[House Hearing, 114 Congress]
[From the U.S. Government Publishing Office]
LEGISLATIVE HEARING ON 21ST CENTURY CURES
=======================================================================
HEARING
BEFORE THE
SUBCOMMITTEE ON HEALTH
OF THE
COMMITTEE ON ENERGY AND COMMERCE
HOUSE OF REPRESENTATIVES
ONE HUNDRED FOURTEENTH CONGRESS
FIRST SESSION
__________
APRIL 30, 2015
__________
Serial No. 114-35
[GRAPHIC NOT AVAILABLE IN TIFF FORMAT]
Printed for the use of the Committee on Energy and Commerce
energycommerce.house.gov
____________
U.S. GOVERNMENT PUBLISHING OFFICE
96-202 PDF WASHINGTON : 2016
________________________________________________________________________________________
For sale by the Superintendent of Documents, U.S. Government Publishing Office,
http://bookstore.gpo.gov. For more information, contact the GPO Customer Contact Center,
U.S. Government Publishing Office. Phone 202-512-1800, or 866-512-1800 (toll-free).
E-mail, [email protected].
COMMITTEE ON ENERGY AND COMMERCE
FRED UPTON, Michigan
Chairman
JOE BARTON, Texas FRANK PALLONE, Jr., New Jersey
Chairman Emeritus Ranking Member
ED WHITFIELD, Kentucky BOBBY L. RUSH, Illinois
JOHN SHIMKUS, Illinois ANNA G. ESHOO, California
JOSEPH R. PITTS, Pennsylvania ELIOT L. ENGEL, New York
GREG WALDEN, Oregon GENE GREEN, Texas
TIM MURPHY, Pennsylvania DIANA DeGETTE, Colorado
MICHAEL C. BURGESS, Texas LOIS CAPPS, California
MARSHA BLACKBURN, Tennessee MICHAEL F. DOYLE, Pennsylvania
Vice Chairman JANICE D. SCHAKOWSKY, Illinois
STEVE SCALISE, Louisiana G.K. BUTTERFIELD, North Carolina
ROBERT E. LATTA, Ohio DORIS O. MATSUI, California
CATHY McMORRIS RODGERS, Washington KATHY CASTOR, Florida
GREGG HARPER, Mississippi JOHN P. SARBANES, Maryland
LEONARD LANCE, New Jersey JERRY McNERNEY, California
BRETT GUTHRIE, Kentucky PETER WELCH, Vermont
PETE OLSON, Texas BEN RAY LUJAN, New Mexico
DAVID B. McKINLEY, West Virginia PAUL TONKO, New York
MIKE POMPEO, Kansas JOHN A. YARMUTH, Kentucky
ADAM KINZINGER, Illinois YVETTE D. CLARKE, New York
H. MORGAN GRIFFITH, Virginia DAVID LOEBSACK, Iowa
GUS M. BILIRAKIS, Florida KURT SCHRADER, Oregon
BILL JOHNSON, Ohio JOSEPH P. KENNEDY, III,
BILLY LONG, Missouri Massachusetts
RENEE L. ELLMERS, North Carolina TONY CARDENAS, California7
LARRY BUCSHON, Indiana
BILL FLORES, Texas
SUSAN W. BROOKS, Indiana
MARKWAYNE MULLIN, Oklahoma
RICHARD HUDSON, North Carolina
CHRIS COLLINS, New York
KEVIN CRAMER, North Dakota
Subcommittee on Health
JOSEPH R. PITTS, Pennsylvania
Chairman
BRETT GUTHRIE, Kentucky GENE GREEN, Texas
Vice Chairman Ranking Member
ED WHITFIELD, Kentucky ELIOT L. ENGEL, New York
JOHN SHIMKUS, Illinois LOIS CAPPS, California
TIM MURPHY, Pennsylvania JANICE D. SCHAKOWSKY, Illinois
MICHAEL C. BURGESS, Texas G.K. BUTTERFIELD, North Carolina
MARSHA BLACKBURN, Tennessee KATHY CASTOR, Florida
CATHY McMORRIS RODGERS, Washington JOHN P. SARBANES, Maryland
LEONARD LANCE, New Jersey DORIS O. MATSUI, California
H. MORGAN GRIFFITH, Virginia BEN RAY LUJAN, New Mexico
GUS M. BILIRAKIS, Florida KURT SCHRADER, Oregon
BILLY LONG, Missouri JOSEPH P. KENNEDY, III,
RENEE L. ELLMERS, North Carolina Massachusetts
LARRY BUCSHON, Indiana TONY CARDENAS, California
SUSAN W. BROOKS, Indiana FRANK PALLONE, Jr., New Jersey (ex
CHRIS COLLINS, New York officio)
JOE BARTON, Texas
FRED UPTON, Michigan (ex officio)
(ii)
C O N T E N T S
----------
Page
Hon. Joseph R. Pitts, a Representative in Congress from the
Commonwealth of Pennsylvania, opening statement................ 1
Prepared statement........................................... 2
Hon. Gene Green, a Representative in Congress from the State of
Texas, opening statement....................................... 4
Prepared statement........................................... 5
Hon. Fred Upton, a Representative in Congress from the State of
Michigan, opening statement.................................... 7
Prepared statement........................................... 8
Hon. Frank Pallone, Jr., a Representative in Congress from the
State of New Jersey, opening statement......................... 9
Prepared statement........................................... 10
Witnesses
Kathy Hudson, Deputy Director for Science, Outreach, and Policy,
National Institutes of Health.................................. 12
Prepared statement........................................... 15
Answers to submitted questions............................... 117
Janet Woodcock, Director, Center for Drug Evaluation and
Research, Food and Drug Administration \1\
Prepared statement \2\
Answers to submitted questions \3\........................... 124
Jeff Shuren, Director, Center for Devices and Radiological
Health, Food and Drug Administration........................... 24
Prepared statement of Dr. Woodcock and Dr. Shuren............ 26
Answers to submitted questions \3\........................... 127
Submitted Material
Discussion Draft, H.R. ___, the 21st Century Cures Act \4\
Statement of the American Health Care Association, April 30,
2015, submitted by Mr. Pitts................................... 74
Statement of the Healthcare Leadership Council, April 30, 2015,
by Mary R. Grealy, President, submitted by Mr. Pitts........... 76
Statement of Health Level Seven International, April 30, 2015, by
Charles Jaffe, Chief Executive Officer, submitted by Mr. Pitts. 82
Statement of the National Association of Chain Drug Stores, April
30, 2015, submitted by Mr. Pitts............................... 84
Statement of the National Marrow Donor Program, submitted by Mr.
Pitts.......................................................... 87
Statement of the Premier Healthcare Alliance, April 30, 2015,
submitted by Mr. Pitts......................................... 92
Letter of March 12, 2015, from Marcia Nusgart, Alliance for HCPCS
II Coding Reform, to Mr. Upton and Mr. Pallone, submitted by
Mr. Pitts...................................................... 95
Statement of the Senior Care Pharmacy Coalition, April 30, 2015,
submitted by Mr. Pitts......................................... 101
Statement of the Cord Blood Association, April 30, 2015, by
Joanne Kurtzberg, President, submitted by Mr. Pitts............ 105
----------
\1\ Dr. Woodcock did not offer an oral statement.
\2\ Dr. Woodcock submitted a joint statement with Dr. Shuren.
\3\ Dr. Woodcock and Dr. Shuren did not answer submitted questions for
the record by the time of printing.
\4\ The information has been retained in committee files and also is
available at http://docs.house.gov/meetings/IF/IF14/20150430/103400/
BILLS-114pih-DiscussionDraft.pdf.
Statement of the Telehealth Working Group, submitted by Mr. Pitts 108
Letter of April 29, 2015, from Ted Thompson, Chief Executive
Officer, Parkinson's Action Network, to Mr. Upton and Ms.
DeGette, submitted by Mr. Lance................................ 109
Letter of March 30, 2015, from Adaptive Biotechnologies, et al.,
to Mr. Upton and Mr. Pallone, submitted by Mr. Butterfield..... 111
Letter of April 13, 2015, from Adult Polyglucosan Body Disease
Research Foundation, et al., to Mr. Upton and Mr. Pallone,
submitted by Mr. Butterfield................................... 113
LEGISLATIVE HEARING ON 21ST CENTURY CURES
----------
THURSDAY, APRIL 30, 2015
House of Representatives,
Subcommittee on Health,
Committee on Energy and Commerce,
Washington, DC.
The subcommittee met, pursuant to call, at 10:01 a.m., in
room 2123 of the Rayburn House Office Building, Hon. Joseph R.
Pitts (chairman of the subcommittee) presiding.
Members present: Representatives Pitts, Guthrie, Barton,
Shimkus, Murphy, Burgess, Blackburn, McMorris Rodgers, Lance,
Griffith, Bilirakis, Long, Ellmers, Bucshon, Brooks, Collins,
Upton (ex officio), Green, Engel, Capps, Schakowsky,
Butterfield, Castor, Sarbanes, Matsui, Lujan, Schrader,
Kennedy, Cardenas, and Pallone (ex officio).
Also present: Representative DeGette.
Staff present: Clay Alspach, Chief Counsel, Health; Gary
Andres, Staff Director; Sean Bonyun, Communications Director;
Leighton Brown, Press Assistant; Noelle Clemente, Press
Secretary; Paul Edattel, Professional Staff Member, Health;
Gene Fullano, Detailee, Telecom; Robert Horne, Professional
Staff Member, Health; Carly McWilliams, Professional Staff
Member, Health; Katie Novaria, Professional Staff Member,
Health; Tim Pataki, Professional Staff Member; Graham Pittman,
Legislative Clerk; Krista Rosenthall, Counsel to Chairman
Emeritus; Chris Sarley, Policy Coordinator, Environment and the
Economy; Adrianna Simonelli, Legislative Associate, Health;
Heidi Stirrup, Policy Coordinator, Health; John Stone, Counsel,
Health; Traci Vitek, Detailee, HHS; Ziky Ababiya, Democratic
Policy Analyst; Jeff Carroll, Democratic Staff Director; Eric
Flamm, Democratic FDA Detailee; Waverly Gordon, Democratic
Professional Staff Member; Tiffany Guarascio, Democratic Deputy
Staff Director and Chief Health Advisor; and Kimberlee
Trzeciak, Democratic Health Policy Advisor.
Mr. Pitts. The Health Subcommittee will come to order.
The Chair will recognize himself for an opening statement.
OPENING STATEMENT OF HON. JOSEPH R. PITTS, A REPRESENTATIVE IN
CONGRESS FROM THE COMMONWEALTH OF PENNSYLVANIA
One year ago today, April 30, 2014, the Energy and Commerce
Committee embarked on an ambitious, bipartisan goal to develop
legislation that would bring the medical innovation cycle of
discovery, development, and delivery into the 21st century and
speed better treatments and, hopefully, more cures to patients
who desperately need them. Since then, this subcommittee has
held over a dozen hearings and roundtables to educate members
on topics ranging from modernizing clinical trials, to
personalized medicine, to digital health care, to incorporating
patient perspective into the development and regulatory
decision-making process. We heard from Government, academia,
patients, providers, manufacturers, and stakeholders from
across the spectrum. The consensus was clear: We can and must
do more to help patients in need and to maintain our Nation's
role as the biomedical innovation capital of the world.
Informed by the continued outpouring of feedback and
constructive criticism from stakeholders across the spectrum,
we have worked tirelessly on a bipartisan basis to develop the
second discussion draft that was released yesterday. While it
remains a work in progress, it is the product of good-faith
negotiations and a significant step forward in this process.
While increasing accountability, this legislation would invest
in the basic research so critical to equipping our Nation's
best and brightest with the tools they need to discover the
underpinnings of disease; it would streamline the development
of new therapies and technologies, which has become
increasingly challenging and resource intensive; and it would
foster a dynamic, continuously learning health care delivery
system. Work continues on several complicated yet critical
issues, including the regulation of diagnostic tests and
telemedicine.
With respect to diagnostics, we remain absolutely committed
to developing a modernized regulatory framework for these
innovative and increasingly important tests and services.
Understanding this is a particularly unique and complex
endeavor. We look forward to working in a deliberative manner
over the coming weeks with Dr. Shuren and stakeholders to
advance legislation.
On telemedicine, I continue to work with my colleagues in
the Energy and Commerce Working Group on Telemedicine towards a
bipartisan proposal that will encourage the use of telemedicine
services to improve health care quality and outcomes, increase
patient access, and control costs.
I want to thank the administration and CBO for their input,
and look forward to our continued collaboration moving forward.
On that note, I would like to specifically thank our three
witnesses today for their assistance throughout this process
and their testimony today.
[The prepared statement of Mr. Pitts follows:]
Prepared statement of Hon. Joseph R. Pitts
One year ago today, April 30, 2014, the Energy and Commerce
Committee embarked on an ambitious, bipartisan goal: to develop
legislation that would bring the medical innovation cycle of
discovery, development, and delivery into the 21st century and
speed better treatments and, hopefully, more cures to patients
who desperately need them.
Since then, this subcommittee has held over a dozen
hearings and roundtables to educate Members on topics ranging
from modernizing clinical trials, to personalized medicine, to
digital health care, to incorporating patient perspective into
the development and regulatory decision-making process.
We heard from Government, academia, patients, providers,
manufacturers, and stakeholders from across the spectrum. The
consensus was clear. We can and must do more to help patients
in need and to maintain our Nation's role as the biomedical
innovation capital of the world.
Informed by the continued outpouring of feedback and
constructive criticism from stakeholders across the spectrum,
we have worked tirelessly on a bipartisan basis to develop the
second discussion draft that was released earlier this week.
While it remains a work in progress, it is the product of good-
faith negotiations and a significant step forward in this
process.
While increasing accountability, this legislation would
invest in the basic research so critical to equipping our
Nation's best and brightest with the tools they need to
discover the underpinnings of disease; it would streamline the
development of new therapies and technologies which has become
increasingly challenging and resource intensive; and it would
foster a dynamic, continuously learning health care delivery
system.
Work continues on several complicated, yet critical issues,
including the regulation of diagnostic tests and telemedicine.
With respect to diagnostics, we remain absolutely committed
to developing a modernized regulatory framework for these
innovative and increasingly important tests and services.
Understanding this is a particularly unique and complex
endeavor, we look forward to working in a deliberative manner
over the coming weeks with Dr. Shuren and stakeholders to
advance legislation.
On telemedicine, I continue to work with my colleagues in
the Energy and Commerce Working Group on Telemedicine toward a
bipartisan proposal that will encourage the use of telemedicine
services to improve health care quality and outcomes, increase
patient access, and control costs. I want to thank the
administration and CBO for their input and look forward to our
continued collaboration moving forward.
On that note, I would like to specifically thank our three
witnesses today for their assistance throughout this process
and their testimony today.
[The discussion draft has been retained in committee files
and also is available at http://docs.house.gov/meetings/IF/
IF14/20150430/103400/BILLS-114pih-DiscussionDraft.pdf.]
Mr. Pitts. And I yield 1 minute to Dr. Burgess at this
time.
Mr. Burgess. Thank you, Mr. Chairman. I do want to thank
you for holding the hearing today.
A lot of bold goals in the 21st Century Cures, but at the
end of the day, it is all about patients. Doctors, of course,
want to heal, and the good news is I really do feel like we are
entering into a golden age of medicine. I think that the
doctors who are in medical school today will have tools at
their disposal to alleviate human suffering that no generation
of doctors has ever known. And it is the work of this
subcommittee that is bringing that possible.
I do have a number of proposals in the newly released
draft, and I look forward to discussing those proposals with
our agencies today. All of these things can be helpful in
speeding the development of new therapies and getting the
needed information into the hands of health professionals.
I do want to highlight, since 2009 we have spent $28
billion to drive adoption of electronic health records, yet
patient health data continues to be fragmented and difficult to
access for health care providers and for patients themselves.
So I am glad to have the chairman's continue support in this
area.
I yield the balance of the time to the vice chairman of the
full committee, Mrs. Blackburn.
Mrs. Blackburn. Thank you. And I think we are also pleased
to see this legislation coming forward and to discuss it with
you.
One of the purposes is to spur innovation and to look for
cures, to help individuals with disease management, and to
focus on those outcomes.
Kind of shift the focus of where we are going a little bit.
I think of it as our moonshot. President Kennedy didn't say we
are going to go increase NASA's budget and go to the moon, he
said we are going to the moon. And that indeed he did. So this
is where we are aiming, to increase these cures and
opportunities.
And I thank you for your time, and I yield back.
Mr. Pitts. Chair thanks the gentlelady.
Now recognize the ranking member of the subcommittee, Mr.
Green, 5 minutes for an opening statement.
OPENING STATEMENT OF HON. GENE GREEN, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF TEXAS
Mr. Green. Thank you, Mr. Chairman. And thank all our
colleagues for being here today.
I want to particularly thank our witnesses and their
colleagues for their expertise and for the countless hours of
work they put in to help us in this effort. It has been 1 year
since the 21st Century Cures Initiative was launched by our
colleagues, Chairman Upton and Congresswoman DeGette.
Yesterday's release of the discussion draft marked a continued
progress toward boosting research and delivering hope to
patients. FDA-approved treatments are the global gold standard
for safety and effectiveness. It is what physicians, patients,
and families trust when making decisions about their health.
Recently, Congress has enacted additional tools, like
breakthrough designation for drugs, to facilitate development
of effective, innovative treatments.
The NIH, the world's leading research institution, is one
of the great success stories of the Federal Government. Our
investment in basic and translational research has led to
advances that have profoundly improved the health and quality
of life of millions of Americans.
The 21st Century Cures Initiative nobly asked for what more
can Congress do to further the public and private efforts to
address today's most difficult scientific challenges and
advance our health care system. Additional funding for NIH is
tantamount to this effort. It is so important that the
initiatives include increased funding for NIH, both through
reauthorization and $10 billion over 5 years in mandatory
funding.
On the regulatory side, the draft includes policies to
incorporate the patient perspective in development process,
facilitate the use of biomarkers, and break down barriers to
collaboration and data sharing. The draft also includes
provisions to modernize clinical trials.
I want to particularly highlight the ADAPT Act, which
Congressman Shimkus and I are working on to provide a
streamlined approval pathway for the next generation of
antibiotics. FDA, and Dr. Woodcock, in particular, has been an
incredible partner on this issue. I want to thank the agency
for their continued commitment to the global crisis of
antibiotic resistance. We are working hard to incorporate
feedback and will have a new draft of the ADAPT to share in a
few days.
The draft also includes a new version of the Software Act,
which I have been working on with Congresswoman Blackburn for a
couple of Congresses. This provision will provide clarity for
developers of software products used in health management and
care. Dr. Shuren and his colleagues at the FDA have been
instrumental to this effort, and I look forward to continuing
to work with you to foster innovation, provide regulatory
certainty, and promote patient safety.
The draft recognizes the importance of improving the
interoperability health of IT systems. Interoperability is
fundamental in realizing the goals of the 21st Century Cures
Initiative, and an interoperable healthcare system can advance
and facilitate research and dramatically improve patient care
and safety.
I thank my colleagues for their commitment. The Cures draft
is a work in progress. There is a lot of work left to do, but
we will continue to move forward and iron out policies that
advance our healthcare system, and live up to the goals of the
21st Century Cures Initiative.
