[House Hearing, 115 Congress]
[From the U.S. Government Publishing Office]




 
      21ST CENTURY CURES IMPLEMENTATION: UPDATES FROM FDA AND NIH

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

                                HEARING

                               BEFORE THE

                         SUBCOMMITTEE ON HEALTH

                                 OF THE

                    COMMITTEE ON ENERGY AND COMMERCE
                        HOUSE OF REPRESENTATIVES

                     ONE HUNDRED FIFTEENTH CONGRESS

                             SECOND SESSION

                               __________

                             JULY 25, 2018

                               __________

                           Serial No. 115-157
                           
                           
                           
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]                           
                           
                           
                           


      Printed for the use of the Committee on Energy and Commerce

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                    COMMITTEE ON ENERGY AND COMMERCE

                          GREG WALDEN, Oregon
                                 Chairman
JOE BARTON, Texas                    FRANK PALLONE, Jr., New Jersey
  Vice Chairman                        Ranking Member
FRED UPTON, Michigan                 BOBBY L. RUSH, Illinois
JOHN SHIMKUS, Illinois               ANNA G. ESHOO, California
MICHAEL C. BURGESS, Texas            ELIOT L. ENGEL, New York
MARSHA BLACKBURN, Tennessee          GENE GREEN, Texas
STEVE SCALISE, Louisiana             DIANA DeGETTE, Colorado
ROBERT E. LATTA, Ohio                MICHAEL F. DOYLE, Pennsylvania
CATHY McMORRIS RODGERS, Washington   JANICE D. SCHAKOWSKY, Illinois
GREGG HARPER, Mississippi            G.K. BUTTERFIELD, North Carolina
LEONARD LANCE, New Jersey            DORIS O. MATSUI, California
BRETT GUTHRIE, Kentucky              KATHY CASTOR, Florida
PETE OLSON, Texas                    JOHN P. SARBANES, Maryland
DAVID B. McKINLEY, West Virginia     JERRY McNERNEY, California
ADAM KINZINGER, Illinois             PETER WELCH, Vermont
H. MORGAN GRIFFITH, Virginia         BEN RAY LUJAN, New Mexico
GUS M. BILIRAKIS, Florida            PAUL TONKO, New York
BILL JOHNSON, Ohio                   YVETTE D. CLARKE, New York
BILLY LONG, Missouri                 DAVID LOEBSACK, Iowa
LARRY BUCSHON, Indiana               KURT SCHRADER, Oregon
BILL FLORES, Texas                   JOSEPH P. KENNEDY, III, 
SUSAN W. BROOKS, Indiana                 Massachusetts
MARKWAYNE MULLIN, Oklahoma           TONY CARDENAS, California
RICHARD HUDSON, North Carolina       RAUL RUIZ, California
CHRIS COLLINS, New York              SCOTT H. PETERS, California
KEVIN CRAMER, North Dakota           DEBBIE DINGELL, Michigan
TIM WALBERG, Michigan
MIMI WALTERS, California
RYAN A. COSTELLO, Pennsylvania
EARL L. ``BUDDY'' CARTER, Georgia
JEFF DUNCAN, South Carolina
                         Subcommittee on Health


                       MICHAEL C. BURGESS, Texas
                                 Chairman
BRETT GUTHRIE, Kentucky              GENE GREEN, Texas
  Vice Chairman                        Ranking Member
JOE BARTON, Texas                    ELIOT L. ENGEL, New York
FRED UPTON, Michigan                 JANICE D. SCHAKOWSKY, Illinois
JOHN SHIMKUS, Illinois               G.K. BUTTERFIELD, North Carolina
MARSHA BLACKBURN, Tennessee          DORIS O. MATSUI, California
ROBERT E. LATTA, Ohio                KATHY CASTOR, Florida
CATHY McMORRIS RODGERS, Washington   JOHN P. SARBANES, Maryland
LEONARD LANCE, New Jersey            BEN RAY LUJAN, New Mexico
H. MORGAN GRIFFITH, Virginia         KURT SCHRADER, Oregon
GUS M. BILIRAKIS, Florida            JOSEPH P. KENNEDY, III, 
BILLY LONG, Missouri                     Massachusetts
LARRY BUCSHON, Indiana               TONY CARDENAS, California
SUSAN W. BROOKS, Indiana             ANNA G. ESHOO, California
MARKWAYNE MULLIN, Oklahoma           DIANA DeGETTE, Colorado
RICHARD HUDSON, North Carolina       FRANK PALLONE, Jr., New Jersey (ex 
CHRIS COLLINS, New York                  officio)
EARL L. ``BUDDY'' CARTER, Georgia
GREG WALDEN, Oregon (ex officio)

  
                             C O N T E N T S

                              ----------                              
                                                                   Page
Hon. Michael C. Burgess, a Representative in Congress from the 
  State of Texas, opening statement..............................     1
    Prepared statement...........................................     3
Hon. Gene Green, a Representative in Congress from the State of 
  Texas, opening statement.......................................     4
    Prepared statement...........................................     5
Hon. Frank Pallone, Jr., a Representative in Congress from the 
  State of New Jersey, opening statement.........................     6
    Prepared statement...........................................     7

                               Witnesses

Francis Collins, Director, National Institutes of Health; 
  accompanied by Stephanie Devaney, Deputy Director, All Of Us 
  Research Program and Norman Sharpless, Director, National 
  Cancer Institute...............................................     8
    Prepared statement...........................................    11
Scott Gottlieb, Commissioner, Food And Drug Administration.......    29
    Prepared statement...........................................    31


      21ST CENTURY CURES IMPLEMENTATION: UPDATES FROM FDA AND NIH

                              ----------                              


                        WEDNESDAY, JULY 25, 2018

                  House of Representatives,
                            Subcommittee on Health,
                          Committee on Energy and Commerce,
                                                    Washington, DC.
    The subcommittee met, pursuant to call, at 9:04 a.m., in 
room 2123, Rayburn House Office Building, Hon. Michael Burgess, 
M.D. (chairman of the subcommittee) presiding.
    Present: Representatives Burgess, Guthrie, Barton, Upton, 
Blackburn, Latta, Lance, Griffith, Bilirakis, Long, Bucshon, 
Brooks, Mullin, Hudson, Collins, Carter, Walden (ex officio), 
Green, Schakowsky, Matsui, Castor, Sarbanes, Kennedy, Cardenas, 
Eshoo, DeGette, and Pallone (ex officio).
    Staff Present: Daniel Butler, Legislative Clerk, Health; 
Jordan Davis, Senior Advisor; Adam Fromm, Director of Outreach 
and Coalitions; Caleb Graff, Professional Staff Member, Health; 
Ed Kim, Policy Coordinator, Health; Ryan Long, Deputy Staff 
Director; Brannon Rains, Staff Assistant; Mark Ratner, Policy 
Coordinator; Kristen Shatynski, Professional Staff Member, 
Health; Danielle Steele, Counsel, Health; Austin Stonebraker, 
Press Assistant; Josh Trent, Chief Health Counsel, Health; 
Hamlin Wade, Special Advisor, External Affairs; Waverly Gordon, 
Minority Health Counsel; Tiffany Guarascio, Minority Deputy 
Staff Director and Chief Health Advisor; Samantha Satchell, 
Minority Senior Policy Analyst; Andrew Souvall, Minority 
Director of Communications, Outreach and Member Services; 
Kimberlee Trzeciak, Minority Senior Health Policy Advisor; and 
C.J. Young, Minority Press Secretary.

OPENING STATEMENT OF HON. MICHAEL C. BURGESS, A REPRESENTATIVE 
              IN CONGRESS FROM THE STATE OF TEXAS

    Mr. Burgess. The Subcommittee on Health will now come to 
order. I recognize myself 5 minutes for an opening statement.
    This morning, I certainly want to welcome our witnesses. We 
are here to conduct oversight and receive updates on the 
implementation on one of the most substantial legislative 
accomplishments in the space of biomedical innovation, the 21st 
Century Cures Act. Cures passed both the House and the Senate 
with wide bipartisan support. It was signed into law December, 
2016. As with all landmark laws, I think it is critical that 
the Congress, especially the relevant authorizing committee, 
engage in oversight to ensure that the agencies are 
implementing the law according with legislative intent, based 
upon the active leadership and robust activities of both the 
National Institutes of Health and the Food and Drug 
Administration.
    I look forward to hearing from Dr. Francis Collins, 
Director of National Institutes of Health, and Dr. Scott 
Gottlieb, the Commissioner of the Food and Drug Administration, 
regarding progress in implementing Cures. I thank our witnesses 
for their willingness to testify on such an important topic.
    The 21st Century Cures Act provides hope to those who need 
it the most, individuals and families suffering from life-
altering, often life-threatening illnesses, whether it is 
cancer, or a rare disease, or Alzheimer's. There are conditions 
that are costly to Americans of all ages and their families. 
Sadly, we each know too well the financial and the human toll 
that diseases place on our friends and our communities.
    One of the most impactful positions of the 21st Century 
Cures created the NIH Innovation Account in Treasury. This 
account funds projects like those related to Precision Medicine 
Initiative, the Brain Research Through Advancing Innovative 
Neurotechnologies Initiative, cancer research and regenerative 
medicine. The pace and breadth of biomedical research continues 
to accelerate, as we now have treatments to cure diseases, such 
as Hepatitis C, which was once unimaginable. Yet there is still 
much we do not know, especially regarding the neurodegenerative 
diseases.
    21st Century Cures included a provision to establish a 
National Neurological Conditions Surveillance System. Prior to 
Cures, there was no requirement or authorization to provide 
surveillance of neurologic disease, but this changed, thanks to 
Cures. Specifically, this section of law requires the Secretary 
of the Department of Health and Human Services to create such a 
system by expanding surveillance, infrastructure, and 
activities, including data collection, to determine prevalence, 
risk factors, and diagnostic and progression markers.
    Preliminary results from an ongoing Multiple Sclerosis 
Society study show that there are nearly 1 million Americans 
living with MS, more than twice the previously reported number. 
The surveillance system included in Cures will provide us 
better information so that we can further our understanding of, 
and eventually cure these diseases. I am especially grateful to 
see progress on this important policy. This began as a 
standalone bill introduced in the previous Congress.
    Additionally, Cures advanced precision medicine, which 
allows physicians to offer their patients truly personalized 
treatment. Achieving the full potential of precision medicine 
will require effort to collect health data in addition to the 
research done by our nation's best research investigators. The 
law codifies the Precision Medicine Initiative, and encourages 
the Secretary of Health and Human Services to carry out the 
goals of the initiative while ensuring confidentiality of the 
patient's information. The All of Us Research Program is a 
major piece of the Precision Medicine Initiative, and has 
already engaged over 1 million volunteers in the United States. 
I think you are to be congratulated for that.
    Clinical trials play a crucial and necessary role in the 
drug approval process. While the Food and Drug Administration's 
traditional clinical trial methods have proven successful, they 
are not always timely or applicable to new types of drugs. 
Cures requires the Food and Drug Administration to evaluate its 
trial designs and issue guidance for the purposes of 
``incorporating complex adaptive and other novel trial 
designs.''
    The innovation and promising results of efforts included in 
Cures will provide Americans suffering from cancer and other 
diseases with the opportunity to undergo successful treatments, 
and to, in some cases, be cured.
    So our thanks to Dr. Collins, Dr. Gottlieb, for giving us 
the updates on the implementation of this new law.
    I will yield the balance of my time to the gentlelady from 
Tennessee, Mrs. Blackburn, for a statement.
    [The prepared statement of Mr. Burgess follows:]

             Prepared statement of Hon. Michael C. Burgess

    Today, we are here to conduct oversight and receive updates 
on implementation of one of the most substantial legislative 
accomplishments in the space of biomedical innovation--the 21st 
Century Cures Act. Cures passed both the House and the Senate 
with wide bipartisan support and was signed into law in 
December of 2016. As with all landmark laws, I think it is 
critical that the Congress, especially the relevant authorizing 
Committee, engage in oversight to ensure that the agencies are 
implementing the law successfully.
    Based on the active leadership and robust activities of 
both the National Institutes of Health and the Food and Drug 
Administration, I look forward to hearing from Dr. Francis 
Collins, Director of the National Institutes of Health (NIH), 
and Dr. Scott Gottlieb, Commissioner of the Food and Drug 
Administration (FDA), regarding progress in implementing 
provisions of Cures. I thank both of our witnesses for their 
willingness to testify on such an important topic.
    The 21st Century Cures Act provides hope to those who need 
it the most--individuals and families suffering from life-
altering, often life-threatening illnesses. Whether it be 
cancer, a rare disease, or Alzheimer's, there are countless 
conditions that are costly to Americans of all ages and their 
families. Sadly, we each know too well the financial and human 
toll that diseases place on our friends and communities.
    One of the most impactful provisions in 21st Century Cures 
created the NIH Innovation Account in the Treasury. This 
account funds projects related to the Precision Medicine 
Initiative (PMI), the Brain Research through Advancing 
Innovative Neurotechnologies (BRAIN) Initiative, cancer 
research, and regenerative medicine. The pace and breadth of 
biomedical research continues to accelerate, as we now have 
treatments to cure diseases such as Hepatitis C, which was once 
unthinkable. Yet, there is still much we do not know, 
especially regarding neurodegenerative diseases.
    21st Century Cures also included a provision to establish a 
National Neurological Conditions Surveillance System. Prior to 
Cures, there was no requirement or authorization to provide 
surveillance of neurological diseases. But this has changed 
thanks to the Cures law.
    Specifically, this section of the law requires the 
Secretary of the Department of Health and Human Services to 
create such a system by expanding surveillance infrastructure 
and activities, including data collection to determine 
prevalence, risk factors, and diagnostic and progression 
markers. Preliminary results from an ongoing MS Society study 
show that there are nearly one million Americans living with 
MS, more than twice the previously reported number. The 
surveillance system included in Cures will provide us with 
better information so that we can further our understanding of, 
and eventually cure, these diseases. I am especially grateful 
to see progress on this important policy, as this part of the 
law began as a standalone bill that I introduced last Congress.
    Additionally, Cures advanced precision medicine, which 
allows physicians to offer their patients truly personalized 
treatment. Achieving the full potential of precision medicine 
will require immense efforts to collect health care data in 
addition to research done by our nation's best research 
investigators. This law codifies the Precision Medicine 
Initiative and encourages the Secretary of HHS to carry out the 
goals of the initiative while ensuring confidentiality of 
patients' information. The All of Us Research Program is a 
major piece of the PMI and has already engaged over 1 million 
volunteers in the U.S.
    Clinical trials play a crucial, and necessary, role in the 
drug approval process. While FDA's traditional clinical trial 
methods have proven successful, they are not always timely or 
applicable to new types of drugs. Cures requires the FDA to 
evaluate its trial designs and issue guidance for the purpose 
of ``incorporating complex adaptive and other novel trial 
designs.''
    The innovation and promising results of efforts included in 
Cures will certainly provide Americans suffering from cancer 
and other diseases with the opportunity to undergo successful 
treatments, and in some cases, to be cured.
    Thank you to Drs. Collins and Gottlieb for giving us 
updates on the implementation of this important law. I look 
forward to hearing your testimony.
    I yield the balance of my time to the gentlelady from 
Tennessee, Ms. Blackburn, for a statement.

    Mrs. Blackburn. Thank you, Mr. Chairman.
    And Dr. Collins and Dr. Gottlieb, we are delighted that you 
are here. Two things that I am going to want to discuss with 
you all. Dr. Collins, the All of Us Research project, and 
making certain that we anonymize, and that we protect the data 
that is in that program, and that the privacy of the patients 
with data is respected as we move forward with this.
    And, Dr. Gottlieb, following up on the SOFTWARE Act and 
making certain that the implementation is going well. And we 
welcome you both. We look forward to the hearing.
    I yield back.
    Mr. Burgess. The chair thanks the gentlelady. The chair 
yields back.
    The chair now recognizes the gentleman from Texas, the 
ranking member of the subcommittee, Mr. Green, 5 minutes for an 
opening statement, please.

