 
to enormous breakthroughs and coverage of cutting-edge drugs 
and devices. FDA reviews and approves drugs faster than any 
other regulatory agency in the world. NIH and FDA are world 
leaders in clinical trial design and in integrating the newest 
science into their policies and approaches while protecting the 
health of the patients. And Medicare has demonstrated 
flexibility in its national coverage determinations so that 
beneficiaries can access these new cures.
    I have a great interest in fostering greater access to 
innovative drugs, devices, and health services. But I also know 
that access to new, innovative medicine alone will not increase 
the quality and outcomes patients experience in our healthcare 
system. Incentives must be in place for providers to furnish 
high quality care to the right patient at the right time in the 
right setting of care.
    The Affordable Care Act was a major advancement in meeting 
these challenges, but we still have work to do. In particular, 
we should enact the delivery reforms contained in our 
bipartisan SGR legislation. We can make another great stride 
forward if we can send this legislation to the President's desk 
before the end of this year.
    I have a little time left, and I would be pleased--anybody 
on our side want it?
    If not, I yield back the time, and let us hear from the 
witnesses.
    Mr. Pitts. The chair thanks the gentleman. As always, 
members' opening written statements will be made a part of the 
record.
    We have one panel today with five witnesses. I will 
introduce them in the order of them making their presentations. 
First, Dr. Josh Rising, Director of Medical Devices, the Pew 
Charitable Trust; Dr. Louis Jacques, Senior Vice President, 
Chief Clinical Officer of ADVI; Mr. Michael Mussallem, Chairman 
and Chief Executive Officer of Edwards Life Sciences 
Corporation; Dr. Gregory Schimizzi, Co-founder, Carolina 
Arthritis Associates, P.A.; and Ms. Mary Grealy, President, 
Healthcare Leadership Council.
    Thank you each for coming. Your written testimony will be 
placed in the record. You will each be given 5 minutes to 
summarize your testimony. And at this time we will recognize 
Dr. Rising 5 minutes for his opening statement.

 STATEMENTS OF JOSH RISING, DIRECTOR, MEDICAL DEVICES, THE PEW 
  CHARITABLE TRUSTS; LOUIS JACQUES, SENIOR VICE PRESIDENT AND 
CHIEF CLINICAL OFICER, ADVI; MICHAEL A. MUSSALLEM, CHAIRMAN AND 
   CEO, EDWARDS LIFESCIENCES; GREGORY SCHIMIZZI, CO-FOUNDER, 
    CAROLINA ARTHRITIS ASSOCIATES; MARY GREALY, PRESIDENT, 
                 HEALTHCARE LEADERSHIP COUNCIL

                    STATEMENT OF JOSH RISING

    Dr. Rising. Chairman Pitts, Ranking Member Pallone, members 
of the committee, I thank you for the opportunity to provide 
testimony. My name is Josh Rising. I am Physician Director of 
Medical Devices at the Pew Charitable Trusts.
    We have an exciting opportunity today to talk about the 
future of healthcare, a future where we can harness electronic 
data to improve patient care. Advances in technology offer 
potential for new approaches to develop medical evidence 
through a continuous cycle that begins before a product is 
approved and continues as the product is used by patients.
    As we move toward this total life cycle approach, we must 
consider two important issues. First, we know that clinical 
trials are the largest contributor to the cost and length of 
product development. We need to use new approaches to decrease 
their length and cost without doing away with these trials and 
the critical data they provide. Second, we must have the tools 
necessary to quickly and efficiently identify problems with 
approved drugs and medical devices, and to assess their 
performance in real world settings that can be different from 
clinical trials.
    We are at a key turning point. Electronic health records 
today collect more data on patient outcomes than we have ever 
had, but we are failing to realize that potential. One 
important innovation to harness data from electronic health 
records is the registry, large databases that collect 
information on groups of patients treated for a particular 
medical condition.
    Now, imagine if we could conduct clinical trials for a 
tenth of the current cost. This is precisely what physicians in 
Sweden recently did using an existing registry. They studied 
heart attack prevention in more than 7,000 patients, comparing 
two different procedures. The data were drawn from electronic 
health records, and the trial cost only $300,000, or roughly 
$50 per patient. Conducting such a study outside of a registry 
in the United States would cost hundreds of millions of 
dollars, if not more. We can do this in the United States, too, 
but only if we fix the lack of interoperability among 
electronic health records and streamline certain electronic 
administrative processes.
    Second, just as important as ensuring prompt access to new 
cures is the ability to detect problems with drugs and medical 
devices on the market and assess their performance in real 
world conditions. Here, too, registries can help. For example, 
an Australian registry of artificial joints found that one type 
of Metal-on-Metal Hip failed at a rate more than two times 
higher than conventional hips, ultimately leading to a 
worldwide recall of the device. Detecting such problems earlier 
is vital for patient safety and could save our healthcare 
system vast sums.
    Pew will soon release a report on registries produced in 
partnership with the Blue Cross and Blue Shield Association and 
the Medical Device Safety Group and the EPINet. In this report, 
we recommend steps to deliver timely, actionable information 
from registries to all stakeholders, including the public.
    Now, there are other ways that electronic data can also 
improve patient care. One is better use of the brand new Unique 
Device Identifier, or UDI, System, which was created by FDA at 
the direction of Congress and will result in a unique number 
assigned to nearly all medical devices. If we now incorporate 
this number into insurance clams, we can use FDA's Sentinel 
System to assess device safety problems the same way we do for 
drugs. Incorporating UDI into claims will also provide payers, 
such as CMS, with the necessary data unavailable elsewhere, to 
evaluate outcomes for patients with implanted medical devices.
    Adding a UDI field to claims has generated support across 
healthcare, including from hospitals such as Geisinger and 
Mercy, health plans like Aetna, physician societies including 
the American College of Cardiology and the Society of Thoracic 
Surgeons, as well as patient and consumer organizations. 
Additionally, HHS Secretary Burwell articulated the benefits of 
adding UDI to claims du ring her Senate confirmation process.
    New mechanisms to collect data both prior to and after FDA 
approval can help facilitate faster clinical trials and ensure 
that any problems are promptly detected. Congress should work 
with the Administration to maximize the potential of these new 
data sources to ensure patient access to safe and effective 
medical devices.
    Thank you again for the opportunity to testify, and I 
welcome your questions.
    [The prepared statement of Dr. Rising follows:]
    
    [GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
    
    Mr. Pitts. The chair thanks the gentleman, and now 
recognizes Dr. Jacques 5 minutes for an opening statement.

                   STATEMENT OF LOUIS JACQUES

    Mr. Jacques. Chairman Pitts, Ranking Member Pallone, and 
members of the subcommittee, my name is Louis Jacques. From 
October 2009 through February 2014 I was the Director of the 
Coverage and Analysis Group at the Centers for Medicare and 
Medicaid Services. I was the Division Director in that group 
from June 2004 until 2009.
    We implemented coverage with evidence development and the 
FDA/CMS Parallel Review Pilot Initiative. We also revised CMS 
regulations pertaining to Medicare coverage and FDA-approved 
investigational device exemption clinical trials, and executed 
a memorandum of understanding between FDA and CMA.
    CMS experience over the past decade is illustrative of the 
challenges to the wide adoption of certain innovative 
technologies. One, there are innovative products and services 
that do not clearly fall within the statutory scope of the 
Medicaid program benefit. Two, the available evidence at the 
time of initial marketing may not clearly establish the 
clinical value of a new technology in the relevant beneficiary 
population. Three, historic coding paradigms can be 
uninformative to the extent that the insurer cannot identify 
the specific item or service for which it is paying. This blind 
buying creates reluctance among insurers and hampers the 
establishment of brand value for high performing technologies.
    I believe there are opportunities. External stakeholders 
have requested more opportunities for coverage with evidence 
development and FDA/CMS parallel review. While these programs 
were articulated in the early 2000s by a prior Administration, 
both are included in the 2012 White House National Bio Economy 
Blueprint.
    Since 2009, CED has essentially replaced non-coverage in 
final national coverage determinations, thereby furnishing 
Medicaid coverage when it would otherwise have not been 
available. By contrast, in the 5 years before 2009, almost half 
of all national coverage determinations ended with non-
coverage.
    Unfortunately, CMS' ability to furnish CED is limited. CMS 
initiates CED under ARC's statutory authority. CMS implements 
CED through the formal national coverage determination process. 
Due largely to staffing cuts the annual number of NCDs 
published has dropped from approximately 12 to 13 in fiscal 
years 2007 and 2008 to only five in 2012 and six in 2013. 
Current staffing is approximately half of 2007 levels.
    Under parallel review, both FDA and CMS maintain their 
separate standards. I have no reason to believe that either 
agency has toughened its process as a process of parallel 
review. While the structure of the pilot contemplates the 
possibility of a national coverage determination, parallel 
review does not inherently require that CMS undertake the NCD 
process. The content of the parallel review engagement depends 
on the product's development stage. Ideally, early discussions 
with CMS could result in more persuasive pivotal trial, 
evidence that leads local Medicare contractors to uniform 
coverage.
    Results to date are encouraging. One product received 
unanimous yes votes and positive comments at its recent FDA 
panel meeting, which the company credited to the discussions 
with both agencies that inform the design of the pivotal trial. 
CMS does not have sufficient staff to match FDA's bandwidth on 
potential parallel review candidates. Despite interest from 
device manufacturers, the parallel review pilot has been 
limited to only two products.
    In conclusion, CMS review of clinical trials serves three 
goals. First, it provides important financial support for 
innovation. Second, the sponsor can obtain CMS feedback on 
whether the initial trial design could persuasively inform a 
coverage decision. Third, CMS can inform the sponsor of 
existing coding or payment paradigms that may apply to the 
product.
    The current vehicles for coverage in clinical trials are 
unnecessarily siloed, preventing the publication of an 
integrated, comprehensive policy. I believe this could be fixed 
with small changes in statute. The definition of a local 
coverage determination could be revised to align LCD authority 
with the actual scope of local contractor claims processing 
responsibility, thereby enhancing transparency and 
predictability. As an alternative to non-coverage, some 
stakeholders have expressed interest in new payment paradigms 
for early stage devices with immature evidence bases.
    Acknowledging the challenges of the Federal administration 
budget, stable funding sources should be considered for these 
initiatives that are expected to produce downstream benefits. 
Their investment requires funding that is more predictable 
potentially from the Medicare Trust Fund itself.
    Thank you for the opportunity to share my thoughts, and I 
would be happy to answer any questions.
    [The prepared statement of Mr. Jacques follows:]
    
