[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]                           


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                    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.]
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