   OPENING STATEMENT OF HON. GENE GREEN, A REPRESENTATIVE IN 
                CONGRESS FROM THE STATE OF TEXAS

    Mr. Green. Mr. Chairman, thank you for holding today's 
hearing on the Implementation of the 21st Century Cures Act. I 
want to thank all our witnesses for being here today, 
especially Dr. Collins from NIH and Dr. Gottlieb from FDA--you 
are pretty regulars at that table--for being here.
    This December will mark the 2-year anniversary of the 21st 
Century Cures Act being signed into law by President Obama in 
his last public signing ceremony. The promise of the 21st 
Century Cures is to advance the discovery and development of 
new treatments, cures, through increased research and improved 
drug approval process.
    This important law dedicated $6.3 billion in new 
investments to support medical research initiatives, like the 
Beau Biden Cancer Moonshot, the BRAIN Initiative, the Precision 
Medicine Initiative within the National Institutes of Health. 
It also provides money to the Food and Drug Administration to 
advance the agency's mission and implement the policies in the 
underlying bill.
    I hope, our committee and Congress, that these investments 
are being put toward finding a cure for many of our nation's 
greatest medical priorities and ensuring the infrastructure is 
in place so the new therapies are accessible to all Americans.
    The NIH was provided $4.8 billion in new funding and 
advanced cutting-edge research initiatives. The FDA was 
provided $500 million over 10 years to improve the agency's 
medical product review process and expedite patient access to 
drugs and devices without compromising standards of safety and 
effectiveness.
    In addition to this much-needed funding, there are many 
provisions in this package worthy of support, from facilitating 
the development of new antibiotics to fight against superbugs 
through advancing the use of modern clinical trial designs to 
investing in the next generation of medical researchers. While 
some of the provisions are technical in nature, the real-world 
impact could not be abstract.
    Patients and families deserve to have their elected 
officials respond to their needs. The 21st Century Cures was 
written to do just that. This morning is an opportunity to hear 
from the heads of the FDA and NIH on implementation of the many 
provisions of this law, from patient-focused drug development, 
medical device innovation, and improving scientific expertise 
and hiring capacity. Many members of our committee were 
instrumental in getting Cures developed and signed into law. 
Most notably, Representative Diana DeGette and Representative 
Fred Upton.
    The 21st Century Cures Act demonstrates what we can 
accomplish when we work across the aisle. I look forward to 
hearing from our witnesses about the oncoming implementation of 
21st Century Cures.
    Mr. Chairman, I yield the remainder of my time to my 
colleague, Congressman Diana DeGette.
    [The prepared statement of Mr. Green follows:]

                 Prepared statement of Hon. Gene Green

    Mr. Chairman, thank you for holding today's hearing on the 
implementation of the 21st Century Cures Act.
    I thank Dr. Collins and Dr. Gottlieb for being here this 
morning.
    This December will mark the 2 year anniversary of the 21st 
Century Cures Act being signed into law by President Obama in 
his last public signing ceremony.
    The promise of the 21st Century Cures Act is to advance the 
discovery and development of new treatments and cures through 
increased research and an improved drug approval process.
    This important law dedicated $6.3 billion in new 
investments to support medical research initiatives like the 
Beau Biden Cancer Moonshot, the BRAIN Initiative and the 
Precision Medicine Initiative within the National Institutes of 
Health (NIH).
    It also provides money to the Food and Drug Administration 
(FDA) to advance the agency's mission and implement the 
policies in the underlying bill.
    It is the hope of our committee and Congress that these 
investments are being put towards finding a cure for many of 
our nation's biggest medical priorities and ensuring the 
infrastructure is in place so new therapies are accessible to 
all Americans.
    The NIH was provided $4.8 billion in new funding to advance 
cutting-edge research initiatives.
    The FDA was provided $500 million over 10 years to improve 
the agency's medical product review process and expedite 
patient access to drugs and devices without compromising 
standards of safety and effectiveness.
    In addition to this much needed funding, there were many 
provisions in this package worthy of support: From facilitating 
the development of new antibiotics to fight against superbugs 
to advancing the use of modern clinical trial designs, to 
investing in the next generation of medical researchers.
    While some of the provisions are technical in nature, the 
real-world impact they could have is not abstract.
    Patients and families deserve to have their elected 
officials respond to their needs. The 21st Century Cures Act 
was written to do just that.
    This morning is an opportunity to hear from the heads of 
the FDA and the NIH on implementation of the many provisions of 
this law, from patient-focused drug development, medical device 
innovation, and improving scientific expertise and hiring 
capacity.
    Many Members of our committee were instrumental in getting 
Cures developed and signed into law, most notably 
Representatives Diana DeGette and Fred Upton.
    The 21st Century Cures Act demonstrates what we can 
accomplish when we work across the aisle.
    I look forward to hearing from our witnesses about the 
ongoing implementation of the 21st Century Cures Act.
    Thank you Mr. Chairman, and I yield the remainder of my 
time to Congresswoman DeGette.

    Ms. DeGette. Thank you so much, Mr. Chairman, or Mr. 
Ranking Member, for yielding.
    And I just want to add my thanks to everybody here, in 
particular, Dr. Collins and Dr. Gottlieb, and all of their 
staff who helped us develop this bill, most especially my 
compadre, Fred Upton, who is sitting over there, who really 
worked with us every day.
    But this really was a work product of this entire 
committee, and it shows the greatness of the Energy and 
Commerce Committee and what we can do when we decide to work 
together to tackle a serious problem.
    Mr. Chairman, I also want to thank you for having this 
series of hearings. It is about a year and a half since the 
bill has been signed, and it is almost exactly 2 years since we 
originally passed it through this committee. We need to make 
sure that everything we intended to do in Cures is happening, 
and if the agencies need modifications or changes or additional 
resources that we give that all due consideration. And the only 
way we can do that is to have hearings like this.
    I do want to say I have been concerned lately reading some 
media accounts that say that some of the Cures money may be 
reprogrammed for other purposes, including for the ORR issues 
of the kids at the border. We should be able to find the money 
to do that without taking money away from important biomedical 
research and drug and device approval at the FDA.
    So I hope that is not the case; and if it is, I hope this 
committee acts swiftly and in a bipartisan way to make sure 
that the intended moneys that we authorized in this committee 
remain, because we still have so many bridges to cross and we 
are going to need every penny that we authorized.
    With that, thank you very much, Mr. Green, for yielding, 
and I yield back.
    Mr. Green. Mr. Chairman, I yield back.
    Mr. Burgess. The chair thanks the gentleman. The gentleman 
yields back. Pending the arrival of the chairman of the full 
committee, the chair now recognizes the ranking member of the 
full committee, Mr. Pallone of New Jersey, 5 minutes for an 
opening statement, please.

OPENING STATEMENT OF HON. FRANK PALLONE, JR., A REPRESENTATIVE 
            IN CONGRESS FROM THE STATE OF NEW JERSEY

    Mr. Pallone. Thank you, Mr. Chairman. I want to thank Dr. 
Collins, Dr. Gottlieb, Dr. Sharpless, and Dr. Devaney for 
joining us to discuss the ongoing work at NIH and FDA to 
implement the 21st Century Cures Act. And, of course, I also 
want to thank our colleagues Diana DeGette and Fred Upton, 
without which the Cures Act would never have become law.
    The Cures Act tasks both of your agencies with implementing 
critical provisions aimed at improving the discovery and 
development of new treatments and cures. At NIH, the law 
provided significant funding for the Beau Biden Cancer Moonshot 
Initiative, the All of Us Research Program, and the BRAIN 
Initiative; and at FDA, the law included provisions to improve 
the medical product review process, as well as new authorities 
and funding to ensure the agency has the resources to recruit 
the best talent.
    At our hearing on this topic last November, you both 
provided promising updates, and I look forward to your 
continued work. As I said before, it is important to hold 
oversight hearings like these that allow us to learn directly 
from the administration how policies are being implemented.
    While I am pleased that the subcommittee has decided to 
conduct continued oversight of the Cures Act, there are several 
topics within the subcommittee's jurisdiction that also deserve 
hearings and oversight. For example, I have asked the majority 
to schedule hearings on a number of issues that are priorities 
for Democratic members of the committee. I have asked for 
hearings on maternal mortality, health disparities, gun 
violence, the Indian Health Service, cosmetics reform, the 
Office of Refugee Resettlement, drug pricing, abuse of the REMS 
program, and marketplace stabilization.
    These are all different issues within the subcommittee's 
jurisdiction, and we may have different opinions on many of 
them. That is exactly why we should at least have a hearing. 
And these are critical issues that this committee should be 
discussing in an effort to find potential solutions.
    So I hope in the coming weeks, after the August recess, 
that the chairman will respond and select a few of these issues 
to hold hearings on in the short time we have left for this 
Congress.
    And unless someone else wants my time, I yield back, Mr. 
Chairman.
    [The prepared statement of Mr. Pallone follows:]

             Prepared statement of Hon. Frank Pallone, Jr.

    I want to thank Dr. Collins, Dr. Gottlieb, Dr. Sharpless, 
and Dr. Devaney for all joining us to discuss the ongoing work 
at NIH and FDA to implement the 21st Century Cures Act.
    The Cures Act tasked both of your agencies with 
implementing critical provisions aimed at improving the 
discovery and development of new treatments and cures. At NIH 
the law provided significant funding for the Beau Biden Cancer 
Moonshot Initiative, the All of Us Research Program, and the 
BRAIN Initiative. And at FDA, the law included provisions to 
improve the medical product review process, as well as new 
authorities and funding to ensure the agency has the resources 
to recruit the best talent.
    At our hearing on this topic last November you both 
provided promising updates and I look forward to your continued 
work. As I've said before, it's important to hold oversight 
hearings like these that allow us to learn directly from the 
Administration how policies are being implemented.
    While I'm pleased that the Subcommittee has decided to 
conduct continued oversight of the Cures Act, there are several 
topics within this Subcommittee's jurisdiction that also 
deserve hearings and oversight. For example, I've asked the 
Majority to schedule hearings on a number of issues that are 
priorities for Democratic Members of the Committee. I've asked 
for hearings on maternal mortality, health disparities, gun 
violence, the Indian Health Service, cosmetics reform, the 
Office of Refugee Resettlement, drug pricing, abuse of the REMS 
program, and marketplace stabilization.
    These are all different issues within this Subcommittee's 
jurisdiction, and we may have different opinions on many of 
them, but that's exactly why we should at least have a hearing. 
These are critical issues that this Committee should be 
discussing in an effort to find potential solutions.
    I hope that in the coming weeks the Chairman will respond 
to my requests and select a few of these issues to hold 
hearings on in the short time we have left this Congress.
    I yield back.

    Mr. Burgess. The chair thanks the gentleman. The gentleman 
yields back. The chair will hold the time for the opening 
statement of the chairman of the full committee, pending his 
arrival.
    We thank our witnesses for being here today and taking time 
to testify before the subcommittee. I am going to give our 
witnesses an opportunity to give an opening statement. That 
will be followed by questions from members.
    Today, we are going to hear from the Honorable Francis 
Collins, the Director of the National Institutes of Health; and 
the Honorable Scott Gottlieb, Commissioner of the Food and Drug 
Administration. And Dr. Collins, it is my understanding you 
have brought a supporting cast of Dr. Norman Sharpless, the 
15th Director of the National Cancer Institute; and Dr. 
Stephanie Devaney, who is the Deputy Director of the All of Us 
Research Program. I don't know if it is the intention for all 
of you to give an opening statement, but we will start with you 
then, Dr. Collins, and you are recognized for 5 minutes for an 
opening statement, please.

    STATEMENTS OF HON. FRANCIS COLLINS, DIRECTOR, NATIONAL 
  INSTITUTES OF HEALTH, ACCOMPANIED BY DR. STEPHANIE DEVANEY, 
    DEPUTY DIRECTOR, ALL OF US RESEARCH PROGRAM, DR. NORMAN 
SHARPLESS, DIRECTOR, NATIONAL CANCER INSTITUTE; AND HON. SCOTT 
     GOTTLIEB, COMMISSIONER, FOOD AND DRUG ADMINISTRATION.

                STATEMENT OF DR. FRANCIS COLLINS

    Dr. Collins. Thank you and good morning, Chairman Burgess, 
Ranking Member Green, and other distinguished committee 
members. It is a great honor to appear before you again today, 
along with my colleague, FDA Commissioner Dr. Scott Gottlieb, 
to give you a progress report on our implementation of the 21st 
Century Cures Act. Also joining me, as you have just 
introduced, the head of the Cancer Institute, Ned Sharpless; 
and Deputy Director of the All of Us Program, Dr. Stephanie 
Devaney.
    I can't emphasize enough for this subcommittee how much we 
appreciate your bipartisan leadership in passing this Act--51 
to nothing, as I recall, out of Energy and Commerce--which aims 
to speed the translation of scientific discoveries into life-
saving treatments and cures. In the written statement I have 
submitted, I have outlined a comprehensive report on how NIH is 
working swiftly to implement the Act's provisions one by one.
    That most especially includes multiyear support for four 
specific areas of scientific opportunity supported by the Act's 
Innovation Fund. Today, I would like to highlight two of these: 
The Cancer Moonshot, and the bold new Precision Medicine 
Initiative, called All of Us.
    The Cancer Moonshot is aggressively pursuing a very 
ambitious goal to accelerate advances in cancer prevention, 
diagnosis, treatment and care. Such advances include 
immunotherapy, in which a person's own immune system is taught 
to recognize and attack cancer cells. After years of research 
supported by NIH, immunotherapy is leading to dramatic cures of 
some cancers, like leukemia, lymphoma, and melanoma. But some 
other cancers, particularly solid tumors, like colon, pancreas, 
breast and prostate, have proven much less responsive.
    Thank you. The slides are now up there.
    I am thrilled to tell you that some of those barriers seem 
ready to come down. Just last month, a team led by NIH's Dr. 
Steven Rosenberg announced a novel modification of an 
immunotherapy approach that led to a complete regression, most 
likely a cure, of widely metastatic breast cancer in a woman 
with previously, universally fatal form of the disease. As 
always, I have to counsel patients this success story for solid 
tumors, like breast cancer, involves very few cases right now, 
and must be replicated in further studies; but without doubt, 
this woman's life-saving experience represents hope for 
millions more.
    As exciting as potential cures like this can be, in 
authorizing and funding the Cancer Moonshot, you wisely tasked 
NIH with advancing not just cancer therapies, but also cancer 
care. Let me tell you about an NIH-funded trial that 
beautifully illustrates the progress we are making in this 
area. Each year, as many as 135,000 American women who have 
undergone surgery for the most common form of early stage 
breast cancer, face a very difficult decision, whether or not 
to also undergo chemotherapy to improve their odds. Now, thanks 
to a large NIH-funded clinical trial called TAILORx, we finally 
have some answers, and they are good answers. It turns out that 
about 70 percent of such women actually do not benefit from 
chemotherapy, and a genomic test of tumor tissue can identify 
them quite reliably.
    Clearly, it is ideal to spare women from the potential 
toxic side effects of chemotherapy, if that is possible, and 
still have a good outcome. On top of that, and this will 
probably warm your heart, because it certainly does mine, this 
move, basically making it not necessary to go through 
chemotherapy for many of those women, will produce a 
significant cost savings for our healthcare system, maybe up to 
$1 billion a year.
    Figuring out what health approaches work best for each 
individual and why brings me to the goal of another important 
investment of the Cures Innovation Fund, and that is, the 
Precision Medicine Initiative. The centerpiece of this 
initiative, the All of Us Research Program, will enroll 1 
million or more people, and we are off to a very strong start. 
On May 6, just 2 months ago, the day we launched national 
enrollment in seven sites across the Nation, we reached more 
than 10,000 people just that day at community events, and 
almost four times that number online.
    As of this week, over 86,000 volunteers have signed up to 
contribute their health data in many ways over many years. Some 
are enrolled through health provider organizations, ten of them 
that are part of our enterprise, and that includes community 
health centers and the Department of Veterans Affairs. Others 
enroll as direct volunteers, who sign up over the internet. 
Altogether, I am happy to tell you that almost half are from 
historically underrepresented racial and ethnic groups, which 
is one of our goals, so that we can utilize this to look at 
health disparities.
    Right now, anyone who is 18 and older, including Members of 
Congress--note the URL if you are interested in learning more 
about how to sign up--you can join. Next year, we will begin 
enrolling children. We decided to first start with adults, but 
next year, children will be added in. And in 2019, we plan to 
open a secure portal to give researchers access to All of Us 
data in a deidentified format with exceptional security.
    With every new person enrolled, every biological sample 
preserved, every electronic health record collected, every 
survey filled out, this data will hold more and more promise 
for advancing human health. And with every new scientist mining 
this data in search of answers to the important biomedical 
questions, the more that promise will be realized. This is 
groundbreaking.
    This exciting progress, along with many other advances in 
biomedical research, is being made possible because of the 
vision of you and your colleagues. So thank you for your 
investment in the 21st Century Cures Act, as well as your 
ongoing support of NIH. We could not do this without you.
    My colleagues and I really look forward to your questions. 
Thank you.
    [The prepared statement of Dr. Collins follows:]
    
    
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    Mr. Burgess. Thank you, Dr. Collins.
    Dr. Gottlieb, you are recognized for 5 minutes for an 
opening statement, please.