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    Mr. Pitts. The chair thanks the gentleman.
    Mr. Mussallem, you are recognized for 5 minutes.

               STATEMENT OF MICHAEL A. MUSSALLEM

    Mr. Mussallem. Yes. Thank you very much, Mr. Chairman 
Pitts, Ranking Member Pallone, Congresswoman DeGette, and 
members of the subcommittee. My name is Mike Mussallem. I am 
the chairman and CEO of Edwards Life Sciences. I am truly 
honored to join the other panelists today to discuss the path 
to revitalizing medical device innovation in the United States.
    I and the other employees of Edwards Life Sciences, from 
our engineers to our valve assemblers, share a passion for 
helping patients. I am privileged to lead a company that is the 
world leader, and has been for 50 years, in heart valve 
replacements.
    The reason I am here is that I am worried about innovation 
in the U.S. and that it is suffering from increasingly costly, 
cumbersome, and risk averse culture in our regulatory and 
payment systems. Our recent experience with a transformative 
therapy to heart valve replacement patients gives us a unique 
perspective on the current climate.
    In short, Edwards Technology allows a heart team to deliver 
a collapsible prosthetic valve through a catheter into the body 
to avoid cracking the chest, stopping the heart, and avoid the 
long and painful recovery that goes along with that open heart 
surgery.
    This has become the most extensively studied heart valve 
ever, including an unprecedented four New England Journal of 
Medicine articles that demonstrated a triple win, which is a 
substantial and sustainable clinical effect, cost 
effectiveness, and extraordinary quality of life improvement.
    We appreciated a productive relationship with Dr. Jeff 
Shuren in FDA, as well as Dr. Patrick Conway and his colleagues 
at CMS, whose approach ensured that there was a balanced and 
reasonable process for this transformative therapy.
    Also in a remarkable effort of groundbreaking collaboration 
between medical societies, regulators, and other stakeholders, 
we built a comprehensive clinical evidence and quality 
measurement tool for this therapy called the TBT registry.
    But there is room for improvement. We all know the path to 
approval and reimbursement is not easy, and it should not be. 
Yet the U.S. approval of this American technology trailed 
Europe by 4 years. We are pleased that the FDA leadership 
viewed this delay as a catalyst to improve, and we see several 
opportunities to remove barriers. I am going to focus on three.
    Number one, evidence development mechanisms can be improved 
to reduce cost and delay. FDA had recently proposed a number of 
improvements to the pre-market clinical trial process and the 
post-market surveillance process that hold the promise. And 
these have been discussed at this committee. In my view, when 
registries are done right, they can yield extremely useful 
information about patients' outcome and device benefits.
    However, the clinical and scientific benefits of registries 
must be balanced with a potentially significant cost burden, 
complexity, and potential misuse of that data. In our case, 
many physicians told us it takes longer to fill out the 300 
fields in the TBT registry than it does to perform the 
procedure itself.
    Number two, reimbursement incentives need to be aligned 
with promoting innovation. Efforts to curb healthcare spending 
could have the unintentional consequence of slowing down 
innovation in our cost-cutting frenzy. It is imperative to 
recognize that medical device innovations become more effective 
and more efficient with time and with experience. We need a 
system that does not shut the door to reimbursement on day one.
    In select cases, coverage with evidence development can be 
a tool that allows promising technologies to reach patients 
sooner while developing evidence to support lasting 
reimbursement. And finally, FDA's vision to improve the 
regulatory process must be accelerated. Dr. Shuren and his team 
have outlined strategic priorities that strike the right 
balance between pre-market and post-market data collection and 
improving customer service.
    We know FDA is a complex bureaucracy to manage, and our 
leaders need a mandate to change more quickly. Congress could 
encourage FDA by providing additional support to expedite these 
changes and give them room to innovate.
    And finally, no discussion about medical technology is 
complete without understanding the true impact that they have 
on patients. And we meet a lot of patients. To mention one, 
Lester Tenney, a true American hero, part of our Greatest 
Generation, survivor of the Bataan Death March, and a Japan 
POW, had long sought an apology from the Japanese government on 
behalf of Federal soldiers. Unfortunately, just as this apology 
was agreed upon, he was diagnosed with disabling and inoperable 
aortic stenosis. He would not survive long, let alone long 
enough to make this trip.
    The good news is that Lester received an Edwards trans-
catheter heart valve, was able to travel to Japan, get the 
apology. This would not have been possible even 5 years 
earlier. And he remains vital to this day and dedicated to 
helping veterans. Lester and tens of thousands of other 
patients we have had the opportunity remind us every day that 
our work is personal. It impacts people individually.
    Thank you for the opportunity to testify today.
    [The prepared statement of Mr. Mussallem follows:]
   
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    Mr. Pitts. The chair thanks the gentleman.
    Dr. Schimizzi, you are recognized for 5 minutes for an 
opening statement.

               STATEMENT OF GREGORY A. SCHIMIZZI

    Dr. Schimizzi. Chairman Pitts, Ranking Member Pallone, 
members of the subcommittee, and honored guests, it is a 
distinct honor to be here today and testify before you. My name 
is Gregory Schimizzi, and I am testifying before you as a 
Member of the Board of Directors and past President of the 
Coalition of State Rheumatology Organizations, or CSRO. And I 
am a private practice Rheumatologist at the Carolina Arthritis 
Associates in Wilmington, North Carolina.
    The CSRO appreciates the opportunity to share our views 
related to barriers to ongoing evidence development and 
communications transparency. Specifically, I will focus on 
situations in which valid communications pathways are being 
hampered by outdated practices of the Food and Drug 
Administration, or FDA, as well as touch upon some unintended 
consequence of the Sunshine Act, or open payments, as 
implemented by the Centers for Medicare and Medicaid Services, 
or CMS.
    The FDA does not allow pharmaceutical companies to actively 
distribute key clinical information even if it is related to 
the on-label indicated, unless it is explicitly referenced in 
the package insert of that product. By limiting the sharing of 
information, physicians are hampered in their ability to gain 
all of the firm scientific rationale and medical evidence 
needed to treat patients.
    So that clinicians may be better informed, the CSRO urges 
the FDA to develop standards for qualifying real world data 
through a public process, to expand the current process of 
review of materials beyond what is included in the package 
insert, to also cover other key data, such as sub-population, 
pharmaco-economic, or comparative cost data, and to ensure a 
timely review process for such information.
    As part of the Affordable Care Act, Congress required the 
Administration to set up a process by which transfers of value 
from certain covered entities, primarily manufacturers of drugs 
and devices to physicians, would be reportable. Such reportable 
information would then be made publicly available. The overall 
goal of this transparency is to make particular potential 
financial conflicts of interest more transparent.
    However, there are considerable problems with the current 
implementation of open payments, including the lack of guidance 
and clarity regarding the physician registration process, as 
well as the review of dispute process lacking necessary 
protection for physicians.
    Finally, a recent CMS-proposed rule related to open 
payments would severely hamper the flow of information. 
Therefore, the CSRO respectfully requests that CMS provide 
additional provider-specific guidance for the registration 
process and adopt policies that allow for flexibility of 
enrollment requirements so that physicians struggling to enroll 
remain able to participate in a meaningful manner, ensure an 
impartial process for disputing the accuracy of financial 
information intended for public disclosure, take steps to 
enhance the fairness and accuracy of the program by ensuring 
that healthcare providers have access to meaningful mechanism 
for limiting the distribution of disputed information, and 
reconsider its proposal to eliminate the continuing medical 
education exemption, and instead appropriately expand the list 
of certified CME accrediting or issuing agencies beyond the 
five currently cited in regulation.
    As I hope I have outlined today, current practices at both 
the FDA and CMS may be inappropriately hampering the exchange 
of information, making it difficult for physicians to receive 
the information they need to make valuable treatment decisions.
    For the FDA, I hope that Congress will examine ways to 
allow for more proactive changes among clinicians with 
appropriate safeguards to ensure that such information is 
truthful and not misleading. For CMS, I hope that Congress can 
urge strategic plan programmatic changes to make the 
transparency process accurate and appropriately descriptive of 
the financial relationships among the various entities.
    Thank you once again for allowing me to speak today and to 
consider my comments today as well as the other information 
captured in my written statement. The Coalition of State 
Rheumatology Organizations looks forward to working with the 
committee to address these issues. I look forward to your 
questions. Thank you very much.
    [The prepared statement of Dr. Schimizzi follows:]
   
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    Mr. Pitts. The chair thanks the gentleman.
    And now, Ms. Grealy, you are recognized for 5 minutes for 
an opening statement.

