[House Hearing, 116 Congress]
[From the U.S. Government Publishing Office]
IMPROVING ACCESS TO CARE: LEGISLATION TO
REAUTHORIZE KEY PUBLIC HEALTH PROGRAMS
=======================================================================
VIRTUAL HEARING
BEFORE THE
SUBCOMMITTEE ON HEALTH
OF THE
COMMITTEE ON ENERGY AND COMMERCE
HOUSE OF REPRESENTATIVES
ONE HUNDRED SIXTEENTH CONGRESS
SECOND SESSION
__________
JULY 29, 2020
__________
Serial No. 116-123
[GRAPHIC NOT AVAILABLE IN TIFF FORMAT]
Printed for the use of the Committee on Energy and Commerce
govinfo.gov/committee/house-energy
energycommerce.house.gov
__________
U.S. GOVERNMENT PUBLISHING OFFICE
55-868 PDF WASHINGTON : 2024
-----------------------------------------------------------------------------------
COMMITTEE ON ENERGY AND COMMERCE
FRANK PALLONE, Jr., New Jersey
Chairman
BOBBY L. RUSH, Illinois GREG WALDEN, Oregon
ANNA G. ESHOO, California Ranking Member
ELIOT L. ENGEL, New York FRED UPTON, Michigan
DIANA DeGETTE, Colorado JOHN SHIMKUS, Illinois
MIKE DOYLE, Pennsylvania MICHAEL C. BURGESS, Texas
JAN SCHAKOWSKY, Illinois STEVE SCALISE, Louisiana
G. K. BUTTERFIELD, North Carolina ROBERT E. LATTA, Ohio
DORIS O. MATSUI, California CATHY McMORRIS RODGERS, Washington
KATHY CASTOR, Florida BRETT GUTHRIE, Kentucky
JOHN P. SARBANES, Maryland PETE OLSON, Texas
JERRY McNERNEY, California DAVID B. McKINLEY, West Virginia
PETER WELCH, Vermont ADAM KINZINGER, Illinois
BEN RAY LUJAN, New Mexico H. MORGAN GRIFFITH, Virginia
PAUL TONKO, New York GUS M. BILIRAKIS, Florida
YVETTE D. CLARKE, New York, Vice BILL JOHNSON, Ohio
Chair 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 TIM WALBERG, Michigan
SCOTT H. PETERS, California EARL L. ``BUDDY
DEBBIE DINGELL, Michigan CARTER, Georgia
MARC A. VEASEY, Texas JEFF DUNCAN, South Carolina
ANN M. KUSTER, New Hampshire GREG GIANFORTE, Montana
ROBIN L. KELLY, Illinois
NANETTE DIAZ BARRAGAN, California
A. DONALD McEACHIN, Virginia
LISA BLUNT ROCHESTER, Delaware
DARREN SOTO, Florida
TOM O'HALLERAN, Arizona
------
Professional Staff
JEFFREY C. CARROLL, Staff Director
TIFFANY GUARASCIO, Deputy Staff Director
MIKE BLOOMQUIST, Minority Staff Director
Subcommittee on Health
ANNA G. ESHOO, California
Chairwoman
ELIOT L. ENGEL, New York MICHAEL C. BURGESS, Texas
G. K. BUTTERFIELD, North Carolina, Ranking Member
Vice Chair FRED UPTON, Michigan
DORIS O. MATSUI, California JOHN SHIMKUS, Illinois
KATHY CASTOR, Florida BRETT GUTHRIE, Kentucky
JOHN P. SARBANES, Maryland H. MORGAN GRIFFITH, Virginia
BEN RAY LUJAN, New Mexico GUS M. BILIRAKIS, Florida
KURT SCHRADER, Oregon BILLY LONG, Missouri
JOSEPH P. KENNEDY III, LARRY BUCSHON, Indiana
Massachusetts SUSAN W. BROOKS, Indiana
TONY CARDENAS, California MARKWAYNE MULLIN, Oklahoma
PETER WELCH, Vermont RICHARD HUDSON, North Carolina
RAUL RUIZ, California EARL L. ``BUDDY'' CARTER, Georgia
DEBBIE DINGELL, Michigan GREG GIANFORTE, Montana
ANN M. KUSTER, New Hampshire GREG WALDEN, Oregon (ex officio)
ROBIN L. KELLY, Illinois
NANETTE DIAZ BARRAGAN, California
LISA BLUNT ROCHESTER, Delaware
BOBBY L. RUSH, Illinois
FRANK PALLONE, Jr., New Jersey (ex
officio)
C O N T E N T S
----------
Page
Hon. Anna G. Eshoo, a Representative in Congress from the State
of California, opening statement............................... 2
Prepared statement........................................... 3
Hon. Michael C. Burgess, 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........................................... 8
Hon. Greg Walden, a Representative in Congress from the State of
Oregon, opening statement...................................... 9
Prepared statement........................................... 10
Witnesses
Linda Goler Blount, M.P.H., President and CEO, Black Women's
Health Imperative.............................................. 12
Prepared statement........................................... 14
Robert Boyd, M.C.R.P., M. Div., President, School-Based Health
Alliance....................................................... 16
Prepared statement........................................... 18
Answers to submitted questions............................... 160
Nancy Goodman, M.P.P., J.D., Founder and Executive Director, Kids
V Cancer....................................................... 25
Prepared statement........................................... 27
Aaron Seth Kesselheim, M.D., J.D., M.P.H., Professor Of Medicine,
Harvard Medical School......................................... 30
Prepared statement........................................... 33
Brian Lindberg, Chief Legal Officer and General Counsel, National
Bone Marrow Donor Program...................................... 43
Answers to submitted questions............................... 46
Prepared statement........................................... 164
Travis T. Tygart, Chief Executive Officer, U.S. Anti-Doping
Agency......................................................... 53
Prepared statement........................................... 55
Answers to submitted questions............................... 168
Submitted Material
H.R. 2075, School-Based Health Centers Reauthorization Act of
2019........................................................... 106
H.R. 4078, EARLY Act Reauthorization of 2019..................... 108
H.R. 4439, Creating Hope Reauthorization Act..................... 110
H.R. 4764, Transplant Act of 2019................................ 112
H.R. 5373, United States Anti-Doping Agency Reauthorization Act
of 2019........................................................ 116
Statement of July 13, 2020, from the National Marrow Donor
Program, submitted by Ms. Eshoo................................ 121
Letter of July 28, 2020, to Mr. Pallone and Mr. Walden, from
Council of State Bioscience Associations, submitted by Ms.
Eshoo.......................................................... 123
Letter of July 28, 2020, to Mr. Pallone and Mr. Walden, from Rep.
Mike Thompson, submitted by Ms. Eshoo.......................... 126
Statement of July 29, 2020, Joanna Kurtzberg, President, Cord
Blood Association, submitted by Ms. Eshoo...................... 128
Report of January 2020, ``Drug Development: FDA's Priority Review
Voucher Programs''\1\
Article of February 2019, ``Impact Of the Priority Review Vouches
Program on Drug Development For Rare Pediatric Diseases,'' from
Thomnas J. Hwang, et al., Pharmaceuticals and Medical
Technology, submitted by Ms. Eshoo............................. 135
Letter of July 23, 2020, to Mr. Pallone and Mr. Walden, from the
Bone Marrow, PBSC, Cell Therapy, and Cord Blood Transplant
Coalition, submitted by Ms. Eshoo.............................. 142
Letter of July 29, 2020, to Ms. Eshoo and Mr. Burgess, by Rachel
Sher, J.D., M.P.H., Vice President, Policy and Regulatory
Affairs, National Organization for Rare Disorders, submitted by
Ms. Eshoo...................................................... 146
Letter of July 28, 2020, to Ms. Eshoo and Mr. Burgess, by
Victoria A. M. Wolodzko, Senior Vice President, Mission, Susan
G. Kome, submitted by Ms. Eshoo................................ 148
Letter of July 28, 2020, to Mr. Pallone and Mr. Walden,
Biotechnology Companies Coalition, submitted by Ms. Eshoo...... 150
Letter of July 22, 2020, to Mr. Alexander, et al., from Coalition
Pediatric Medical Research, submitted by Ms. Eshoo\2\
Letter on July 29, 2020, to Mr. Pallone and Mr. Walden, by Nancy
Houlihan, MA, RN, AOCN, President, Oncology Nursing Society,
submitted by Ms. Eshoo......................................... 152
Slides of July 29, 2020, by Robert Boyd, President, School-Based
Health Alliance, submitted by Ms. Eshoo \3\
Guide, School-Based Health Alliance, submitted by Ms. Eshoo...... 153
Addendum, Slide Presentation--Written Testimony for the Record,
submitted by Ms. Eshoo......................................... 154
----------
\1\ The information has been retained in committee files and also
is available at https://docs.house.gov/meetings/IF/IF14/
20200729/110949/HMTG-116-IF14-20200729-SD007.pdf.
\2\ The information has been retained in committee files and also
is available at https://docs.house.gov/meetings/IF/IF14/
20200729/110949/HMTG-116-IF14-20200729-SD013.pdf.
\3\ The information has been retained in committee files and also
is available at https://docs.house.gov/meetings/IF/IF14/
20200729/110949/HMTG-116-IF14-20200729-SD015.pdf.
IMPROVING ACCESS TO CARE: LEGISLATION TO REAUTHORIZE KEY PUBLIC HEALTH
PROGRAMS
----------
WEDNESDAY, JULY 29, 2020
House of Representatives,
Subcommittee on Health,
Committee on Energy and Commerce,
Washington, DC.
The subcommittee met, pursuant to call, at 10:00 a.m., via
Cisco Webex online video conferencing, Hon. Anna G. Eshoo
(chairwoman of the subcommittee) presiding.
Members present: Representatives Eshoo, Engel, Butterfield,
Matsui, Castor, Sarbanes, Schrader, Kennedy, Cardenas Welch,
Ruiz, Dingell, Kuster, Kelly, Barragan, Blunt Rochester, Rush,
Pallone (ex officio), Burgess (subcommittee ranking member),
Upton, Guthrie, Griffith, Bilirakis, Long, Bucshon, Brooks,
Mullin, Carter, Gianforte, and Walden (ex officio).
Also present: Representatives O'Halleran and Johnson of
Ohio.
Staff present: Joe Banez, Professional Staff Member; Billy
Benjamin, Systems Administrator; Jeffrey C. Carroll, Staff
Director; Kimberly Espinosa, Professional Staff Member; Waverly
Gordon, Deputy Chief Counsel; Stephen Holland, Health Counsel;
Aisling McDonough, Policy Coordinator; Meghan Mullon, Policy
Analyst; Joe Orlando, Staff Assistant; Kaitlyn Peel, Digital
Director; Tim Robinson, Chief Counsel; Rebecca Tomilchik, Staff
Assistant; Kimberlee Trzeciak, Chief Health Advisor; C. J.
Young, Press Secretary; Nolan Ahern, Minority Professional
Staff, Health; Mike Bloomquist, Minority Staff Director; S. K.
Bowen, Minority Press Secretary; William Clutterbuck, Minority
Staff Assistant; Theresa Gambo, Minority Human Resources/Office
Administrator; Caleb Graff, Minority Professional Staff,
Health; Tyler Greenberg, Minority Staff Assistant; Tiffany
Haverly, Minority Communications Director; Peter Kielty,
Minority General Counsel; Ryan Long, Minority Deputy Staff
Director; James Paluskiewicz, Minority Chief Counsel, Health;
Brannon Rains, Minority Policy Analyst; Kristin Seum, Minority
Counsel, Health; Kristen Shatynski, Minority Professional Staff
Member, Health; and Everett Winnick, Minority Director of
Information Technology.
Ms. Eshoo. OK. I am going to gavel in, and the Subcommittee
on Health will now come to order.
Due to COVID-19, today's hearing is being held remotely,
and all members and witnesses will be participating via video
conferencing.
As part of our hearing, the microphones--as you know, but
it is a reminder--the microphones will be on mute to eliminate
background noise, and members and witnesses will need to unmute
your microphone each time you wish to speak. So that is every
time. So turn it on when you go to speak. Turn it off so that
there isn't any background noise when you are not speaking.
Documents for the record can be sent to Meghan Mullon at
the email address that we have provided your staff with, and
all documents will be entered into the record at the conclusion
of the hearing.
The Chair now recognizes herself for 5 minutes for an
opening statement.
OPENING STATEMENT OF HON. ANNA G. ESHOO, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF CALIFORNIA
Today our subcommittee is considering five bills to
reauthorize important public health programs that support and
improve the health and well-being of children in our country,
as well as adults.
While so much of our subcommittee's focus and work is on
the COVID-19 pandemic that has taken the lives of 150,000
Americans, we also have to continue our essential work during
the crisis, which means making sure that health programs
nearing their expiration are continued, that they are improved,
and in some cases expanded.
So today we are going to hear testimony on five bipartisan
reauthorization bills. Several of these bills support
individuals in the fight against cancer.
The Creating Hope Reauthorization Act, sponsored by
Representatives Butterfield and McCaul, help children access
pediatric cancer drugs.
Pediatric cancer is the number one disease killer of
American children, but pharmaceutical companies often avoid
developing pediatric cancer drugs because of the very small
market and the high risks that are associated with studying and
testing drugs for children.
The Creating Hope Act provides incentives for the research
and development of pediatric cancer drugs by providing the
developers with the valuable priority review vouchers which
allow the recipient to speed up the FDA review of any one of
its new drug products.
The next bill, the TRANSPLANT Act, sponsored by
Representatives Matsui and Bilirakis, helps those with blood
cancers, like leukemia and lymphoma, to be matched with a
potential bone marrow and cord blood donor.
Through this matching program, over 100,000 lives have been
saved, so clearly this works. And that law is a source of pride
to all of us.
The EARLY Act, sponsored by Representatives Wasserman
Schultz and Brooks, increases funding for the successful CDC
outreach and education campaign that informs young people about
breast cancer risks.
Each year, over 300,000 women are diagnosed with breast
cancer. I have spent my entire service in Congress to make sure
women have access to breast cancer treatment, including
reconstructive surgery after mastectomies, and I am really
pleased to consider this important program today.
Now, outside of public health programs that help in the
fight against cancer, we are also considering a bill to
reauthorize the School-Based Health Center Program, sponsored
by Representatives Sarbanes and Stefanik.
About 2,500 school-based health centers serve 3.6 million
American children. These health centers provide children with
immunizations, mental health support, asthma, and allergy
screenings, and many other vital services. It really is a
terrifically effective program.
And finally, we are considering a bill to make sure that
the U.S. Anti-Doping Agency is prepared for the 2028 Olympics
in Los Angeles. The sponsors are Representatives Mike Thompson,
Diana DeGette, and Mr. Johnson.
The Agency is an independent body that manages the anti-
doping program for America's athletes to ensure they are
playing clean.
So as we struggle with the pandemic, with COVID-19, and the
crisis that it is in our country, we have to keep up our work
for the American people, those with cancer, our Nation's
children, and our athletes training for future events, and that
is exactly what we are doing today. So I stand ready to work
with each of you to make sure that these programs are
reauthorized.
[The prepared statement of Ms. Eshoo follows:]
Prepared Statement of Hon. Anna G. Eshoo
Today our Subcommittee will consider five bills to
reauthorize important public health programs that support and
improve the health and wellbeing of children and adults.
While so much of our Subcommittee's focus and work is on
the COVID-19 pandemic which has killed 150,000 Americans, we
also have to continue our essential work during the crisis,
which means making sure that health programs nearing their
expiration are continued, improved, and in some cases expanded.
Today we will hear testimony on five bipartisan
reauthorization bills.
Several of these bills support individuals in the fight
against cancer.
The Creating Hope Reauthorization Act sponsored by
Representatives Butterfield and McCaul helps children access
pediatric cancer drugs.
Pediatric cancer is the number one disease killer of
American children, but pharmaceutical companies often avoid
developing pediatric cancer drugs because of the small market
and the high risks associated with studying and testing drugs
for children.
The Creating Hope Act provides incentives for the research
and development of pediatric cancer drugs by providing the
developers with the valuable Priority Review Vouchers which
allow the recipient to speed up the FDA review of any one of
its new drug products.
The TRANSPLANT Act sponsored by Representatives Matsui and
Bilirakis helps those with blood cancers like leukemia and
lymphoma to be matched with a potential bone marrow and cord
blood donor. Through this matching program, over 100,000 lives
have been saved.
The EARLY Act sponsored by Representatives Wasserman-
Schultz and Brooks increases funding for the successful CDC
outreach and education campaign that informs young people about
breast cancer risks. Each year, over 300,000 women are
diagnosed with breast cancer.
I've spent my entire service in Congress to make sure women
have access to breast cancer treatment, including
reconstructive surgery after mastectomies, and I'm pleased to
consider this important program today.
Outside of public health programs that help the fight
against cancer, we're also considering a bill to reauthorize
the School-Based Health Center program sponsored by
Representatives Sarbanes and Stefanik. About 2,500 school-based
health centers serve 3.6 million children. These health centers
provide children with immunizations, mental health support,
asthma and allergy screenings, and many other vital services.
Finally, we're considering a bill to make sure that the
U.S. Anti-Doping Agency is prepared for the 2028 Olympics in
Los Angeles. The sponsors of this bill are Representatives
Thompson, DeGette, and Johnson. The Agency is an independent
body that manages the anti-doping program for America's
athletes to ensure they're playing "clean."vAs we struggle with
the COVID-19 crisis, we have to keep up our work for Americans
with cancer, our nation's children, and athletes training for
future events.
I stand ready to work with my colleagues to make sure these
programs are reauthorized.
Ms. Eshoo. And with that, I will yield back my time. And
the chair now recognizes Dr. Burgess, the ranking member of our
subcommittee, for 5 minutes for his opening statement. And
please remember to unmute.
Thank you, colleagues.
OPENING STATEMENT OF HON. MICHAEL C. BURGESS, A REPRESENTATIVE
IN CONGRESS FROM THE STATE OF TEXAS
Mr. Burgess. Very well. I thank the Chair. I trust I have
successfully unmuted.
This is an important hearing today to discuss the
reauthorization of five public health programs: the school-
based health centers, the young women's breast health education
and awareness, the Stem Cell and Therapeutic Research Act, and
the United States Anti-Doping Agency Reauthorization Act, and
perhaps most importantly, the Pediatric Priority Review Voucher
Program.