                STATEMENT OF DR. SCOTT GOTTLIEB

    Dr. Gottlieb. Thank you, Mr. Chairman, Mr. Ranking Member, 
and members of the subcommittee. Almost 2 years ago, the 
members of this committee hailed the passage of Cures as a 
potential game-changer for patients. I agree. I have provided a 
comprehensive list of our Cures activities in my written 
statement, but I would like to focus my remarks on one 
crosscutting priority under Cures, and that is modernizing 
clinical trials.
    FDA has embraced innovative trial designs and novel 
endpoints in the patient-centered trials envisioned by the 
Cures Act. Our aim is simple: Innovative, advanced, evidence 
generation, to assure the timely availability of safe and 
effective therapies. The Cures Act is catalyzing these 
approaches and catalyzing the development of new precision 
medical technologies that are enabling us to target, arrest, 
and cure intractable conditions. These advances aren't cheap. 
Access and cost is a big issue.
    And I know some question whether our market base system for 
medical innovation is financially sustainable. They ask if we 
can afford this coming wave of precision-guided therapies. I 
would say, we couldn't sustain our system without them. New 
advances, like regenerative medicine and gene therapy, can 
displace costs associated with serious illness by restoring 
function and reducing reliance on costly medical care delivered 
in hospitals and nursing homes. The best solution isn't to 
reduce market incentives for innovation, but to make it easier 
to bring new innovations and competition to the market, all 
without compromising one bit FDA's rigorous gold standard for 
product regulation.
    That brings me back to modernizing clinical trials. Rising 
trial costs and complexity undoubtedly impacts market 
competition and drug pricing. It can be a barrier to getting 
timely competition to newly approved branded innovative drugs. 
One reason we may be seeing higher costs is because it is 
taking longer for competition to emerge in some of these 
categories where specialty drugs are targeting unmet medical 
needs. We studied this question at FDA, and the data confirms 
these trends. I want to share a snapshot today of what we 
found. We plan to publish the full results really soon.
    For nonorphan drugs that treat conditions affecting larger 
patient populations, 41 percent of the first-in-class drugs 
approved between the years of 1991 and 2000 had at least one 
competitor in the same class within 5 years. This rate dropped 
sharply over the next decade. For the same kind of drugs 
approved between 2001 and 2010, only 18 percent had a within-
class competitor after 5 years.
    Another way of interpreting the data is to describe the lag 
in competition. For the drugs approved in 1991 to 2000, nearly 
a quarter had a competitor within 2 years. For the cohort of 
drugs where the first-in-class medicine was approved between 
2001 and 2010, it took an additional 5 years for there to be 
nearly as much competition; and by year 7, competition still 
lagged, with only 22 percent of the newer cohort of drugs 
having any competitor.
    We see similar patterns in most rare disease treatments as 
well. These trends mean that costlier branded drugs may enjoy 
longer periods without facing competition from products in the 
same class. And this may increase their pricing power. We need 
to understand why. Part of this has to do with the difficulty 
of running clinical trials with a second-in-market drug, 
especially if there is available therapy for a significant 
unmet need. It is becoming harder and harder to be second, and 
that is a problem.
    Efficient, modern approaches to designing and conducting 
trials can address some of these challenges and help us get 
more information about safety and effectiveness at the same 
time. To advance these and complementary goals, the FDA is 
pioneering a number of critical advances in clinical trial 
design.
    First, our master clinical trial protocols. These include 
basket trials, umbrella trials, and platform trials. These 
approaches can sharply increase trial efficiency and lower 
costs. They move away from one-drug/one-disease trials and 
allow the testing of multiple drugs against one or more 
diseases, or disease subtypes, using a common clinical trial 
infrastructure.
    Another approach is seamless trial designs that compress 
the traditional three phases of trials into one continuous 
trial. Through these approaches, you run one continuous trial; 
and as you enroll new patients, you expand subsequent cohorts 
of enrolled patients, using the information you learn about the 
features that help predict benefit from a new treatment.
    We are going to be publishing a guidance very soon that 
lays out how product developers can conduct these seamless 
trials and how to expand cohorts as trials progress, and a 
clinical criteria that can be used to expand cohorts as these 
trials advance. A lot of time and cost of clinical development 
is spent waiting in between the starting and stopping of the 
three phases of trials, and seamless trials can compress this.
    Every American has already, or will one day, face a serious 
medical diagnosis, either personally or through a loved one. We 
need to reduce the burden and the cost of advancing care. The 
clinical trial reforms we are making today will help ensure 
more patients who find themselves in these hard circumstances 
have a better chance of finding a cure, and that market 
competition helps make these treatments accessible to everyone. 
Thanks a lot.
    [The prepared statement of Dr. Gottlieb follows:]
    