And again, I want to thank our witnesses. And I would like
to yield the remainder of my time to Congresswoman DeGette.
[The prepared statement of Mr. Green follows:]
Prepared statement of Hon. Gene Green
Good morning and thank you all for being here today.
I particularly want to thank our witnesses and their
colleagues for their expertise, and for the countless hours of
work they have put in to help us in this effort.
It has been 1 year since the 21st Century Cures Initiative
was launched by my colleagues, Chairman Upton and Congresswoman
DeGette.
Yesterday's release of a discussion draft marked continued
progress toward boosting research and delivering hope to
patients.
As we know, FDA-approved treatments are the global gold
standard for safety and effectiveness. It is what physicians,
patients and families trust when making decisions about their
health.
Recently, Congress has enacted additional tools--like the
breakthrough designation for drugs--to facility the development
effective, innovative treatments.
The NIH, the world's leading biomedical research
institution, is one of the great success stories of the Federal
Government.
Our investment in basic and translational research has led
to advances that have profoundly improved the health and
quality of life of millions of Americans.
The 21st Century Cures Initiative nobly asked what more can
Congress do to further public and private efforts to address
today's most difficult scientific challenges, and advance our
health care system.
Additional funding for NIH is tantamount to this effort.
It is so important that the Initiative includes increased
funding for NIH, both through reauthorization and $10 billion
over 5 years in mandatory funding.
On the regulatory side, the draft includes policies to
incorporate the patient perspective in the development process,
facilitate the use of biomarkers, and breakdown barriers to
collaboration and data sharing.
The draft also includes provisions to modernize clinical
trials.
I want to particularly highlight the ADAPT Act, which
Congressman Shimkus and I are working on, to provide a
streamlined approval pathway for the next generation of
antibiotics.
FDA, and Dr. Woodcock in particular, has been an incredible
partner on this issue.
I thank the agency for their continued commitment to combat
the global crisis of antibiotic resistance.
We are working hard to incorporate recent feedback and will
have a new draft of ADAPT to share in the coming days.
The draft also includes a new version of the SOFTWARE Act,
which I have been working on with Congresswoman Blackburn.
This provision will provide clarity for developers of
software products used in health management and care.
Dr. Shuren and his colleagues at FDA have been instrumental
to this effort, and I look forward to continuing to work with
you all to foster innovation, provide regulatory certainty, and
promote patient safety.
The draft recognizes the importance of improving the
interoperability of health IT systems.
Interoperability is foundational to realizing the goals of
the 21st Century Cures Initiative.
An interoperable health care system can advance and
facilitate research, and dramatically improve patient care and
safety.
I thank my colleagues for their commitment to continuing
this effort.
The Cures draft is a work in progress.
There is a lot of work left to do, but we will continue to
move forward and iron out policies to advance our health care
system and live up to the goals of the 21st Century Cures
Initiative.
Thank you again to our witnesses, and I yield the remainder
of my time to Congresswoman DeGette.
Ms. DeGette. Thank you so much.
In the year since Chairman Upton and I announced this 21st
Century Cures effort, I have constantly been impressed by the
engagement and consensus of people across the healthcare
landscape. From the beginning, we sought suggestions from
everyone, and we have worked diligently to reflect those ideas
in the discussion draft we have before us. I also want to add
my heartfelt thanks to everybody, both in this room and across
the country, who have helped Chairman Upton and myself, and all
of the members of this committee, work to deliver treatments
and cures for patients.
The draft makes important improvements to our biomedical
research system and our process for assessing and improving new
therapies, drugs, and devices for patients. After years of
resource erosion and cuts, we deliver important new resources
to the National Institutes of Health. We placed the patient
perspective at the heart of the FDA's drug approval process. We
will develop disease registries to pull information, and help
researchers drill into the unique and sometimes subtle needs of
patient populations. We will help new scientists begin their
careers in research so that our great minds tackle great
biomedical challenges. Any of these ideas would be worth doing
on their own, but, frankly, this committee's ambitions stretch
across the century, and so we want to do everything we can to
improve the process of discovering, developing, and delivering
new biomedical advances.
So in that spirit, as you can see, we have a great deal
more work to do. This discussion draft has brackets around many
sections of text, and we have much more work to do, but it is
certainly not through lack of trying on all of our parts over
the last year. One specific issue that deserves singling out is
the fact that we are asking FDA to make many changes to its
current operation. We need to make sure that the agency has the
resources to carry out these duties.
Mr. Chairman, I want to thank you, I want to thank Chairman
Upton, and I want to just reflect back to the time when we made
that kind of hokey video launching this effort, but we have
made tremendous progress. We have a lot more to do, and in that
spirit, I want to thank you, Chairman Upton, Chairman Pitts,
Mr. Pallone, Mr. Green, all of the staff. It has really been a
great effort, and I look forward to moving along this road so
that we can actualize this important, important piece of
legislation. Thank you.
Mr. Pitts. The Chair thanks the gentlelady.
And now recognizes the distinguished chairman of the full
committee, Mr. Upton, 5 minutes for an opening statement.
OPENING STATEMENT OF HON. FRED UPTON, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF MICHIGAN
Mr. Upton. Well, thank you, Mr. Chairman.
First, I want to talk a little bit about how we got here
today. These two little girls, my friends, Brooke and Brielle,
of Mattawan, Michigan, served as an inspiration for the 21st
Century Cures. They are battling SMA, and they are two of the
brightest stars that I know. Their motto is, we can and we
will.
At our very first 21st Century Cures roundtable last
spring, I commented that I think that we can all agree that we
can always be doing more to help biomedical innovation. We have
come a long way, yes we have, but those words still hold true.
In fact, since our launch a year ago today, we have heard from
our colleagues in the Senate, and yes, they are interested in
these same goals, and President Obama even included precision
medicine as part of his State of the Union Address in January.
There is clearly an opportunity to make a real difference. And
all of us here have traveled the country to listen to as many
stakeholders as we could to get more knowledge to make this
bill as solid as we can.
At that first roundtable in this room last year, we asked,
``What steps can Congress take to accelerate the discovery-
development-delivery cycle in the U.S. to foster innovation,
bring new treatments and cures to patients, and keep more jobs
in the U.S.?'' The bipartisan discussion draft that was
released yesterday makes meaningful investments and still will
be fully paid for, includes a number of policies that seek to
answer those same questions. We started this journey because
all of us know patients and families who are desperate for
hope. We have also seen and read about the incredible advances
made in science as well as in technology. But it has become
increasingly clear in recent years that our regulatory policies
have not kept pace with innovation, and there is much more that
we can be doing to provide that hope to folks, and that is what
this bill does.
This discussion draft, the product of eight hearings, more
than two dozen roundtables, and hundreds of discussions, a
number of white papers, incorporates the patient perspective
into the regulatory process. It will increase funding for the
NIH. It modernizes clinical trials, including allowing for more
flexible trial designs so that we can customize trials based on
the unique characteristics of patients most likely to benefit.
Twenty-first Century Cures will unlock the wealth of health
data available to patients, researchers, and innovators, and
can communicate and keep the cycle of cures constantly moving
and improving.
We still have important issues to resolve over the next
couple of weeks. One placeholder included in the draft is on
rescuing and repurposing drugs for serious and life-threatening
diseases and disorders. As we move through the process to
markup, we will continue to work on a policy to provide
incentives to develop drugs that, while they may have failed in
trials for one indication, show promise to treat patients
facing other serious or life-threatening diseases. We need to
ensure the scientific promise to help patients play a more
important role than patients in drug development. This policy
also will include incentives for doing research on drugs that
are FDA-approved but can be repurposed to help patients with
different types of illnesses.
On the important issue of diagnostics, we remain committed
to developing a modernized regulatory framework for these
products and services. We look forward to working with Dr.
Shuren and stakeholders with hopes of having a legislative
hearing in July. This hearing and the 1-year anniversary of
21st Century Cures are important milestones, but much more work
remains to get the bill to the President. Along with the wealth
of ideas and support shared over the last year, we have heard
repeatedly that patients can no longer wait. We must get this
done this year.
I want to thank all of my colleagues on both sides of the
aisle who have participated in this effort, thank the patients
who have shared their stories, administration officials, staff,
and other experts. I particularly want to thank Ms. DeGette,
Mr. Pitts, Mr. Pallone, and Mr. Green for their countless hours
and, indeed, partnership. Ms. DeGette joined me in Kalamazoo
just this last week where we gained valuable feedback from a
number of great groups--innovators, medical students, community
leaders--and I look forward to going to her district in the
next month or so.
Yes, we still have work to do, but it is important to
recognize the incredible progress of this past year and remain
focused on our common goal of helping patients. We have a
chance to do something big, and this is our time. It is Brooke
and Brielle's time as well.
Yield back.
[The prepared statement of Mr. Upton follows:]
Prepared statement of Hon. Fred Upton
First, I'd like to talk about how we got here today. These
two little girls [holds up photo with the girls], my friends
Brooke and Brielle Kennedy, served as an inspiration for 21st
Century Cures. They are battling SMA, and are two of the
brightest stars I know.
At our inaugural 21st Century Cures roundtable last spring
I commented, ``I think we all agree that we can always be doing
more to help biomedical innovation.'' We've come a long way,
but those words still hold true. In fact, since our launch 1
year ago today, we have heard from our colleagues in the Senate
that they are interested in these same goals, and President
Obama even included Precision Medicine as part of his State of
the Union Address in January. There is clearly an opportunity
to make a real difference.
At that first roundtable we asked, ``What steps can
Congress take to accelerate the discovery-development-delivery
cycle in the U.S. to foster innovation, bring new treatments
and cures to patients, and keep more jobs in the U.S.?'' The
bipartisan discussion draft released yesterday, which makes
meaningful investments and still will be fully paid for,
includes a number of policies that seek to answer those
questions.
We started this journey because al I of us know patients
and families who are desperate for hope. We've also seen and
read about the incredible advances made i n science and
technology. But it has become increasingly clear in recent
years that our regulatory policies have not kept pace with
innovation, and there is much more we can be doing to provide
that hope to folks. That's what this bill does.
This discussion draft, the product of eight hearings, more
than two-dozen roundtables, and several white papers,
incorporates the patient perspective into the regulatory
process. It will increase funding for NIH. It modernizes
clinical trials, including allowing for more flexible trial
designs so we can customize trials based on the unique
characteristics of patients most likely to benefit. 21st
Century Cures will unlock the wealth of health data available
so patients, researchers, and innovators can communicate and
keep the cycle of cures constantly moving and improving.
We still have important issues to resolve over the next few
weeks. One placeholder included in the draft is on rescuing and
repurposing drugs for serious and life-threatening diseases and
disorders. As we move through the process to markup, we will
continue to work on a policy to provide incentives to develop
drugs that, while they may have failed in trials for one
indication, show promise to treat patients facing other serious
or life-threatening diseases. We need to ensure the scientific
promise to help patients plays a more important role than
patents in drug development. This policy also will include
incentives for doing research on drugs that are FDA-approved
but can be repurposed to help patients with different types of
illnesses.
On the important issue of diagnostics, we remain committed
to developing a modernized regulatory framework for these
products and services. We look forward to working with Dr.
Shuren and stakeholders with hopes of having a legislative
hearing by July.
This hearing and the 1-year anniversary of 21st Century
Cures are important milestones, but much more work remains to
get this bill to the president. Along with the wealth of ideas
and support shared over the last year, we heard repeatedly that
patients can no longer wait. We must get this done this year.
I want to thank all of my colleagues who have participated
in this effort, thank the patients who have shared their
stories with us, as well as the administration officials,
staff, and other experts. Yes, we still have work to do, but it
is important to recognize the incredible progress of the past
year and remain focused on our common goal of helping patients.
We have a chance to do something big, and this is our time. And
it is Brooke and Brielle's time.
Mr. Pitts. Chair thanks the gentleman.
Now yields to the ranking member of the full committee, Mr.
Pallone, 5 minutes for an opening statement.
OPENING STATEMENT OF HON. FRANK PALLONE, JR., A REPRESENTATIVE
IN CONGRESS FROM THE STATE OF NEW JERSEY
Mr. Pallone. Thank you, Mr. Chairman.
Let me thank you, Chairman Pitts, and also Chairman Upton,
Ms. DeGette, and Ranking Member Green. Today's hearing will
examine the draft released yesterday that is the result of
months of discussion. It has changed significantly from the
draft the chairman released earlier this year. While it is by
no means perfect, it does reflect hard work by staff, true
collaboration between Republicans and Democrats, stakeholders,
and the administration, and I am hopeful we can bring this
legislation to a successful conclusion.
There are a large number of policies in the draft and not a
lot of time to cover them all, but let me just highlight a few.
Most notable in the new draft, and the one that I am most proud
to see, is $10 billion in mandatory funding for NIH over the
next 5 years. It also includes a $1.5 billion increase in NIH
discretionary authorization over the next 3 years, and this is
a real win for researchers, patients, and industry alike. I
believe Federal funding is the foundation of our biomedical
ecosystem, and is one of the most promising ways to spur
economic prosperity and treatments and cures for the 21st
century.
We also need to ensure that policies in this draft do no
harm. I have said all along that broadly extending drug
exclusivity will not solve the problems 21st Century Cures sets
out to address, so I am glad to see that this new draft
includes placeholder language for a much more tailored approach
at solving a targeted problem. We are going to continue
discussions on how we can incentivize development of a narrow
class of drugs that have been abandoned because of inadequate
remaining patent life. Dr. Collins has spoken about the need to
provide limited additional exclusivity for drugs that have been
found to be safe in clinical trials. Even though they failed
the trials for effectiveness, it may be possible to repurpose
them for a different indication, or for a different population
for which they may be effective. If such drugs fill an unmet
medical need for treating a serious or life-threatening
disease, it may be appropriate to provide companies with
limited additional exclusivity for companies to spend the
resources needed to determine if they work. And I appreciate
the chairman's commitment to continue to discuss this policy
and ensure that it is targeted to where it is needed. I do not
want to undermine the balance between protection and
competition that Hatch-Waxman has been so successful in
achieving.
Mr. Chairman, with the hard work of staff, I believe we
have come a long way; however, there are other complicated
policies like interoperability and telehealth which still need
thorough vetting and further consideration. And I have said
since I became the ranking member, I am serious about finding
common ground on important issues. True bipartisanship is
critical to achieving successful and broadly supported
policies, and I am confident that this much-improved
collaborative process can continue.
[The prepared statement of Mr. Pallone follows:]
Prepared statement of Hon. Frank Pallone, Jr.
Thank you Chairman Pitts. And let me thank Chairman Upton,
Ms. DeGette, and Ranking Member Green. Today's hearing will
examine a draft released yesterday that is the result of months
of discussions. It has changed significantly from the draft the
chairman released earlier this year. While it is by no means
perfect, it does reflect hard work by staff, true collaboration
between Republicans and Democrats, stakeholders, and the
administration, and I am hopeful we can bring this legislation
to a successful conclusion.
Let me also thank HHS for the expert advice and help along
the way. I know how many resources have been spent on this
effort as well, and this draft is a better product because of
their guidance.
Now I would have liked Members and their staff, and our
witnesses, to have had more time with the draft before a
legislative hearing. The ambitious timeline has been a
challenge. Iwant to be clear that I am committed to ensuring
that every Member is comfortable as this process moves forward
so that a final product gains broad support.
There are a large number of policies in this draft--and not
a lot of time to cover all of them. But let me highlight just a
few things.
Most notable in the new draft, and the one that I am most
proud to see, is $10 billion in mandatory funding for NIH over
the next 5 years. It also includes a $1.5 billion increase in
NIH discretionary authorization over the next 3 years. This is
a real win for researchers, patients and industry alike. I
believe Federal funding is the foundation of our biomedical
ecosystem and is one of the most promising ways to spur
economic prosperity and treatments and cures for the 21st
Century.
While this a great development, I hope that we can also
ensure that FDA has the needed resources to implement the many
additional policies put forth in this draft. We cannot divert
already scarce resources nor impede the progress FDA has
already made to advance the development and review of medical
products.
We also need to ensure that policies in this draft do no
harm. I have said all along that broadly extending drug
exclusivity will not solve the problems 21st Century Cures sets
out to address. So I am glad to see that this new draft
includes placeholder language for a much more tailored approach
at solving a targeted problem. We are going to continue
discussions on how we can incentivize development of a narrow
class of drugs that have been abandoned because of inadequate
remaining patent life. Dr. Collins has spoken about the need to
provide limited additional exclusivity for drugs that have been
found to be safe in clinical trials. Even though they failed
the trials for effectiveness, it may be possible to repurpose
them for a different indication or for a different population
for which they may be effective. If such drugs fill an unmet
medical need for treating a serious or life threatening
disease, it may be appropriate to provide companies with
limited additional exclusivity for companies to spend the
resources needed to determine if they work. I appreciate the
chairman's commitment to me to continue to discuss this policy
and ensure that it is targeted only to where it is needed.
Mr. Chairman, with the hard work of staff, I believe we
have come a long way. However, there are other complicated
policies, like interoperability and telehealth, which still
need thorough vetting and further consideration.
As I've said since I became Ranking Member, I am serious
about finding common ground on important issues. True
bipartisanship is critical to achieving successful and broadly
supported policies. I am confident that this much improved
collaborative process can continue. There is still much more
work to be done, but today's hearing is an important step and I
look forward to our continued partnership on this initiative.
Mr. Pallone. I would like to yield now a minute initially
to Representative Schakowsky, and then the remaining minute or
so to Representative Matsui.
So I will yield now to the gentlewoman from Illinois.
Ms. Schakowsky. Thank you, Congressman Pallone.
I want to highlight how vital it is that we provide
additional funding to NIH, both mandatory and discretionary.
For years, NIH has seen stagnant funding, a trend that simply
must be reversed, and I am so pleased to see this legislation
includes both $10 billion in mandatory spending as well as an
increase in their discretionary authorization over the next 3
years. I also am encouraged by removal of many of the patent
exclusivity provisions that were initially included in the
draft released by the majority in January. Added exclusivity is
not needed to bring new cures to patients.
Lastly, I believe that we must have a serious conversation
about the high cost of medications, and we must do more to
address this growing problem. If we are spending billions of
dollars to incentivize the development of new drugs, we need to
ensure that patients have affordable access to those therapies.
I am drafting legislation that would allow HHS to negotiate for
better prices on certain specialty drugs and biologics. I
strongly hope that giving HHS this authority would help to
ensure that our healthcare system can sustain the treatments
that we hope to advance this legislation.
I want to end by expressing my gratitude to all the leaders
of this effort for giving the rest of us the privilege of
giving real hope to millions of Americans who are longing for
cures.
And I yield back.
Mr. Pitts. Gentlelady yields to Ms. Matsui.
Ms. Matsui. Thank you. Thank you for yielding.
I believe in this 21st Century initiative to take advantage
of innovation and to get breakthroughs of cures and technology
to patients faster. I believe many of us have friends or family
members who were too late to it, and so we should use their
courage to spur us on forward.
This legislation really does serve to address the
roadblocks, and we must continue to get it right. I would like
to thank Chairman Upton, Ranking Member Pallone, and
Subcommittee Chairman Pitts for working with a bipartisan
Working Group on Telehealth. Technology has huge potential to
both improve patient care and reduce healthcare costs. Our
ultimate goal as a working group has been to advance quality
telehealth services within the Medicare Program while
recognizing that telehealth can save the system money. We must
continue to work with that.