All five of these bills provide tools for individuals in
all stages of life to stay healthy and to save lives. So I am
grateful that the committee has organized this discussion on
these important measures of expiring reauthorizations and
potentially providing hope and reassurance to many Americans in
what has turned out to be a very difficult time.
Often serving as a lifeline for access to care to many
children, H.R. 2075 would reauthorize Federal support for
school-based health centers, while also including federally
qualified health centers as eligible providers.
Generally administered as a partnership between hospital,
schools, and local organizations, school-based health centers
provide comprehensive care for students through important
services, such as primary medical care, behavioral care, and
even substance disorder counseling.
These services are offered in school, a setting with which
students are familiar and comfortable. This convenient setting
makes these services more accessible for many students,
especially those high risk, and certainly underscores the
importance of opening schools.
Furthermore, ensuring access to preventive care allows for
early intervention and treatment before a condition might
worsen.
This was first authorized in 2010. The EARLY Act
reauthorizes the Young Women's Breast Health Education and
Awareness Requires Learning Young Act. This piece of
legislation provides women with meaningful information,
teaching women, especially young women, the importance of
breast health and the risk factors associated with breast
cancer, education and awareness, and the critical steps in
preventive care.
With a history starting in the 1980s, the C.W. Bill Young
Cell Transplantation Program has supported over 92,000 blood
stem cell transplants. With 12,000 Americans diagnosed with
blood cancer and disorder every year, H.R. 4764, the TRANSPLANT
Act, would reauthorize the C.W. Bill Young Transplantation
Program and the National Cord Blood Inventory, providing
resources and support to those who need a donor or a cord blood
unit.
Diseases like sickle cell anemia or blood cancer often rely
on bone marrow or cord blood transplant for treatment. However,
70 percent of those with a blood disorder or cancer do not have
a matched donor. H.R. 4764 will help maintain that program.
And I do want to acknowledge over the years this program
has been one that has been championed by Representative Chris
Smith of New Jersey, and certainly he has been responsible for
getting us to where we are today with the cord blood industry.
In 1999, the U.S. Olympic Committee launched the United
States Anti-Doping Agency to oversee and enforce anti-doping
programs for our Nation's athletes. The Anti-Doping Agency
Reauthorization Act would ensure that the USADA has the
resources needed to encourage healthy sportsmanship among
American athletes, especially as sports teams begin playing
again.
Our athletes often serve as role models for our children,
making the mission of USADA all the more significant. Our
children must learn the value and importance of clean
sportsmanship.
Parenthetically, it is interesting that in 2014 one of the
earliest attacks of Russian interference was in the anti-doping
agency that was actually administered through, it turned out to
be, the Russian military.
Finally, I am encouraged to see H.R. 4439, the Creating
Hope Reauthorization Act, included in today's hearing. It is an
important bill that would make permanent the Pediatric Rare
Disease Priority Review Voucher Program, an incentive program
to encourage American drug innovation for rare and pediatric
diseases.
Because of the complexity and expense required to invest in
pediatric drugs, the FDA had only approved two pediatric
oncology drugs in 22 years leading up to the Creating Hope Act,
which was first signed into law in 2012.
Since the enactment of this program, the FDA has approved
22 drugs. Unfortunately, the program does expire on September
30 of this year, so it does require our immediate attention.
So I hope we can continue to work on these bills in a
bipartisan manner and get these reauthorizations across the
finish line. They are all critical.
I thank our witnesses for sharing their time and expertise
today.
And I will yield back the balance of my time.
[The prepared statement of Mr. Burgess follows:]
Prepared Statement of Hon. Michael C. Burgess
Thank you, Madam Chair. Today we are here to discuss the
importance of reauthorizing five public health programs:
School-Based Health Centers, the Young Women's Breast Health
Education and Awareness Requires Learning Young Act, the Stem
Cell Therapeutic and Research Act, the United States Anti-
Doping Agency Reauthorization Act, and the Pediatric Priority
Review Voucher Program. All five of these bills provide various
tools for individuals in all stages in life, to stay healthy
and even save lives. I am grateful that the committee organized
this discussion on these important measures, potentially
providing hope and reassurance to many Americans depending on
these programs in this difficult time.
Often serving as lifeline for access to care for many
children, H.R. 2075, would reauthorize federal support for
School-Based Health Centers while also including Federally
Qualified Health Centers as eligible providers. Generally
administrated as a partnership between hospitals, schools, and
local organizations, school-based health centers provide
comprehensive care for students through important services such
as primary medical care, behavioral care, and even substance
disorder counseling to 3.6 million k-12 students nationwide.
These services are offered in school, a setting with which
students are familiar and comfortable, this convenient setting
makes these services more accessible for many students,
especially those of high-risk.
Furthermore, ensuring access to preventative care allows
for early intervention and treatment before the condition
worsens. First authorized in 2010, The EARLY Act reauthorizes
the Young Women's Breast Health Education, and Awareness
Requires Learning Young Act. This piece of legislation provides
women with meaningful information, teaching women, especially
young women, the importance of breast health and the risk
factors associated with breast cancer. Education and awareness
are a critical steps in preventative care, and with the breast
cancer survival rate averaging 93 percent if detected at an
early stage, we know it can help save lives.
With a history starting in the 1980s, the C.W. Bill Young
Cell Transplantation Program has supported over 92,000 blood
stem cell transplants. With 12,000 Americans diagnosed with
blood cancer and disorders every year, H.R. 4764, the
TRANSPLANT Act, would reauthorize the C.W. Bill Young Cell
Transplantation Program and the National Cord Blood Inventory,
providing resources and support to those who need a donor or
cord blood unit. Diseases like sickle cell anemia or blood
cancer often rely on bone marrow or cord blood transplant for
treatment; however 70 percent of those with a blood disorder or
cancer do not have a matched donor. H.R. 4764 will help
maintain this program.
In 1999, the U.S. Olympic Committee launched the United
States Anti-Doping Agency, or USADA, to oversee and enforce
anti-doping programs for our Nations' athletes. The Anti-Doping
Agency Reauthorization Act would ensure that the USADA has the
resources needed to encourage healthy sportsmanship among
American athletes, especially as sports teams begin playing
again. Our athletes often serve as role models for our
children, making the mission of the USADA all the more
significant. Our children must learn the value and importance
in clean sportsmanship, and what better way to learn about
these values than through the athletes they so admire.
Finally, I am encouraged to see H.R. 4439, the Creating
Hope Reauthorization Act, included in today's hearing. This
important piece of legislation would make permanent the
pediatric rare disease priority review voucher program, an
incentive program to encourage American drug innovation for
rare pediatric diseases. Because of the complexity and expense
required to invest in pediatric drugs, the FDA had only
approved two pediatric oncology drugs in the twenty years
leading up to the Creating Hope Act, which was first signed
into law in 2012. Since the enactment of this program, the FDA
has approved 22 drugs for rare pediatric diseases. Consider all
the young lives this program has helped improve and maybe even
save. Clearly these outcomes signal success and the importance
in passing this piece of legislation.
I do hope we can continue to work in a bipartisan manner to
get these reauthorizations across the finish line in a timely
manner. I thank our witnesses for sharing their time and
expertise today.
Ms. Eshoo.
Mr. Pallone. I couldn't hear Anna. Am I supposed to speak
now?
Ms. Eshoo. Yes. I just introduced you.
OPENING STATEMENT OF HON. FRANK PALLONE, Jr., A REPRESENTATIVE
IN CONGRESS FROM THE STATE OF NEW JERSEY
Mr. Pallone. All right. Thank you.
Mr. Pallone. So today we continue the committee's ongoing
work to improve the health and well-being of Americans by
discussing legislation that will reauthorize five important
public health programs.
While we continue to prioritize the critical response to
the COVID-19 pandemic that is devastating our Nation, it is
also essential that we continue our work to improve access to
care.
So we are going to hear from public health experts about
what is working and what considerations this committee should
take into account as we move forward on the five bills. I know
that Chairwoman Eshoo has already described the bills, but let
me just a say little more about them.
The first bill, H.R. 2075, the School-Based Health Centers
Reauthorization Act, authored by Representatives Sarbanes,
Tonko, and Upton, would reauthorize these centers.
Those health centers are a powerful tool for achieving
health equity among children and adolescents who unjustly
experience disparities in outcomes because of their race and
family income. The authorization for school-based health
centers lapsed in 2014, and it is important that we strengthen
those programs with additional Federal funds.
The next bill, H.R. 4078, The EARLY Act Reauthorization was
introduced by Representatives Wasserman Schultz and Brooks, and
it authorizes the Centers for Disease Control and Prevention to
develop initiatives to increase knowledge of breast cancer
among women, particularly for women under the age of 40 and
those at heightened risk for developing the disease.
Breast cancer, we know, has impacted too many families, and
it is important that the CDC continue its work.
We are also going to bring up H.R. 4439, the Creating Hope
Reauthorization Act, cosponsored by many members of the
committee. This bill would permanently authorize the Rare
Pediatric Disease Priority Review Voucher Program.
This program has provided value to pharmaceutical companies
who have made investments in rare disease programs, but it also
places a strain on the FDA. So we have to keep in mind that we
should think carefully about whether a permanent authorization
makes sense as opposed to shorter term.
Next, H.R. 4764, the TRANSPLANT Act of 2019, reauthorizes
the C.W. Bill Young Transplantation Program. This provides
patients who need life-saving bone marrow and umbilical cord
blood transplants with info and support as they go through the
process. It also maintains an efficient process for identifying
donor matches, increases the number of nonfamilial donors
available for transplant, and expands data and research to
improve patient outcomes.
Our colleagues Representatives Matsui and Bilirakis have
authored this and worked to secure funding for this program for
many years.
And finally H.R. 5373, the U.S. Anti-Doping Agency
Reauthorization Act. The Anti-Doping Agency is the national
organization in the U.S. for Olympic, Paralympic, Pan American,
and Parapan American sports. This reauthorization would not
only promote clean sports through testing, education, and
research, but also use a portion of its funding to promote a
positive youth sport experience.
So I want to thank all the witnesses, and I would like to
yield the time that remains to Representative Sarbanes.
[The prepared statement of Mr. Pallone follows:]
Prepared Statement of Mr. Pallone
Today we continue this Committee's ongoing work to improve
the health and wellbeing of Americans by discussing legislation
that will reauthorize five important public health programs.
While we continue to prioritize the critical response to the
COVID-19 pandemic that is devastating our nation, it is also
essential that we continue our work to improve access to care.
Today we will hear from public health experts about what is
working and what considerations this Committee should take into
account as we move forward on these five bills.
The first bill, H.R. 2075, the "School-Based Health Centers
Reauthorization Act," which is authored by Representatives
Sarbanes, Tonko, and Upton, would reauthorize school-based
health centers. These health centers are a powerful tool for
achieving health equity among children and adolescents who
unjustly experience disparities in outcomes because of their
race and family income. The authorization for school-based
health centers lapsed in 2014, and it is important that we
strengthen these programs with additional federal funding.
The next bill, H.R. 4078, the "EARLY Act Reauthorization,"
was introduced by Representatives Wasserman Schultz and Brooks.
The EARLY Act authorizes the Centers for Disease Control and
Prevention (CDC) to develop initiatives to increase knowledge
of breast cancer among women, particularly for women under the
age of 40 and those at heightened risk for developing the
disease. Breast cancer is a tragic disease that has impacted
too many families and it is important that the CDC continues
this work.
We will also discuss H.R. 4439, "The Creating Hope
Reauthorization Act," which is cosponsored by many Members of
this Committee. This legislation would permanently authorize
the Rare Pediatric Disease Priority Review Voucher Program.
While this program has provided value to some pharmaceutical
companies who have made investments in rare disease programs,
it also places a strain on the Food and Drug Administration
(FDA). We must keep that in mind and we should think carefully
about whether a permanent reauthorization makes sense at this
time.
Next, H.R. 4764, the "TRANSPLANT Act of 2019," will
reauthorize the C.W. Bill Young Transplantation Program. This
important program provides patients who need life-saving bone
marrow and umbilical cord blood transplants with information
and support as they go through this process. It also maintains
an efficient process for identifying donor matches, increases
the number of non-familial donors available for transplant, and
expands data and research to improve patient outcomes.
Representatives Matsui and Bilirakis, who have authored this
legislation, have worked to secure funding for this program for
many years.
Finally, we have H.R. 5373, the "United States Anti-Doping
Agency Reauthorization Act of 2019." The U.S. Anti-Doping
Agency is the national anti-doping organization in the U.S. for
Olympic, Paralympic, Pan American, and Parapan American sports.
This reauthorization will not only promote clean sport through
testing, education, and research efforts, but it will also use
a portion of its funding to promote a positive youth sport
experience.
I want to thank all of the witnesses for joining us today,
and I would like to yield the remainder of my time to
Representative Sarbanes.
Mr. Sarbanes. Thanks, Mr. Pallone.
I want to just thank Chairwoman Eshoo and Chairman Pallone
for having this very important hearing today regarding
reauthorization of all these key public health programs.
I have been working on the School-Based Health Centers
Reauthorization Act for a very long time, and I am very
appreciative that that is a bipartisan bill, and we certainly
have bipartisan sponsorship of it within the committee.
And it would authorize, as you have indicated, Federal
support for school-based health centers through 2024, which
provide critical primary and mental health services to
vulnerable and youth, certainly very important in this moment.
I have seen it in Maryland for sure, and I know that the
witness we have today, Robert Boyd, who is president of the
School-Based Health Alliance, will certainly be able to testify
to this as well. And that is that when a student has access to
school-based health centers the negative health outcomes, such
as asthma, morbidity, rate of hospital admissions, those
decrease, while the educational outcome, such as school
performance and graduation rates, increase.
And now, of course, the services of school-based health
centers are needed more than ever given the coronavirus
pandemic, young people grappling with changes in their lives,
the uncertainty. And being able to get care in a familiar and
supportive setting is very important.
So I look forward to the testimony today. Again, thank you,
Chairwoman Eshoo, Chairman Pallone, and my colleagues, for
support of this bill. And I yield back.
[The prepared statement of Mr. Sarbanes follows:]
Ms. Eshoo. The gentleman yields back.
The Chair now has the pleasure of recognizing Mr. Walden,
the ranking member of the full committee, for his 5 minutes for
an opening statement.
OPENING STATEMENT OF HON. GREG WALDEN A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF OREGON
Mr. Walden. Thank you very much, Madam Chair. Thanks for
having this hearing. As we have heard, these are really
important pieces of legislation. I am glad we are having the
hearing on them.
Obviously, there is a lot of uncertainty about the
authorizations as they are due to lapse or expire in just a
matter of months. So reauthorizing the funding of these public
health programs could be literally life-saving for Americans,
and because of that it is critical. I hope we swiftly move
these bills.
And as we have heard, the first bill, H.R. 2075, the
School-Based Health Centers Reauthorization Act of 2019, by
Representatives Sarbanes, Upton, Tonko, and others,
reauthorizes funding for school-based health center programs,
which support health centers operating as a partnership between
a school and a community health organization in order to
provide quality healthcare for our students.
H.R. 4078, the EARLY Act Reauthorization of 2019, sponsored
by Representatives Wasserman Schultz and Brooks, reauthorizes
programs related to breast cancer outreach, on which you have
been a real leader, Madam Chair, on this effort, and other
education initiatives, along with survivor support services at
the Centers for Disease Control and Prevention.
H.R. 4764, the TRANSPLANT Act of 2019, spearheaded by
Representatives Matsui and Bilirakis, reauthorizes the C.W.
Bill Young Cell Transplantation Program. That provides critical
support and life-saving treatments to patients in need of bone
marrow or umbilical cord blood transplants.
There are approximately 7,000 rare diseases affecting an
estimated 30 million people. It is also estimated about half of
these diseases affect children. That is why I am glad that we
are also considering H.R. 4439, the Creating Hope
Reauthorization Act. This bipartisan legislation makes the
Priority Review Voucher Program permanent for rare pediatric
diseases.
The PRV was first created in 2012 with the passage of the
Food and Drug Administration Safety and Innovation Act to
incentivize the development of therapies to treat rare
pediatric diseases.
While progress has been made in the development of
pediatric therapies, in fact, 22 therapies have been approved
for the treatment of 18 rare pediatric diseases since the
inception of the pediatric PRV program, we all know we still
have a long way to go. Nearly 95 percent of all rare diseases
do not have an FDA-approved treatment, leaving many patients
and their families with no options.
Research and development for rare disease therapies is
often scarce because each drug is only intended to serve a very
small population, and of course the opportunity to recoup
investments is limited.
Drug development is extremely costly, as we all know. It is
often time-intensive and often requires billions of dollars and
nearly a decade before receiving FDA approval, if it makes it
all way through the process.
The permanent reauthorization of the pediatric PRV program
would provide some certainty to drug developers considering
whether to invest in therapies for rare pediatric diseases as
they evaluate the feasibility of devoting significant resources
to products that may not provide a return on significant
investments given the limited population of patients, but that
could be life-changing for those that receive them.
So again I want to thank all of our witnesses for
participating in our hearing today, and I want to thank the
chairwoman for having this hearing and helping move these bills
along. They are commonsense, they are bipartisan, and we hope
to get them through the markup quickly and on down to the White
House.
So with that, Madam Chair, I yield back the balance of the
time.
[The prepared statement of Mr. Walden follows:]
Prepared Statement of Hon. Greg Walden
Madam Chairwoman, I am glad we are convening this hearing
to discuss the reauthorization of several important public
health programs, many of which face uncertainty as their
authorizations have either lapsed or will expire in just a
matter of months. Reauthorizing the funding of these public
health programs could be life-saving for many Americans and
because of that it is critical that we swiftly move these
bills.
The first bill, H.R. 2075, the School-Based Health Centers
Reauthorization Act of 2019, sponsored by Reps. Sarbanes,
Upton, and Tonko, reauthorizes funding for the school-based
health center program, which supports health centers operating
as a partnership between a school and community health
organizations in order to provide quality health care services
to students. H.R. 4078, the EARLY Act Reauthorization of 2019,
sponsored by Reps. Wasserman Schultz and Brooks reauthorizes
programs related to breast cancer outreach and education
initiatives, along with survivor support services at the
Centers for Disease Control and Prevention (CDC). H.R. 4764,
the TRANSPLANT Act of 2019, spearheaded by Reps. Matsui and
Bilirakis, reauthorizes the C.W. "Bill" Young Cell
Transplantation Program, which provides critical support and
life-saving treatment to patients in need of bone marrow or
umbilical cord blood transplants.
