[House Hearing, 112 Congress]
[From the U.S. Government Publishing Office]
REAUTHORIZATION OF MDUFA: WHAT IT MEANS FOR JOBS, INNOVATION, AND
PATIENTS
=======================================================================
HEARING
BEFORE THE
SUBCOMMITTEE ON HEALTH
OF THE
COMMITTEE ON ENERGY AND COMMERCE
HOUSE OF REPRESENTATIVES
ONE HUNDRED TWELFTH CONGRESS
SECOND SESSION
__________
FEBRUARY 15, 2012
__________
Serial No. 112-116
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Printed for the use of the Committee on Energy and Commerce
energycommerce.house.gov
_____
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COMMITTEE ON ENERGY AND COMMERCE
FRED UPTON, Michigan
Chairman
JOE BARTON, Texas HENRY A. WAXMAN, California
Chairman Emeritus Ranking Member
CLIFF STEARNS, Florida JOHN D. DINGELL, Michigan
ED WHITFIELD, Kentucky Chairman Emeritus
JOHN SHIMKUS, Illinois EDWARD J. MARKEY, Massachusetts
JOSEPH R. PITTS, Pennsylvania EDOLPHUS TOWNS, New York
MARY BONO MACK, California FRANK PALLONE, Jr., New Jersey
GREG WALDEN, Oregon BOBBY L. RUSH, Illinois
LEE TERRY, Nebraska ANNA G. ESHOO, California
MIKE ROGERS, Michigan ELIOT L. ENGEL, New York
SUE WILKINS MYRICK, North Carolina GENE GREEN, Texas
Vice Chairman DIANA DeGETTE, Colorado
JOHN SULLIVAN, Oklahoma LOIS CAPPS, California
TIM MURPHY, Pennsylvania MICHAEL F. DOYLE, Pennsylvania
MICHAEL C. BURGESS, Texas JANICE D. SCHAKOWSKY, Illinois
MARSHA BLACKBURN, Tennessee CHARLES A. GONZALEZ, Texas
BRIAN P. BILBRAY, California JAY INSLEE, Washington
CHARLES F. BASS, New Hampshire TAMMY BALDWIN, Wisconsin
PHIL GINGREY, Georgia MIKE ROSS, Arkansas
STEVE SCALISE, Louisiana JIM MATHESON, Utah
ROBERT E. LATTA, Ohio G.K. BUTTERFIELD, North Carolina
CATHY McMORRIS RODGERS, Washington JOHN BARROW, Georgia
GREGG HARPER, Mississippi DORIS O. MATSUI, California
LEONARD LANCE, New Jersey DONNA M. CHRISTENSEN, Virgin
BILL CASSIDY, Louisiana Islands
BRETT GUTHRIE, Kentucky KATHY CASTOR, Florida
PETE OLSON, Texas
DAVID B. McKINLEY, West Virginia
CORY GARDNER, Colorado
MIKE POMPEO, Kansas
ADAM KINZINGER, Illinois
H. MORGAN GRIFFITH, Virginia
_____
Subcommittee on Health
JOSEPH R. PITTS, Pennsylvania
Chairman
MICHAEL C. BURGESS, Texas FRANK PALLONE, Jr., New Jersey
Vice Chairman Ranking Member
ED WHITFIELD, Kentucky JOHN D. DINGELL, Michigan
JOHN SHIMKUS, Illinois EDOLPHUS TOWNS, New York
MIKE ROGERS, Michigan ELIOT L. ENGEL, New York
SUE WILKINS MYRICK, North Carolina LOIS CAPPS, California
TIM MURPHY, Pennsylvania JANICE D. SCHAKOWSKY, Illinois
MARSHA BLACKBURN, Tennessee CHARLES A. GONZALEZ, Texas
PHIL GINGREY, Georgia TAMMY BALDWIN, Wisconsin
ROBERT E. LATTA, Ohio MIKE ROSS, Arkansas
CATHY McMORRIS RODGERS, Washington JIM MATHESON, Utah
LEONARD LANCE, New Jersey HENRY A. WAXMAN, California (ex
BILL CASSIDY, Louisiana officio)
BRETT GUTHRIE, Kentucky
JOE BARTON, Texas
FRED UPTON, Michigan (ex officio)
(ii)
C O N T E N T S
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Page
Hon. Joseph R. Pitts, a Representative in Congress from the
Commonwealth of Pennsylvania, opening statement................ 1
Prepared statement........................................... 3
Hon. Michael C. Burgess, a Representative in Congress from the
State of Texas, opening statement.............................. 5
Hon. Frank Pallone, Jr., a Representative in Congress from the
State of New Jersey, opening statement......................... 5
Hon. Joe Barton, a Representative in Congress from the State of
Texas, opening statement....................................... 7
Prepared statement........................................... 8
Hon. Tim Murphy, a Representative in Congress from the
Commonwealth of Pennsylvania, opening statement................ 9
Hon. Henry A. Waxman, a Representative in Congress from the State
of California, opening statement............................... 9
Hon. Fred Upton, a Representative in Congress from the State of
Michigan, opening statement.................................... 223
Witnesses
Jeffrey E. Shuren, Director, Center for Devices and Radiological
Health, Food and Drug Administration........................... 11
Prepared statement........................................... 13
Answers to submitted questions............................... 224
David Perez, President and Chief Executive Officer, Terumo BCT... 79
Prepared statement........................................... 82
Elisabeth M. George, Vice President, Global Government Affairs,
Regulations, and Standards, Philips Healthcare................. 88
Prepared statement........................................... 90
Ralph F. Hall, Professor of Practice, University of Minnesota Law
School......................................................... 100
Prepared statement........................................... 102
Ross Jaffe, Managing Director, Versant Ventures.................. 129
Prepared statement........................................... 131
Aaron S. Kesselheim, Assistant Professor of Medicine, Harvard
Medical School, Division of Pharmacoepidemiology and
Pharmacoeconomics, Brigham and Women's Hospital................ 155
Prepared statement........................................... 157
Art Sedrakyan, Associate Professor and Director, Patient-Centered
Comparative Effectiveness Program, Weill Cornell Medical
College and New York Presbyterian Hospital..................... 164
Prepared statement........................................... 166
Lisa Swirsky, Senior Health Policy Analyst, Consumer Union....... 174
Prepared statement........................................... 176
James Shull, Browns Mills, New Jersey............................ 182
Prepared statement........................................... 184
Submitted Material
Letter, dated October 14, 2011, from Alan Mertz, President,
American Clinical Laboratory Association, to Mr. Burgess,
submitted by Mr. Burgess....................................... 72
Letter, dated October 14, 2011, from Michael A. Peat, Managing
Director, Texas Gulf Coast Business Unit, Quest Diagnostics
Inc., to Mr. Burgess, submitted by Mr. Burgess................. 73
Letter, dated October 14, 2011, from Donald E. Horton, Jr., Vice
President, Public Policy & Advocacy, Laboratory Corporation of
America, to Mr. Burgess, submitted by Mr. Burgess.............. 74
Letter, dated October 18, 2011, from Edward R. Ashwood, President
and CEO, ARUP Laboratories, Inc., to Mr. Burgess, submitted by
Mr. Burgess.................................................... 75
Letter, dated October 25, 2011, from Eric Rieders, President and
CEO, NMS Labs, to Mr. Burgess, submitted by Mr. Burgess........ 76
Letter, dated November 3, 2011, from Sherri J. Bale, Managing
Director, GeneDx, to Mr. Burgess, submitted by Mr. Burgess..... 77
Letter, dated January 25, 2012, from Mark S. Birenbaum,
Administrator, American Association of Bioanalysts and the
National Independent Laboratory Association, submitted by Mr.
Burgess........................................................ 78
Statement, dated February 15, 2012, of Michael A. Carome, Deputy
Director, and Sidney M. Wolfe, Director, Health Research Group,
Public Citizen, submitted by Mr. Pallone....................... 197
Statement, dated February 15, 2012, on behalf of the American
Urogynecologic Society and the American Congress of
Obstetricians and Gynecologists, submitted by Mr. Pallone...... 207
Article, ``Postmarketing Surveillance of Medical Devices--Filling
in the Gaps,'' undated, in The New England Journal of Medicine,
submitted by Mr. Pallone....................................... 211
Article, ``Regulation of Medical Devices in the United States and
European Union,'' undated, in The New England Journal of
Medicine, submitted by Mr. Pallone............................. 214
REAUTHORIZATION OF MDUFA: WHAT IT MEANS FOR JOBS, INNOVATION, AND
PATIENTS
----------
WEDNESDAY, FEBRUARY 15, 2012
House of Representatives,
Subcommittee on Health,
Committee on Energy and Commerce,
Washington, DC.
The subcommittee met, pursuant to call, at 10:17 a.m., in
room 2322 of the Rayburn House Office Building, Hon. Joe Pitts
(chairman of the subcommittee) presiding.
Members present: Representatives Pitts, Burgess, Shimkus,
Rogers, Murphy, Blackburn, Gingrey, Latta, McMorris Rodgers,
Lance, Cassidy, Guthrie, Barton, Bilbray, Bass, Pallone,
Dingell, Towns, Engel, Capps, Schakowsky, Matheson,
Christensen, and Waxman (ex officio).
Staff present: Clay Alspach, Counsel, Health; Nancy Dunlap,
Health Fellow; Paul Edattel, Professional Staff Member, Health;
Debbee Keller, Press Secretary; Ryan Long, Chief Counsel,
Health; Carly McWilliams, Legislative Clerk; Chris Sarley,
Policy Coordinator, Environment and Economy; Heidi Stirrup,
Health Policy Coordinator; Alli Corr, Democratic Policy
Analyst; Eric Flamm, FDA Detailee; Karen Nelson, Democratic
Deputy Committee Staff Director for Health; and Rachel Sher,
Democratic Senior Counsel.
Mr. Pitts. This subcommittee will come to order.
The Chair recognizes himself for 5 minutes for an opening
statement.
OPENING STATEMENT OF HON. JOSEPH R. PITTS, A REPRESENTATIVE IN
CONGRESS FROM THE COMMONWEALTH OF PENNSYLVANIA
Congress first authorized a medical device user fee program
in 2002, in the Medical Device User Fee and Modernization Act,
MDUFMA. We last reauthorized the program in the Medical Device
User Fee Amendments of 2007, MDUFA, which expires September 30,
2012.
While I am glad that FDA and industry have reached recently
a proposed medical device user fee agreement, the committee did
not receive it by the January 15, 2012, deadline, as set in
statute. As it is already late, I would encourage FDA and the
administration to expedite their review of the agreement so
that the committee receives it at the earliest possible date.
The proposed agreement will provide $595 million in user
fees for fiscal year 2013 through fiscal year 2017, a sum that
is more than double the current user fee level of $287 million.
A key goal of the agreement is to increase predictability
and transparency. Under the agreement, together with regular
Congressional appropriations, FDA should be able to hire 240
full-time review process employees, including 140 reviewers
specifically for devices, over 5 years. The increased user fees
will pay for additional training for device reviewers and
information technology upgrades to improve the review process.
With these new resources, FDA has agreed to measure review time
in calendar days, not FDA days, which is an important step to
providing increased predictability.
Under the proposed agreement, FDA and industry will
communicate more often, and earlier in the review process,
where FDA will provide the feedback that manufacturers need to
go forward.
The United States is the world leader in medical device
innovation. This not only benefits patients who need new,
innovative treatments, it benefits our economy. In 2008,
according to the Lewin Group, the medical device industry
employed 422,778 workers nationwide, paid $24.6 billion in
earnings, and shipped $135.9 billion worth of products.
In 2008, in my home State of Pennsylvania, the medical
device industry employed 22,233 people and paid Pennsylvania
workers over $1.1 billion in earnings.
These are good jobs. Nationally, jobs in medical technology
pay almost 40 percent higher compared to the national earnings
average.
What is best for patients and what is best for jobs is to
have a device review process that is clear, transparent,
predictable and accountable, and I hope that that is what the
proposed agreement accomplishes.
I would like to thank all of our witnesses on today's
panels.
[The prepared statement of Mr. Pitts follows:]
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Mr. Pitts. I would like to yield the remaining time to Dr.
Burgess, the vice chairman of the committee.
OPENING STATEMENT OF HON. MICHAEL C. BURGESS, A REPRESENTATIVE
IN CONGRESS FROM THE STATE OF TEXAS
Mr. Burgess. Thank you, Mr. Chairman, and Dr. Shuren,
again, thank you for being here. You are going to hear today
some concerns from people on the dais and from our subsequent
panel, from patients and innovators.
As the chairman points out, funding was increased in fiscal
year 2008 and fiscal year 2010 by nearly 35 percent, and during
that time the average review time for lower-risk devices
increased by 43 percent, higher-risk devices by 75 percent, so
we have got an official Washington conundrum. Resources are
increasing, performance is decreasing, and you need to be the
very best you can but it doesn't look like we are there yet.
Delays in reviews through inconsistencies certainly harm public
health but they also stifle innovation and cost jobs.
We don't want the FDA to approve anything that harms
patients, and that is your mission, but a little predictability
could go a long way. The industry should not have to double
user fees in order to get the very basics of customer service.
So the question is, have you become more interactive,
predictable and innovative? Those should be the goals of the
basic agreement but they also are tenets of a well-run
organization. We worry about the jurisdictional creep that has
been going on where you seek to grab as much regulatory
territory as possible, oftentimes through draft guidance,
absent legislative direction. Things like mobile apps and
laboratory-developed tests are things that you want to do but
we are not sure you are doing what you are supposed to do. We
shouldn't enable your efforts to duplicate efforts of other
Federal agencies.
Mission creep may be a cry for help, and Doctor, this
morning we are here to try to provide that help for you. But
some days we wonder if you don't need a bigger check but you
need a check on what is exactly happening at the level of your
agency. We want to help. I think we all admit that there are
problems in our device approval regimen that hurt patients and
it is just critical that we get it right for them.
I yield back the balance of my time, Mr. Chairman.
Mr. Pitts. The Chair thanks the gentleman and now
recognizes the ranking member of the subcommittee, Mr. Pallone,
for 5 minutes for opening statement.
OPENING STATEMENT OF HON. FRANK PALLONE, JR., A REPRESENTATIVE
IN CONGRESS FROM THE STATE OF NEW JERSEY
Mr. Pallone. Thank you, Chairman Pitts. I welcome every
here for our third installment of the UFA hearings.
Today we will be discussing the reauthorization of the
Medical Device User Fee Agreement, known as MDUFA, and let me
say at the outset that we are all very relieved and encouraged
by the current circumstances. There was grave concern that the
parties would be unable to reach a compromise, and I am happy
that things are moving forward.
While there is still no legislative language, there is an
agreement in principle that we will be discussing at length. It
includes $595 million in fees over 5 years, specific goals for
total review times, additional meetings with sponsors, third-
party analysis of the FDA's review process as well as other
program improvements. In addition, I understand that the
additional funding would allow FDA to hire over 200 new full-
time workers by the end of the 5-year program.
Now, we have consistently heard for a long time about the
need for FDA to improve the predictability, consistency and
transparency of its premarket review program. This agreement
will not solve all of those issues overnight but it certainly
sets FDA on a good path moving forward with important tools and
more resources at their disposal. It also provides the industry
with some much-needed insight into the review process and
better metrics to measure the FDA's performance, and these are
quality enhancements that should allay those concerns.
I know that Congress and the FDA greatly appreciate the
industry's investment in this program. This proposal represents
a strong compromise, and I commend the hard work of both
parties in getting to this place I am confident will help the
agencies continue to improve efficiencies.
Let me also say that I have been encouraged by FDA's
commitment both over the past year and as part of this user fee
agreement to recognize the need for some internal
transformations. Change doesn't happen overnight, and
regardless, Dr. Shuren, your center has been more than willing
to listen and learn from member stakeholders and industry on
how to shift and adapt in ways to make these processes better
for companies and consumers. You have recognized some of the
inadequacies of the agency and maintained an open mind on
fixing what is broken. At the same time, you have also
maintained the policies are important to patient safety and
device effectiveness. You and the Commissioner were kind enough
to visit my district and talk one on one with me and New Jersey
companies about these processes, so I appreciate that and I
look forward to working with you to continue to improve the
center.
Today's hearing will also touch upon a number of FDA policy
proposals from my Republican colleagues. In general, I have
concerns with some of these bills and I look forward to
discussing them further. Specifically, I wonder whether these
proposals could make it difficult for the agency to meet its
negotiated commitments. I also think it is critical we
understand at length the intended impact, justification and
potential unintended consequences of these proposals before
moving forward.
I will just close by stating what I have said a number of
times. I agree that MDUFA is of the utmost importance. I agree
that FDA should facilitate an environment that doesn't create
added unnecessary burdens upon innovating companies, but we
must not make FDA policy changes at the expense of patient
safety. The public health must be our number one goal above all
else. We need to take a long, hard look at any potential policy
that could make it more difficult for FDA to protect patient
safety, and I know there are a number of witnesses joining us
today that will talk about that important aspect. I look
forward to that.
But I wanted to especially welcome Jim Shull--I hope I am
pronouncing it right--from Browns Mills, New Jersey, who is
here to share his personal story.
Thank you, Mr. Chairman. I yield back.
Mr. Pitts. The Chair thanks the gentleman and now
recognizes the chair emeritus of the full committee, Mr.
Barton, for 5 minutes for opening statement.
OPENING STATEMENT OF HON. JOE BARTON, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF TEXAS
Mr. Barton. Thank you, Mr. Chairman. I am not going to take
5 minutes. I believe I am supposed to yield to Dr. Murphy.
I have an opening statement that I will put in the record.
I hate to be the skunk at the garden party, but every now and
then I am. These user fees are not something that have been on
the books for a hundred years. We first put them in place in
2002 and we have reauthorized them once. Currently, it is about
$287 million, I believe. I think it is a lot to ask this
committee to swallow a doubling of the user fee budget to
almost $600 million. I checked yesterday, and I understand that
it may be the tradition but I couldn't find that any member or
any staff member of the majority or the minority had been
involved in these negotiations with the FDA and the industry.
If we came in and asked to double the income tax receipts, we
would be laughed out of Congress, and to have a proposal put
forward that doubles the user fee with the performance or lack
thereof that has accompanied the last 3 or 4 years is something
that I am not going to condone.
Now, I haven't talked with Chairman Upton or Chairman
Pitts, and I am sure that there is another side to the story.
But put me down as extremely skeptical that this is a good deal
for the consumer or for the small medical device industry.
I had a company in my office just this week, or late last
week actually, that has been making a device and marketing it
for 30 years, and all of a sudden now they have been asked to
have to go through the entire premarket approval process for
something. I just don't accept that.
So Mr. Chairman, I am extremely glad that you are holding
this hearing but don't ask this member to rubberstamp a
doubling of a user fee when we have the program performance or
lack thereof at this FDA.
And with that, I would yield the balance of the time to Dr.
Murphy of Pennsylvania.
[The prepared statement of Mr. Barton follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
OPENING STATEMENT OF HON. TIM MURPHY, A REPRESENTATIVE IN
CONGRESS FROM THE COMMONWEALTH OF PENNSYLVANIA
Mr. Murphy. I thank the gentleman.
A few weeks ago, several of my colleagues and I met with
Professor Ralph Hall, who will be testifying a little bit later
today on a panel. At that meeting, Professor Hall explained how
the review process at the FDA is driving investment in medical
technologies overseas as well as sending jobs overseas. Now,
according to Professor Hall, 40 percent of venture capitalists
have already reduced investment in medical technology in the
United States and many more are planning this. About 61 percent
of venture capitalists cite regulatory challenges with the FDA
as having the greatest impact on their investment decisions.
Now, this may seem like financial jargon but in reality, it
points to a tragic bottom line: no money, no research, no
treatments, no cures. This is about saving lives of people with
untreatable diseases who are waiting in line for Washington's
rules and bureaucracy to get out of the way and for the
treatment and cures to move forward. It is cruelty, not
comfort, when a doctor must tell a patient that bureaucratic
barriers prevent patients in the United States from getting the
treatment that they need.
We need to and we must help American patients have better
access to the latest, safest medical advancements while also
improving FDA's review process to allow more investment in U.S.
medical technology. It is something we ought to be doing out of
compassion for people who are sick.
And with that, I yield back to Mr. Barton.
Mr. Barton. I have no further comments. If there are other
members, I will be happy to yield, Mr. Rogers or Mr. Latta,
anybody? I yield back to the chairman.
Mr. Pitts. The Chair thanks the gentleman. The Chair now
recognizes the ranking member of the full committee, Mr.
Waxman, for 5 minutes for an 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, for holding
this important hearing.
Our goal today is to start the process of reauthorizing the
Medical Device User Fee Act, and I commend FDA and the industry
for finally coming together to agree on a user fee proposal. I
know it was a hard-fought compromise and I look forward to
seeing the details. But I am pleased that there has been an
agreement because I have very little faith that Congress is
going to provide the appropriations for the FDA to do the job
without a user fee. I would prefer we do it that way, and those
who don't like the user fee will have to acknowledge that FDA
will be short-funded and we won't get these devices approved as
quickly as possible.
The funds collected under this act will provide FDA's
device program with critical dollars that enable the agency to
fulfill its public health mission: to ensure that only safe and
effective medical devices are marketed in the United States.
That is our essential goal here. We should work together on a
bipartisan basis to get it done.
The real compassion in this country is to make sure that we
can get drugs and devices that work and that are safe to
consumers, not just to get them out on the marketplace because
it is no one's benefit to have drugs that are not safe or
medical devices that are not safe or effective. The FDA, the
device industry and American patients are counting on us to do
our job.
I am concerned that some may try to hijack the
reauthorization to advance proposals that would put the health
of patients at risk. Last year, Republican members of the
committee introduced a slate of 10 bills that would make
significant and harmful changes, in my view, in FDA's device
program. Unless we can reach consensus on these proposals, they
should not be inserted into this must-pass reauthorization.
The newspapers are full of articles about the dangers of
improperly designed medical devices. The prestigious Institute
of Medicine concluded that our medical device laws need to be
significantly strengthened. But many of these bills ignore the
need for reforms that would protect patients. Instead, they
read like a wish list assembled by lobbyists for the device
industry.
The device industry claims that FDA regulation is killing
jobs, stifling innovation, and depriving American patients of
new medical devices. But there is no evidence to back these up
except anecdotes. Anecdotes from some individual companies are
not enough. And I think the industry knows that they need an
FDA that is going to do its job if they are going to have
credibility in the marketplace.