And critical to the efforts of both Telehealth and Cures is
the interoperability of health IT systems, which facilitate
population health research and improve patient care. We need to
continue to work on this as well.
Thank you, and I yield back the balance of my time.
Mr. Pitts. The Chair thanks the gentlelady.
That concludes the opening statements. As usual, all the
opening statements of members, if you provide them in writing,
will be made a part of the record.
I have a UC request. I would like to submit the following
documents for the record: statements from the American
Healthcare Association, Healthcare Leadership Council, Health
Level Seven International, National Association of Chain
Drugstores, National Marrow Donor Program, the Premiere
Healthcare Alliance, the Alliance for Healthcare Common
Procedure Coding System Reform, Senior Care Pharmacy Coalition,
and the Cord Blood Association, and a statement from the
bipartisan Telehealth Working Group.
And without objection, so ordered.
[The information appears at the conclusion of the hearing.]
Mr. Pitts. We have on our panel today three witnesses, and
I will introduce them in the order of their presentation.
First, Dr. Kathy Hudson, Deputy Director for Science,
Outreach, and Policy at the National Institutes of Health.
Secondly, Dr. Janet Woodcock, Director of the Center for Drug
Evaluation and Research at the Food and Drug Administration.
And finally, Dr. Jeff Shuren, Director of the Center for
Devices and Radiological Health at the Food and Drug
Administration.
Thank you very much for coming today. Your written
statements will be made a part of the record. You will each be
given 5 minutes to summarize your testimony.
And so, Dr. Hudson, at this point, you are recognized for 5
minutes for your summary.
STATEMENTS OF KATHY HUDSON, DEPUTY DIRECTOR FOR SCIENCE,
OUTREACH, AND POLICY, NATIONAL INSTITUTES OF HEALTH; JANET
WOODCOCK, DIRECTOR, CENTER FOR DRUG EVALUATION AND RESEARCH,
FOOD AND DRUG ADMINISTRATION; AND JEFF SHUREN, DIRECTOR, CENTER
FOR DEVICES AND RADIOLOGICAL HEALTH, FOOD AND DRUG
ADMINISTRATION
STATEMENT OF KATHY HUDSON
Dr. Hudson. Good morning, Chairman Pitts, Ranking Member
Green, members of the subcommittee, Chairman Upton, and
Congresswoman DeGette. I want to thank the members of the
subcommittee, and especially your amazing staff for all the
work that you have done over the past year to move forward this
21st Century Cures Initiative.
I am pleased to testify this morning alongside of my
colleagues from the Food and Drug Administration. We work side
by side every day to advance the issues that you are attempting
to address in this important bill.
How can we accelerate the pace of medical breakthroughs in
the United States? How can we get cures to patients faster? Too
often, patients and those who love them run out of options. We
don't know what the disease is, we don't have effective
interventions for them, we simply don't have the answers. Our
shared goal is to usher in an era in which we have the answers,
and we have effective ways to diagnose, treat, and prevent
disease and disability.
Investments in the National Institutes of Health have
resulted in dramatic increases in lifespan, and marked
reductions in devastating diseases and disabilities. Take HIV/
AIDS. When I was a graduate student in California in the early
'90s, I was attending far too many funerals of friends, fellow
classmates and family members who had succumbed to the HIV
virus. Today, it is unlikely that young people will attend the
funeral of someone who has succumbed to AIDS because of the
remarkable advances in treatments and preventions that have
been made possible by NIH-supported research. While we have
much to do, this is a remarkable success story, but we need
more.
Today, I want to talk about a few of the areas in which
your draft bill can facilitate scientific innovation and
collaboration, and increase efficiency through reducing
administrative burdens on scientists.
First, you have proposed to increase the funding available
to support NIH research. Thank you. Thank you. Thank you. Thank
you. The research community is ecstatic to see this new
provision in the bill, and we are deeply appreciative. After a
number of years of reduced ability to support research, and
diminishing ability to pay for great ideas that are brought
before us, this is a dramatic and important moment, so thank
you very much. We hope that this increase in support for NIH
will be undertaken as a part of broader efforts to support
important programs across Government.
Second, the draft bill includes a number of proposals to
enhance accountability, and we support those. That is why Dr.
Collins and his leadership team are undertaking a number of new
ways to enhance our stewardship of the resources that you and
the American people provide. These include investments in
making sure we are investing in the highest research
priorities, fostering creative collaborations, and making sure
that we are sustaining the biomedical workforce.
Third, I think that we can all agree that scientists should
be spending their time doing science and bringing cures to
patients. Unfortunately, researchers are spending too much time
filling out forms that benefit no one. Your effort to
streamline the ability of NIH intramural scientists to attend
scientific meetings is one important step. NIH is taking
additional steps to reduce burden on our grantees, and we
appreciate the inclusion in the draft bill of an exclusion for
scientific research from the paperwork-inducing Paperwork
Reduction Act.
Fourth, on data sharing, and you mentioned this,
dissemination of research findings is fundamental, and we are
using all sorts of new technologies and opportunities to make
sure that the results of our investments in research are made
available to other researchers, to patients, and to providers.
We appreciate very much the inclusion in this draft bill of a
specific provision that allows the NIH director to require data
sharing for NIH-funded research.
And fifth and finally, while we need to ensure the rapid,
unencumbered sharing of data from biomedical research, we also
need to protect the privacy of those who volunteer to
participate in biomedical research. Although we have taken a
number of steps to protect research participants, there are
ways in which Congress can be of assistance. Specifically, a
statutory change establishing that individual level genomic
data are confidential would provide research participants with
more robust privacy protections, and enhance public trust and
confidence in medical research. This will be particularly
important as major new research efforts, such as the Precision
Medicine Initiative, move forward.
This concludes my testimony, Mr. Chairman. NIH looks
forward to working with you and your staff as you continue to
remove the brackets from the draft bill. And I welcome your
questions. Thank you.
[The prepared statement of Dr. Hudson follows:]
[GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
Mr. Pitts. Chair thanks the gentlelady.
Now recognizes Dr. Woodcock, 5 minutes for an opening
statement.
Dr. Woodcock. Thank you. Dr. Shuren will be presenting our
oral statement.
Mr. Pitts. Dr. Shuren?
Dr. Shuren. It is in the spirit of greater efficiency.
STATEMENT OF JEFF SHUREN
Dr. Shuren. So, Mr. Chairman and members of the committee,
on behalf of Janet and myself, thank you for inviting us to
testify regarding the committee's 21st Century Cures proposal.
We share your desire to accelerate the development of safe and
effective medical products. We would like to thank Chairman
Upton, Representatives Pallone and DeGette, other members of
the committee, for reaching out to FDA over the past many
months to ask for our insights on opportunities to reduce the
costs and time involved in studying new medical products, while
continuing to protect patients who use those products.
We also want to recognize Congress' critical role in
establishing user-fee programs that have led to faster product
reviews, and greater collaboration between the agency,
companies, and our stakeholders. With your partnership, FDA has
been successful in accelerating drug and medical device review
times, even as FDA's regulatory review process has remained the
gold standard worldwide.
While working together with the committee on the Cures
legislation, we are continually cognizant of the agreements
made between the agency and the industry, and enacted by
Congress under the Prescription Drug User Fee Act, the Medical
Device User Fee Act, and appreciate the importance of assuring
that new provisions not impede or conflict with the important
ongoing work pursuant to those user fee agreements.
We appreciate the chance to provide input throughout the
drafting of the legislation. As we have previously indicated to
the committee, we believe there are opportunities to accelerate
medical product development. For example, by supporting
patient-centered medical product development, encouraging
development and qualification of biomarkers, utilizing real
world evidence in the review process, reducing barriers to the
use of central IRBs for device trials, and strengthening FDA's
ability to hire and retain highly qualified experts. We are
encouraged that these things have been addressed in this
legislation, and look forward to providing additional feedback
on these proposals as we evaluate the details of the draft.
There are also several areas that we believe require
further improvement to ensure that they do not compromise the
safety and effectiveness of American medical products. For
example, we appreciate that the committee has been working with
FDA and stakeholders to encourage the development and
qualification of drug development tools. We look forward to
continuing to work with you to ensure that this language does
not divert from important resources, and take those away from
drug review activities. We share the committee's goal on
advancing the development of new antibiotics through a new
approval pathway focused on drugs intended for limited
populations of patients with few or no available treatment
alternatives, and streamlining the process for updating
antibiotic breakpoints.
We thank Representatives Shimkus and Green for their
leadership on this important topic, and look forward to
continuing to work with the committee on the remaining issues,
including the inclusion of a branding element within the
labeling of such products that will alert healthcare
communities to these products that they are special, and should
be treated as such, as well as provisions related to meetings
and agreements. We recognize the interest of manufacturers in
communicating with health insurers about healthcare economic
information, and are evaluating this new language. We will
provide feedback on this topic as soon as possible.
We thank Representatives Blackburn and Green, as well as
the committee staff, for the opportunity to work with the
committee and stakeholders to ensure that medical software is
regulated in a manner that ensures appropriate oversight of
higher risk software to protect patient safety, while limiting
requirements on other products. In many cases, software is
essential to the safe functioning of medical devices used in
the diagnosis and treatment of patients. Removing particular
types of software from the statutory definition of medical
device requires careful consideration to avoid unintended
consequences.
We look forward to continuing to work together to address
remaining issues, including avoiding the imposition of
unnecessary burdens on the agency's effort to streamline its
approach to device software that would delay rather than
accelerate these actions. We look forward to providing you with
additional feedback as we review this new draft, and to
ensuring that it meets our shared goal of accelerating
innovation, without jeopardizing the safety and effectiveness
of medical products. The American public benefits from the
efficient and expeditious development and review of innovative
medical products, and the safety and effectiveness of those
products depends on the high quality of the input and review of
FDA.
Thank you, Mr. Chairman, and we look forward to your
questions.
[The prepared statement of Dr. Woodcock and Dr. Shuren
follows:]
[GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
Mr. Pitts. Thank you. All right, we will begin questioning.
And I will recognize myself 5 minutes for that purpose.
We will start on patient center drug development for Drs.
Woodcock and Shuren. Patients are the cornerstone of the 21st
Century Cures Initiative, incorporating patient perspective
into the regulatory process, and the benefit-risk discussion is
a pivotal change to our regulatory structure. The patient focus
drug development section builds on the work FDA started with
FDASIA in 2012, and I know that both, Dr. Woodcock, Dr. Shuren,
both your centers have made progress incorporating the patient
perspective in different ways for drugs and devices. What have
you done since the enactment of FDASIA in this regard?
Dr. Woodcock, we will start with you.
Dr. Woodcock. Certainly. We have held--we are supposed to
hold 20 meetings. They are The Voice of The Patient. They are
for specific diseases, and we hear from patients, and it is a
facilitated discussion of the burden of disease, what is their
experience of the disease, what are the various burdens,
because really, there is a whole spectrum of burden for
patients. One patient's experience doesn't represent the
experience of everyone who has a disease. So we hear from a
spectrum of patients, and then we write a report called ``The
Voice of the Patient.'' And then in some cases, we have issued
guidance afterward on drug development, talking about, for
example, with chronic fatigue syndrome, about how you would
develop a drug for that condition.
So what we have really learned is that patients are experts
in their disease, people with chronic diseases are experts, and
we really need to hear from them, both the burden of their
disease, and also how well the treatments that exist, if any,
are doing, and what needs to be improved. And what we have
learned though is we need a much more structured and organized
way to incorporate this input into drug development. And we
think that what is laid out in the discussion draft will really
help with that.
Mr. Pitts. Thank you. Dr. Shuren?
Dr. Shuren. Well, in 2012, we put out a framework on the
factors we consider for benefits and risks, and weighing
benefits and risks, and approving high-risk and innovative
lower-risk devices. One of those factors that we would take
into account is patient's perspective on benefit and tolerance
for risk. We have been working on draft guidance about how
patient perspectives would be included in premarket review, and
in support of device approvals. We have been working as a part
of the Medical Device Innovation Consortium, a public-private
partnership with industry, patient advocacy groups, nonprofits,
and Government, and that includes NIH, on a compendium of tools
for assessing patient preferences, to then inform product
approvals. They are also working on a framework for sponsors
for what to take into consideration on patient preferences.
We have also worked with RTI to develop a tool for
assessing patient preferences for patients with obesity and the
treatments that would best benefit them. The results of that
survey were used to inform our decision to approve the very
first device treatment for obesity since 2007. So we are
actually already incorporating such information into our
decisions. And, of course, we attend the drug meetings as well.
Mr. Pitts. Thank you. Now, next question for all of you;
one on interoperability, and one on pediatric clinical trials.
This legislation is based on the innovation cycle, the way
medical products are developed through the regulatory system
from discovery, development, to delivery. Some of the
fundamental problems we have identified as the challenges of
working together, but the committee has identified how working
together is critical for 21st century innovation, and a
paramount piece of this is interoperability. Imagine a world
where your cell phone would not work with a landline, or if my
cell phone did not connect with other networks. Ridiculous.
Well, that is the world of electronic health records, and that
is the world of health data patients with devices such as
diabetes patients, numerous devices collecting data that never
get compiled or looked at by a physician.
We are not using this information to innovate and empower
patients, and interoperability is the barrier, how
interoperability and data collection could be used at your
agency to accelerate the science and gain understanding of
diseases. The first question, and then comment on how will a
global pediatric clinical trial network help accelerate
pediatric research in medical products? Dr. Hudson?
Dr. Hudson. So let me begin in addressing the question of
interoperability. Our colleagues in the Office of the National
Coordinator for Health IT are working very hard at fixing the
problems of interoperability, and making sure that all of our
healthcare providers, and we all have many, are actually able
to communicate with each other, and equally importantly, able
to share that information in a ready way with us.
I moved my mother from Texas to Minnesota in November, and
I ended up carrying two boxes of paper medical records with me.
I hope that that doesn't happen in the future, and I think we
are moving quickly to solve that problem.
Certainly, interoperability for patient care is
extraordinarily important, but having interoperable medical
records is also vital for research. And so making electronic
medical records, electronic health records, available and
accessible for research will be important, especially as we
move forward with the Precision Medicine Initiative.
Do you want to----
Mr. Pitts. So if you would supply in writing to us the
response to those questions.
I will now recognize the ranking member, Mr. Green, 5
minutes for questions.
Mr. Green. Thank you, Mr. Chairman. Among the provisions,
the draft includes key improvements to FDA's premarket program
for medical devices. I believe most significant of these
provisions is the establishment of an expedited pathway for
breakthrough and innovative technologies. This has the
potential to increase the efficiency and predictability of the
agency's review process, and improve patient access.
Dr. Shuren, can you comment on the provision creating a
breakthrough pathway for medical devices? Is this complementary
to actions that the FDA has already underway?
Dr. Shuren. Yes, it is. So we think this is a very
important provision. It essentially codifies a program that we
just launched the other week that we call the Expedited Access
Pathway Program. It is something we have been piloting since
2011. This is an attempt to sort of speed access to very
important medical devices. It includes greater collaboration
and interaction with the sponsor who is developing the product,
but also the opportunity, where appropriate, to shift some data
we would otherwise collect premarket, to the post-market
setting and gather it then.
Mr. Green. OK. Basic research and translational research
are critical to scientific advancement. Dr. Hudson, we heard
that certain modifications to give increased flexibility would
help NIH to leverage funding and advance promising research.
The discussion draft includes a provision that removes
restrictions on the National Center for Advancing Translational
Sciences', or NCATS', ability to utilize its authority and
foster development. Can you explain how increased flexibility
on the use and funding of NCATS and Other Transactional
Authority will help advance scientific research?
Dr. Hudson. Thank you very much for the question. So NCATS,
the National Center for Advancing Translational Sciences, is
our newest center at the National Institutes of Health, and it
ironically has this limitation on being able to pursue beyond
Phase 2(a) clinical trials.
The way that NCATS works is largely in collaboration with
other institutes at the NIH to pursue new innovative
approaches, to design of clinical trials and the like, and so
it having this restriction on being able to move forward in
later-stage clinical trials has really limited its ability to
do important research. So we appreciate very much the lifting
of that restriction in the draft discussion.
Mr. Green. OK. Thank you.
Dr. Woodcock, during our roundtables and hearings, we heard
a great deal about the promise of biomarkers. The science is
incredibly complex, and the scientific community has a wide
variety of views on the issue. The discussion draft includes
language on FDA's treatment of biomarkers, but outstanding
policy questions need to be answered. We must ensure that
legislation provides a clear and workable solution that
recognizes the underlying science. Can you share with us your
view of what additional authorities would be most helpful to
the FDA to facilitate and advance the use of biomarkers in the
approval process?
Dr. Woodcock. I am not sure that additional authorities are
needed. For those who are not experts in this, biomarkers are
measurements that are made on people, and these measurements
help us decide whether a person has a disease, whether giving
treatment might help them or not, and also to monitor treatment
once they are on therapy. And we have thousands of biomarkers
that are now used in clinical trials, but clearly, the new
biomarkers, the genetic biomarkers, proteomics, all these new
technologies, are going to be very important in helping us do
precision medicine and develop new cures. And their progress is
slow, and their regulatory acceptance is slow, because not
enough evidence is usually generated to decide whether they are
worthy of making decisions about human lives. You have to know
those biomarkers are reliable before you are willing to take a
chance on a human life.
And so the question is what processes should be put in
place that help develop these biomarkers and make them robust.
The discussion draft codifies some procedures that we have in
place called the biomarker qualification process, and during
that process, we give advice to developers who are usually
consortia, because another problem is there is nobody really in
charge of this, and so these consortia come together--patient
groups, others come together--and develop the evidence on these
biomarkers. And we provide advice about what would be needed to
get them to the stage where you would be willing to use them to
make decisions about people.
So I think the discussion draft has made a lot of progress,
and we really look forward to working with you on finalizing
this very important issue.
Mr. Green. OK. Thank you, Mr. Chairman. I am out of time,
but I know we will have some other questions to submit.
Appreciate it.
Mr. Pitts. All right, thank you.
The Chair now recognizes the chairman of the committee, Mr.
Upton, 5 minutes for questions.
Mr. Upton. Well, thank you again, Mr. Chairman. And, you
know, as I reflect on this overall bill, one of the things that
I am most proud of is the money for the NIH. And, Dr. Hudson,
appreciate your kind words when I talked to Dr. Collins a
couple of times over the last week or so, he was very excited.
And I just want to read--there was a statement that Andy von
Eschenbach, who has been very helpful as well, former FDA
Commissioner, of course, he said, and I quote, ``I think it has
the potential''--this bill is what he is referring to--``has
the potential of being one of the most transformational pieces
of legislation that has come along since the National Cancer
Act of '71.'' And he praised the bill for looking at the entire
ecosystem on medical product discovery, development, and
delivery, and figuring out how to achieve more synergy between
the groups involved, the basic medical research, drug
development, approval, and reimbursement.
And I can remember the first roundtable that we had in this
room, of course, it was Henry Waxman and myself that led the
effort in the House to double the money for the NIH back in the
'90s. We teamed up with Paul Wellstone and John McCain in the
Senate to get it done. Had a lot of discussions since then,
even yesterday with Cory Booker and Durbin, and, you know, it
is something that Frank Pallone and Diana, then Joe and--we are
all very much onboard to try and increase that money.