There are approximately 7,000 known rare diseases affecting
an estimated 30 million people. It is also estimated that about
half of these diseases affect children. That is why I am glad
that we are also considering H.R. 4439, the Creating Hope
Reauthorization Act. This bipartisan legislation will make the
priority review voucher (PRV) program permanent for rare
pediatric diseases. The PRV was first created in 2012 with the
passage of the Food and Drug Administration Safety and
Innovation Act (FDASIA) to incentivize the development of
therapies to treat rare pediatric diseases. While progress has
been made in the development of pediatric therapies--in fact,
22 therapies have been approved for the treatment of 18 rare
pediatric diseases since the inception of the pediatric PRV
program--we still have a long way to go. Nearly 95-percent of
all rare diseases do not have an FDA-approved treatment,
leaving many patients without options.
Research and development for rare disease therapies is
often scarce because each drug is only intended to serve a
small number of patients, meaning the opportunity to earn a
profit is limited. Drug development is extremely costly and
time-intensive, often requiring billions of dollars and nearly
a decade before receiving FDA approval--if it makes it through
the approval process at all. The permanent reauthorization of
the pediatric PRV program would provide some certainty to drug
developers considering whether to invest in therapies for rare
pediatric diseases as they evaluate the feasibility of devoting
significant resources to products that may not provide a return
on the significant investment given the limited population of
patients, but that could be life-changing for those that
receive them.
I thank the witnesses for being here today and look forward
to hearing their testimony. I hope we can move these
commonsense, bipartisan bills through the mark up process as
quickly as possible. I yield back.
Ms. Eshoo. The Chair thanks the ranking member for his good
comments, and he yields back.
The Chair would now like to remind members that pursuant to
committee rules, all members' written opening statements shall
be made part of the record.
I now would like to introduce our witnesses that are with
us today.
First, Ms. Linda Blount. She is the President and CEO of
the Black Women's Health Imperative.
Welcome to you. We are thrilled that you are here with us
today.
Mr. Robert Boyd, the president of the School-Based Health
Alliance.
Thank you, Mr. Boyd, for your willingness to testify today.
Ms. Nancy Goodman. She is the founder and executive
director of Kids v Cancer.
Thank you for your extraordinary work, and we are thrilled
to have you as a witness.
And Dr. Aaron Kesselheim. He is a professor of medicine at
Harvard Medical School.
Welcome, Doctor, and thank you for your willingness to be
here with us.
Mr. Brian Lindberg is the chief legal officer and general
counsel of the National Bone Marrow Donor Program.
Welcome to you.
And Travis Tygart is the chief executive officer of the
U.S. Anti-Doping Agency.
So welcome to each one of you. Thank you for being willing
to share your time, your important time, with us on these
important bills.
I now will recognize Linda Blount.
You are recognized for 5 minutes. And please remember to
unmute. Welcome. It is wonderful to see you.
STATEMENTS OF ROBERT BOYD, M.C.R.P., M.DIV., PRESIDENT, SCHOOL-
BASED HEALTH ALLIANCE; LINDA GOLER BLOUNT, M.P.H., PRESIDENT
AND CEO, BLACK WOMEN'S HEALTH IMPERATIVE; NANCY GOODMAN,
M.P.P., J.D., FOUNDER AND EXECUTIVE DIRECTOR, KIDS V CANCER;
AARON SETH KESSELHEIM, M.D., J.D., M.P.H., PROFESSOR OF
MEDICINE, HARVARD MEDICAL SCHOOL; BRIAN LINDBERG, CHIEF LEGAL
OFFICER AND GENERAL COUNSEL, NATIONAL BONE MARROW DONOR
PROGRAM; AND TRAVIS T. TYGART, CHIEF EXECUTIVE OFFICER, U.S.
ANTI-DOPING AGENCY
STATEMENT OF LINDA GOLER BLOUNT
Ms. Blount. Thank you very much. Good morning, Chairwoman
Eshoo, Ranking Member Burgess, members of the Committee on
Energy and Commerce. Thank you for the opportunity to appear
before this committee to discuss the EARLY Act Reauthorization
of 2019, introduced by Representatives Wasserman Schultz and
Brooks.
I am testifying today as president and CEO of the Black
Women's Health Imperative. And this week, we celebrate our 38th
year as the only national organization solely focused on
improving the health and wellness of our Nation's 21 million
Black women and girls.
We are proud to work with Congresswomen Robin Kelly, Yvette
Clarke, and Lisa Blunt Rochester, members of the Congressional
Black Caucus and of this committee.
In this role, and as a Black woman, I see every day how
young Black women are disproportionately impacted by this
terrible disease, and I know we can do more to prevent needless
suffering and death from breast cancer through early education
and screening.
Black women develop breast cancer on average 5 to 7 years
younger than White women, and until recently, the good news was
that Black women got breast cancer at lower rates than White
women. But as of 2015, that is no longer the case.
But that is where the good news ends. Black women are 40
percent more likely than White women to die of breast cancer.
This is, in part, for three reasons.
First, Black women are more likely than other racial and
ethnic groups to have an aggressive breast cancer subtype.
Second, they are less likely to receive the most effective
therapeutics for their cancers.
The first is an issue of biology, and the second is an
issue of behavior.
The third reason is why we are here today. Black women die
at such high rates because their breast cancers are too often
detected at late stages when treatment of any kind is less
effective. And researchers know that most breast cancers are
detectable long before a woman gets a mammogram.
Consider that 30 percent of all breast cancers in Black
women occur under the age of 50; 18 percent occur under the age
of 45. The CDC projects that for this year roughly 30,000
breast cancers will be diagnosed in women under 45. That is
5,000 among Black women under 45.
We know that early education, awareness, and screening
saves lives. When breast cancer is detected early and quality
treatment is received, the 5-year survival rate is 100 percent
for all women. If we just increase the rate of screening
mammography by 50 percent among women 45 and under, the
survival rate would be increased by an additional 3,000 women
every year, and that is 700 to 1,000 more Black mothers,
daughters, and sisters. That is good news.
Younger women are less likely to undergo breast cancer
screening than women age 50 to 74, and they are less likely to
have access to the latest digital breast screening
technologies, including 3D tomosynthesis, which have been shown
to detect breast cancers earlier.
We must do better to educate and improve access, which is
where the EARLY Act comes in. The EARLY--Breast Cancer
Education and Awareness Requires Learning Young--Act, sponsored
by Representatives Wasserman Schultz and Brooks, would
reauthorize and increase funding originally authorized in 2010,
giving needed attention to the education of younger and high-
risk women about their breast health.
The program not only educates women ages 45 and younger on
breast cancer risk, but it supports initiatives and research to
identify high-risk women, collect family histories, and educate
healthcare providers.
The EARLY Act has already benefited women. Mortality rates
from breast cancer have dropped in the past ten years, in large
part due to early detection.
The need for earlier screening and diagnosis is critical
for women with inherited genetic mutations, such as BRCA genes,
Ashkenazi Jewish women, and women who were treated with
radiation therapy for cancer as children.
I add Black women to this list not because of biology or
genetics, but because of the systemic racism that has limited
their access to preventive care.
The COVID-19 pandemic is yet another example of disparities
in our public health approach to the Black community. Black
people are at much higher risk of contracting COVID-19, and
they are much more likely than White people to die from the
virus.
We are on the verge of a seismic shift in this country when
it comes to valuing Black lives and health. I see a future when
we can stop talking about how Black women disproportionately
and needlessly die from breast cancer. Reauthorize the EARLY
Act and make 2020 the year we do what we know works and save
lives.
Thank you.
[The prepared statement of Ms. Blount follows:]
[GRAPHIC] [TIFF OMITTED] T5868.008
[GRAPHIC] [TIFF OMITTED] T5868.009
Ms. Eshoo. Thank you very much for your excellent
testimony, Ms. Blount.
The Chair now recognizes Mr. Robert Boyd.
You are recognized for 5 minutes for your statement.
STATEMENT OF ROBERT BOYD
Mr. Boyd. Thank you, Congresswoman Eshoo. Good morning to
you and to Ranking Member Burgess and to members of the Health
Subcommittee. Thank you for inviting me to speak with you this
morning on H.R. 2075.
I would like to thank Congressman Sarbanes for being the
tireless champion for school-based health centers. Without your
leadership and your dedicated staff, we wouldn't be here today.
My name is Robert Boyd, and I am the son and nephew of
public school teachers. I am here today as the President of the
School-Based Health Alliance, a nonprofit organization based
here in Washington, DC. Since 1995, the Alliance has served as
the national voice for more than 2,500 school-based health
centers that collectively provide healthcare for over 3.6
million children, youth, and others from predominantly low-
income families from across the country.
School-based health centers have been located in public
schools since the early 1970s. As you all know, schools are
more than just places of learning. Schools are pillars of the
community, whether located in large cities, suburbs, or rural
America.
For millions of low-income students, school-based health
centers are their sole source of healthcare. They allow parents
to remain at work and students to stay in school while getting
the healthcare that they need.
Now, the data is clear: Healthy kids learn better, healthy
kids earn better grades, and healthy kids achieve higher
promotion and graduation rates. Healthy kids also grow up to be
healthy adults. School-based health centers sit at the critical
intersection of education and healthcare.
Currently, we hear extensive discussion about how schools
will operate this fall. It will be difficult to safely resume
in-person learning. Many schools will continue remote learning
or in some hybrid combination.
But what cannot be up for debate is the ability of school-
based health centers to continue providing essential healthcare
services to our most vulnerable students.
Many people do not realize it, but even during the pandemic
many school-based health centers are still delivering ongoing
care.
Throughout the pandemic, school-based health centers have
continued to provide health services to K-12 students directly,
sometimes onsite at the school, sometimes at school-linked
clinics, via mobile vans, even drive-through visits in school
parking lots.
Some students have been able to receive life-saving
medications, immunizations, and even participate in socially
distanced counseling sessions.
Unfortunately, far too many have not.
We also provide care through telehealth services. As a
result of the pandemic, the U.S. Centers for Medicare and
Medicaid Services has given State Medicaid programs increased
authority and flexibility to expand telehealth services,
including telephonic and video conference care, and has removed
some cross-State licensing requirements.
Going forward, telehealth will remain an important strategy
for increasing access to care, allowing us to reach the
students and families with the greatest needs, including almost
one-third of school-based health centers located in rural
communities.
Telehealth is not a substitute for in-person care. It is a
technological enhancement in the tool chest of healthcare
providers.
Ladies and gentlemen, please be clear, the School-Based
Health Alliance believes in a comprehensive definition of
health that includes protecting the mental, emotional, and
social health needs of students.
Even as education and health leaders are urgently
purchasing hand sanitizer and marks, we cannot forget to care
for the entire student. We are here to help.
The mental and emotional health of students is an issue
that has too often been overlooked in the current debate about
reopening school buildings. It is more important than ever that
we think comprehensively and act with urgency. We must treat
this pandemic as we would a mass incident, like tornadoes,
hurricanes, or school shootings, that wreak multiple levels of
havoc on an entire community.
As with a mass incident, some of the pandemic's harm is
visible and immediately apparent. But other damage may be less
visible, such as increases in depression, anxiety, sleep
problems, hunger, and stress, caused by children's struggles
with online learning and social isolation.
A lingering concern is the potential for child abuse to
remain unchecked, given that the primary reporters of violence
and neglect are educators and healthcare workers.
Communities need maximum flexibility to ensure that the
doors of school-based health centers, both our literal doors as
well as our virtual online gateways, remain open to deliver
critical primary and mental healthcare services to students.
By passing the bipartisan School-Based Health Centers
Reauthorization Act, you will recognize the critical role that
school-based health centers play in both the immediate and
long-term health needs of our Nation's schoolchildren.
Thank you, and I am happy to answer questions.
[The prepared statement of Mr. Boyd follows:]
[GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
Ms. Eshoo. Thank you very much, Mr. Boyd, for your
excellent testimony.
The chair now recognizes Ms. Nancy Goodman for 5 minutes
for your testimony.
Welcome again, and thank you.
STATEMENT OF NANCY GOODMAN
Ms. Goodman. My name is Nancy Goodman, and I am here as
founder and executive director of Kids v Cancer, a nonprofit
that has been the driving force of the Creating Hope Act, as
well as the RACE for Children Act, which requires novel
therapies to be studied in children's cancers.
Kids v Cancer promotes programs that are in the best
interest of seriously ill children, regardless of whether the
pharmaceutical industry supports or opposes them. Toward that
end, we do not accept donations from drug companies.
In addition, I have drafted and am solely responsible for
the following comments.
I am here not only in my capacity as the executive director
of Kids v Cancer but also as a mother. More than a decade ago,
I lived in Manhattan with my husband and sons, Jacob and
Benjamin.
When Jacob was eight years old, he began waking up with
nausea and headaches. We went to our pediatrician again and
again, and on a Sunday morning, sitting on an examination bed,
wearing a Mickey Mouse tie, our pediatrician told us Jacob
might have a brain tumor.
We learned Jacob had medulloblastoma, a rare pediatric
cancer. The drugs available to treat Jacob were 40 years old,
and they were unlikely to work. How could that be, I wondered,
when there are so many exciting innovations in adult cancer
therapies?
Jacob died in the early hours of a wintry Friday morning.
He was ten years old. That day I founded Kids v Cancer.
We started with drafting the Creating Hope Act. The
challenge we sought to address was that there were inadequate
market incentives to bring drug companies to develop drugs for
small children with life-threatening illnesses. Yet we wanted
to create a program largely funded by major pharmaceutical
companies.
So let me repeat that. The Creating Hope Act basically
transfers value from large pharmaceutical companies to smaller
and oftentimes more innovative biotech companies to facilitate
their investment in creating drugs for seriously ill children.
Since the passage of the Creating Hope Act, the Pediatric
Voucher Program has been very successful and has created a
pathway for companies to develop many drugs for children with
life-threatening illnesses. So let me review some of these
measures of success.
The first measure of success is the value of the vouchers.
The Pediatric Voucher Program has mobilized well over $1
billion in incentives in the form of payments for vouchers,
creating opportunities for market-based, risk-adjusted returns
on investment, and rare pediatric disease drug development.
A second measure of success is the number of new drugs that
have been approved for seriously ill kids since the Creating
Hope Act's passage, more than 20 so far.
A third mark of success is the number of drugs in the
development pipeline. As measured by the number of rare
pediatric disease designations, this number is increasing by
leaps and bounds, from 3 in 2013 to over 60 today.
Fourth, as Dr. Kesselheim, my co-witness, notes in his
Health Affairs article, drugs qualifying for a pediatric
voucher are more likely to proceed through the phases of drug
development and at the speed at which it would do so.
Fifth, and perhaps the most important, at the time of
Jacob's illness there were no companies whose core mission was
pediatric cancer drug development. Now there are several.
The Creating Hope Act has been a great success, but its
short duration has created uncertainty and limited its impact.
Since 2012, the program has been reauthorized three times, each
for 1 to 4 years. However, this time horizon for drug
development from idea to FDA approval can be 7 to 10 years or
longer.
The start of the drug development process is a critical
moment. It is when developers choose whether to develop a drug
for kids. If we reauthorize the Pediatric Voucher Program on a
permanent basis, we can affect this early stage of drug
development as well.
The Creating Hope Act has increased the number of reviews
in the FDA's Priority Review Program. That is why, when
originally drafting the Creating Hope Act, we were careful to
include a provision by which the FDA could compensate itself
for any additional burden, allowing the FDA to set voucher user
fees.
In 2020, voucher user fees are $2.1 million on top of a
$2.9 million user fee. If there are remaining issues around the
management of this program, I thank the FDA leadership as they
work to address them head on so that we do not have to abandon
or shorten a program that promises hope to so many critically
ill children.
All evidence available indicates that the Pediatric Voucher
Program is effective. I urge you to support its permanent
reauthorization by passing the Creating Hope Reauthorization
Act.
Thank you for including me in today's hearing, and I am
happy to take your questions.
[The prepared statement of Ms. Goodman follows:]
[GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
Ms. Eshoo. Thank you very much, Ms. Goodman. I can't help
but think that, as a mother myself, out of the extraordinary
tragedy and loss of your son, your work is nothing short of
remarkable. And it is so important for us to take up this
reauthorization and that it be made permanent, as you
indicated. So thank you very, very much. You are a source of
inspiration to us.
Next, Dr. Aaron Kesselheim, you are recognized for 5
minutes for your testimony. And thank you again for your
willingness to be with us today.
STATEMENT OF AARON KESSELHEIM
Dr. Kesselheim. Thank you. Chairwoman Eshoo, Ranking Member
Burgess, members of the subcommittee, thank you for this
opportunity to talk today.
I am an internal medicine physician and lawyer, and I lead
the Program on Regulation, Therapeutics, and Law, one of the
largest and most prolific nonindustry-funded research groups in
the country that focuses on the pharmaceutical market.
My remarks today will cover the rare pediatric disease
priority review voucher, or PRV, why it is a poor way to
support research into rare pediatric disease treatments, and
what some better options are.
A PRV entitles drugmakers to speed FDA review of the 10-
month timeframe it applies to standard drugs that lack any sort
of special innovative or clinical qualities to the priority
review 6-month timeframe reserved for drugs representing
therapeutic advances, like those meeting unmet medical needs.
Economists assume that drugmakers would value reaching the
market four months sooner, so Congress created the voucher in
2012 to stimulate investment into rare diseases affecting
children, unquestionably an important public health issue.
Unfortunately, it hasn't turned out that way. There are
four major problems: It doesn't work, it wastes public and FDA
resources, it is potentially dangerous, and it isn't very
valuable.
First, from 2012 to 2018, the FDA awarded 14 rare pediatric
disease PRVs, which is more treatments than in years before
2012, but the important question is whether the initiation of
the PRV program changed the research trajectory.
To study this question, in a study led by my colleague here
at Harvard, Thomas Hwang, we compared how drugs treating rare
pediatric diseases progressed through development in the years
before and after the voucher program with drugs treating rare
adult diseases, which would not earn a voucher.
We found no variation in the rate at which drugs eligible
for a rare pediatric PRV were introduced into clinical testing.
That is, the baseline trajectory didn't change. Rather, there
has been an increase in recent years in rare diseases of all
kinds, pediatric and adult. That is obviously great, but it
doesn't mean that the PRV works, and I would be happy to submit
this paper for the record.
Second, the voucher wastes public resources. It derives its
market value from the prospect that manufacturers with more
likely run-of-the-mill drugs would come to market sooner and
start selling those drugs at high prices to be reimbursed by
payers like Medicare and Medicaid.