                    STATEMENT OF MARY GREALY

    Ms. Grealy. Mr. Chairman and members of the subcommittee, 
thank you for the opportunity to testify this afternoon. And 
thank you as well for the attention you're bringing to the 
future of American healthcare, and the ability of the 
healthcare system to develop, communicate, and utilize the data 
that can lead to 21st century cures.
    I am here today representing the Healthcare Leadership 
Council, a coalition of leaders from all sectors of American 
healthcare. I am very proud that our membership includes 
innovators, like Mr. Mussallem, also on today's witness panel.
    Our members share this committee's goals for a healthcare 
system that is affordable, sustainable, and of the highest 
attainable quality, and that is also on path toward curing the 
diseases and illnesses that have cost us far too much both in 
lives and resources.
    Each year, those involved in all aspects of healthcare 
generate literally trillions of decisions, communications, 
interventions, consultations, treatments, therapies, and 
clinical trials. The key to achieving progress lies in 
harnessing this massive amount of information and setting 
policies and practices in place to productively share and to 
use this data.
    HLC members have been engaged in this challenge for some 
time both as individual innovative companies and collectively. 
What I share with you today is our broad-based, multi-sector 
perspective on how we can create an environment in which data 
can be used to strengthen the entirety of the healthcare 
continuum.
    There are three areas where I will focus my comments today. 
One, the role of the HIPAA privacy law; two, the need for 
Federal data policies that enhance access to information to 
enable health system improvements and accelerated medical 
research; and three, the potential impact of the new Sunshine 
Act on the physician industry collaborations that are critical 
engines of healthcare advancement.
    On the first point, the HIPAA privacy and security laws are 
generally serving patients in the healthcare system well, and 
should continue to be the guiding rule regarding the 
appropriate and effective use of patient health data. There are 
certain aspects of HIPAA, however, that warrant continuing 
review and discussion.
    We need to keep in mind that HIPAA was created at a time in 
which policymakers were not thinking about the knowledge that 
could be gained by accessing data residing in large databases 
and the technological ability to process that data very 
rapidly. It may be necessary to adjust the authorization 
components of HIPAA to ensure that data can be used effectively 
for research.
    Also, in order to transmit data and collaborate in its use, 
we need to review the utility of having 50 separate sets of 
State privacy laws and regulations instead of a single national 
standard.
    On the issue of Federal data policy, Healthcare Leadership 
Council members have developed a set of consensus multi-sector 
principles on data policy that I have submitted for the record. 
One of these key principles is our belief that access to 
Federal health data should no longer be denied to entities 
perceived to have a commercial interest. The profit status of 
an organization should not take precedence over the larger 
question of how best to conquer disease and improve population 
health.
    Any standard that restricts access to critical, federally-
held health data is, in fact, detrimental to our shared goals 
for medical and human progress. We must put the benefit to 
patients first.
    Finally, we believe strongly that Congress must diligently 
monitor the impact of the Physician Payment Sunshine Act. This 
is not a criticism of transparency, which our member companies 
practice and HLC strongly endorses. We are concerned, though, 
about the transparency without context. We are concerned that 
physicians may feel stigmatized by the Federal reporting of 
their interactions with manufacturers in a way that does not 
communicate the patient benefits of such collaborations.
    Some of our member companies are already witnessing 
physicians withdrawing from collaborative activities, which can 
have a devastating impact not only on innovation, but also on 
product efficacy and safety. Congress should monitor the 
implementation of this law to ensure that both transparency and 
innovation are fully achieved.
    Mr. Chairman, thank you again for the opportunity to 
testify today. We believe that this committee's bipartisan 
vision for 21st century cures is an achievable reality, one 
that can be accelerated by creating a pathway for the 
productive use of data that we already possess.
    Thank you, and I will be happy to answer any questions.
    [The prepared statement of Ms. Grealy follows:]
    