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        Mr. Burgess. Thank you, Dr. Gottlieb.
    And, again, thanks to all of our witnesses for agreeing to 
be here. We will now proceed to the member question-and-answer 
part of the hearing, and we will still yield to Chairman Walden 
when he comes in for an opening statement. But let me recognize 
myself 5 minutes for questions.
    Dr. Collins, thanks for being here. Thanks for bringing 
your backup. So let me ask you a question. On the 21st Century 
Cures, we tried to identify ways to get regulations and 
policies that were inconsistent, and to give you some 
flexibility to move past some of these that are overlapping and 
unnecessarily duplicative to relieve some of the administrative 
burden. I think the Act asked you to review that.
    So can you, perhaps, share with us where you are in the 
review and how NIH has identified some opportunities to relieve 
the burden on investigators?
    Dr. Collins. Yes, I am happy to do so. And, again, thanks 
to the committee for making all of those changes part of this 
bill. Some of them may seem kind of bureaucratic and 
administrative, but they make a huge difference to us in terms 
of the ability to carry out our mission.
    We have been asked, for instance, to look at the way in 
which we have asked our grantees to deal with financial 
conflicts of interest, which we think are very important for us 
to track, but where the mechanisms for doing that tracking were 
seen as unduly onerous. And we are in the process of going 
through that with our colleagues at HHS, and I think there will 
be some changes that will make that a more efficient process.
    Another thing, again, that sounds pretty down in the weeds, 
but it mattered a lot to us, is the degree to which we need to 
do monitoring of what you would call a subrecipient, where you 
give a grant to a particular institution, and then they have a 
subpart of that to another individual. And in the past, our 
need to reach through and do very detailed monitoring, even of 
subrecipients, was something that we were required to do 
without a whole lot of reason for that. And so we have already 
moved to simplify that process.
    Financial expenditure reporting, a lot of the reports that 
have been done in the past produce data that not very many 
people look at, and yet at the same time, we need to be sure 
that we are being good stewards; and that is also being 
simplified.
    We are looking right now at animal care and use, and the 
oversight that is necessary, of course, to be sure that we are 
dealing with animals in an ethical way. But some of those 
particular oversight mechanisms are now being reviewed, and we 
put out an RFI and got 19,000 comments back from people who 
have opinions about how we might streamline this process.
    Those are a few of the examples. Again, it was really 
helpful having those features into 21st Century Cures to give 
us the authority to do those things.
    Mr. Burgess. And obviously, ongoing, it is going to be 
important for us to communicate on this, and if there are ways 
where we can provide additional legislative help on that, I 
think you would find the committee willing to have those 
discussions.
    Dr. Collins. Thank you.
    Mr. Burgess. Dr. Gottlieb, thank you for your comments on 
the clinical trial reform. And I always felt while we were 
doing the roundtables for Cures, that that is likely where the 
big money was. If we could reduce the time in trial, if a 
product was going to fail, allow it to be identified and fail 
early so we didn't spend a lot of time chasing something that 
was not going to pan out.
    So I really like the concept that you just elucidated about 
the seamless trial concept and condensing the time between 
Phase I, Phase II, and Phase III clinical trials. I think that 
is likely to have a significant impact.
    One of the things that has always concerned me is that we 
have had some legislation, there has been a little controversy, 
but the ability for all--we don't have a payer here, we don't 
have CMS, but for the researcher, the regulator, and the payer 
to communicate before something comes.
    I always got the impression that when Hepatitis C, when 
SOVALDI came down the pike, that the payers at the State level, 
certainly the Medicaid payers, were not ready for what was 
happening, but they had been prohibited from having those 
discussions because of current law.
    So I am hopeful that we are able to do something, because 
particularly in gene therapy or gene editing, rather, where you 
have one shot that is going to cure something that is a 
lifelong problem, that is huge, but it is likely to be priced 
out of the range of most average people's budgets.
    Can you comment on that, on our ability to communicate 
prior to approval?
    Dr. Gottlieb. Thank you, Mr. Chairman. I think we, I would 
say, fully addressed this issue. And your points are well-made, 
and I share your concerns and have shared your concerns in the 
past. I think we have fully addressed this issue with the 
guidance that we issued earlier this year. That was, in part, 
built off of a Cures provision related to payer communications 
with product developers for purposes of engaging in value-based 
contracting and other economic kinds of discussions, where we 
have essentially established a safe harbor for those kinds of 
communications where the FDA isn't opining on whether or not it 
has the legal authority to provide any regulation to that 
context, and some would argue we don't on First Amendment 
grounds. But even if we did, we would not choose to exercise 
that authority because, as a matter of public health, we 
believe there should be robust discussions between product 
developers and payers for the purposes that you suggest, so 
they can engage in value-based contracting, other novel ways to 
try to pay for these very novel therapies.
    Mr. Burgess. Great. Thank you. Thank you for that answer.
    I yield back my time and recognize Mr. Green, 5 minutes for 
questions, please.
    Mr. Green. Thank you, Mr. Chairman. And, again, I welcome 
our whole panel here.
    Let me give an example of what has happened in the last 4 
years. In 2014, we had an outbreak of Ebola in West Africa. 
There was no treatment for it. And today, what we are seeing in 
the Democratic Republic of Congo, we have a vaccine. What has 
happened in those 4 years? Now, Cures came on in 2016. But can 
Dr. Collins or Dr. Gottlieb share that? How did that become? I 
know Merck actually has the vaccine now, but how did we get 
there? Because back in 2014, there was no vaccine.
    Dr. Gottlieb. I will let Francis talk, because the vaccine 
came from him, and I will comment on where it is now.
    Mr. Green. It has to be approved.
    Dr. Gottlieb. Exactly.
    Dr. Collins. So NIH actually began working on an Ebola 
vaccine back in the 1990s, when nobody had really paid that 
much attention to this, but we were concerned that this might, 
at some point, become important. So there was already a 
fundamental amount of basic science and work toward a vaccine 
happening for a good 15 to 18 years before the crisis that 
struck in West Africa in 2014.
    That being the case, this was, in fact, a program that 
developed a vaccine in record time, but still not, in fact, for 
West Africa in time to have much of an impact on the actual 
outbreak, because by the time the vaccine was being 
distributed, good public health measures were already resulting 
in the epidemic waning rather quickly, which was a good thing.
    With the DRC, this vaccine, which is manufactured by Merck, 
but based upon a close collaboration with NIH--in fact, the 
Phase I trials for this very vaccine were done in the NIH 
Clinical Center up here in Bethesda, showing that it was, in 
fact, safe and seemed to generate good antibody titers. That 
was distributed in DRC in a ring vaccination strategy, and also 
giving them to healthcare workers.
    And we were very happy to see yesterday, the declaration 
that this epidemic is now over. Did the vaccine contribute to 
that? It is a little hard to tell, because, fortunately, this 
was a limited outbreak. DRC moved quickly with traditional 
measures. But I think, again, we are very well-situated now to 
deal with an Ebola outbreak in the future. In this instance, it 
was great to see how quickly the vaccine was available, got 
distributed, and was made available to those who needed it.
    Dr. Gottlieb. I will comment just to build off Dr. Collins' 
comments. I make two points up front: First, I think it is hard 
to understate what a game-changer this is. This is the first 
time that we now have a technology available in the setting of 
Ebola to intervene to stop the spread with something other than 
just traditional public health tools of isolation.
    I also think we should make note of the efforts of the 
manufacturer in this case, Merck; the doses that were deployed 
in the DRC were donated. They shipped about 13,000 doses to 
WHO. WHO handled the transport to the DRC. It was delivered 
under an expanded access protocol that was administered by the 
WHO. So this was very much an altruistic effort that helped in 
that setting.
    I will say that we are working efficiently to try to move 
toward a licensed product here in the U.S. Obviously, I need to 
be cautious what I say, but I feel optimistic that this will be 
something that we can accomplish in the near term, and we can 
have a fully licensed vaccine.
    Mr. Green. Well, and that is just an example, because we 
had a gentleman from West Africa come to Dallas, Texas, and the 
protocols weren't followed, and so it impacted our own country. 
But using this as a paradigm on what we can do for other 
terrible illnesses that are developing, and I tell people on 
any given day, we have tuberculosis in the city of Houston, 
because it is an international city, and you just don't check 
everybody. So we have to be on our toes to be able to do that.
    And the 21st Century Cures, I think, gives you some focus. 
But looking at the Ebola vaccine, in 4 years, we went from 
people worried it is going to get--and I was trying to explain 
to folks, more of my constituents will die of the flu because 
they didn't get a flu shot than will ever be exposed to Ebola. 
And we want to keep it that way, though.
    But I am concerned about the growing threat of the 
antibiotic resistance, and that is why I sponsored a provision 
in 21st Century Cures to create a pathway for these antibiotics 
that would meet unmet medical needs. The provisions directed 
FDA and CDC to coordinate efforts with respect to monitoring 
antibiotic resistance, and any other drugs approved under the 
limited population pathway for antibiotic and antifungal drugs.
    Dr. Gottlieb, what steps has the FDA taken to coordinate 
with the CDC to support policy that promotes judicious 
antibiotic use and antibiotic stewardship?
    Dr. Gottlieb. We have taken a number of steps. We are going 
to be taking some additional steps to look at antibiotic use in 
animal feed, the length, the duration of use and the 
indications in which they are used, and plan to have some 
additional policy steps that we should be announcing within the 
next couple of months to continue to advance what we have 
already done in that regard to reduce the use of antibiotics in 
animal feed and limit one route by which we are seeing the 
resistance develop.
    And as well, with both the provisions in FDASIA, the QIDP, 
Qualified Infectious Disease Product provisions, as well as the 
LPAD that was also enabled in legislation passed by this 
committee, we have been able to create new pathways to try to 
provide additional incentives and additional efficient pathways 
to get new drugs to the market that attack some of these 
multidrug resistant pathogens.
    I would just close by saying I think it is still important 
that we focus on trying to develop new pull incentives and new 
ways to potentially reimburse some of these limited-use anti-
infectives as a way to create additional incentives for the 
development of these products.
    And we are working on one such idea in conjunction with our 
colleagues at CMS to try to move towards a different 
reimbursement paradigm for drugs that are the kinds that you 
want to just put on the shelf and never have to use, moving 
toward a site licensing model as opposed to a pay-per-use 
model, which has been the traditional way that we have paid for 
drugs.
    Mr. Green. Thank you, Mr. Chairman. I know I am way over 
time and I will yield back the time I don't have.
    Mr. Burgess. The chair thanks the gentleman.
    The chair recognizes the gentleman from Kentucky, Mr. 
Guthrie, the vice chairman of the subcommittee, 5 minutes for 
questions please.
    Mr. Guthrie. Thank you, Mr. Chairman.
    I have a couple of questions, but first I want to say I 
know there is a lot going on in this great city and this great 
country moving forward. And a lot of times what is happening in 
this hearing--a lot of hearings make television. This one 
probably doesn't make a lot of prime time television, but it is 
important. It is extremely important what happened when a bill 
like the 21st Century Cures passed, I think got a voice vote 
out of this committee, overwhelmingly bipartisan, and really 
makes a difference in people's lives.
    And, Dr. Collins, you and I had a meeting the other day 
with a member of the band, U2. I only point that out because he 
was talking about somebody that was close to him that had had 
childhood cancer. And I remember walking away, because I had a 
friend of mine that passed away when I was about 11 or 12, and 
she would be alive today if she had the same disease this time, 
or probably would be alive today. And the reason I point out 
U2, because I think they, being Irish, really love this country 
and they really point out why America inspires the world and 
does things throughout the world.
    And people throughout the world do research, but nobody 
compares to what you are doing at NIH, what we are doing as a 
country, what our healthcare system, what our industry, public-
private partnerships. And it is just a shame that--the old 
Annie Murray song, We Could Use a Little Good News Today. There 
is a lot of good news, and a lot of it is happening in what 
people and this panel is doing. We really appreciate it very 
much.
    But I do have a couple questions for Dr. Gottlieb. One is 
specific on the Cures Act. It codified many practices in the 
Office of Combination Products, but also included provisions to 
clarify regulatory requirements, improve processes. Can you 
explain how the FDA is delivering on these efforts in the 
complex area of regulation?
    Dr. Gottlieb. We have put forward a number of additional 
guidances as well as staff manual guidances on how we approach 
combination products in the agency. Historically, it has been 
challenging for the agency. I think we have made a lot of 
progress in recent years. I think we have made a lot of 
progress owing to some of the provisions that flow out of the 
Cures Act as well.
    We set up a Combinations Council to try to adjudicate who 
has primary jurisdiction over these products, and we will be 
putting out a guidance sometime probably this fall, end of the 
summer, early fall, that is going to make some further process 
reforms that I think is going to make it more efficient for 
products that sit on that cusp to move into the device realm, 
which I think is the preferred route for a lot of product 
developers, if they can get there.
    So we are going to look for ways to find the most efficient 
route while being mindful of our public health obligations in 
these circumstances.
    Mr. Guthrie. Well, thank you. And also, you and I talked 
just recently on the telephone, and several of us here sent a 
letter concerning drug shortages, drug supply shortages. I know 
I have had an emergency room physician from my district say 
that sometimes they don't have just the simple things they 
need. They have to be more creative. They have to figure out 
other things to move forward.
    I know some of it was tied to the natural disaster in 
Puerto Rico. I think you guys really went in and handled it. 
That is what people need to know. There are people in 
government that are trying to make things work. We have issues 
we need to address. But what happened in Puerto Rico to get 
pharmaceuticals moving again, what you guys have done. But I 
know there are still issues with drug shortages. I hear from 
EMS, emergency room physicians, anesthesiologists, just basic 
things.
    Could you just talk to me about a minute and a half what 
you explained to me on the phone and what you are doing with 
your Drug Task Force and what is pressing and what is moving 
forward?
    Dr. Gottlieb. So the challenges here are structural in my 
view, Congressman. If it is not one thing in shortage today, it 
is going to be another thing tomorrow. And we are dealing 
specifically with sterile parenteral drugs, the sterile 
injectable drugs, and those are the ones that seem to be 
chronically in shortage.
    I think there are structural problems in that. Frankly, 
reimbursement has been driven down so low. Many of these drugs 
are manufactured slightly above cost of goods. So the only way 
to profitably manufacture them is to do it at tremendous scale. 
So we have seen a lot of consolidation in the space, but we 
have also seen underinvestment in manufacturing, because there 
aren't a lot of margins to reinvest in manufacturing.
    And I will say manufacturing these isn't trivial. It is one 
thing to manufacture a small-molecule drug and have a margin 
that is slightly above cost of goods. It is quite different 
when you are trying to manufacture a sterile, injectable drug. 
So things go wrong, and when things do go wrong, since this 
space is consolidated, if one facility gets shuttered it could 
take down 30, 40 percent of the market.
    We think there are things we can do, apply an additional 
regulatory touch to some of these critical drugs, if we could 
define sort of a category of what we would say are critical 
access drugs that we don't want to go into shortage. But, quite 
frankly, I am concerned that my regulatory touch is only going 
to exacerbate the problem insofar as it will increase cost.
    So what we are trying to do with our shortages task force 
is look at this holistically, and bring in our colleagues from 
the VA and CMS and look at how we might couple some additional 
regulatory steps that we could take to mitigate shortages, or 
try to prevent them with some potential changes in the 
reimbursement structure for certain of these drugs.
    We talk a lot about drugs that are priced too high. And 
there are drugs that are very costly, and probably exceed the 
value that they are delivering. But there are drugs also that 
probably are priced too low relative to their importance and 
the cost of manufacturing them. And I think we need to take 
another look at how we reimburse these old sterile, parenteral 
generic drugs, off-patent drugs.
    Mr. Guthrie. Well, thank you very much. And my time is 
expired and I yield back. Thank you all for being here.
    Mr. Burgess. The chair thanks the gentleman. The gentleman 
yields back.
    The chair recognizes the gentleman from New Jersey, Mr. 
Pallone, the ranking member of the full committee, 5 minutes 
for questions, please.
    Mr. Pallone. Thank you, Mr. Chairman.
    First, I have a question for Dr. Gottlieb, and then I want 
to go to Dr. Collins for a second one.
    Last week, FDA announced the release of its biosimilar 
action plan, which strives to encourage more innovation and 
competition in the biologics market. And I believe such action 
is critical and necessary if patients are to realize the full 
benefits of biosimilars. While 8 years have passed since the 
Biologics Price Competition and Innovation Act, only three 
biosimilars are marketed in the U.S., despite FDA having 
approved 11 of them. So as you noted yourself, Dr. Gottlieb, 
competition in this space is anemic.
    So I have two questions: First, you are well-aware that the 
high cost of prescription drugs, including biologics, continues 
to be a barrier for many patients. As a part of the Biosimilars 
Action Plan release, you revealed that a recent FDA analysis 
that found that if all FDA-approved biosimilars were available 
to American consumers, that significant savings could be 
realized.
    Could you discuss further FDA's analysis and the savings 
potential that biosimilars could offer?
    Dr. Gottlieb. I appreciate the question. We are going to be 
publishing the full results of that analysis soon, but 
essentially what we did is, we looked at the experience in the 
European market where we saw product introductions, and 
extrapolated that experience to what could have happened in the 
U.S. market if the same product had been introduced in the U.S. 
market.
    So we effectively took the competitive landscape from 
Europe, made some corrections for the fact that the dynamic in 
the European market is slightly different than the dynamic in 
the U.S. market, but looked at the percentage price reductions 
when one, two, three, four biosimilars entered the market. And 
when we extrapolated those findings back to the U.S. market, 
and then assumed that if every biosimilar that was approved in 
the U.S. market had launched, we extracted that an additional 
$4.5 billion would have been saved in 2017.
    So the savings are quite significant, and I think, if 
anything, we probably erred on the side of underestimating them 
by being very conservative in how we did our analysis.
    When you look at the European experience, the savings 
aren't of the same magnitude that you see in the small molecule 
world, but they are quite significant. They start to approach 
the small molecule type of savings once you see four or five 
product introductions.
    Mr. Pallone. Thanks a lot, really.
    The second question, I always want to commend you for your 
continued dedication to curbing gaming tactics used by certain 
manufacturers to delay or impede competition, but a question 
is, how can Congress work with the FDA as you implement the 
Biosimilars Action Plan to help facilitate greater competition 
in the biosimilar space?
    Dr. Gottlieb. I appreciate the question, Congressman. Yes, 
we are going to look at some of these more difficult scientific 
questions that I think could facilitate more competition, like 
interchangeability.
    But one of them, in particular, that we are going to be 
looking very hard at is being able to use the European product 
as a reference standard. We have situations where a biosimilar 
might be manufactured in the same facility and distributed in 
both the U.S. and Europe. We know that, but our knowledge of 
that constitutes commercial confidential information. So we 
have to require a biosimilar entrant to run the study against 
the U.S.-referenced product, even though the European product 
is the same thing, and the European product might be cheaper 
and easier for them to source.
    So that there are opportunities, I think, to have cost 
savings if you could source a reference product globally. And I 