It also wastes regulatory resources. FDA officials have
raised concerns that the PRV interferes with the way it
prioritizes drugs by hastening review of unremarkable products
that would not otherwise merit an expedited timeline, straining
resources since the FDA cannot quickly hire and train new staff
with the necessary expertise to review the drugs.
Third, the voucher is dangerous, since too speedy FDA
review may lead to bad decisionmaking. For drugs that are a
major advance in treatment, accelerating FDA review is
reasonable, but one study showed the risks of imposing
arbitrarily short deadlines on FDA review times for drugs that
did not deserve such acceleration, such as increased risk of
subsequent safety-related withdrawals or botched warnings.
And finally, the vouchers' value has plummeted. Because
vouchers can now be earned for FDA-approved drugs for neglected
tropical diseases and medical countermeasures, as well as rare
pediatric diseases, there are a lot of vouchers available on
the market.
In recent years, vouchers have sold for approximately $80
to $110 million, a far cry from the lofty values predicted by
the economists who devised this scheme.
One way to try to address these issues would be to make
necessary improvements in the voucher program. For example,
drugs qualifying for the voucher could be limited to first-in-
class products or products meeting unmet medical need.
Manufacturers should also not be allowed to earn a voucher
immediately for drugs approved via the "special accelerated
approval" pathway, since accelerated approval of drugs has not
yet demonstrated meaningful patient benefits.
Most importantly, rare pediatric disease manufacturers
earning a voucher should be required to ensure that the product
is sold to U.S. patients at value-based prices, or at no higher
than prices sold to rare pediatric disease patients in other
high-income countries, to help ensure that the children who
need the drug can be assured of access to it.
A better pathway forward for Congress would be to consider
more direct ways of encouraging drug development for such
medical conditions. For example, the U.S. Government supports
pharmaceutical innovation with substantial amounts of taxpayer
dollars each year, mostly through the NIH. One study found that
all new drugs approved from 2010 to 2019, or their molecular
targets, could be linked back to this government-funded
research.
The U.S. Government also offers various tax concessions and
refunds directed at research and development spending by
private firms. Thus, greater upfront funding, or tax credits,
could be offered for research into rare pediatric diseases. And
unlike the PRV, this method of stimulating innovation has a
track record of success.
Another approach would be to provide greater support for
late-stage development, perhaps through nonprofit organizations
via public-private partnerships. Recent funding through BARDA
to support vaccines for COVID-19 is related to this model.
Naturally, such partnerships should include guarantees
about affordable access to the rare pediatric disease drugs
that emerge because of the public's involvement in reducing the
risks and costs of drug research and development.
Children with rare diseases need more investment in
scientific discovery and clinical trials to get them the
treatments that they deserve. It is time to move past the
pediatric rare disease priority review voucher mistake and let
it sunset, and instead direct efforts towards better solutions
known to lead to transformative drugs, including direct public
investment in research and development for treatments for rare
pediatric diseases.
Thank you very much.
[The prepared statement of Dr. Kesselheim follows:]
[GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
Ms. Eshoo. Thank you very much, Doctor.
Now we will recognize Brian Lindberg.
Thank you again for being with us today, and you have 5
minutes for your testimony.
STATEMENT OF BRIAN LINDBERG
Mr. Lindberg. Madam Chair, Ranking Member Burgess, and
distinguished members of the subcommittee, I am Brian Lindberg.
I have the honor to serve as chief legal officer and chief
policy officer of National Marrow Donor Program/Be The Match.
For over three decades, through public-private partnership
with the Federal Government, we have been entrusted to operate
the C.W. Bill Young Cell Transplantation Program, which
provides life-saving bone marrow and umbilical cord blood
transplants to patients in need.
As you know, this is the program that houses the national
registry of volunteer bone marrow donors, as well as the
National Cord Blood Inventory and the Stem Cell Therapeutics
Outcomes Database.
On behalf of the entire team at NMDP/Be The Match and the
patients we serve, I would also like to thank all the members
of the Health Subcommittee for the opportunity to speak with
you today and for allowing me the privilege to share with you
the successes of the program over the past 5 years, but more
specifically during the current crisis of the global pandemic.
Finally, I would also like to extend my special thanks to
Congresswoman Matsui and Congressman Bilirakis for their
leadership in introducing H.R. 4764, to reauthorize the program
for another five years. Ms. Matsui and Mr. Bilirakis have been
tireless champions for patients whose only chance at life is
bone marrow transplant.
Today, I am so pleased to be able to tell you that this
past December, NMDP/Be The Match marked the 100,000th time it
facilitated a transplant in which a heroic volunteer donor
stepped up to save the life of a person they had never met by
making this life-giving gift of bone marrow donation.
This is because 35 years ago the founders of our program
refused to accept the conventional wisdom that a national
registry wouldn't work because, after all, who would do this,
who would step up to donate their marrow for a stranger.
They persevered because they knew that a national registry
would work, that this must work, and needed to be established
by Congress.
I can say without hesitation that this same spirit, that
refusal to take "no" for an answer, has served this program
well over the past year. In addition to celebrating our
100,000th transplant in 2019, we have facilitated more
transplants than in any other year in our history.
And then global pandemic arrived, and it threatened the
very foundational underpinnings of our ability to fulfill our
obligation to those patients. And that is specifically why I am
here before you today.
As you are no doubt aware, the success of this program and
the lives of the patients we serve rely on our ability to move
couriers who carry donated cells, not only across the country
but also across the world.
The complexity of matching patients and donors results in
half of these cellular donations crossing an international
border. Domestic and international travel bans, border
closings, declining availability of passenger airline service,
quarantine and shelter-in-place orders, and an ever-changing
patchwork of State and local restrictions during the COVID
pandemic have created near catastrophic barriers to our ability
to facilitate timely transplant and threaten to impede every
one of these life-or-death missions at multiple points along
what is now a tenuous path from donor to waiting patient.
But despite all of that, I am thrilled to be able to report
to you today that throughout the course of the pandemic, NMDP/
Be The Match, through innovation and determination, has since
March delivered more than 2,500 life-saving therapies to
patients who are relying on them without a single failure. Not
one patient prepped for transplant has gone without.
Everyone associated with this program understands the
consequences of failure are the direst consequences of all.
But these successes have been hard-earned, and in many
cases involve days-long, around-the-clock, extraordinary
interventions across multiple Federal agencies, as well as
State and foreign governments.
Due in material part to the national program status awarded
us under the authorizing statute, over the past few months we
have been granted a waiver in the national interest by the CDC
to the European travel ban, allowing international bone marrow
couriers to deliver products to U.S. patients.
We have designated our couriers and donors as essential
critical infrastructure by the Department of Homeland
Security's CISA.
We have leveraged private aircraft, donated for use by HHS,
to deliver life-saving cells to patients when commercial
flights were no longer available.
We have accessed support from U.S. embassies and consulates
to obtain from foreign governments timely travel authorizations
for donor and courier travel and to coordinate private and
humanitarian flights in and out of countries that had otherwise
closed their borders.
And we have been granted clearance from Customs and Border
Protection that ensures donations coming from Canada to
American patients will have uninterrupted transport into the
country.
The examples I have just described share two common but two
extraordinarily important threads. First, this would not have
been achieved without our national program status. And second,
patients in the U.S. would have died had these things not
happened.
And that is why I am before you today, urging your help to
ensure timely reauthorization of the program to mitigate any
unnecessary risk to patient life.
Thank you again for the opportunity to submit this
testimony and for the committee's longstanding support of this
program that has given so many a second chance at life.
On behalf of those whose lives have been extended, those
who are searching the registry for a match donor today, and
those who will need to look to the program for help in the
future, we urge to once again reauthorize the C.W. Bill Young
Cell Transplantation Program, and we would respectfully ask you
do so prior to the program lapsing at the end of September.
Thank you.
[The prepared statement of Mr. Lindberg follows:]
[GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
Ms. Eshoo. Thank you so much, Mr. Lindberg. What a powerful
story, given all the challenges of COVID that you have had to
deal with.
Each one of you has a powerful story in your testimony, and
Mr. Lindberg's was as well. Thank you.
I served with Bill Young, and I think that he would be
very, very proud. Very proud.
Mr. Lindberg. Thank you.
Ms. Eshoo. He was a gentleman, a lovely, good man.
Next, the chair recognizes Travis Tygart.
Mr. Tygart, you are recognized for 5 minutes for your
testimony, and thank you for being with us today. Welcome.
STATEMENT OF TRAVIS T. TYGART
Mr. Tygart. Thank you. Chair Eshoo, Ranking Member Burgess,
members of the subcommittee, good morning. My name is Travis
Tygart. I am the CEO of the United States Anti-Doping Agency,
or USADA. Thank you for inviting me to appear before you today
to discuss our reauthorization and our important mission.
It is a huge honor to be here representing our North Star,
which are our clean athletes. We greatly appreciate the ongoing
support of Congress and the President's Office of National Drug
Control Policy, or ONDCP, to protect, along with us, the
health, safety, and rights of clean athletes to a fair and
level playing field.
USADA, as you know, is charged with implementing a robust,
fair program, which includes both in- and out-of-competition
testing, results management, and athlete education for all U.S.
Olympic, Paralympic, and Pan Am athletes.
Importantly, we also contribute to the advancement of all
clean sport through scientific research and education
initiatives focused on values and healthy and safe competition.
Chair Eshoo, members of the subcommittee, we cannot perform
this essential mission without your support. As many of you
know, we opened our doors in late 2000, thanks to the
bipartisan efforts of Congress in recognizing the need to
create an independent anti-doping system.
Throughout the 1990s, many viewed the U.S. and our athletes
as dirty. There was no independent effort, because the Olympic
sports movement, whose job it is to promote sport, was also
handling the anti-doping program. It was the epitome of the fox
guarding the henhouse.
As a result, Congress took action and established an
independent organization to implement a fair and robust
national program.
This landmark decision literally changed the game for U.S.
athletes for the good. This independence is our lifeblood, and
it is possible only through government support that we receive
each year.
We received Federal funding in the fall of 2000 and every
year since. We were first formally authorized by the committee
and Congress in 2006, and we were reauthorized in 2014. Our
congressional funding comes through ONDCP and is combined with
our private funding to achieve our mission.
Since we opened, we have educated tens of thousands of
athletes and others on the reason that playing fair is the only
way. Our True Sport Partner program has reached over 13.8
million since its founding in 2017.
Deterrence is the foundation of the entire program, and we
have conducted approximately 175,000 drug tests. In 2019, we
completed a total of 7,300 blood and urine tests.
We have also earned the trust that we fairly enforce the
rules even when not easy or popular to do so. This is essential
to our success. In 2019, we received 533 tips to our Play Clean
whistleblower line and resolved 49 cases.
Importantly, when we were sued by Lance Armstrong, a
Federal court ruled that the USADA process provides due
process. This will likely be the most important legal legacy
ever, that due process is afforded our Olympic and Paralympics
athletes.
The final component is our scientific and research efforts.
Along with others, we have advanced the science to now have
reliable laboratory methods to detect and, thus, deter things
like EPO or human growth hormone or designer steroid use.
We also joined forces and raised additional private money
with Major League Baseball and the National Football League to
establish a scientific research entity that has become a world
leader.
Of course, you can see from our independent audit that we
send to Congress every year we are efficient and we are good
stewards of the funding. We are lean and mean, and for just a
small amount of government funding we have changed the game for
the good.
Comparatively, the U.S. Government is funding less on
average per athlete than many other countries. As one example,
the U.K. Government provided its agency a million dollars more
in funding than us, yet they have less than half the athletes
we do.
USADA's reauthorization, as was mentioned by the chair, is
even more important this time around since the Summer Olympic
and Paralympic Games are coming to the United States in L.A. In
2028. By passing this reauthorization, you can help make sure
the 2028 Olympic Games are the cleanest games ever.
Finally, our authorization strengthens our ability to
influence globally. Congress' backing gives us the standing to
advocate so that our athletes' rights are also protected when
they compete abroad. USADA's independent model has become a
beacon to others, and we are humbled to be in the position to
lead within the global community.
Just last month, at the request of Congress, ONDCP sent a
report to Congress detailing the World Anti-Doping Agency's
governance problems, including its lack of independence and
failure to provide fair athlete and U.S. representation.
We applaud ONDCP for its work on this issue. And hopefully
you all have seen that several leading athlete groups from
Canada, the United States, Germany, and others have come out
strongly in favor and support of these principles.
Chair Eshoo, Ranking Member Burgess, members of the
subcommittee, Congress did the right thing almost 20 years ago
by establishing an independent anti-doping organization, and
you all have continued to support this model to this day.
Along with the millions of athletes we represent, we thank
you for your ongoing support and hopefully for the swift
passage of H.R. 5373, and I look forward to taking any of your
questions.
[The prepared statement of Mr. Tygart follows:]
[GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
Ms. Eshoo. Thank you for your wonderful testimony and for
being with us today.
We will now move to member questions, and I recognize
myself for 5 minutes. To the witnesses that I don't get to with
questions, it doesn't mean that I don't respect your program.
You have all given superb testimony. They are very important
programs.
I want to zero in on the Creating Hope Reauthorization Act,
because we have some differing views here.
So to Dr. Kesselheim, the GAO found that drug developers
said the voucher program plays a major part--has played a major
part in their decision to invest in the development of rare
pediatric drugs, but your research found no increase in the
development of rare pediatric drugs after the voucher program
began.
So my first question to you is--and I want to get my
questions in first and then take the answers--how do you
explain the differences in these findings?
To Ms. Goodman, Dr. Kesselheim's research paper used drug
development for rare adult diseases as the comparison group for
rare pediatric diseases. But in your experience, how is the
drug development process different for pediatric drugs versus
adult drugs?
And to both of you, do you believe that the Pediatric
Voucher Program has existed long enough to prove whether it is
beneficial or harmful, and if so, what benefits have you seen,
and what harm?
And so why don't we start with Dr. Kesselheim. Please try
to keep your comments brief so that I can get all of the
answers to my questions.
Dr. Kesselheim. I will do my best, Chairwoman, to be brief.
I would say that the difference is that the GAO report was
based on anecdotal interviews with individual companies, and
the study that we did was a comprehensive study that looked at
all new drug development. And so it would be possible for, in
subjective interviews, for companies to say, sure, why not, but
if you actually look at the data across the board, it doesn't
look like there is any difference.
Ms. Eshoo. Well, I represent Stanford University, the
Stanford Medical Center, and Dr. Crystal Mackall, she is the
leading cancer researcher at the Stanford Medical School, and
she is a clinician. She said the voucher program has been
remarkably impactful for childhood cancer. She said before the
program, she used to go with her hat in hand to beg investors
to consider a potential drug, and now people take a second look
and are interested in developing drugs. And she named them. She
said something very interesting, that a bean counter may be
disappointed by the sheer numbers of approved pediatric drugs.
But she said that that disregards the lengthy drug development
cycle, and it is more important to look at the impact of the
approved drugs. And then she named them. So I just wanted to
insert that.
When you say the GAO interviewed people and it was
anecdotal, the GAO usually does very thorough work. So I don't
know how you--how did you get to identify the GAO's report as
anecdotal?
Dr. Kesselheim. Because that is how the GAO describes it in
its report. It says that the way that they identified the--how
drug companies thought--what drug companies thought about it is
by interviewing some of them.
Ms. Eshoo. OK. Ms. Goodman, can you answer my questions,
please, and anything else you want to add?
Ms. Goodman. Yes, and thank you so much for giving me this
opportunity. Look, I think there is a lot of evidence that this
has been a very successful program. There are more approved
drugs. There are loads of new drugs in the development pipeline
which we can measure by designation. And how do we explain that
away? Even Mr.--Dr. Kesselheim, in his health affairs piece,
notes that the program has been--you know, has shown that for
drugs that qualify for vouchers, they speed through the drug
development process faster, and they are more likely to go from
one phase to the other.
It is true that the program's weakest point of
effectiveness is, at the very start, when drug developers
decide will I develop a drug for seriously ill children or for
some other indication, right?
And so I agree with Mr. Kesselheim--Dr. Kesselheim, in part
on that point. But the reason there is this problem is that
drug development takes, you know, ten or more years, and we
keep on reauthorizing this program for very short periods of
time. If we reauthorize it for permanent length of time, we are
going to see Dr. Kesselheim very happy about the effect of this
program on early drug decisions.
Dr. Kesselheim. If I could just clarify, just a mentioned
mistake about the way that my--what my study actually shows. So
my study didn't show any difference in the ways that drugs
progressed from phase 2 to phase 3, or phase 3 through
approval. Although, it did show that there was a statistically
significant effect on the progress from phase 1 to phase 2.
Phase 1 is also--tends to be the shortest phase of clinical
trials and drug development, so it is unclear of how meaningful
that is overall. The most important finding was that it doesn't
appear that there are any new drugs that come into the system
before or after the voucher program as compared to rare adult
diseases. And I will just point out that over the last couple
of decades----
Ms. Eshoo. Doctor, my time has run out. But I think it is
important to note of the drugs that have been approved, as Dr.
Mackall says at Stanford Medical School, and she also works out
of Lucile Packard Children's Hospital in my district.
It is now--let's see, we go to--who is next? Who is? To the
ranking member of the subcommittee, Dr. Burgess, for his
questions, 5 minutes.
Mr. Burgess. I thank the chair. Maybe we can continue this
discussion that we just started a little bit. Dr. Kesselheim,
Ms. Goodman mentioned in her testimony that the number of rare
pediatric disease drugs approved by the FDA since the enactment
of the Creating Hope Act--the Creating Hope Act has increased
by over 120 percent. Do you have a sense of how the United
States compares to other nations when it comes to rare
pediatric drug development?
Dr. Kesselheim. Well, I believe that the United States is--
you know, helps contribute substantially to rare pediatric
disease drug development because of the substantial amount of
money through the NIH that goes into the discovery and
development of the--of some of the key, you know, drugs that--
that emerged for all kinds of conditions, including rare
pediatric diseases. And I--my presumption is, is that those
drugs also are available in other countries around the world,
although, I haven't studied that.
Mr. Burgess. But as far as development for a drug to treat
a rare pediatric cancer--I would suspect that it is likely,
more likely to happen in the United States than other
countries. But perhaps we can find that out on our own.
Ms. Goodman. If I could intervene?
Mr. Burgess. Yes, yes. Sure.
Ms. Goodman. Yes, you know, the vast predominant--the vast
majority of drugs developed for seriously-ill kids are right
here in the United States. And if we don't get our incentives
right, kids all over the world are going to suffer. And so I
think this is a real issue. I think you hit it right on the
head.