I have been appalled by the quality of the so-called
``studies'' that industry is using to advance these bills. Last
July, I asked the editors of our Nation's top medical journals
to examine the methodology used in the leading industry papers
asserting that FDA is too slow, burdensome, and unpredictable.
The editors said there were serious methodological flaws in
both studies--biased samples, small sample size and botched
statistical analysis, just to name a few--rendering them
essentially useless as part of any discussion of FDA's
regulatory system. None of the editors felt that the
methodology of these studies was worthy of publication in a
peer-reviewed journal, and yet they are put forward as a reason
why we ought to change the law here in Congress.
Many in the device industry argue that Europe should be our
model and they say new technologies are available years before
they are on the market in the United States. But just
yesterday, the New England Journal of Medicine published a
study by Dr. Aaron Kesselheim finding numerous examples of
high-risk devices that were first approved in the E.U. but
either showed no benefit, or, worse, had substantial safety
risks. I am glad that Dr. Kesselheim is here today to testify
about this study.
FDA's job is to protect the public health. Part of
advancing public health is helping manufacturers win approval
for innovative new devices. But FDA's core responsibility is
ensuring that only safe and effective devices are permitted on
the market.
When FDA falls short and allows dangerous devices like
surgical mesh and metal-on-metal hip implants to be implanted
in patients, the suffering of victims can be incalculable. That
is why I joined with Mr. Pallone, Mr. Dingell and Ms. DeGette
in requesting that the committee hear from witnesses about the
risks from dangerous devices, and I want to thank Subcommittee
Chairman Pitts and full Committee Chairman Upton for working
with us to allow these witnesses to testify today on the second
panel.
The reauthorization of MDUFA should be bipartisan, so I
urge all members of the committee to work together on this
critically important program.
Thank you, Mr. Chairman.
Mr. Pitts. The Chair thanks the gentleman.
Our first panel will have just one witness, Dr. Jeffrey
Shuren, Director of the Center for Devices and Radiological
Health at the FDA. Dr. Shuren is accompanied today by Mr.
Malcolm Bertoni, Assistant Commissioner for Planning for the
Office of the Commissioner. We are happy to have you with us
today, Dr. Shuren. You are recognized for 5 minutes to
summarize your testimony. Your written statement will be
entered into the record.
STATEMENT OF JEFFREY E. SHUREN, DIRECTOR, CENTER FOR DEVICES
AND RADIOLOGICAL HEALTH, FOOD AND DRUG ADMINISTRATION
Mr. Shuren. Mr. Chairman and members of the subcommittee, I
am Dr. Jeff Shuren, Director for the Center for Devices and
Radiological Health, or CDRH, at the FDA. Thank you for the
opportunity to testify today.
I am pleased to tell you that on February 1, FDA and
representatives from the medical device industry reached an
agreement in principle on proposed recommendations for the
reauthorization of the Medical Device User Fee Act, or MDUFA.
These recommendations would authorize FDA to collect $595
million over 5 years to help fund a portion of the agency's
medical device review program with FDA agreeing to certain
overall performance goals. The final details of the agreement
will be resolved very soon, and as required by law, we will
hold a public meeting and seek public comment on the proposed
package before sending a final package to Congress.
When I came to CDRH in 2009, in response to concerns
expressed by industry and others, we initiated a review of our
device premarket review programs. The following year, we
released two reports that concluded, as I have testified
before, that we had not done as good a job managing the review
programs as we should have. The number one problem we found was
insufficient predictability, which was leading to
inefficiencies, higher cost to industry and FDA, and sometimes
delays in bringing safe and effective products to market.
In January 2011, we announced a plan with 25 specific
actions that we would take that year to improve the
predictability, consistency and transparency of our premarket
programs. As of February 2012, 75 percent of these actions plus
eight additional actions are already completed or well
underway. They are intended to create a culture change toward
greater transparency, interaction and the appropriate balancing
of benefits and risk. They focus on assuring predictable and
consistent decision-making and application of the least-
burdensome principle and implementing more efficient regulatory
processes.
We believe these actions have had and will have a visible,
positive impact by providing greater predictability about data
requirements through guidance, reducing unnecessary or
inconsistent data requests through training and policy and
process changes, implementing policies that lead to
appropriately balanced benefit-risk determinations, using
external experts more extensively and effectively, creating
incentives to conduct clinical studies first in the United
States, speeding up clinical trial approval decisions and
implementing the innovation pathway.
Preliminary data indicates that the actions we have taken
have started to bear fruit. For example, the backlog of 510(k)
submissions that had been steadily increasing from 2005 to 2010
decreased for the first time last year. However, we still have
much work to do.
Reauthorization of MDUFA will provide the resources that
CDRH needs to continue improving the device review programs and
help reduce the high staff turnover that has adversely affected
review predictability and consistency. The proposed MDUFA
recommendations we have agreed upon with industry will also
include several important process improvements. For example, if
a performance goal on a device application is missed, the MDUFA
proposal would require FDA and applicants to work out a plan to
complete work on the submission, ensuring that no submission is
left behind, and requiring new substantive interaction between
FDA and an applicant halfway through the targeted time for
reviewing the application would help to assure sufficient time
for the applicant to properly respond to appropriate questions.
Clear criteria for when FDA will refuse to accept a complete
application means more efficient use of resources to the
benefit of both FDA and industry. These and other proposed
enhancements are intended to achieve a shared outcome goal of
reduced average total time to decision, which we and industry
believe is an important indicator of a successful premarket
review program.
The agreement in principle we have reached with industry
strikes a careful balance between what industry agreed to pay
and what FDA can accomplish with the amount of funding
proposed. However, we are concerned that even if device user
fee resources are increased under MDUFA III, additional new
legislative mandates imposed on CDRH could divert resources and
undermine FDA's ability to achieve the new performance goals.
When PDUFA was last reauthorized in 2007, the addition of new
policy-related requirements ultimately resulted in FDA's drug
review program having to temporarily suspend meeting its PDUFA
review goals in order to meet the statutory mandates. We want
to avoid such a situation so that CDRH can focus on meeting the
ambitious new proposed PDUFA program goals and achieving timely
patient access to safe and effective devices, which is an
objective that we share with industry, health care
practitioners, patients and consumers, and I know you as well.
Mr. Chairman, I commend the subcommittee's efforts and am
pleased to answer any questions the subcommittee may have.
[The prepared statement of Mr. Shuren follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Mr. Pitts. The Chair thanks the gentleman, and I will now
begin the questioning and recognize myself for 5 minutes for
that purpose.
Dr. Shuren, Chairman Upton and I have set a deadline of
reauthorizing the user fees by the end of June. We received the
three other user fee proposals by January 15th but we did not
receive the medical device user fee proposal as required under
statute. Given the need to reauthorize the user fees as soon as
possible, let me ask you a two-part question. Number one, when
will FDA send us the legislative language and proposed
agreement for the medical device user fee so that the committee
can begin its work, and two, what specific steps does the
administration plan to take to expedite the process so the
committee can get the device information as soon as possible?
Mr. Shuren. So the plan we have put in place and what we
have asked of the administration is for expedited review of a
proposal so that we can get the proposal out to you and out to
the public as we move into March, and so you will be able to
see what we are proposing, we will get the public comments, we
will wrap up on that. We have to follow that process. And then
we will have the final package. But you will be able to see
that proposed package, and our goal is to try to do that in the
next few weeks.
Mr. Pitts. By mid-March?
Mr. Shuren. That is approximately the time, and that is
what we have been asking the administration to support us in
doing.
Mr. Pitts. All right. The medical device legislation
introduced by our committee members and Mr. Paulson of
Minnesota contains critical improvements aimed at making FDA's
regulation of medical devices both premarket and postmarket
more predictable. This predictability is critical to getting
life-saving devices to our Nation's patients and their
families, as we have heard from Marty Conger, Carol Murphy and
Pam Sagan at our O&I hearing in July. It is also critical in
keeping medical device jobs in the United States, as we have
heard from numerous innovators throughout the past year.
We have heard some argue that these device bills aren't
necessary because FDA is fixing the problem. That is a little
hard to believe. For example, that is what FDA has told us
about the pre-amendment class III devices for the past 20
years, and the problem still isn't fixed. Class III devices are
still going through the 510(k) process. Frankly, we don't have
20 years or even 6 months to wait for FDA to fix the problems.
Our Nation's patients and innovators need help now. So my
question is, will you commit to working with us on this
legislation so we can help our Nation's patients and help keep
American device jobs here in the United States?
Mr. Shuren. Mr. Chairman, we would welcome the opportunity
to work with you on legislation.
Mr. Pitts. We will follow up with that. Thank you.
What is the status of the unique device identifier rule?
Mr. Shuren. So we have completed the rule. It is now
currently under review at the administration and we are waiting
for their approval to move forward with it.
Mr. Pitts. Five years ago, the committee passed the
reauthorization of the medical device user fee, and when we
voted for that bill, we did so expecting that FDA would meet
its end of the deal. It appears that that hasn't happened. FDA
has failed to meet many of the MDUFA goals, and during the past
5 years, we have seen the total time it takes from submission
to FDA decision rise dramatically. Given that track record, why
should we believe that you are going to meet the goals you
agreed to in the proposed user fee package?
Mr. Shuren. Well, I won't belabor the point that there are
some things that but for the user fee act, we would not have
been able to enhance, but we agree, we are not happy with where
the program is; industry is not happy with where it is. There
are fundamental problems right now. Some of that is on our
part, and that is why I made a public commitment to make those
changes and started last year, regardless of whether we saw
user fee dollars or not, and we are moving forward on those.
But by the same token, there are problems with the program
that we cannot solve without funding. I have high staff
turnover rates, just like the drug program had 10 years ago,
because of too much work on their plate. We don't have enough
managers to provide good oversight. The ratios are running from
1:14 up to 1:25 under a front-line manager. That is untenable
in any business, and I can't solve that with changes in
policies and processes. I can only change that with having the
people to do the work, enough managers and enough staff to do
the work. That is what comes out of the user fee dollars. And
together, making those program improvements that we have
underway, having the dollars from industry and having smart
performance goals in place can help us achieve a successful
program and the outcome we all want to see from device review.
Mr. Pitts. I have just 20 seconds left. What metrics are
included in the agreement to make sure you can meet your goal?
Mr. Shuren. In the MDUFA agreement?
Mr. Pitts. Yes.
Mr. Shuren. So there are performance goals that pertain to
FDA time but also to the average total time to the decision. So
these are the things that happen that are not quite under our
control but by putting in certain process improvements of
greater engagement and interaction with industry, with the
companies as we move forward during the review, our hope is
that with that and with the more staff on board, we can
actually bring down the total time for making a decision, which
we think is an important indicator, through those improvements.
We also have goals that go towards--it is predominantly to the
performance on different kinds of applications.
Mr. Pitts. The Chair thanks the gentleman and recognizes
the ranking member, Mr. Pallone, for 5 minutes for questions.
Mr. Pallone. Dr. Shuren, I wanted to ask about the 510(k)
process, and first commend you for the focus you have given to
improving it. I have been interested in how to fix it for a
long time. In fact, when I was the chairman of the
subcommittee, we held a hearing in 2009. Quite frankly, both
before and after that hearing, I was of the opinion that the
510(k) process was broken, so I am glad that FDA has focused
its attention on resources and how to improve it.
I have seen your 510(k) action plan and the amount of work
that CDRH did on this topic is pretty impressive. What is your
sense of the 510(k) program now? Is it operating better? Is
there more predictability and consistency? And what steps on
your action plan would you categorize as game changers?
Mr. Shuren. So the program is not where we would like it to
be. We are not seeing the performance from it that we would
like to have, but we are starting to see some early indicators,
if you will, the canaries in the coalmine, suggesting instead
of them dying from gas, that actually they are doing better. So
starting almost 10 years ago, we saw the requests for
additional information on 510(k)'s go up and up and up
steadily. We saw total review times going up and up and up. We
saw the backlog of 510(k)'s going up and we saw the percent of
510(k)'s being cleared going down. In 2011, for the very first
time we are seeing the percent of additional requests on
510(k)'s starting to dip for the first time the other
direction. We are seeing that the percent of 510(k)'s being
cleared has been going up. I put all this information, by the
way, in my written testimony. In 2012, that number, that
percent of clearance actually went up beyond 2011. We are
seeing the backlog go down. So all of these are early signs but
I don't think you are going to see the real benefit from it
until many of our policies go into effect.
Game changers right now--simple smart business process
improvements to assure that critical decisions like asking for
additional information are not made in the lowest parts of our
center but they are made at the right level of management,
which is why I need enough managers to provide that oversight.
In fact, we created a Center Science Council of our most senior
people to oversee the most important decisions. We are putting
in new policies to incentivize starting clinical trials in the
United States earlier. You get the clinical studies started
here first, you keep the technology here because the companies
come back to the same doctors over and over again, and also
having benefit-risk framework that is much more focused on
taking into account what patients are willing to tolerate for
risk because they are the ones who get the devices, not my
reviewers.
Mr. Pallone. Thank you. Let me ask you about the conflict
of interest in these scientific experts for the advisory
panels. We have heard from a number of parties that the
conflict of interest provisions are not working and are
excluding legitimate experts. When the Commissioner was here 2
weeks ago, she indicated that there have been challenges at FDA
in filling the advisory panels. Would you agree that CDRH is
having similar challenges?
Mr. Shuren. We do face challenges in moving forward, which
is why we agree with you. You consider this an important issue;
we consider this an important issue. And although we have not
found a legislative fix yet that has a significant difference,
we think this is something worth exploring. One of the reasons
I would like to take the chairman up on his offer to work on
legislation focused on this area is one of those areas. We are
looking at internal process changes, are there other things we
can be doing to sort of reduce those challenges we face.
Mr. Pallone. I know when you testified before the Senate
Health Committee in November, you indicated willingness to
engage with the Senators, so I guess I am getting the same
assurance from you today on this.
Mr. Shuren. Yes.
Mr. Pallone. All right. Chairman Pitts talked about the
UDI, and I think it is unfortunate that after 5 years, I think
we should be closer on implementation on what I consider a very
critical component. But what I wanted to ask you is, could you
explain how UDI will interact with other postmarket authorities
that FDA has in the device space and other initiatives that you
have underway?
Mr. Shuren. So unique device identifier will allow us to
link the use of a device with a patient's experience with the
device. So data is collected every day as a part of routine
clinical practice, and we can't tap into that without a UDI.
That is why that unique device identifier is a game changer,
and it will allow us to move forward to have more robust
postmarket surveillance systems that then industry and we can
take advantage of and health practitioners and others in the
following ways. If we have more robust postmarket surveillance,
when there are problems, if we can identify them more quickly
and get on top of them, it doesn't mean the device comes off
the market. It means that we address it, and you don't get the
front-page stories in the newspapers because you don't have so
many people exposed. You have a better infrastructure that
allows companies to conduct postmarket studies at lower cost
because the infrastructure is there, and it will allow us to
make better use of postmarket data to reduce the burden for
premarket data requirements for some devices. In fact, if we
are properly authorized, we may be able to even shift some of
the premarket data requirements to the postmarket setting. But
these are all things we could do in the future and a unique
device identifier is critical to making that happen.
Mr. Pallone. Thank you.
Mr. Pitts. The Chair thanks the gentleman and recognizes
the vice chairman of the committee, Dr. Burgess, for 5 minutes
for questions.
Mr. Burgess. Thank you, Mr. Chairman.
Dr. Shuren, in this committee we worked on this a lot over
the years, and it seems like there is a repetitive stream of
people in my office talking about difficulties they are having
in this arena. So I don't think there is any question that we
have a problem. The problem generally seems to be with
predictability and consistency at your agency, and whether we
all agree with where the problems are and whether we all agree
with how much activity is leaving our shores, I don't think
there is any question that some is, and the President's own
Jobs Council has raised this issue, and specifically they
commented, quoting from them, ``Our medical innovation system
is in jeopardy. Investment in life science area is declining at
an alarming rate because of the escalating cost, time and risk
of developing new drugs and devices. While many factors
contribute to the decline, an important factor is the
uncertainty surrounding the FDA regulatory environment.''
So this is not House Republicans, this is the President's
Jobs Council. This is the administration that is voicing
concern with the predictability and consistency within the FDA.
How do you respond to what the Jobs Council is telling us?
Mr. Shuren. I think you can add me and my own staff, who
have our own concerns about the program as well, and I will say
in terms of the Jobs Council, when they then came out and said
what things you might want to look at for the medical device
program, one of their recommendations was to have a benefit-
risk determination framework that is much more focused on
looking at patient tolerance for risk. We appreciate that,
because when they came out with that recommendation, we had
actually already proposed such a framework over the summer. In
fact, we are finalizing it right now and we have committed and
are already set to put out the final document and implement it
come the end of March.
Mr. Burgess. But again, you know, I just can't stress this
enough. There is a steady stream of people that come in to see
me and I suspect other Members of Congress have similar stories
where there is a problem, and the problem seems to be centered
at the Center for Devices and Radiological Health. It is
clearly something that needs your highest attention and I look
forward not just to your framework but we actually look forward
to some performance on this, and as I reference in the opening
statement, we can't just be upping in the dollars and
decreasing the performance, and unfortunately, that seems to be
the direction we are going.
Let me ask you a couple of specifics on some of the things
I referenced. Some of the draft guidance that is coming out of
your area where it appears that you are increasing your
jurisdiction and you territory, and I am not sure that is in
everyone's best interest and specifically in your best
interest, but what about the draft guidance for industry and
staff on the in vitro diagnostic products that are labeled for
research use only and investigational use only? This is
something that came out of your office, and depending upon the
stage of development, such components are officially labeled
research use only, investigational use only. That means they
are neither sold nor marketed as clinical devices nor offered
as services such as laboratory-developed tests, but they may be
useful in developing new devices. So now it looks like your
agency is wanting to regulate even the devices that are used to
help develop the devices. Have I read that correctly?
Mr. Shuren. Well, components that are being used as a part
of the device are part of the device, and we regulate that. You
know, the policy----
Mr. Burgess. Well, let me ask you this then. Specifically,
what are some of the deficiencies that you saw that required
you to issue this draft guidance?
Mr. Shuren. That there were companies who were actually
saying that this particular device or analyte was for research
purposes. They were actually marketing it for commercial use.
So this policy is to clarify in terms of what you need to do to
be very clear on, is this truly for research and how you handle
that, or is this actually being used in patient care, and that
is what it is trying to clarify.
Mr. Burgess. And again, give us an idea of the scope of the
problem of this. Is this something that you are bumping up
against all the time or is this something that has happened and
you are trying to get in front of it?
Mr. Shuren. No, it is something we have been running into
and we continue to see, and that is why we have a policy to
clarify it.
Mr. Burgess. And can you provide us on the committee with
some examples of that so we can better understand why this
mission creep is going on at your center?
Mr. Shuren. We would be happy to come back and give you
some very specifics, give you a list of examples.
Mr. Burgess. And once again, this doesn't seem to be the
flexibility built into this. It is kind of an all-or-nothing
phenomenon, and one of the complaints we get is, there is no
flexibility within the Center for Devices and Radiological
Health. Is that something that you can help us with?
Mr. Shuren. First of all, I would say actually we are more
flexible than people give us credit for.
Mr. Burgess. Fair statement, because you are not getting
any credit at all right now.
Mr. Shuren. I know. I mean, I will give you an example. We
just recently approved a device for tears in the large artery
in the chest, and in terms of flexibility, we actually approved
that device based upon just 51 patients followed for just 30
days, very small, not randomized, no controls, and we did it in
less than 180 days. So the opportunities are there. The changes
we are trying to make in the program are also to ensure we have
flexibility where we need to do it but we are also consistent
in how we apply it, and like I said, we made some process
improvements that just went in the end of last year. There are
a lot of policy changes, good policy changes, but as you know,
as a Federal Government agency, we have to get public comment.
That is a good thing. We get lots of perspectives. The downside
is, it takes more time. So most of the things we are trying to
improve actually don't start getting finalized and kicking in
until this year.
Mr. Burgess. We want you to be consistent. That is part of
our goal as well, but I would appreciate you providing us some
data on this because some of the stuff we are hearing does not
comport with what you are telling us.
Thank you, Mr. Chairman. I will yield back. Maybe we will
have time for a second round.
Mr. Pitts. The Chair recognizes the ranking member of the
full committee, Mr. Waxman, for 5 minutes for questions.
Mr. Waxman. Thank you, Mr. Chairman.
Dr. Shuren, one of the bills included in the Republican
package would make significant changes to the device center's
so-called third-party review program. Currently, that program
permits third parties to review certain 510(k) applications and
provide recommendations to FDA on whether the agency should
clear a particular device, then FDA has 30 days to make a final
decision. That is what the law is now. The Republican bill
would alter this scheme to make the third party's
recommendation binding on FDA if FDA fails to respond in 30
days. The bill also would expand the types of devices that
these third parties are permitted to review to include
permanently implantable or life-sustaining or -supporting
devices. These outside reviewers are not currently allowed to
review these devices. I think these changes are very worrisome.
Would FDA be concerned about these kinds of changes to the
program?
Mr. Shuren. We are deeply concerned about these changes. I
mean, the hard stop, the default about their decision going
into effect if we don't make a decision actually can have the
perverse impact also of our being in a position to actually not
approve that product. That actually can spell the death knell
for the third-party review program, and I don't think that was
really the intent behind the bill but that is probably the
outcome that will likely happen.
Expanding the scope of the devices, I will tell you, there
are over a thousand devices that are already eligible for
third-party review. I mean, for 510(k), most of the 510(k)'s
would be eligible. We have gone through the different
categories and we have said almost 75 percent--the number may
in fact be higher--could then be eligible of that set for
third-party review. The problem is, that program hasn't worked
all too well, and one of the big challenges we face is that
those third parties don't have access to the confidential
information that we do. So as a result, they end up coming back
sometimes with decisions that are not fully informed.
For example, we may have already spoken to a company about
what they need to do, they came to us, and then they go
separately to a third party. They have no idea what that
conversation was, and as a result, they can't take advantage of
it. That is the challenge we really face in getting that
program----
Mr. Waxman. Well, I was concerned about this program when
we implemented it in 1997. I was never comfortable with the
concept of having external third parties who have the potential
for conflict of interests on their own reviewing these
important devices. So when I read this bill, I was very worried
about the changes that they put in place. After hearing your
further description of the impact it would have, it makes me
even more concerned and I feel very uncomfortable with these
further changes. It is like the XL pipeline resolution. When
you force a decision, you get a bad decision.