will add that about half the cost right now of developing a 
biosimilar--it is highly variable, but, on average, about half 
the cost of developing a biosimilar are the costs of acquiring 
the branded biologic to run the trial, to run the comparative 
trial. So the savings could be significant.
    Mr. Pallone. Thank you.
    Let me quickly get to Dr. Collins. You mention in your 
testimony that NIH is currently building the All of Us data 
resource, which ultimately help researchers study the data 
collected from participants and connect to other large 
datasets. Making this data available to a broad range of 
researchers obviously has pros and cons, and the two major 
issues that come to mind are data security and participant 
privacy.
    So let me quickly put two questions into one. Given that 
NIH is asking participants to share intimate details about 
their health and lifestyle, how do you plan to ensure data 
privacy and data security, and are there protocols in place to 
share updates or study results with participants?
    Dr. Collins. I am going to ask Dr. Devaney, the Deputy 
Director, who is all over these issues, to respond to your 
question.
    Mr. Pallone. Thank you.
    Ms. Devaney. Thank you for the question and thank you for 
having us here today.
    So we are thrilled about where we have come with the All of 
Us Research Program. We have over 86,000 participants. Of 
course, we know, as we try to engage a community of diverse 
participants, up to a million, or maybe even more, we need to 
gain their trust. And so privacy is one of our most important 
priorities.
    All of the data that comes into the database source is 
encrypted and deidentified, and goes into a secure cloud 
environment. And once we open up access to researchers next 
year, they will have to abide by a code of conduct, and no data 
will be allowed to be downloaded from that environment.
    In addition, and just really briefly, I want to thank the 
committee here for giving us really strong privacy protections 
within the Cures Act, including certificates of 
confidentiality, which cover all of our data and allow us to 
really provide robust protections to our participants' 
confidentiality.
    Mr. Pallone. Thank you. Thank you, Mr. Chairman.
    Mr. Burgess. The chair thanks the gentleman. The gentleman 
yields back.
    The chair recognizes the gentleman from Michigan, Mr. 
Upton, the author of the Cures legislation, 5 minutes for 
questions, please.
    Mr. Upton. Well, thank you, Mr. Chairman.
    I just want to remind everyone here that this was a great 
committee process that passed 51 to nothing. And I want to say 
that everybody that was on the committee in the last Congress 
actually had a piece of this, because we listened to everybody 
here. Staff was terrific, that is for sure, but so were the 
agencies, because you helped lead us to where we wanted to go. 
The administration. The Senate. We had all the disease groups. 
We had lots of players, and we appreciated that input.
    And I think, based on the hearing today and what we really 
thought was going to happen, it has fulfilled our expectations, 
and we have got a lot of time left to play. It is a game-
changer. And I got to say, way back when, I was one of the 
participants with John McCain and Paul Wellstone and Henry 
Waxman, who doubled the money for the NIH back in the 1990s. 
The four of us were the sponsors of that bill to get it done. 
And it was a very important element that we actually increased 
the money for the NIH, knowing that we had a terrific steward 
in Dr. Collins, who was going to be able to lead us there.
    And I can remember sitting upstairs with Mr. Pallone and 
Ms. DeGette, my partner, and a few others to talk about how 
much money we wanted to add to that baseline. And we got it up 
to a $45 billion increase over a 10-year span. And the 
leadership demanded that we have pay-fors for it, and we came 
up with those not once, but twice, as they were stolen the 
first time.
    And a comment that was made earlier, and it might be more 
important to have maybe someone from HHS here, but I was too 
alarmed last week when I read a story that HHS was perhaps 
reprogramming some of the money that we had done a dollar-for-
dollar, year-by-year table to make sure that that money went 
for the NIH.
    And I don't know, Dr. Collins, if you have knowledge if 
there is some truth to the published reports that money was 
being taken away from what we were able to do and put to 
something else. I don't know if that is happening. And if it is 
happening, how do we get it back?
    Dr. Collins. So I am not aware of the published report you 
are mentioning. There is, I believe--and we should probably 
check this--language in 21st Century Cures that prohibits 
transfer of the funds that are allocated in the Innovation Fund 
by the Secretary for any other purposes. So I think this is 
nicely designed so that those funds are intended to go for your 
original plan.
    Mr. Upton. That is good to hear. I want to talk a little 
bit about All of Us. And I think privacy protections are 
important, and it is very important people know about them and 
that they don't have to worry about that data being stolen. 
And, as we found, particularly from our roundtables, 
individuals were more than willing to share what their own 
experience was, knowing that it was going to protect someone 
else from maybe having that same ailment.
    So it is exciting to hear that 87,000 people have already 
signed up, really, just in a couple weeks. I know for me, 
particularly with the privacy protections, we would like to 
maybe set up a caucus here, and All of Us caucus that can be 
bipartisan to encourage members and staff to participate. We 
have got about, oh, like 25,000 people that work here on the 
Hill. Maybe we can get a good percentage that will come down to 
the Gold Room around the corner to figure that out.
    I know that researchers are going to have access to this 
information in about a year or so. How quickly do you think 
some of that data might be able to be utilized and figure out 
the right pathway for some real important research to be 
promising?
    Obviously, the news in the last couple weeks about the 
chemo, and not having chemo for breast cancer was very 
important, heralded around the country nicely. But how fast do 
we expect that maybe some of this All of Us information might 
be able to be used by the researchers with some concrete result 
of positive?
    Dr. Collins. Ms. Devaney.
    Ms. Devaney. Sure. Yes, it is a great question. We are 
really excited to get some of the brightest minds around the 
country actually accessing the data. And as you mentioned, 
86,000 participants, that data is just going to get richer and 
richer as we add more participants, and as we get more 
electronic health record data in on all the participants.
    I would imagine that once we give access to researchers, 
that we would pretty shortly start to see some pretty 
significant findings. And, of course, the data gets richer with 
more participants, but over the long term as well, as we get to 
see people who are healthy, stay healthy, get disease, how they 
respond to therapies. All of that will be----
    Mr. Upton. Just real quickly, because my time is expiring, 
how long does it take for someone, if they sign up to do this, 
what is the time element for them to participate?
    Ms. Devaney. Sure. So the online enrollment process takes 
about 45 minutes to go through the consent and to authorize 
your electronic health record. And then we have the in-person 
visit where people donate blood and undergo a physical 
measurement. That takes somewhere between 45 minutes and an 
hour as well. And then over time, we will be asking 
participants to continue to fill out surveys and do other 
things digitally, and maybe even come back for in-person visits 
over the years.
    Mr. Upton. I yield back.
    Mr. Burgess. The chair thanks the gentleman. The gentleman 
yields back.
    The chair recognizes the gentlelady from California, Ms. 
Eshoo, 5 minutes for questions, please.
    Ms. Eshoo. Thank you, Mr. Chairman.
    Good morning, Doctors. It is an honor to have you here. And 
I can't help but think that while most people in the country 
don't know our names or what we do, they are all counting on 
you. So thank you for what you do. I think that you represent 
part of the real genius of our country. And I always say, NIH 
stands for our National Institutes of Hope. And you gave us 
even more hope with your report, Dr. Collins.
    Dr. Gottlieb, I know that you are aware that the 
legislation that my wonderful colleague, Congresswoman Susan 
Brooks and I wrote, the Strengthening Public Health Emergency 
Response Act, was included in the 21st Century Cures Act. And 
among other provisions, the bill established a priority review 
voucher to encourage the development of medical countermeasure 
drugs and vaccines at FDA.
    Now, when we developed the bill, the FDA, at that time, 
expressed concerns that allowing biodefense medical 
countermeasures to qualify for the PRV, would dramatically 
increase the number of PRVs awarded. The first product was just 
approved earlier this month. So does the FDA still have 
concerns about the number of products that will qualify for the 
PRV under the legislation? Can you tell us if there has been 
good interest on the part of companies to apply for it? Can you 
just maybe briefly inform us about that?
    Dr. Gottlieb. I appreciate the question, Congresswoman. And 
I think we all appreciate the effort of this committee to try 
to provide incentives to these important products. As you 
noted, the first product that qualified for one of these PRVs 
was just approved by the FDA. And it is potentially a very 
important product, a treatment rather than just purely a 
vaccine for smallpox. The Holy Grail was always to have a 
treatment in the event of smallpox being used as a bioweapon.
    I don't know the position of my colleagues who were here 
before me, but I certainly wouldn't argue that we have too many 
medical countermeasures coming into the agency and too many 
PRVs being issued in this context. I think, if anything, it is 
probably quite the opposite. We would still like to see more 
drug development in this realm, and there are still economic 
challenges to developing these kinds of medicines.
    Ms. Eshoo. I appreciate that.
    I don't know whether it is Dr. Collins or Dr. Sharpless. 
Pancreatic cancer is still a death sentence. I did legislation 
some years ago regarding it. Can you give us an update of if 
the needle has actually moved? I know when I did the 
legislation--I can't remember the year--that the needle really 
hadn't moved in about 40 years. So can you give us just a quick 
update on that? And then I would like to go to Dr. Devaney for 
a question.
    Dr. Sharpless. Sure. I think the answer is yes, the needle 
has begun to tick ever so slightly up in pancreatic cancer. I 
was speaking recently at the PanCAN, one of the leading 
advocacy groups in this space on this topic.
    Ms. Eshoo. Marvelous advocates.
    Dr. Sharpless. They do great work. And I think we have seen 
some really good stuff. So if you can resect pancreatic cancer, 
so you get a so-called R zero resection, those patients are 
doing pretty well. So resectable pancreatic cancer is a 
minority of those patients, but that group is growing. And we 
just had a positive trial, a randomized trial announced at ASCO 
with a significant survival rate for adjuvant chemotherapy in 
those patients.
    Other patients who do not have the RAS mutation, so the so-
called RAS wild-type patients, which may be about 10 percent, 
some of those patients are responding to novel kinase 
inhibitors, for example, that----
    Ms. Eshoo. I think I would like to follow up with you----
    Dr. Sharpless. Sure.
    Ms. Eshoo [continuing]. Because I am not a doctor, so I 
don't understand everything you just said. But I know that you 
can break it down for me if we have a conversation or a 
meeting. I would appreciate it.
    Dr. Devaney, thank you for your wonderful report. One of 
the places where the program is taking place is in my 
congressional district at the Palo Alto VA. Can you give me 
just a quick update if you know about the veterans that signed 
up to participate, how your experience working with the Palo 
Alto VA? I am very proud of it. It is quite an enterprise 
there. I have heard from many constituents who were interested, 
who are interested in participating in All of Us. And I would 
also like to know how you are advertising to the general 
public?
    Ms. Devaney. Sure, yes. I will start with the VA, and--
thank you for that by the way--we have a really great 
partnership with the VA and have for years. And of course they 
have been running the Million Veteran Program and they have 
670,000 veterans or more already enrolled. So we have learned a 
lot from them.
    Dr. Phil Tsao, who is the P.I. at Palo Alto, has been a 
great partner. They launched a few months ago and have had 
great success getting veterans enrolled into our program, which 
has been--it is a really important population for us, because 
of course the health outcomes that are so important there. And 
we have also a VA site in Boston. So we are excited to get even 
more VA sites launched around the country.
    Ms. Eshoo. Great and how are you advertising to the 
public----
    Ms. Devaney. Sure. Yes. We are using a number of different 
tactics, learning over time, how do we actually engage across a 
study that is trying to be national. So we are using different 
marketing techniques, digital marketing, we are also doing in-
person engagement. The healthcare provider organizations, 
including the VA site, are really critical for us because they 
have those direct relationships with their patients.
    Ms. Eshoo. Thank you very much.
    Thank you, Mr. Chairman. I yield back. Thank you again to 
all of you.
    Mr. Burgess. The chair recognizes the gentleman from Ohio, 
Mr. Latta, for 5 minutes for your questions, please.
    Mr. Latta. Thank you, Mr. Chairman. And thank you very much 
to our panel for being here. I really appreciate it and the 
testimony you have provided. It is important for this country. 
And also the gentleman from Michigan's not here right now, but 
I also want to thank him for his leadership on 21st Century 
Cures and getting us to this point where we are today.
    Dr. Collins, if I could start with a question for you in 
your testimony you talked in regards to the brain initiative. 
And especially at the end of testimony talking about where it 
will lead to increased understanding for brain health and a 
means of preventing brain disorders such as Alzheimer's 
disease, Parkinson's, Schizophrenia, autism, drug addiction and 
traumatic brain injury. And I know I also see that there has 
been about $1.5 billion that has been allocated over the next 
10 years for the studies that is going on, but I know that when 
I have been at Ohio State and seeing what they are doing with 
Parkinson's in their studies. But the one area I would like to 
focus in on today is on Alzheimer's, because as we look across 
the Nation today and we are seeing what is happening, and the 
reports especially from what Alzheimer's association's reports 
that you are looking at that the cost could rise as high as 
$1.1 trillion by the year 2050.
    The sixth leading cause of death in the United States 
today. One in ten Americans over 65 will have it. We have about 
5 million Americans living with Alzheimer's today. One-third of 
Americans over 85 are afflicted with it. But unfortunately, 
start looking down the road over the next almost 30 years, 
people over the age of 65 there will be about 14 million people 
afflicted. Where do you see this going right now with 
Alzheimer's?
    Dr. Collins. This is an area of intense interest as a major 
challenge for the world and certainly that is very true of the 
United States. You have quoted the frightening numbers of 
individuals that are projected to be affected and the cost that 
that is going to apply to our healthcare system.
    And even more than that, the personal tragedies that happen 
every time a diagnosis of this sort appears, it affects not 
only the individual but their family and the caregivers. We are 
all in on trying to come up with an aggressive strategy to 
identify a pathway toward prevention and treatment of the 
disease. And we are grateful to the Congress because over the 
course of the last 3 years, Congress has been progressively 
giving us greater resources to work specifically on 
Alzheimer's. We are also doing that in a very I think visionary 
productive partnership with industry in something called the 
Accelerating Medicines Partnership to try to be sure that the 
basic science that we are doing on the brain, trying to tell 
what are the earliest signs of Alzheimer's gets translated 
quickly into therapeutics.
    One of the things we are learning is that Alzheimer's 
begins long before the first symptom, in terms of cognitive 
function. And if we are really going to be successful in 
delaying or preventing it, we need to find people at risk at 
the earliest possible moment and not wait until they are 
already in a circumstance of having lost a lot of neurons, 
because they will be hard to get back at that point.
    I am a guardedly optimistic that we are on the right path 
with some of the trials that are underway right now where we 
have the opportunity to treat people before they have any 
symptoms, but we know they are on a dangerous pathway and can 
watch and see what is happening.
    We are also learning a lot more about roles that play a 
significant hand in what happens with Alzheimer's and the 
immune system which we hadn't really realized before. And 
inflammation seems to be important in this condition. And all 
of the focus that we have had on amyloid and tau, the two 
proteins that build up in the brain are important, but that may 
not be the whole story. I could give you a long lecture on 
this, and I will stop now because of the timetable. But I do 
want to reassure you that between NIH basic and clinical 
science working with industry and with close collaborations 
with the advocates, we are pushing this as fast as we possibly 
can.
    Dr. Gottlieb. And I would just add if I may, we work 
closely with Dr. Collins in these efforts and we recently 
issued a new guidance document updating the agency's guidance 
on clinical trial end points in this setting, in this 
particular disease setting.
    Historically there was a perception that you had to show 
improvement in both functional status and cognition to win 
approval or at least show that you slowed the decline in 
functional status and cognition. That is challenging because by 
the time your functional status declines as an Alzheimer's 
patient, you probably had a pretty significant decline in 
cognition.
    And now that we are able to identify patients earlier with 
more sensitive tools to detect changes in cognition, also 
process prespecify patients who are at high risk of developing 
Alzheimer's, what we want to do is intervene earlier when there 
are slight changes in cognition, try to arrest that process. 
And so what the agency has said is in the right circumstances 
if you can show an improvement of cognition alone, that could 
potentially qualify for approval for a drug that would slow the 
progression of Alzheimer's disease in the right prespecified 
population that properly identify patients. So we are trying to 
work closely with NIH as well to help facilitate this 
innovation.
    Mr. Latta. Thanks Scott.
    Dr. Collins. That is really important to have that as an 
endpoint.
    Mr. Latta. Well again, thank you very much for our panel. 
And Mr. Chairman, I yield back.
    Mr. Burgess. The chair thanks the gentleman.
    Mr. Green. Mr. Chairman, could I ask for the next Member of 
Congress, Ms. Diana DeGette, could come up and sit in the 
ranking chair as a cosponsor of the bill.
    Mr. Burgess. No objection.
    The gentlelady is recognized for 5 minutes for questions.
    Ms. DeGette. Thank you so much. I just thought Mr. Green 
wanted a break. That was very nice of him to recognize me.
    And again, I want to add my thanks to all four of our 
witnesses up here for their extraordinary help, not just in 
passing this bill, but also in implementing it. It really warms 
our hearts to see how much progress we have made in such a 
relatively short time. And it also encourages us to think how 
much more we can make.
    So I just have a few questions for each of our agencies 
that are here today. I think I will start with the FDA. So Dr. 
Gottlieb, the biomedical community has really made great 
strides in developing clinical therapies from a marking of the 
potential system cells, to creating therapies tailored to the 
unique genetic fabric of a patient.
    And one of the things we have been concerned about for a 
long time is that overseeing and regulating these cutting edge 
therapies really requires an experienced, well trained, robust 
public health workforce.
    And so in 21st Century Cures we had a provision for new 
hiring authorities known as HR Cures. I am wondering if you can 
talk to us about how the HR Cures authority has helped the 
agency recruit and retain its talent.
    Dr. Gottlieb. Thank you, Congresswoman. We are in the 
process of implementing that. I think it is going to be 
transformative for the agency in terms of our ability to 
recruit people with specialized skills. We have identified 38 
occupations that meet the requirement for the alternative pay 
system. We have made the first two hires under the new cures 
authority, both deputy center directors, but I think this is 
going to be very important for the agency.
    And just to briefly pick up on your point, when we are 
talking about new treatments like gene therapy and cell base 
regenerative medicine and even things we are seeing on the 
medical device side of the house, the clinical characteristics 
of the products relate very closely to the product features. 
And the product features change very quickly. The underlying 
technology of the product itself, these aren't all pills 
anymore.
    Ms. DeGette. Right.
    Dr. Gottlieb. And so it used to be that if you had 
expertise in the scientific field or the clinical field you 
could adjudicate all the different kinds of drugs. But now it 
requires expertise also in the products themselves. The gene 
therapy platforms, there are regenerative medicine platforms. 
And we see this a lot on the medical device side of the house 
where there is a lot of novel technology. You have to have 
engineering skills on that specific technology. That is where 
this is going to be particularly helpful, trying to find those 
people with the very discrete, specialized----
    Ms. DeGette. These are people you just can't hire someone 
out of grad school. These are people with really specialized 
areas of knowledge.
    Dr. Gottlieb. That is right. And they are people who are 
often employed in endeavors in the private sector where they 
are highly paid, highly skilled so we are competing against 
others for the same small pool of talent.