Mr. Burgess. So let me just ask you, because you brought up
the point about the reauthorization date, and I don't know if a
lot of people are aware of--this program was started in a Food
and Drug Administration reauthorization act back in 2012, but
it is not linked to the reauthorization or the FDA
reauthorizations that we do every five years. So this program
expires September 30th kind of all on its own--talk about an
orphan--and it is out there by itself. And you may have an
excellent point that it ought to be reauthorized, but perhaps
the permanence or a longer period of time because we do have a
long time horizon with these products that are in development.
Ms. Goodman. That is correct. Look, the first drug to get a
voucher for pediatric cancer is the Dinutuximab by Unituxin,
and this drug was in development for 30 years. So even if we
do--even if we extend this program by 7 or 10 years, there is
still going to be a large portion of drugs that we don't
incentivize and we can't support.
Mr. Burgess. You know, as I was listening to you and one of
the earlier witnesses, of course, we are so fortunate on this
committee, the full committee, to have worked on the 21st
century CARES Act and championed and got through. Also pointed
out to me in 2015, having been no new therapeutic for sickle
cell disease approved for 40 years, FDA approved therapy for
sickle cell for 40 years, very reminiscent of your comments
about no new children's drugs developed for 40 years. So we
don't--we don't always do things perfectly in this committee,
but sometimes we can make things a priority that actually do
make a difference in people's lives. And from time to time, we
have to acknowledge that, celebrate that success, and recognize
that that is equity that we can push forward if we are willing
to do that. So definitely making things a priority, thumbs up.
That is an important point.
Now, Mr. Lindberg, just before my time runs out, I had
signed up to be a donor. I have been in the donor program to
see if I can be a donor match, because there was some drive
going on in my community, long before I ever came to Congress.
I have been here for a while. Is that--is that enlistment still
active? Does a citizen need to reup that from time to time?
What can you tell us, as we are here highlighting your program,
what the people need to know about signing up and the length of
time that they may be signed up for a marrow donor program?
Mr. Lindberg. Thank you, Ranking Member Burgess. So--thank
you for signing up to join the registry. Our criteria--
eligibility criteria do state that once a volunteered registry
member reaches the age of 61, that person ages off the
registry. That is a cell efficacy issue.
Mr. Burgess. What difference does it make? I object.
Mr. Lindberg. We like people objecting to aging off. One
thing that we know is that over 30 years of this program and
our work with outcomes database, we know that--we know that
despite a very good HLA or DNA match, that as a donor ages,
that there are poorer outcomes for the recipient. So we know
that our network transplant physician community looks for
young, healthy donors, and will prioritize those over those--
like me--who are a bit more aged than others.
Mr. Burgess. OK. Well, I actually did not know that. So
that is useful information that came in this morning. I didn't
realize it had been so insensitive as to bump me off for being
old.
Thank you, Madam Chair. Again, I have learned something
this morning. I will yield back.
Ms. Eshoo. The gentleman yields back.
The chair is now pleased to recognize the chairman of the
full committee, Mr. Pallone, for his 5 minutes of questions.
The Chairman. Thank you, Madam Chair. I wanted to ask my
questions of Dr. Kesselheim and they are all about the Rare
Pediatric Disease Priority Review Voucher. I wanted to say
that--while I believe that we--while I think we all agree that
more has to be done to expand research into treatments and
cures for diseases that affect children and adolescents, and
that those treatments and cures should be accessible to those
who need it, I think the committee needs to carefully consider
whether the Rare Pediatric PRV Program is tailored to achieve
those ends. And that requires an analysis of the cost and
benefits.
And when making policy decisions to reauthorize programs,
generally, we have to analyze how they have worked in the past,
and that can be particularly challenging when we are looking at
an incentive program, we have to determine whether the desired
effect would have happened without the incentive provided by
Congress.
So, Dr. Kesselheim, with that, by way of background, I
interested in the portion of your testimony when you described
how you do an empirical study into how the Rare Pediatric PRV
program acted an incentive. Could you describe the way or the
methods you used in your study in your findings in that regard?
Dr. Kesselheim. Sure. So what we did was we identified all
new drugs that are coming into the pipeline, not drugs that are
FDA-approved, drugs that coming into the pipeline, and we
identified whether or not they were directed towards pediatric
rare diseases and adult rare diseases. And then we looked to
see in the years before the Pediatric Review Voucher Program in
2012 was extended to rare pediatric diseases, and in the years
after, whether or not there was a difference between the number
of new rare pediatric drugs entering into the pipeline and the
number of new adult rare drugs entering into the pipeline,
which theoretically wouldn't have been incentivized at all,
because they wouldn't have gotten--they wouldn't have been
eligible to get a voucher. We found no difference in the
trajectory before and after.
What we did notice was that the trajectory has been
increasing, which is a good thing, but what it just means is
that, it just means that rare diseases, in general, are a point
of a lot of research for drug companies and the--and public
funding, and we are just getting a lot more new drugs for rare
diseases across the board over the last couple decades. The
Rare Pediatric Disease Voucher Program was extended to rare
pediatric diseases in 2012 doesn't seem to have affected the
trajectory at all.
Mr. Pallone. Well, now, let me ask you this: I know the
program does not have a budget score or really cost the Federal
Government any money, but there may be, you know, other costs
associated with this. So in that regard, can you describe how
the program affects FDA resources?
Dr. Kesselheim. Sure. And I will actually disagree that the
program doesn't cost the government any money. The government--
the program does cost the government a lot of money, because
what the program does is it lets run-of-the-mill, non-
innovative drugs onto the market sooner so that they can start
being charged extremely high prices to Medicare and Medicaid,
which, of course, are government-funded insurance programs.
That is where the voucher gets its value from.
But in terms of the FDA, yes, in those--in that same GAO
study where they interviewed drug companies, they also
interviewed people at the FDA, and the people of the FDA said,
Look, this is a real burden on our resources, even though there
is an additional user fee associated with it, it is just not
something that it--you know, it strains our ability to
prioritize what we think are important public health drugs and
put those at the top of the queue.
Mr. Pallone. Well, let me ask you one last question. The
bill before us would fully remove the sunset and authorize the
program indefinitely. I know in your testimony you said it
would prefer to see this program expire. But if the program
were to continue, which I think is likely, what improvements
would you recommend?
Dr. Kesselheim. Well, so first of all I would say, I would
say, first of all, that the PRV should not be able to apply to
drugs that are--that it should only be able to apply to drugs
that are first in class or to drugs that meet an un-medical
need. I would say that the PRV should not be able to apply to
drugs that are approved via the accelerated approval pathway,
because those drugs do not have a demonstrated effect on actual
patient outcomes. And I would say that any drug that gets a PRV
should be required to make the drug available to patients at a
value-based price that is consistent with the same price for
which it is available to pediatric rare disease patients in
other countries, so that patients can have access to the drugs
that they really need.
Ms. Goodman. Congressman, if I may jump in here for a
moment. And I am going to also say that I would disagree with
Dr. Kesselheim on every single point he has made. Let's just
start with the pricing issue.
You know, there are two kinds of ways to provide access to
drugs, access to drugs for patients. And there are two groups
of patients. One are patients who already have drugs. And so
for those patients, of course, we would like the drugs to be as
low as--the pricing as low as possible. But the other group of
patients don't have the therapies to treat their illnesses yet,
and that includes my son, Jacob. And we can't expect that we
are going to give that class of patients best access to drugs
if we are going to be suppressing prices of therapies that
haven't even been developed yet.
The second point I just want to make is with respect to,
you know, we are talking a lot about this health affairs
article. I think Dr. Kesselheim is, you know, playing fast and
loose with a bunch of numbers. If you look at his charts, there
are notable deltas, which means pediatric and adults, the adult
curves. And, of course, the weakest point in the efficacy of
the program is right at the start when people--when developers
decide do they want to develop drugs for kids? That is why we
need a permanent program.
Mr. Pallone. My time has expired, but--what did you say,
Doctor? I am sorry.
Thank you, Madam Chair.
MS. Eshoo. Yes, well, I know that it went over by almost a
minute, but it is a very important discussion, and perhaps
other members will follow up on the--on the points that are
being made.
It is a pleasure to recognize the gentleman from Michigan,
the former chairman of the full committee, Mr. Upton, for his 5
minutes of questions.
Mr. Upton. Thank you. Thank you, Madam Chair, and I am
delighted to be here. And I also just want to say, I want to
thank Mr. Sarbanes and others--Republicans and Democrats that
are a cosponsor of this important legislation on school-based
health centers, something that we need to reauthorize. For me,
I have been terribly supportive of this program. I would note
that in my district I have a number of these facilities. And I
am very concerned that, particularly, as schools are going to
have limited openings, it appears, from e-learning, limited
days, how exactly are we going to make sure that our kids, and
school kids and their families then are able to benefit from
these school-based clinics? And I guess that question is for
Mr. Boyd--Dr. Boyd, if you mind commenting on that. I will put
the earphones back on. Thank you.
Mr. Boyd. Yes, Congressman, it is "Mr."
Schools did not close. They went to remote learning.
School-based health centers, to the best of their abilities,
did not close, they went to remote operations through
telehealth. Our dilemma--and I am not an alarmist, I worked
with Congresswoman Brooks for a number of years on school
safety--we have a pending healthcare crisis coming in our
schools that is shared by all of the major education groups,
all of the major school-based health partners that we have, and
the nurses, the psychologists, the counselors, and the social
workers. And it is not just mental health. Kids are not getting
their vaccines. They are not getting their lifesaving
medications for diabetes and treatments for asthma. We have to
address these issues.
The school-based health centers are going to continue to
try to serve their students. I know anecdotally, we don't have
a lot of good data, but we have been in close touch with
school-based health centers around the country. I know
anecdotally of stories of clinicians asking school resource
officers and educators to go check up on children that they
haven't heard from. We don't know how many Title 1 school kids
have not been served by the continuing of education through
remote learning, or the continuing of healthcare through
telehealth. We just don't know what the numbers are yet.
So, I want to make clear that--that we are going to
continue to operate to the best of our abilities, whether
school buildings reopen or not.
One of our great concerns at the Alliance is the ability of
school-based health centers to reopen. A number of them are
sponsored by federally qualified health centers, some by public
health agencies, some by hospital systems, some by school
districts.
In the cases where they could take those workers and put
them back on the front line, keep them on the front line, in
the primary community health center or the primary hospital,
they did just that. The question will be whether or not they
will be able to move them back to the schools should that
building reopen.
And so, we have a pending crisis and, you know, we are
going to be right there on the front lines with our partners in
education to address the needs of the students that we serve,
but the resources are short. The resources are scant.
Mr. Upton. So a quick question to finish up on my time. So
schools in Michigan pretty much closed in March, in March, and
they were shut down through their Easter break. And it is
somewhat unclear as schools struggle--I talked to a number of
our superintendents--my superintendents this past week as they
are trying to prepare for opening up at some point in the next
number of weeks. Usually, the end of August is when they do it.
Do we have any sense of how many children in any of the
States actually missed a vaccination, or perhaps got into--any
even anecdotal evidence of kids running into trouble because
their health needs were unmet as it related not only to the
school closings in the spring, but, also, the preparations as
they--they have to include that when they begin to open up this
fall?
Mr. Boyd. Yes, it is only anecdotal at this point, Mr.
Upton. It is--it is not--there is no data at this point for us
to be able to know how many children have missed vaccinations
and how many children have--that no one has even heard from.
Anecdotally, I have heard from colleagues at the Coalition
of Community Schools that it could be as many as a third of
Title 1 school children that no one has heard from in some
districts. That is a scary number, because it is not just the
education side that we are not hearing from them, they are not
getting their healthcare because the school-based health center
was their health home. We may see that start to turn up in
reports from emergency rooms, because that is where they would
seek help if they are not getting it at the school. But we
don't have those kinds of numbers. They have got their hands
full right now just with the coronavirus.
We do know that we are all very concerned, as I said in my
remarks, about abuse. That we have no idea what the numbers are
of--going on with school children around abuse. We just don't
know.
Mr. Upton. Thank you. I know my time has expired. I yield
back. Thank you very much.
Mr. Boyd. Thank you, sir.
Ms. Eshoo. The gentleman yields back.
A pleasure to recognize the gentleman from North Carolina,
Mr. Butterfield, for his 5 minutes of questions.
Mr. Butterfield. Thank you very much, Ms. Eshoo, for
convening this hearing today, and thank you for your passionate
leadership, not just on these issues, but so many issues that
we have dealt with as a committee. Thank you to our full
committee chairman, my friend, Frank Pallone, I thank you,
Frank, for your leadership. I certainly respect your views on
PRVs and just look forward, look forward to working with you to
get this right. I know you have a strong conviction for helping
young children who are affected with cancer, and I know we
will--we will find some common ground to get this thing right.
And, finally, thank you to the six witnesses, thank you so
very much for your passion. I can literally feel your passion
as you testify today.
Let me just hone in on Ms. Goodman, and I have met you
before, Ms. Goodman, and just thank you for your strength and
what you bring to this debate.
Ms. Goodman, your testimony indicates that the Rare
Pediatric Disease Priority Review Voucher--of course, we call
it PRV--that this program is working as intended. You mentioned
that both the number of rare pediatric disease designations and
the number of drugs approved for these conditions actually
increased after the creation of the program.
A scientist at Duke University, which is located in my
congressional district in North Carolina, whose invention is in
clinical trials in children with brain tumors, this, this doc
told my office that even though brain tumors are the most
common cause of death from cancer in children and infants, they
are still rare. And for most children, there is no effective,
safe therapy available. He said that this program serves a
very, very unique role in assuring continued clinical research
efforts on behalf of a vulnerable and underserved patient
community.
And so, Ms. Goodman, it has been eight years, eight long
years since the rare pediatric disease, the PRV was created,
yet we know that that--that it often takes a decade, ten years
or more to develop the drug. My bill, which is cosponsored by
16 members, and I think one more has joined us this morning, 17
members of this committee, would permanently reauthorize this
lifesaving program.
How would a long-term extension of the PRV impact research
and drug development for children? Please help me with this,
and put your comments in the record.
Ms. Goodman. Thank you, Congressman Butterfield. Look, even
though this program has been in effect for eight years due to
your leadership, each time we have had to reauthorize the
program, we have only had an opportunity to do reauthorizations
of three months, one year, one year and four years. So drug
developers, at the very beginning of the drug development
process, when they are looking 10 or 15 years out, they don't
have any assurance that the program is going to be there for
them. And so they may or may not be persuaded by the existence
of the Pediatric Voucher Program, and they may or may not use
the possibility of getting the voucher if the program is
reauthorized to start developing drugs for seriously-ill
children.
If we have a permanent program, we are going to see many,
many more drugs for seriously ill kids.
Mr. Butterfield. Well, you know, as legislators, we are
always concerned about the cost of a program to the taxpayer.
Can you tell me if this program has cost our taxpayers any
money at all?
Ms. Goodman. No, sir. No money at all. The CBO scored this
program at zero.
Mr. Butterfield. Very good. Ms. Goodman, as Jacob's mom,
and as the founder of Kids Versus Cancer, do you think--do you
think that the Rare Pediatric Disease Program should be
permanently reauthorized, not only your view, but the viewpoint
of our stakeholders and our friends that you associate with
every day?
Ms. Goodman. I do. I believe that permanent reauthorization
will ensure that this incentive has maximum effect. And I have
not received a cent from any of these vouchers, but I don't
mind if other biotechs are well-funded so that they develop
drugs for seriously-ill kids.
Mr. Butterfield. And, finally, let me close out by speaking
directly to Mr. Boyd. Dr. Boyd, thank you very much for your
testimony today. I realize that COVID is different in every
State, in every jurisdiction. But does it matter that a school
district has insufficient funding? The tier 1 school systems
that I specifically have in mind, has funding become a factor
in the decision to reopen?
Mr. Boyd. Yes, as of right now, sir, we don't receive any
funding from educational sources. Our funding is through grants
that help get the school-based health center open, and then
from reimbursements from, primarily, Medicaid and SHIP.
Although, some are set up to take private health insurance. But
for the most part, the kids we serve don't--don't have private
insurance.
We are hopeful in the Senate bill that is being considered,
the Republican Senate bill that school-based health centers
will become an eligible use, so we would hope that you would
consider that if it comes before the House so that we can have
money to reopen and restart.
Mr. Butterfield. Thank you very much. Those negotiations
are taking place as we talk. Thank you very much.
Ms. Eshoo. Am I unmuted?
Mr. Butterfield. I yield back.
Ms. Eshoo. The gentleman yields back.
A pleasure to recognize the gentleman from Kentucky, Mr.
Guthrie, for his 5 minutes of questions.
Mr. Guthrie. Thank you, Madam Chair. Thanks for having this
and hosting this meeting.
And to Ms. Goodman, thanks for being here and honoring your
son Jacob with your effort. I guess, we are--according to your
timeframe, so when I was in junior high, which was about 40
years ago, I lost a friend. And every time I see St. Jude ads,
I always think of her. We were from the South, and so she went
to St. Jude. And in this day and with her category, she
probably would have survived. So what we are doing here is
important, and research really changes family's lives. So
thanks for that.
My question, though, is for Mr. Lindberg. And I am on the
registry as well. We had a big bone marrow drive for someone in
our community's son, there was a young person too that needed
bone marrow, so I had a huge drive. And I didn't match him, but
later on, I matched and got called and went and did all of the
process. It is a lot when you--when you donate, actually. And I
got right to the point where I was supposedly scheduled to go
donate, and it was cancelled. And I always hoped and prayed
that it was because they found a better match. They wouldn't
tell me why. That is what I always kind of wondered, who that
other person was. We never were able to get that far down the
process.
But, hopefully, he or she found a better match and is still
with us today. I just would love to know that, but I know there
is no way to know.
But during June, what is remarkable is that the bone--your
program facilitated more transplants in June than ever before,
and it was encouraging just to see during the pan--in the
middle of a global pandemic.
So my questions are, could you please speak to your work
with the Health and Human Services in order to ensure these
transplants were possible in June? And moving forward, what
needs to be done to ensure transplants can continue during the
coronavirus pandemic, and how do we--how can we prepare better?
So you--as you work at HHS, to ensure that we can continue
moving forward, and then what do you think we can do to make it
better during--if another pandemic comes to be better prepared?
Thank you.
Mr. Lindberg. Thank you, Congressman Guthrie. And thank you
for going through the process, almost getting to that point
where you donated.