Another of the Republican slate of bills, the Premarket
Predictability Act of 2011, would make certain changes to three
key areas of FDA's device regulation: one, to FDA's oversight
over the investigational device exemption, two, to the so-
called least-burdensome provisions, and three, to the
procedures for appealing decisions through the Center for
Devices. I want to start with the changes to the least-
burdensome provision because those are the most troubling to
me.
This language was added to FDA's statute as part of the
1997 Food and Drug Administration Modernization Act at a time
when the industry was asserting that FDA was requiring too much
of device manufacturers and stifling innovation, strikingly
similar to what we hear still today, and in essence, these
provisions say that FDA must consider the least-burdensome
means of demonstrating that a device is effective when the
agency makes its approval or clearance decisions. So in other
words, FDA should consider whether clinical data are necessary
if there are other less-burdensome means for demonstrating that
a device can be marketed.
The Premarket Predictability Act would change this
provision by adding more-specific language like requiring FDA
to consider alternative approaches to clinical data in
evaluating device effectiveness ``in order to reduce the time,
effort and cost'' and directs FDA to consider ``alternatives to
randomized controlled clinical trials and the use of surrogate
endpoints'' when clinical data are necessary. This seems to me
overly prescriptive. Why would Congress be dictating to our
premier scientific regulatory body what type of clinical data
it should consider? It is also concerning because it seems that
it can make it harder for FDA to require clinical data even
when the agency believes it is necessary. I know that some of
the language in this bill was lifted from FDA's 2002 guidance
implementing the least-burdensome provision but it looks like
there were some changes to that language that could be
significant. Can you comment on this?
Mr. Shuren. Yes. First, let me say, I support the least-
burdensome principle. I think as a general matter, it is good
government and I support the policy we put back in our guidance
in 2002. That is why I reemphasized it to my staff last year in
email. It is why we are actually tailoring our guidance so we
apply it specifically to specific devices.
I do have concerns regarding this legislation because as it
is drafted, we are reading it as lowering the standards in the
United States for devices coming on the market, and that
concerns us, and also to the extent there is a difference in
that language in the bill versus our guidance, we have to
reconcile those differences, which means we have to change the
current policy. If folks think we have the right policy but we
are not applying it consistently, that is a different issue.
Now, we do have concerns about not applying it consistently and
that is why we put in process improvements to assure that we
are getting the right level of sign-off on any decisions for
actually trying to ask for more information or doing something
different than we did before, and oversight on decisions to
make sure we are applying the least-burdensome principle. That
is the problem we think needs to be fixed and that is the one
we are already working on.
Mr. Waxman. Thank you.
Thank you, Mr. Chairman.
Mr. Pitts. The Chair thanks the gentleman and recognizes
the chair emeritus of the committee, Mr. Barton, for 5 minutes
for questions.
Mr. Barton. Thank you, Mr. Chairman.
I think it is better to have a third-party review than to
have it sit on a bureaucrat's desk at the FDA and not get
reviewed at all, but that is just me.
Mr. Chairman, I want to put into the record a study of
October 2011 by the National Venture Capital Association and
the Medical Innovation and Competitive Coalition. I am going to
put the entire study in the record, but I want to just give
some of the bullet points.
This study was done in October of last year, and its
conclusion and summary is that venture capital companies in the
United States are decreasing their investment in biotechnology
and medical device startups in the United States. They are
reducing their concentration in critical therapeutic areas and
they are shifting their focus away from the United States
towards Europe and Asia. The primary reason is because of FDA
regulatory challenges. In the last 3 years, they have decreased
by 40 percent their investments in medical devices. In the next
3 years, they expect to decrease it again by 42 percent, and 61
percent of the respondents cited as their primary reason
regulatory challenges at the FDA. I am sure that you have seen
this study or at least the summaries of it, Doctor?
Mr. Shuren. Yes, I have seen it.
Mr. Barton. Now, the proposal that the industry and your
department have agreed to doubles the user fees per year for
the next, I think, 4 or 5 years. The current PMA fee right now
I believe is $220,000. Is that correct?
Mr. Shuren. That is correct, for full fee. If you are a
small business, it is $55,000.
Mr. Barton. What does it go to in this proposal that we
have yet to see?
Mr. Shuren. So we are finalizing those details but we are
thinking at the end of 5 years it would be about $267,000,
$268,000, so it will go up by about $48,000, and it was
actually a little bit higher last year. We reduced it, because
by law, if we collected a little bit more money, we had to
reduce the fees so we reduced the fees this year.
Mr. Barton. And what does the small company fee go up to?
Mr. Shuren. I think it is about $67,000.
Mr. Barton. And what is the level at which you are eligible
for the small company fee?
Mr. Shuren. If your annual sales or receipts are $100
million or less.
Mr. Barton. And is that what it is in the current? So is
that changed or unchanged?
Mr. Shuren. No, that has remained the same, and you can
compare this on the drug side. NDA is the complement on the
drug side. That fee is $1.8 million.
Mr. Barton. And I am sure, Doctor, that you are aware that
in the new health care law that passed several years ago, there
is a 2.3 percent tax on medical device companies, and it is
expected to raise $20 billion over the next 10 years.
Mr. Shuren. I am aware of the tax.
Mr. Barton. Why could we not use some of that money and
have no fee increase at all?
Mr. Shuren. The tax isn't under our purview. That is a
question for the administration. But I will say the concern
about dollars, and I recognize, you know, for industry, to ask
them to pay more, you know, they are figuring out how to do
that. But I will you, $595 million over 5 years, compared to
what you heard the other week on the Generic Drug User Fee Act,
over 5 years, they are going to collect about $1.5 billion, and
the Prescription Drug User Fee Act over 5 years is going to
collect almost $3.5 billion. So I appreciate the industry
paying more and they made compromises, we made compromises to
get to where we are, but to look at us and say that we are
asking for way too much, the drug program is going to get six
times the amount in user fees over 5 years than us. Even
generic drugs, a smaller program, is going to get 3 times the
amount.
Mr. Barton. I appreciate that, but your current medical fee
is $287 million, and under this proposal, it doubles.
Mr. Shuren. Well, not the individual fees to companies, the
collections. You know, things like--most of the small companies
make the 510(k) devices, and the fee right now is about $2,000,
and under the changes being made over 5 years it would go up to
about $2,600. They also pay a registration fee, and many of
them have one facility. That right now is about $2,300, and it
might go up to $3,800. If you look at the drug side, a
registration fee for a facility is a little over a half a
million dollars.
Mr. Barton. My time is expired, Mr. Chairman, but put me
down as very skeptical. I will look at this with an open mind,
but if I had to vote today, I would vote no and I would really
ask the committee staff on both sides that once we get the
proposal to really scrub it and let us make sure that we
protect our device user companies and the consumers who are
going to have ultimately pay the increase in these fees. With
that, I yield back.
Mr. Pitts. The Chair thanks the gentleman, and if you will
provide a copy of that study for the minority, they would like
to see it before we enter it into the record.
Mr. Barton. Sure.
Mr. Pitts. The Chair recognizes the ranking member
emeritus, Mr. Dingell, for 5 minutes for questions.
Mr. Dingell. Thank you, Mr. Chairman.
Dr. Shuren, nowhere in the legislation is any money being
diverted from the clearance of devices or pharmaceuticals. Is
there any diversion of the fees to be collected under this
legislation from the actual clearance in any of the programs at
FDA?
Mr. Shuren. No.
Mr. Dingell. Now, do the agreed-upon user fees give FDA
resources necessary to ensure safety and efficacy of medical
devices? Yes or no.
Mr. Shuren. Yes.
Mr. Dingell. Insufficient staffing at FDA and high employee
turnover rates were mentioned by you, and they are a matter of
concern. Will the agreed-upon user fees allow FDA to hire staff
to carry out functions necessary to protect patient safety and
improve new innovative devices? Yes or no.
Mr. Shuren. Yes.
Mr. Dingell. Will the agreement allow FDA to improve
training and staff to ensure consistency in the review process?
Yes or no.
Mr. Shuren. Yes.
Mr. Dingell. Do you believe the additional staff and
professional development will help lead to reduced employee
turnover? Yes or no.
Mr. Shuren. Yes.
Mr. Dingell. This authorization of medical device user fees
includes several accountability provisions. The independent
assessment of the review process is one of these provisions. Do
you believe that this independent evaluation of the device
review process and the recommendations from this evaluation
will help FDA to identify needed areas of improvement? Yes or
no.
Mr. Shuren. Yes.
Mr. Dingell. And will you put effort into seeing to it that
that transpires?
Mr. Shuren. Yes.
Mr. Dingell. Now, will the independent assessment help
industry and FDA to evaluate how FDA is using these resources
from the user fee program? Yes or no.
Mr. Shuren. Yes.
Mr. Dingell. Dr. Shuren, would you agree that user fees are
necessary to supplement the rather miserable level of
appropriations provided by Congress to FDA for the purposes in
the legislation?
Mr. Shuren. Yes.
Mr. Dingell. Now, Doctor, I have a concern here. If a high-
risk device was put on the market with no trials for efficacy
whatsoever, let us say a pacemaker or a heart valve, do you
believe that a provider would reasonably know when or under
what conditions to prescribe the particular pacemaker to an
individual?
Mr. Shuren. No.
Mr. Dingell. So we have a real problem. If we don't assure
that these things are safe, we might be putting in a hip or a
knee or a heart valve or a pacemaker that wouldn't work and
then we would have a fine mess on our hands, would we not?
Mr. Shuren. Yes.
Mr. Dingell. All right. Now, again, if a high-risk device
was put on the market with no trials for efficacy, do you
believe a patient would be sure of the efficacy of the
particular or specific pacemaker for their particular heart
condition? Yes or no.
Mr. Shuren. No.
Mr. Dingell. If a high-risk device was put on the market
with no trials for efficacy, can a patient or provider know
that the device is efficacious for the heart conditions you are
trying to treat? Yes or no.
Mr. Shuren. No.
Mr. Dingell. In my opinion, demonstrating efficiency and
efficacy in postmarket trials as opposed to premarket approval
would weaken the high standard that patients have come to
expect. Do you agree, yes or no?
Mr. Shuren. Yes.
Mr. Dingell. Now, even industry associations have made it
clear that they support the regulatory framework currently in
effect at FDA. Do you agree that maintaining this framework
will preserve America's leadership in medical device
innovation? Yes or no.
Mr. Shuren. Yes.
Mr. Dingell. We are not going to be benefited by approving
devices that are not efficacious and that don't help the
patient, are we?
Mr. Shuren. No.
Mr. Dingell. That is going to have a bad effect on our
sales of devices, is it not?
Mr. Shuren. Yes.
Mr. Dingell. Now, I want to go back to a little bit of
history on this. This whole business started when I was
chairman of the committee and chairman of Oversight. We found
that there was a massive amount of abuse at FDA, that there
were gratuities taken and all matter of difficulties. We found
that a lot of this was judgments that were being abused by FDA
because it didn't have the money to do the job, and we found
that industry had this awful problem of not being able to get
clearance. So we found in the case of pharmaceuticals that
pharmaceuticals were laying around and not getting approved and
sometimes on a 17-year patent that was taking 7 to 10 years to
get that done. A major U.S. pharmaceutical company would lose
during that time $250 million a year. The consequences of that
were very serious. So the Congress was always plagued with
legitimate demands by industry to give them an extension of
patent, and I supported many of these things, simply because it
was basic fairness. But we figured out that the only way to do
this was to see to it that they got their clearance quickly. So
with agreement of industry, the first thing we did was to move
this into the pharmaceuticals, and then the over-the-counters
came in and said it would be a good idea if you did this for us
because it would help us, and then we found that others would
agree to it, although I have to say the device manufacturers
had some difficulty in swallowing it, but they ultimately did,
and they found it worked and they found that they all made more
money because they were getting their patents cleared in a
faster and better fashion.
I hope my colleagues will learn a little bit about that
history. This gives cleaner and better service to the people.
It saves money. It helps innovation and it helps our
manufacturers to make decent money out of their patents without
the delay that was occurring previous to these events.
Thank you, Mr. Chairman.
Mr. Pitts. The Chair thanks the gentleman and now
recognizes the gentleman from Illinois, Mr. Shimkus, for 5
minutes for questions.
Mr. Shimkus. Thank you, Mr. Chairman.
Thank you, Dr. Shuren, for being here. Do you agree that
the Institute of Medicine study on the 510(k) process was
widely rejected? Yes or no.
Mr. Shuren. One of their recommendations was widely
rejected.
Mr. Shimkus. So that would be a yes?
Mr. Shuren. Partial yes.
Mr. Shimkus. I will take partial. I am under Mr. Dingell's
standards here.
How much did you pay for that study?
Mr. Shuren. About $1.3 million.
Mr. Shimkus. Did you ask for your money back? I am glad we
got some giggling. The reality is, I was at a breakfast this
morning and someone was asking for additional Federal money,
only $21 million. The reality is, you are sitting here saying
we don't have enough money, but then we fund a study through
the Institute of Medicine that costs $1.3 million that is
widely rejected, and we don't get our money back. So these
dollars all add up, and we are in a Congress now that says, you
know, this whole saying, if you worry about the pennies, the
dollars take care of themselves. So as we are talking about Mr.
Barton, why is he doubling a user fee? Well, if we take care of
the pennies, the dollars will take care of themselves, and in
this case, I don't think we got our money's worth out of the
Institute of Medicine's report.
Mr. Shuren. And I will say, I appreciate those concerns.
They actually had a number of other recommendations that we are
following up on, and if it is of interest to the committee--and
I don't want to eat up your time--I would be happy whenever it
is convenient, now or set a separate time, to walk through what
we will be doing with the Institute of Medicine's
recommendations in their report and the ones that we deferred a
decision on to give them an opportunity to weigh in.
Mr. Shimkus. And I appreciate that, and obviously we are
not pleased with the response so far.
Tell me again, we will go to the yes or format, is it
important that we require reviewers to prove scientific or
regulatory rationale for major decision-making?
Mr. Shuren. There needs to be a scientific rationale.
Mr. Shimkus. Is that a yes? Come on. You can do it for Mr.
Dingell. I mean, why can't you say yes or no? Maybe because he
is on the other side of the aisle.
Mr. Dingell. I would suggest if the gentleman does need
help, I will be glad to assist him.
Mr. Shimkus. Do you want to read these for me?
Mr. Shuren. Let me say with a caveat within those
constructs of the question but some of the wording I might have
put differently so the real meaning isn't conveyed to the
committee.
Mr. Shimkus. Maybe I should share my questions with you
prior to the hearing as other folks do to get a clarification
of that in the question and answering.
Do you think it is important that we establish an expedited
appeals process for any challenges to those decisions?
Mr. Shuren. Yes.
Mr. Shimkus. Thank you. Do you think it is important to
have qualified, trained reviewers handling applications for
submissions?
Mr. Shuren. Yes.
Mr. Shimkus. Do you think it is important that we have FDA
publish detailed review summaries of 510(k) clearance of
premarket approval and HDE and de novo?
Mr. Shuren. Yes, with a caveat. I mean, all of the----
Mr. Shimkus. We are getting there.
Mr. Shuren. Some of these go to legislation that----
Mr. Shimkus. Amen, brother. That is what we are talking
about.
Mr. Shuren Would actually----
Mr. Shimkus. You know, and legislation that was lampooned
by the ranking member of the full committee here. I mean, he
specifically took crosshairs on legislation putting it in its
worst light where based upon some of your answers, maybe some
of those have some merit, and that is what we do. I mean, that
is what our hearing is about.
Mr. Shuren. I know, and we would like to work through
those, but some of these things in the bills and even things
like detailed decision summaries if you are talking about the
summaries that we are doing as opposed to what we are doing
now, those have costs to them. They will divert and----
Mr. Shimkus. Well, we have got Obamacare, million dollars
of tax increases now and fee increases, so we are not sure it
is all about money. We see that the medical device folks are
really ponying up a lot money now. They are doing it in the
Obamacare tax and they are doing it with this agreement.
Let me go to a final point. FDA leadership--you kind of
mentioned this earlier but I wanted to follow up. FDA
leadership explicitly directed staff in a memo dated November
23, 2008, to remove the ``least burdensome language'' from
guidance documents, and of course, we have pieces of the
legislation here that says the importance of the least-
burdensome provision. What are you doing to make sure reviewers
actually apply to the least-burdensome standard in practice?
Mr. Shuren. So what we did is, we took out--there was
boilerplate that was inconsistently being used. It was creating
more confusion and actually wasn't helping our staff apply
least burdensome, so we are doing the following. First of all,
you should also have--I communicated with my staff about how
important is it to follow the least-burdensome principle. That
went out also as a subsequent email. Secondly, what we are
doing is trying to apply the least-burdensome principles to
specific devices so manufacturers don't just hear, ``Well, you
apply least burdensome,'' to show them in fact how it can be
applied to their device. That is significantly more meaningful.
We put processes in place to try to assure we have got
management input so that we are applying the least-burdensome
principle consistently in our decision-making. And I think
those changes are starting to kick in in the program. Those are
meaningful, important changes to make.
Mr. Shimkus. Thank you, Dr. Shuren. Thank you, Chairman.
Mr. Pitts. The Chair thanks the gentleman and recognizes
the gentlelady from California, Ms. Capps, for 5 minutes for
questions.
Mrs. Capps. Thank you so much for your testimony, Dr.
Shuren. I appreciate the work that has been done to reach the
MDUFA deal, and I think this is a very important moment to
balance the needs of the companies for increased predictability
at the agency but also to increase patient safety. Congress
needs to uphold our part of the deal.
As I have mentioned in previous hearings, these user fee
agreements do not supplant Congress's role in ensuring that FDA
has the necessary resources to do its job. I hope we can work
together to ensure adequate appropriations for the agency.
Before I begin with my questions, I want to quickly raise
the issue of the unique identifier policy for medical devices
that is currently stuck in OMB. No matter what one's position
on the policy itself, everyone is stuck in a holding pattern
until this is released. Getting this policy out of OMB is
important for industry and consumers alike, and I wanted to put
on the record that Representative Schakowsky and I have sent a
letter to OMB urging them to move forward on releasing the
policy on the unique device identifier system. I appreciate,
Dr. Shuren, FDA's work on the policy and I look forward to its
release.
Now, shifting gears with my question, Dr. Shuren, reports
by the Institute of Medicine and the GAO have expressed that
women have been historically underrepresented in medical
research, particularly so for cardiovascular and other device
trials. But due to proprietary data issues, it is hard to know
for sure what is and what is not getting reported to FDA, and
that is why my bill, the Heart for Women Act, which has passed
the House twice with near-unanimous support, would require the
GAO to examine whether clinical trial and drug and medical
device safety and efficacy data are being reported by sex, by
race, and by age. Perhaps we can make some headway here.
I understand that as part of MDUFA's agreement, the FDA and
industry members will conduct an initial meeting to set goals,
timelines and expectations. Is that correct?
Mr. Shuren. Yes.
Mrs. Capps. Can you discuss to what extent the FDA will
inquire about the devices use in the diverse population of
patients? And, if the device is intended to be used in a
diverse patient population, could the FDA use this time to
encourage enrollment of a representative group on clinical
trials so that the trials fully represent and reflect the usage
of the product and prevalence of the disease?
Mr. Shuren. So we have been stepping up our efforts to have
better representation in medical device clinical trials, and
that has been through guidance, that has been through workshops
and that has been through one-on-one engagement with companies.
So we believe it is important and it is something we are
pursuing.
Mrs. Capps. And it is something you can give measurable
results on?
Mr. Shuren. To look at what may be changing in terms of
representation in clinical trials, yes, that kind of data we
could be able to provide.
Mrs. Capps. Would it be transparent enough for us to be
able to see the data, or at least to get the assurances that
you are giving us that there is a level of understanding and
that it is a fully representative sample?
Mr. Shuren. Yes. We will go back, because we have been
trying to be more transparent about information that we are
using in our decisions, and we actually have a tool starting to
put up information on the clinical trials that are used in
support of device approvals, and I think that is one of the
components in there, but we can double-check and get back to
you.
Mrs. Capps. I would appreciate that we have some follow-up
on this particular question and look forward to working with
you on it.
I want to bring up another topic in my remaining time.
Several weeks ago, I asked your colleague, Dr. Hamburg, about
the Sentinel system for postmarket surveillance. The PDUFA
agreement will allow user fees to go towards using Sentinel for
postmarket surveillance of prescription drugs, thereby
protecting the public health, saving money on research and
staying ahead of the curve on drug recalls, and from reports,
most of the work Sentinel has done to date has been in the drug
space. Now, let me ask you, can Sentinel be used in the medical
device space?
Mr. Shuren. It can be used. We have been a part of the
discussions. The holdbacks right now is, one, we need unique
device identifiers. Until we have that, we can't do it. The
second is, I will say when Congress put the mandate to have a
program for drugs, that got a lot of people to step up to the
plate to participate, and it is a very non-regulatory program.
But because it is not mentioned specifically for devices, it
has not had that same level of enthusiasm.
Mrs. Capps. I wanted to ask you to expand upon the barriers
that might exist to expanding it to the device side, and you
kind of hinted. Would you go further in the remaining few
seconds to talk about some ways that you see as barriers that
perhaps then we could--somehow there could be a pathway through
to making it be effective there?
Mr. Shuren. Well, the unique device identifiers, we need to
have that system in place, and I think the fact that the
legislation that passed just mentioned drugs put a lot of
attention and for the folks who have data, the focus went to
drugs because devices wasn't----
Mrs. Capps. Are you saying the legislation needs to be
revisited that includes devices?
Mr. Shuren. I think if the legislation mentioned devices,
we would get more interest in having such a program for medical
devices.
Mrs. Capps. I yield back. Thank you.
Mr. Pitts. The Chair thanks the gentlelady and recognizes
the gentleman, Mr. Rogers, for 5 minutes for questions.
Mr. Rogers. Thank you, Mr. Chairman.
Thank you, Dr. Shuren, for being here. I can see how it
gets confusing. This committee asked the FDA just a very short
number of years ago to regulate tobacco, and they are going to
generate some $2 billion over 5 years on a product that if you
use as directed will kill you. That is a fairly confusing
message to the FDA, so for that, I am going to apologize for
what Congress did to you, and I certainly could find lots of
places for that $2 billion when it comes to medical research to
do something pretty spectacular that is not going to find its
way there.
But I guess what confuses me, and I too have been looking
at the National Venture Capital Association, mainly because
they are the canary in the coalmine. If they are the first ones
to give an indication if in fact they are going to change their
investment habits to companies who are innovating when it comes
to medical devices and the survey results are a bit
frightening. So you believe that medical devices that are
approved by the FDA, they advance American public health. Would
you agree with that?