    Ms. DeGette. In the FDA June 2018 report called 21st 
Century Cures Workforce Planning you talk about this patchwork 
of hiring authorities that have created challenges. Are you 
beginning to be able to address that?
    Dr. Gottlieb. We have had historical challenges with the 
overall hiring framework at FDA going back 15 years or longer. 
Some of the same challenges that I am grappling with now I saw 
commissioners grappling with when I was at the agency in 2003 
and again in 2007.
    We are addressing it very directly. We are trying to do it 
top down, wholesale change of the hiring process to make it far 
more efficient. We started a pilot which dramatically 
streamlined the on boarding process, the hiring process. And we 
focused that pilot on the PDUFA slots, on the user fee slots. 
What we have decided to do is basically take that structure of 
that pilot and apply it to the whole hiring process and not 
just sort of bifurcate off one set of hiring----
    Ms. DeGette. I hate to cut you off, but I can't ignore Dr. 
Collins. And I have about 10 minutes of questions for you, but 
you are going to have 1 minute to answer them.
    Let me just ask you what kind of research is going on over 
in your agency on alternatives to opioids for pain management? 
Because the opioid crisis is one of the big issues that has 
really been facing this committee for the last 1 year or 2.
    Dr. Collins. Thank you for raising this. This is an 
incredibly important issue. We are grateful to the Congress 
that in the omnibus an additional $500 million was put into our 
budget to work on the opioid crisis. And a significant fraction 
of that is being devoted to just the thing you are raising 
here, the need to develop affective and nonaddictive pain 
medicines for those 25 million Americans who suffer from 
chronic pain every day and for whom opioids is not the answer.
    Ms. DeGette. Right.
    Dr. Collins. And actually it is quite harmful in many 
instances. This goes all the way from basic science, to 
discovering new targets, to working with industry to help 
actually free up some ideas that had been on the slow boat and 
now can be speeded up. Working in a collaborative way. And 
setting up a clinical trials network so that we can quickly 
test and see whether new therapeutics that are not addictive 
work, and in what setting did they work, because all pain it 
not the same. Low back pain may not be the same as the pain you 
get from trigeminal neuralgia, and we need to have all ready to 
go the kind of clinical trial networks that currently doesn't 
exist.
    Ms. DeGette. I think, Mr. Chairman, this might be a good 
subject for a whole separate hearing. Thank you.
    Just one last thing, Dr. Gottlieb, I had some questions 
about some rules that have been pending about shock therapy in 
the FDA. We don't have time to have you answer those questions 
right now, but I am going to be contacting your agency to find 
out why those rules haven't been approved. They have been 
pending since 2016. And I promised my constituents I would ask 
you about this so we----
    Dr. Gottlieb. I would be happy to discuss that with you 
moving forward thank you.
    Ms. DeGette. Thank you, very much. Mr. Chairman.
    Mr. Upton. Thank you, Mr. Chairman. I appreciate your 
holding this hearing. And I want to thank all of our witnesses 
today and the very talented men and women that you are speaking 
on behalf of your agencies who do amazing work in the area of 
healthcare in America Cures. The legislation was passed, 
medical innovation, reforms to America's and mental health 
services.
    And this is why I thought today was really an important 
day, an important hearing to continue our work to legislate and 
evaluate and legislate and evaluate and see what else we need 
to do going forward. What is working, what is not, and what do 
we need to change.
    I know I have had a lot of roundtable discussions, and 
hospital board rooms, community centers townhalls with people 
in my district who are very pleased with the investments we 
have made in medical research and in turn the work that your 
scientists are doing.
    To turn that money and research into reality, and medicine, 
and cures that goes from one end of this table to the other. 
And so we appreciate the work you are doing. A friend in Bend 
say Carol, who is an MS advocate, has said Cures was a great 
step toward making it possible to find a cure to MS. And I know 
you all hear that every day. People are pretty excited about 
precision medicine, the cures that are out there waiting to be 
found and the work that you are doing.
    Dr. Gottlieb, in your testimony today you suggested there 
is a correlation between lowering medical product costs and 
promoting modern clinical trial designs. Can you elaborate on 
that, because we are all very concerned about the costs of 
healthcare, the cost of medicines. And clinical research isn't 
always as efficient as it could be, and how biomarkers and 
other drug development tools help to make that more efficient 
in patient centers.
    Dr. Gottlieb. I appreciate the question, Mr. Chairman. I 
think while clinical trial costs don't directly translate into 
how a product is priced, we know that. People price products 
based on what the market's going to bear and the value that it 
is delivering. We know that clinical trial costs, cost 
development costs do factor into the rising costs of the 
overall development and competitive nature of this landscape. 
What I talked about in particular today is the complexity of 
clinical trials, especially in areas of unmet medical need when 
there is already available therapy is in my view delaying to 
market the second and third in class drugs.
    So these specialty drugs that target unmet medical needs 
are enjoying monopolies for longer periods of time. And if you 
believe that subsequent competition and the data shows this is 
when competition market prices come down, that competition 
isn't entering and the prices aren't coming down. And we now 
have data to demonstrate this. We will be publishing that soon. 
I gave a snapshot of it here today.
    I think there are things we can do to try to facilitate 
more efficient routes to market the second in class drugs and 
third in class drugs while at the same time increasing our 
assurance in safety and effectiveness, not sacrificing it one 
bit. And those are the kinds of development reforms that we are 
focused on.
    Mr. Upton. And trials for rare diseases can be difficult to 
conduct, especially if there is already a first FDA approved 
treatment. How do you spur additional innovation and greater 
market competition for rare diseases?
    Dr. Gottlieb. And this gets to some of the changes in the 
clinical trial designs. One thing in particular is in very rare 
diseases looking at natural history models to model the 
behavior of the control arm, or using modeling as simulation, 
so you don't have to droll placebo trials in those settings, 
because if there is already available therapy, patients aren't 
going to want to go on to a placebo if they can otherwise use 
the available therapy. They are going to want to use an active 
drug.
    So this is a place where we could benefit from more disease 
models. We should be able to create those. There is a request 
in the President's budget for about $20 million which would 
help facilitate that. I know we have support from this 
committee and others in Congress for some of those resources so 
we are very grateful for that. But I think there are things we 
can do working together.
    Mr. Upton. I appreciate that and a good reminder. The 
promotion for the President's budget, well done, well played. 
Send that to the White House.
    Drs. Collins, and Devaney, and Sharpless, I saw the NIH 
recently announced an effort called respond or research on 
prostate cancer in men of African ancestry defining the roles 
of genetics, tumor markers and social stress. So that study 
funded in part by 21st Century Cures Cancer Moon Shot 
initiative will investigate the environmental and genetic 
factors of prostate cancer in African-American men. Can you 
tell us more about this important study and how it will combine 
molecular approaches in environmental science?
    Dr. Sharpless.
    Dr. Sharpless. Thank you for the question. This is alluding 
to a trial just announced from the NCI. That will be the 
largest trial on this topic in our history, so it is going to 
take 10,000 patients newly collected and 10,000 historical 
patients and try to analyze the genomes and social factors that 
contributes to this important disparity. African-American men 
are likely to be diagnosed with advanced and prostate cancer 
and are more likely to die of prostate cancer. So understanding 
this important disparity in our country is really a crucial 
question and the response trial are exciting.
    Mr. Upton. All right. Anything else anybody want to add?
    Dr. Collins.
    Dr. Collins. I think I just have to point again to the All 
of Us study as a platform that we will be able to utilize going 
forward for answering many of that sort. We have this goal and 
we aim to reach it that about 50 percent of the participants in 
All of Us are going to come from traditionally underrepresented 
groups, racial and ethic groups.
    And so if you want to really examine health disparity 
circumstance, you are going have a million participants who are 
highly motivated to take part and research on whom we collected 
a great deal of data. We are going to learn a lot about this 
kind of health disparity, about all diseases once we have that 
platform up and going.
    Mr. Upton. Excellent. Excellent. Thank you all. Appreciate 
your continuing involvement with our committee. It has been 
positive and effective I think for the American people. And we 
look forward to doing more together.
    And Mr. Chairman, I yield back.
    Mr. Burgess. The chairman thanks the gentleman. The 
gentleman yields back. The chair recognizes the gentlelady from 
Florida for 5 minutes for questions, please.
    Ms. Castor. Thank you, Mr. Chairman. And welcome to all of 
our witnesses today.
    Dr. Collins, I share your enthusiasm over the future of the 
Cancer Moon Shot in precision medicine. Part of my enthusiasm 
stems from the fact that back home in Tampa I represent the 
Moffitt Cancer Center community, the only NCI designated 
comprehensive cancer center in the State of Florida.
    And I love meeting routinely with the young scientists and 
researchers who now feel like we have given them a new 
commitment where they were very concerned in the past on the 
future of NIH funding as it competes with all the other needs. 
Now they feel like, OK, 21st Century Cures, these new 
investments for the Cancer Moon Shot, gives them hope for all 
of their research and the care they are providing.
    Dr. Duvaney, the Moffitt Cancer Center has been a national 
leader in building large datasets, tissue samples before and 
working with other institutions, especially for those in 
underrepresented communities.
    You have now given us a bit of an outline on the importance 
of protecting privacy for the people who participate. Can you 
tell us a little bit about the protocols going forward for 
researchers? Will they be required to share their research? 
When would that happen? Will those research results be 
available to all for other researchers to build upon?
    Ms. Devaney. Great question, thank you for that. So, we 
have been thinking a lot about the--to your point about young 
investigators, the diversity of researchers also that can 
access the data, making sure that the platform we are building 
is open to everyone, and feel responsible for ensuring that the 
things that we learn on the data, that is so generously 
provided by our participants, is returned to the scientific 
community and to our participants in fact.
    So we are developing policies around ensuring that 
researchers who access the data required to follow code of 
conduct and share their results within a specific amount of 
time. And we are still finalizing those policies in 
anticipation of opening up the data next year, and policies 
around returning results back to participants who participate 
in the study as well.
    Mr. Costello. Because Dr. Collins, in the past it has been 
kind of siloed. If certain researchers had kind of kept it, 
held it close, and didn't make all the data available, what 
does the future hold do you think?
    Dr. Collins. I appreciate the question. And I appreciate 
21st Century Cures gave me, as the NIH director, authority, 
that I did not have before, to require data access for studies 
that we support. I could cajole in the past. I could try to 
embarrass people, but I didn't really have the clout to say, 
this is a requirement. If you are getting a grant from NIH, you 
are required to make your data accessible.
    You all gave me that and that has been a very useful tool. 
And we are very engaged in the process of trying to establish 
exactly what that needs to look like for a wide variety of 
studies. We have done it particularly for genomics where we 
have a very well worked out genomic data sharing policy, and we 
are in the process of working that out for things like imaging 
and electronic health record information and so on. But this is 
a very high priority. We hate those silos too and we have been 
having a good time knocking them down.
    Ms. Castor. Great. We look forward to that.
    Dr. Sharpless, your predecessor at NCI, Dr. Doug Lowy was 
well-known for his research on HPV and the HPV vaccine. Part of 
the Cancer Moon Shot isn't just the cancer research, but it is 
what NCI can do to help prevent cancers and protect them. Over 
the past few years, there has been new NCI focus on making sure 
that there is greater uptake of the HPV vaccine across the 
country.
    Do you intend to continue with that? If we discovered the 
cure for cancer, there would be parades in the streets, but 
here is an actual vaccine that prevents cervical and a whole 
host of other cancers, I think it is important. Are you 
committed to continuing those initiatives?
    Dr. Sharpless. Very much. I should mention that Dr. Lowy 
continues as my deputy director. He has been incredibly 
invaluable in that regard, because entering the Federal 
processes as an academic is challenging so I really appreciate 
Doug and his work in this area is wonderful.
    Yes, we have a robust, huge portfolio in this area. I think 
one of the most important studies is a trial of one versus two 
doses of the vaccine. Right now the recommended is three doses. 
That is a big issue in terms of implementation in the 
community. So if one dose worked well, that could be game 
changing for subjects in the United States as well as globally 
with cervical cancer is a bigger problem.
    Ms. Castor. I guess it is a good reminder, it is back to 
school time. This is a vaccine that is appropriate. It is 
important for boys and girls, middle schoolers to get the HPV 
vaccine to prevent cancer in the future. Is that correct?
    Dr. Sharpless. Yes, right. A number of vaccines that 
prevent cancer HPV, Hepatitis B, for example, the dissemination 
of those things that work into the community is a real 
challenge for us in terms of the inflammation science to make 
these effective therapies more available.
    Ms. Castor. Thank you very much. I yield back.
    Mr. Burgess. The chair thanks the gentlelady. The chair 
recognizes the gentleman from New Jersey, Mr. Lance 5 minutes 
for questions, please.
    Mr. Lance. Thank you, Mr. Chairman. Good morning to you 
both and thank you for your enormous public service.
    For Dr. Gottlieb, the Orphan Drug Act has helped to create 
a market based system, to encourage the development of new 
medicines for people living with rare diseases, most of whom 
are children. As you know, I am the chair of the Rare Disease 
Caucus in the House.
    In exchange innovators are granted 7 year monopolies in a 
specific disease area. Since the act was established in 1984, 
medicines have been brought to market for only some rare 
diseases, and there are many rare diseases as you both know 
where there are no medicines at all.
    For many of these diseases, however, there has been zero 
second generation, newly innovative medicines brought to market 
for patients. In the field of Lysosomal storage disorders, for 
example there have been enzyme replacement therapies for eight 
different rare diseases. The cost on average is nearly $500,000 
per patient per year. However, with the exception of Gaucher 
disease, not a single second generation therapy has been 
approved for any of these diseases, since these first 
generation ERT approvals, and most of those approvals date back 
at least a decade.
    In your view, Dr. Gottlieb, have we created perpetual 
monopolies in many rare diseases, especially in the areas I 
have discussed? And what are the barriers to moving innovation 
forward to spur competition and innovation and to bring newer, 
better medicines to patients with rare diseases as rapidly as 
possible?
    Dr. Gottlieb. Thank you for the question, Congressman. I 
think this cuts to some of the other issues we discussed here 
today so it is a very relevant question.
    I do believe that there are settings where it is harder to 
bring second to market competition into certain categories. I 
would argue this is one of them. It is particularly the case 
when you have drugs that target degenerative diseases, 
significant unmet medical needs, small populations. It is hard 
to run clinical trials with new drugs once one therapy is 
available. Typically, the subsequent drugs will have to be 
studied on top of the available therapy and you will have to 
show improved efficacy, with combination therapy, as opposed to 
just monotherapy. It is hard to run head-to-head comparative 
studies when already effective therapy is available. People 
don't want to forego an effective treatment, especially when 
you are dealing with a child with a degenerative disease.
    I think there are a lot of things we can do. Earlier this 
year we published it in conjunction with our counterparts in 
Europe, our regulatory counterparts in Europe, a master 
protocol, if you will, for Gaucher's disease and how you can 
study multiple drugs within the context of the same clinical 
trial. Structure for Gaucher's, we have talked earlier about 
trying to develop natural history models to model. The behavior 
of patients who go untreated. We know how these diseases 
progress.
    We know how they affect patients. We should have really 
robust natural history models to model be able to model that 
affect so we don't have to enroll patients on placebo trials. 
It relies much on placebo and that would make trials easier to 
conduct.
    So there is a whole host of clinical trial reforms that I 
think we can pursue to try to facilitate second to market 
innovation, which I agree with you is critical in these areas.
    Mr. Lance. Is there something more we should be doing 
statutorily here in the legislative branch of government, 
Doctor?
    Dr. Gottlieb. I would be happy to have that discussion with 
you. I think there is a lot of things that we can do, that we 
are trying to do, and will do. I think one thing that I would 
just affirm--and I had this discussion with the chairman about 
the natural history models--we allocated $6 million earlier 
this year to develop six natural history models, Myotonic 
dystrophy 1, a natural history model for the affects of sickle 
cell disease on the kidney, but we did in our budget this year 
request additional funds to develop up to an additional 20 
natural history models, and those would be focused on these 
diseases areas, these very orphaned, if you will, super 
orphaned diseases we would focus on the resources there.
    Mr. Lance. Thank you, I look forward to working with you as 
we have in the past. I have been the chair of the Rare Disease 
Caucus for quite some time. And this is completely bipartisan 
in nature. And we look forward to continuing the discussion. 
Dr. Collins, it is always a pleasure to see you.
    I note in the audience John Crowley who has been very much 
involved in the rare disease space and his daughter Megan, and 
they are residents of New Jersey. And there is no one who is 
fighting for progress in the rare disease space more than my 
friend Mr. Crowley.
    Thank you, Mr. Chairman. I yield back the balance of my 
time.
    Mr. Burgess. The chair thanks the gentleman. The gentleman 
yields back. The chair recognizes the gentleman from Virginia, 
Mr. Griffith, 5 minutes for questions, please.
    Mr. Griffith. Thank you very much, Mr. Chairman. I do 
appreciate it. I appreciate you all being here today.
    On June 8, 2018, Dr. Gottlieb, the committee sent FDA 
bipartisan oversight requests for information about the FDA 
increasing criminal enforcement at ports of entry to combat the 
opioid epidemic, an initiative that you have championed and we 
support. The letter has only six requests, some of the requests 
were first posed by an email to the FDA Office of Legislation 
on January 30th of year. It is now late July and the FDA 
continues to tell the committee staff of letters in clearance.
    Commissioner, could you help expedite the clearance of this 
letter so the committee has the FDA's response before the end 
of July?
    Dr. Gottlieb. I absolutely will. It is in clearance. And we 
in an effort to try to improve transparency and get the 
information out publicly, I did make a lot of the information, 
if not most of the information that is going to be in that 
letter, available publicly in some remarks I gave at a forum we 
had to try to work with internet stakeholders to address opioid 
sales online. So I have made the information public. I can make 
those remarks available to you. I think we have made them 
widely available. But I recognize the importance of getting the 
formal response back to Congress and I am on it. I have some of 
my own challenges up my line.
    Mr. Griffith. I appreciate it. Well, and you have so much 
to do and I really thank you for the good work that you are 
doing. And there is a lot of different areas that we have been 
working on together.
    Dr. Gottlieb. Thank you.
    Mr. Griffith. And I appreciate that very much.
    What further improvements will FDA make to the expanded 
access program to ensure the process is effective and efficient 
for providers and patients, particularly now that the Federal 
rights to trial legislation has passed? And while I like the 
House version better than the Senate version, we have got the 
Senate version. Give me an update on that.
    Dr. Gottlieb. Well, we have a process underway right now, a 
working group that is looking at what steps, if any, we ought 
to take to facilitate the proper implementation of the right to 
try measure. It is going to be a pathway that sits alongside 
our extended access program. We are still going to operate our 
extended access program.
    We did bring in an outside group, an expert group to take a 
top down look at our expanded access program and make some 
recommendations to the agency on how we can improve it. I am 
going to make that public soon to provide some transparency 