I can tell you that after 15 years of working with this
program myself, just that hope that having a donor, a
potentially matching donor available for a patient, that hope
means an awful lot. And, like you, I will hope that there was
just simply a better DNA match donor for that patient out
there.
Thanks for your question. With respect to HHS, we have
enjoyed so, so many decades of public private partnerships with
our colleagues at HRSA and HHS. They have been extraordinarily
helpful to us throughout the course of pandemic. We work
shoulder to shoulder with them.
One example that I will provide is that as we were very
much struggling with commercial flights being cancelled, one of
the things that I will mention to the committee quickly is that
we take these courier trips so seriously, many times, many,
many times patients have been mild-like, meaning their immune
systems have been removed before a product that is collected
internationally can be delivered to their transplant center. So
if we--if we miss, that has very, very dire consequences. So we
can't miss.
We take--our couriers take these products in coolers, and
they enter into the passenger compartment of a commercial
airline many times and fly overseas to bring those products
back to the patient's bedside.
As those commercial flights started to dry up, as schedules
started to be eviscerated as the pandemic hit, one of the
things that we have worked with HHS around was they had
received an in-kind donation of private flight time from an
entity that I can't name because they have added--a private
entity that has asked to remain confidential.
But those--those flight hours were donated to us, and we
were able to work through HHS to procure these private flights
to move donors and couriers overseas, so that we didn't miss--
like I said, we didn't miss one time. I think that is a
remarkable thing. And it is just--it is grit and determination
and incredible help.
So I am so proud of my colleagues here at National Marrow
Donor Program/Be the Match, and I am proud of our partnership
with the government over 30 years. It has been extraordinary.
In terms of what we can do going forward, this program
reauthorization and doing it in a timely way is of immediate
importance to us. And my colleagues here at the NMDP, so much
of our success over the course of the last four months has
pivoted on our national program status.
I fear that if a program lapses, we may lose some of that
really important leverage that we have had with foreign
governments, in particular. If we lose the full force of the
Federal Government behind us, all of a sudden we are a
nonprofit here sitting here in Minneapolis, Minnesota. It is a
really great profit----
Mr. Guthrie. I am about out of time.
Mr. Lindberg. Yes. Thank you.
Mr. Guthrie. I am out of time. Thank you very much. I am
56. So, hopefully, I will get called within the next 5 years.
Mr. Lindberg. Thank you, sir.
Mr. Guthrie. So I appreciate it very much. And thank you
all for everybody else, and I am sorry I didn't get to your
questions. I yield back.
Ms. Eshoo. The gentleman yields back.
It is a pleasure to recognize our wonderful colleague from
California, Ms. Matsui, for her 5 minutes of questions.
Ms. Matsui. Thank you very much, Madam Chair, for having
this important hearing. And I thank the witnesses for being
here today. These are all important bills, and we all want them
to pass. And a special thank you to Mr. Lindberg for testifying
today about the importance of the National Bone Marrow Program
and Cord Blood Inventory. You can tell it is a popular subject,
because many people have already talked about it, and some of
the questions have already been covered. But I think we should
keep talking about it more, because the bone marrow program is
really very critical.
In fact, my late husband, Bob Matsui, had MDS, and there
was a time, I guess, 15 years ago or more, when he was ill,
when it wasn't really possible for him to get the bone marrow
that he needed. So the more we have, the better it is.
Every three minutes, we know that someone is diagnosed with
blood cancer. And for patients and families facing these fatal
diseases, a bone marrow or cord blood transplant may be the
best treatment or only potential for a cure.
Congress recognized a need to coordinate these lifesaving
transplants in creating the C.W. Bill Young Cell
Transplantation Program, a strong bipartisan public health
priority. I am proud to say that in working with my colleagues,
the program's authorization has never lapsed since it was first
enacted in 2005.
Because most patients diagnosed do not have a suitable
donor in their family, the program's national registry known as
the Be The Match which matches patients in need with possible
unrelated volunteer bone marrow, or cord blood donors, is truly
a lifeline. We have to continue to encourage donors and give
these patients with otherwise fatal blood cancers a second
chance at life. And that is why I was pleased to join
Representatives Bilirakis and Pingree to introduce the
Transplant Act of 2019, legislation to reauthorize the C.W.
Bill Young Cell Transplantation Program and the National Cord
Blood Inventory for another five years.
So, anyway, just to continue on, you mentioned in your
testimony some of the roles that the National Marrow Donor
Program plays in addition to running the national registry. Can
you elaborate a bit on the work that Be The Match and NMDP
plays?
Mr. Lindberg. I would be thrilled to do that, Congresswoman
Matsui. And thank you, again, for unwavering support over the
years. I see a direct line of sight between your advocacy for
this program and thousands of patients' lives saved here, so
thank you for that.
NMDP/Be the Match, is the largest, I would argue, most
sophisticated registry of volunteered bone marrow donors in the
world. We have, I think, been now, again, a world leader over
the course of the last four months as the globe has been hit
with a pandemic. We have been able to, with the help of you and
your colleagues, pull levers that we would not have been able
to pull otherwise, with the State and foreign governments in
order to continue to move these lifesaving products in a timely
way all across the world. And we continue--and we intend to
continue to do that.
Ms. Matsui. OK. You know, the number of transplants of
racial and ethnic minority patients has increased substantially
in the year 2000 to today. What effort is Be The Match making
continue to expand the diversity of the registry to ensure that
minority patients can find matches?
Mr. Lindberg. Yes. Thank you for that question,
Congresswoman Matsui. I was about to go there and decided that
I would hold a moment. I wanted to mention that our 5-year
strategic plan--our mission is to save lives through self-
therapy. Our vision to do that is--we talk about democratizing
self-therapy. And what we mean by that when we say that, is
that no matter who you are, no matter your race, your
ethnicity, your religion, your creed, your economic status,
that it is our obligation to find for you that match and to
deliver that match to you and hope that your life can be saved.
And what we know today is that because of the peculiarities
of our DNA and our HLA, that it is unfortunately far more
difficult for those who are in historically underserved
populations to find that good match.
So we have doubled down on our efforts to serve underserved
communities, and we have set up a metric that we are moving
well towards to double the number of transplants in ethnic
populations in five years.
Ms. Matsui. Well, thank you very much. I appreciate your
hard work. And we know this has to be reauthorized. It is such
important work. So, thank you very much, and I yield back.
Ms. Eshoo. The gentlewoman yields back.
The gentleman from Virginia, Mr. Griffith, is recognized
for his 5 minutes of questions. We are so happy to see you and
know that you are doing well.
Mr. Griffith. Thanks. I am glad to be with you all. And,
yes, things are going very well. It is really interesting, I
was the 17th member who signed on to continuing and permanently
funding Chairman--Mr. Butterfield's bill. And I will just
relate to you what happened. Sometimes timing is important. I
was at the pool within the last couple of days with my boys who
are 14 and 12. And I was talking to a dad whose son is entering
the 6th grade, and he has just gotten over leukemia, or is at
least in remission. We were talking about this very program,
and I reiterated to him my support for it. I couldn't think of
a better way to show that support than signing onto the bill,
and, hopefully, we can get this through and we were very
pleased to have done that. But we all know somebody who is
affected, or might be affected by this, and it just brings it
all home.
That being said, and I appreciate that. I do want to go
to----
Mr. Butterfield. Thank you, sir. Thank you.
Mr. Griffith. Thank you. I appreciate you carrying it. I do
want to go to the Anti-Doping Agency, and I want to talk a
little about that.
Mr. Tygart, are you available for a question there, or I
guess you are somewhere----
Mr. Tygart. I sure am. Yes, sir.
Mr. Griffith [continue]. In the internet? In your written
testimony you talk about the World Anti-Doping Agency, and you
reference that the United States is not able to be on the
executive committee. What is the stated reason for that?
Because we have been leading and pushing hard to make the
Olympics better. Why aren't we on that executive committee of
the World Anti-Doping Agency?
Mr. Tygart. Yes, the process for selection of the countries
around the executive committee, we fall into the Americas
region, which is 43 countries in South Latin, the Caribbean,
North America as well. And they had a rule change that says the
chair of a council of sports ministers gets the seat at the
WADA executive committee. And you may know, we don't have a
sports minister in the United States. So while you know there
may be some technical ability to take that position, I think
the reality is that no way the U.S. is going to have that seat.
Both us and Canada have been extremely disappointed because we
are the two largest funders to WADA as well.
Mr. Griffith. And do you think the rules change,
specifically, to exclude Canada and the U.S. because of our
strong anti-doping position?
Mr. Tygart. It is a--certainly, that suggestion has been
put out there. I don't have the--as a lawyer, I don't have the
proof that I would want to prove it, but certainly that
skepticism and question has to be asked.
Mr. Griffith. But what you are saying is, is that it is not
just a conspiracy theory, there might be a little something
there, if there is smoke, there might be some fire?
Mr. Tygart. It is just hard for me to believe that the
largest funder and one of the powerful sport countries as well
as a country that has been leading with an independent model
since 2000 does not have a seat at the most powerful committee
at the World Anti-Doping Agency, and, realistically, does not
look to have one in the near future.
Mr. Griffith. And that is very disappointing. While I
certainly hope that we will continue to fund your agency, is
there any action we should be taking to, as a Nation, to
express our disappointment and perhaps roll back our funding to
that World Anti-Doping Agency if they are going to exclude us?
Mr. Tygart. I think what ONDCP and we, as I said in my
testimony, applaud the effort that they went to, at the request
of congressional appropriators for ONDCP to provide them a
report, they lay it out perfectly well, and they ask for the
opportunity to have discretion in their funding. I think up
until now, they have seen it only as a contract that they had
to pay, regardless of what WADA did, reform-wise or otherwise.
And so we would--we would love--and we were thrilled that the
House Appropriations Subcommittee FSGG has given them, in its
markup, the discretion that they asked for, and we hope that
the Senate does the same, and then allow ONDCP, who has the
status within WADA's eyes, to be able to sit down with WADA and
negotiate before just removing the money. But at the end of the
day, without any leverage, money being the biggest leverage,
the likelihood of change there is not realistic.
Mr. Griffith. Well, and I think that is important as well.
As many members of this committee may recall, and I don't
remember if it was this subcommittee or not, but we had Michael
Phelps and a number of others in a couple of years ago talking
about this very issue. And I think it is important--I think it
is extremely important, and I think this committee thinks it is
important that we make sure that we don't have doping in our
sports in the United States, or in our sports across the world
as our athletes compete. But I think do appreciate your hard
work. Is there anything else that you want us to know before I
finish my questioning?
Mr. Tygart. I think you are absolutely right, and there is
no better testament to the number of household names in the
Olympic movement, Paralympic movement who stand beside us, arm
in arm, to fight for their right to clean and healthy and safe
sporting. And we really appreciate the government's effort on
this to give them the hope that they can compete in the right
way.
Mr. Griffith. Well, I appreciate that. I see my time is up.
Madam Chair, I yield back.
Ms. Eshoo. The gentleman yields back.
It is a pleasure to recognize the gentlewoman from Florida,
Ms. Castor, for her 5 minutes of questions. Nice to see you,
Kat.
Ms. Castor. Nice to see you. Thank you, Chairwoman Eshoo.
This is an important hearing with a lot of terrific bills, so
thank you. And thanks to our witnesses who are here today. We
need more school-based, safe health centers across America, so
I want to thank my colleagues, Representative Sarbanes,
Representative Tonko, for leading that effort. I am a cosponsor
of that bill. I also appreciate the focus on rare pediatric
diseases and Creating Hope for Families, and what else we need
to do in stem cells and then anti-doping sphere as well.
I want to focus on the EARLY Act. I am a cosponsor of the
Early Act, and I am grateful to Congresswoman Debbie Wasserman
Schultz and Congresswoman Brooks for joining together to stand
up for young women.
Ms. Blount----
Ms. Blount. Yes.
Ms. Castor [continue]. Congratulations to you. I understand
the Black Women's Health Imperative is celebrating 38 years of
advocacy----
Ms. Blount. We are.
Ms. Castor [continue]. Advocacy for health equity for Black
women and girls across the country. I am hopeful that the EARLY
Act will help improve the diagnosis rates for African American
women contracting breast cancer. And I hope the EARLY Act would
also help--help improve their care after they are diagnosed.
In your testimony, you cite a number of very troubling
statistics. Black women develop breast cancer typically 5 to 7
years younger than White women. And African American women are
40 percent more likely to die after a breast cancer diagnosis.
That is not acceptable in this country. Would you mind walking
us through the reasons for that, and what else we should be
doing about it?
Ms. Blount. Yes, thank you for your question and your
concern, Congresswoman, I appreciate that. There are a number
reasons, as you might imagine. A lot of researchers will point
to obesity rates, they will point to underlying chronic
conditions, they will point to childbearing patterns among
Black women as an explanation for why they are getting breast
cancer younger. And that may be true, to some degree, but that
can't explain it all, because if you look at Black women and
White women in control for obesity rates, control for
childbearing patterns, age at when they had their first child,
that doesn't explain the difference in breast cancer rates.
So the answer is, we don't know exactly. There is something
going on. And what a lot of researchers are now exploring, and
frankly, have been for quite a number of years is looking at
the impact of elevated cortisol levels. Black women have, on
average, about 15 percent more cortisol in their bloodstream
than White women. And we know a lot about what that does to the
body. It triggers our inflammatory response, which, in fact,
raises our risks for chronic disease. But we also now know that
elevated--chronically elevated cortisol also raises our risk
for things like incompetent cervixes. Infant mortality and
maternal mortality have been demonstrated for years.
So what we need to understand more about is what this--what
is happening, because we know it is not biology, we know it is
not genetics, but what is happening in the lived experiences of
the Black woman that actually raises their risk for chronic
diseases, particularly, breast cancer?
Ms. Castor. So what is the most effective--what successful
outreach initiatives have you seen that you would recommend to
us?
Ms. Blount. Well, I have seen a number of community-based
outreach initiatives. I mean, our organization, is of course,
among many to make sure to educate Black women on the
importance of screening mammography, in particular, and
starting at age 40. This is very much a grassroots kind of
activity where we have got to make sure that providers
recommend screening mammography. You would be amazed at the
percentage of providers who don't suggest that their patients
get the screening mammogram, starting at age 40, and to explain
the process.
So we and other organizations have been involved working
with federally qualified health centers, and with the NMA and
other medical associations to help educate Black women on the
importance--and Latinas on the importance of getting that first
mammogram, understanding your family history, and getting that
early mammogram so that you know what your risks might be, and
can take the proper steps.
Ms. Castor. That would save lives.
Ms. Blount. It would indeed.
Ms. Castor. Thanks for your important work.
Ms. Blount. Thank you, Congresswoman.
Ms. Eshoo. The gentlewoman yields back.
It is a pleasure to recognize--oh, God, every time I say
Representative Brooks, my heart sinks because we don't want you
to leave. But I am going to recognize you for your questions.
It is wonderful to see you.
Mrs. Brooks. Thank you, Madam Chairwoman, And thank you so
much for those kind words. But thank you to all of the
witnesses for your passionate testimony. All of these bills are
critically important in ensuring that we reauthorize all of
these very important five public health bills.
It is also good to see you, Mr. Boyd. We have done work
together in the past on school safety. And as the mother of a
Title 1 teacher, all that you said about the concerns for our
school-based programs, health centers are critically important.
I want to focus my time today, though, and continue to
follow up on what my colleague, Ms. Castor, was just talking
about with respect to breast cancer. It is not just another
disease. It is personal for so many of us. I think it is safe
to assume that everyone in this hearing has been impacted in
one way or another by someone who suffered from breast cancer.
And, in fact, our colleague and dear friend, Debbie Wasserman
Schultz, suffered from early breast cancer at the age of 41
back in 2007, and she has been a passionate advocate, as has
the chairwoman in fighting to make sure that young women have
access to screening.
And I want to continue to talk about the importance of
early detection, because we know that--we know that the higher
chance a woman has in getting detected, they will beat that
cancer.
And, then, Ms. Blount, you stated in your testimony, I
think it is worth repeating, that if we can increase breast
cancer screenings in women under 45 by just 50 percent, 3,000
more women per year will survive their diagnosis. Yet, I think
many young women, in particular, don't think about the
importance of getting screened. And as you have said, the
providers often don't encourage them to get screened.
So while we are--let's talk a little bit further about what
is the state of our Nation's public awareness and education
efforts, in your opinion, particularly focused on younger
women? Can you please share with us what you view as the state
of our Nation's public awareness campaigns and education
efforts?
Ms. Blount. I can. And thank you for that question,
Representative Brooks. It is certainly not where it needs to
be. You know, young people often feel invincible, as we have
seen with COVID-19 recently, and, you know, a young woman
doesn't want to think about breast cancer. I understand it. I
understand it completely. But we have got to do more about
making sure that she understands her risk and, particularly,
her family history.
So, you know, we have got the National Breast and Cervical
Cancer Early Detection Program which helps, but we also know
there are funding challenges with that program. In some States,
the program may run out of money 6, 7, 8 months into the year.
And while there is outreach that is done, and we hope treatment
occurs when there is a suspicious finding, you know, there is
not the resources to reach all the women and, particularly,
young women, frankly, in the way they need to hear the message.
Our public health conversation, our public health programs, the
kind of language, imagery we use is, frankly, fairly old
school.
So, a lot of what we in my organization are trying to do is
use modern-day images, terminology, communications mechanisms
to make this message relevant to where women are today. And if
we can do that, we can get them into screening early.
Mrs. Brooks. Thank you very much. Can you actually discuss
a few of the strategies that your organization, Black Women's
Health Imperative, is using to educate these women? What kind
of strategies, specifically, is your organization using, and
how can we ensure that these young women get the access that
they need?
Ms. Blount. We are, of course, using social media. We are
going right to where these young women are on Instagram to talk
about the importance of breast health. We have launched what we
call Black & Well TV where we have conversations with notable
influencers and celebrities and medical professionals and
public health professionals to talk about the importance of
early screening. It is, you know--it is delivered via
streaming. And we hope in the not-too-distant future to
actually have a partnership where we can have a more national
spokesperson convey this kind of information in the way Black
women need to hear it in order to act on it, so that they
understand that this is important, but, most importantly, that
they don't need to be afraid.
We are particularly concerned now that COVID-19 may
actually keep women out of mammography centers, and that
screening rates may go down. So we have got to make sure that
Black women understand that it is still important to do, and
that there are safe ways to do it.