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    [The attachments to Ms. Grealy's testimony have been 
retained in committee files and can be found at http://
docs.house.gov/meetings/IF/IF14/20140722/102524/HHRG-113-IF14-
Wstate-GrealyM-20140722.pdf.]
    Mr. Pitts. The chair thanks the gentlelady. Thanks to all 
the witnesses for their testimony. I will begin questioning and 
recognize myself for 5 minutes for that purpose. I will start 
with you, Dr. Schimizzi.
    Different uses for FDA-approved drugs and devices are 
constantly being discovered, many times for treatment of 
different conditions and diseases or for different populations. 
Manufacturers of these products have access to robust data sets 
and information that is not always limited to the specific 
indications listed in their package inserts.
    Why is it important that we responsibly allow providers to 
have access to such information to ensure that the most 
appropriate treatment options are being considered?
    Dr. Schmizzi. Well, thank you, Mr. Chairman. In 
rheumatology we see many patients with rare diseases and 
unusual autoimmune problems. And we also see patients who are 
referred to us by other specialists for autoimmune problems in 
their specialty that they do not know how to handle, so they 
send them to us.
    In our armamentarium of medications, we have an array of 
medications that work very well. Some of them are of low 
toxicity, and some of them are of high toxicity. In the event 
of a new agent being brought to the United States medical arena 
having a high safety profile, but lack an indication for an 
orphan disease or a critically important problem in another 
organ system, like the eye, for example, use of those 
medications would be miraculous and have a high margin of 
safety if we had access to information. I am just using the eye 
as an example. There are other instances as well. Primary 
muscle disease is another one.
    Medications are available, but the indications are not 
there, and they probably never will be because the numbers of 
patients who have these diseases is so small, it would take 
many years to discover that the indications were there and 
millions of dollars, perhaps tens of millions of dollars, to 
identify that.
    So if we had access to information that these new 
medications might be effective in certain other small diseases 
that may have been gleaned from the data that was derived from 
the direct clinical trials, then that would be extremely 
helpful to us and help guide us in a direction that would 
increase efficacy, and increase safety, and maybe even decrease 
cost and poor outcomes.
    Mr. Pitts. Do you believe that the current restrictions on 
off label communications are limiting healthcare professionals' 
ability to provide the most appropriate treatment to patients? 
And if so, what needs to happen?
    Dr. Schmizzi. What was the last part?
    Mr. Pitts. If so, what needs to happen?
    Dr. Schmizzi. Yes, I do believe that the limitation of 
exchange of information is hampering the delivery of healthcare 
to some of these patients, especially in my sub-specialty. What 
needs to happen is that we need to have access to information 
that is locked up in vaults in pharmaceutical companies, locked 
up in data sets in study information.
    For instance, here is a great example that I can spread to 
rheumatology. There is a great drug that came out many years 
ago to prevent ulcers and to treat ulcer disease and 
esophagitis called Prilosec. The generic name was omeprazole. 
Prilosec was a mixture of two different mirror image molecules, 
D-enantiomer and an L-enantiomer. It is like a right hand and a 
left hand.
    Well, it came to light that one of the enantiomers was much 
more effective at treating ulcer disease and esophagitis, so 
out came esomeprazole, or Nexium, which has proven to be more 
effective.
    What if there were medications on the market right now that 
we have that could treat diseases but have side effects, and 
yet if we isolated the D-enantiomer and the L-enantiomer, we 
would identify which one was effective and which one caused the 
problems. I submit to you that there are drugs in our 
compendium right now that have D and L isomers, and the data 
sets are probably available in the vaults of pharmaceutical 
manufacturers that show the D isomer is more effective than the 
L isomer. The L isomer has more complications than the D 
isomer. So that would be a dramatic improvement.
    So such data sets are locked up. We do not have access to 
them, and I do not know that we ever will.
    Mr. Pitts. Quickly, Ms. Grealy, you mentioned HIPAA. What 
kind of changes should we consider to HIPAA to ensure that big 
data can be used effectively for research purposes while still 
protecting patient privacy?
    Ms. Grealy. Well, when the HIPAA law was originally passed, 
there was an exemption for healthcare operations, and that 
included the treatment and payment for patients. But sort of 
outside that scope was research activity.
    So I think a very simple approach would be let's include 
healthcare research as if it is a natural part of healthcare 
operations.
    There are probably several other recommendations that we 
could make, but I think the key here is to make sure that we 
have an appropriate balance between protecting patient 
information, and we believe very strongly in that. We also do 
not want to erect barriers to having access to that data.
    I think a big part of this is having informed consumers, 
educated patients, and especially as we are seeing patients 
engage more and more in the management of their own healthcare.
    Mr. Pitts. The chair thanks the gentlelady, and my time has 
expired. The chair recognizes the ranking member, Mr. Pallone, 
5 minutes for questions.
    Mr. Pallone. I wanted to start with Dr. Jacques, and then 
if I have time, ask Dr. Rising a question. But, Dr. Jacques, I 
wanted to better understand what you mean when you talk about 
the confusion created by Medicare's vague authority and lack of 
administrative agility in Medicare coverage and payment 
policies for new innovation technologies.
    Could you briefly describe the statutory limitations that 
apply to Medicare coverage determinations, both as they relate 
to coverage with evidence development and local coverage 
determinations? And what are your recommendations for how to 
streamline these authorities? And then maybe how does this 
existing authority impact the decision making framework for CED 
study questions, and what data is needed to trigger and end the 
CED study?
    Mr. Jacques. The reasonable and necessary standard, which 
is essentially the coverage standard for Medicaid, those 
provisions are 1862(a)(1) of the Social Security Act, which is 
then followed by subsections (a) through (p) that parse things 
out for prevention hospice and things along those lines.
    CED itself is not defined in the Social Security Act, so 
CMS has had to rely on the Agency for Healthcare Research and 
Qualities Research Authority under Section 1862(a)(1)(E) of the 
Act. Thus ARC has to approve every CED decision.
    While the scope of a national coverage determination is 
described broadly in statute as a decision under Title 18, 
local coverage determinations are defined in the Act only as 
decisions under 1862(a)(1)(A). Thus, an LCD could not implement 
coverage with evidence development. So even if there were an 
item or service that is only furnished within one contractor 
region of the entire country, a national decision would be 
required to implement CED.
    I have been told by various stakeholders that CED could be 
approached more eagerly if it were not tied to the formal NCD 
process. The current framework forces CMS to apply the CED 
requirement to all beneficiaries receiving the item or service 
in question, regardless of where they live. This is a 
particular challenge for beneficiaries who live in the remote 
parts of the country where clinical studies do not normally 
happen and clinical trial enrollment is, frankly, unrealistic 
for many.
    A more agile CED paradigm would permit CED to occur in 
parallel with other forms of coverage rather than requiring 
everyone to fit through the same door.
    Mr. Pallone. Now, you also highlight a rapid decline in the 
number of national coverage determinations in the last few 
years. How has the lack of staff resources within CMS impacted 
that decline, and what, if any, impact has this had on coverage 
with evidence development?
    Mr. Jacques. I believe that staff reductions are the 
largest single cause of the decline in number of national 
coverage determinations. And the impact of this decline is 
broader than CED because it impacts the Agency's ability to 
respond to other requests for coverage.
    CED itself generally requires more internal staff work to 
develop, and it creates an ongoing need after the publication 
of the final decision to interact with sponsors who might want 
to conduct clinical trials. By my own estimate, it takes about 
three times as much internal effort for CMS staff to do CED 
than it does to simply say yes or no.
    Mr. Pallone. All right. Thank you. And I am going to try to 
get this in. Dr. Rising, you note in your testimony that the 
taste trial conducted in Europe on heart attacks cost a tiny 
fraction, perhaps one-one hundredth of what it would cost in 
the U.S., because it was able to make use of patient 
registries. However, we also heard in Mr. Mussallem's written 
testimony that registries can be very expensive to set up and 
maintain, and threshold questions must be answered to determine 
when and how registries should be used for post-market data 
collection.
    Now, I am familiar with registries from the law creating 
the 9/11 Health Program. It included a provision to authorize a 
registry of people who were exposed to toxic dust from the 
attack on the World Trade Center on 9/11. But I do not know 
much about registries for assessing medical products.
    Can you explain how these registries work, and can you 
describe what source of impediments to the use exist, including 
why they may be harder, expensive to set up and maintain. And I 
would like to know your thoughts and what can be done to 
facilitate their use.
    Dr. Rising. Sure.
    Mr. Pallone. You do not have a lot of time to do it.
    Dr. Rising. I will in 30 seconds.
    Mr. Pallone. OK.
    Dr. Rising. So medical product registries, kind of like the 
9/11 responder registry, will follow a group of patients with 
one exposure for a period of time. So, for example, we heard a 
little bit from Mr. Mussallem about their trans-catheter valve 
registry, which follows patients who have gotten a valve for a 
period of time in order to assess their long-term outcomes.
    Now, while registries can be a tremendous source of 
information on the post-market performance of devices, there 
are some barriers to setting them up. And one of the biggest 
barriers is the lack of interoperability between systems. So, 
for example, clinical staff need to enter data in the TBT 
registry and then enter a lot of the same data again in 
electronic health records. So this kind of added burden on the 
staff is one of the biggest drivers for why registries are 
currently inefficient in the United States.
    Now, in addition to these post-market benefits, registries 
can have tremendous benefits for innovation as well. One of the 
other benefits that we have seen for the trans-catheter valve 
registry that Dr. Shuren talked about at the first hearing was 
that data from the registry was used to expand an indication 
for the device.
    So if we are able to take some steps in this country 
forward for registries, we should be able to see significant 
benefits both on the safety side and then also on the 
innovation side of things.
    Mr. Pallone. All right. Thanks a lot. Thank you, Mr. 
Chairman.
    Mr. Pitts. The chair thanks the gentleman, and now 
recognizes the vice chairman of the full committee, Ms. 
Blackburn, 5 minutes for questions.
    Mrs. Blackburn. Thank you, Mr. Chairman. I have got just a 
couple of questions that I want to go to, and again, I thank 
you all for participating with us and working with us.
    There is a lot of bipartisan agreement on this. You have 
heard different members of the committee speak to that, finding 
a pathway forward so that we deal with the regulatory 
framework, provide some certainty, and speed up the process by 
which innovation and cures are going to get to our patients is 
a shared goal. And so, we thank you for that.
    Mr. Mussallem, let us go back to the topic of the registry. 
We have talked a little bit about that, and you have all talked 
about basically the data and the value of the data that can be 
found within those registries and the benefit to our--to 
patients from being able to utilize the data in those 
registries.
    So let us talk a little bit about risks that are there for 
the patients or cost that is there. And can you give me just a 
little bit of an articulation looking at the other side of this 
with risk and cost, both the actual dollar cost, or, as Ms. 
Grealy mentioned, the privacy, some of the privacy concerns?
    Mr. Mussallem. Yes, thank you very much, Member Blackburn. 
And I applaud the bipartisan cooperation toward this shared 
goal. Registries can be a powerful tool, and we by and large 
think they could be very appropriately used. My only caution 
that I mentioned in my testimony is that there are some cases 
where technologies and/or therapies are well enough known that 
can establish a safety and effectiveness standard without going 
through that sort of process.
    In the case of the TBT registry, in particular, that I 
mentioned, the large group of stakeholders ended up generating 