Mr. Shuren. Yes.
Mr. Rogers. And would you agree with Commissioner Hamburg
that the FDA has a role to play in ensuring that medical device
companies stay in the United States and want to bring their
products to the market here first? There is some advantage to
that, is there not?
Mr. Shuren. Yes.
Mr. Rogers. And I know we are saying to some degree nothing
to say here, we are moving on, we are trying to get through
this, and I hope that you do, but would you find it concerning
that according to this survey, that 44 percent of American
venture capital firms are now going to invest in life science
companies in Europe and Asia? I mean, it is clearly a shift. Is
that concerning to you?
Mr. Shuren. Well, it does concern me to see investments not
going into development of products here for the United States,
and I have to tell you, I have been on the record with that
beforehand, and one of the drivers for some of the policies we
have in place, we have been out meeting with the venture
capital community. Ross Jaffe is going to be up here
testifying. Ross and I have spoken on many occasions, and Ross
can tell me if I am not telling the truth, but, you know, some
of the top things of their concerns was I mentioned that
benefit-risk determination, taking into account patient
tolerance for risk, recognizing that when you have truly novel
first-time technology that you can't expect it to be a home
run, you have to view that a little bit differently. All of
that is baked into this framework, a common framework between
us and industry that is explicit, that will be a part of the
record.
A second is incentivizing getting the early clinical
studies to start here in the United States, and those policies
were developed in part directly out of those concerns, the
innovation pathway. Features of that were things that the
venture capital community had raised as could be helpful to
them to help some of these breakthrough products get to market.
We have taken that----
Mr. Rogers. Reclaiming my time. I appreciate those efforts,
but what they are also saying is that the reason that
investment shift is because ``the unpredictability at the
FDA.'' So I understand you tried to make some changes. Did you
hear that from those venture capital firms about the
unpredictability of the FDA?
Mr. Shuren. Yes, and that is why a number of the actions we
are taking are meant to address predictability in terms of
better guidance, better decision-making in terms of better
oversight on the decision-making that we put in place.
For folks who may be interested, we did put out an overview
that covers all the actions that we are taking and it puts a
list of everything we are doing and if we achieved it, a link
to all that information. I will make sure that our Office of
Legislation--I think that has been passed out. We will make
sure that is sent to everyone, and that is updated every time
we take----
Mr. Rogers. The one thing that worried me is a little bit
is, you said you sent out an email to your staff on the less-
burdensome approach. Sorry, but that doesn't sound like a great
plan to me.
Mr. Shuren. Well, that is why we follow that up in terms of
specifically addressing----
Mr. Rogers. OK, but my point being here, Dr. Shuren, I
appreciate it. I hope you understand the gravity of it. And
just putting out a report certainly hasn't deterred the long
list of folks who come into Congress every day and saying they
are having these huge problems. Investment is shifting
overseas. The smaller folks are losing investment as we speak.
And so we need a little fire in the belly here. If you are
truly trying to change that equation, it has to happen now. We
don't have time for reports and lighthearted emails about how
we ought to change for the future. I appreciate you having to
defend this, but at the same time, if we don't change it, we
jeopardize having to have our devices manufactured and
innovated in Asia and Europe. I don't think that is good for
U.S. consumers. Oh, and by the way, we made it more difficult
because we also applied a tax to the companies who were
successful enough to get through what is a very unpredictable
FDA process, which means they are also hiring less and
innovating less. I mean, the policies here don't work together,
and that is why I think people like me are very, very
frustrated with the FDA, knowing that we have asked you to do
really dumb things in the past, but this stuff is so crucially
important for our consumers and the folks who need these
medical devices. We have to have a little urgency in our
approach here, and I just don't see it.
Mr. Shuren. Well, I would say actually we have had the
urgency. You know, in 2010, we went out and we went across the
country to get input from industry, from others. We pushed very
quickly to get out reports and recommendations. I will tell
you, I got letters from some of your colleagues telling me to
slow down. I heard from industry folks, slow down, more time
for conversation, and our feeling was, we can't wait. We know
there are these issues and that is why we moved forward, we put
in our plan in the beginning of 2010 and we have been marching
relentlessly forward. I keep hearing from people, industry has
even said, can you slow down, you are putting too much stuff,
and it is sort of, there is a lot of things that if we don't
work them together and fix, rather than just a few little
things, we won't have the impact we want to have. And that
email I sent out is not fluff. Quite frankly, leadership starts
at the top, and to do that and communicate with my staff, I
have to be out there, I have to be out in front. I have to put
my name on it, and that is what that email did.
Mr. Pitts. The Chair thanks the gentleman and recognizes
the gentleman, Mr. Engel, for 5 minutes for questions.
Mr. Engel. Thank you, Mr. Chairman.
Thank you, Doctor. Talking about medical devices, the 2011
Institute of Medicine's report on the FDA's 510(k) processes
raised significant concerns about the current premarket
approval and postmarket monitoring processes for these medical
devices. We would all agree, I don't think there would be any
disagreement on this, that there is a necessary balance between
premarket and postmarket FDA processes. No matter how stringent
the premarket requirements, it is obviously not possible to
know everything about the safety and effectiveness of new
products until they have been in use for some significant
period of time, and as we improve the processes for getting
products to patients more quickly, I believe we need to improve
FDA's ability to detect problems that occur once products are
on the market.
So let me ask you this. Can you please describe the role
that postmarket data collection and surveillance play in the
current FDA device approval framework, and secondly, what
additional authorities or resources does FDA need to address
the problems highlighted in the IOM report?
Mr. Shuren. Well, we do use information from the postmarket
setting to help inform on the premarket side. Many of the
devices that are made, they constantly come back in the door
through incremental innovation. So having real-world experience
on those devices is critically important. Our systems in the
United States are pretty good. It is not really the system the
Nation deserves. We have adverse-event reporting that gives us
some information, but we don't have a truly robust data
collection that we really need. The Institute of Medicine
highlighted that point, and we agree with them. We need to
pursue that at a national level, and that is why as a strategic
priority we put out last month, we said we will go forward and
put out a draft national strategy for postmarket surveillance
in the spring. We will have a public meeting. We will have a
public dialog how to do this because ultimately this will help
companies, can help companies keep products on the market, can
help companies get products on the market, can also help
protect patients. It is a win-win, we need to work together,
and I think things like Sentinel, unique device identifier are
all critical aspects, having more registries. We have been
stepping up our efforts on registries.
I will tell you, Europe has a lot of issues with the
postmarket side. One thing they sometimes will do a little bit
better than us is having a national registry for certain
devices. I will give you an example. Just very recently we
worked with the American College of Cardiology, the Society of
Thoracic Surgeons and with a company, Edwards Life Sciences, on
a registry for heart valves that are being inserted through
blood vessels, revolutionary technology, and this now will be a
national registry, not only getting information on that device
but subsequent devices that come forward and you can actually
do postmarket studies buried within that registry, can reduce
future costs for doing those kinds of examinations.
Mr. Engel. Thank you, Doctor. Let me ask you a question
about the regulation of laboratory-developed tests. The FDA's
oversight of medical tests, the LDTs, have become controversial
of late. As I understand it, there are several issues in play
here. First, there are a wide variety of tests, everything from
blood tests to genetic tests that can predict whether a patient
would benefit from a particular therapy. Secondly, the FDA
regulates the actual tests themselves while CMS oversees the
administration of these tests called CLIA, the Clinical
Laboratory Improvement Amendments. It is clear that the FDA has
jurisdiction over these tests but the agency has historically
exercised enforcement discretion with respect to so many of
them but there are recent signs that the agency is going to
begin regulating a subset of these tests again.
The reason I ask that is because one of the Republican
medical device bills, the Modernizing Laboratory Test Standards
for Patients Act, which is H.R. 3207, I believe would make
radical changes in its regulatory scheme. The bill would remove
FDA from the picture entirely and give complete control of
these tests to CMS. My understanding is that CMS does not
believe this is a good approach.
So let me say this. I am very concerned about the direction
of this bill, and by all accounts, these tests are at the
cutting edge of new medical therapies, and to take the
responsibility of ensuring that these tests are clinically
effective away from the FDA, our premier scientific regulatory
body, and give it to one that lacks entirely the scientific
expertise to me makes absolutely no sense. Do you have concerns
about the approach to laboratory-developed tests laid out in
H.R. 3207?
Mr. Shuren. We do have concerns about it, and we appreciate
the fact that the bill recognizes the fact that finally
laboratory-developed tests need to be regulated. The days of
the Wild West need to stop, that CLIA is not adequate for the
oversight of that. The law as it currently stands is not good
enough, and the standard of analytical validity and clinical
validity, the standard that FDA uses, that it is the right
standard. The problem is, it creates a duplicative Federal
bureaucracy at a much higher cost, grows government
unnecessarily and it maintains an unlevel playing field between
traditional manufacturers and labs who make the exact same kind
of test, and as a result, just continues to stifle innovation
and can actually kill jobs on the flip side, and then it allows
those tests to come out on the market and then for CMS to make
a decision after it goes on the market. So you can have a bad
test that is out there, and we have seen plenty of laboratory-
developed tests, ones for diagnosing ovarian cancer that have
been inaccurate, so women are having their ovaries out and
didn't need to, making decisions about treatment for breast
cancer, treatment on chronic Lyme disease, I mean tests for
autism that are just wrong and they need to be regulated but
they need to be regulated right, and CMS did say they are not
the right place for it, they don't have the expertise, and the
cost would be at least $50 million to $100 million a year plus
$20 million startup. For our framework in the first few years,
we are talking about a cost that is probably less than $3
million in fees to industry, so I don't know why we want a more
costly, less effective kind of approach and this duplicative
oversight that actually would not help.
Mr. Engel. Thank you. I agree.
Thank you, Mr. Chairman.
Mr. Pitts. The Chair thanks the gentleman and recognizes
the gentleman from Kentucky, Mr. Guthrie, for 5 minutes for
questions.
Mr. Guthrie. Thanks, Dr. Shuren. We had a meeting in your
office about this important issue. I am from a manufacturing
background and a big believer in making in the USA and remaking
it in the USA and have been concerned about some companies
making them in the Europe because of the regulatory
environment. We talked about that.
I actually have a bill on guidance documents, and a lot of
companies like guidance documents because it gives them
regulatory predictability, but some of the problems--your
process for reviewing internal guidance documents because some
companies have said that they have submitted a guidance
document--that guidance document no longer reflects FDA
thinking, and so what process do you review those and because
how they can submit to you or to a dated guidance document?
Just kind of talk about what you are doing with the guidance
process to improve it.
Mr. Shuren. Yes. So with guidance documents, you can
actually continue to submit comments about it after the comment
period has closed. It is different from rulemaking. So that
docket remains open and we will look to see if new comments
come in. We made a concerted effort to improve our guidance
development process. In fact, in 2011, our production of
guidance documents improved by about 22 percent over 2010, and
2010 was better than 2009, but we sort of squeezed, you know,
the fruit and gotten maybe about as much juice as we can from
the internal processes improvements, and it is one of the
reasons as a part of the MDUFA III reauthorization agreement we
are getting a little bit of extra dollars, about five
additional people to help us for the oversight of guidances.
What is critical is, we need people who are more technical
writers on guidances so our experts who are doing reviews can
provide their expertise but not write the documents themselves.
That is what is going on now. And so they get diverted away
from doing premarket reviews. The little bit extra help will
help us take some of that tension off. It will also help us do
a better job at looking at guidances that have already been put
out to see if changes need to be made and also to try to make
sure that we are finalizing draft guidances more quickly.
Mr. Guthrie. And one other point I wanted to bring up. On
page 7 in your testimony, there is a chart that says about from
2000 to 2011 has been increasing additional request additional
information from 510(k) requests whereas now it says in 2011
three-fourths of all 510(k)'s had additional information
requests coming back. And I think the implication is that
companies aren't submitting the information that you need,
therefore, you haven't asked for more, and I am a manufacturing
person, quality engineer, so I used to be responsible for
submitting our tool and dies once they came in and we got paid
based on them being approved, and let me tell you, they were
only wrong if I didn't have the right information because I had
to answer to somebody because literally once our customers
signed off on that, they were by contract supposed to cut a
check. So sometimes I felt delayed because the other parts of
the project weren't ready.
So the question is, you see the trend. Are three-fourths of
the applications really inadequate or are you not letting them
know what you need? I mean, that is the question that I have.
Because it does seem like a disturbing trend to go from a third
to three-fourths.
Mr. Shuren. Yes, and actually because it was a disturbing
trend, we did an analysis of 510(k) decisions, the first 130 we
had done, or 110 in 2010. We put that analysis on our Web site,
and it is a mixed bag. I mean, there are times----
Mr. Guthrie. You have been willing to show that. I
appreciate these charts because it does show the issues, and I
appreciate that.
Mr. Shuren. Yes, but it also shows the problems have been
longstanding, like a decade, and this was a canary in the
coalmine that then led to increased total times for review. The
data just marches up starting around 2002. But when we looked
at it, so a number of different reasons behind it. There are
companies who we have put out very clear guidance on what to do
and they opted not to follow it, and they could do something
different but they didn't even justify doing something
different. I mean, even where for years you provide a little
bit of clinical data. If you want to measure oxygen through the
skin, you take a blood sample and compare it. A company comes
in and never even did the blood samples. We go back, do the
blood samples.
Mr. Guthrie. That is legitimate. That is absolutely
legitimate. It is hard to believe companies whose products are
based on that.
Mr. Shuren. Believe it or not, it happens, but then we have
companies where if we had better clarity on what to do, that
would help, and the last is, there are times where we ask for
things that we shouldn't be asking for, and that was one of the
drivers behind our changing our decision-making within the
center, making sure we have that level of oversight that the
staff can't suddenly decide to ask for something extra until
you have the proper level of sign-off. In fact, if you want to
ask for a new kind of clinical study across a type of device,
that is made at the highest levels in the center by the Center
Science Council where those kinds of decisions in fact should
be made. I just need enough managers to provide that oversight.
Mr. Guthrie. I have a chart here from the venture
capitalists, like 38 percent of their decisions, FDA
regulations are about 38 percent of their decision whether to
invest, and about two flights down there is a meeting now, and
I am going to run back to it, on manufacturing and so we have
talked about that. That is a concern. That is why we are here
and why we are real concerned about it because we want it made
in America and made safety and securely and efficiently. I
appreciate your efforts. Thanks.
Mr. Pitts. The Chair thanks the gentleman and recognizes
the gentlelady from Illinois, Ms. Schakowsky, for 5 minutes for
questions.
Ms. Schakowsky. Thank you, Mr. Chairman.
You have heard a lot today from many that the FDA has
become too risk-averse in terms of what the agency requires
device manufacturers to do in order to obtain FDA clearance or
approval, and we have heard that the FDA is insisting on too
much clinical data prior to approval and that this has resulted
in a decrease in venture capital investment as well as an
export in innovation and jobs abroad, and to help address the
situation, some have suggested that the FDA's mission statement
should be changed to include things like job creation and
innovation, and a bill has been introduced that would
accomplish this. But even if we assume there is some truth to
these reports, and I think there is a lot of evidence to
suggest that in fact there is not, revising FDA's mission
statement seems drastic to me. So I wanted you to comment on
the implications of revising the FDA's mission statement to
include things like job creation.
Mr. Shuren. Well, we are concerned about a change to
mission statement that would include job creation, economic
growth, competitiveness because we read that, so are we looking
at job growth in the context of product approvals? Are we now
going to--I mean, to do that, then we are asking for financial
data on the companies, we are looking at reimbursement
opportunities, market analyses become part of approval
decisions, and then whose jobs? Jobs in the United States or
jobs overseas? What about jobs of the competitors? I mean, the
devices most at risk will actually be the most disruptive
technologies because they are more likely to adversely affect
the competitors in the short term and could hurt job growth in
that direction.
So those are the kinds of, I really think, unintended
consequences happen with those changes, and there are a number
of other things in this bill as you march down the list that
would lead to, we think, very troublesome changes in what we
do. It can change the standard for evidence for our product
approval decisions. I mean, one of them is on public
participation. So we then say OK, so we are talking now about
public participation in product approval decisions. That means,
so should we revisit what information we have considered
confidential and start making more of that information public
and some people may think it is a good thing. We hear from
industry, please don't do that, but that is where this bill is
actually directing us. It talks about using the most, you know,
innovative tools. Well, innovative doesn't mean it is the best
tool. So we start using bad tools and we talk about, well, make
sure you are using modern tools. Well, sometimes the newest
tools aren't the best ones. Old ones are just as good but why
we should change the goalpost on industry every time there is
some modern tool? It may not be necessary to do that.
Ms. Schakowsky. So you think that this could slow down,
complicate and actually make less efficient the process?
Mr. Shuren. Oh, yes. I think it could lead to some fairly
dramatic changes in how we make product approval decisions and
I think it would adversely affect industry and adversely affect
patients.
Ms. Schakowsky. If you look at the language of the bill,
and that is called the Food and Drug Administration Mission
Reform Act, there is some language that may on its face seem
less controversial like changing the mission to require FDA to
take into account the risks that certain patients are willing
to take. Am I correct in saying that these are things the FDA
is already doing, and if so, proponents of the bill would argue
that there should be no harm in revising the mission statement
to encompass things that the FDA is already doing, and I
wondered if you could comment on that.
Mr. Shuren. Yes, this is something we already are doing as
part of the benefit-risk determination framework we put out.
That is already out there publicly, and it will go final and
begin implementation at the end of March. That is going to
happen.
But this is an activity. It is not really a mission. And so
this isn't exactly the right way of sending a message about
having a benefit-risk determination framework because it is
really an activity. It is an action.
Ms. Schakowsky. Well, I am concerned about revising FDA's
mission statement. I think it is a pretty drastic step and it
doesn't seem that there is a record for why such a dramatic
change would in fact be necessary.
So I thank you for your comments, and I yield back. Thank
you.
Mr. Pitts. The Chair thanks the gentlelady and recognizes
the gentleman from Louisiana, Dr. Cassidy, for 5 minutes for
questions.
Mr. Cassidy. Dr. Shuren, a friend of mine, an orthopedist,
went to--I am a doctor--went to a conference in San Francisco
and said he was struck that there was, relative to previous
years, a paucity of new equipment being displayed. So what I am
speaking of is somewhat influenced by the conversation I had
with him. I assume there must be some difference in terms of
how you regard the bigger manufacturer or the bigger innovative
company versus the smaller. Fair statement?
Mr. Shuren. Yes. Actually, we try to do a lot more hand-
holding with the smaller companies.
Mr. Cassidy. What in this bill--I mean, if I were to go and
say to those smaller companies, first, how do you define a
small company, and secondly, if I were to go to those
innovators and say these are the specific provisions that
pertain to you, what would be your summary?
Mr. Shuren. So small businesses for purposes of the user
fee act is $100 million or less in annual sales or receipts.
Mr. Cassidy. I want to have such a small business, by the
way, but continue.
Mr. Shuren. And what we will do is actually work with them
in terms of what they may need to do to bring a product to
market. We are very used to dealing with small companies
because they make up the largest segment of the device
industry, although most of the devices on the market are made
by big companies. But I will tell you, one of the challenges we
are seeing is some of the data suggesting we are seeing an
uptick of some of the first-time sponsor companies coming to
us, and because they are small companies, they oftentimes don't
have a good understanding of what they need to do to come to
market. I quite frankly think----
Mr. Cassidy. But that suggests a regulatory complexity as
much as anything, correct?
Mr. Shuren. No. You come to it with what you know, and for
people who understand that system, can work a lot better. I
think you don't suddenly--you need to have efficient systems,
you need to have clear systems. They need to be predictable and
consistent. But you don't just suddenly lower the bar simply
because someone says----
Mr. Cassidy. That is a fair statement. Are your fees the
same for larger and smaller companies?
Mr. Shuren. No, they are smaller for smaller companies.
Mr. Cassidy. And do they remain constant relative to the
previous authorization or do they increase or decrease for
smaller companies in this regard?
Mr. Shuren. So in MDUFA III, they will go up, and what we
are talking about now is for PMA going from about $55,000 now
to $67,000 by 2017, and the first PMA for a small business is
free. It is on the house.
Mr. Cassidy. Now, I presume that if you have a small
company, you would still be required for the double blind
control trial insofar as that is practical to test your
invasive device. I assume that is the case?
Mr. Shuren. The evidence you have to provide wouldn't
change. I mean, the device is the device. It shouldn't change
based upon who made it. That has been one of the issues with
laboratory-developed tests.
Mr. Cassidy. That is a fair statement.
Mr. Shuren. But by the same token, we are trying to apply
least burdensome, so actually most of our clinical trials are
not placebo-controlled double blind clinical trials. They are
either not practical or they may not be necessary.
Mr. Cassidy. Now, let me ask you as regards the increased
revenue you all are requesting, I have again seen stuff and I
have learned to say what I have been told, not what I know. Let
me first say that. But you in your testimony can see that there
is an increased time for approval over the last several years.
You are working to address that.
Mr. Shuren. Yes.
Mr. Cassidy. But I have also seen that your revenue
increased under the last MDUFA authorization. Your revenue
significantly increased, and I think I know that your number of
employees similarly increased. And so it seems like the lack of
resources was not there. I mean, you have the resources. You
had more money, you had more people, and yet the time to
approval increased. So since we are being asked to give you
more resources, why did more resources not work last time but
they are going to work this time?
Mr. Shuren. So two parts to that. One, there are program
issues that need to get fixed, and those are things we have
identified and we are fixing, and that is separate from
resources if you are going to make it work.
But the second is the resources we got weren't sufficient
for the work we had to do, and one of the things in MDUFA II
was we didn't take into account the increase in workload that
would occur. So we got more people to try to meet the goals but
then the workload was also going up and sort of outpaced the
resources we got, and we never addressed the fundamental issue
of having enough people to do the work and enough managers to
provide oversight, and so we constantly have this high turnover
rate, which industry has complained about because it disrupts
the review of the device.
Mr. Cassidy. I see you have a high turnover rate, but you
did increase your number of employees. So what you are saying
is, you just needed to increase them even more?
Mr. Shuren. That is correct, and we have the same problem,
by the way, in the drug program. About a decade ago, they had
the same high turnover rate, same issues. The drug industry
said--and they were not concerned about--they were very
concerned about performance. And so what happened was, there
were process improvements in the drug program and they got more
money. They were able to get over that hump and they were able
to put the drug program on the right track.