Mrs. Brooks. Well, thank you. Thank you for your important
advocacy, and we do know that early detection works and will
save lives. So thank you for your important work. I yield back.
Ms. Blount. Thank you.
Ms. Eshoo. The gentlewoman yields back.
It is a pleasure to recognize our colleague from
California, Mr. Cardenas, for his 5 minutes of questions. You
need to unmute. Mr. Cardenas?
Mr. Cardenas. Yes. Thank you. Can you hear me? I am on. OK.
Thank you. Thank you, Madam Chair, and Ranking Member Burgess,
for holding this important legislative hearing. Reauthorizing
critical public health programs is very, very important to our
country, and I hope people understand that critical need,
especially during this pandemic. And a big thank you to our
witnesses. I really appreciate your expertise and your opinions
today.
My first question has to do with schools. So I am going to
ask Mr. Boyd a question. I am a longtime supporter of the
School-Based Health Centers Program, and I am very glad to see
prioritizing legislation today to reauthorize this program that
provides healthcare for 3.6 million youth, predominantly from
low-income families.
I am very, very proud to say that I grew up in the second
largest school district in the country. And the school-based
health clinic--I think it was the first one in the entire
district, was at San Fernando High School, my high school. And
what is really important in all aspects of healthcare is
cultural competency, and especially when it comes to linguistic
differences.
So that school was very--and that clinic that sponsored it
was very smart in carrying my brother, who was a clinical
psychologist of linguistics and competent in two languages,
Spanish and English, and also culturally competent as well. He
grew up in that neighborhood. He and my ten brothers and
sisters went to that high school. So he was able to really
provide a service above and beyond what he learned in college
to get his degrees.
So I worry about the massive wave of behavioral health
issues experts warn about when we are talking about this
pandemic, staying-at-home orders, virtual learning, and more.
People having to educate their own children, that in and of
itself is a big issue these days.
My question has to do with how could additional school-
based health funding support students with mental health and
substance abuse disorders? Like I said, my brother, the
psychologist, was at the school-based clinic. So it was mental
and physical health provided there. Mr. Boyd?
Mr. Boyd. Yes, Congressman, you raised two important
issues: one, access. The number of school-based health centers
from my perspective, from our organization's perspective is
unacceptable. There are few within 3,000 of us pre-pandemic,
and we have got 25,000 fully eligible Title 1 schools. So we
have got to grow the number. That then poses a human capital
problem. We can't take people out of the--out of colleges and
just throw them to work in school-based health centers. They
have got to licensed, certified, trained professionals. So over
the next ten years, we have to address that human capital
issue, and it has to address cultural competence.
In seminary, I was always taught to look for the good news.
The good news in the pandemic is that a lot of behavioral
health specialists have said, OK, we accept the fact that
telehealth is here to stay. Now the opportunity that that
presents is how do we get culturally competent mental health
professionals to the schools that need them, potentially using
telehealth? And that is something that a number of us are
working on right now, trying to put together a strategy to
bring to the Federal agencies to say, we have the capability of
doing this through telehealth. We have got to address the State
licensing issues post-pandemic. Right now, it is not as much of
an issue.
But you are right on point: Human capital is the number one
issue as we look to expand the number of school-based health
centers over the next ten years.
Mr. Cardenas. Thank you, Mr. Boyd. And I just want to state
for the record: Tomorrow, I am introducing the bipartisan bill
with some of my NC colleagues, behavioral health technical
assistance and training centers for schools within SAMHSA.
SAMHSA, for those who don't--who are viewing this, is the
Substance Abuse and Mental Health Service Administration. This
would help schools and school systems better support students
with mental health and substance use disorders.
I hope this important legislation gains momentum and would
welcome support from the School-Based Health Alliance.
Mr. Boyd, what impact do you think COVID-19 will have on
the health and well-being of our students? And, also, how can
we better help prepare you all to address this in the coming
school year?
Mr. Boyd. It is--like I said, it is a disaster. It is a
disaster on the mental health side. It is going to be a
disaster for the children that need daily medicine management
and behavioral health services. We need more of us. The
opportunity using telehealth is a great one because the cost of
privacy-protected platforms have come down, and the cost of
equipment that primary care providers use has come down
literally 90 percent in the last 18 months. Where it was
$23,000 a unit this time last year, it is $2,300 a unit for
that primary care equipment.
So, you know, to those of you on Energy and Commerce that
are also on Communications, turning to the FCC and saying, Use
some of the universal services fund to enable us to buy that
equipment and provide those platforms through school-based
health centers, is critical. We need those resources. We simply
don't have them. It is one thing to get reimbursed, but if you
don't have the capacity to be there and you don't have the
trained professionals to serve, it is not going to matter. We
have got to have the equipment, and we have got to have that
kind of funding.
Mr. Cardenas. Thank you, Mr. Boyd.
Madam Chair, if you will allow me a point of personal
privilege about our colleague, Mrs. Brooks. I agree with your
sentiment, sad to see her leave. And I just want to quote my 2-
year-old granddaughter who is bilingual. Every time I leave,
she touches me by saying, "No te vayas, no te vayas." That is
"Don't go. Don't go." Take care.
Ms. Eshoo. That is beautiful. Thank you. And the gentleman
yields back. Pleasure to recognize the gentleman from Montana,
Mr. Gianforte, for his 5 minutes of questions.
Mr. Gianforte. Yes, thank you, Madam Chair.
Ms. Eshoo. Oh, I am sorry. I am sorry, I am sorry. I need
to withdraw that. I was wrong.
Mr. Mullin, our colleague from Oklahoma, I am sorry. I
apologize to you, Mark. You are now recognized for 5 minutes.
Mr. Mullin. Madam Chair, thank you so much.
Ms. Eshoo. Mr. Gianforte, promise you.
Mr. Mullin. Well, Madam Chair, thank you so much. I would
have gladly yield to hopefully the next Governor of Montana,
Greg Gianforte. But I got to say this, Tony, you said that you
hate to see Susan go, I am mixed feelings because I don't want
her to go, but at the same time, I get to move up on the dais
as she does. So I am kind of mixed on this one. No, I thank you
so much for having this important hearing, Madam Chair.
Ms. Goodman, my question is going to be referred to you,
and I just got to--reading your story about your son, I just
want to tell you how much I appreciate you continuing, even a
decade later, advocating for these important causes. Literally,
I just ran out of where I am staying temporarily. I am not even
at my house or in the office, because my son had a major brain
injury, and I had to just drop him off with specialists at the
Centre For Neuro Skills in Bakersfield and ran back in here to
answer questions.
And as you know with pediatric care, there is a disconnect
between adult care and pediatric care; what is available to our
children, versus what is available to adults, and it is
disproportionately wrong. And, so, I just want to tell you how
much I appreciate your advocacy, but not just on medication,
but on rehab and development, and the encouragement of biotech
companies to be involved in this area.
So personally, Ms. Goodman, I just want to tell you, thank
you so much for being such an advocate for all of our kids.
In your testimony, you mentioned pediatric rare disease
drug development is mostly done by small biotech companies,
which is true. Can you comment on how Rare Pediatric Disease
Priority Review Voucher Program works to get more funding to
these small inventors?
Ms. Goodman. Sure. Thank you so much, Congressman Mullin,
and first of all, we are all keeping your son in our thoughts
and our prayers for a, you know, a full and speedy recovery.
Mr. Mullin. Thank you.
Ms. Goodman. So the voucher program is really critical for
biotech, many of which are thinly capitalized, because the
opportunity for them to create a voucher, it gives them a
chance to attract additional investors and even to sell their
designation early in the drug development process and monetize
the designation, you know, phase 1 or phase 2, as soon as they
receive it, so that they can use that funding for development
of the drugs for kids who really need these novel therapies.
Mr. Mullin. How is the program funded to support the FDA
reviewers as needed to review products on the expedited
timeline? Are you familiar with that?
Ms. Goodman. Yes, sir. Thank you for that question. So
every year, the FDA calculates what the expense was to the FDA
of this program, and the FDA sets a new pediatric voucher user
fee for the following year. And the current user fee is $2.1
million, and that is on top of the PDUFA user fee that a
sponsor would pay, which is about $2.9 million.
Mr. Mullin. Do you know if the program has a CBO score on
either what it costs or what it saves the American taxpayers?
Ms. Goodman. Yes. The score is zero.
Mr. Mullin. And I think, obviously in asking these
questions, I know the answer, but I think a lot of people need
to understand that, that we are not--we can invest in this
without costing us anything because of the return.
And once again, your advocacy on this is something that I
think every parent that has been through what you have been
through, commends you on, and I just can't thank you enough.
What measures does the FDA have in place to ensure that the
safety and the efficiency of these drugs go through the
expedited approval process?
Ms. Goodman. So, I think your question is, how can we be
sure that the FDA approves drugs in a safe manner?
Mr. Mullin. Yes.
Ms. Goodman. Is that your question?
Mr. Mullin. Yes. Absolutely, yes.
Ms. Goodman. Look, I think that people who choose to work
at the FDA are just smart and incredibly impassioned and
dedicated people, and they make sure that any drugs that they
approve, you know, meet their rigorous standards. The targets
they need to meet under this, or any other PDUFA program, are
not required. They are not mandatory on the FDA, they are
optional. They are only--FDA is only going to approve drugs if
they are safe.
Mr. Mullin. Right. And I, once again, know the answer to
this, but without congressional action, the Pediatric Rare
Disease Priority Review Voucher is set to expire at the end of
this year. What would that mean to you, and to parents that are
going through the same things that you went through?
Ms. Goodman. Thank you. You know, we have had almost two
dozen new drugs for seriously ill kids approved in the past
eight years since the program was developed, and I think it is
going to dry up.
Mr. Mullin. Right. Well, Ms. Goodman, thank you again for
your time and for being here.
Madam Chair, thank you for holding this important hearing,
and with that, I yield back.
Ms. Eshoo. The gentleman yields back. It is a pleasure to
recognize the gentlemen from Maryland, Mr. Sarbanes, for 5
minutes, for his questions.
Mr. Sarbanes. Thank you very much, Madam Chair. Can you
hear me?
Voice. Yes, sir.
Mr. Sarbanes. OK. Appreciate all of the bills that we are
discussing here today. Again, very gratified that among them is
the School-Based Health Center Reauthorization bill, and I want
to thank my colleagues for their support of it and their
interest in the topic.
And Mr. Boyd, I want to thank you for your leadership at
School-Based Health Alliance and the testimony that you have
given us today. I can say, having visited many, many school-
based health centers over the last few years, trying to
understand all the dimensions in which they can provide support
for children and families, that some of the most impressive
practitioners and health professionals I have ever met are the
people that staff these school-based health centers.
The amount of support they give to the school, to the
students, to the families, and to the community, really can't
be overstated. So you are representing a very proud and
resourceful group of people, and we thank you for being with us
today.
The school-based health centers, I kind of look at them as
having two basic functions, or opportunities. One is obviously
to serve what is a captive audience, which are the children
that are located in that school, and to take full advantage of
the fact that you have them there, or that you are connected to
them if we look at it now through the lens of the pandemic and
what that is doing to kind of change the status quo.
But the other is that through children who come to these
school-based health centers, or are served by them, they act as
a link to the families of those students and can help connect
families to healthcare resources that are more broadly
available. Sometimes that is through actual partnerships with
community health clinics, including federally qualified health
centers. Other times it is more through referral to other
providers that are in the community.
But maybe you could speak a little bit to that, how the
health centers in these schools can be a gateway of opportunity
to access broader health supports, whether it is physical
health or mental health, emotional health, that are needed in
those communities?
Mr. Boyd. That is a great question, Congressman. Pre-
pandemic--and I have to keep speaking of that because we don't
know how many school-based health centers are going to have the
resources to reopen--but pre-pandemic, the majority, over 51
percent of the school-based health centers were sponsored by
federally qualified health centers. So that gave a tremendous
link between the families' overall healthcare needs and
addressing the healthcare needs of the students.
The NACHC, the National Association of Community Health
Centers, has identified, and put in their strategic plan for
this next year, an interest and a direction for federally
qualified health centers to look more carefully at school-based
health centers as an opportunity for them to expand their
business models.
You speak to the quality of the people that work in the
school-based health centers. Clearly, they could make more
money. This is their calling. They are like teachers. They are
not there because it is the best-paying job in the world; they
are there because of their dedication and their commitment to
serve, and particularly to serve children, and in particular,
to serve children in low-income communities because that is
where most of the SBHCs are located.
So, yes, absolutely, and pre-pandemic, we were in process
of arranging a visit, for us to go with you to visit some of
those centers in your district, and hopefully we will get the
opportunity to do that in the future.
Mr. Sarbanes. Well, I look forward to that, and I
appreciate very much your raising the issue of compensation for
those who staff the school-based health centers. If you think
about it, there is no more important position, given all of the
different dimensions that can be brought to bear by those
professionals. And we got to make sure that we recruit them
with the opportunity to, you know, make a good living, and that
we keep them.
Because that is critical as well, because they build
relationships. That was one of the most powerful testimonies I
got from a most recent visit to a school-based health center,
was the relationship that that center had built with certain
students--this was a high school--over the course of their
time. And it meant when those students had issues and stresses,
they felt like it was a place to turn, there was a relationship
there. That is exactly what we need to provide for our young
people.
So thank you for your testimony, we appreciate it very
much, and I yield my time back.
Mr. Boyd. Thank you, sir.
Ms. Eshoo. The gentleman yields back. A pleasure to
recognize the gentleman from Montana once again, Mr. Gianforte,
5 minutes for questions.
Mr. Gianforte. Thank you, Madam Chair. I appreciate the
committee meeting today to advance several bipartisan
reauthorization bills. As we know, we are reauthorizing the
School-Based Health Center Program, a program to promote
awareness and education about breast cancer in younger women,
the Cord Blood Inventory and Bone Marrow Transplant Programs,
and the U.S. Anti-Doping Agency. This is the entity that
ensures that Olympic and Paralympic athletes are not using
performance-enhancing drugs. I know we are all looking forward
to having the Olympics next year and cheering for the Americans
that will be competing.
The piece of legislation we are working on today that I am
most appreciative of is the Creating Hope Reauthorization Act.
This bill reauthorizes the Rare Pediatric Disease Priority
Review Voucher Program. This program creates an incentive for
drug companies to develop therapies for rare pediatric
diseases.
If we develop a therapy for one of these diseases affecting
children, they get a voucher to speed the FDA review of another
drug. This program has already led to the development of 22
therapies. I have heard from Montanans dealing with the loss of
their children from DIPG, a currently untreatable brain tumor.
DIPG is the second most common cause of child cancer deaths.
Encouraging more therapies and treatments for these rare
pediatric diseases is something we should all support.
Ms. Goodman, thank you for sharing your story with us
today, and you were very articulate in your response to
Markwayne Mullin's questions. Is there anything else you would
like to add about the reauthorization of this program and the
impact it has been having for children in this country.
Ms. Goodman. Thank you, Congressman Gianforte for that
question. I would just really like to just emphasize that this
program has, as you said, 22 new drugs, many new drugs in the
pipeline. The FDA has the opportunity to incorporate all of the
costs of executing this program in their user fee, and so, I
hope we can find a way to reauthorize it on a permanent basis.
Mr. Gianforte. Well, I think we have strong bipartisan
support, and you being here today and telling your story has
had quite an impact. I want to thank you for that.
I look forward to supporting these pieces of legislation,
and with that, Madam Chair, I yield back.
Ms. Eshoo. The gentleman yields back. A pleasure to
recognize the gentleman from Massachusetts, Mr. Kennedy, for
his 5 minutes of questions.
Mr. Kennedy. Madam Chair, thank you, and thank you for
holding this important hearing, as always.
Mr. Boyd, in your testimony, you mentioned that it is still
unclear how and if students will be able to go back at in-
person learning in the fall. It is a hard thing to balance--the
importance of in-person education, while also ensuring the
health and safety for our children, their families, and their
communities.
Because opening schools will not just impact students, it
impacts everyone around them. Because of this, we need to
ensure that if schools do allow for in-person learning, we
follow scientific guidelines, learn from other countries that
either began too soon, or took a more strategic approach, and
ensure we have all the necessary stakeholders at the table in
making these decisions.
So Mr. Boyd, I want to ask you if you can talk a little bit
about how you think school-based health centers should be
involved in these decisions and the planning processes for the
re-open.
Mr. Boyd. That is a great question. Thank you, Congressman.
We are part of a series of networks in Washington that include
all of the major organizations on the education side, including
the unions, the superintendents, the principals, as well as on
the health delivery side, in the nurses, the counselors, the
psychologists, the social workers, and us. Everybody, across
the board, sees the value of the work that we do. We don't see
ourselves in competition with each other. We see ourselves in
full support of each other.
We are not sure how schools should open. We think that is a
local decision that is going to have to be made on a scientific
basis, case by case. But again, I want to come back to the
point that schools are--schools, for the most part, remained
open. The question was the ability of the school to deliver
their services. It was the buildings that closed.
I don't think that most school districts today are looking
necessarily at not reopening. They are looking at whether or
not to reopen their buildings. School-based health centers have
to be a part of that conversation, and we are, at the national
level; it has to filter down to the district level. School
districts can't put out plans that don't include parents, and
in some situations, students, as well as major employers in
their communities, as well as all of the disciplines that I
have referenced.
Mr. Kennedy. And I wanted to build on that a little bit,
Mr. Boyd, because you spoke about the importance of mental
health and emotional well-being of children during this
pandemic, and it has been a big area of focus of mine, as
perhaps you know. We have seen how the pandemic has highlighted
the gaps in our own health system when it comes to mental
health, and now what has happened with the need to shelter in
place, with additional isolation and diminished care
connections and how that can negatively impact one's mental
health.
We have seen the devastating impacts of a far increased
need for mental and behavioral health services, increasing
rates of suicide, long-standing racial inequities that still
exist between Black, Native, and Latinx children and families.
So just walk me through how you are thinking of providing--
meeting that need for mental/behavioral health services for our
children and in our schools?
Mr. Boyd. Yes, I think there is several challenges.
Telehealth does provide that opportunity. For many mental
health care providers throughout this pandemic, that has been
the telephone. And while children are left--schoolchildren are
left reluctant to do that kind of a telehealth visit, it has
been the adults that have resisted it. I think many have gotten
past that.
Our dilemma then is to make sure that we protect the
privacy of those students. It is to give them a greater
opportunity to participate, but make sure that when they are
participating, they are doing it in an environment that allows
them to be honest and open without sharing confidences or the
invasion of their privacy because someone's able to hear it, or
someone's able to hear the healthcare provider on the other
end.