around what they found.
    But we think that there are additional process steps that 
we can take to make it easier for people who aren't as 
sophisticated or physicians who haven't done this before to 
access that. We are also working with Friends of Cancer 
Research and the Reagan-Udall Foundation to create a platform 
to have sort of a one site of entry, if you will, to get 
information about what expanded access programs exist.
    I think the challenge still remains that the difficulty, 
sometimes reluctance, but a lot of times difficulty of sponsors 
to actually make drugs available especially when we are dealing 
with things like some of these cell based therapies or 
biologics cost aren't trivial, when you are a small company you 
barely have enough product for the clinical trials. And I have 
been on the other side of this, so I know how that is. And so I 
think we still need to look for ways to provide proper 
incentives to try to make sure that when we have really really 
promising therapies we might be able to have supply available 
too to make that available to patients.
    Mr. Griffith. Well I appreciate that. I have been an active 
proponent for right to try for many years. And if people are 
facing death, they will take that Hail Mary pass and take 
whatever they need to, if they can. Let us know what we can do 
to assist you on that as well.
    Dr. Collins, I am going to ask you a couple of questions 
just to give me update. I have a partial answer that I already 
found on the internet and that is with Lou Gehrig's ALS, I 
understand you all had a breakthrough this spring. But I also 
am interested in Huntington's chorea, and I obviously ask about 
these because I have friends who have been afflicted with both 
of those.
    Dr. Collins. Those are both terribly important and often 
tragic neurological diseases that result in neurodegeneration. 
With ALS, as you mentioned, there has been encouraging 
development of a new therapeutic approach this spring still 
very much in the process of being evaluated.
    Huntington's disease I will actually reflect a bit on, this 
was 25 years ago that my own laboratory was involved in the 
discovery of gene that is responsible for that condition. And 
it has been very gratifying to see in the course of just the 
last year or two, a still and a mouse model, very encouraging 
information about using a genetic approach to try to block the 
production of the toxic protein.
    And now in human clinical trial, which we are waiting for 
the full data to be rolled out, but sounding encouraging, a 
similar possibility that utilizing a molecular therapy injected 
into spinal fluid, which then gets into the brain, may very 
well be able to provide benefit.
    And goodness knows, we have waited a long time for that 
kind of thing to happen. So it is pretty exciting to see. We 
have a long way to go I think before we can say really have the 
answers, but this is a lot different than saying we have 
nothing to offer.
    Mr. Griffith. I appreciate that very much and yield back.
    Thank you, Mr. Chairman.
    Mr. Burgess. The chair thanks the gentleman, the gentleman 
yields back. The chair recognizes the gentleman from 
California, Mr. Cardenas, for 5 minutes for questions please.
    Mr. Cardenas. Thank you very much, Mr. Chairman and ranking 
member. I appreciate the opportunity to openly discuss with the 
witnesses the progress that we have made. And not every day do 
we as United States Congress actually pass laws that we can 
actually look at and say, I think we did something good. And so 
I want to thank all of the implementers for doing good work.
    My first question has to do with the diversity in research 
of subjects. So thank you for testifying today. I was excited 
to read in your testimony, Dr. Collins, that about 85,000 
individuals have already started the enrollment process for the 
All of Us Research Program; ``70 to 75 percent are from 
communities who have been historically unrepresented in 
biomedical research and almost 50 percent are specifically from 
racial and ethic groups who have not been included in previous 
research.''
    As you all know, diversity in medical research has long 
been an issue. I believe one way to decrease health disparities 
in this country is to ensure that we are including individuals 
of all races and ethnicities in medical research.
    So with that, my question is what is NIH doing to ensure 
that All of Us research program recruits a diverse group of 
participants that is racially and ethnically diverse?
    Dr. Collins. Well we track that week by week. I personally 
am asking for that information every week to see how the 
enrollment is going. But I am going to ask Dr. Duvaney to say 
how this is being done because this is unprecedented to have 
this level of diversity.
    Ms. Devaney. Yes. And thank you for the question. This is 
perhaps our strongest priority as a program, understanding that 
ultimately a lot of the data scientists are using today is not 
reflective of the diversity of the country and that what we are 
learning is not applicable to all communities.
    We have a number of different ways in which we are 
attempting to reach out to diverse communities. And just go 
into it with the understanding that we are going to try things, 
and it is not going to work, and we are going to go have to 
shift and try new things.
    One of the things I would just like to highlight is working 
with community partners. We have a number of community 
engagement partners across the country who are helping us to 
build trusted relationships with their communities at a local 
level. And our chief engagement officer, Dr. Dara Richardson-
Heron at the NIH has been really working with those groups in a 
very robust way. And beyond our first foreign inaugural 
partners we have about 30 plus partners that are national that 
are helping us to build awareness within our communities.
    Mr. Cardenas. So you are talking about groups that are more 
local and actually might have a propensity for working with a 
particular population, whether it is an immigrant population, 
or a Hispanic population, or a Black population, et cetera. So 
working with local groups who already have relationships with 
those communities?
    Ms. Devaney. That is right, exactly. Yes.
    Dr. Collins. When we did the launch on May 6 in seven 
different places I was in New York at the Abyssinian Baptist 
Church with an African-American community that was totally 
revved up about the opportunity to be included this time.
    And I have to give credit to 21st Century Cures about this 
focus on inclusion. You have given us some additional tools 
there in many different projects across the board, but All of 
Us is really turning out to be a flagship here about how to do 
this in the most visionary way.
    Mr. Cardenas. OK. So this opportunity for us to remind 
ourselves that diversity and research when it comes to subjects 
in particular communities, et cetera, because we all 
genetically have different reactions, et cetera, and different 
propensities when it comes to certain diseases and cause and 
effect, correct?
    Dr. Collins. Absolutely. And one should not assume that 
when you see a health disparity that that is something that is 
readily understandable on the basis of looking at one thing. 
Usually these are a combination of environmental exposures, of 
stress levels, of some genetics in there. But one shouldn't 
overinterpret that part because we are awfully similar at the 
DNA level. And if you really want to understand the health 
disparities, you need to have these comprehensive studies that 
collect data from all those perspectives and then figure out 
how to intervene.
    Mr. Cardenas. So it is better to have good research rather 
than having to force yourself to extrapolate out, and then that 
is more guesswork and that is not necessarily scientific.
    Dr. Collins. You want to have the data on the people for 
whom you are going to then offer some answers. We just heard 
from Dr. Sharpless a few moments ago about this big study they 
are just starting with African-American prostate cancer. 
Another example, where we don't understand, why men who have 
prostate cancer, who are Black, have a higher likelihood of 
dying and a higher likelihood of aggressive disease. We need to 
know that.
    Mr. Cardenas. OK. So what are some of the challenges 
associated with building a cohort that is racially and 
ethnically diverse? Do we have some findings or things that we 
are hoping to tackle in the near future that maybe you learn by 
venturing further into this diversity effort than ever before?
    Ms. Devaney. Yes. Well, we are just at the beginning of 
this journey. So we would love to follow up with your office 
and talk about this more. I will say we have community advocacy 
groups and participant advisory boards, at all of the health 
care organizations that are part of our program that are 
helping us understand what is working, and what is not working, 
messaging what is not working as part of--even things like your 
parking isn't close enough to the clinic, and therefore it is 
too hard for me to make the appointment time to come in and 
donate my samples. So we have been learning a lot just from 
participant feedback and would be happy to share as we learn 
more.
    Mr. Cardenas. So as an engineer myself, having a good 
feedback system and adjusting all the way is important so you 
are doing that.
    Ms. Devaney. Yes.
    Mr. Cardenas. OK. Thank you very much Mr. Chairman and 
thank you for the generosity of time.
    Mr. Burgess. The gentleman yields back. The chair 
recognizes the gentleman from Missouri, Billy Long, for 5 
minutes for questions, please.
    Mr. Long. Thank you, Mr. Chairman.
    And I would like to get one more shout out to Diana 
DeGette. Diana is my co-chair on a congressional study group on 
Japan. We travel to Japan together every year. And also to Fred 
Upton for their tireless work on this 21st Century Cures and 
they just did yeoman's work as a lot of you know. So I want to 
give one more shout out to them.
    My youngest daughter when she was 25 years old diagnosed 
with Hodgkin's lymphoma. Today she is 29 years old, she is 3 
years past chemo. So the developments over the years as Dr. 
Collins was talking earlier have been phenomenal. And I want to 
thank everyone.
    Dr. Sharpless, we were happy to support the Cancer Moon 
Shot funding in the 21st Century Cures and are looking forward 
to hearing about progress for patients. Can you tell us about 
the most exciting thing that is being supported in the Cancer 
Moon Shot?
    Dr. Sharpless. Well, that is really tough. There is so much 
good stuff. I think this is a very meaningful law for patients 
with cancer. And I think it is impacting in many ways. We 
already talked about the response studies, maybe I will talk 
about the rare tumor initiative, which is also a very exciting 
initiative. We mention rare diseases, many rare cancers 
actually are hard to study for some of the reasons that came 
up. They are rare, they are hard to do clinical trials.
    And the NCI is a good place to study those things. We can 
have patients with a certain rare disease come to the NCI and 
get their care here. And this paradigm works and the Moon Shot 
trying to build on that experience. So for example, in the area 
of a disease called RASopathies where different mutations that 
activate RAS, a driver protein in cancer, those patients 
generally present with childhood tumors.
    And the NCI now has a recent trial that we just had 
presented at ASCO shown here on the left is a slide of Andrea 
Gross showing, one of our scientists--showing results of 
selumetinib trial in this RASopathy patients.
    So this is a patient with NF1 deficiency. And my executive 
summary was the tumors shrink, the kids feel better and the 
drugs seem safe. Yay. And then a few parents contacted me and 
said, my child was on this trial, actually and the responses 
have been marvelous.
    And so shown is the picture of the child pretreatment on 
the left. This is Phillip after treatment. And you can see his 
windpipe, his airway is not being compressed by the tumor 
anymore, and he has not the social stress of going to school 
with a big lesion like that. So this is a sort of a precancer 
syndrome. This is mutation that activates RAS, this cancer 
gene. And this kind of study is a really great thing for the 
NCI to take on because of our integral program.
    Mr. Long. OK. And in the testimony it says we must 
transform the way we conduct research, the way we share 
results, and the way we get discoveries into patient's care. 
Could you discuss how NIH is helping transform infrastructures 
needed to help meet these goals?
    Dr. Sharpless. For junior scientist you mean?
    Mr. Long. Yes.
    Dr. Sharpless. Yes. Yes. So the new funding from Congress 
has made it possible to increase the success rates for new 
scientists applying for their first grants, so it is called R01 
grant. So in NCI for example has set aside enough funds to 
increase that amount by 25 percent. We similarly are trying to 
link in the period of the award. We think that that will allow 
the scientists to concentrate on their science and not much 
bureaucracy in terms of writing grants.
    So we are testing out a 7-year award as opposed to a 5-year 
award. And one of the things we are really doing is thinking 
about the training opportunities we are providing for young 
scientists, both in the postdoctoral setting and in the faculty 
setting, to make sure they get the right skills, for example in 
big data, which is something that we really need more training 
for.
    Dr. Collins. If I may, just across all of NIH, and again 
thanks to the 21st Century Cures and Next Generation researcher 
initiative, which is part of this bill, we have put into place 
efforts to provide a better chance for early stage 
investigators coming to NIH with their first major grant 
application to get funded, and then to have sustainable support 
so they know they have a career.
    This year, fiscal year 2018, we are predicting we will fund 
the largest number of first time investigators ever at NIH 
because we have made this shift in priorities and you helped us 
with that, with 21st Century Cures.
    Mr. Long. Thank you. I had a chance to meet with two early 
career scientists from the American Association of Cancer 
Research. And I was very impressed with the excitement and 
passion they have for careers in cancer research and helping 
cancer patients. And once again as a father of a cancer patient 
survivor it really means a lot to me.
    Thank you all for being here, and thank you for being here 
today, you all too.
    So I yield back.
    Mr. Burgess. The chair thanks the gentleman. The gentleman 
yields back. The chair now recognizes the gentleman from 
Maryland, Mr. Sarbanes, for 5 minutes of questions, please.
    Mr. Sarbanes. Thank you, Mr. Chairman. Thank you all for 
coming today. Whenever you testify I am reminded of the 
tremendous responsibility that you have heading the 
organizations that you do. And I am going to thank you for your 
work to implement 21st Century Cures, which obviously as you 
know is a point of real pride for this committee. So thank you 
for that.
    Commissioner Gottlieb, I wanted to ask, I know there have 
been some discussions already about the biosimilars action 
plans that you released. I commend you for that. I have just 
introduced in the last few days something we are calling the 
biosimilars competition act, which is try to get to some of 
these pay for delay agreements that are operating in the 
biologic, biosimilar space in the same way that we have given 
authority to the FTC to kind of police that conduct with 
respect to brand name drugs and in generics where it has been 
consequential for sure. That the authority is there, that they 
can look at these agreements, they can judge whether they are 
fair and appropriate, vis a vis the consumer or not--there are 
tremendous savings to be had there.
    I think maybe the figure is that brand names occupy about 
20 percent of the drugs that are produced every year, but it is 
still 75 percent of the cost that is out there. And I think 
that includes the sort of biologics, biosimilar distinction as 
well. Can you just speak to anything that is a good idea for 
the FTC to have this authority?
    And also, I think you understand from the plan that you put 
forward in your comments generally that you look for ways to 
cooperate with an agency like the FTC around this kind of thing 
to make sure that we are getting these drugs at the price point 
that they should be at.
    Dr. Gottlieb. I thank you for the question, Congressman. I 
haven't looked specifically at the measure, but I will. It is a 
little bit outside the scope of my authorities. I have my own 
equities with FTC in trying to work with them to try to see how 
we can facilitate their interests and potentially bring in 
cases related to delaying tactics it might involve for example 
the REMS where we can help provide information that could 
prompt them to take a look at whether those practices are 
anticompetitive and that has been a big bugaboo of mine as 
well.
    Generally speaking, we like to approve safety biosimilars 
and we like to see them marketed and we like to see patients 
benefiting from them.
    So culturally we like to see drugs marketed. It is the fact 
that most of the biosimilars that have been approved have not 
been marketed for various reasons. The biggest reason is in 
patent delays. It is also a fact that a growing number of small 
molecule generic drugs end as being approved but the drugs are 
never being marketed because of the changing economic dynamic 
to that market as well as. And I think we need to keep a close 
eye on that as well, because competition may be declining in 
the small molecule generic world as well.
    Mr. Sarbanes. Thank you. And we will try to get this tool 
into the toolkit that the government has broadly to make sure 
that we are protecting consumers in that space.
    Let me switch gears real quick. To you, Dr. Collins. And I 
know you got a question earlier I believe about Alzheimer's 
research generally and some of the clinical trials associated 
therewith. But there was an article in The New York Times 
yesterday that pointed specifically to the challenge that is 
presented by trying to find sufficient people to participate in 
these trials.
    There are a lot of trials that are underway or are on deck 
that would suggest the need for up to 25,000 participants in 
these trials. And some of them could be really breakthrough, 
but I gather that this is a real problem, challenge, trying to 
find enough participants.
    Can you speak to what you know about that and what can be 
done about it?
    Dr. Collins. Yes, thank you. That was based upon comments 
made by Dr. Marie Bernard who is the deputy director on the 
National Institute on Aging at the major Alzheimer's meeting 
that was held in Chicago.
    This is a challenge because we are now at the point where 
we are trying to recruit individuals who don't yet have 
cognitive decline, but who are at high risk for Alzheimer's by 
increasingly accurate means that we have, some of them using 
imaging, some using genetics, and we need a lot of participants 
in order to do that. But now we are reaching out to people who 
may not be that motivated to take part in research because they 
are fine, right now.
    We are arguing that that is the best time to intervene, but 
it is not so easy to enroll. I will say one of dreams I have of 
the All of Us program that we have been talking about there 
program where you have a million participants who are 
preconsented for recontact for reach protocols that that would 
be a fantastic group to be able to enroll participants in 
studies like this for common disease, whether it is diabetes, 
or hypertension, or Alzheimer's.
    But right at the moment where we don't have that platform, 
we really have a challenge trying to convince people that this 
is going to be something they want to take part in. We are 
pulling out all the stops. And again the Congress having made 
Alzheimer's such a high priority for us. We have resources in 
order to do that kind of recruitment, but it is not simple 
because it is a different model than what people are used to 
where you get approached about a clinical trial when you 
already have the diagnosis. Here we are approaching people who 
don't have that diagnosis trying to figure out how to prevent 
it.
    Mr. Sarbanes. Thank you very much. I yield back.
    Mr. Burgess. The chair thanks the gentleman. The gentleman 
yields back. The chair recognizes the gentleman from Indiana, 
Dr. Buschon, for 5 minutes of questions, please.
    Mr. Buschon. Thank you, Mr. Chairman. Thank you all for 
being here. Honestly it is pleasure for me to be here. As a 
physician, I never thought I would get to talk to you all. And 
I was at an event with Dr. Sharpless with Steve Rosenberg and 
Diana DeGette recently. And the work that he has done over the 
years. I can't even express how much of an impact he has had.
    Dr. Collins, you just said high risk. Who is high risk for 
Alzheimer's?
    Dr. Collins. Well, anyone who gets to be 85 or 90 is at 
high risk.
    Mr. Buschon. No, I am saying that people who don't know 
they have it, if your mom has it, your dad has it, your 
grandma? Maybe if one of your family members has it, you are 
high risk. I don't know.
    Dr. Collins. So there are there ways in which we are 
currently identifying such folks. One is we that you have a 
very strong family history, almost inherited in a dominant 
fashion. In fact, it is a dominant fashion. And there are those 
families that if you are in one of those circumstances we can 
track the gene in the family and figure out who has it long 
before any symptoms.
    Another way is to look at a genetic risk factor called 
ApoE4. If you have one copy of that, your risk goes up 
threefold. If you have two copies, one from each parent, it 
goes up 15 fold so those people are very high risk. And the 
third way is scanning using a PET scan that picks up amyloid, 
because amyloid starts depositing in your brain probably 20 
years before the first cognitive decline symptoms. So if people 
who are worried about this are willing to do a scan, we may be 
able to say, hey, you are one of those that ought to get into 
this clinical trial.
    Mr. Buschon. So if somebody out there is watching C-SPAN 
right now and they are sitting there going, hmm, I wonder if I 
am high risk. I understand the technical tests and things that 
you do, but people need to say well, my mom had it so I am high 
risk. You know what I am saying?
    Dr. Collins. And indeed that is one.
    Mr. Buschon. That is one of them, right?
    Dr. Collins. There are other ways to try to be more precise 
about it. If people are watching C-SPAN out there and are 
wondering hey should I take part in this? We just heard how 
difficult it has been sometimes to get the word out there. The 
place to go is clinicaltrials.gov which posts the trials on 
Alzheimer's disease and everything else where you can find out 