this long list of items to be collected. I mentioned 300 data 
fields. Maybe when a technology is brand new and unknown you 
want to learn an awful lot about it. The problem is that 
becomes quite costly. And at some point, it gets too expensive 
to maintain.
    Ideally you would have a registry that could be whittled 
down to those things that are really most critical that you 
would like to measure, and there may be a way to populate it 
with electronic data that is already being generated, such that 
a registry could be a very cost-effective tool.
    In the case of the TBT registry, it literally cost seven 
figures plus per year for that total cost. That is shared by a 
lot of constituents, including manufacturers. But a lot of the 
burden rests on hospitals. They have a burden where they 
actually pay a fee every year and additionally have to put on 
dedicated staff just to fill out those fields. So not something 
to be taken lightly.
    Mrs. Blackburn. If you had to give us a list of guiding 
principles as we look at a framework for developing some of the 
registries, what are those three or four principles that you 
would articulate?
    Mr. Mussallem. I think it is most important to have a clear 
risk benefit analysis and also have clear goals set out by the 
registry. There should be a set of rules around the registry 
and some governance guidelines around it.
    Dr. Rising spoke to the work that Pew Foundation has done 
in this area. It is actually very thoughtful, done with a broad 
group of stakeholders about the value of registries. And I 
think that is not a bad guidepost.
    Mrs. Blackburn. OK. And let me ask you this. Do you 
envision any of these registries moving to the point where the 
patient could populate some of those cells and fields 
themselves?
    Mr. Mussallem. Ideally registries would not be expensive to 
populate, and any time that they could be filled out 
automatically in an electronic patient record or even, as you 
suggest, that a patient could do it themselves, this is 
important. I mean, some simple things. Is the patient alive? Is 
the patient going through a routine of exercise, or what is the 
patient's diet? All these things are very potentially powerful 
variables that could provide insight to the value of 
technologies.
    Mrs. Blackburn. Well, we would hope that anybody populating 
one of these with an app on an iPad would indeed be alive and 
not have their avatar doing it for them.
    Mr. Mussallem. Well said.
    Mrs. Blackburn. So, oK. Dr. Schimizzi, let me ask you just 
a couple of things on off label use. You had mentioned that, 
and I am intrigued by this. I think this is an area that it 
holds some promise. It is a legal practice, correct?
    Dr. Schmizzi. Yes.
    Mrs. Blackburn. OK. Do you consider it a best practice to 
inform a patient that a therapy that is being prescribed is off 
label?
    Dr. Schmizzi. I think that is best practice, yes, and I 
always do.
    Mrs. Blackburn. If informed doctors can legally prescribe 
off labeled patients who are also well informed, what would be 
the current barriers to that practice?
    Dr. Schmizzi. Well, the barrier is we need the information 
to pick which drug to use in a difficult situation. And that 
information is not always available to us.
    In the immune system, there are different cells that are at 
work, we know that a certain cell is active in one disease. And 
if you suppress that cell, we can suppress the disease or cure 
the disease. And that agent might be available for a cancer, 
but if we can transpose and use that in this patient, that 
would probably work. It would be very nice to have that 
information from the pharmaceutical company or manufacturer or 
innovator who developed that product that, yes, this is very, 
very likely an effective way to use this medicine, but we are 
never going to study it because they probably never will.
    Mrs. Blackburn. OK. I will yield back. I am over time. 
Thank you, Mr. Chairman.
    Mr. Pitts. The chair thanks the gentlelady, and now 
recognizes the ranking member of the full committee, Mr. 
Waxman, 5 minutes of questions.
    Mr. Waxman. Thank you, Mr. Chairman. The Affordable Care 
Act strengthened our movement away from a healthcare system 
that rewards providers for the volume of services they provide 
and toward a system that fosters and promotes high quality, 
high value care. The bipartisan SGR legislation is to make this 
committee's perspective a permanent change in the reimbursement 
rate for physicians under Medicare. That legislation passed out 
of this committee and the other committees' jurisdiction and 
then furthered that aim by incentivizing care delivery that is 
coordinated in alignment with consensus guidelines and best 
practices, and as efficient as it is appropriate.
    Dr. Jacques, in your testimony you speak of the broad 
national goals of Federal health agencies to improve public 
health and protect beneficiaries' access to products and 
services that demonstrate genuine benefit. You suggest that FDA 
approval for drugs and devices puts products on the shelves, 
but a prudent purchaser should not blindly pay for those 
products without regard to how useful or appropriate they are.
    Could you speak to this point: should Medicare really be 
paying for products that have no real value or paying more for 
products that have no added value? How do we balance a desire 
for rapid adoption of new technologies with ensuring that 
providers can be confident in the safety and benefit of new 
technologies as they are held accountable for their use?
    Mr. Jacques. New technologies remind me of teenagers, and 
both of my children are adults, so I survived raising teens. We 
see glimpses of their future promise, but we also recognize 
that not all of them are going to be good drivers as soon as we 
put them behind a wheel. As a society, we accept this and we 
balance their independence with our risks through a variety of 
mechanisms, whether it is a learner's permit or a prohibition 
on consuming alcohol or driving with friends.
    I believe in an ideal health technology system. We would 
have one where lessons are learned quickly and disseminated 
broadly. That depends on reliable collection, analysis, and 
publication of real world data that arise from using patients 
who are more typical than those studies in pivotal trials and 
who are treated in their communities by their own physicians.
    Mr. Waxman. OK. But what does that mean if a doctor wants 
to use a new technology, and he has to be confident that this 
is going to be safe and it is going to benefit the patient?
    Mr. Jacques. I am sorry if I was obtuse. What I was trying 
to convey is that the timing of calling the question is as 
critical as the content of the question itself. And especially 
for new technologies, the issue is being asked to call the 
question arguably prematurely to give it a thumb's up or a 
thumb's down when, in fact, what you actually have is an 
adolescent technology that has promise, but you do not really 
know the final answer.
    Mr. Waxman. And should we be paying for that through the 
Medicare system when we do not know whether it is going to add 
any value to what we already have available to us?
    Mr. Jacques. There are people who feel strongly on both 
sides of that question, sir.
    Mr. Waxman. So when we do we call the previous question to 
get their vote?
    Well, we hear a lot of concern raised from manufacturers on 
the cost of data collection to the healthcare system both in 
real terms and in delays of bringing new technologies to 
patients. However, as you suggest under the 510(k) paradigm, 
some devices may be cleared for marketing with no relevant 
clinical trial evidence at all. Could you discuss your concerns 
with that program and the potential risk to the healthcare 
system of Medicare covering such technologies even under its 
coverage with evidence development authority?
    Mr. Jacques. Yes. While that paradigm is appropriate for 
many low-risk devices, I would focus my own attention on that 
subset of cleared devices where untested claims of enhanced 
benefit are made beyond the predicate device, or where 
subsequent evidence may raise questions about the fundamental 
impact of the technology.
    I think the premise of the 510(k) program makes it more 
difficult for a sponsor to articulate an enhanced value 
proposition for a technology when it has been found to be 
substantially equivalent to an old technology.
    And that to me is the critical point in terms of paying for 
value. That value proposition that you are essentially better 
than something is hard to make if you have not actually been 
compared to anything else.
    Mr. Waxman. So we may have a 510(k) to get the device 
approved, but we ought to know before we start paying a lot of 
money for it that it is going to work.
    Mr. Jacques. Yes, sir.
    Mr. Waxman. Thank you. Thank you, Mr. Chairman.
    Mr. Pitts. The chair thanks the gentleman, and now 
recognizes the vice chairman of the subcommittee, Dr. Burgess, 
5 minutes for questions.
    Mr. Burgess. Thank you, Mr. Chairman. And I want to thank 
the ranking member for his generosity in mentioning H.R. 4015, 
which was the repeal of the sustainable growth rate formula, 
which did come through this committee. We are about at the 1-
year anniversary of that 51 to zero vote. That was a landmark 
occasion for the committee. And in many ways, the development 
of that SGR policy was very similar to what is happening with 
the Cures Initiative. So I think that provides a template that 
ultimately could speak to success for the Cures Initiative that 
as we opened the doors up, we took information, asked for 
information from physicians, from patients, as to what they 
needed to see in the repeal of the sustainable growth rate. As 
a result, nobody got exactly what they wanted, but we got a 
product that was ultimately supportable by both Republicans and 
Democrats on this committee, and ultimately did pass the floor 
of the House, though we are still waiting on the Senate.
    Ms. Grealy, I need to ask you a question about--there is a 
bill that Donna Christensen and I have done, H.R. 2663, which 
deals with CBO scoring, because oftentimes it seems like there 
are good ideas that are developed within the healthcare sphere, 
but then CMS will say, but you know what? All we can do--or the 
CBO will say CMS just tells us about the cost, so all we can do 
is report to you on the cost. So the ability to implement this 
new regimen is, in fact, a cost driver for the system, and 
cannot be regarded as a cost saver.
    And, in fact, in this committee, even though I did not 
support the Affordable Care Act, on this committee I recognized 
a great deal of anxiety on the part of my Democratic 
counterparts in dealing with Mr. Elmendorf at the Congressional 
Budget Office. Wait a minute, we get no credit for all of the 
savings we are going to get from treating things in a more 
timely fashion.
    So in your role at the Healthcare Leadership Council, have 
you studied that issue at all?
    Ms. Grealy. Yes, Mr. Burgess, and we strongly support the 
legislation----
    Mr. Burgess. I felt you would say that.
    Ms. Grealy. You think so.
    Mr. Burgess. That is why I asked you.
    Ms. Grealy. And delighted that it is bipartisan legislation 
as well. But as you know, innovation plays a strong role in 
wellness and prevention. And what our members have seen are 
long-term savings when you make that investment in wellness and 
prevention.
    And as you point out, unfortunately CBO, in their 
traditional scoring methods, does not give you credit for those 
long-term savings. And we know that 70 to 80 percent of 
healthcare costs today are going towards the treatment of 
preventable chronic disease, and we know that if we make an 
investment over the long term, we will see a dramatic reduction 
in those healthcare costs. So your legislation would not 
mandate that CBO have this longer scoring window, but at least 
we would have that option so that you as members of Congress 
could see that information and then make your decision on 
making those investments, which may have a short-term cost, but 
we know in the long term will result in better health and lower 
costs for the healthcare system.
    Mr. Burgess. Well, oftentimes it seems today we only end up 
talking about the costs of a therapy and we do not recognize 
the fact that, my goodness, we have beaten one of the big 
scourges of people's health. The hepatitis C treatment comes to 
mind. Instead of talking about the victory over hepatitis C, a 
disease that did not even have a name when I was in residency. 
We called it non-A/non-B hepatitis. And now there is a 
treatment for it that is, in fact, a cure. That is pretty big 
news.
    So it is my hope that the Cures Initiative will be able to 
focus on those things. Yes, we will talk about price and we 
will talk about cost as we go through. But the big news, the 
headline is hepatitis C vanquished in our lifetimes, and that 
is a big deal.
    Dr. Schmizzi, I wanted to ask you a question on the 
Sunshine Act and the Sunshine Act provision that was contained 
in the Affordable Care Act. It does seem like it was written 
pretty broadly, and now the implementation is or runs the risk 
of hindering communication and information sharing among 
physicians.
    So a rule that came out over the 4th of July weekend may 
prevent some of the country's most qualified physicians from 
giving lectures to fellow physicians through continuing medical 
education. Have you heard of providers that are having 
difficulty getting access to medical studies or even finding it 
more difficult to access continuing education because of the 
way this law is being implemented?
    Dr. Schmizzi. Excuse me, Congressman. I do not hear of 