The question I have, Dr. Hudson, for you is, is the TAP
Program, and as you know, the practice of taking away 2-1/2
percent of NIH's research budget through the evaluation TAP,
Section 241 in the Public Health Services Act, I have to
confess, must create some difficulties when planning.
Can you walk us through the challenges and added burdens
that you face when dealing with TAP and its effect on the
stability of NIH funding, and would it be in the public's best
interests for the NIH to be exempt from that requirement, as I
understand we did in the Cromnibus piece of legislation last
year?
Dr. Hudson. Well, first of all, I want to reiterate my deep
appreciation on behalf of the entire biomedical research
community and also patients for the increase in the NIH budget
that is proposed in this bill. It is a welcome change and
really quite remarkable.
In terms of the TAPS, they are complicated. They were
particularly complicated this year in the omnibus and how they
were orchestrated. It requires somebody from the Budget Office
to actually walk us through this, but it is--basically, we
still have the TAPS but they are rerouted into NIH with a
reduction in the base budget of one of our institutes, the
National Institute of General Medical Sciences. That is not an
ideal fix for this situation. The TAPS are fairly predictable,
and so we are able to base our projections of what we are going
to be able to fund, taking into account that we know that these
TAPS always come about, and that we account for them in our
budgetary and programmatic planning each year.
So they are not unexpected, they support important
programs, including programs at the National Institutes of
Health. So some of those planning and evaluation dollars come
back to us to support important programs----
Mr. Upton. Do you know about what share of that money comes
back?
Dr. Hudson. I don't know off the top of my head, but we can
certainly provide that to you. It is a nontrivial amount that
comes back to us as P&E money for us.
Mr. Upton. We are just thinking that as we try to make sure
that you have a steady stream, and one that is going up----
Dr. Hudson. Yes.
Mr. Upton [continuing]. That that is a source that ought to
be, you know, I think, for me, I would feel more--just think
that--knowing that it is used directly for research seems to
me, a better thing.
Dr. Hudson. Um-hum.
Mr. Upton. Dr. Shuren, you know that as we are developing
legislation on a new diagnostics framework, and by the way,
appreciate your help across the country as well as we have
developed this legislation, we believe that that new framework
could serve as a cornerstone to the advancement of the
provision medicine and support development of diagnostic tests.
And I just want to get your thoughts and continued commitment
to work with us as we see this proposal through.
Dr. Shuren. Mr. Chairman, we would be happy to work with
you. It is also our hope that we can all commit that the final
version on any legislation will have the support of the labs,
of the device industry, of all of you, and of course, the FDA
as well.
Mr. Upton. And I want to give you a backhanded compliment
as well. When Ms. DeGette and I were in Kalamazoo last week,
the folks at Striker Medical said very good things about the
role that you have been playing and appreciate all that you do.
So with that, Mr. Chairman, I yield back.
Mr. Pitts. Chair thanks the gentleman.
Now recognize the ranking member of the full committee, Mr.
Pallone, 5 minutes for questions.
Mr. Pallone. Thank you. I wanted to ask a question of Dr.
Woodcock first.
It seems to me that we are asking the FDA to take on a lot
of new responsibilities in this discussion draft, and the draft
would require FDA to issue more than 15 guidance documents and
implement a variety of new processes. For example, the section
on antibiotic drug development would require FDA to create a
separate approval process for antibiotics and antifungal drugs
intended to treat serious and life-threatening infections for
certain populations.
So can you talk about the time and resources that will be
necessary to implement these provisions and issue these
guidance documents?
Dr. Woodcock. Well, I think there is a trade-off between
putting out new guidances and implementing new programs, and
actually getting the work done, giving advice to sponsors and
reviewing applications in a timely manner. And I believe that
the FDA Amendments Act, which had a large number of provisions
in it that we had to implement, shows what can happen. This
chart shows that right after--in the green is our performance
of getting things done on time; drug applications, reviewing
those new products and getting them out on the market.
Immediately after the Amendments Act, and for many years after,
we were not on time with our review work, and that was because
we were implementing the provisions required under the
Amendments Act, which were important, but we did not receive
additional resources in many cases to do this other work.
So I would say, we have a saying in medicine which is,
``first, do no harm,'' and it is very important, I think, in
enacting new legislation to make sure that you don't break what
is fixed. And currently, our drug review program is really
going full-speed, we are making all our deadlines, and we would
like to keep it that way.
Mr. Pallone. Well, as you know, the current draft does not
authorize any additional funding for FDA to take on these
additional responsibilities, so can you talk about how
implementation of these provisions will divert resources from
the work that the Center for Drug Evaluation and Research is
currently doing?
Dr. Woodcock. Well, to the extent that the requirements are
statutory, and we have to get guidances out or do other work,
set up new programs in a specific amount of time, those are
directions from Congress, and those will come first. All right?
And we do try to meet all our user fee goals and exceed them
because those are the new products that need to get on the
market. And, for example, the breakthrough therapy, we try to
get those products out the door even faster than the goals
because, really, those are products that are going to be life-
changing for people. And it is no doubt though that statutory
instructions will come first, and we will have to prioritize
our resources toward getting what Congress has instructed us to
do, done.
Mr. Pallone. Well, Dr. Hudson--thank you.
Dr. Hudson, with regard to NIH funding in antibiotic
research, NIH funding has also been responsible for generating
investment in dry development pipelines, particularly areas of
critical public health need, and one such area that needs
increased investments is that of antimicrobial development,
which the World Health Organization has named as a top public
health threat. How could NIH use increased funding to support
antibiotic research and development initiatives, including
efforts to improve effectiveness and to help ensure proper
stewardship of antibiotics in our healthcare system?
Dr. Hudson. So I appreciate the question. Certainly, there
are opportunities to explore new--development of new
antibiotics. In fact, there was recently, with the support of
NIH, the discovery of a new antibiotic from a soil bacteria, as
it turns out. So we certainly have opportunities to explore the
development of new antibiotics, and also to explore the
development of approaches to treat antibiotic-resistant
microbes. That is a serious and growing problem across the
country, and we need to focus additional resources on that
serious concern.
Mr. Pallone. All right, thank you.
I am just trying to get one more question to Dr. Woodcock.
In addition to increased NIH funding, which has long been a
priority, one of the provisions in this discussion draft that
is especially important is the FDA Grant Authority for studying
the process of continuous drug manufacturing, and the
conventional process of batch manufacturing is outdated, but
continuous manufacturing will benefit patients and
pharmaceutical companies by increasing quality and efficiency.
Dr. Woodcock, can you talk about the difference between
batch manufacturing, continuous manufacturing, and what
advantages does continuous manufacturing provide, and what do
you think--or why do you think it is more widely used in this
country for drug manufacturing?
Dr. Woodcock. I----
Mr. Pallone. You have 7 minutes.
Dr. Woodcock. I don't know why----
Mr. Pallone. Seven seconds.
Dr. Woodcock [continuing]. It is not more widely used
because if you think of batch manufacturing, it is like
cooking, and instead of having like a little cake mixer, that
you have a gigantic cake mixer. And then you take all that
stuff and you put it into some other machine, and that is what
they mean by batch. So you do one operation, then you transfer
it to another operation, then you transfer it. There is a
tremendous amount of waste, and there is a tremendous amount of
opportunity for not getting things right when you do this mass
mixing and so forth, and you want to get it into little pills
at the end.
So continuous manufacturing at its best, you take the
ingredients at one end, the chemicals, and you make the active
and then add whatever else you are putting in it, in a
continuous stream. So it comes out at the end all done, one end
to the other. And you can measure it carefully. Each tablet you
can measure, whether you made it right or not, by computer. And
so this is the future of drug manufacturing. It is much more
efficient. It also can bring manufacturing back home because
there is no reason to do that all around the world, like there
is now with these gigantic factories that are needed.
So this cannot be accelerated enough, in my opinion.
Mr. Pallone. Thank you. Thank you, Mr. Chairman.
Mr. Pitts. Chair thanks the gentleman.
Now recognize the vice chair of the full committee, Mrs.
Blackburn, 5 minutes for questions.
Mrs. Blackburn. Thank you so much, Mr. Chairman.
And, Dr. Shuren, I want to say thank you to you and your
team for working with my team and also Congressman Green, as he
mentioned earlier, on our Software Act, which is a part of this
legislation. We think we are in a better place on that, and we
thank you for your participation.
Dr. Hudson, I want to come to you with some questions. The
Cromnibus that we passed last December required NIH to do an
NIH-wide strategic plan. I want to know where you all are in
that process, when it is going to be completed, and are you
incorporating some of the elements we are discussing today?
Dr. Hudson. Thank you very much for the question.
So we are, in fact, in the process of developing that
strategic plan. We have put together a group of NIH leaders
that includes some of the directors of the institutes and
centers across the NIH who have begun this process. The
Cromnibus requires that we complete this strategic plan by
December, and we intend to meet or beat that deadline. We are
excited about integrating the overarching strategic plan for
the National Institutes of Health with the strategic plans that
are already required and provided by each of the 27 institutes
and centers. And so those will be linked together in
fundamental ways.
We appreciate some of the modifications that were taken
into consideration in the revision of the discussion draft;
removal of some of the more onerous requirements for the
strategic plan and related provisions, but we are well on our
way and look forward to sharing that strategic plan----
Mrs. Blackburn. Wonderful. We look forward to getting it.
We think it is an important part----
Dr. Hudson. Um-hum.
Mrs. Blackburn [continuing]. What we are trying to do
through the Cures legislation, that we be focused and
strategic, and that we set some goals. And also we think that
accountability and transparency is an important part of this
process, and in that, we want to make certain that you all are
prioritizing your spending. And so as you go through this
process of developing that plan, that is something we are going
to be looking for. And I wondered, as we were looking at this,
as you look at your spending, do you look at portfolio analysis
and conduct that, and you want to speak to that for a second?
Dr. Hudson. I do. I do. I appreciate the interest. And we
have been looking very carefully, in part because of the
constriction and the available budget for the NIH, it has even
been more important that we make sure that we get as much value
of every dollar that we invest as possible, and that we are
investing in the right opportunities to address the challenges
that face us, and translating basic science into translation
into the clinic. So we have--are in the process of enacting a
series of stewardship reforms to make sure that we are looking
carefully across the portfolio, and of course, we have the
technologies today to be able to do that. It used to be with
paper records we couldn't really do that. Now, with the press
of a button and some new nifty tools, we can look across and
see what are we funding in a particular area, what are other
Government agencies funding in a particular area, and where are
there opportunities that we need to focus more attention on. So
those are great opportunities that we are looking at to make
sure that we are spending all of our dollars very wisely.
Mrs. Blackburn. Yes. I was recently at Vanderbilt
Children's Hospital in Nashville, and we were discussing a
little bit about some of the childhood diseases and research.
So talk to me about what you are doing with children. As you
look at this portfolio analysis about children benefitting from
the cures and the scientific advances that are there through
NIH funding.
Dr. Hudson. So we are going to be going down to Vanderbilt
the--later in the month of May for our working group meeting on
precision medicine. We are really looking forward to that. So
we spend probably 10 percent of our budget focused specifically
on pediatric research. That doesn't say that kids are not
included in other studies, but about 10 percent are directly
focused on children.
Mrs. Blackburn. OK. Now, let me ask you this.
Dr. Hudson. Yes.
Mrs. Blackburn. I am under the impression that you all do
not have a method to track all children in all studies. Is that
correct?
Dr. Hudson. So we do have mechanisms to be able to know
that children are or are not included in the studies. It is a
question that is asked of applicants in the grant application.
We also have means of being able to follow whether or not
children were or were not included in trials in the course of
progress reports, and in Clinicaltrials.gov, which is now being
upgraded and implemented in full force, there is a
requirement----
Mrs. Blackburn. OK, my time is expiring, and I want a
fuller answer on this, and I know----
Dr. Hudson. I look forward to providing that.
Mrs. Blackburn [continuing]. You would like to give it.
Dr. Hudson. But I think that what we would like to do is be
sure that you have a better system for tracking children so
that they are included in the appropriate studies, and I would
look forward to working with you on that.
And I yield back.
Dr. Hudson. Likewise. Thank you.
Mr. Pitts. Chair thanks the gentlelady.
And now recognize the gentleman from Maryland, Mr.
Sarbanes, 5 minutes for questions.
Mr. Sarbanes. Thank you, Mr. Chairman. Appreciate the
testimony today, and I want to congratulate the members who
have been working on this piece of legislation for some time
now, obviously making tremendous progress with it.
I wanted to follow up a little bit on what Representative
Pallone was asking about in terms of the resource challenge
potentially for the FDA, Dr. Woodcock and Dr. Shuren.
Obviously, I don't have the handle on the inner structure of
FDA that you do, but just conceptually, I imagine that there is
basically a main review process that exists, and then what
seems to have happened over the last few years, for
understandable reasons, is we keep pulling things out and
creating priority reviews, and expedited processes and so
forth. And I wonder if there comes a point at which, if you
kind of expedited every last part of what the original main
review process was, that you kind of slice the agency up into
so many little component parts that you would stand back and
look at it and say, well, if we had just gone ahead and
expedited the overall main process, we would probably have a
more efficient allocation of resources, and we might even have
faster review in place.
So could you just comment on, sort of, if you take this out
to the nth degree, or to its logical conclusion in terms of
constantly expediting what you have to do, whether you end up
with some kind of structural distortion in the way you are
supposed to operate, that even with additional resources, which
I think are important, would mean that you couldn't get to the
efficiency that you ultimately want to have, and that the
public and that we want to see you have. And it may be that
that tension I am describing is really not as much of a
challenge as it appears to me, but I would like to get your
thoughts about it.
Dr. Woodcock. Well, basically, we have expedited review for
everything because of the Prescription Drug User Fee Act that
Congress has passed multiple times, and then the Generic Drug
User Fee Act. We have timelines for everything, all the
applications we review, and under the PDUFA we have timelines
for meeting with companies, and for getting minutes back to
them. We track tens of thousands of different activities that
we are supposed to do. And so it is all part of the review
program. And the same people then have to do the pediatric
program that Congress passed, and they have to do the
breakthrough program, and they have to do many other programs
that we have that, of course, people have been very interested
in. And so I think these things from the drug center point of
view could be accomplished with adequate resources, but we are
at the point where we add more programs on, with the same
people trying to implement them, and we slow the whole thing
down, as happened in 2007.
Dr. Shuren. So it is a similar situation on the device
side, and that is not a criticism about good things people want
to do, it is just recognizing the fact that our people are
people and they have a lot of work on their plates, and we have
commitments to meet, and the more things that get piled on, the
more we are set up for failure. It is one of the reasons why I
deal with a high turnover rate in our review divisions and in
the center, because their workload is high and the more that
goes on, the more challenging it is.
You know, when we looked at our budget--what we get for our
budget authority for this year, compared to 10 years ago, even
though there were some increases, and none since 2011, if you
factor in increased inflation and mandatory pay increases, our
purchasing power today is the same as it was 10 years ago, but
our responsibilities went up. And our only real increases in
funding come from industry. They pay for it, but they pay for
services they get in return, not for the other things we do.
And we are excited that NIH will get more support, but all
those great things don't get forward out to the market and
those assessments on whether or not they are safe and effective
unless we are in the position to do our work.
Mr. Sarbanes. Well, and the other, I guess, the bottom-line
issue is that this effort for expedited review and processing
of things creates expectations on the part of the public, and
if you can't meet those expectations because of resources then,
you know, you end up creating a more kind of cynical public as
a result. So I think it is really important that this resource
piece be addressed and be robust.
And with that, I yield back.
Mr. Pitts. Chair thanks the gentleman.
Now recognize the chair emeritus of the committee, Mr.
Barton, 5 minutes for questions.
Mr. Barton. Thank you, Mr. Chairman.
Before I ask my questions, I want to compliment you and
Chairman Upton and Mr. Pallone, Ms. DeGette, and others for
this discussion, for this draft that we released yesterday on
the 21st Century Cures. It is literally transformational.
Healthcare has been a priority of mine in the time I have been
in the Congress. I helped lead the effort to reauthorize the
NIH back in 2006. I have helped in bills to reform the FDA, but
I would say this piece of legislation, if it goes forward, and
hopefully it will, will be a landmark not just for this
Congress, but for many, many Congresses. So I want to
compliment you and all the people that have worked on it. I am
extremely pleased with what is in the draft. Now, there are
some things that are not that I wish were. I had hoped that my
Ace Kids Act, which is bipartisan, bicameral, with over 120
cosponsors, was in the discussion draft. It has been deleted
from this draft. I hope to have discussions about that and
perhaps get a hearing just on that piece of legislation because
it is certainly worthy of being included, or moving as a
standalone bill.
Dr. Hudson, you are the deputy director. I spent quite a
bit of time with the director, Dr. Collins, out at the Milken
Institute this past weekend in California. I was on a panel
with him Monday morning, so I am very pleased that, if he
couldn't be here today, that you are here. I am going to ask
you some specific questions about what is in the draft, and
hopefully you can make your answers succinct so that we can get
through a number of questions.
The discussion draft creates a review--a new review panel
called Biomedical Research Working Group, to identify and
provide recommendations to the NIH director on ways to reduce
the overhead burdens. You have existing at NIH a Scientific
Management Review Board which is already set up, already
established, and basically, either is doing or could do the
same thing. In your opinion, could the Scientific Management
Review Board that already exists do the function that the new
Biomedical Research Working Group is tasked with doing in the
draft?
Dr. Hudson. So it is certainly a possibility. Either the
SMRB could undertake this review, or a working group of the
SMRB could undertake this task. Similarly, it could be a
working group of the advisory committee to the director. There
is also a National Academy of Sciences Study that has just been
undertaken to look at scientific burden. This is an important
administrative burden on scientists. This is an important
problem we need to solve.
Mr. Barton. Well, I am certainly not opposed to there being
a review of biomedical research, but in my opinion, to create a
brand new group doesn't make sense when, as you just pointed
out, you have several groups that are already in existence, and
the overhead is there, the staff is there, we could just give
them that task.
The draft has a creation of an Innovation Fund that it
funds at $2 billion for 5 years. Again, I support the concept.
In 2006, we created the Common Fund, and we set a minimum of
1.8 percent, which is about 6 or $700 million.
Dr. Hudson. Um-hum.
Mr. Barton. That Common Fund has done great work, but it
has never been increased in funding. It stayed about 1.6 to 1.8
percent of the budget. It is authorized up to 5 percent. In
your mind, could not we put this $2 billion that we earmarked
for the Innovation Fund and put it into the existing Common
Fund, because that was the whole purpose of the Common Fund
which was give the director the ability to move money where it
would do the most good?
Dr. Hudson. So the Common Fund has been an amazing asset
for the NIH, and I appreciate you having created that in the
2006 Revitalization Act. The--an Innovation Fund that is
proposed in this discussion draft does include $2 billion, and
has two specific purposes, and one other purpose that is yet to
be defined. And we look forward to working with you on that.
The specific part of the Innovation Fund that I think is
important is that it permits the distribution of those funds to
the institutes and centers for innovative research. And so I
think that we need the ability to be able to funnel those funds
to important opportunities across the institutes and centers.
