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93–247 2015

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 

( 

Printed for the use of the Committee on Energy and Commerce 

energycommerce.house.gov 

(II) 

COMMITTEE ON ENERGY AND COMMERCE 

FRED UPTON, Michigan 

Chairman 

RALPH M. HALL, Texas 

JOE BARTON, Texas 

Chairman Emeritus 

ED WHITFIELD, Kentucky 

JOHN SHIMKUS, Illinois 

JOSEPH R. PITTS, Pennsylvania 

GREG WALDEN, Oregon 

LEE TERRY, Nebraska 

MIKE ROGERS, Michigan 

TIM MURPHY, Pennsylvania 

MICHAEL C. BURGESS, Texas 

MARSHA BLACKBURN, Tennessee 

Vice Chairman 

PHIL GINGREY, Georgia 

STEVE SCALISE, Louisiana 

ROBERT E. LATTA, Ohio 

CATHY MCMORRIS RODGERS, Washington 

GREGG HARPER, Mississippi 

LEONARD LANCE, New Jersey 

BILL CASSIDY, Louisiana 

BRETT GUTHRIE, Kentucky 

PETE OLSON, Texas 

DAVID B. MCKINLEY, West Virginia 

CORY GARDNER, Colorado 

MIKE POMPEO, Kansas 

ADAM KINZINGER, Illinois 

H. MORGAN GRIFFITH, Virginia 

GUS M. BILIRAKIS, Florida 

BILL JOHNSON, Missouri 

BILLY LONG, Missouri 

RENEE L. ELLMERS, North Carolina 

HENRY A. WAXMAN, California 

Ranking Member 

JOHN D. DINGELL, Michigan 

Chairman Emeritus 

FRANK PALLONE, JR., New Jersey 

BOBBY L. RUSH, Illinois 

ANNA G. ESHOO, California 

ELIOT L. ENGEL, New York 

GENE GREEN, Texas 

DIANA DEGETTE, Colorado 

LOIS CAPPS, California 

MICHAEL F. DOYLE, Pennsylvania 

JANICE D. SCHAKOWSKY, Illinois 

JIM MATHESON, Utah 

G.K. BUTTERFIELD, North Carolina 

JOHN BARROW, Georgia 

DORIS O. MATSUI, California 

DONNA M. CHRISTENSEN, Virgin Islands 

KATHY CASTOR, Florida 

JOHN P. SARBANES, Maryland 

JERRY MCNERNEY, California 

BRUCE L. BRALEY, Iowa 

PETER WELCH, Vermont 

BEN RAY LUJAN, New Mexico 

PAUL TONKO, New York 

JOHN A. YARMUTH, Kentucky 

SUBCOMMITTEE ON HEALTH

JOSEPH R. PITTS, Pennsylvania 

Chairman 

MICHAEL C. BURGESS, Texas 

Vice Chairman 

ED WHITFIELD, Kentucky 

JOHN SHIMKUS, Illinois 

MIKE ROGERS, Michigan 

TIM MURPHY, Pennsylvania 

MARSHA BLACKBURN, Tennessee 

PHIL GINGREY, Georgia 

CATHY MCMORRIS RODGERS, Washington 

LEONARD LANCE, New Jersey 

BILL CASSIDY, Louisiana 

BRETT GUTHRIE, Kentucky 

H. MORGAN GRIFFITH, Virginia 

GUS M. BILIRAKIS, Florida 

RENEE L. ELLMERS, North Carolina 

JOE BARTON, Texas 

FRED UPTON, Michigan (ex officio) 

FRANK PALLONE, JR., New Jersey 

Ranking Member 

JOHN D. DINGELL, Michigan 

ELIOT L. ENGEL, New York 

LOIS CAPPS, California 

JANICE D. SCHAKOWSKY, Illinois 

JIM MATHESON, Utah 

GENE GREEN, Texas 

G.K. BUTTERFIELD, North Carolina 

JOHN BARROW, Georgia 

DONNA M. CHRISTENSEN, Virgin Islands 

KATHY CASTOR, Florida 

JOHN P. SARBANES, Maryland 

HENRY A. WAXMAN, California (ex officio) 

(III) 

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. 

(1) 

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 

2 

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 

3 

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 indeVerDate Nov 24 2008 14:42 Jun 04, 2015 Jkt 037690 PO 00000 Frm 00007 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 113\113-163 CHRIS
4 

pendence 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. 

5 

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. 

6 

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 

7 

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-onMetal 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:] 

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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. 

14 

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 postmarket 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 

22 

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 onlabel 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 providerspecific 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 

37 

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 

54 

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-WstateGrealyM-20140722.pdf.] 


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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 

67 

Prilosec. The generic name was omeprazole. Prilosec was a mixture 

of two different mirror image molecules, D-enantiomer and an Lenantiomer. 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? 

68 

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 

69 

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 

70 

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. 

71 

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? 

72 

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. 

73 

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 

74 

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/nonB 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 

75 

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 transcatheter 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? 

76 

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 proVerDate Nov 24 2008 14:42 Jun 04, 2015 Jkt 037690 PO 00000 Frm 00080 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 113\113-163 CHRIS
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tecting 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. 

78 

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 dosVerDate Nov 24 2008 14:42 Jun 04, 2015 Jkt 037690 PO 00000 Frm 00082 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 113\113-163 CHRIS
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age 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 

80 

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 organiVerDate Nov 24 2008 14:42 Jun 04, 2015 Jkt 037690 PO 00000 Frm 00084 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 113\113-163 CHRIS
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zations. 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 

82 

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. 

83 

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. 

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 posVerDate Nov 24 2008 14:42 Jun 04, 2015 Jkt 037690 PO 00000 Frm 00087 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 113\113-163 CHRIS
84 

sible. 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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