anything yet, but I can certainly see it coming that the 
Sunshine Act provision, the way it is written, can actually 
inhibit speakers from wanting to attend or be participants in a 
medical conference because of the information that will be 
published about them being paid and where the money comes from.
    Most institutions, most professional associations get their 
funding from member dues, but they also get funding from 
industry support in the form of gifts or donations. And those 
gifts and donations, if they are identified to be tied to CME 
credits, can actually impair the desire of academicians and 
thought leaders in medicine to give those presentations in 
front of those societies. So it can have a real negative impact 
on that. I do not believe it has happened yet in my sub-
specialty, but it certainly is possible.
    Mr. Burgess. Thank you for that answer. Mr. Chairman, I 
hope that is something that this committee will keep in mind 
and continue to monitor as we go forward. I will yield back.
    Mr. Pitts. The chair thanks the gentleman, and now 
recognizes the gentleman from Texas, Mr. Green, 5 minutes for 
questions.
    Mr. Green. Thank you, Mr. Chairman. And again, thank our 
witnesses for being here. A central component in improving the 
quality of our healthcare system and developing 21st century 
cures must be data-driven innovation. Mr. Mussallem, in your 
testimony you talked about the coverage for evidence 
development CED determination, how it can be useful if used 
appropriately.
    However, the challenge of ensuring CED is a tool for the 
reimbursement system to give patients access to groundbreaking 
therapies rather than the burden that ultimately limit 
innovation remains before us. Can you tell us how we might be 
able to handle that?
    Mr. Mussallem. Sure. Particularly the use of CED, I think, 
is valuable for therapies that are new and really have not been 
evaluated in the past. In many cases, the therapies that can be 
reimbursed are ones that are well understood, and you could 
establish the safety and a safe and necessary threshold. But in 
the case where you just do not know much because they are 
novel, it is helpful to be able to apply CED.
    It is not always clear in the beginning of the CED process 
exactly what evidence is going to be collected and how much is 
necessary. And one of the things that is also not clear about 
CED is when does it come to an end? At some point in the 
initial stages of a technology, it is very valuable to learn as 
much as you can and collect that evidence. But once you have 
done that for some period of time, it is appropriate for CED to 
sunset so that it does not just become another layer of cost 
that sits on the healthcare system.
    And so, it is important, I think, to define CED more 
thoughtfully and carefully as we think about using it as a 
tool. But it has great promise for entering areas that are 
unknown.
    Mr. Green. OK. Do you have any mechanisms you would suggest 
to enhance the coverage of these innovative therapies?
    Mr. Mussallem. It is not a simple question. In the case of 
trans-catheter technology, CED was used, and it was used that 
allows for this important aspect of medical device development 
to be evaluated, different than a drug.
    Medical technology is one that is an iterative process. 
Because we make tools for physicians, often we get a lot of 
feedback from physicians and they say, could you make it 
better? Could you make it smaller? Could you make it do things 
that it does not do today? And we respond to that. And through 
those changes, therapy improves dramatically.
    And so, a coverage evidence development tool that is 
flexible, and this is what was done in the case of trans-
catheter heart valves, allowed for the system to accommodate 
new generations, new indications, as the evidence supported it. 
So that is a powerful use of that tool.
    Mr. Green. Dr. Jacques, is there any other mechanism 
available to provide coverage to these new innovations?
    Mr. Jacques. There are other mechanisms, including 
regulations concerning Medicare coverage for FDA-approved 
Category B investigational device exemption trials, the 
challenge being that aside from CED and those IDEs, there is 
not an obvious path for other sorts of valuable research.
    Mr. Green. OK. Anyone else on the panel for those issues or 
those questions?
    If not, our entire healthcare system is shifting to a model 
that embraces shared decision making by informed patients whose 
views are valued and considered at every stage of the 
treatment. We have heard a great deal about the potential value 
for patient preference information and regulatory risk benefit 
determinations, particularly in the context of medical device 
pre-market approval.
    The FDA has emphasized that patient tolerance for risk and 
perspective on benefit is an important consideration. It makes 
sense for the innovators and regulators to consider patient 
perspectives as they develop and evaluate medical devices.
    Mr. Mussallem, again, what potential benefit do you see 
from incorporating patient preference information in regulatory 
determinations, and do you have any suggestion on how it could 
be incorporated in the process?
    Mr. Mussallem. Sure. All medical technology and all medical 
advancements are not created equal. Some can have a profound 
impact on patients' lives. In our case, sometimes it is the 
only difference between life and death for these patients. So 
when you are making that sort of a consideration as a 
regulator, you would really love that the regulators, they have 
the ability to apply a risk benefit analysis when they are 
thinking about what they should do in terms of allowing this 
technology to come to patients.
    If you keep the bar too high in the pre-market approval 
setting, what you might do is in an effort to achieve great 
science, again allow patients to not benefit and, in fact, die 
or live very poor quality of life. And sometimes it would make 
some sense to allow a certain element of risk, certainly to 
safeguard against safety concerns, and have a basic level of 
evidence, but to study in a post-market setting the true depth 
of efficacy in a real-world setting, and then apply that and 
make adjustments.
    So this is one that you would not want to be unfettered, 
but to give regulators, in effect, not only the ability to, but 
the mandate to take a risk benefit analysis I think would be a 
powerful enhancement for the system and make it a learning 
system rather than what we have today.
    Mr. Green. Thank you, Mr. Chairman.
    Mr. Pitts. The chair thanks the gentleman, and now 
recognizes the gentleman from New Jersey, Mr. Lance, for 5 
minutes for questions.
    Mr. Lance. Thank you, Mr. Chairman, and good afternoon to 
you all. Ms. Grealy, in your testimony you state that the key 
to harnessing the potential of real time data lies in putting 
the policies and practices in place that allow us to harness 
this data. You then go on to state the importance of protecting 
confidential health information while also making data 
appropriately accessible under HIPAA.
    In several of our recent hearings, witnesses have pointed 
out the challenges that arise in ensuring that this innovative 
technology is HIPAA compliant. Are there ways in which HIPAA 
inappropriately restricts the sharing and use of patient data 
by researchers and medical providers?
    Ms. Grealy. Well, I think it is an ongoing challenge. And 
really what are trying to do is find the appropriate balance 
between protecting that patient information, but not stifling 
the innovation or access. And so we constantly have to keep 
that in mind.
    And periodically proposals are put forward that really 
would consume a lot of resources and time, and really do not 
create any particular value for the patient. I will use an 
example of that disclosure of everyone who has had access to 
the patient information, whether they are within that 
healthcare operations that I discussed, which is reasonably 
expected by patients.
    So I think it is all about making sure that we do not try 
to micromanage this, and we really put the patient at the 
center of it. And how can we create better value for that 
patient? And so, as we are looking at new ways and new access 
to information, and I will use as an example of that, as I 
mentioned earlier, patient engagement and the new tools for 
that, the mobile apps.
    And we are spending a lot of time, those of us that have 
been minding the HIPAA world for many, many years, working with 
those app developers and telling them, as you are approaching 
this, we do not want to hinder your innovation, but try to 
build into your system up front the appropriate patient 
protections and information protections. But again, the key is 
let us not stifle that innovation by them, and let us not defer 
a whole lot of resources that could be going towards patient 
care and treatment and innovation by getting caught up in too 
much compliance activity.
    Mr. Lance. Is there something we should be doing here in 
Congress to make this a better situation?
    Ms. Grealy. I would almost say do not do too much.
    Mr. Lance. First do no harm.
    Ms. Grealy. Yes, first do no harm.
    Mr. Lance. To coin a phrase.
    Ms. Grealy. Yes. And as we are looking, we have heard a lot 
today about registries and how we can use that information. I 
would say, again, let us not stifle that access and the use of 
that information.
    And the other very powerful thing that we are seeing that I 
think is going to be make all of this much more available and 
much more usable is what is happening with health information 
technology. I do not think any of us could have imagined even 5 
years ago how rapidly we are nowable to build these databases 
and how rapidly we are not able to access that information. And 
more importantly, get those best practices to the physicians 
right as they are treating the patients and having those 
practice guidelines, which is going to go a long way towards 
creating that value that we have all talked about in our 
healthcare system.
    Mr. Lance. Thank you. The Physician Payments Sunshine Act, 
usually known as the Sunshine Act, requires manufacturers of 
drugs and medical devices that participate in Federal health 
programs to report payments to physicians in teaching 
hospitals. In your judgment, has that data sharing in this 
regard been beneficial to medical innovation? Ms. Grealy?
    Ms. Grealy. Again, I would caution, and I think we heard a 
lot today on this panel. We all believe in transparency. We 
think that is important, and having the disclosures about 
collaborations between physician and industry.
    What we are most concerned about, and what we have actually 
seen already is the chilling effect, that physicians are 
concerned. Wait a minute, this is a minimum amount of money. It 
is just not worth it to have my name on a list when there is no 
context about what was the value of that collaboration.
    And I think you heard Mr. Mussallem talk about their 
interactions with physicians as they are developing new cures, 
new devices. It is absolutely critical. And their partnerships 
with academic health institutions, absolutely critical.
    So again, it is about balance. We think there should be 
reporting this information, but it needs to be in context so 
that people know what is the value of having physicians working 
with manufacturers, and how does that benefit patients.
    Mr. Lance. Thank you. My time has expired. Thank you, Mr. 
Chairman.
    Mr. Pitts. The chair thanks the gentleman, and now 
recognizes the gentleman from Florida, Mr. Bilirakis, 5 minutes 
for questions.
    Mr. Bilirakis. Thank you, Mr. Chairman. I appreciate it 
very much. First question is for Dr. Schmizzi. There are about 
7,000 diseases and only about 500 treatments available. 
Patients with rare diseases frequently have no approved 
treatments. This forces these patients to find off label usage 
for medication to treat their condition. If the FDA has rules 
limiting information to doctors and patients, this could harm a 
patient's health. And I know this was touched on earlier.
    Our health system should be patient-centered type of care, 
in my opinion. Given that, how can we ensure that patients and 
their physicians have access to information, whether it be on 
label or off label uses, so that it can determine the best 
course for treatment?
    Dr. Schmizzi. Thank you, Congressman. I think the best way 
to do that is to ask the FDA or direct the FDA by legislation, 
or statute, or regulation changes, to allow that communication 
to go forward. Right that now communication is badly stifled, 
and much of the information that pharmaceutical manufacturers 
and innovators have is hidden from our view.
    Rheumatology treats many diseases that have no defined 
treatment. There is no medication that is defined to treat 
Sjogren's syndrome. There is not defined treatment, no 
medication specifically defined to treat chondrocalcinosis. 
Some of these unusual diseases that are not really rare. We see 
a lot of people with that, but we have no defined mechanism or 
medicine that is approved for the use in these diseases.
    But things like Sjogren's syndrome, I am certain that the 
pharmaceutical company that has manufactured some of the 
medications available today have had crossovers with patients 
who have Sjogren's syndrome, and they have data on how those 
patients' Sjogren's symptoms improved or worsened, which is 
also important to know. Did a particular medication make that 
particular subset of symptoms worse?
    Those things are important for us to know, but those things 
may not be readily available to us. And those would be very 
helpful to have.