Mr. Cassidy. So you feel like your process improvements are
not enough, just to use your existing employees with existing
revenue more efficiently, but rather you need both efficiency
and much more money?
Mr. Shuren. That is correct.
Mr. Cassidy. I yield back.
Mr. Pitts. The Chair thanks the gentleman and recognizes
the gentleman, Mr. Matheson, for 5 minutes for questions.
Mr. Matheson. Thank you, Mr. Chairman.
Thank you, Dr. Shuren, for being here today. I am glad that
Mr. Barton and Mr. Rogers both made reference to the Venture
Capital Association study. I was going to note that, but I
think they covered what the substance is, is the troubling
trend of investment going offshore. I have grave concern for a
couple of reasons. One is, of course, I want folks in the
United States to have access to the best devices possible to
maintain their health and safety, number one, and secondly, the
medical device industry is the great U.S. success story over
time and it has tremendous presence throughout the country
including in my home State of Utah, and I am worried about
investment shifting offshore.
I do applaud your goal that you stated of bringing greater
consistency and efficiency and transparency at the device
center, and I want to ask you about your proposed guidance
document on when device modification requires a 510(k). Last
year, as you know, FDA released its draft guidance to industry
detailing when a manufacturer needs to submit a new 510(k) for
a change to an existing device. Obviously, FDA has had a policy
on the books for many years that industry understood and was
well accepted, but the new policy could, from what I have been
told, dramatically increase FDA's workload, by estimates of 200
to 500 percent, I mean, that many more applications coming to
the FDA for 510(k). Is it your interpretation of the guidance
document that it would require manufacturers to file 510(k)'s
in that much of an increased magnitude in terms of workload
within the FDA?
Mr. Shuren. It is not, and we had put out the guidance
actually to clarify when to submit a modification,
predominantly in areas that were gray where we didn't provide
clarity in the past, and we were not intending to raise the bar
but to clarify to make it easier. We recognized, though, the
concerns that had been raised by industry. We take them
seriously. And I will tell you, we have got companies in, we
have had trade associations in, and we are actually working
very closely with them, sort of marching through to see what
would be the real impact, did we get some things wrong, did we
not clarify properly and we are going through that. We are
doing that very methodically.
You know, one of the downsides is, one of the bills on
guidance document development would actually limit the time
frame to get a final guidance out, and if that was in effect
and we had just the one year to do it, I would be in a position
to take that guidance and rush to finish it whereas I would
rather take the time and work with industry to get it right. I
think that is ultimately the right thing to do and that is what
we are trying to do now.
Mr. Matheson. Let me ask you a specific component of the
guidance. Is it your interpretation that the new guidance would
require manufacturers to file a 510(k) when a manufacturer
would need to change suppliers due to a supplier goes bankrupt
or there is a fire or some other emergency? Would they need to
file a new 510(k) with the agency?
Mr. Shuren. Just to change suppliers, no. They would have
to document it as part of their design controls. That is just
internal records. But they don't have to submit a 510(k).
Mr. Matheson. It is my understanding that the guidance
proposed last year would require manufacturers to file 510(k)'s
for likely uses. Can you comment as to how or why the FDA would
require manufacturers to anticipate likely off-label uses of
their devices and file a 510(k)?
Mr. Shuren. They would not have to file a 510(k) for off-
label uses. They don't have to go and say well, it could be
used this way so I have to file a 510(k) then. That is the
guidance.
Mr. Matheson. But there is something in the guidance about
likely uses. Is that correct?
Mr. Shuren. There is something in there about if the
manufacturer on their own puts a contraindication in their
labeling about a particular likely use, then there is something
called a changes being affected manifestation that they would
submit to us. So it just that one circumstance where they are
actually making this change in the labeling and it is just a
certain kind of update to 510(k).
Mr. Matheson. So absent the manufacturer listing on their
labels another likely use, you are suggesting that if there
some off-label use, the manufacturer is not going to be
compelled to file a 510(k)?
Mr. Shuren. That is correct.
Mr. Matheson. OK. Thank you, Mr. Chairman. I yield back.
Mr. Pitts. The Chair thanks the gentleman and recognizes
Ms. McMorris-Rodgers for 5 minutes for questions.
Mrs. McMorris Rodgers. Thank you, Mr. Chairman, and thank
you, Dr. Shuren, for being here. This is a very important
discussion, and when it comes to new cutting-edge medical
research, exciting new medical devices, the FDA can either help
make it happen or the FDA can close the doors to an entire
industry, and as Mr. Matheson just said, the medical device
industry in America is a great success story over the last 50
years, and we have been the world leader. Americans have
benefited and lives have been saved. And yet today we hear
because of the FDA, we hear about delays, we hear about
increased cost, increased user fees. We hear about regulatory
unpredictability. And it is not just--it is not the regulations
themselves, it is the fact that the goalpost changes so often.
And then along with that, we know that this industry is also
facing huge tax increases because of the President's health
care bill. We also know that it takes on average now 4 years
longer in America to bring a new device to market than in
Europe, and I don't believe that Europe is using bad tools and
I don't believe it means that we have to lower the bar, but we
do need to address what is happening.
And so my first question is, do you believe that the
current regulatory environment at FDA is negatively impacting
the development of new medical devices here in America and
sending jobs overseas?
Mr. Shuren. I think the program that we have here needs to
be improved so that we are actually having devices, more
devices developed over here and that we are keeping and
actually creating more jobs over here in the United States, and
I take it seriously very much from a public health standpoint.
I am a physician myself. I would like to see more treatments
and diagnostics for patients. I am a neurologist. That space,
if there is ever a space that could use more help, that is the
one. But I don't think Europe is the answer. Europe actually
does have a lower standard. You don't show effectiveness over
there. You don't show that there is any benefit to patients,
and as a result, you do have products--we are finding more
products that have been approved over there later shown through
subsequent studies, often through the United States, that it is
unsafe or it is ineffective, but they don't have a centralized
database of their approvals so it is very hard to follow much
of this.
And there has been a growing chorus in Europe for change,
particularly for high-risk devices. Like the European Society
of Cardiology, the British Medical Journal are all coming out
to say high-risk devices should be treated more like the United
States: demonstrate effectiveness, more robust clinical trials
over there, putting out guidance to clarify what to do. Believe
it or not, for the need for more guidance, we put more guidance
than Europe does. So I don't think the answer is that the
United States should become Europe. I think we should keep the
American standard but the program behind it needs to be
predictable, consistent, transparent and timely. I don't know
what----
Mrs. McMorris Rodgers. Do you believe that that program
currently is predictable?
Mr. Shuren. Well, I don't think it is sufficiently
predictable, consistent, transparent, and we have said that,
and I wouldn't be making these changes, I wouldn't have my
staff spending the time to make those changes if we didn't
believe it, and I will tell you, in spite of their working hard
to try to get products out and the added effort to make these
changes in the program, we are actually now starting to see
early signs of improvement in performance. It is going to take
a little time to really show bigger impact but it goes to show
you, making those investments on our part can pay off
dividends, but what we really need is, we need the support to
go ahead and do it and then ultimately between our changes and
the extra dollars with the user fee program, we can get
ourselves back on track and we can keep the American standard.
Mrs. McMorris Rodgers. Well, at the current rate, we are
going to run out of time, and I have introduced legislation
regarding harmonization, and I wanted to ask you what role you
believe harmonization with other countries could play in terms
of getting devices to market more quickly.
Mr. Shuren. I actually consider harmonization critically
important. We had what is called a global harmonization task
force, which was us, European Union, Canada, Australia, Japan
working on harmonization. I will tell that most of the members
of that group had felt that that group had kind of run its
course. We put out----
Mrs. McMorris Rodgers. Now, when was this?
Mr. Shuren. This is the global harmonization task force,
and it put out many high-level documents that were more helpful
to developing countries who didn't have a regulatory program in
place or just developing but didn't lead to a lot of true
harmonization. We, the United States, I will tell you I
personally felt we needed to do better and so we put a new
proposal on the table for an international medical device
regulators forum to broaden the participation. It can't just be
those few countries because the rest of the world was at risk
of moving in different directions. We had to broaden our scope
and we had to focus on real implementation on harmonization,
and that group, I will tell you, to the credit of the members
of GHTF, they agreed to do it and the very first meeting of
that new forum is at the end of this month.
Mrs. McMorris Rodgers. So are you seeing products being
brought to market any quicker because of these efforts?
Mr. Shuren. No, this effort is going underway. That was the
problem with GHTF. We actually weren't focusing on critical
questions about could we actually be relying on data submitted
or in some cases decisions being made by other regulatory
bodies in support of bringing the product here to the United
States.
Mrs. McMorris Rodgers. Thank you. I have run out of time.
Bottom line, we are running out of time and we have to start
making it happen. Thank you.
Mr. Pitts. The Chair thanks the gentlelady and recognizes
Ms. Blackburn for 5 minutes for questions.
Mrs. Blackburn. Thank you, Mr. Chairman, and I thank you
all for being here.
And Dr. Shuren, I hope that you realize and appreciate that
we would like to see a sense of urgency coming from you to do
more than just talk about issues but actually have some
demonstrable actions, and when you talk about a global task
force, when you talk about, you know, time, as Ms. McMorris-
Rodgers said, we are running out of time with a lot of our
constituents and their companies who complain about the way
they are dealt with by the FDA, and in their mind, time is
money.
Now, you all in government have an additional, a continuing
appropriation but I think it is important that you realize what
we see from you is that you may not get additional money. The
Federal Government doesn't have additional money to give.
Taxpayers are saying we want to see them show some successes
and some changes in behavior, and right now, perception is
reality, and the reality is, the FDA is a very difficult agency
with which to deal. You can look at the Jobs Council. You can
look at the ODE annual report, the GAO, the Venture Capital
Alliance. You can look at all of these, and there are problems
dealing with you and the regulatory burden that you impose and
the method in which you impose that.
Now, let me ask you a question. You may have seen this
article about mobile devices. This is something that is
important to my constituents in Tennessee. And this is from
February 7th Washington Times. So I want to ask you about
mobile devices, and how do you plan to move forward with
regulation of mobile devices? Do you think you have got enough
on your plate with that? And if you do move forward with mobile
devices, do you intend to subject them to the device tax? If
somebody goes out and buys their iPad and places a mobile
device on that, some monitoring device on this, are they going
to be subject to the device tax? So please speak specifically
to the mobile device.
Mr. Shuren. So specifically for mobile devices, we actually
took a very unique approach for FDA. Normally if something is a
device, you regulate it like a device, and we said, ``Wait a
minute, why do we need to do that?'' Quite frankly, if there is
not sufficient value added to do that, and keeping in mind the
value of having certain technologies out there and recognizing
the more rapid innovation cycles we see, then we shouldn't do
it. So the policy we put out--and that article is dead wrong.
They got it wrong, and you should see the commentary in other
publications on that article saying what was this person
thinking. No, what we actually said is, while the world of
mobile apps is maybe this big for devices, we are only
interested in this, and in reality, what we are interested in
is, it is the same thing as devices we already regulate. It
shouldn't matter if the device is on a desktop versus on a
mobile application. It is still a device. It is something we
already regulate. That doesn't change it. And that is really
the very narrow universe that we focused our attention on. That
is essentially it. That makes a lot of sense.
What we got back from comments is, can you provide more
clarity on the boundaries, give us more examples about it, but
for the most part, the read we have been getting from people is
that very narrow look makes a lot of sense, and for the rest we
have said even if you are a device----
Mrs. Blackburn. What about expediency? Because right now it
is taking about 3 years and about $75 million to get something
through your process, and I have to tell you, some of the
innovators that I am talking with, they don't think this was
completely wrong. They saw a lot of commonalities in the
article, and so I would just highlight with you, when you look
at the speed of innovation that is taking place in the medical
mobile applications that you can't spend 3 years trying to get
through all of your filings and reviews and the repetitiveness
and switching reviewers. Sir, there is a tremendous amount of
frustration with the FDA by our innovative community. So talk
with me about expediency.
Mr. Shuren. Sure, and again, when we are talking about the
mobile apps that we are looking at, it is things like you have
technology that is pulling down X-rays and reading the X-rays,
I mean, the stuff we normally regulate, or EKG machines to
measure heart rhythm. We have been regulating those for years.
But when we deal with just software, we recognize too that the
paradigm we have, the framework we have in place for devices
does not work well. Actually, that was one of the
recommendations from the Institute of Medicine to look at
software because it was so challenging. So maybe we don't have
to get the $1.3 million fully backed. We can let them keep a
few dollars. But we are actually underway to sort of revisit
our entire framework as regard software, recognizing exactly
the point that you make, that you have these rapid changes, and
you need to allow for that kind of business model and constant
updates. By the same token, there may be other ways to assure
you have a good product that we might be able to avoid even
looking at it premarket, and the other is, there is a whole
bunch of things for clinical decision support, things to help
you make decisions that while they could be medical devices, we
are going through it and saying leave it alone, just leave it
alone completely, and that is what we are working on by way of
policy. Because we agree, we have to have a rationale approach.
Mrs. Blackburn. When do you think that your policy will--
when are you going to have some guidance? And my time has
expired. I will ask you to answer, and yield back.
Mr. Shuren. OK. Our goal is on mobile medical apps to close
out that one this year and also to put out the draft policy on
the clinical decision support software this year as well.
Mr. Pitts. The Chair thanks the gentlelady. That concludes
the questions by the members of the subcommittee. Without
objection, we will go to members of the committee for
questions. Dr. Christensen, you have been very patient, you
were here the whole hearing. We will recognize you first for 5
minutes for questions.
Mrs. Christensen. Thank you, Mr. Chairman and Ranking
Member. It has been very informative to sit here and listen to
the questions and the answers.
I wanted to follow up on Mr. Waxman's questions about the
Premarket Predictability Act of 2011. The bill would make
changes in two areas in addition to the least-burdensome
provisions, one, to the investigational device exemption, and
then second, to the procedures for appealing decisions through
CDRH.
On the first, the bill would change the investigational
device exemption process in ways that appear designed to permit
companies to conduct studies that are not necessarily geared
towards an approval or clearance decision. That seems to run
counter to the company's interest, so can you explain where
this is coming from, if you know, and whether you believe a
change like this is necessary?
Mr. Shuren. Well, we actually find problematic the change
that is put in there because that change in standard for
approving a clinical trial will mean that we will approve a
clinical trial that is supposed to be the pivotal trial to show
it is safe and effective and we will approve a trial that isn't
going to be good enough so it will go forward, and then when
the product comes back in the door with the results, we want to
approve the product. And we suffered in that circumstance
previously and so we were watching our approval of products
going bad. It wasn't working well.
Now, on the flip side, we sort of changed that but didn't
change it well enough so that we said look, let us stop doing
it, but what we didn't allow is, there may be extra questions
we don't need an answer to right now, and they are nice to know
but we shouldn't worry about them, and so we put out new policy
in November of 2011 to actually set that balance right on
approving clinical trials, and we think that is the smart
approach. That will get us to actually approving clinical
trials more quickly but appropriately. This change in the
standard will actually adversely affect products coming on the
market.
Mrs. Christensen. That was my impression as well.
And the Premarket Predictability Act would also make
changes to CDRH's appeals process to make it easier to have you
as the center director be directly involved in appeals. In
fact, it appears that under that bill, you would not be doing
much else other than just dealing with appeals. So can you
comment on that section of the bill and what impact those
changes to the appeal process would have on the center?
Mr. Shuren. Well, if folks would prefer that I just work on
appeals and not improving the premarket program and making the
changes necessary to do, this is a good way to do it. I would
actually prefer just being sent on vacation, but that is a
problem with this bill. And I will tell you, most appeals
actually get resolved at the office level. In fact, of the
appeals filed in the past 2 years, 26 to 28 percent wind up
getting changed in whole or in part. So it goes to show you,
the appeal process can actually work.
Mrs. Christensen. Thank you. I just wanted to get that on
the record.
And on the guidance issue that was raised, H.R. 3204, the
Guidance Accountability and Transparency Act of 2011, appears
aimed at making FDA guidance development a more public process
and ensuring that they remain up to date. I think we all agree
that government procedures should be as transparent as possible
and that the ability of government to make informed and
sensible decisions is dependent on receiving and making use of
information stakeholders, and we certainly agree that guidance
should be finalized in a timely manner and kept up to date.
At the same time, though, I think we all understand that
the principal purpose of FDA guidance is to enable the agency
to provide advice in a more timely and flexible manner than it
can through regulations. For instance, when FDA learns of new
information relevant to certain product approvals, the agency
needs to be able to communicate this information to the
regulated industry as quickly as possible. Otherwise the
industry could waste valuable time and money doing clinical
trials on other work that won't necessarily help with approval
of clearance of their product. So we need a workable process
that balances the need.
But I am concerned that the processes that would be
required would actually make the guidance more onerous and more
time consuming. So as my time is getting short, I know that the
legislation would apply to all FDA guidances but could you tell
me how it would affect CDRH and are there any aspects of that
legislation that you agree with that might be helpful?
Mr. Shuren. The bottom line is, we will issue fewer
guidance and there will be less predictability in our programs.
I mean, there are all these additional hoops and hurdles. You
have to announce that you are going to do this particular
guidance 3 months in advance. We already put out a list. Then
we have to meet both before and after putting out the draft so
the cost just dramatically increases, and where we have been
trying to improve our productivity, productivity is going to go
into the toilet and we know that is not good for industry.
Mrs. Christensen. And if you have to issue your final
guidance in 12 months, that just makes you say no, I can't do
it, so----
Mr. Shuren. Well, that is one of the problems, and industry
sometimes asks for longer comment periods because they want
more time to look at it. I can't grant the longer comment
period. Modifications guidance, we couldn't be working through
those issues. And if I have HHS or OMB who are reviewing it,
that just adds on a lot of additional time. We understand the
need to kind of try to move quickly and rapidly but this
actually would have unintended consequences. And the other part
about expanding what is under a guidance document actually can
have adverse consequences for patient safety because it
includes notices that involve a complex scientific issue. Those
are public health notices that we have to get out quickly to
tell the public about a big public health concern would not be
subject to this good-guidance practice more onerous. So we
would have to say there is something coming up on this device,
we will announce it in 3 months, stay tuned. That doesn't help
patients.
Mrs. Christensen. Thank you for clarifying those issues for
us. Thank you.
Mr. Pitts. The Chair thanks the gentlelady and recognizes
Mr. Bass for 5 minutes for questions.
Mr. Bass. Thank you very much, Mr. Chairman, and I
appreciate your accommodation. I am also not a member of this
subcommittee.
Dr. Shuren, I represent a State, New Hampshire, with a
number of important medical device manufacturers as well as
laboratories that are at the forefront of developing new
medical devices, some of which are very common now and in use
not only in America and around the world, and to say that some
of them at least are very frustrated with the length of time
and the quality of the decisions that are coming out of the FDA
on the medical device side would be an understatement and
perhaps in some cases we can work together on some of these
issues.
But I am here to ask you a question about a bill that I
have introduced as part of, I think there are 10 altogether, on
MDUFA having to do with humanitarian-device reform. As you
know, we haven't had nearly as much success since the 1990s in
developing humanitarian devices for rare diseases as we have
had with the orphan drug program, just 55 devices compared to
350 orphan drugs. But that isn't FDA's fault or the industry's
fault. There are flaws in the law that chill investigator and
sponsor interest and demand targeted reforms. The bill that I
have agreed to introduce, H.R. 3211, the Humanitarian-Device
Reform Act of 2011, would lift the profit restriction on
current law but maintain FDA's current oversight of
humanitarian devices. The Act would simply do it for adult HDEs
what the 2007 pediatric device law has already done for
pediatric HDEs. Today, there is evidence that this has already
led to more interest in pediatric HDEs.
My question to you is, do you agree that lifting the no-
profit restriction on adult HDEs while maintaining FDA
oversight is a win-win reform that would encourage more
innovation, ensure safety and result in more treatment for
rare-disease patients?
Mr. Shuren. So the honest answer is, I don't know what the
ultimate impact would be on the flip side for pediatric
devices. We happen to agree with you that there is a need for
more incentives to develop devices for these rare conditions. I
know the National Organization for Rare Disorders has said
look, lift the cap on adult products. That makes a lot of
sense. The American Academy of Pediatrics has a concern that if
you broaden it, then manufacturers won't make devices for the
pediatric population, and we have seen a fivefold increase in
companies coming forward to actually get a fivefold increase in
designations for humanitarian-device exemption for pediatric
indications.
So this is exactly the kind of topic, quite frankly, that
we agree Congress should be tackling. We would like to be a
part of that conversation. We suggest get all the players in
there, because I don't think we have enough information to make
a firm decision but we fully support this is an area that it is
critical that we take a closer look at.
Mr. Bass. I appreciate that, and I appreciate the fact that
you are willing to work with me and other members of the
subcommittee. I would point out that there are other patient
groups that disagree with AAP, and the reality is that we could
really benefit significantly if we had an honest debate and
could work out some sort of a legislative remedy for this.
And with that, Mr. Chairman, I will yield back. Thank you,
Doctor.
Mr. Pitts. The Chair thanks the gentleman. That concludes
the first round of questioning. We will now take one follow-up
per side. I recognize Dr. Burgess for 5 minutes.
Mr. Burgess. Thank you, Mr. Chairman.
I vowed to be good today, but someone on the other side
took the first shot, so let us talk about laboratory-developed
tests for just a moment and the reason why H.R. 3207 was in
fact necessary because of draft guidance coming out of your
shop, the Center for Devices and Radiological Health, appeared
to be overstepping the boundaries. In fact, there appeared to
be a basic change in the standard regulatory paradigm that had
been established, and if one even wanted to draw it to its
further conclusion, there appeared to be violations of the
Administrative Procedures Act coming out of your office by
issuing this draft guidance. You are going to require people to
do things that had never previously been required, and this was
all happening without any legislative authority. It was simply
happening upon the will and the whim of the Center for Devices
and Radiological Health.
So I have got several letters from laboratories across the
country that are in support of keeping this jurisdiction within
CMS, within the purview of CLIA. Laboratory tests must be
accurate, they must have clinical utility, and that is the
correct place. To ask these companies to literally be sucked
into the maelstrom of the regulations of the devices, you can't
do what you are already supposed to be doing and you are asking
for more jurisdiction. How is this helpful? How does this move
anything in the proper direction?
So Mr. Chairman, I did want to submit these letters on the
laboratory-developed tests for the record, because again, I
think this is an important part of the discussion. Maybe this
legislation is not the correct final product but this
discussion needs to be part of the reauthorization of the user
fee agreements. I will certainly allow you time to respond.