Mr. Barton. OK. And finally, my last question. The
discussion draft creates a biomedical--I mean in the discussion
draft--it is not discussion, it is a draft now, a bill, we--it
requires each institute director to look at biomedical research
at the institution. Congressman Harris, who is on the
Appropriations Committee, and myself have a bill that creates a
biomedical research officer at OMB, because OMB looks at all
the agencies. Which approach do you think is better; letting
each institute director do this review, or having somebody at
OMB who looks at all the agencies and that is their only job?
Dr. Hudson. So I think that we need to have scientific
decisions made by people with scientific expertise who have a
focused disciplinary background. So I would prefer that those
kinds of decisions remain at the NIH. The institute directors
and their Advisory Councils have an important responsibility to
not just consider the priority score that comes out of peer
review, but also to consider other factors, and we are making
sure that those best practices are shared across the institutes
and adopted.
Mr. Barton. That is not the answer I wanted, but I got two
out of three so I am going to declare victory and turn it back
to the chairman.
Mr. Pitts. That was excellent. The Chair thanks the
gentleman.
Now recognize the gentlelady from California, Ms. Matsui, 5
minutes for questions.
Ms. Matsui. Thank you, Mr. Chairman.
Before I begin my questions about specific provisions, I
would like to reiterate points my colleagues have made about
how critical it is that we adequately fund agencies to do all
the work that we expect them to do. I am pleased that we were
able to include both strong discretionary and mandatory funding
screens for NIH research in this legislative draft. I urge my
colleagues to provide similar financial support for the FDA as
we move forward. We expect the FDA to make sure that our food
and our drugs are safe and effective, and it is our
responsibility as Members of Congress to ensure the FDA has the
resources to do so.
There are several provisions in this legislative package
that would help patients with rare diseases. I support the idea
of incentivizing the development of new and existing drugs that
will make a difference in patients' lives, especially rare
disease patients who may not yet have the treatments or cures
that they need. However, I am cautious to balance the
incentives for development with the ability for generic
competition to come onto the market, as that is a key aspect of
drug access and affordability.
This bill isn't perfect and there are many pieces that
still need to be worked on, but I would like to highlight a few
pieces that have the potential to really get at the goal we are
all after in an effective and balanced way.
Dr. Woodcock, as you know, patients with life-threatening
conditions are often willing to try riskier treatments than
other types of patients. The FDA has the Expanded Access
Program to increase access to experimental drugs for these
patients. 21st Century Cures includes a provision based on the
Andrea Sloan CURE Act, which I cosponsored with my colleagues,
Representatives McCaul and Butterfield.
Dr. Woodcock, can you comment on FDA's Expanded Access
Program and how the related provision will help patients who
seek increased transparency in the program?
Dr. Woodcock. Well, currently patients in the United States
can get access to investigational drugs if their doctor applies
to the company. FDA facilitates these interactions and rarely,
rarely turns them down. So thousands of patients--a 1,000
patients or patients every year get expanded access. However,
there isn't transparency on company policies on whether or not
they will be providing such access and how. And so the bill
does urge companies to post a policy so that people would know.
We think that having a point of contact also would be
helpful because sometimes we don't know who to call to find out
how to arrange expanded access for a patient. So we believe
that transparency would be helpful, and we believe, in our
conversations with the community, that entities will step
forward to help broker those connections between the healthcare
professionals and the companies so that there is much more
transparency in this.
Ms. Matsui. Thank you.
Dr. Hudson, a part of seeking cures for patients should
include collecting data about their conditions and current
treatments in order to better understand their diseases. A
couple of provisions of this package would enhance data
collection. I want to ask about the Neurological Disease
Surveillance System for diseases like Parkinson's and MS, since
CDC is not here as a witness. But surveillance is an important
public health function, and I support that provision.
Dr. Hudson, can you describe the idea in Section 1123 to
establish a partnership between NIH, FDA, industry, and
academia to establish or enhance an IT system to manage data on
the natural history of diseases, especially rare diseases?
Dr. Hudson. So I believe that section actually provides the
authority to the Secretary, and so it will be up to her to make
the decision about how that is implemented. And I will turn to
my colleagues at FDA to weigh-in on this as well.
There are a number of ongoing activities that provide
information especially about rare and neglected diseases, both
through the National Library of Medicine and through the Office
of Rare Diseases at the National Center for Advanced and
Translational Sciences, and what I would like to do as we move
forward with this bill is to make sure that these new
information systems are compatible and synergistic, in fact,
with existing systems so that we don't end up having many, many
different places for information about rare disorders, so that
when people are encountering a situation where they have a
child, for example, without a diagnosis, that they don't have
to go to multiple places to find the information they are
looking for, but can readily find it.
Ms. Matsui. But I just want to ask how would NIH and FDA
work with non-governmental organizations like NORD to
incorporate existing disease registries?
Dr. Hudson. Go ahead.
Dr. Woodcock. Yes. Well, we are very interested in and, in
fact, have been working with NORD, and have talked to other
stakeholders as well. When planning a trial of a new
intervention into a rare disease, you have to know what happens
to the people or you can't make a plan----
Ms. Matsui. Sure.
Dr. Woodcock [continuing]. And that is why we need to
collect data over time on people with very rare diseases and
what happens to them. And so we are very interested in these
tools that will help patient groups actually collect the data,
and have a repository so we can plan trials better and
developers can understand what they need to do.
Ms. Matsui. I thank you very much.
And I yield back.
Mr. Pitts. Chair thanks the gentlelady.
Now recognize the vice chair of the subcommittee, Mr.
Guthrie, 5 minutes for questions.
Mr. Guthrie. Thank you, Mr. Chairman.
Dr. Shuren, the provisions of Cures are both big and small,
and they all were created to improve the way we develop access
to cures. One provision which I have championed is Section
2218, which seeks to create more clarity around the CLIA Waiver
process for both the benefit of industry and for the FDA. Can
you tell me your thoughts on the benefits of clarifying the
CLIA Waiver Program?
Ms. Shuren. Yes, we had put out guidance in 2008 to attempt
to provide greater clarity, and we understand there really is
more flexibility out there for what companies can do, but we
haven't provided that sufficient clarity, both for them and,
quite frankly, for our own staff. So we support moving forward
to update that guidance and provide that level of clarity and,
of course, work with the community on a final product.
Mr. Guthrie. Thank you, Dr. Shuren.
And, Dr. Woodcock, matter of fact, Mr.--Congressman Pallone
kind of got into the continuous manufacturing, and I am a
manufacturing background and so we are looking at this as we
are moving forward, and going from batch to continuous, if it
is efficient and--it seems like that would develop naturally
through the marketplace. But my understanding, and so I ask
that question, is the regulatory uncertainty is what authority
you have to grant, and what authority the manufacturers have if
they change, does that change the whole process, so we put a
provision in to have a grant program to invest in, so it is not
just happens just like the marketplace outside because of the
regulatory process. So why is it important that we invest, and
why do you--why is this necessary to move to a more continuous
manufacturing program?
Dr. Woodcock. Well, there have been many factors that have
led to this industry making such valuable products actually
having its manufacturing processes not be state-of-the-art. And
some of that has been regulation, because the old manufacturing
processes are so uncertain, because of the nature of the bulk
efforts that they are doing, they are very strictly regulated
and any changes the manufacturer makes--any substantive
changes, they have to apply to us and get approval and so
forth. And it takes quite a while. Not necessarily us, but
doing all the documentation. And so that has been one factor
that has held back innovation in this area.
Another factor, though, is that these products, I think,
are so valuable, but I don't think the industry, until
recently, felt manufacturing was a competitive advantage. And
so the R&D people got all the glory, and the manufacturing
folks were told just get the product out the door and don't
change anything. So now, because of various changes, that is
altering, and we are seeing applications with continuous
manufacturing, and we are working with companies. We are not a
barrier, but we need more of an academic base in this to feed
ideas into the manufacturing sector. And that is where we would
like to provide more grants and so forth, more funding of some
sort, to enable academia to contribute to this revolution.
Mr. Guthrie. All right, thank you very much. I appreciate
that answer.
And, Mr. Chairman, while representatives from CMS are not
here today, I do believe it is important to touch on an area
that will be addressed in Cures for which more work needs to be
done. The national and local coverage discrimination process
within CMS are the processes whereby new technologies gain
entrance to the Medicare Program, and I have heard numerous
concerns about the current processes, specifically for LCDs,
that need to be addressed, and I certainly deeply appreciate
the bipartisan support for the narrow provision that is
included in this bill. However, I believe there is still more
to be done, and I plan on gathering more information on this
topic and working with stakeholders to gather more ideas on
ways to improve the LCD process.
I look forward to working with the committee and the
Administration as I move forward. And thank you, Mr. Chairman,
and I yield back.
Mr. Pitts. Chair thanks the gentleman.
Now recognize the gentleman from Massachusetts, Mr.
Kennedy, 5 minutes for questions.
Mr. Kennedy. Thank you, Mr. Chairman. I want to thank the
witnesses for your testimony today. Thank you for coming. I
also want to thank the chairman of the subcommittee and ranking
member, and Chairman Upton, Mr. Pallone, Ms. DeGette, for all
their hard work in bringing this bill to this place where it
is. It has obviously undergone an awful lot of work, and from
somebody in Massachusetts who has a vocal constituency that is
very much looking forward to the movement of this bill through.
Excited to see the progress, and obviously, a lot of work that
still needs to be done.
But I wanted to focus a little bit, if I can, back at
funding mechanisms for NIH. And, Dr. Hudson, maybe to start
with you. Obviously, Federal investments in medical research
have, and continue, to transform healthcare, advance new
treatments, therapies and screenings. Nowhere is this more
evident than at NIH. In fact, the 2011 Health Affairs Studies
found that nearly \1/2\ of all patents for new drugs cite
public sector patents or research in their applications.
Increased investments in NIH yields groundbreaking research,
fuels industry, serves as a foundation for this Nation's
greatest scientists. Funding has obviously stagnated for years.
And as I indicated, this is a huge--not at--certainly not a
week goes by, and often not a day goes by when I don't have
constituents that come into our office and indicate that this
is a huge priority for Massachusetts.
Thrilled to see the increase in funding that is included in
this bill. And wanted to dig in a little bit to your thoughts
around the Innovation Fund. So the first priority there is
precision medicine which, again, from Massachusetts, we have
some great companies that are developing life-changing
precision medicines to treat cancer, cystic fibrosis, Gaucher's
Disease, and--just to name a few. There is a lot of progress
there--or promise there. I think we have to work through some
still--challenges as the process goes forward, but I was hoping
you could dive into the precision medicine funding mechanisms a
bit. Another priority there is young scientists which, again,
comes on a daily or weekly basis to me from our hospitals and
provider communities saying that they are losing young,
talented scientists to other industries, or even to other
countries. Wanted to see if you could touch on that.
And the third piece that--I know it might be a bit
premature, but--is that other bracket. So what do we think
other might mean? And I don't mean to put you on the spot, but
if you can flush that out a little bit, I would be grateful.
Dr. Hudson. Thank you very much. So on precision medicine,
we are still in the early stages of trying to really sketch out
a specific plan for the national cohort part of this in which
we want to invite a million or more Americans to share with us,
share with researchers their health information, genomic
information, and environmental exposures, behavioral
information and the like. And patients are eager to do that.
They want to make sure that the best information is made
available to advance their heath and that of their families and
other Americans. So that plan is being developed. We are really
excited about it, and hoping to use new innovative mechanisms
of being able to fund that research, and also leverage the
resources of others in the private sector to do some
collaborative work together.
On emerging scientists, this is a substantial problem. We
need to reach sort of an equilibrium in the workforce pipeline
so that we can attract new investigators in. Certainly, young
people are going to see this $2 billion mandatory funding
stream as an opportunity to--and encouragement to stay in and
dig in, and stay with the biomedical research enterprise.
And then in terms of that other category, which is
intriguing and we haven't had a lot of opportunity yet, since
it has only been out for 24 hours, to talk about it with the
leadership at NIH, but I think initial considerations are we
would really like to be able to make sure that we are funding
innovative investigator initiative research. The best ideas
come from the best brains across America, and we don't
necessarily anticipate what those ideas are going to be until
they come before us. And right now, we are only paying 18
percent of the grants that come to us, and we know we are
leaving great science unfunded. And so being able to pay more
of that good science would--might be a priority as well as the
brain initiative.
Mr. Kennedy. I have a minute left and so----
Dr. Hudson. Yes.
Mr. Kennedy [continuing]. I wanted to get a brief
discussion from the rest of the panelists as well.
You, Dr. Woodcock, I think indicated that basic tenet of do
no harm. We are putting a lot of exciting opportunities at your
doorstep. Do you--as contemplated, does FDA have the resources
to actually make these transitions and make these investments
as effectively and as efficiently as possible, particularly
when part of the challenge, at least that I hear, again, from
my communities back home, is how long it takes to get some of
these drugs and devices approved?
Dr. Woodcock. Well, I think we are very stretched. I think
we are up against the wall always. We are always asked to keep
doing more with less. We do not take a long time to get things
approved. They take a long time to get developed. And it is our
advice that is so important, and that would be one of the first
things to go because that is more discretionary, but it has
been shown that we can cut years off of company's development
time by giving them--if they come in for timely advice, because
we see across the board all the development programs. But yes,
we are very stretched in our resources. And, of course, some of
the hiring and assistance that is contemplated in this draft
would be helpful as well because we are also below our
ceilings.
Mr. Kennedy. Great. Thank you.
And, Dr. Shuren, apologies, but I am over time. So thank
you very much for your testimony and thanks for coming today.
Mr. Pitts. Chair thanks the gentleman.
Now recognize the----
Mr. Kennedy. Chairman, thank you.
Mr. Pitts [continuing]. Gentleman from Illinois, Mr.
Shimkus, 5 minutes for questions.
Mr. Shimkus. Thank you, Mr. Chairman. It has been a long
time since Mr. Green was asking his questions, but there is one
point of what he was asking that I just wanted to build upon in
the Subtitle K. So, Dr. Shuren, can you tell me the types of
resources contained with the priority view for breakthrough
devices section of this bill, and how important they can be to
the FDA and industry when seeking approval of a breakthrough
product?
Dr. Shuren. So we do think this is an important program. It
is something we had launched. It can tremendously help
important technologies getting to market, getting to patients,
but still be safe and effective technologies. Our challenge
will be having the people to do this work. We know from
piloting the innovation pathway in 2011 it requires a lot more
people to do it. I think Janet and her program on the drug side
found it requires a lot more people to handle breakthrough
drugs.
When we proposed our program, we said we would do it
resources permitting, because we do not want to jeopardize the
commitments we made under the User Fee Act or the other work we
have to do. With the statutory provision, the challenge we have
is this is mandated, we have to do it, and the law says so. And
we are concerned that when we move forward on this, we will not
have the people to succeed at all the things we have to do, and
the things that are important to do for patients.
Mr. Shimkus. So in going to Subtitle L, which contains a
number of regulatory improvements for both the FDA and
industry, for instance, Section 2201, the third party quality
system assessment can lower the burden on both FDA and the
industry when such actions are warranted.
I am wondering if you can spend a few minutes and tell us
how the FDA sees this section improving the Cures delivery
cycle.
Dr. Shuren. So this program is--pertains to modifications
that are made to high risk devices under PMA, and moderate
devices under a 510K. And it looks at a subset of modifications
that, if we had assurances the company had what we call a good
quality system, it is essentially their system for designing,
making changes, supplier controls, manufacturing, that we would
not need to see those modifications. We could rely on a third
party assessment of that quality system for those device types.
And we think that would be very helpful to industry. We looked
at it--will this be an efficiency for us?--and it turns out
probably not, and here is why: It will cost us money to set up
the program and maintain it, to have the people that go out
training the third parties and auditing them. At the same time,
we might free up some of the work we do in reviewing these
submissions. They tend to be less work for those kinds of
submissions for modifications. On the other hand, we lose all
of the user fee revenue we would have gotten. So when we
crunched the numbers, this may actually cost us money.
We still think if we can work this through it could be a
very good thing to do, but we have to be cognizant about the
resource implications.
Mr. Shimkus. Thank you. That is very helpful.
Yes, and for the chairman and the ranking member, I know
Mr. Green and I are pleased that adapt language in the draft is
in this current draft, and give credit to Dr. Gingrey, former
member, who was really a pusher of that in the last Congress.
And I have been pleased to take a lead with Mr. Green on this
process. It is reported, as you know, over two million
Americans each year get sick due to antibiotic resistant
bacteria, and tens of thousands die as a result. And I can go
over all the stats, we all know them. I guess getting just to
the question, it is really--I still--even though I am happy
with the draft, there is still, I think, a need, if we want to
respond and we want to expand immediately and more
appropriately for continued incentives.
So, Dr. Woodcock, would you want to speak on that issue?
Dr. Woodcock. Yes, we probably can't do enough to get this
crisis addressed. We are doing more under GAIN. GAIN was very
helpful. We thank you. We think that a limited population
approach will be very helpful as an incentive because it has
fewer patients and fewer costs associated with it, and it will
be faster. We still believe, of course, we don't think we need
a new program, and we would really like to see a logo or some
kind of statement in the label. However, even if this program
is enacted, I think it will attract investment because it is a
very limited development program, and so the bar is lower.
However, I don't know that that will be enough.
Mr. Shimkus. So, Mr. Chairman, just--so you are saying
probably additional incentives might be needed?
Dr. Woodcock. Well, we can't do enough to address this
crisis in my opinion.
Mr. Shimkus. So you are saying additional incentives might
be needed.
Mr. Pitts. Chair thanks the gentleman.
And now recognizes the gentlelady from Florida, Ms. Castor,
5 minutes for questions.
Ms. Castor. Well, thank you, Mr. Chairman, for calling the
hearing today.
I am very pleased with the progress on the 21st Century
Cures Initiative by the committee, and want to thank Chairman
Upton and Ranking Member Pallone, and my good friend
Congresswoman DeGette, and Congressman Green and Chairman Pitts
as well. I think it is moving in the right direction.
One of my top priorities as a Member of Congress has been
to ensure steady and robust funding for the National Institutes
of Health. Today, medical research in America is entirely
discretionary. So that means that it is at the mercy of all of
the congressional budget battles and sequester, and that brings
on a lot of uncertainty. And I know all of my colleagues hear
the same thing from research institutes and scientists in their
own district. We will only save lives unless we have robust
funding of medical research in America. And I think Dr. Hudson
really said it in a very kind way, that we have a diminishing
ability to pay for the treatments and cures of the future. We
have really fallen behind. There was a recent Journal of
American Medicine that went into how we are at risk of losing
our competitive edge to other countries around the globe. And,
in fact, in the last 2 years, I have offered amendments in the
Budget Committee to the Federal budget to shift medical
research funding from the discretionary category into the
mandatory section because I don't believe that medical research
in America anymore is discretionary. This is something that we
have to demonstrate a commitment to. But, you know, those
amendments were always voted down on a party line vote, but the
dialogue was very interesting because there was a great sense
that something needed to be done. So I think it is appropriate
that it is the Energy and Commerce Committee and the
authorizing committee that begins to take that step towards
moving research funding into the mandatory section.