    Mr. Bilirakis. Thank you. Would it improve the standard of 
care to have these indications on the label, such as ensuring 
correct dosage and access to insurance reimbursement? And 
should we incentivize sponsors to do the additional studies to 
get these off label uses on label?
    Dr. Schmizzi. I think incentivization to do some of these 
studies on these small diseases would be very, very helpful. It 
took 15 years to define that dermatomyositis was treatable with 
a medication that has been on the market for 20 years. It took 
that long to get a large enough sample size to prove that the 
medication really worked. And dermatomyositis is a devastating 
inflammatory disease of the muscle that destroys muscle tissue 
and skin.
    So incentivizing those types of things would go a long way. 
And the National Institutes of Health already does that, and 
they were the ones who sponsored the actual study on 
dermatomyositis. But incentivizing manufacturers to go forward 
with some of these smaller diseases would be very helpful, yes.
    Mr. Bilirakis. Very good. Thank you very much. Mr. 
Mussallem, you mentioned that it was 4 years after the EU 
approval before the FDA approved the SAPIEN valve. Is the U.S. 
typically behind the EU for device approval? Why, and how can 
we accelerate the process?
    Mr. Mussallem. Yes, it was 4 years' difference. I would say 
generally in medical devices and medical technology, 
manufacturers would introduce their products first in Europe. 
The burden of proof to be able to introduce in Europe is much 
lower than the burden of proof required by the FDA.
    There is a level of safety that needs to be established in 
Europe, but a much lower level of efficaciousness that is 
required that is required before it moves to the marketplace. 
And it is left to the judgment of physicians and patients on 
whether it should be used, and the FDA requires a much higher 
level of science to bring it to the United States.
    Mr. Bilirakis. How can we accelerate the process here in 
the United States?
    Mr. Mussallem. Well, there are several ideas, and a number 
of them have actually been mentioned by Jeff Shuren, who is 
responsible for CDR-8, including trying to think more carefully 
and take a risk benefit analysis, and think carefully about 
what might be collected in a pre-market setting versus a post-
market setting.
    In the cases where patients really need the benefit, if you 
were to have a pre-market setting that was not so onerous, but 
rather have more extensive study in the post-market setting, 
what you could do then is potentially speed these cures to 
people that really need it when in the judgment of FDA it was 
the right thing to do. And at the same time, make sure that you 
are collecting the evidence so that therapies that are winners 
get supported and losers get stopped.
    Mr. Bilirakis. Well, thank you very much. Appreciate that. 
And I yield back, Mr. Chairman.
    Mr. Pitts. The chair thanks the gentleman, and now 
recognizes the gentlelady from North Carolina, Ms. Ellmers, 5 
minutes for questions.
    Mrs. Ellmers. Thank you, Mr. Chairman. And thank you to our 
panel, especially Dr. Schimizzi--welcome--from Wilmington, 
North Carolina, one of my very favorite places and just down 
the road from Dunn, North Carolina.
    Dr. Jacques, I have a question for you. You state that CMS 
needs unambiguous authority to review clinical trials when 
claims related to these trials will be submitted for Medicare 
payment. In what ways is CMS authority in this respect limited 
now, and how does this impact the search for cures?
    Mr. Jacques. At a fundamental level, one would expect that 
the Medicare program or any insurance company would know what 
it is paying for as opposed to paying blindly. And my 
understanding is periodically Congress asks Medicare how much 
research are you paying for, and my understanding is the Agency 
has been unable to actually produce a number. So that would be 
helpful to know.
    I think that Medicare engagement on research would actually 
serve a number of purposes because I have found much to my own 
frustration while I was a civil servant that there would often 
be times when companies would have come in with the data that 
they had, and we would sit there around the table going, if 
only 2 years ago you had made this small change it would have 
made a very large course direction in where you came up.
    So the challenge is that Medicare covers routine costs in 
clinical trials based on a White House executive order from the 
end of the Clinton Administration. There is then a distinct 
regulation on FDA Category B investigational device exemptions, 
and then there is CED. And in any particular trial, there may 
be an overlap of those things, so CED would include routine 
costs, for example. There may be CED that might also be 
combined in the context of an FDA IDE approval study.
    And because all of these things are siloed, it is very, 
very difficult at the staff level when a prospective 
investigator comes in and says, oK, here is my clinical trial. 
These are all the things I want to do. Can you tell me if it is 
covered or not. And that can be a conversation that takes 
months to get to all the details.
    And I believe that if CMS simply had a singular authority 
that would relate to this, it could then publish an actual 
integrated policy where all the pieces actually fit, and you 
were not running all over the place trying to find different 
parts of an answer.
    Mrs. Ellmers. Mr. Mussellum and Dr. Schimizzi, it looked 
like you were very intrigued by Dr. Jacques' answer. Is there 
anything that either one of you would like to comment on?
    Mr. Mussallem. Yes. We think that just by listening to 
comments of Dr. Jacques and others in CMS, we have heard that 
there are limitations to what policy allows CMS to do. And also 
that there are limitations associated with their staffing 
levels, and that is concerning to us. We are dependent on 
payment to be able to bring these technologies to patients.
    One of their particular things that are most noteworthy is 
much of the great medical breakthroughs come from individuals, 
or very small companies, or somebody that just has a great 
idea. And being able to take that from a napkin to reality is 
becoming longer and longer and more and more costly.
    And to be able to have a conversation with CMS that clearly 
defines a predictable process would be very powerful to those 
organizations. And this unpredictability has a chilling effect 
on innovation, so that kind of clarity would be very positive.
    Mrs. Ellmers. Dr. Schmizzi?
    Dr. Schmizzi. I have found that what Dr. Jacques mentioned 
about staffing problems being an issue with national coverage 
determinations and local coverage determinations, contrasting 
one another, conflicting, very intriguing especially because 
that is a topic that has really hit us very hard this last year 
when we have a patient who may live in North Carolina part of 
the year and New York another part of the year, and they have 
Medicare. They may be able to get the medication in North 
Carolina, but when they go to New York the medication is denied 
because the carriers are different and the coverage 
determination is different.
    It would be really nice to have a uniform set of rules.
    Mrs. Ellmers. Centralized.
    Dr. Schmizzi. Yes. I mean, that is essentially what the 
national coverage determination was meant to do. But I can now 
understand, given what I have heard today, that it might indeed 
be a staffing problem that does not allow CMS to act on the 
national level, and allows individual carriers to make 
different determinations in different States, which makes it 
difficult for a patient to get the same care in different areas 
of the country.
    Mrs. Ellmers. Right. Well, thank you. And my time is about 
expired, so I will leave it at that. Thank you, Mr. Chairman.
    Mr. Pitts. The chair thanks the gentlelady, and now 
recognizes the gentlelady from Colorado, Ms. DeGette, 5 minutes 
for questions.
    Ms. DeGette. Thank you very much, Mr. Chairman, and thank 
you again for your commitment to these hearings.
    Mr. Mussallem, in your written testimony you mentioned some 
of the advantages of registries to help with post-market 
surveillance. And you talk about the American College of 
Cardiology and the Society of Thoracic Surgeons working 
collaboratively to create the STSACCTVT registry. Can you talk 
to us a little bit more about that registry, when it was 
formed, the cost, and who can access that data and information?
    Mr. Mussallem. Yes, thank you. It was a remarkable 
collaboration. And again, routinely when a new technology is 
approved, FDA would mandate a post-approval study. In this 
case, the idea of mandating a post-approval study took a couple 
of forms, and an alternative was presented to collect data in a 
registry rather than collect more extensive data on a smaller 
group of people.
    Through conversations with CMS--as a matter of fact, this 
actually became one where CMS also became partners in this 
discussion as well as a number of other stakeholders. It 
actually became part of the national coverage determination 
because the national coverage determination said we will pay 
for this new procedure if you are collecting evidence. And they 
did that under the provisions of coverage with evidence 
development.
    So this registry became multi-purpose, and it did a few 
things. One is it became the post-approval study for FDA and to 
follow patients on a long-term basis with this new therapy, and 
every patient gets this, so it is a very large and powerful 
database. It became the tool used for evidence collection for 
CMS in terms of their ultimate decision on coverage for 
evidence development. And it just became very powerful to the 
medical community because here was a set of data that rather 
than being managed by a company----
    Ms. DeGette. Excuse me. Excuse me. They only give me 5 
minutes.
    Mr. Mussallem. I am sorry.
    Ms. DeGette. And I appreciate every minute of that. Who can 
access that data and information from that registry?
    Mr. Mussallem. Yes. That is exactly where I was going.
    Ms. DeGette. OK.
    Mr. Mussallem. This data is managed by the American College 
of Cardiology and the Society of Thoracic Surgeons as a matter 
of fact. So it is housed within their organization, and so they 
have access to it. There is an advisory board that includes 
many of the members of those societies that actually ride herd 
over that data and publish results from that data on a routine 
basis.
    Ms. DeGette. OK. Now, there are some limitations, I think, 
that you and others have pointed out with registries. And I am 
wondering, do you think it is just because we do not have a lot 
of experience with it?
    Mr. Mussallem. I think there is concern that we do not have 
experience with registries, and that is certainly true. We have 
no experience, for example, in our field of heart valves.
    Ms. DeGette. Right. Right. So we just need to learn. Dr. 
Rising, I wanted to ask you quickly along these lines, in your 
testimony you talked about the Sentinel Initiative as a 
possible alternative or supplement to registries. Can you talk 
about how those can work for data collection?
    Dr. Rising. Sure. I would be happy, thanks. So Congress 
instructed FDA to establish the Sentinel Program in 2007 to 
proactively monitor for problems with drugs and biologics on 
the market. And in 2012, Congress instructed FDA to expand 
Sentinel to include medical devices.
    Now, what Sentinel is it uses claims data, almost 
exclusively claim data, housed by payers to look for 
associations between exposure to a particular product and then 
a particular health outcome.
    Now, Sentinel could be expanded to devices except that 
right now there is no specific information on a device that is 
used in care on the claims form. So a payer might have 
information that they did a hip replacement that they are 
paying for, but they have no information on the specific hop 
replacements that were used.
    Ms. DeGette. Right. Right.
    Dr. Rising. So to expand Sentinel to include devices, a new 
field needs to be placed on the claims form. And in general, 
and we are a big supporter of using existing structures, such 
as claims forms, to capture new information like this.
    Ms. DeGette. And, Mr. Mussallem, in your testimony you said 
that we need more resources and support for FDA. And I am 
wondering what types of resources you think we need. We have 
heard others talking about CMS. I am wondering about FDA.
    Mr. Mussallem. You know, I think the leadership at FDA has 
a pretty clear vision of some things that need to change, and 
they have done a pretty good job of articulating that through 
their strategic plan.
    Ms. DeGette. Right. They have also told us about it, too. 
Thank you very much. Thank you, Mr. Chairman.
    Mr. Pitts. The chair thanks the gentlelady. I have a 
unanimous consent request. I would like to insert a letter from 
the Lupus Foundation of America into the record.
    Without objection, so ordered.
    [The information appears at the conclusion of the hearing.]
    Mr. Pitts. This has been another very informative and 
important hearing. Thank you very much for testifying today. We 
will have a lot of follow-up questions I am sure from members. 
We will send those to you. We ask that you please respond 
promptly. Members will have 10 business days to submit their 
questions for the record. That means they should submit their 
questions by the close of business on Tuesday, August the 5th.
    Without objection, the subcommittee is adjourned.
    [ Whereupon, at 4:39 p.m., the Subcommittee was adjourned.]
    [Material submitted for inclusion in the record follows:]