Mr. Pallone. Mr. Chairman, I would have to review those
before I could agree to unanimous consent to put them in the
record.
Mr. Pitts. OK. We will provide copies to you.
Mr. Shuren. So laboratory-developed tests, we have been
clear for years, they are medical devices. I mean, it is the
test. It doesn't matter who makes the test and that is how the
law is, but we have exercised enforcement discretion but the
world changed, and we have more-complex tests that are actually
putting patients at significant risk. I would be very
interested to see the framework you are talking about because
we actually never issued draft guidance, so maybe it is another
group that put it out there, but we have yet to put anything
out there for people to react to. But it makes absolutely no
sense to have the same kind of test that is regulated by two
different government agencies, depending upon who makes it.
And CMS has been clear when they looked at the legislation,
this is not the right place for doing it. In fact, one of the
changes under CLIA was about where you make determinations in
terms of the risk on the test, and it moved from CDC to FDA,
specifically to reduce duplication and try to have more of one-
stop shopping, and this actually goes the opposite direction
of----
Mr. Burgess. No, sir. The indications of the draft guidance
you were going to put out, that would be the duplication that
this legislation is seeking to avoid. And CLIA, remember, in
its inception in the late 1980s, I was never a big fan of CLIA
as a practicing physician but their whole purpose, the purpose
that Senator Kennedy and others worked on this was so that
laboratory tests could be certified as accurate and have
clinical utility. That is their job. Don't tell me they don't
want to do their job. If a Federal agency doesn't want to do
its job, then perhaps we will have that discussion, but this is
their job. This is what they were required to do under the
amendments in 1988.
Mr. Shuren. No, the amendments actually don't address these
issues on analytical and clinical validity. In fact, your bill
now changes that so you have to provide the data to actually
show that. The problem is, it is not set up in a good way to
get there and it creates duplicative government.
This is actually a problem for personalized medicine. We
have heard this from companies who are making drugs and then
devices to actually have the devices diagnose who is the right
population to get the drug, and you now have companies, they
make the device, they make the drug, they do the data.
Everything works out and moves forward. In fact, one of them,
two of them that just came out, we and our Center for Drugs, we
approved it, both the drugs and the diagnostic, in less than 5
months. But then the day that they go out with their test and
with their drugs, labs come out and say oh, I have got the
exact same thing and in fact we are better. Really? And so now
people can go use those other tests. Who knows if they are
actually any good. Because none of the studies was even done
with the drug. It is not even out there. And so what do you
have now? Now you have tests that actually may be directing
patients to get treatment they shouldn't get or not get a
treatment they should get, and that is a disaster.
Mr. Burgess. Well, I would submit that the duplication
actually exists within your center, and albeit there is work to
be done here but to simply ignore that there is a problem is to
do no service to anyone at all.
Thank you, Mr. Chairman, for your indulgence and I will
yield back.
Mr. Pitts. The Chair thanks the gentleman and recognizes
the ranking member for 5 minutes for follow-up.
Mr. Pallone. Thank you, Mr. Chairman.
Dr. Shuren, H.R. 3202, the Novel Device Regulatory Relief
Act, appears to be intended to streamline the de novo process
for FDA approval of medical devices. Although it is important
to ensure that FDA review processes are efficient, I am sure we
would all agree that the fundamental goal of the FDA is to
ensure the safety of the public and to protect Americans from
unsafe and ineffective medications and devices.
The proposed new language in this bill would allow device
companies to require that their new device be evaluated under
the de novo process without first submitting a 510(k)
application demonstrating a substantial equivalence to another
device already on the market, which is what is currently
required under the de novo procedures, and it changes the
timelines under which a de novo application must be submitted.
So my question is, do you think this change under this
proposed legislation would add to the efficiency of your
clearance process? Does it give you enough time to do the
reviews for products that presumably will be more novel than
most 510(k) submissions?
Mr. Shuren. We do think that the change of not having to be
required to submit a 510(k) before going down the de novo
pathway makes sense. So taking that requirement out of the law
makes sense. Giving us only 60 days to do it, however, isn't
enough time. I mean, even a 510(k), which is less complicated,
is 90 days by law, and even that, we all know that that is not
enough time for many of these as well. So not enough time but
it is the right thing to do to take out the 510(k) if they
don't want to submit it. Some companies, you actually don't
know and they don't know, and they submit a 510(k) and then we
will look at it. They actually never the requirements for a
510(k).
Mr. Pallone. All right. Then I wanted to ask you a second
question. As you know, the Safe Medical Devices Act of 1990
mandated that FDA evaluate pre-amendment class III devices and
on a case-by-case basis either reclassify them to class I or II
or require them to go through premarket approval as most post-
amendment class III devices. What I would like to know is why
FDA hasn't completed its mandated task of reclassifying pre-
amendment class III devices or requiring them to go through
premarket approval. Can you tell us how far you have gotten in
this activity and how many devices remain, and are there
unnecessary procedural hurdles in the law that keep you from
finishing this activity?
Mr. Shuren. So when I came on board, we put a new refocused
energy into trying to get these done, and we have on our Web
site each of the devices that we have to go through and where
they are in the process. There are five steps. Four of them, we
have wrapped up on. Another six we have proposals out and we
will be issuing some actually final rule coming up and another
proposed rule. So we are marching down the list. The challenge
for us are the statutory requirements to go through this
process, advisory committee meetings and doing rulemaking. In
fact, this challenge--I mean, you all in legislation are
telling us do this faster. This is a challenge when we have to
change classification on a product. It is by rulemaking, and it
cuts both ways. On the one hand, it is a weakness with 510(k).
If you have a device that is in the 510(k) pathway and we have
new data to say there are concerns, it should not be under
510(k), it should have been under PMA, a higher classification.
It will take us several years to go there and puts a terrible
quandary on doctors and patients who are out there and have the
technology and they don't have the data behind it, or we take
it completely off the market and that doesn't make sense in a
lot of cases. We want to leave it there. That process is too
burdensome.
On the flip side--and that is a safety issue. On the flip
side, though, when we want to down-classify so we have
something at a high risk or moderate risk and we want to make
it lower risk and reduce regulatory burdens, we have so many
statutory burdens on us, it is hard to do that. So it is hard
for us to be deregulatory and it is hard for us to set the bar
in the right place. And if that were fixed, that would solve a
big challenge. It would actually buttress things like the
510(k) program where the attention goes on these few devices
where there are a lot of issues but it will also allow us to
free up resources by down-classifying devices that should be
subject to a lower standard.
Mr. Pallone. You know, just an editorial comment. I don't
envy you your job because it is a constant problem which is on
the one hand, we want innovation, we want approvals to move
more quickly, but we also have to balance that with public
safety, and we get it at both ends. I mean, I as a politician
get that from both ends, you know, ``Why aren't you moving
quickly?'' On the other hand, everything has to be safe. You
know, it is tough. I mean, I know a lot of my colleagues,
particularly on the other side of the aisle, have been saying
there are too many hurdles, but you can't sacrifice public
safety, either, so it is a difficult quandary. Thank you.
Mr. Shuren. I appreciate that. Actually, not even my dog is
talking to me these days.
Mr. Pitts. The Chair thanks the gentleman.
The Chair has two unanimous consent requests. One, the
report by the National Venture Capital Association entitled
``Vital Signs.'' You have seen that?
Mr. Pallone. That is fine.
Mr. Pitts. Without objection.
[The information appears with Mr. Jaffe's prepared
statement.]
Mr. Pallone. And the other being from----
Mr. Pitts. Mr. Burgess's letter?
Mr. Pallone My colleague is fine too, yes.
Mr. Pitts. Without objection, those will be entered in the
record.
[The information follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Mr. Pitts. That completes panel one. Thank you very much,
Dr. Shuren. We look forward to sitting down with you and
working with you as the process goes forward.
At this point we will take a 5-minute recess while panel
two sets up on the table, and we will reconvene in 5 minutes.
[Recess.]
Mr. Pitts. I will ask all of our guests and witnesses to
please take their seats, and I will introduce the second panel.
First of all, thank you all for agreeing to testify before the
subcommittee today. Let me quickly introduce each one of you,
and you can present your testimony, summarize your statements
in this order. Mr. David Perez, the President and CEO of Terumo
BCT; Ms. Elisabeth George, Vice President of Global Government
Affairs, Regulations and Standards for Philips Healthcare; Mr.
Ralph Hall, Professor at the University of Minnesota Law
School; Dr. Ross Jaffe, Managing Director of Versant Ventures;
Dr. Aaron Kesselheim, an Internal Medicine Physician at Brigham
and Women's Hospital; Dr. Art Sedrakyan, an Associate Professor
at Weill Cornell Medical College; Ms. Lisa Swirsky, Senior
Health Policy Analyst at Consumers Union; and Mr. Jim Shull
from the State of New Jersey.
Again, thank you all for coming. We have your prepared
statements, which will be entered into the record. Mr. Perez,
we will begin with you. You are recognized for 5 minutes to
summarize your testimony.
STATEMENTS OF DAVID PEREZ, PRESIDENT AND CHIEF EXECUTIVE
OFFICER, TERUMO BCT; ELISABETH M. GEORGE, VICE PRESIDENT,
GLOBAL GOVERNMENT AFFAIRS, REGULATIONS, AND STANDARDS, PHILIPS
HEALTHCARE; RALPH F. HALL, PROFESSOR OF PRACTICE, UNIVERSITY OF
MINNESOTA LAW SCHOOL; ROSS JAFFE, MANAGING DIRECTOR, VERSANT
VENTURES; AARON S. KESSELHEIM, ASSISTANT PROFESSOR OF MEDICINE,
HARVARD MEDICAL SCHOOL, DIVISION OF PHARMACOEPIDEMIOLOGY AND
PHARMACOECONOMICS, BRIGHAM AND WOMEN'S HOSPITAL; ART SEDRAKYAN,
ASSOCIATE PROFESSOR AND DIRECTOR, PATIENT-CENTERED COMPARATIVE
EFFECTIVENESS PROGRAM, WEILL CORNELL MEDICAL COLLEGE AND NEW
YORK PRESBYTERIAN HOSPITAL; LISA SWIRSKY, SENIOR HEALTH POLICY
ANALYST, CONSUMERS UNION; AND JAMES SHULL, BROWNS MILLS, NEW
JERSEY
STATEMENT OF DAVID PEREZ
Mr. Perez. Thank you, Chairman Pitts, Ranking Member
Pallone and members of the committee for this opportunity to
testify today.
My name is David Perez and I am the President and Chief
Executive Officer of Terumo BCT and Chairman of Terumo
Corporation's Blood Management Business board, and I am
responsible for leading the strategic direction, the growth and
the execution of this global organization.
At Terumo BCT, we believe in the potential of blood to do
even more for the world than it does today. This belief unites
our organization, inspires our innovation and strengthens our
collaboration with customers, which ultimately benefits the
patients that we all serve. Working with the American Red
Cross, community blood centers throughout the United States as
well as hospitals, we unlock the potential of blood as we
strive to make even safer high-quality transfusions available
to people. We help our customers bring even more treatment
options to patients with advanced blood therapies, and we
support researchers in developing cell therapies that may
fundamentally improve health care.
I want to thank you for convening today's hearing and for
your interest in improving medical device regulation for
patients in our industry.
Over the course of the last year, members of this committee
have demonstrated their focus on improving the efficiency and
effectiveness of FDA regulation in your outreach to the agency
and to the policy proposals that show your commitment to this
important issue.
The medical technology industry is an American success
story. Our industry directly employs more than 400,000 workers
nationwide including 22,000 in the State of Pennsylvania,
20,000 in New Jersey and over 11,000 in my home State of
Colorado, making these among the States with the largest med
tech employment. In 2011, our company alone added 297 jobs, 224
of which were in manufacturing.
Whether the firm is large or small, success in our industry
comes only from innovation, the creation of diagnostics,
treatments and cures that extend and enhance lives. While we
are very proud of our contribution to the U.S. economy, we are
even more proud of our contributions to improving patient care.
Even though we are making progress in improving patient
care and see immense future opportunities, we are also very
worried. Today, America is the world leader in medical
technology but there are warning signs that our lead is
slipping, and a key factor in our loss of competitiveness has
been the decline in the FDA's performance. Put simply, FDA is a
crucial partner to our company's efforts to bring safe and
effective medical devices to patients. Without a strong,
effective and efficient FDA, we cannot have a strong and
competitive industry.
While the FDA has consistently maintained an excellent
record of assuring the safety and effectiveness of the products
it reviews, delays in product approval, inconsistency in the
review process and the resulting downstream effects on
investment and innovation have undermined the competitiveness
of our industry and harm patient access to new treatments,
diagnostics and cures.
I am pleased to be able to report that after extensive
negotiations, industry and FDA recently reached an agreement in
principle for a new user fee package, which we believe has the
potential to help achieve meaningful change in FDA performance
through groundbreaking accountability and transparency
measures.
The FDA leadership and Dr. Shuren in particular have
recognized the need to vigorously address the issues affecting
the device center, and I want to applaud them for this
commitment. The user fee agreement is a huge step in the right
direction. It is good for industry, it is good for the FDA, and
most of all, it is good for patients.
The user fee agreement builds the conditions for success in
a number of major ways. For the first time ever, this user
agreement establishes average total time goals for FDA product
review. All previous agreements have set goals in terms of time
on the FDA clock. What matters to companies like my own and
patients is the time it actually takes to get the product to
patients. By setting in place this new goal, we will helping
the FDA focus on the metric that is truly the most important to
all concerned.
The agreement also includes process standards that we
anticipate will improve the consistency and timeliness of the
review process independent of the specific time goals, and the
agreement provides for meaningful pre-submissions interactions
where agreements reached will not change so that companies know
what the FDA expects and the FDA is bound by its commitments.
And a new procedure, what we call No Submission Left Behind,
will be instituted so that if the FDA time target is missed,
the company and the FDA will meet to work out a schedule to
resolve the remaining issues so that the submission doesn't go
to the bottom of the pile.
The agreement also provides for greater accountability so
that FDA's success will be transparent to FDA management, to
industry, to patients and to Congress so that any problems that
arise can be corrected promptly. There will be quarterly and
annual reporting on key metrics both the FDA and the industry
have agreed are very important. In addition, this agreement
requires analysis of FDA's management of the review process by
an independent consulting organization coupled with an FDA
corrective action plan to address opportunities for change and
improvement.
Finally, to give FDA additional tools to meet these goals,
the agreement provides $595 million in user fees, additional
reviewers, lower management-to-reviewer ratios, enhanced
training, and other resources provided by the agreement will
give FDA what it needs to improve performance.
I appreciate the committee's work and its focus on
enactment of this reauthorization package as soon as possible,
and once again, I thank you for the opportunity to testify.
[The prepared statement of Mr. Perez follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Mr. Pitts. The Chair thanks the gentleman and now
recognizes Ms. George for 5 minutes for an opening statement.
STATEMENT OF ELISABETH M. GEORGE
Ms. George. My name is Elisabeth George and I represent
Philips Healthcare as their Vice President of Global Government
Affairs, Regulations and Standards. I want to start by thanking
Chairman Pitts and Ranking Member Pallone for holding today's
hearing. I also want to thank you for your particular interest
in medical innovation and for leading a policy discussion on
how we can work together to collectively improve the medical
device user fee program.
It is clear to me that we all share the goal of getting
safe and innovative products to U.S. patients in a timely and
predictable manner. Philips Healthcare employs over 15,000
hardworking Americans in cities and towns across the country.
We are just one in a global industry. Philips Healthcare's
current activities are organized across four businesses:
imaging systems, patient care and clinical informatics, home
health care solutions, and customer services. We have
appreciated your steadfast support in ensuring the access to
medical technology and particularly imaging and its important
appropriate use for patients.
I have worked for Philips Healthcare for more than 15
years. I have managed strategic planning and technical aspects
for global affairs, regulations and standards. I have also
served on multiple FDA advisory panels through the years and
have most recently represented the medical imaging industry
during the MDUFA negotiations with the FDA. As an industry
negotiator, I am pleased to talk with Congress today about the
agreement in principle between the medical device industry and
FDA. We believe that this agreement will facilitate improved
transparency and consistency leading to better predictability
and more timely access for patients.
After negotiating for more than a year, we believe that
this agreement is balanced and is fair to all stakeholders. We
hope this package will lead to a timely reauthorization of the
medical device user fee program. The goal of this agreement is
to ensure timely patient access to safe, effective treatments
and diagnostics. Although it is not formerly proposed to
Congress until it receives full administrative approval and the
FDA completes its public commenting period, the package as
negotiated includes commitments from the agency that will
improve the device review program through additional
predictability, transparency and accountability. In a time of
tremendous advances in medical technology, the agreement
enables the industry to bring innovative, lifesaving
technologies to market faster so that patients receive the
highest quality care.
The explicit goal of the device user fee program has been
to achieve more timely clearance of safe and effective devices
by providing the FDA with supplemental funds to independently
evaluate applications. However, despite clear Congressional
intent, FDA performance has declined steadily over the past
several years. For example, fiscal year 2006, it took an
average of 105 calendar days to make a final decision on a
submission. The number increased to 154 days in 2009 despite
the fact that the user fees had increased by over 50 percent
over the same period. The decline in timeliness has been an
overarching concern for industry. Our goal in this agreement
was to reverse this downward trend and to ensure value for our
user fee investment for both patients and innovators. The
increase in resources to the agency under this agreement
corresponds to a more timely approval process, which will
benefit patients and the manufacturers who develop these
innovative technologies.
The agreement includes several new quantitative goals to
hold the FDA accountable. These goals include total time for
decisions as well as improved annual targets for 510(k)
applications. The agreement also works to ensure an improved
review process that is more predictable and transparent for
manufacturers, patients and other stakeholders such as through
enhanced clarity in the pre-submission process, enhanced
guidance development and an independent assessment of the FDA's
performance. These improvements are important for patients,
innovation and jobs in America.
I believe it is important that Congress do everything
possible to encourage high-tech 21st century industries like
the medical device manufacturing that will continue to create
jobs and necessary to grow the U.S. economy. We are very
appreciative of members of this committee who have held a
series of hearings and introduced a number of bills in an
effort to respond to these concerns and improve the FDA review
process for medical devices. I believe that our collective
efforts will lead to constructive improvements.
Thank you for your consideration of these important issues.
As the legislative process moves forward, we look forward to
continuing to work with Congress and the administration to
ensure patients are guaranteed timely access to medical
technologies.
I again thank you for this invitation.
[The prepared statement of Ms. George follows:]
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Mr. Pitts. The Chair thanks the gentlelady and recognizes
Mr. Hall for 5 minutes for an opening statement.
STATEMENT OF RALPH F. HALL
Mr. Hall. Chairman Pitts, Ranking Member Pallone, members
of the committee, I appreciate the opportunity to address you
on these important issues of medical device regulation. I serve
on the faculty of the University of Minnesota Law School. I am
also part-time counsel with Faegre Baker Daniels and am CEO of
a four-person startup company.
I am here to focus on two matters: the agency's authority
in the area of medical device regulation and the safety
performance of FDA in its actual review. I believe it is
important to differentiate between questions of authority from
questions of implementation. Authority is whether the agency
can act or has the power to compel action, while implementation
goes to issues such as resources, skill sets, timing,
processes, etc. The user fees that are under discussion
specifically today primarily address implementation challenges
and are intended to address those.
On the authority front, the agency has extensive authority
for the entire lifecycle or, as we call it, total product
lifecycle, of a device from initial design to final
obsolescence. There are of course improvements, some of them
which have been discussed in the de novo process or HDEs, for
example, but fundamentally, the agency has the current
authority to require products to meet the statutory standard of
a reasonable assurance of safeness and effectiveness. This is
true under both the 510(k) system and the PMA system. There are
differences in how we achieve that objective or that test but
that same statutory standard applies to all products.
Along the same lines, the agency has extensive postmarket
authority. Examples include the MDR system, the 522 orders,
MedSun, registries, and there have been discussions about
registries. It is important to note again on the authority
front that the agency currently has the authority under the
510(k) system to mandate patient registries for products for
which it believes such registries are appropriate and valuable.
The agency likewise has extensive authority in the areas of
recalls and dealing with product issues including the authority
to ban products where that is necessary and the authority to
mandate recalls.
The major question then is, how is the agency performing on
the safety aspects. I leave to others the issues of impact on
innovation, timeliness, predictability, etc. We have performed
a study looking at medical device recalls. We have analyzed 5
years of data. We are actually in the process right now of
analyzing another year's worth of data. That is not yet
completed. The conclusion of this study is that the agency is
doing a very good job on the safety aspect. The vast majority
of products that get through their system do not have
significant safety issues. It is obvious and critical to
remember that all medical devices have risks and the statutory
standard is a balance between the benefit and the risk of the
product. So one of the key aspects and requirements of the
system is to identify the risks so that a knowing balance can
be made between the risks and the benefits, and when you look
at the data, you can see that greater than 99.5 percent of all
product approvals do not result in a class I recall, that the
majority of recall safety issues that do occur are postmarket
issues: manufacturing mistakes, labeling errors, etc. And
changes to a premarket system obviously can impact events that
take place after product approval.
Quality systems are the key to improving product safety. Of
all recalls, as we have looked at the data, approximately 90
percent of all of those have some relationship to quality
systems and improvements in quality systems therefore provide
the greatest leverage. Very preliminarily, we have looked at
2010 data, as I mentioned. That data seems consistent with what
we have seen to date with the other data, with a slight
increase in manufacturing issues. We are not clear if that is
statistical or not. We have also taken a look at class II
recalls, and preliminarily, the reasons for recall appear to be
consistent between class I and class II recalls.
So in conclusion, the agency has multiple control points to
ensure product safety and effectiveness, not just one: quality
systems, premarket approval, postmarket approval. The agency
has authority, extensive authority both pre- and postmarket,
and the agency's safety record has been very good over the past
years.
Thank you very much.
[The prepared statement of Mr. Hall follows:]
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Mr. Pitts. The Chair thanks the gentleman and recognizes
Dr. Jaffe for 5 minutes for an opening statement.
STATEMENT OF ROSS JAFFE
Mr. Jaffe. Chairman Pitts, Ranking Member Pallone, members
of the subcommittee, thank you for the opportunity to testify
today. My name is Ross Jaffe. I am a physician trained in
internal medicine who for the last 21 years has had the
privilege of working to help develop innovative medical
technologies.