I am also very pleased with the precision medicine portion
and the Innovation Fund. Under what is currently happening at
NIH, I know $200 million of that will go to expand cancer
genomics research. And there is a very exciting collaboration
underway at the Moffitt Cancer Center in Tampa, along with Ohio
State and the new partners of University of Colorado, New
Mexico, University of Virginia. And what they are going to do
is launch a database with more than 100,000 patients who have
consented to contribute tissue and clinical records for
research to understand cancer at the molecular level. They are
going to use the total cancer care protocol to create a
collaborative environment.
I know, Dr. Hudson, you had mentioned that before, and it
appears you believe that this bill continues to give NIH the
flexibility that you need to move forward on those kind of
initiatives, is that right?
Dr. Hudson. It does, and we deeply appreciate the new
investment in NIH, or proposed investment in NIH. We agree that
investments in medical research really are mandatory. We must
invest in medical research in order to bring cures to patients.
Ms. Castor. Thank you. And, Dr. Woodcock, on the precision
medicine provisions in this draft bill, is the same true for
FDA? I know the Center for Drug Evaluation and Research has
been actively working for a number of years with a particular
focus on pushing for the development of targeted therapies. I
understand CDER has approved 30 such therapies since 2012. This
new section in the draft is intended to help you, but tell us,
does it help, is it counterproductive, does it need additional
work?
Dr. Woodcock. Well, the basic research that underlies
understanding disease can only help in developing treatments
for those diseases. So, yes, I think that investing in
biomedical research to understand diseases will generate a new
level of understanding that will lead to more targeted
therapies for a wide variety of diseases.
Right now, it is concentrated in cancer, in rare diseases,
and in a couple of other areas, and the goal here, I think, is
to make precision medicine more broadly available by
understanding the genetic basis of these.
Ms. Castor. OK, that is very helpful.
And I would also like to add my concern for not having the
ACE Kids Act included in 21st Century Cures, and I look forward
to working with my good friend and colleague, Congressman
Barton, to work on that. That is the Advancing Care for
Exceptional Kids Act to improve how we deliver care to children
with complex medical needs. And I thank Congressman Barton,
chairman emeritus, for raising the issue today.
Thank you, and I yield back.
Mr. Pitts. Chair thanks the gentlelady.
Now recognizes the gentleman from Pennsylvania, Dr. Murphy,
5 minutes for questions.
Mr. Murphy. Thank you, Mr. Chairman. It is great to see
this panel here. Thank you so much for your valuable input.
Couple of quick questions. Dr. Hudson, in the bill on page
65--you don't have to look it up--but the draft version of the
21st Century Cures legislation it states, and I will read it
for you, ``medical research consortia consisting of public-
private partnerships of Government agencies, institutions of
higher education, patient advocacy groups, industrial
representatives, clinical and scientific experts, and other
relevant entities and individuals, can play a valuable role in
helping develop quality biomarkers.''
Can you give me some input on what you see is the value of
these public-private partnerships as laid out in the
legislation for biomarkers?
Dr. Hudson. So there certainly are opportunities for
representatives from different sectors to come together to
explore what are the challenges and opportunities in being able
to develop biomarkers. And as Dr. Woodcock mentioned,
biomarkers are really measurements of something that is going
on, and those are used sometimes in preclinical research, and
are extraordinarily valuable, but the ones, of course, that are
of highest interest are those biomarkers that are used as
surrogate endpoints in clinical trials that are related to drug
development. And so we can certainly work collaboratively
together, and are. There is a biomarkers consortium that
involves FDA and NIH and others. There is the Critical Path
Institute that is involved with multiple stakeholders and
looking at biomarker issues. The Accelerating Medicines
Partnership, a great new public-private partnership that was
launched just over a year ago that includes us, FDA, and a
number of pharmaceutical companies and patient groups. It is
also looking at biomarkers development, especially in
Alzheimer's Disease.
Mr. Murphy. I think I am going to come back to Alzheimer's
in a moment.
Dr. Woodcock, I want to ask both of you this question too.
Consortia like this are key in biomarkers for mental illness,
it seems to me. In July of 2014, the Psychiatric Genomics
Consortium identified 128 independent associations spanning
108--that are common in schizophrenia. It was a major, major
breakthrough. So how will the 21st Century Cures legislation
help translate some of these insights derived from this
research to new medical treatment such as drugs to treat
serious mental illness? Either of you comment on that?
Dr. Hudson. Well, certainly, the increased investments in
NIH will allow us to support additional research, particularly
at the National Institute of Mental Health. And I know you have
had many conversations with Dr. Insel about the investments and
their importance. So that would be the primary benefit of the
new 21st Century Cures legislation for us and moving that field
forward.
Dr. Woodcock. Well, as I have said many times, I believe
there is somewhat of a gap between the basic discovery of these
and the evidence you need to generate to understand which one
of them is actionable. We would really like to be able to
subset schizophrenia. We would really like to be able to do
earlier diagnosis. Right? We would really like to be able to do
early intervention, but how do you get from identifying these
genes and actually to something you can take action on? And
that is evidence generation of some of the things that
consortia are doing, but I feel that enough of it is not
occurring.
Mr. Murphy. Well, let me add to this, you know, we are
dealing here also with really alleviating a lot of pain and
suffering from patients and their families. We heard from the
President's Council on Science and Technology on the costs
imposed by major chronic illnesses like Alzheimer's, and
stunningly, the President's Council noted that Alzheimer's
imposes a huge financial burden on America's economy with an
annual cost of about $200 billion. The National Institute of
Mental Illness, Dr. Insel, I think he wrote that there is about
$57 billion cost also, which is equivalent to the cost of
cancer, just for treating severe mental illness, but those
numbers are probably way low. NAMI estimated that for bipolar
alone, the costs were $45 billion per year. And yet I am
frustrated, as I am sure NIH and NIMH are, that we spend only
about $900 million a year on researching mental illness, this
devastating brain disease.
So do you see, I would like to ask this panel, do you see
this bill in helping us move forward then, and do we need to
tweak anything in getting more funding, more research, more
focus on these devastating brain diseases such as Alzheimer's
and severe mental illness? I will let you go across the panel.
Dr. Hudson. So I think that mental illnesses are
particularly challenging. We don't understand very much about
how the brain actually works, and understanding the normal
function of the brain and the abnormal function of the brain is
going to be critical in order for us to make breakthroughs in
terms of treating many of these devastating mental illnesses.
One opportunity and where we can certainly have increased
investment is in the brain initiative in order to understand
the networks and circuitry in the brain, both in the normal
human brain and in the abrupt, misfiring human brain. That will
help in a whole host of mental illnesses and in neurological
diseases as well. And so that is an area where I think is ripe
for investment. The Blue Ribbon Panel that set forth the
spending plan for that, we have not yet made those budgetary
targets, and we would be happy to move those numbers up.
Mr. Murphy. I recognize, Mr. Chairman, my time is up, so
perhaps the rest of the panel could submit the questions for
the record--their answers for the record. I would appreciate
that.
Mr. Pitts. Chair thanks the gentleman.
And now recognize the gentlelady from Illinois, Ms.
Schakowsky, 5 minutes for questions.
Ms. Schakowsky. Thank you, Mr. Chairman. I just want to say
I feel a sense of bipartisan mission here, some excitement that
we are standing on the brink of some very important
discoveries. It is a wonderful feeling that we seem to be in
agreement, and the--all the gratitude that has gone to the
leaders is certainly well deserved to bring us to this point.
I wanted to specifically follow up on a question on the--on
Representative Castor's line of questioning. And so I wanted to
ask you, Dr. Woodcock, given the efforts that FDA has already
taken to advance precision medicine, do you believe you need
additional authority from Congress? Do you need new authority
to pursue the goals laid out in the President's Precision
Medicine Initiative?
Dr. Woodcock. We don't believe we need new authorities for
precision medicine. Actually, diagnosis, you know, is the
foundation of medicine, and for hundreds of years doctors have
been getting diagnosis more and more precise. And the precision
medicine, we are really trying to use new molecular knowledge,
like gene knowledge, to get even more precise. But that is sort
of how drugs--drug regulation works. We figure out what patient
population could benefit, and then they are treated. And so we
have been doing this--we perceive a great groundswell of
activity, we hope--we all hope, over the next few years in
precision medicine, but it is an extension of the way drugs
have been used for a very long time, and we just hope to get a
lot better at it.
Ms. Schakowsky. So that is helpful. And as you know, there
is a new precision medicine section that is in this draft. I
believe it is intended definitely to further your efforts in
this area. Can you tell us if you think it will accomplish that
goal, this new section, recognizing that it may still need some
tweaking? I think we all want to be helpful here and don't want
to do anything that might be counterproductive.
Dr. Woodcock. OK. We look forward to working with the
committee on this. The version that was in yesterday was
changed from previously, and we need to take a close look at
that, and we really look forward to working with you on it.
Ms. Schakowsky. Very good. I wanted to--while we are all
forward-looking today, I think it may be helpful to just look
back on what happens a little bit when we don't adequately fund
NIH. I know that over--between 2003 and 2015, NIH actually lost
about 22 percent of its funding. So, Dr. Hudson, I know--I
remember Francis--Dr. Francis Collins talking about how we may
have been more advanced in Ebola research, for example, and
even some sort of vaccine had we had the funding to do it. I
wonder if there are other examples of things that maybe we can
do now that we couldn't do because of the lack of funding?
Dr. Hudson. I think probably one of the most devastating
effects of the budget constrictions over the last several years
has been the lack of appeal for careers in biomedical
research----
Ms. Schakowsky. Um-hum.
Dr. Hudson [continuing]. For young people. So as I go to
scientific meetings and conferences, and often with Dr.
Collins, we hear repeatedly the sort of chronic depression of
youngsters who are questioning whether or not it is worth
pursuing a career in biomedical research, and that is
particularly true for MDs or MD-PhDs who could instead be in
clinical practice where there is a more secure career
trajectory, rather than in biomedical research where the
success rate right now, and we hope now to see this rise, is 18
percent. And so people are spending a lot of time writing
grants and not getting them funded. I had a meal this weekend
with a girlfriend of mine who I went to graduate school with
who won a Nobel Prize, and she was talking to me about how she
has been really desolated by the budget cuts and by young
people now not being interested in coming to work in her lab to
pursue important research questions. So I think we are--we have
gone from a very--we are potentially going from a very dreary
phase in biomedical research to a much brighter phase, and for
that we are very grateful.
Ms. Schakowsky. I hope so. The--also start and stop in
terms of research funding makes it difficult, so I hope this is
the beginning of continued funding going forward.
Thank you so much. I yield back.
Mr. Pitts. Chair thanks the gentlelady.
Now recognize the gentleman from Texas, Dr. Burgess, 5
minutes for questions.
Mr. Burgess. Thank you, Mr. Chairman. And before I start, I
just want to underscore that the interoperability of electronic
health records is a top priority for me. And I know reading in
the press this morning that my bandwidth has been exhausted by
finally achieving success on the sustainable growth rate
formula, I just want to assure everyone that I have good minds
working in my office on this issue of interoperability, and it
will remain a top priority. I am, of course, relieved that
Chairman Pitts and Chairman Upton and Ranking Members Pallone
and Green also have made a similar commitment to this issue,
and it is my sincere hope to have this issue advanced by the
time we get this draft to markup.
So I have talked in the past about my own frustrations with
electronic health records, and here we are years later and I am
still hearing from doctors that electronic health records
failed to deliver on the promise. Patients seen in the
emergency room with chest pain, follows up with their
cardiologist, that doctor should be able to review the
patient's health information recorded by the hospital without
the patient having to request that it be faxed, without the
secondary doctor having to pay an exorbitant fee, without
having to agree to use the same electronic health record vendor
as the hospital, and yet many times that is the way our world
is working. And it is frustrating for doctors, and it is bad
for patients. Doctors and hospitals have invested time and
money to make this switch to electronic health records, and we
in this committee, under the Stimulus Bill and to some degree
under the Affordable Care Act, have invested 28 billion
taxpayer dollars to support this transition. Developments in
the technology have far outpaced the capabilities of the
systems. This is not a tech problem, this is a bureaucracy
problem, and we can fix it.
So, Dr. Hudson, let me ask you, if people were able to
seamlessly share their health information in electronic form
with the National Institute of Health, would it improve
researchers' ability to identify patterns in diseases?
Dr. Hudson. Yes.
Mr. Burgess. Thank you. Thank you for being succinct.
Another issue, and I am very committed to protecting First
Amendment rights of clinicians, to share and receive truthful
medical information. The current draft, in my opinion, must do
much more in this area.
So, Dr. Woodcock, given that approximately half of the
medicines prescribed to treat cancer patients in oncology
centers are used by physicians off-label, and over 60 percent
of pediatric prescriptions are off-label, wouldn't it benefit
patients if the manufacturers of these medicines could provide
physicians and payers with the most up-to-date truthful, non-
misleading information about drugs with no delay?
Dr. Woodcock. Well, there are multiple pathways, of course,
that clinicians can get information from manufacturers, they
can talk to them, there are scientific meetings, there are
publications, and so forth, and there are downsides to
establishing essentially a market for a drug before it has been
tested for a given indication. Now, for economic purposes, for
payers, formulary committees, we understand that a free flow of
information is needed, and we look forward to working on that.
Mr. Burgess. Right. There are First Amendment
considerations here, but it seems like the FDA should allow a
company to distribute to a physician the peer-reviewed New
England Journal of Medicine article, for example, that may have
been important in getting this product approved in the first
place.
And before my time has expired, I really do appreciate, Mr.
Chairman, you holding this hearing today and I appreciate our
witnesses being here. And I know it is a long hearing, and to
some degree, we are all somewhat longwinded and drawn out.
On the issue of precision medicine, on the issue of
personalized medicine, I do worry that some of the things that
have happened recently, within the last year and a half, have
kind of put the brakes on what should be happening in that
space, and specifically, I am referring to genomic information
which should--why is my genomic information that 23andMe has,
why is it locked up and why is it locked away from me now? Why
can I only get ancestral information from 23andMe? It is great
to know my mother was descended from Jesse James--I always
suspected that--but actually it would be more useful if I knew
whether or not I was at risk for multiple sclerosis, for
example. And on the concept of precision medicine, we have
dealt with laboratory-developed tests before. The ability of a
doctor to get a more precise diagnosis sometimes hinges upon
getting those laboratory-developed tests and not impeding their
development. And then finally, the whole concept of medical
apps. It is one that has exploded since really we have begun
having some of these hearings, and I very much look forward to
the day where medical apps, laboratory-developed tests, and
consumer-directed genomic information can help direct that
precision medicine.
Mr. Chairman, I will yield back.
Mr. Pitts. Chair thanks the gentleman.
And now recognize the gentleman from Oregon, Mr. Schrader,
5 minutes for questions.
Mr. Schrader. Thank you, Mr. Chairman.
Go back to maybe a little more basic questions, as a new
member of the committee and stuff. What--how does both FDA and
NIH prioritize the research, trying to juxtapose that research
that gives the biggest bang for the greater population at large
versus making sure that there are these opportunities for
subgroups and breakthrough populations, and will this be part
of your addressing this bill?
Dr. Hudson. So the way in which priorities are selected and
funding decisions are made is a combination of factors. First,
we want to fund only the very best, most meritorious science,
and that is determined through a process of peer review, which
is sort of the gold standard. But that is only one measure of--
one input for our funding decisions. Another is what are the
diseases and disorders that are most profoundly affecting our
population. And so that certainly weighs into our
considerations as well. What is our existing portfolio of
investments, and where are there potential gaps that we need to
fill. And then lastly, where are there specific scientific
opportunities. And sometimes that comes because there was a
breakthrough in another area that shined some light on another
unexpected area----
Mr. Schrader. Um-hum. Um-hum.
Dr. Hudson [continuing]. And then we need to chase after
that, and we need to do that with some alacrity. And so those
are really the 4 basic mechanisms. And we are able to go out to
the community and say we are interested in looking in these
specific categories of research. They are high priority to us,
come in with your best ideas. At the same time, leaving open
the door for people who have their own ideas of the next best
thing, that they can come to us with their great innovative
ideas, investigator-initiative research, often basic research
that is vital to our entire portfolio.
Mr. Schrader. FDA, same question.
Dr. Woodcock. Well, for the Center for Drugs, we have
really a miniscule research budget. We are not really a
research institution, all right, and we do testing--a lot of
testing, say, counterfeit drugs and things like that. We also
do applied research on matters that relate to regulating drugs,
like how would you establish that a biosimilar drug is
biosimilar.
Mr. Schrader. Um-hum.
Dr. Woodcock. And so we have to have scientists who
actually do that hands-on in the lab, so they are capable of
evaluating an application when it comes in.
Mr. Schrader. So both of you have strategic plans then to
address how you prioritize the testing and/or the things you
actually research.
If my office could get a copy of that just so we have some
idea of how to approach.
I guess the second question would be on the continuous
manufacturing opportunity. The question I have is, you know,
are there cost differences between that and the batch
manufacturing that has been traditional within the industry?
Dr. Woodcock. There is going to be sort of an entry cost
that will be high to switch over to this technology, and so we
expect that, say, generic manufacturers may not switch over for
quite a while because it needs to get established, the
equipment manufacturers need to have stable offerings, and so
forth. Once you get into continuous manufacturing, we would
expect it generally to be less expensive because it has a much
smaller footprint, much less waste, many fewer failures, and is
higher quality actually. So--but getting into it is a radical
departure from the way it is done now----
Mr. Schrader. Sure.
Dr. Woodcock [continuing]. And so will take investment.
Mr. Schrader. Would the, you know, would the pharmaceutical
companies and device manufacturers agree with that?
Dr. Woodcock. Well, I don't know that it is relevant to
devices so much, Jeff can speak to that, but yes, I think now
the innovator industry really understands the opportunity for
them----
Mr. Schrader. Sure.
Dr. Woodcock [continuing]. And so they are moving very
briskly into this area, whereas the generic industry, which
actually supplies most of the drugs that Americans take every
day, operates on smaller cost margins, their profit margins,
and so I think they will be slower to enter this area.
Mr. Schrader. Yes, I just wanted to make sure, you know,
the manufacturers in our country, by and large, do a very good
job. We have, I think, some of the safest drugs in the world,
and you and others make sure that that occurs, which I
appreciate. So I was just trying to get to the cost benefit
type of playback that would be there.
I guess the last question would be for our NIH folks, Dr.
Hudson. How do you work with pharmaceutical companies on the
antibiotic, antifungal research, make sure you are not
duplicating--many of them have huge R&D budgets, how do you
make sure you are not duplicating what they are doing?
Dr. Hudson. So there is a network of investigators who
specifically work on antibiotic research, and they are closely
coordinating and communicating with the private sector on where
our research investments are, and I would be happy to provide
additional information on that for the record.
Mr. Schrader. Great, thank you very much.
I yield back.
Mr. Pitts. Chair thanks the gentleman.
Now recognize the gentleman from New Jersey, Mr. Lance, 5
minutes for questions.
Mr. Lance. Thank you, Mr. Chairman. I would like to submit
for the record a letter from the chief executive officer of the
Parkinson's Action Network here in town regarding the
legislation, especially regarding the integrated electronic
health records with the Clinicaltrials.gov, and I would ask
that this be submitted for the record.
Voice. Without objection.
Mr. Lance. Without objection.