who is doing a trial, what are the enrollment criteria, who do 
you contact to learn more about it? All of this we do very 
closely with the FDA as a partner.
    Mr. Buschon. Great, thank you.
    Dr. Gottlieb, my wife's an anesthesiologist she is out in 
the field today practicing medicine. She just texted me. She 
didn't even know you guys were here. And she says, hey, can you 
check into the fentanyl shortage, because--no, I am not kidding 
she just texted me.
    Mr. Long. She is watching C-SPAN.
    Mr. Buschon. No, she doesn't. She is actually practicing 
medicine. And we have demonized Fentanyl, the illicit forms of 
Fentanyl, but this is a very, very common anesthetic agent. And 
apparently there is going to be a long term back order on 
Fentanyl. Which is honestly going to be a huge problem.
    You don't need to answer the question, I just want to point 
out that what you are doing on drug shortages has real 
everyday, and you know this, but for everyone else out there, 
clinical implications in the practice of medicine. She says 
Zofran, other common paralytic agents that she uses daily in 
her anesthesia practice are really in short supply. Fentanyl is 
a new one on me though, she just texted me that.
    So thank you for work on that and your response earlier on 
what you all are doing on that. If you have any other further 
comments. Because I have one other question for you.
    Mr. Gottlieb. I will just say these are structural 
problems, as I mentioned before, and whatever is in shortage 
say 6 months from now I guarantee it is going to be something 
different. I have grappled personally with these as well, as a 
hospital based physician, so this impacts patient care. We are 
working very hard on this.
    Mr. Buschon. I know you are. So 21st Century Cures sought 
to implement personalized medicine and seek cures. This is a 
little proprietary question here, for diseases such as cancer 
which rely on diagnostics, and I know your staff probably has 
told you I might ask about this, unfortunately the current 
diagnostic frameworks for laboratory developed tests, LDTS in 
vitro diagnostic test kits haven't been updated since 1988, 
1976, and 1988. That is why Diana DeGette and I and other 
members of the committee have released the Diagnostic Accuracy 
and Innovation Act, DAIA, so to speak, to update how 
diagnostics are regulated.
    We have had a discussion draft out there for a long time 
you all have had that and we really appreciate your input. And 
we have talked to laboratories, patient groups providers and 
others and we really need reform now.
    And so I know you are familiar with the issue and I brought 
it in the past, as well as the committee's last hearing. You 
recently gave us a narrative on what the FDA provided us 
earlier this year in response to DAIA, but I want to get 
diagnostic reform done this Congress hopefully.
    So we are waiting for the red line of the bill from FDA and 
I wanted to know if you have any insight in that progress?
    Dr. Gottlieb. It is very close, Congressman. We have 
completed most of our work and I hope to have it to you very 
soon. I would be happy to come into your office and brief you 
on it as well, but I think we are very close.
    Mr. Buschon. Great. I just want to reiterate you have been 
very helpful and I am very appreciative of that. And we are 
hopeful to get this accomplished this Congress.
    Thank you, Mr. Chairman. I yield back.
    Mr. Burgess. The chair thanks the gentleman. The gentleman 
yields back. The chair recognizes the gentlelady from Indiana, 
Ms. Brooks, 5 minutes for questions, please.
    Mrs. Brooks. Thank you, Mr. Chairman and thank you all for 
your service to our country and for all of the incredible 
medical innovation. I just want to thank you, because you all 
could probably be doing very, very different things in our 
country and yet you are here, working on behalf of all 
citizens, not just here but actually around the globe.
    Dr. Collins, I would like to ask in your testimony you 
mention that there are provisions that provide direction from 
Congress to ensure specific subgroups of patients are 
specifically included in research in clinical trials. I am 
particularly interested in childhood disease and cancers.
    And having heard from constituents who have lost a child to 
cancer and are currently fighting the disease, we know that NIH 
is focused on improvements for children. That is an area that 
has been lacking in the past. Can you elaborate on the 
implementation of the recently passed Childhood Cancer STAR 
Act, which was signed into law in June and what kind of 
innovation are we focused on for childhood cancers and disease?
    Dr. Collins. I ask Dr. Sharpless to respond to that.
    Dr. Sharpless. I thank you for the question on childhood 
cancer. It is important to say two things about childhood 
cancer. While it is true we have made tremendous progress, 
fewer children are dying of cancer than ever, we are still 
having, also true, too many children die of cancer.
    And in particular, even when we are able to cure kids of 
cancer, we often leave them with lifelong toxicity, so they 
have survivorship issues. And I think that is one of the 
principal issues the STAR Act tried to address is the issues of 
novel therapies for kids with cancer, and then also, the burden 
of survivorship that some of these patients incur through their 
curative therapy.
    And so the NCI is really interested in this topic. This was 
a personal research interest of mine when I was in academics, 
so I know firsthand the pain that many of these patients go 
through or the suffering that this entails.
    And I think we have a focus on new research efforts related 
to survivorship, biospecimen acquisition, and then getting new 
advices and new voices into the NCI, to make sure that we are 
adequately advised from the expert community on pressing issues 
in survivorship.
    So thank you for the leadership on that important act.
    Mrs. Brooks. Thank you very much.
    Dr. Gottlieb, as my colleague, Anna Eshoo, talked about, in 
our focus on PAHPA reauthorization, which it is critically 
important we get that reauthorized by the end of September, one 
of the things that has come up in various discussions with Dr. 
Kadlec and others is the importance of platform technology, the 
use of platform technology when it comes to innovation with 
vaccine.
    Can you talk about that to some extent, because there has 
been frustration on this committee about egg-based vaccines 
versus platform technology? Or if anyone would like to comment. 
I am starting with you, but----
    Dr. Gottlieb. I would like to start, if I can. I appreciate 
the question very much. And we have a proposal in the 
President's budget for some additional funding for the agency 
to try to support continuous manufacturing, alternative 
manufacturing. And we think this is really the direction that 
we want to head in, where you have closed, continuous 
manufacturing platforms, particularly with recombinant 
technologies, where you can effectively have, like, cassettes 
that you basically plug into the platform and can allow 
continuous manufacturing of a vaccine.
    And if you want to modify the vaccine, you can literally 
plug in another cassette that codes for a different permutation 
of the same vaccine, same recombinant vaccine.
    We think that this is really the solution or a solution for 
influenza seasonal flu, the ability to scale up manufacturing 
more quickly andproduce vaccines closer to the flu season so 
you can guess the strain better as well as have a platform 
available in the event of a pandemic flu. And these same 
technologies can be used to try to scale up manufacturing of 
other vaccines.
    In an ideal circumstance, what we would have isn't 
mothballed vaccine that degrades over time if it is not stored 
correctly and takes up a lot of space and is costly, but 
platforms that allow quick manufacturing of vaccines should we 
need it.
    Mrs. Brooks. I am assuming, Dr. Collins.
    Dr. Collins. NIH works very closely with FDA in this space. 
If Tony Fauci was here, he would go into this in some detail, 
that the idea of having to build things in eggs is so much 
yesterday's technology.
    And the concrete example this past year, with the flu 
vaccine having been surprisingly ineffective. It turns out that 
the virus mutated in the process of being grown in the eggs; 
and so, therefore, it turned out not to be a particularly 
effective vaccine for something that we didn't have control 
over.
    These new platforms, which allow you to build vaccines in a 
much more rapid fashion, and much more directed fashion, DNA-
based vaccines, RNA-based vaccines. Once you have that platform 
going, you can, very quickly, adapt it to many different 
pathogens. And that is certainly something we are working on 
now, to develop this universal influenza vaccine, which is a 
very high priority, and Congress has given us some additional 
funds for that, so that we wouldn't have to have the yearly 
effort to try to guess right. You would have a vaccine that 
works against virtually all strains and would also be effective 
against that next 1918-style pandemic, which we are all worried 
about and which is overdue.
    Mrs. Brooks. Thank you. And we did include a Pan Flu 
provision in the PAHPA legislation passed out of this 
committee.
    With that, I yield back. Thank you for your work.
    Dr. Collins. Thank you for that.
    Mr. Burgess. The chair thanks the gentlelady. The 
gentlelady yields back.
    The chair recognizes the gentleman from Florida, Mr. 
Bilirakis, 5 minutes for questions.
    Mr. Bilirakis. Thank you, Mr. Chairman. I appreciate it. 
And I thank the panel for their testimony today as well.
    Well, the first question I wanted to ask is, we talked 
about Alzheimer's disease, of course, and what about 
Parkinson's? We are talking maybe how do you know if you are a 
candidate for Parkinson's disease to participate in these 
clinical trials, if there are no symptoms? It may be in the 
early stages. And if you could answer that question, sir, I 
would appreciate it.
    Dr. Collins. There are parallels here that are notable. 
There are genetic risk factors for Parkinson's disease, which 
is interesting, because when I was in training and I asked my 
professor, ``Are there any diseases that don't have genetic 
contributions?'' And he said, ``Oh yes, everybody knows 
Parkinson's is always totally random and sporadic.'' Well, he 
was really wrong.
    So if you have a variant, for instance, in a gene called 
LRRK2, your risk of Parkinson's goes up. If you have a variant 
in a gene alpha-synuclein, your risk goes up. We are beginning 
to, therefore, be able to identify people at high risk and 
invite them to take part in prevention trials.
    Another big thing that has happened in Parkinson's disease 
in just the last 6 months is the formation of a partnership 
with industry called the Accelerating Medicines Partnership for 
Parkinson's Disease with FDA as a critical partner in this as 
well, and really, now, figuring out how we could learn from a 
very large amount of data that is out there, but hadn't been 
brought together, what are the next generation of drug targets 
for Parkinson's disease and how do we accelerate the process of 
getting there? Because we have treatments, L-dopa has been 
around a long time, but we certainly don't have things that 
actually prevent progression. They more treat the symptoms. And 
we believe we could do better with that.
    I should also say the BRAIN Initiative, which is this very 
bold effort supported by 21st Century Cures and the Innovation 
Fund, is learning things about the wiring diagram of the brain 
that is going to be very relevant to some of the things that 
are being done for Parkinson's disease, with direct brain 
stimulation, where you actually put an electrode into the brain 
to try to take care of some of the motor problems. What we do 
right now is kind of clunky. As we learn the wiring diagram, we 
could be much more precise and effective about that.
    Mr. Bilirakis. Well, thank you very much. Anyone else want 
to add something with regard to that? OK. Thank you.
    Dr. Gottlieb, Section 3088 clarifies that FDA has the 
authority to grant emergency use authorization for animal 
drugs, allowing the agency to approve the GMO mosquitoes for, 
again, Florida's Zika problem. We are planning ahead.
    Would you provide an update on the implementation of 
Section 3088, specifically as it relates to approval of the GMO 
mosquitoes?
    Dr. Gottlieb. I can get back to you with a more detailed 
update on that, Congressman. But I will tell you, there has 
been some discussion about the nexus of authority with EPA for 
some of these products. But we did provide a guidance earlier 
this year, I believe, that addressed some of these issues. So I 
can get back to you with more specifics about where that 
stands.
    Mr. Bilirakis. I appreciate that very much.
    Dr. Gottlieb, a second question: As a long-time champion 
and supporter of policies that seek to promote a deeper level 
of patient engagement in the therapy development processes, I 
am pleased with the progress the agency has made under your 
leadership--so congratulations, and we appreciate all you do--
including the FDA's moving ahead to implement the Patient-
Focused Impact Assessment Act provision of Cures that requires 
the FDA to disclose how patient engagement data informed a 
review of any approved product.
    Where is the agency presently in implementing this 
provision, particularly efforts to standardize the inclusion of 
such information in the record of approved drugs so that it is 
accessible and understandable?
    Dr. Gottlieb. We have issued one of four guidances that we 
intend to relay to patient-focused drug development, and we 
have standardized a format for the presentation of patient-
related information in clinical trials.
    So when a clinical trial is submitted to us, there is a 
discrete, a very explicit section for patient-focused 
information. And on the medical device side of our house, we 
have done some similar things: We are seeing a very high rate 
of the use of patient-focused information, and PROs, in the 
development of medical devices as well. So this is a cross-
agency effort across all of our Medical Product Centers. We 
also just stood up a Patient Affairs Office inside the Office 
of the Commissioner reporting in to the principal deputy that 
is going to help advance some of these policies, really, a 
coordinating office to provide a focus of access for patient 
groups, but also, a focus of policymaking when it is cross-
agency policymaking around these issues.
    Mr. Bilirakis. OK. Thank you very much.
    I yield back, Mr. Chairman.
    Mr. Burgess. The chair thanks the gentleman. The gentleman 
yields back.
    The chair recognizes the gentleman from Georgia, Mr. 
Carter, 5 minutes for questions, please.
    Mr. Carter. Thank you, Mr. Chairman. And thank all of you 
for being here and thank you for your efforts in implementing 
21st Century Cures, legislation that I personally feel like is 
some of the best legislation we have passed in Congress in 
quite a while, so we are very proud of, particularly coming out 
of this committee.
    Dr. Collins, I will start with you. I wanted to ask you 
particularly about one of the initiatives of 21st Century 
Cures, and that was to, really, review the regulations and 
policies with respect to research in laboratory animals. And as 
I understand it, you are working with USDA now and the FDA to 
try to complete a review of that. And I just wanted to ask you 
if you could tell us the current status of that review, and 
when do you anticipate the completion of that review?
    Dr. Collins. I appreciate the question. We are very 
seriously engaged in this. And, again, 21st Century Cures gave 
us some clear guidance about what we ought to engage in. We did 
put out an RFI back in March to ask for comments in this area, 
in terms of whether the oversight that we currently apply to 
animal experimentation is sufficient or whether it has areas 
that are overly bureaucratic, which has been a concern.
    Obviously, we are deeply concerned about maintaining our 
ethical responsibilities, in terms of how we take care of 
animals that are subjected to various experimental approaches, 
from which we learn a great deal that has led to many medical 
advances.
    We got 19,000 responses to that request, and they are 
currently being sorted at the moment. We would expect, 
therefore, to have, based upon those, a draft set of 
recommendations about animal care and use, sometime probably in 
September. We will then need to have responses to that. And so 
we would hope to have a final version of this by December or 
early in 2019.
    Mr. Carter. Great, great. Any opportunities that you have 
identified thus far that may help you?
    Dr. Collins. I think there are concerns that some of the 
requirements we put on grant applicants in terms of animal care 
and use could be delayed until the award is actually made as 
opposed to asking them to have all of those things in place 
when they submit an application, because that can add a lot of 
time and effort.
    And obviously, our concern is, if we are going to actually 
make the award, we want to be sure that the animal care is 
being done in the best possible way. That is one area. 
Obviously, there are differences of opinion here.
    Mr. Carter. Yes.
    Dr. Collins. And we are seeing those in those 19,000 
responses. And at some point, we have to try to come down in 
what we think is a fair and balanced approach.
    Mr. Carter. And I am sure you will reach that. I am very 
confident. Let me switch gears here and talk about something 
that is very exciting to me as a pharmacist, and that is 
precision medicine. And that is something that I see in genomic 
testing and everything is something I see as the wave of the 
future, and great opportunities for us in healthcare.
    But I am concerned, when we get all this data in, we are 
struggling already with our electronic health records. How are 
we going to handle this? I want to ask you that, and then I 
would like to ask Dr. Gottlieb as well.
    Dr. Collins. Well, I will say a word, and then I will ask 
Dr. Devaney to say a little more, as the Deputy Director of the 
All of Us Program. We are very invested in looking at this in 
the fashion that is cutting-edge, as far as dealing with very 
large datasets, putting the data into the cloud.
    We just had a very interesting all-day workshop on Monday 
on artificial intelligence and machine learning and how that 
can be applied to these unprecedented datasets to glean the 
maximum amount of information out of there while, at the same 
time, maintaining the confidentiality, the security systems 
that the participants in this are going to expect about their 
deidentified data.
    But maybe, Dr. Devaney, you want to say a little more?
    Mr. Carter. Sure, please.
    Ms. Devaney. I would love to add to that. So one of the 
data types that is going to be, I think, the most essential to 
precision medicine, or one of is information from electronic 
health records. And this is one of the largest challenges for 
our program.
    We have direct partnerships with many healthcare 
organizations, and we are getting those data continuously from 
those partnerships, but we are also working on other 
strategies, including one in partnership with four of the 
largest her vendors right now, to work on making the data 
transmission much more seamless across provider lines and into 
the program when a participant authorizes it.
    Mr. Carter. Great. Dr. Gottlieb, I know this is important 
in drug data development as well.
    Dr. Gottlieb. I appreciate the question. I would just take 
a step up the continuum to try to make effective use of the 
data. Because there is so much information, how can we make 
effective use of the data in a way that it can translate to 
clinical benefits to patients. And I think this is where some 
of the issues we talked about earlier around clinical trial 
innovation comes in where you have the ability now, with a 
seamless clinical trial design, to effectively buy us 
enrollment in the trial for some of the genomic information and 
predictive information that is likely to predict who is more 
likely to benefit from a treatment, and who is less likely to 
experience a side effect.
    And so if we can use this information in that way to 
structure trials and enrollment, we can end up with much more 
information about who is likely to benefit from a drug and more 
tools to make sure the right drug gets to the right patient at 
the right time. We have talked about this for decades. We now 
have that technology at hand.
    Mr. Carter. And I can sense the excitement in your voice. 
And it is exciting for me, as a healthcare professional as 
well. I just look into the future of this and just think, Wow, 
what we have got to look forward to. Thank you.
    And I yield back.
    Mr. Burgess. The chair thanks the gentleman. The gentleman 
yields back.
    The chair needs to state that, without objection, all 
members' opening statements will be made part of the record.
    The chair asks if the gentleman from Texas has a unanimous 
consent request concerning Alzheimer's. Were you going to ask 
that additional information be forwarded to the committee about 
Alzheimer's?
    Mr. Green. If you could just send it to the committee on 
some of the information you couldn't have given us today 
because of the time limits.
    Dr. Collins. Happy to do that.
    Mr. Green. Thank you. I forgot what I was going to ask.
    Mr. Burgess. And also, the chair would like to make the 
observation that, Dr. Collins, you started out this hearing 
with the remark about the immunotherapy and some of the 
dramatic things that have occurred. And it predated our work, 
our passage of the Cures bill, but a former President of the 
United States, in July of 2015, went public with the 
information that he had metastatic melanoma to the brain and 
the liver. And remembering my time in medical school, my 
initial thought was, we will not have this individual with us 
by Labor Day. But it has really been dramatic to see him a year 
later deliver a speech at the Democratic Convention. A year and 
a half, then he was present at the inauguration in January 
2017. I don't know what his clinical status is now, but it was 
truly dramatic.
    And, again, all of you are to be congratulated for making 
that possible. And hats off to former President Carter for 
going public with the information and entering a clinical 
trial, because that is the way information is gathered and 
learned. So, again, I felt obligated to make mention of that 
milestone.
    Seeing that there are no further members wishing to ask 
questions, I do want to thank our witnesses for being here 
today. Pursuant to committee rules, I remind members they have 
10 business days to submit additional questions for the record. 
I ask the witnesses to submit their responses in receipt of 
those questions.
    Without objection, the subcommittee is adjourned.
    [Whereupon, at 11:17 a.m., the subcommittee was adjourned.]