In my role as a physician and venture capitalist, over the
last few years I more and more frequently face a frustrating
paradox. On the one hand, we live in a time of incredible
innovation in science and medicine that I see embodied in
fascinating technologies every day. On the other hand, more and
more often I am forced to turn down many of these important
medical innovations because our unpredictable regulatory system
here in the United States has stretched development time frames
and increased capital requirements needed to fund these
technologies, precluding adequate investment return for my
investors.
It is important to note that our investors are primarily
university endowments, foundations and pension funds, which
rely on us to generate a positive return on their capital. If
we do our jobs well, not only do patients benefit and
physicians have access to more innovative medical technologies,
high-quality jobs are created, universities can educate more
students, foundations can do more good works, and people can
retire in greater comfort, a real win-win-win system that
supports medical innovation and the U.S. economy.
Colleagues of mine who have testified during previous
hearings have described how most medical innovation comes from
small venture-backed companies. However, the growing
uncertainty with the FDA has dramatically reduced the amount of
investment available to fund innovative medical companies.
According to data from PriceWaterhouseCoopers, in 2007, 116
early-stage companies raised approximately $720 million in
initial financing. In just 4 short years, that investment
amount has dropped by more than 70 percent to just 55 companies
raising only $200 million. To put this in perspective, 2011 saw
the lowest level of venture capital investment in medical
startups in the last 16 years.
In a recent survey by the National Venture Capital
Association, which has been referenced this morning, 42 percent
of health care venture firms expect to decrease investment in
medical device companies over the next 3 years. In addition, 31
percent of firms expect to shift health care investment and
operational focus away from the United States towards Europe
and Asia. In both cases, regulatory challenges here in the
United States were cited as the primary factor for declining
investment and driving investment overseas. Indeed, it is now
common for many innovative lifesaving technologies, for
example, percutaneous heart valves, to be available for
patients in Europe years before they are available to patients
here in the United States.
Fortunately, within the last year or so, the FDA leadership
including Dr. Shuren has acknowledged how regulation is slowing
innovation and driving product development overseas. They have
begun internal efforts to improve FDA processes as illustrated
by a series of draft guidance documents released over the past
few months.
One notable efforts seeks to make explicit FDA
considerations and risk-benefit determinations for premarket
approval. Under the law, FDA is supposed to assess medical
technologies to assure that the probable benefits are greater
than the probable risks. Unfortunately, over the past few
years, many FDA reviewers appear to be applying a different
standard, weighing the probable benefit against any possible
risk, which is not the standard in the law. If implemented
appropriately, this guidance should make risk-benefit
determinations more patient-centric and evidence-based and
therefore improve the transparency, consistency and
accountability of FDA decision-making, and I was pleased to
hear today that that should be moving forward very quickly in
the next few months.
Beyond administrative changes under consideration by the
FDA, the MDUFA reauthorization being discussed at this hearing
will include additional process enhancements as well as needed
resources to increase the predictability of the process.
However, resources alone are not enough. We also need
meaningful operational improvements, not only through MDUFA but
also through additional legislation that leads to better
application of the least-burdensome principle, streamlining the
de novo process and revision of conflict of interest policies
to allow more leading experts to sit on FDA advisory panels.
In closing, let me be clear about one thing. We are not
asking for increased regulatory predictability, consistency or
efficiency at the expense of public safety. Innovation and
safety are not a tradeoff. It is not an either-or. We
absolutely need both. As investors, my colleagues and I pursue
medical innovations precisely because they are safer and more
effective for patients, preferably when they also can reduce
health care costs. We need to work together to assure a
regulatory system that supports the timely development of
innovative products and therefore enables safer and more
effective patient care.
Thank you.
[The prepared statement of Mr. Jaffe follows:]
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Mr. Pitts. The Chair thanks the gentleman, and Dr.
Kesselheim, you are recognized for 5 minutes for an opening
statement.
STATEMENT OF AARON S. KESSELHEIM
Mr. Kesselheim. Chairman Pitts, Ranking Member Pallone and
members of the Subcommittee on Health, thank you very much for
the chance to share my thoughts with you today about the
regulation of medical devices. I am Assistant Professor of
Medicine at Brigham and Women's Hospital and Harvard Medical
School in the Division of Pharmacoepidemiology and
Pharmacoeconomics.
One essential question being addressed in today's hearing
is whether requiring the FDA to loosen its standards for
medical device regulation would encourage innovation and help
patients. Some offer the European Union as a model because
high-risk devices generally make it to market sooner and more
easily there. The main reason is that E.U. device approval
usually only requires studies in small numbers of patients
showing the device appears to be safe and performs as expected.
Such evidence could include demonstrating that a new stent
expands appropriately in the coronary artery. There are no
requirements in the E.U. that companies demonstrate that their
devices benefit patients. By contrast, the FDA requires more
robust evidence of safety and effectiveness for many of these
implantable or high-risk devices. Thus, approval for the same
coronary stent might require showing fewer cardiac events or
the need for another invasive procedure.
The current E.U. system for approving medical devices
recalls the U.S. prescription drug market before 1962 when the
FDA only required limited studies of purity or safety before a
drug could be marketed, but after the thalidomide public health
crisis, legislation gave the FDA authority to compel reasonable
safety and efficacy data before a new drug could be sold. This
reform was almost derailed by accusations that it would
threaten the viability of the pharmaceutical industry, but what
happened instead was that the U.S. pharmaceutical industry grew
into one of the most profitable in the world. Why? FDA
validation meant that physicians could prescribe drugs
confident that a neutral expert body had certified their
efficacy and safety. Requiring companies to demonstrate that
their products were effective also created incentives for
manufacturers to impose a higher standard on their product
evaluation, leading to their developing some of the most
important medications we have, and today, nobody seriously
advocates returning to a time when we essentially let any drug
on the market and then figure out afterwards which ones were
useful or dangerous based on haphazard patient experience.
But this is indeed what is happening in the E.U. for
approval of even the highest-risk medical devices. For example,
the French company PIP is now under criminal investigation for
using non-medical-grade silicone in breast implants. PIP's
silicone implants were never submitted for marketing in the
United States. Or take the case of the PleuraSeal lung sealant
system, which was approved in the E.U. in 2007 to treat air
leaks after pulmonary resection surgery. A clinical study
conducted as part of an FDA premarket approval application
showed in 2011 that it had triple the rate of adverse events
compared to standard techniques. As a result, the device was
rejected by the FDA and a worldwide recall was initiated. Or
the CorCap cardiac support device, a harness for patients with
heart failure to improve their cardiac output. The device was
granted E.U. approval in 2000 but a pivotal U.S. premarket
trial conducted by 2004 showed no change in mortality, had
numerous irregularities including missing data for about 40
percent of patients, and it was not approved by the FDA. Thus,
the FDA requirement for premarket testing helped identify
unsafe or ineffective devices or prevented companies from
introducing substandard products, sparing U.S. patients from
being exposed to them.
But the FDA approval process is not perfect. Rigorous
premarket testing cannot identify all safety concerns, and the
FDA must use a least-burdensome approach in working with
manufacturers to decide what clinical data will be required. In
addition, experts have identified the clearance of high-risk
devices through pathways designed for low-risk devices as an
important inconsistency between the FDA's mandate and practice.
Thus, patient safety also requires enhanced postmarket testing
of new devices.
In the drug world, one of the lessons from the Vioxx
episode was that safety surveillance cannot be dependent on the
recent of adverse-event reports alone. More active postmarket
device surveillance would include development of national
registries with mandatory reporting of all implanted devices
along with automatic review of clinical experiences for certain
devices after a period of years to ensure that they are
producing the expected benefits. With today's advances in
informatics and epidemiological surveillance techniques, this
would not be problematic in terms of either cost or regulatory
burden.
In summary, patients and physicians do not want access to
any latest drug or device. Rather, they want access to products
that have meaningful clinical benefits with reasonable
assurance of safety. The Medical Device User Fee Act should
bolster this essential role of the FDA by increasing funding
for inspections of manufacturers, hiring of more reviewers or
safety experts, and by providing for more rigorous postmarket
surveillance so that devices proven to be effective and safe
can be used confidently by physicians for the benefit of their
patients.
Thank you very much.
[The prepared statement of Mr. Kesselheim follows:]
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Mr. Pitts. The Chair thanks the gentleman, and Dr.
Sedrakyan, you are recognized for 5 minutes for an opening
statement.
STATEMENT OF ART SEDRAKYAN
Mr. Sedrakyan. Thank you very much, Chairman Pitts and
Ranking Member Pallone and members of the subcommittee. It is a
pleasure to talk today. I am Art Sedrakyan. I am an Associate
Professor at Weill Cornell Medical College, and I am directing
the Patient-Centered Comparative Effectiveness Research Program
that is focusing on safety and effectiveness of devices. In my
career, I have been exposed to regulatory, academic and
manufacturing perspectives.
In the past decade, we have seen a lot of groundbreaking
devices that will change the practice of medicine. However, at
the same time, we have seen a number of high-profile failures
of approved medical devices. Many of these failures occurred
through these pathways which was called substantial equivalency
pathway, which was 510(k) pathway.
The mere presence of this pathway creates an environment
that is making people prone to committing errors. The absence
of funding for robust postmarket surveillance is an even more
important issue that we need to consider. The Centers for
Devices and Radiological Health recognized the limitations of
postmarket surveillance infrastructure today and they set up a
program called Medical Device Epidemiology Network, and it also
created a new entity that will look for a specific example, an
orthopedic device. It is called International Consortium of
Orthopedic Registries that is planning to bring together 15-
plus nations and registries from around the world to create an
infrastructure that will enhance postmarket surveillance in the
area of orthopedic devices. However, there is limited funding
to sustain and replicate this effort in many other areas.
The absence of robust postmarket infrastructure system, in
the absence of that, we need to make only gradual adjustment to
the balance of pre- and postmarket evaluation. It is important
for us to build these large comprehensive registries and
registry consortia and also advance the registry science. The
process will be through evidence-based innovation and will
protect manufacturers as well. Only after we build this strong
postmarket surveillance infrastructure will we accumulate
evidence of device performance in a variety of device
performance in a real-world setting. We can make those
adjustments at the premarket threshold.
Let me discuss the issue that shows the limitations for
both premarket and postmarket infrastructure and the investment
we have to make to ensure that we don't get disasters in the
future. There are over 270,000 hip replacement devices used in
the country, and this is a very safe operation. There are some
devices that are very successful and have 95 percent success
rate over a 10-year period. Even in this environment where
there are very successful devices on the market, through the
510(k) pathway new devices were introduced, so-called metal-on-
metal devices, and a specific example is the ASR device. The
device has been approved through the path of substantial
equivalency and used a predicate device of the same company
that if you look closely does not really resemble the original
predicate device. It has undergone substantial transformation.
Over the iterative cycle, I was able to--these products entered
the market because you could--that if you use one predicate as
a predicate for another device and then so forth encourages
vicious cycle for bringing device that might be dissimilar to
the previous device that has been approved.
Without any evidence, these metal-on-metal devices were
quickly adopted by surgeons and registries around the world
reported really disastrous outcomes with this particular
implant. DePuy recalled 93,000 of these devices out of the
market, and the evidence has been summarized in our paper and
also well covered by Barry Meyer at New York Times.
Interestingly, there would be more than 50,000 patients that
will undergo this serious revision surgery in the next 10
years, and this is going to cost American taxpayers billions of
dollars of additional costs, and this has--I am not aware of
any discussion between CMS and manufacturers to cover side
effects related to faulty medical products.
So I have some graphic pictures in my testimony that show
that these revision surgeries that are happening are not really
trivial problems. People have substantial suffering related to
these procedures.
I have to also note that even though European registries
were the first and Australian registries were the first to see
these problems, they are not necessarily the best registries
that we have today in the world and we should build much more
robust infrastructure system in this country and sometimes
multinational infrastructure to be able to prevent this
happening in the future, and one of the most important ways
that we can do that is through public-private partnership, and
a public-private partnership that can be led by FDA and involve
stakeholders in partnership with manufacturers and insurers.
Thank you very much.
[The prepared statement of Mr. Sedrakyan follows:]
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Mr. Pitts. The Chair thanks the gentleman and recognizes
Ms. Swirsky for 5 minutes for an opening statement.
STATEMENT OF LISA SWIRSKY
Ms. Swirsky. Good afternoon. My name is Lisa Swirsky and I
am a Senior Health Policy Analyst at Consumers Union. Consumers
Union is the publisher of Consumer Reports magazine and Best
Buy Drugs. We also have a Safe Patient Project, which is a
campaign to improve the safety and efficacy of devices. We are
also member of the Patient Consumer and Public Health
Coalition, which is a broad coalition of public interest groups
interested in the safety and efficacy of drugs and devices, and
some of our comments today reflect the broader interest of that
community.
Consumers Union urges Congress to take a balanced approach
to reauthorizing MDUFA, focusing both on the real need to keep
deficient devices off the market while also providing timely
access to safe and effective devices. Safety failures such as
those that occurred with metal-on-metal hips and surgical mesh
resulted from failures in the device regulatory system,
particularly the problem 510(k) process. But we would also urge
Congress to understand that behind those failures, there are
real people. Lana Keaton is one such consumer. She was a
previously healthy woman who was treated for what was a pretty
routine condition for a middle-aged woman, incontinence. She
went on for surgery for insertion of a synthetic mesh bladder
sling, which is a product that was cleared through the 510(k)
system. She awoke from surgery in extreme pain due to
complications from the mesh, and she has had to undergo 17
surgeries, and she has another one upcoming.
CU urges Congress to remember the experiences of hundreds
of thousands of people like Lana who have been injured by
defect devices as it considers reauthorization of the medical
device user fee program. Our priority is that these devices
work and that they don't hurt people, and we believe that with
proper resources, we can have a streamlined timely system
without sacrificing safety.
To this end, we would ask Congress to strengthen the
premarket approval process for devices. In particular, Congress
should pass legislation ensuring that recalled devices cannot
be used as a predicate for subsequent devices. Congress should
also shore up the system for monitoring devices once they are
already on the market by providing FDA with the authority to
require postmarket studies when it deems necessary to ensure
the safety of devices and also to improve postmarket
surveillance tools such as Sentinel and the adverse event
reporting system.
CU has reviewed provisions of the agreement as described in
the minutes from the FDA's January 31st meeting with industry,
and we offer the following comments and concerns on the
outlines of the agreement in principle.
Overall, we feel that the user fee amount in inadequate.
During the course of negotiations with industry, the FDA
indicated it would need somewhere between $770 million to up to
$1 billion to implement the program enhancements that it was
asking for. Now, while we understand that FDA has since scaled
back those proposals in light of the lower-than-expected user
fee, nonetheless, a lot of those program enhancements still
remain in the agreement and we are concerned that as long as
they remain in the agreement without dedicated funding, they
will become an unfunded mandate on an agency that is already
struggling to meet current requirements. And we would ask that
if Congress thinks that these enhancements are beneficial, that
they appropriate adequate funding.
We also are concerned that the agreement overemphasizes the
achievement of performance goals when device applications are
reviewed and processed within a reasonable time frame because
the application is sound and the device is safe and effective.
This is obviously a win-win for consumers and industry.
However, there is no mention in the agreement that these goals
are conditioned on the overall quality of the products, the
complexity of the products, the benefit of the products to
consumers or really any factors that may be relevant to
protecting the public health. Notably, the word ``safety'' does
not appear once in the minutes from the meeting where industry
and FDA came to agreement. We consider this a striking
omission, given recent notable safety lapses by the device
industry.
Even more worrisome, the agreement in principle references
total time to decision, goals based on calendar years in
addition to the goals based on FDA days. Current performance
goals stop the clock when the FDA sends an application back to
a device manufacturer when the agency needs additional
information. Under the agreement, the FDA is kept on the clock
even when it needs to get further information. CU opposes any
kind of binding of the FDA to get the information that it needs
to ensure the safety and adequacy of devices.
We have further concerns about provisions in the agreement
that call for incorporating the patient perspective and risk-
benefit considerations. The industry has requested that groups
that represent patients with a specific disease represent the
patient perspective. However, in our experience, many of these
patient groups are heavily funded by industry. Patient
representatives used for these purposes should be held to
conflict of interest standards and should be required to
disclose any financial ties with industry.
Finally, as Congress considers MDUFA, we urge it to provide
a direct seat at the table for consumers in future
reauthorization negotiations. While the stakeholder meetings
that FDA conducted with consumer groups was an advancement over
prior authorization processes, they still kept consumers at
arm's length from negotiations that have significant
implications for the public health.
Thank you.
[The prepared statement of Ms. Swirsky follows:]
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Mr. Pitts. The Chair thanks the gentlelady and now
recognizes Mr. Shull for 5 minutes for an opening statement.
STATEMENT OF JAMES SHULL
Mr. Shull. My name is Jim Shull. I am from Browns Mills,
New Jersey. I would like to thank the committee for allowing me
to speak here today.
My story goes back to 2005 when I was told I had a hernia.
I woke in the recovery room from the surgery in excruciating
pain. Two days later, I was in such pain that I couldn't stand
up straight or barely walk. I called my surgeon's office and he
told me to meet him at the emergency room. He took me into an
examination room, looked at the surgical site and told me that
it was very infected. He prescribed an antibiotic, and morphine
for the pain, but nothing seemed to help. The infection was so
bad that I had streaks running down my groin.
I continued to call the surgeon over the next 2 weeks only
for him to tell me that I am a slow healer. At my 6-week
follow-up I explained again to my surgeon that I was in
unbearable pain, so, he decided to inject my groin with
Novocain right through the incision and sent me back to work.
The pain I was feeling was as if there was a sharp object
left inside of me. After continuously going back to the surgeon
he decided to send me to pain management, where over the course
of 6 weeks the pain doctor injected my groin upwards of 70
times.
Nothing would help the pain so I decided to investigate
myself. I went back to the surgeon and explained to him what I
had found. Only then did he tell me that he had put a synthetic
mesh inside of me and told me that it was not the mesh, because
the mesh is inert and my problem has to do with the nerves in
my groin. I tried to go back to work because I couldn't afford
not getting a paycheck, but the pain was so unbearable that I
ended up in the ER. The doctor in the ER did a CT scan only to
find nothing. That is because the mesh is transparent and
cannot be seen on X-rays. The doctor in the ER told me that I
probably had diverticulitis and that I needed to follow up with
a GI specialist. Those tests came back negative also.
I decided to get a second opinion from another surgeon and
asked if he could remove the mesh from inside of me. He told me
that he couldn't remove the mesh but could do an exploratory
surgery to see if the nerves were stitched up. This surgeon did
cut and tie off one of the nerves in my groin and thought that
it would ease my suffering. After returning to him for 6 weeks
in unbearable pain, he told me that there was nothing else he
could do for me. So I was on my own.
I finally did find a surgeon in another State and he agreed
to see me. When he examined me he told me that he knew exactly
what was wrong with me but to be sure he sent me to have an
MRI. I went back to this surgeon and he showed me the problem.
There it was: a hardened piece of synthetic mesh inside of me.
So finally after almost 2 years of unbearable pain, I found
someone who could give me some answers. The surgery to remove
the mesh took 3-1/2 hours. When I awoke in the recovery room,
the surgeon was at my bedside. He told me that he was sorry and
that I would be in pain for the rest of my life. The surgeon
explained to me that he had removed a balled-up piece of
concrete from my groin, that the mesh had hardened and balled
up, and had encapsulated the other two main nerves in my groin.
In order to get the mesh out, the nerves had to be severed. He
explained to me that the mesh was so hard, that when I moved it
was acting like a saw and cutting into the surrounding tissue.
I had a 3-inch gash in my pelvic floor along with hundreds of
smaller cuts and tears.
In 2008 I was diagnosed with a degenerative nerve
condition. The pain that I suffer through on a daily basis
consists of constant burning and sharp pains in my groin and
upper thigh. My groin and upper thigh are purple and brown
color because of the nerve condition I now have. I must take
three strong medications--OxyContin, Percocet and Tramadol--
just for the pain alone. Every 6 months I have to have radio
frequency ablation done at the spinal level where the nerve
roots are located. It is very uncomfortable for me to sleep at
night without the help of medication. Because of this product I
am no longer able to work as a printer.
When I was a teenager, I had a hernia. That hernia was not
repaired with mesh, but was stitched back together. Thirty-four
years later and I still have no problems with that repair. The
mesh that was put inside of me caused so much damage that none
of the nerves will ever be able to be repaired and will never
grow back. I live a life of pain because of a product that
never had any kind of clinical testing and slipped through the
back door of what you know as the 510(k) process based on the
use of predicate devices. I am left disabled because the FDA
considered surgical mesh equivalent to that of sutures and
allowed it to be implanted in patients like me.
After years of people reporting problems and investigations
into synthetic mesh, the FDA published a public health warning.
Unfortunately, the warning was only for synthetic transvaginal
meshes that are used in woman. There was no public health
notification for hernia meshes, which are just as tragic and
cause horrible complications for men and women alike. Failing
to address the hernia mesh issue puts too many people in
danger. I think synthetic mesh should not be on the market
because it is unsafe and I have proudly taken the challenge to
work to prevent this from continuing to happen to others.
In closing, I would like to say that I am only one face in
thousands of people that this has happened to, and the sad part
of it all is that I feel that I may be one of the lucky ones.
This committee can change the laws to improve the safety of
medical devices and put patients first. Surgical mesh and other
medical devices should be tested for safety before they are
allowed to be implanted into people like myself. We also need a
national system to track what happens to patients like me after
devices are implanted, to catch these problems as soon as
possible.
Thank you.
[The prepared statement of Mr. Shull follows:]
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Mr. Pitts. The Chair thanks the gentleman and thanks to all
the panel for your testimony, and we will now begin
questioning, and I will recognize myself for 5 minutes for that
purpose.
Dr. Jaffe, you presented some very compelling data in your
testimony, and it reiterates what we have been hearing from
medical device innovators who have testified before this
committee and those we speak with back in our home districts.
PWC reports show that in 2007, 116 medical device startups had
$720 million in funding, and that last year, 55 companies
received under $200 million. This reflects more than a half-
billion-drop in funding of medical device startups. Can you
explain the impact of this alarming drop in funding, the impact
on patients and jobs?
Mr. Jaffe. Thank you, Chairman Pitts. Let me start with the
jobs issues first. Clearly, each of these companies may only
have five to ten employees who start up funding, but if they
are successful, they will grow, and many successful medical
device companies we are involved with have hundreds of
employees. We also know from data that for every one job we
create in a company, there are three or four created in the
community to support to those jobs, so clearly there is an
economic impact.