Mr. Pitts. Without objection, sure.
[The information appears at the conclusion of the hearing.]
Mr. Lance. Thank you.
I was pleased to see in the latest iteration of the
legislation a placeholder to incentivize and advance the
repurposing of drugs to address serious and life-threatening
diseases, and I have been working on this for quite some time.
I am glad that there is a bipartisan agreement that this issue
deserves our focus, and ultimately real policy solutions as
part of the larger legislation.
Dr. Collins alluded to some of the challenges in bringing
cures and treatments to patients during one of our many
roundtables last year, and I am deeply appreciative of that.
Dr. Collins noted specifically that this was a problem where
compounds failed to gain approval, but researchers later
discovered potential new uses for cures and treatments for
patients.
Director Hudson, can you give us a sense of how NIH has
encountered and observed some of these challenges through its
drug repurposing initiatives?
Dr. Hudson. I would be happy to, and thank you for the
question.
So at our newest center, the National Center for Advancing
Translational Sciences, one of the first programs that we
started in that program--in that institute, and I was honored
to be the deputy--acting deputy director there at its onset,
was a drug reuse program. And it is a wonderful partnership
between a number of pharmaceutical companies, ourselves, and
academic partners. And really, it is intended to take compounds
that have proven to be safe in humans, but have failed in
efficacy or have been abandoned for business reasons, economic
reasons. And companies have been willing to share those
compounds and provide them to us, and then they are offered up
for academic researchers to see whether or not those molecules
might actually be effective for a new use. And there was a
recent paper that was quite dramatic in which a drug that had
originally been developed by AstraZeneca for cancer, a
researcher at Yale was looking at the available compounds. He
had done some research on Alzheimer's and found that there was
a particular kinase that was activated in Alzheimer's. He saw
this kinase inhibitor that was available from AstraZeneca
through our program, got it, used it in mice, restored neuronal
synaptic activity, and restored some memory loss in these mice
models. And it has moved very briskly into clinical trials in
humans. So in 18 months, we have moved a compound that had
failed in cancer, into phase two studied in humans. It is a
pretty remarkable progress, and more programs like that would
be very beneficial. We need to make sure at the end of the day
that somebody is going to commercialize those. And so we look
forward to working with you on the specific provision in the
bill.
Mr. Lance. Thank you, and I hope that this is included in
the legislation that reaches the subcommittee, the committee
and on the floor of the House.
I would like to discuss briefly a different provision of
the legislation that I have been working on with my colleague,
Mr. Griffith, related to Clinicaltrials.gov. Last year, a
constituent of mine contacted me expressing his deep concern
and frustration with Clinicaltrials.gov. His young son had
recently passed away from brain cancer, and over the course of
his son's treatment, my constituent looked to
Clinicaltrials.gov in the hopes of finding a trial for his son.
Not only did the site lack a significant amount of information,
but it was confusing and ultimately unusable. The legislation
we have been working on aims to correct this by clarifying and
streamlining the information included in Clinicaltrials.gov,
and making the site an effective resource for both patients and
physicians. And it conforms to what others are already doing,
and I urge NIH to support this effort and make these meaningful
changes.
Dr. Hudson, in your testimony, you stated the scientific
community and the public expect data generated, that Federal
funds will be shared to enable further insights to be gained.
This is exactly why we are supporting these provisions, and why
I hope that this is in the legislation. Would you please
comment on your views on this?
Dr. Hudson. So thank you for your interest in
Clinicaltrials.gov. I have a particular passion about this
database and making sure that it is exceptionally useful to
patients and providers and to researchers. I have to say that
when I started getting engaged with Clinicaltrials.gov, I
learned that it was very difficult for researchers to try to
submit their trials into the database, it was difficult for
patients and families and providers to easily search the
database, and as a result of that, we have made specific
targeted investments to increase the usability of
Clinicaltrials.gov. We have a notice of proposed rulemaking, we
have gotten comments back, we will be finalizing those rules to
make sure that every single applicable clinical trial under the
regulation, and all NIH-funded clinical trials, are registered
and their data are submitted, and that the data is available.
There are some specific provisions in the draft where
data--structured data elements are suggested, where I think
they may be less than helpful at the end of the day. And we
would be interested in working with you to make sure that there
are ways in which people can get the information without
placing inordinate burdens on the researchers, and without
actually trying to box up information in ways that ultimately
it is less useful for being able to retrieve it. We have
sophisticated search functions, we can be able to provide this
information. I think we received the same letter that was sent
to you from your constituent, and we are going to do better.
Mr. Lance. Thank you. My time has expired. This is an
important issue and I hope to continue to work on it.
Thank you, Mr. Chairman.
Mr. Pitts. Chair thanks the gentleman.
Now recognize the gentlelady from California, Mrs. Capps, 5
minutes for questions.
Mrs. Capps. Thank you, Mr. Chairman. And thank you to all
our witnesses for your testimonies.
I am so pleased we are here discussing investments in
critical research and innovation, and want to commend the
committee staff who have worked so hard to improve the latest
draft of this bill.
Early on in my time in Congress, that was over 15 years
ago, I was very proud that we were able to work across the
aisle to nearly double the budget of the National Institutes of
Health. I think it was a high-water mark for this Congress. We
continually see how vital these Federal research dollars are to
medical innovation. NIH supports the best research in the
world, and has contributed to dramatically improving the lives
of so many Americans, but there still is much more to be done.
That is why it is so crucial that this bill provides an
increase of $10 billion for NIH research. It is important that
we provide the necessary support that NIH requires to continue
to be the gold standard in research and development. I have
always believed that supporting NIH is one of the smartest
investments that this Congress can make. As we all know, NIH is
driven by innovation, however, we still face significant
barriers in turning scientific knowledge into new therapies and
effective treatments.
Last Congress, the National Pediatric Research Network Act
was signed into law. This legislation was led by myself and
Congresswoman McMorris Rodgers, and it targeted the
difficulties in pediatric disease research, especially for
research on rare diseases. The low prevalence of these diseases
makes them particularly hard to research, but for those
affected, a new cure or treatment could mean a world of
difference.
So my first question, again, Dr. Hudson, I am kind of--we
are picking on you today. Can--could you talk briefly, I have
three questions for you, but first, how the National Pediatric
Research Network Consortia--Consortium described in the bill
might have an impact on the study of rare pediatric diseases or
birth defects?
Dr. Hudson. So there are a number of pediatric research
centers and networks that already exist, close to 100 different
research centers and networks, and those networks already
provide important infrastructure for being able to do critical
research on pediatric diseases, especially rare diseases. So we
have newborn research network, we have a number of networks
that are already in place. We look forward to building this new
network and making sure that it is complimentary to, and not
duplicative with, the existing research networks that we have
in place.
Mrs. Capps. Thank you. My colleagues have heard me talk
before about a family in my district with spinal muscular
atrophy, and you know these rare diseases affect not just the
person who is involved, but the entire family, and many times a
wider network of folks as well. That is why devoting resources
toward gaining better understanding of treatments of these
particular diseases is so crucial to entire communities. As NIH
takes on this critical research, we must ensure robust funding
for this important program. That is my pitch, myself and my
colleagues.
Another question for you. We know children also have unique
healthcare experiences. Treatment needs research challenges.
Children are not just little adults, and medical discoveries
that apply to adults don't necessarily apply to children. NIH
has had a policy in place for almost 20 years requiring that
children be included in NIH studies unless there is a good
reason not to do so. While I applaud this policy, I believe
that we can do a better job of not only tracking the number of
children in research, but also distinguishing between subgroups
like infants and teens where there are tremendous differences.
As many of you know, NIH tracks specific populations such as
the number of women and minorities who are enrolled in the
studies of funds, and this information is available on
Clinicaltrials.gov. But now my question is to you, Dr. Hudson.
I believe NIH should track the number of children it enrolls in
studies and their ages on these Web sites as well because there
are such major differences between them. Adding to this
Clinicaltrials.gov could achieve--adding this to
Clinicaltrials.gov could achieve the goal of more robust data
regarding children in NIH studies. Do you agree?
Dr. Hudson. So certainly, the inclusion of the ages that
are sought for inclusion within clinical trials----
Mrs. Capps. Right.
Dr. Hudson [continuing]. Is being included in the
registration information for Clinicaltrials.gov, and then when
the summary data is reported, the ages are also included in
that but in an aggregate form. I think we could also do more,
especially with new technologies, electronic technologies and
data technologies, to extract more information earlier in the
process so when we are looking at the grant applications, when
we are looking at the progress reports, that we would be able
to monitor in a more robust way the inclusion of children
before the study is already awarded and the trial is underway.
And so we look forward to working with you to make sure that we
are----
Mrs. Capps. Great.
Dr. Hudson [continuing]. Paying close attention, using all
the technologies that we have.
Mrs. Capps. And, Mr. Chairman, I realize my time is up, but
I have one more additional question to you, Dr. Hudson. Perhaps
I will submit it in writing. Thank you.
Mr. Pitts. Chair thanks the gentlelady.
Now recognize the gentleman from Virginia, Mr. Griffith, 5
minutes for questions.
Mr. Griffith. Thank you, Mr. Chairman. I would be happy to
yield a minute to the gentlelady if she has one more question.
Mrs. Capps. Well, that is really thoughtful of you. Thank
you very much.
The question--because it follows in a line with these
others, I wonder if you could describe how this data sharing
might increase our understanding of potential differences in
the way medical treatments affect women and minorities as well.
I mean, this kind of provision would help us, would it not,
better understand the effects of treatments on differing
populations and subsets? I hope NIH continues its work to
include more women and minorities in clinical research as well
as children, and look forward to working with you. But is it
just perhaps an extrapolation?
Dr. Hudson. And we are, in fact, looking forward to being
able to have these kinds of data so that we can draw
conclusions of data in sets rather than individually, to draw
important conclusions about disparities in health and health
outcomes----
Mrs. Capps. Great.
Dr. Hudson [continuing]. That would direct us for future
research. So we have the tools now to be able to deploy to
really ratchet up our attention to these issues.
Mrs. Capps. Thank you very much. And I yield back.
Mr. Griffith. Taking back my time. Let's stick with
Clinicaltrials.gov. You heard both the gentlelady before me and
Congressman Lance talking about some of the concerns from some
of the folks there, and I don't want to put words in your
mouth, but I gathered from some of the comments you made back
to Congressman Lance that you are not completely supportive of
Section 1102 that deals with making sure that there are certain
data points in there. How would you improve--we certainly want
to work with you on it, but we also--I feel very strongly, and
I know others do too, that we continue to improve this to make
it easier for patients and others to get the data they need.
What particularly do you have a problem with in 1102, and what
would you think that we needed to add to it?
Dr. Hudson. So there are a number of elements there that
the draft suggests be provided a structured data field, and
they are pretty straightforward and we can certainly do that.
We certainly have proposed that in the notice of proposed
rulemaking. We are currently evaluating the 800 or so comments
that came in in response to that, largely overwhelmingly
positive. So we are excited about that and getting a final rule
out, and we want to do that soon.
In terms of the elements where we have more concerns about
whether or not you can actually put it into a discreet category
really concerns the eligibility and exclusion criteria. For
clinical trials, often the inclusion and exclusion criteria are
complex and aren't easily definable into subunits, and so by
forcing investigators to put inclusion and exclusion criteria
into structured data elements may actually lose some of the
wealth of information that we would want to have available to
patients, providers, researchers, research reviewers, et
cetera. So that is really the area that we have the largest
concern, and we would be happy to sit down and talk to you in
more detail about that specific provision.
Mr. Griffith. Well, I certainly hope that we can work on
that because----
Dr. Hudson. Yes.
Mr. Griffith [continuing]. We don't want to exclude folks,
but we also want to make sure the data is out there, and right
now, as you have heard, there is a lot of concern about whether
or not the data is really out there.
Dr. Hudson. Yes.
Mr. Griffith. So we need to make sure it gets out there.
Dr. Hudson. Yes. We----
Mr. Griffith. Because that is one of the things we see as
very important with this, and with the next section in the
draft bill, which is 1121, the clinical trial data system. And
I believe the more that we can make that data available, the
more likely we are--obviously, you have to make sure that you
take away the personal identifiers, but there have been all
kinds of studies that say that we can do that.
Dr. Hudson. Yes.
Mr. Griffith. And I think that means that we are going to
find better ways to move forward.
Dr. Hudson. Yes.
Mr. Griffith. You were talking about a drug recently that
there had been a failure in in one area, but it worked
somewhere else.
Dr. Hudson. Yes.
Mr. Griffith. That is the kind of data, I think, if we can
enact this section, and again, it is a draft proposal, we can
tweak it, but if we can get this section drafted where we can
get that information out there to as many researchers as
possible and to as many people as possible, I think we are
going to be able to find, just like that researcher, and I have
forgot the university, was it----
Dr. Hudson. Yale.
Mr. Griffith. Yale. Who suddenly said, hey, I think this
will work over here, when it didn't work for cancer, it did
work perhaps----
Dr. Hudson. Yes.
Mr. Griffith [continuing]. For Alzheimer's. I think that is
the beauty of that particular section. I feel very strongly
about that section staying in this bill as it goes forward
because I believe that the more people who look at the data,
somebody is going to have an ah-ha moment, a eureka, and jump
out of the bathtub exclaiming that they have suddenly figured
out how to solve the problem.
Dr. Hudson. May I comment? So----
Mr. Griffith. Yes.
Dr. Hudson. So that provision specifically requires that
NIH or the Secretary contract to an outside entity----
Mr. Griffith. Um-hum.
Dr. Hudson [continuing]. Who would then collect patient-
level data from clinical trials that are supported by the NIH.
It is not clear to me, frankly, that having us contract with an
outside entity is the most effective way to get data available,
and we are already experimenting with a number of mechanisms of
making patient-level data available from specific programs
where, in the RFA, we say we want to do it and then we do it,
and we--there are different models that havej been tried by
different institutes. And I think we need to look carefully at
what we are learning from that experience to--before we sort of
jump into a statutory mandated requirement for all NIH clinical
trials. This is going to be a burden on our investigators, and
we have not yet established the value for all clinical trials,
as opposed to----
Mr. Griffith. What we want to try to do----
Dr. Hudson [continuing]. Particular subsets.
Mr. Griffith [continuing]. Is to ease the burden on
patients and ease the burden on those who are trying to find
cures for the patients' diseases. And I think it is important
that we move forward with the taxpayers' money to make sure
that as many people as possible can have access to that
information.
And my time is up, so I will yield back.
Mr. Pitts. Chair thanks the gentleman.
Now recognize the gentleman, Mr. Butterfield, 5 minutes for
questions.
Mr. Butterfield. Chairman Pitts, I thank you for holding
today's hearing on the most recent legislative draft of the
21st Century Cures Initiative. I certainly appreciate the hard
work of members, and particularly our staff. I look forward to
continuing to work with you and our colleagues to see that 21st
Century Cures meets and crosses the finish line.
I understand, Mr. Chairman, that our staffs have worked
beyond the call of duty, and I just wanted to personally thank
each one of them on both sides of the aisle.
By all accounts, Mr. Chairman, this has been a bipartisan
process. I have had the pleasure of working with my colleagues
on this committee, Congresswoman Renee Ellmers and Congressman
Gus Bilirakis, and even with Congressman Mike McCaul, who is
not on this committee but we all know him very well, on
advocating for our shared priorities that span political
parties. I am appreciative of the inclusion of some of my
priorities in today's draft, including Subtitle D on disposable
medical technologies. I must say, however, that I was very
disappointed to learn that H.R. 1537, the Advancing Hope Act,
was not included, nor was language that would achieve the same
goal. The Advancing Hope Act would permanently reauthorize the
Pediatric Priority Review Voucher Program, which has proven to
be tremendously successful. Since its introduction, I have
received overwhelming support from biopharmaceutical innovators
and over 140 patient groups and rare disease organizations who
have urged this committee in writing to include provisions in
this initiative that would make the Pediatric PRV Program
permanent.
And so I would ask unanimous consent, Mr. Chairman, that
these letters dated March 30 and April 13 be inserted in the
record.
Mr. Pitts. Without objection, so ordered.
[The information appears at the conclusion of the hearing.]
Mr. Butterfield. Mr. Chairman, the Pediatric PRV Program
addressed the market failures we have seen as rare pediatric
disease drugs have struggled to market by creating financial
incentives for rare pediatric disease drug development in the
form of vouchers. The PRV Program cost taxpayers absolutely
nothing--let me repeat: nothing--while at the same time helping
to speed treatments and potential cures to pediatric rare
disease patients who desperately need them.
So, Mr. Chairman, I hope that this committee will seriously
consider including legislative language that would make the
Pediatric PRV Program permanent in any subsequent 21st Century
Cures drafts. I respectfully make that request of you, Mr.
Chairman, and to all of my colleagues, and I look forward to
working with you to see that that happens.
I have several questions, Mr. Chairman. In the interest of
time and because I have an ambassador sitting in my office
waiting for me right now, I will submit my questions for the
record, if that would be acceptable.
Mr. Pitts. That is acceptable.
Mr. Butterfield. Thank you, Mr. Chairman. I yield back.
Mr. Pitts. Chair thanks the gentleman.
And now recognizes the gentleman, Mr. Bilirakis, 5 minutes
for questions.
Mr. Bilirakis. Thank you very much, Mr. Chairman. I
appreciate it. Thank you folks for your testimony this morning.
Dr. Woodcock and Dr. Shuren, anticipating more combination
products in the future, can you tell the committee what steps
FDA is taking to refine its current approach to facilitate the
development of these innovative combinations?
Dr. Woodcock. Well, we have a combination product office
that carries out the directions of Congress in trying to figure
out whether there is a drug lead or a device lead for products.
The device center and the drug center work very closely
together in working on these products, but I must say that the
statutes governing devices and the statutes governing drugs
were put in place a long time ago, and they didn't really
contemplate, I think, these new products, which are probably
part of the future of medicine. And so we are working very hard
to try and make these two statutes congruent.
Dr. Shuren. That is a place that does require probably
further discussion, and whether or not there are changes to be
thought about to make that intersection work better than it
currently does.
Mr. Bilirakis. We might have some suggestions for you, so I
would love to----
Dr. Shuren. We would be happy to have the conversation.
Mr. Bilirakis. Thank you.
Second question. During the 21st Century Cures roundtables,
we often heard about the cures gap, the enormous gulf between
approved therapies and known diseases, which leave many
patients with no treatment to turn to. Patients in the rare
disease community understand this challenge, where market
realities often make it more difficult to develop therapies for
diseases with smaller patient populations. I believe there is
great promise in repurposing drugs. In fact, earlier this year,
I introduced the Open Act with my colleague, Representative
Butterfield, who had to leave to see the ambassador. It would
foster research to increase the number of safe, effective, and
affordable rare disease medicines for patients by incentivizing
drug manufacturers to repurpose their approved products for
rare disease indications, by providing an additional 6 months
of market exclusivity when a product is repurposed and approved
by the FDA for the treatment of a rare disease. Ninety-five
percent of rare diseases have no FDA-approved treatments.
My first question is to Director Hudson, and of course, to
Dr. Woodcock. Can you comment on how repurposing already
approved drugs may hold therapeutic promise for rare disease
populations?
Dr. Hudson. So I think there are a number of examples where
drugs that were initially approved or pursued for one
indication have proven to be effective for other indications.