                 Prepared statement of Hon. Fred Upton

    When we first launched the 21st Century Cures initiative in 
April, we had a pretty good idea that learning about the 
benefits and risks of a drug or device doesn't end when FDA 
initially approves or clears the product for use in certain 
patients with a specific disease or set of conditions. Since 
then, we have heard repeatedly that in many ways it is only 
just beginning.
    Different uses for drugs or devices are constantly being 
discovered by physicians, researchers and scientists in 
academia and industry. Particularly in the context of devices, 
improvements are continually made to products based on new 
evidence being developed about how certain patients are 
responding to certain treatments, technologies, or combinations 
thereof. We must work to ensure that our regulatory and 
reimbursement policies encourage this iterative process and do 
not stifle innovation.
    This type of ongoing evidence development, collaboration, 
and communication must be facilitated, not hindered by any 
policies in place that do not ultimately benefit patients. As 
part of the 21st Century Cures initiative, I am committed to 
evaluating how Congress can play a role in breaking down any of 
these legal or regulatory barriers and encouraging 
communication and collaboration between and among patients, 
doctors, and scientists regarding new data, research, and 
results.
    At our digital healthcare roundtable, we learned about the 
many exciting opportunities to capture and analyze data in 
real-world delivery settings to generate meaningful insight and 
specific evidence about which type of treatments are working 
better on which type of conditions or diseases in which type of 
patients-often right down to the molecular level. During our 
joint hearing of the Health and Telecommunications 
subcommittees, we learned more about the role electronic health 
records and increased data sharing can play in that process, 
but also heard about the challenges and privacy issues we must 
address in order for such potential to become reality.
    As we stated from the outset in our first Cures white 
paper, the policies we have in place must allow for health care 
delivery to serve as a platform for new discovery and 
development. This hearing will provide a great opportunity to 
learn how we can encourage and reward ongoing evidence 
development and not unduly limit how such evidence is discussed 
or communicated to patients and providers.
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             Prepared statement of Hon. Frank Pallone, Jr.

    Thank you Chairman Pitts. Today's hearing is a broader 
effort to better understand how data collection and the 
exchange of patient information can be improved to help 
facilitate 21st Century Cures.
    The development process of medical products, as we have 
learned, has many layers. Throughout our meetings on this 
initiative we have heard that FDA and NIH are driving medical 
advances and innovative approaches to clinical trial designs. 
NIH also develops and funds the basic research that makes 
medical advances possible. FDA, meanwhile, has made full use of 
early indicators of effectiveness, when the science justifies 
their use, to enable it to approve drugs based on more limited 
data than would otherwise be possible.
    But there are still challenges to taking full advantage of 
these advances. For example, we've heard that there are 
obstacles to patient recruitment for clinical trials. Today I 
hope we can better understand about what methods can be used to 
facilitate initial product development but also allow for 
further evaluation of the effects of drugs and devices already 
on the market. I am particularly interested in the role patient 
registries and electronic health records can play.
    We all want the best cutting edge medicines and treatments 
to get to the patients who need them. But we must also ensure 
that we have good tools for post market monitoring. So I'm also 
interested in how electronic health records can facilitate such 
monitoring and enable greater participation in clinical trials, 
while also safeguarding patient privacy under HIPAA.
    Another topic we will hear extensively about today is how 
drugs and devices, once developed, get reimbursed--highlighting 
the process by which new drugs and devices under federal health 
programs like Medicare gain coverage. Clinical trials don't 
always provide the necessary clinical evidence to enable the 
Medicare program to determine whether the coverage of a 
particular drug or device is reasonable and necessary for its 
particular patient subpopulation. With the inability of 
Medicare to negotiate prices and the increasing price of new 
drugs and biologics, it is incumbent upon Medicare to be very 
diligent in its coverage decisions.
    Getting a treatment or a cure to a patient has implications 
for industry, payors and patients alike. So how do we ensure 
access to these products? In addition, medicines and treatments 
alone will not ensure the best outcomes. Providers have a 
critical role to play in the quality of care patients receive.
    Mr. Chairman, these are complicated issues. I want our 
researchers and scientists to have access to the funding 
necessary to make discoveries; I want our companies to operate 
in an environment where innovations can flourish; and I want 
patients to have access to safe and effective treatments. I'm 
not entirely sure a package of laws is needed to accomplish all 
of these goals, but I'm hopeful that Democrats and Republicans 
can work together moving forward to accomplish these goals.
    Thank you.
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