The more important issue, though, is really the impact on
patients and potential technologies for those patients. I have
an unusual job in the sense that I invest in things I hope I
never have to use personally and I hope none of you or your
loved ones ever need any one of the products we develop. But if
you are someone with the issue that our technologies address,
you will be very grateful they were developed. And the sad part
of all this is that there are many technologies that I
mentioned earlier that I see every day that deserve development
but I can't pursue because the time and capital requirements
would be too great to allow me to make returns I need to
satisfy my investors' requirements, and it is the challenge of
the system we all need to work on.
Mr. Pitts. Thank you.
Mr. Perez, can you give us an example of difficulties your
company has had with the FDA? Have you experienced an increase
in how long it takes to get through the FDA process, and why do
you believe that doubling the amount you pay in user fees is
going to solve what is partly a management issue?
Mr. Perez. Well, I think the performance metrics that are
specifically addressed in the MDUFA agreement go back to some
of the issues that we have had with the FDA. I will give you an
example. We had a pre-submission hearing with the FDA on a
technology, and then we went almost 14 months before we heard
back from the FDA, and a lot of that had to do with the fact
that there was not agreement within the FDA on how to go
forward with the approval process of a product like this, and
this specific MDUFA agreement addresses that where we have a
pre-submission meeting, there has to be agreements and those
agreements can't be changed. We had another example where we
had an agreement with the FDA on a clinical trial. We moved
forward on the clinical trial. We got about halfway through the
clinical trial and the requirements of that trial were changed.
So once again, I think some of the things that we are
trying to address regarding predictability and accountability
are specifically addressed in this MDUFA agreement, and I think
some of the challenges that we have, I am not saying they are
all going to go away but I think some of the specific
challenges that we have had will be addressed with this new
agreement.
Mr. Pitts. Ms. George, how does the proposed user fee
agreement improve predictability and consistency with respect
to FDA's review of medical devices, if you can be specific?
Ms. George. I believe that there are a couple of areas that
it does that. First off, that through the pre-submissions
process, as was stated by Mr. Perez, there would be agreement
as to what the requirements are ahead of time, early, prior to
submission, so that the manufacturer when they submit their
510(k) it includes the requirements up front so that it can
flow through the process more quickly. I also think that the
interaction requirement that we have put into the agreement of
having earlier interaction with the FDA so that we know what
the questions might be if they are going to have them, that
will support it, and then the added management as through the
resources that are going to be added, that will ensure
consistency in how they make those determinations so that a
reviewer by themselves doesn't have to make that decision.
Mr. Pitts. Professor Hall, from what I understand, FDA has
extensive postmarket authority for medical devices. Would you
walk us through that authority, please?
Mr. Hall. There are a number of authorities the agency
currently has. They include obtaining information through
medical device reports, so-called MDRs, the MedSun process,
which is an active postmarket surveillance system linking about
350 hospitals. There is a 522 order process. You have special
controls that specifically include the statutory authority for
postmarket surveillance obligations, patient registries and
other tools. In the PMA world, you have conditions of approval.
The QSR systems include postmarket surveillance. We call them
CAPA, corrective and preventive action, processes that, for
example, require product trending, root-cause analysis, etc. So
those are just a number of the statutory systems that are
currently in place.
Mr. Pitts. Thank you. My time is expired. The Chair
recognizes the ranking member, Mr. Pallone, for 5 minutes for
questions.
Mr. Pallone. Thank you, Mr. Chairman.
I wanted to ask Ms. Swirsky and Ms. George, only because of
time limitations, because of these advisory committees and
conflict of interest. As you know, industry and some patient
groups have focused on removing limits on how many experts with
financial conflicts of interest may serve on the committees.
Many consumer groups are concerned that for FDA and the public
to be confident in the objectivity of the advice FDA receives,
every effort must be made to minimize the number of conflicted
experts that serve on these committees. I would like to ask Ms.
Swirsky, if you could suggest ways that FDA could broaden its
pool of experts. Let me start with that and then I will go to
Ms. George. How would you suggest the FDA could broaden its
pool of experts?
Ms. Swirsky. I want to say first off, I think the FDA has
already suggested that those caps on the waivers, which I think
are the subject of many of the bills in the House and some in
the Senate, haven't really been at issue. They are not using
the existing caps.
Mr. Pallone. Right. She mentioned that when we had the
Commissioner here last week.
Ms. Swirsky. So that suggests to us that there is some
broader problems.
Mr. Pallone. Right. Just give me your suggestions, because
I don't have a lot of time.
Ms. Swirsky. I am sorry. So some of our suggestions, we
would hope that the FDA would be ripe for a task force to bring
in stakeholders, various stakeholders, consumer groups and
industry to sort of come together to look at some of the
barriers and identify some solutions. But some of the solutions
I think we and other consumer groups have thought about is
first of all, creating better awareness of advisory panels. I
think right now there isn't great awareness of it, and so what
you have now are self-selected folks who sign up for these
advisory panels, and some ideas include trying to work with
medical schools to make this a part of their curriculum so we
can create more prestige around the advisory panels. Obviously
we can pay them more, which is probably not in the cards for
the short term. But also I think there is a lot of evidence
that about 50 percent of academic researchers aren't conflicted
at all so we need to tap into that pool, and research suggests
that academic medical centers have fewer conflicted members,
and so bringing them into the process and getting their input
in how we can make it more attractive to them.
Mr. Pallone. Thank you.
Ms. George, first I wanted to thank you for coming to that
FDA roundtable we had at Rutgers with the Commissioner, but
would explain why elimination of the caps on waivers would be
helpful, given as Ms. Swirsky said, that the FDA hasn't come
anywhere near reaching its cap to date? Do you think it would
be helpful? And if you want to comment on broadening the pools
also but----
Ms. George. One of the challenges----
Mr. Pallone Quickly because I have one more question.
Ms. George. One of the challenges I think that does occur
with the panels is, anything that goes to panels is innovative.
It is new technology. It is new clinical science and there are
not a lot of available people out there to actually come in to
be those experts, to come in and answer the questions, to be
able to ask industry the questions. So one of the challenges
that we have as a manufacturer if we bring something to panel
is, we have probably already tapped a lot of those people to
help us in the development and in the creation of the
technology or science and so the FDA has limited people
available that they could use, so that does cause some aspect
of conflict.
Mr. Pallone. Let me ask Mr. Perez, I have one more
question. I have about a minute left. You know, I understand
the negotiation over the medical device agreement wasn't easy,
but we have heard from the drugs and biologics trade
associations that they are committed to a clean PDUFA, and
while they may have some additional legislation they would be
happy to see enacted as part of the UFA legislation package,
they don't want anything that would slow down or jeopardize the
passage of that package. So I just wanted to ask you, are you
committed to seeing that nothing slows down or stands in the
way of passage of MDUFA as part of the package of FDA
legislation? I am asking you to take the same pledge.
Mr. Perez. I think we share a common goal here, that we
want to get this done in a very timely manner. We know many
members have already introduced some legislation all in an
effort to improve and help the FDA be more successful but I
think right now we need to make sure that we balance those
efforts with trying to get the MDUFA passed in a very timely
manner. So we would like to work with the members of the
committee, to listen to them, and I think it is very, very
important to get this done. Dr. Shuren outlined a timetable and
I hope we can stick to it.
Mr. Pallone. All right. Thank you so much.
Mr. Pitts. The Chair thanks the gentleman and recognizes
the vice chairman of the committee, Dr. Burgess, for 5 minutes
for questioning.
Mr. Burgess. Thank you, Mr. Chairman.
Mr. Perez, a valid point, what a lot of people don't
realize about the user fee agreements is when they expire on
September 30, this is not like the typical Congressional action
where we can say the dog ate my homework so I am going to give
myself an IOU for the next couple of months. These are
voluntary funds that are provided by the industry, and without
the user fee agreement and in force, those monies simply stop
on October 1st. Is that correct?
Mr. Perez. That is correct.
Mr. Burgess. So this timeline that we are looking at now is
one with a great deal more severity than the usual
Congressional timelines. I mean, I forget, we had, what, 35
different temporary patches to the FAA reauthorization in the
last 10 years. We can't do this.
Mr. Perez. I agree. We have to get it done.
Mr. Burgess. We have to get it done, and so I appreciate
all of you being here and Dr. Shuren being here because I think
this is--you know, we may disagree about some parts of this but
we all understand how important it is to get this done.
Dr. Jaffe and Dr. Kesselheim, let me just take advantage of
the fact that you two are sitting next to each other and you
seem to have vastly different views of the world. You both
heard each other's testimony. Is there any common ground
between you or are we left with this rather stark definition on
either side of what an ideal user fee agreement would look
like?
Mr. Jaffe. Well, I don't know where the differences are
between us on the user fee agreement. I certainly didn't hear
any concerns about the need for more resources for the FDA and
for process improvements.
Mr. Kesselheim. I would agree with that. I mean, I think
that the need for greater funding for a lot of the essential
work that the FDA does is essential and it would be my
preference to see that money come directly from Congress, but
since that is not going to happen, I think that the user fee
agreement is essential and a lot of the issues we will deal
with by improving the----
Mr. Burgess. Let me interrupt you in the interest of time
because they just called a vote. Dr. Jaffe, you describe a
world in which the risk-averse nature of the agency charged
with protecting the public interest, the risk-averse nature has
damaged your business model. Is that correct? Did I
misinterpret that?
Mr. Jaffe. Yes, Dr. Burgess.
Mr. Burgess. And Dr. Kesselheim, your view seemed to be
that it doesn't matter about the damage because these companies
are out there trying to push products out on the American
public, the unsuspecting American public that are bad products
and the FDA has to stand as the last bastion of defense against
the industry and these bad products. Did I miss something in
the testimony of two individuals?
Mr. Kesselheim. Well, so I would first say that for many
products in the 510(k) clearance process, for 95 percent of
products the time to market in the United States and the
European Union is not different, that what we are talking about
are the highest risk products that arrive at the E.U. market
sooner, and I think as I said before, the essential reason for
that is that they are just not being tested for efficacy and
for----
Mr. Burgess. Dr. Jaffe, do you agree with that?
Mr. Jaffe. I don't fully agree with that, I must say. You
know, we do go to Europe early because there is a more
straightforward path but we do test products in Europe. They do
have to have data to get approved. We have a company selling in
Europe a leadless cardiac defibrillator which could be a major
improvement over the problems we have had with leads here in
the United States. That product has been on the market for 3
years in Europe and it will probably be several more years
before it is approved here.
Mr. Burgess. Now, let me ask you something. Do they have a
postmarket surveillance program in Europe?
Mr. Jaffe. The company has continued to do studies but I am
not sure--I am not directly involved in it. I don't know if
they are required to but the company has continued to do
studies of that product both in Europe and it has completed a
clinical trial here in the United States which is submitted.
Mr. Burgess. Now, will that company be able to use any of
that data when it goes to the FDA to present its case?
Mr. Jaffe. I do not know the answer to that question. I am
not directly involved.
Mr. Burgess. Mr. Hall, do you know?
Mr. Hall. It is possible, assuming that it meets the U.S.
criteria for informed consent, data, validity, etc., but there
are many situations where data can be used.
Mr. Burgess. Now, I have got a list of a number of things
where the postmarketing authority exists in the device world
and is missing from the drug world. Now, there are some things
where drugs and devices share some postmarketing authority,
things like adulteration, misbranding, manufacturer changes
both drugs and devices are required to report but you look at
things like classification based on risk, devices have it,
drugs don't; user reporting, devices have it, drugs don't;
reports of removals or corrections, devices have it, drugs
don't; tracking, devices have it, drugs don't. I mean, it looks
like the Food and Drug Administration is already applying many
of these standards in the device world maybe even a little bit
more stringently than the drug world. Do you agree with that,
Mr. Hall?
Mr. Hall. There are obviously a number of differences
between drugs and devices. The agency has a plethora of
postmarket authorities in the device world. Some of them do not
exist in the drug world. In part, that is because of the
differences between drugs and devices. You don't have an
implantable drug, you know, as a general rule.
Mr. Burgess. You do for some hormonal agents.
Mr. Hall. As a general rule, is what I am trying to say.
Mr. Pitts. The Chair thanks the gentleman and recognizes
the ranking member emeritus--I mean ranking member of the full
committee, Mr. Waxman, for 5 minutes.
Mr. Waxman. Thank you. I will be emeritus when we get the
control back and then I will be chairman, but thank you very
much for calling on me and I thank this panel for their
testimony. I had a chance to review some of the testimony, and
I have had my staff here throughout your presentation.
Dr. Kesselheim, I must express alarm over your article
describing the harms caused by the devices approved in Europe
first and then later found to be ineffective or, worse, harmful
to patients. This is important information for us to have given
that so many in the device industry have complained that FDA is
depriving Americans of the innovative devices patients in the
E.U. get so early. Obviously as you have shown, this is not
always such a good thing. Your New England Journal article also
describes what are some critical fundamental differences
between the E.U. and the U.S. systems. You say that ``the E.U.
system is a part of a framework for commerce which originated
as a means of streamlining trade and coordinating
manufacturing, safety, and environmental standards'' in the
E.U. Your article also states that so-called notified bodies,
which are for-profit independent companies that specialize in
evaluating many products, not just medical devices, are not
``designed to work as public health agencies,'' and the
approval standards in the E.U. are quite different from ours.
Device manufacturers have only to prove that the device works
as intended, not that it is effective at treating or curing the
particular indicated condition.
So yet in recent months, many have argued that we should
reformulate our device regulatory system so that it more
closely resembles the E.U. Let me ask you, based on what you
have learned from your study, do you agree that we should look
to the European system as a model for how we regulate devices
in the United States?
Mr. Kesselheim. Absolutely not. You know, there is no
evidence that I have found in all the places that I have looked
that suggests that the model for device approval in the E.U. in
any way benefits patients overall as compared to the U.S.
system, and indeed these notified bodies have major problems
with conflicts of interest and their independence, and in fact,
they only evaluate devices for approval whereas the competent
authorities in the E.U. are the ones charged with safety
evaluations. So the safety and the approval evaluations in the
E.U. are separate and that is just not the way to effectively
protect the public health.
Mr. Waxman. Some of the bills that are being proposed
change FDA device regulation to make our system look a lot more
like the E.U. system. Let me ask you about one of them that
would expand the device center's so-called third-party review
program. Currently, that program permits third parties to
review certain 510(k) applications and provide recommendations
to FDA on whether the agency should clear a particular device.
FDA has 30 days in which to make a final decision, but it is
FDA that has the final say. That is existing law. One bill has
an alteration of the scheme to make the third party's
recommendation binding on FDA if FDA fails to respond in 30
days. The bill would also expand the types of devices that
these third parties are permitted to review to include
``permanently implantable or life-sustaining or supporting
devices.'' These outside reviewers are not currently allowed to
review these devices.
Dr. Kesselheim, as an expert on the U.S. and E.U. systems
of medical device oversight, do you believe this legislation is
a move in the right direction? Would you be concerned about
these kinds of changes to the program?
Mr. Kesselheim. Yes, I believe this is definitely a move in
the wrong direction, and I would be concerned about these types
of changes. First of all, there is plenty of peer-reviewed
evidence showing in the drug realm that decisions made at the
end of a fixed regulatory period end up more likely leading to
drugs that have safety problems later on down the road, so
imposing this 30-day fixed time limit on the FDA in terms of
devices is bad policy, and I also think that increasing the
role of these independent agents into the evaluation of the
most highest-risk devices would again move us more towards the
E.U. equivalent, notified bodies, and it would be bad policy,
and there is very little individual oversight of what these
notified bodies are able to do. Manufacturers are able to game
the system in a way and select which notified bodies they want
to based on which are known to provide a faster path to
approval, and I just think it would be a bad idea.
Mr. Waxman. It is ironic that Governor Romney is attacking
President Obama saying he wants us to be more like the
Europeans. That may or may not be right, but in this case, we
don't want to be more like the Europeans. The FDA gives a seal
of approval that is respected all around the world for our
drugs and devices and we are better able to protect the public
health with our present system.
Mr. Kesselheim. Indeed, I do, and in fact, a lot of the
European authorities rely on the studies done for FDA approval
in order to make decisions about payments and use of the
devices there. So indeed, you know, authorities around the
world rely on the FDA system.
Mr. Waxman. Thank you, Mr. Chairman.
Mr. Pitts. The Chair thanks the gentleman. We are going to
try to wrap this up. We are in the middle of a vote. Dr.
Cassidy, 5 minutes for questions.
Mr. Cassidy. So Mr. Hall and Dr. Jaffe, just to be on
record, are you all in favor of this bill, the number three, if
you will?
Mr. Jaffe. The MDUFA reauthorization? Yes.
Mr. Cassidy. And Mr. Hall, are you?
Mr. Hall. The agency needs adequate resources. I am Don
Quixote on this. I prefer the funding to be from public
sources. I recognize the practical aspects and problems with
that right now.
Mr. Cassidy. OK. That sounds good.
Now, Dr. Kesselheim, I think William Moser said let us use
our drugs while they still work, and that was obviously way
back when, when there was poor regulation. You suggest it still
may be true in Europe of medical devices. And Dr. Jaffe,
obviously there is tension there that was earlier alluded to. I
am way out of field. I am a gastroenterologist. But don't I
recall something--I was looking at but I couldn't find it--that
there was an artificial disc that was being used by maybe
orthopods or spine surgeons that had been implanted in lots of
folks and turned out not to be efficacious?
Mr. Kesselheim. As far as I am concerned, yes, there have
been examples of those sorts of orthopedic spine devices that
turn out later to have been unsafe or not work, yes.
Mr. Cassidy. Now, Dr. Jaffe, how would you--understanding
there has to be a kind of movement towards innovation but
understanding that there are these instances where things are
not efficacious, that they are approved and they are put in a
lot of people and they cost a lot of money. How would you
balance that tension?
Mr. Jaffe. Congressman, I just wanted to say clearly that
we have not advocated for any type of European system here in
the United States, and we still believe in the importance of
good clinical safety and efficacy studies. The challenges we
have with the FDA are less around those standards than they are
about the unpredictability and the delays and the difficulty in
getting decisions made that cost our companies millions that
stretch time frames in a great distance.
Mr. Cassidy. So you are not so concerned with the paradigm
that they use, rather how they implement it, if you will?
Mr. Jaffe. Exactly. It is more their internal management.
That is why these guidance documents that Dr. Shuren referred
to are so important, making the clinical risk-benefit
determination much more transparent and clear and accountable
so we can review over time, make sure that we are in agreement
to start and we are in agreement at the end of the process
using the same standards because we have seen standards change
as reviewers change. We have seen delays in getting to
decisions. We see----
Mr. Cassidy. I have limited time, so Dr. Kesselheim, again,
I am just kind of curious about this, and again, I am trying to
dig from the recesses of my memory, so if I say something
stupid, it won't be the first time. Somebody has pointed out to
me that some of the things that are approved, maybe certain
types of stents for cardiac disease, turn out not to be
efficacious but there is no vested interest in terms of
learning efficacy in terms of your outcome data is--if your
outcome data is mortality, it is a long study, very expensive,
etc. Surrogates may not be adequate markers for the ultimate
outcome. And Dr. Sedrakyan, I think I saw you nodding your
head. Would you all comment on that? Because again, I am trying
to understand this issue. I am not challenging anybody. I am
just trying to understand.
Mr. Sedrakyan. I can answer that. In many situations, it is
possible that a device will take time until side effects will
develop, and a large number of products will be already on the
market with consequences for public health. Now, the best
answer to that kind of problem is to have a worldwide network
that will help us determine the side effects early.
Mr. Cassidy. But side effects is lack of clinical efficacy.
It may decrease angina, for example, but it may not prolong
life. Do we need 10,000 people and 5,000 get a stent and 5,000
don't? Do you see what I am saying? Can we use surrogate
markers?
Mr. Kesselheim. I mean, I think that there are surrogate
markers that have been validated as relatively well predicting
final outcomes, and in those cases, surrogate markers are
useful. There are also, you know, new techniques for doing
randomized trials in detecting efficacy so that they can be
done in a more expedited way, and I am also more in favor of
promoting an efficient and predictable FDA regulatory process
as well, but I think that at the end of the day----
Mr. Cassidy. Let me cut you off because I told my colleague
I would give him the remainder of my time, because I think I
got your point.
Mr. Burgess. I thank the gentleman for yielding.
Dr. Kesselheim, if I could just ask you very quickly, are
you currently involved either with the plaintiff or defense in
any of the product liability lawsuits involving, say, the
artificial hip?
Mr. Kesselheim. No.
Mr. Burgess. And the same question to you, Dr. Sedrakyan?
Mr. Sedrakyan. No.
Mr. Burgess. Mr. Shull, let me just ask you, your story is
very compelling. Certainly at some point there has been a
lawsuit involved, I would assume.
Mr. Shull. Yes.
Mr. Burgess. And currently your lawsuit is against whom?
Mr. Shull. It has settled.
Mr. Burgess. With whom did you settle?
Mr. Shull. That would be the doctor.
Mr. Burgess. Was the product you referenced in your case,
was that product ultimately recalled from the market?
Mr. Shull. No, it was never recalled.
Mr. Burgess. Did you file suit against the company?
Mr. Shull. I did, but the product was deemed used off label
and----
Mr. Burgess. So it was the physician involved, not the
company?
Mr. Shull. The company exchanged testimony for me to drop
the suit against them.
Mr. Burgess. All right. I thank you for that.
I will yield back, Mr. Chairman.
Mr. Pitts. The Chair thanks the gentleman. We have a
unanimous consent request.
Mr. Pallone. Mr. Chairman, I would ask unanimous consent to
enter into the record first the testimony from Public Citizen;
second, testimony from American Congress of Obstetricians and
Gynecologists; and third, two New England Journal of Medicine
articles, one, ``Postmarketing Surveillance of Medical
Devices--Filling in the Gaps,'' and second, ``Regulation of
Medical Devices in the United States and European Union.''
Mr. Pitts. Have you shared that with us?
Mr. Pallone. Yes.
Mr. Pitts. Without objection, so ordered.
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Mr. Pitts. That concludes the second panel. I would like to
thank the witnesses and members for participating in today's
hearing. I remind the members that they have 10 business days
to submit questions for the record, and I ask the witnesses to
respond promptly to the questions. Members should submit their
questions by the close of business on Thursday, March 1.
Without objection, the subcommittee is adjourned.
[Whereupon, at 1:57 p.m., the subcommittee was adjourned.]
[Material submitted for inclusion in the record follows:]
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