And in some cases, those have been rare and neglected diseases.
We appreciate very much your interest in this area, and really
look forward to working with you to come up with a provision
that would be appropriate for being able to actively pursue
this area where there is such opportunity to accelerate the
delivery of new medications for patients that really need them.
Mr. Bilirakis. Thank you. Dr. Woodcock?
Dr. Woodcock. Well, I think, in rare diseases, you need to
understand something about the disease, and then, of course,
having a range of therapies that you can try, and being able to
pick from those because you understand something about what
might work--which is the example Dr. Hudson just gave about
Alzheimer's. So obviously, there is a whole range of treatments
out there, and those that have not made it to the market would
expand that universe of things that could be tried. So I think
as disease understanding improves in rare diseases, there is an
opportunity to try many compounds.
Mr. Bilirakis. Thank you. My next question: What incentives
are currently available that encourage research into rare and
orphan applications in drugs that are already approved by the
FDA for a separate indication? We will start with Director
Hudson, and then Dr. Woodcock.
Dr. Hudson. So there are specific research programs at the
NIH, including the Office of Rare Diseases, the Therapeutics
for Rare and Neglected Diseases, there are a number of programs
that are specifically focused on supporting research for
diseases that affect a small number of people in the
population. And then in addition, and Dr. Woodcock can address
this, there are incentives and a poll from her end as well.
Dr. Woodcock. Yes, the Orphan Drug Act was a very
successful program that has brought many, many treatments to
rare diseases, and it includes incentives during the
development, as well as exclusivity provisions after a drug is
marketed for that indication.
Mr. Bilirakis. Thank you. Sir, would you like to comment as
well?
Dr. Shuren. So we have a program, the Humanitarian Device
Exemption, to facilitate and incentivize the development of
devices for rare disorders, and I actually want to compliment
the committee because there is a provision in this bill that
will now change the cap for HDEs, and I think potentially
provide greater incentives for device development in this area.
Mr. Bilirakis. Very good. Thank you very much.
And, Mr. Chairman, I will yield back. I do have another
question, but I will submit it for the record. Thank you.
Mr. Burgess [presiding]. Chair points out the gentleman's
time has expired.
The Chair would recognize the gentleman from New York, Mr.
Engel, 5 minutes for questions, please.
Mr. Engel. Thank you. Thank you very much, Mr. Chairman.
Throughout my time in Congress, I have been a very strong
advocate for those suffering from rare diseases. I authored the
ALS Registry Act and the two most recent Muscular Dystrophy Act
reauthorizations. I know the 21st Century Cures Initiative
holds great promise for the patients and families afflicted
with rare diseases if it is done well, and I am encouraged by
the progress made with the latest discussion draft, and hope
that continued refinements will lead to legislation that we can
all support.
Dr. Woodcock, one of the concepts I am pleased to see
included in the latest discussion draft is the section related
to biomarker development qualification. I know that the FDA
utilizes biomarkers often in making drug approval decisions,
but to date there is not, I believe, a formal process to put in
place to qualify biomarkers. So while I understand that FDA
approves many products based on surrogate endpoints, I have
also heard that the FDA has only qualified only a handful of
biomarkers. So could you explain how the FDA currently uses
biomarkers, and what the difference is between qualified
biomarkers and surrogate endpoints?
Dr. Woodcock. Sure, although it may take your whole 5
minutes.
Mr. Engel. That is OK.
Dr. Woodcock. Generally speaking, drug developers, during
their development program, can come into FDA under the user fee
agreements, and they can get agreement that is more or less
binding with the FDA on their pivotal trials. And those trials
might include a surrogate endpoint, which is not a clinical
measurement like do you feel better, but is your tumor stable,
all right, not--or it could include selection criteria which
might be by biomarkers. Do you have a certain tumor marker or
do you just have certain genetic mutation that would match with
this therapy. All right? And we can agree with that, but that
whole process is confidential. And that is how most of these
have gotten on the market, for rare diseases and regular
diseases, is the companies have gone through a process which is
confidential, we agree with their use of the biomarker, they
use it, and then the review process occurs.
To use biomarkers more generally, a number of years ago we
started a qualification process which was considered to be
different. It would be public. And there we would want everyone
to be able to use the biomarker, not just the company within
its development program. So those are different kind of
biomarkers usually, and the groups that have come into us are
consortia, patient groups, and so forth, because they are
looking, say, at safety biomarkers, something that an
individual company might not be interested in developing, but
this would apply to all drugs. For example, we are going
through qualification now for drug-induced kidney injury and
markers of that. It will be much better than the markers we
currently have if they are accepted.
So we have actually approved 12 separate biomarkers through
our qualification process, we have qualified those, but they
were in five different programs. So people say we had five
different biomarkers, but we have really had 12. All right? But
there are many more in the process. They are not under review
by us. We are giving them advice on how to develop these
biomarkers, and generate the evidence needed to make decisions
about human lives or human kidneys, or whatever. So we have a
robust qualification process going on right now. It is not in a
statute, it is something that we put out in guidance, and that
we manage. And the European Medicines Agency, we also worked
with them, and they have a parallel process. We often do this
qualification together.
Mr. Engel. Thank you. And you didn't take up the full 5
minutes, so I can get in one more question.
And let me ask this question for anybody who cares to
answer it. I am fully supportive of the goals behind the 21st
Century Cures Initiative, but I think that we really know it
won't be possible to achieve the ambitious goals set forth in
the discussion draft without providing adequate resources to
the FDA, CMS, and NIH. I didn't vote in support of the Budget
Control Act, but I know that all of our witnesses have faced
significant cuts to their budgets over the last several years
as a result of sequestration. And I know that our witnesses
have not had a lot of time to review the discussion draft
released yesterday, but can each of you, or whoever cares to do
this, share in broad terms what kind of staff and financial
resources you believe will be necessary to meet the
requirements outlined in this discussion draft?
Dr. Woodcock. We would be glad to get back to you on that.
I don't think we have had time to analyze this draft, but we do
feel it will have significant resource implications for the
FDA.
Mr. Engel. Do the others agree?
Dr. Hudson. So the discussion--the draft includes a
significant increase in funding for NIH, which we think we can
spend in effective ways, although we are concerned about other
agencies and making sure that, as we address resource issues,
that we also address resource issues for FDA and other agencies
across Government.
Mr. Burgess. All right----
Mr. Engel. All right.
Mr. Burgess [continuing]. Gentleman's time has expired.
The Chair recognizes the gentleman from Missouri, Mr. Long,
5 minutes for any questions please.
Mr. Long. Thank you, Mr. Chairman. And thank you all for
being here today in this important hearing.
And, Dr. Woodcock, does the FDA have a Twitter page and a
Facebook page?
Dr. Woodcock. I don't know whether the FDA does, but I know
that my staff does things on Twitter.
Mr. Long. It is my understanding that they do have a
Twitter page and a Facebook page, and when the FDA puts out
tweets about new drug approval, it is limited to 140
characters, so generally, they don't include the safety
information and warnings about a drug within the Tweet itself.
If you don't know they had one, I don't know how you can answer
this, I guess, but let's assume they do have one.
Dr. Woodcock. Well, generally, it's just a factual
statement about the drug approval and the indication.
Mr. Long. OK. So in a social media post, the agency does
not include the information in the body of the message which,
again, in Twitter is 140 characters, and instead notes the new
approval, and then provides the rest of the safety and
effectiveness information in a detailed link. So the question
that I have is, when regulating manufacturers' use of social
media, wouldn't a similar commonsense approach make sense to
let the manufacturers do the same thing?
Dr. Woodcock. Well, I think the reasoning that has been
pursued is that manufacturers have a different stake in
presenting the information than does the agency.
Mr. Long. A different what?
Dr. Woodcock. Stake.
Mr. Long. Stake?
Dr. Woodcock. Yes.
Mr. Long. OK.
Dr. Woodcock. In other words, that we are, you know, we are
presenting this information as a factual matter from a
Government agency that does not market the drug.
Mr. Long. So would it be unreasonable for a company to use
the name of the drug and have proved indication in a Tweet?
Dr. Woodcock. We have issued some draft guidance on this,
and I think we would be glad to get back to you. We are
currently re-evaluating our policies on regulation of drug
advertising in light of recent jurisprudence, and we would be
happy to discuss that further with you.
Mr. Long. But doesn't it benefit patients in discussions
with their doctors to know about new medical advances,
including the names of new drugs and their approved
indications? Wouldn't that be beneficial to the patients?
Dr. Woodcock. Yes, and there are multiple pathways for that
information to get out there now.
Mr. Long. OK, well, don't you think the FDA should
encourage this type of communication, rather than making it
more difficult, assuming that the information is accurate, to
be able to do the same thing that the FDA does as far as
getting out the information and linking to other things?
Dr. Woodcock. We can get back to you on what our current
guidance says about this on social media, and what we, you
know, and the----
Mr. Long. I know what your current guidance says, but I
would like to have your word that you will work with the
committee and work with my office as far as trying to put these
commonsense approaches into place, because I think that it is
beneficial to the patients and to the doctors. So I just would
like to have your word that you will look and work in that
direction, as I have been told off-the-record that the FDA will
be able to----
Dr. Woodcock. Yes, we will be happy to work with you on
this.
Mr. Long. OK, I appreciate that. And thank you all for
being here today.
And with that, Mr. Chairman, I yield back.
Mr. Burgess. Chair thanks the gentleman.
Chair recognizes the gentleman from New York, Mr. Collins,
5 minutes for your questions please.
Mr. Collins. Thank you, Mr. Chairman. This has been a great
hearing, and I want to thank Dr. Woodcock for taking the time
earlier this week to meet with me and talk about some issues,
and certainly my bill on the Bayesian statistical model for
adaptive trials, and I appreciate your support of that. I
think--this is the 21st century, not 1950, and I think that is
going to be good for all of us.
I was also very impressed with your knowledge and your
dedication to safely getting new drugs to market, and that is
what we are all about. But with all the novel and the
complicated issues that we are asking the FDA to analyze and
approve, I do worry that the FDA may not have the latitude and
the Government hiring process to hire the best and the
brightest minds in the field. Now, HHS currently works under a
cap on the number of senior biomedical researchers, that
applies to the NIH and the FDA, and also salary caps. Now, the
good news is the draft that we have now eliminates the cap on
senior biomedical researchers. It also substantially increases
the pay, I think it is to the level of pay up to that of the
President of the United States, which is substantially more
than we have now, and hopefully will make you competitive. But
I do worry that there are 2 other barriers and, Dr. Woodcock, I
would like you to maybe speak to those. The first one is the
hiring process itself, where these are unique individuals,
these are very high-paid individuals with very specific traits
that are necessary for you to do the job that we are asking you
to do, but yet, as I understand it, you are stuck in the
traditional hiring process. It can take you 9 months, you may
not even get the name of the person you want to hire on the
list. So if you could speak to that, and hopefully, what we can
do here is eliminate that and allow you to have, for these
levels of folks, the ability to hire the people you need. And
then the other one is the little nuanced issue of one of these
folks coming out of big pharm, Pfizer, something like that,
with stock, and that, while they are willing to put them in a
blind trust, which I am thinking is all we should ever ask,
that is not currently allowed in your hiring process, and that
could stop you from hiring someone. So if you could speak to
those two issues and, frankly, give us your recommendation how
we can still, in this draft, make changes.
Dr. Woodcock. Thank you. Yes, I am sure that Dr. Shuren has
this same challenge, and I know it occurs across the FDA. The
science right now is exploding, the new products are extremely
innovative. That is wonderful, but we need to have some good
scientists who can go toe-to-toe with the best in industry, and
industry can afford the best scientists. And we have great
difficulty hiring at that senior level. As you said, there have
been caps on the hiring authorities, there are caps on how much
we can pay the people, there are actually caps on how much we
can give them to promote them, that create tremendous
disparities internally in how people are paid, depending on
when they came into the Government. And we have extreme
difficulty hiring senior people who have worked outside the
Government because of their holdings, and the conflict of
interest rules, and we can't use blind trust for them to deal
with their stocks. So recently, I had someone who said, you
know, I really want to come, this was a very senior doctor, he
said I really passionately believe in the mission, but I can't
give up my family's future to do this, and I just can't do it.
And we have heard that again and again. So we have major
barriers to hiring senior people.
Dr. Shuren. I would add we have the exact same problem. I
have lost great people as a result. On the flipside, we have
great people at the center, but because I can't pay a
competitive salary, we essentially are the training ground for
industry. That is what the American taxpayer is paying for. And
so we train them, they are terrific, they leave, they take that
knowledge with them, and that disrupts our reviews, it makes it
much harder for us to have the good people, and ultimately it
hurts patients.
Mr. Collins. So, I mean, let's go back to the specifics. We
have addressed two of the issues in this draft, but I am
assuming you would like us to also get language in there that
allows you the discretion to hire the people you need without
going through the bureaucratic hiring practice, and number two,
allow these senior folks to put their holdings in a blind
trust, and therefore, be able to come to work for HHS. Is that
correct, those two would be very helpful?
Dr. Woodcock. Yes. I don't understand the rules about
financial arrangements well enough to know, you know, how that
would be done, but it is clear that it is a huge barrier right
now, and we can't get people who are experienced from all these
industries we regulate. And direct hire is a kind of authority
that is very helpful to us when we have it. We can just
identify people and bring them in. I mean, as you know, the
Federal hiring system is worried we are all going to hire our
relatives, but I don't have too many relatives who are PhD
neuropharmacologists, and so there are so many safeguards and
everything, we can't reach the people who we need. And that
would be tremendously helpful. I am not sure how that should be
done----
Mr. Collins. Well----
Dr. Woodcock [continuing]. But it would be helpful.
Mr. Collins. I think that is one of the things we can try
to work through as this draft moves along, and I thank you all
for your testimony today.
And I know my time has expired, but I still yield back.
Mr. Burgess. Gentleman's time has expired.
Chair now recognizes the eternally patient Ms. DeGette for
5 minutes for your questions, please.
Mr. Green. Mr. Chairman, before you let her time start, I
would like to say, Congresswoman DeGette, like Chairman Upton,
has worked so hard on this for the last year, I want to thank
her, but her patience was shown today, not only working on this
legislation but also sitting here. And by the way, former
Congresswoman Karen Thurman, who came in with me a few years
ago and--from Florida, has been here also very patiently, along
with a lot in our audience. Thank you, Diana.
Ms. DeGette. Thank you. Thank you very much. Well,
actually, I have a leg-up, having sat through this whole
hearing today because now I know what everybody thinks. That is
very useful as we move forward. And I kind of consider myself
to be the clean-up batter here at the end of this hearing.
Mr. Chairman, I really want to thank you and Mr. Pitts, and
I want to thank Mr. Green and Mr. Pallone again. Mostly, I want
to thank all of our staffs who have been really working night
and day. And as I said, the best time to work is really the
weekends because there are no distractions. So it has been
really great.
And, Dr. Hudson, Dr. Woodcock, and Dr. Shuren, you and your
staffs have just been tremendous in giving us technical
assistance. So that is the good news. The even better news from
my perspective is we are going to have a lot more work to do
here moving forward in the next few weeks, but I think the
amount of consensus that we have is striking and positive. We
still have a lot of those brackets in our discussion draft, and
a lot of that is just hammering out language that we still need
to agree on, but I am here to report that Chairman Upton is
planning subcommittee and full-committee markups soon. He wants
to keep the momentum of this bill going, and so we really are
going to have to redouble our efforts to get everything worked
out. We have to get it scored, we have to find the money to do
what we are going to do. I know a lot of people ask me, well,
how could we possibly spend the money, and I said, because we
need to. And I think that is the general view on both sides of
the aisle, it is the general view in the patient community, and
among the administration, and, lo, we are doing it here. We
still need to find a way to fund the FDA for the things that we
are asking you to do, and we know that. So we are going to do
all of that. We also, as we learned today, need to continue to
work with members on language for issues that they care deeply
about, and we are going to do that.
And so in these last few seconds that we have, I want to
ask the administration, aside from resources, which we know we
need to get you, what else do we need to consider that is not
in this discussion draft? Dr. Hudson, I will start with you.
Dr. Hudson. Well, first of all, congratulations on this
triumph really to get us to today, and the route ahead is
really exciting. Your--the--many of the issues that we wanted
to have included within this bill have been addressed. The
ability of the NIH director to require data sharing, for
example, the increased level of resources. There are a number
of the specific provisions that we really wanted to see into
the bill that are now here. There are a couple of places where
we have some concerns. I mentioned some of those with the--with
regard to individual patient-level data sharing mandates this
early in the process, but we are very happy with where this
bill stands----
Ms. DeGette. Great.
Dr. Hudson [continuing]. And I am not sure that we have any
outstanding--we--probably some technical--small technical
fixes, but nothing major that we are----
Ms. DeGette. Nothing that we have left out?
Dr. Hudson. No.
Ms. DeGette. If you think of something, let us know. And
keep----
Dr. Hudson. We absolutely will let you know.
Ms. DeGette. And, of course, we look forward to having your
input on those other issue.
Dr. Woodcock?
Dr. Woodcock. Well, one thing I think that I am somewhat
concerned about is that children with cancer--most childhood
cancers are very rare, and they are currently being left out of
the precision medicine, or whatever you want to call it,
targeted therapy revolution because the way we have looked at
pediatric disease is we have said there is a disease in adults,
and then there should be a disease in children. But, in fact,
in the targeted therapy, there is a pathway that is targeted in
adults, and then is there a pathway that is the same in
children. And I think we should think about that because there
is no current way to bring that about.
Ms. DeGette. And I will tell you, Dr. Woodcock, that is--
pediatric cancer, that is an issue we have really been talking
about. It is not in here because we haven't gotten to yet, and
so we need help getting to that.
Dr. Hudson?
Dr. Hudson. Just respond quickly.
Ms. DeGette. Yes.
Dr. Hudson. So in the Precision Medicine Initiative, there
is a cancer section, and in that cancer section there is adult
clinical trials and understanding resistance to oncology drugs,
and there is a pediatric section for that. And we would be
happy to have----
Ms. DeGette. So let's do some work on that.
Dr. Hudson. Yes.
Ms. DeGette. Thank you.
Dr. Hudson. Absolutely.
Ms. DeGette. Dr. Shuren?
Dr. Shuren. Well, I will just say on behalf of the agency,
you know, we just got the draft, we are going to go through it,
and we appreciate the opportunity and would like to put that
placeholder in of coming back if there are additional things
that----
Ms. DeGette. Yes, and that is why I said this is not just
for the agency, but also for others, if they have suggestions
of what they are not seeing in here, please bring them forward,
again, expeditiously, because we are moving on this.
And thank you again, Mr. Chairman.
Mr. Burgess. Gentlelady yields back.
Chair thanks the gentlelady, and again thanks her for her
patience.
I want to thank all of our witnesses today for your
testimony. It has been a long morning, but I think it has been
an important morning.
I do want to remind all members they have 10 business days
to submit questions for the record. And I ask the witnesses to
respond to the questions promptly. Members should submit their
questions by the close of business on Thursday, May 14.
Without objection, the subcommittee is adjourned.
[Whereupon, at 12:52 p.m., the subcommittee was adjourned.]
[Material submitted for inclusion in the record follows:]
[GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
[all]