We have got to come up with technological solutions and
platforms that work for the kids but also protect their privacy
and work for the practitioners. That is going to be the major
challenge and the major opportunity for us going forward if we
wind up being in this situation for an extended period of time,
as it seems we are going to. We are going to have to use
technology.
Mr. Kennedy. Time is limited, so I might follow up with a
question for the record for you, but I am grateful for your
time and your willingness to highlight these issues as we try
to navigate our way forward. Thank you very much, sir.
I yield back.
Mr. Boyd. At your convenience, sir.
Ms. Eshoo. The gentleman yields back. The chair now
recognizes the gentleman from Florida, Mr. Bilirakis, for his 5
minutes of questions.
Mr. Bilirakis.
Ms. Eshoo. Are you unmuted, Mr. Bilirakis?
Mr. Bilirakis.
Ms. Eshoo. Mr. Bilirakis, you need to unmute. I guess if I
sing it, it doesn't make any difference, right?
Mr. Bilirakis.
Ms. Eshoo. Let's see, how are we going to get his
attention? Unmute.
Voice. Looks like he is trying to unmute.
Ms. Eshoo. Let's see if we have--why don't we go to
Congressman Ruiz of California, and then circle back with Mr.
Bilirakis, because I think he has some technological issues
there.
Mr. Ruiz. OK.
Ms. Eshoo. Somebody is laughing. I don't know.
Mr. Ruiz. My good friend, Gus, you are going to have to go
get a staffer to help you, my friend. I have been there. It is
a black hole for sure.
So thank you, everybody, for being here. School-based
health centers are essential for many families in my district
and around the country. I grew up in an underserved community
with poor access to healthcare. It is a farm worker community
of Coachella, California. In fact, my alma mater, Coachella
Valley High School, has a school-based health center.
While some of these, quote/unquote, health centers consist
of one exam room, limited supplies, and are often in a tiny
trailer on campus, they are critical to a child's health and
well-being, providing services such as dental prevention and
treatment, health education, mental health services, and
preventative health screenings.
Not only that, but they also serve the families of the
students, providing important continuity of care within the
family. Students and their families rely on school-based health
centers for their healthcare, because oftentimes, that is the
only access to healthcare that the family has.
These facilities tend to serve families in otherwise
underserved areas where health disparities are acute, where
people might not have insurance, or maybe can't find a doctor
who takes Medicaid, or can't take off work in the middle of a
Tuesday to take their child to the doctor. School-based health
centers fill this need, and they are critical to families who
have limited access to care.
My question to Mr. Boyd, how do school-based health centers
address health equity and the needs of underserved children and
adolescents?
Mr. Boyd. Thank you, Congressman. You raised three
important issues, and the first one I want to speak to is the
infrastructure. The average age of a public school building in
the United States of America is north of 50 years--I have built
a lot of buildings in my life--and the guts, the roofing
systems, the HVAC, et cetera, are built with a 20- to 30-year
life. So they are holding them together with nothing, and the
reason you probably had a trailer was, there was no room in the
school building for them to put that school-based health
center, but they really wanted one.
The other issue you raise is access, and I am a believer
that if you took all of the rural schools in America and you
put them into one school district, you would probably have the
poorest school district in America. So even if there was a
desire to build out space for that school-based health center
in your community, they probably did not have the resources.
The pandemic has given us an interesting opportunity,
technology, to be able to expand the reach and beam into that
trailer other kinds of services that might not be available
readily in the community, is a unique one for this time that we
are in. That is why I say to those of you that sit on the
Communications Subcommittee, make the FCC give us money for
telehealth. It is critical--
Mr. Ruiz. Thank you.
Mr. Boyd [continue]. If we are going to continue to serve
and expand. It is all about what Linda Blount said. It is all
about equity.
Mr. Ruiz. Thank you, Mr. Boyd. You said in your testimony
that there is an intersection between education and health.
What are the health outcomes of these centers, and can you
expound on the correlation between health and education
outcomes?
Mr. Boyd. I love--thank you for that. I love when people
ask questions that other people have answered. Included in your
written testimony is a document from the Community Preventative
Services Task Force of the CDC, and they list access to all of
their findings, but they point out two major ones.
School-based health centers led to improved educational
outcomes, including school performance, grade promotion, and
high school completion. School-based health centers also led to
improved health outcomes, including the delivery of
vaccinations and other recommended preventative services, and
decreases in asthma morbidity, and emergency department and
hospital admission rates.
Mr. Ruiz. That is very interesting, because, oftentimes, in
poor communities, the rate of asthma is high, and that is
particularly true in my desert, rural community with farm
workers.
It seems like a lot of people will refer to cost-
effectiveness of these programs. Are there any data that shows
the cost effectiveness of these school-based health centers?
Mr. Boyd. I may have to get back to you on that one.
Understand that school-based health centers don't cost the
schools any money. They are reimbursed by Medicaid, and
oftentimes, if that child is not being seen by a school-based
health center, they may not be seen by anybody. They are not
necessarily going to the local, federally qualified health
center or to an independent pediatrician.
Mr. Ruiz. Well, I think we can agree that if we have
preventative services, mental health services, that we provide
in the schools efficiently, then it lowers healthcare costs
overall in the long run.
Mr. Boyd. Absolutely. Healthy kids grow up to be healthy
adults and are less of a drain on our financial system.
Absolutely.
Mr. Ruiz. Thank you very much. I yield back.
Mr. Boyd. Thank you.
Ms. Eshoo. The gentleman yields back. Let's circle back to
our wonderful colleague, the gentleman from Florida. Mr.
Bilirakis. Are you unmuted?
Mr. Bilirakis. Can you hear me?
Ms. Eshoo. Now I can. Now we can.
Mr. Bilirakis. OK. Very good. Yes, we--I had to switch to
my iPhone because the computer was not working. I did unmute
several times, and I apologize for that, Madam Chair.
Many of you know that the National Marrow Donor Registry
was established more than 30 years ago by our former colleague
and friend, Bill Young. You mentioned that, Madam Chair. What a
wonderful man. Actually, my district was adjacent to his. He
was passionate about this program, and often said it was his
proudest accomplishment in Congress. That is saying something.
Because of Bill Young and, of course, Chris Smith's
passion, the gentleman from New Jersey, I am familiar with the
work of the National Registry. However, what I didn't realize
until I met with Mr. Lindbergearlier this year is the special
requirement that bone marrow must be hand-carried by volunteer
couriers from donor to patient.
Because of this perishability, there are serious time
constraints. Many times, marrow travels internationally. So
again, the question is for Mr. Lindberg--by the way, an
outstanding job on your presentation today, sir--the question
is, can you discuss the challenges this creates for the
program, especially during this pandemic, please? Thank you.
Mr. Lindberg. Mr. Bilirakis, thank you. Thank you for
cosponsoring H.R. 4764, and for picking up the torch that Mr.
Young lit. We appreciate that so very much.
You know, as you can imagine, the COVID pandemic has
created incredible barriers for us, obstacles that perhaps back
in March I wasn't sure we were going to be able to overcome.
But I am so proud of my colleagues who have done incredible
work to make sure that no patients have missed their
transplant.
I will share briefly, only anecdotally, things like, Mr.
Bilirakis, there was a donor in the country of Colombia. That
donor, because of inability to collect her cells in Colombia,
needed to get to the United States. We needed to get her to the
United States in a matter of days. I will share only briefly
that on a Saturday afternoon at 2:00, I was told by an embassy
official, Brian, it is impossible, this can't--we are not going
to be able to make this work in time. And by 10:00 that
evening, we had the authorization for her to leave the country.
We had the authorizations to open up a closed airport. We
brought in a private aircraft and flew this young woman out of
the country of Colombia. She flew to your home State, Mr.
Bilirakis, and donated her bone marrow just a couple of days
later in time for that patient's life to be saved.
So, yes, this has been an incredibly trying time, but I
have been so thankful for my colleagues, and, frankly, the
support of you, your colleagues, and the Federal Government
agencies in allowing us to make that happen.
Mr. Bilirakis. Well, that shows you, we should never give
up. And I know Harry Glenn has been working on this as well. He
is the former chief of staff for Bill Young.
So let me go on to the next question. As referenced
previously, the nonpartisan Government Accountability Office
stated in their January 2020 report on Priority Review
Vouchers, that all seven drug developers we speak to indicated
that the vouchers were a factor in their decision.
So this question is for Ms. Goodman. The bottom line, as a
patient advocate, do you believe the Rare Pediatric PRV
Program, has been effective at incentivizing the development of
new target therapy and immunotherapy drugs for rare, pediatric
cancers, to extend and save lives and provide hope to pediatric
patients and their families?
If so, can you also describe for this committee what would
occur if this program failed to be reauthorized or reauthorized
permanently? That is the question. And these are all great
bills, I tell you what. This is a wonderful hearing. We hope we
put them in the markup in September. But anyway, if you could
answer, Ms. Goodman, I would appreciate that very much.
Ms. Goodman. Thank you so much, Congressman Bilirakis, and
thank you for your support on this bill throughout the past 10
years. Look, the evidence that the program is successful goes
from the 22 drugs that have been approved since it was passed,
to the over 60 drugs in the development pipeline for seriously
ill kids.
You know, we don't want to lose this opportunity by letting
this program lapse, and by making it permanent, we can hit the
one area that we haven't--where we haven't maximized the
incentive, and that is at the very earliest stage of drug
development when drug developers say, am I going to develop a
drug for seriously ill kids; they need to know the voucher is
going to be out there whenever they get the drug approved so
that they can develop those drugs for kids. Thank you.
Mr. Bilirakis. Thank you.
A question for Mr. Lindberg, for many diseases, including
blood cancers and sickle cell anemia, cellular therapy offers
the best hope for a cure. The question is for Mr. Lindberg. Has
the calculated need for unrelated cellular transplants, in
other words, non-family members increased? If so, what
demographic has seen the largest increase in need? If you can
share that with the committee, I would really appreciate it.
Mr. Lindberg. Thanks, Mr. Bilirakis. I will do that with
pace here. Yes. We know as the number of diseases that are
treatable by bone marrow transplant, purple blood stem cell
transplant, and other cell therapies increases, the need
thereby increases as well.
And we know that our largest obstacles are making sure that
as we become more and more diverse as a country, that we are
able to serve more and more diverse patients through more and
more members of our registry of varying ethnic descents that
are willing to step up and make those donations.
Mr. Bilirakis. Thank you very much.
I think my time is expired, Madam Chair. Is that correct? I
believe so.
Ms. Eshoo. Your time is expired----
Mr. Bilirakis. Thank you very much. Appreciate it.
Ms. Eshoo [continue]. And the gentleman yields back. Glad
you got the----
Mr. Bilirakis. Yes, we got it straightened out.
Ms. Eshoo [continue]. Got it all straightened out there.
Mr. Bilirakis. Thank you.
Ms. Eshoo. OK. It is a pleasure to recognize the very
patient gentlewoman from New Hampshire, Congresswoman Kuster,
for your 5 minutes of questions.
Ms. Kuster. Wonderful to be with you. Thank you, Madam
Chair.
Ms. Eshoo. Sure.
Ms. Kuster. I want--can you hear me? We are good?
Ms. Eshoo. Yes.
Ms. Kuster. It is a very important discussion, and thank
you for hosting this hearing today. While this committee has
been keenly focused on the coronavirus pandemic, there are many
public health programs that we need to continue to support, and
one of those is school-based health centers. So I am a proud
cosponsor of H.R. 2075, introduced by my colleague, Congressman
Sarbanes.
These health centers are critical in providing
comprehensive care, including care to identify at-risk students
before they develop substance dependence and addiction.
In New Hampshire and communities across the country, we are
battling two crises: the opioid epidemic and the COVID-19
epidemic. In fact, preliminary data shows that COVID is
worsening preexisting issues with substance misuse, and the
current pandemic and resulting economic downturn are
exacerbating behavioral health risk factors, such as social
isolation and stress.
Prior to COVID-19, I heard from teachers regarding the
generational effects of the opioid epidemic and how children
cope with adverse childhood events, or ACEs, outside of their
home.
As the COVID-19 pandemic continues, how can school-based
health centers ensure that we continue to address adverse
childhood events closer to the occurrence of the event, which
increases the ability to treat an acute condition before it
becomes chronic?
And I am asking Mr. Boyd, if you could speak to the role of
school-based health centers in nurturing age-appropriate
resilience that is helpful to mitigate self-medication, and to
further substance-use disorders in early adulthood?
Mr. Boyd. Yes, this is another great question. We work very
closely with youth as well. We have a youth advisory committee
that, you know, feeds into our programmatic work, and actually
a part of one of our Federal grants is to work with the youth.
The dilemma right now, as you know, is that the pandemic
has exponentially grown, potentially, the abuse and use of
alcohol and drugs. We have seen it in liquor store sales. We
don't have, you know, good numbers on any increases in sales of
opioids, in particular.
Our need right now is to get the school-based health
centers that we have reopened, and to expand that reach. Part
of expanding that reach is also expanding the services that are
offered, and substance-abuse services are critical in school-
based environments. One of our----
Ms. Kuster. Thank you. My time is limited. I am sorry to
have to cut you off.
Mr. Boyd. No problem.
Ms. Kuster. I really find this very important, and I want
to support you and your work and your colleagues all across the
country, including here.
Another program I want to focus on is the USADA, United
States Anti-Doping Agency, recognized by Congress as the
national anti-doping organization for our Olympic community,
and I am the niece and cousin of Olympic alpine ski racers.
And I wanted to ask you, Mr. Tygart, in your testimony, you
discuss a culture change in terms of the way the United States
was perceived on the international sports stage. Can you talk
about this shift, and why it is so important for youth when
they look up to Olympic athletes, to know that they are drug-
free?
Mr. Tygart. Yes, ma'am, and thank you for the question.
Well, it is absolutely critical to have true heroes today, and
we may be more desperate today in the world of sport than we
ever have been, to have people that our young kids can look up
to and try to emulate and attempt to become.
And so when Congress set up an independent organization to
ensure that the fox was no longer guarding the henhouse, what
it did was gave confidence to athletes that this independent
organization is not there just to promote them, but is there to
ensure that they follow the rules.
And we know whether a young person becomes an Olympic
athlete and becomes one of those heroes, or goes into any
other, you know, industry or career in their life outside of
sport, the lessons they learn on the playing field are
important life lessons that they will take with them.
And respecting the rule of law, respecting, yes, we want to
win, but we want to win the right way, is absolutely essential.
And our athletes today, you know, 20 years ago, since we were
established, have embodied that notion and are truly the heroes
that we want them to be. Not to say that some won't attempt to
cut the rules, but we are here to have a fair program that
ensures that their decision to do it right is enforced.
Ms. Kuster. Well, my time is up, but I can certainly say as
a parent, I appreciate the role that you play, and thank you
again.
Thank you, Madam Chair, and I yield back.
Ms. Eshoo. The gentlewoman yields back. It is a pleasure to
recognize the only pharmacist in the Congress, my colleague,
Mr. Carter, from Georgia, for 5 minutes of questions.
Mr. Carter. Thank you, Madam Chair, and thank everyone for
being on this call. This is certainly important.
I want to start with you, Ms. Goodman. There are those who
have been somewhat critical, if you will, of the Rare Pediatric
Disease Priority Review Voucher, and have said that it hasn't
been successful in achieving what it was intended to. You cited
some figures, I believe, when Congressman Mullin was asking you
some questions. Could you repeat those and the number of drugs
that have come out as a result of the PRV, and how you would
respond to those claims, those critics who say that it hasn't
operated like it should have?
Ms. Goodman. Sure. You know, there have been 22 new drugs
approved by the FDA--that is a very high bar, approval--since
the program was enacted in 2012. I think that is pretty good
proof that the program has been successful.
Mr. Carter. How was it before the program? Can you compare
it before and after?
Ms. Goodman. Sure. Well, in the case of pediatric cancer,
which is not all rare diseases of course, there had only been
two drugs approved expressly for kids with cancer in the 25
years leading up to the Creating Hope Act enactment. Just
couldn't get funding.
Mr. Carter. OK. Good. Obviously, the program has helped.
Obviously, we have seen results.
Ms. Goodman. The program has helped with small biotechs,
with academics who want to get their ideas out into industry.
It has just really been a very successful program.
Mr. Carter. Well, staying on that, on the program itself
and the vouchers that get the priority review by the FDA, and
allowing them to be completed in six months, do you have any
evidence that any of the drugs approved with a voucher under
this program have had to be pulled from the market because it
was unsafe? Have you seen any instances of that at all?
Ms. Goodman. So that is a terrific question, Congressman
Carter. I am going to have to get back to you on specifics, but
I will say that, you know, because of the voucher program,
because of the user fees charged for this program and for
PDUFA, FDA has been able to almost double the number of
employees working at the FDA on reviewing drug approvals from
2008 until now. So we really have so many more people, so much
more FDA technology, and better management practices reviewing
these drugs. I really trust the FDA not to be--do a quick and
dirty job. I really trust them to only approve drugs that are
safe.
Mr. Carter. Well, you know, I am one who believes. And
listen, as was mentioned earlier, as a practicing pharmacist
for over 30 years, I have seen this, and I have dispensed some
of these medications, and I can tell you that they are needed,
and we need to improve the process by which they are approved.
And certainly, you know, we still need to be careful, there is
no question about that, but I am still one who believes that,
you know, no matter how long the process is, you still run that
risk. There will be some that--and I have seen it over my years
of practice--of drugs that have gone through a thorough review,
that regardless of how good a review it was, we had to pull
them from the market at some point. And that is going to
happen, but to keep them from getting on the market, I think,
is far worse than what we have experienced.
Dr. Kesselheim, are you still with us? Yes.
Mr. Kesselheim. I am. And happy to answer questions.
Mr. Carter. Yes. Have you seen any instances where any
drugs have had to be pulled back as a result of this
accelerated approval program?
Mr. Kesselheim. Yes. There was a study that was published
in The New England Journal of Medicine about a decade ago that
said that drugs that are approved within a short period of
time, just before the FDA approval deadline, were more likely
to be pulled from the market, were more likely to have boxed
warnings added to it or other safety-related information added
to it.
And I think that study shows that if you impose arbitrarily
fast deadlines on reviews, that that can increase the risk of
those kinds of things happening. Again, I agree it doesn't
happen very often, but we want to try to minimize it happening
as much as possible.
The FDA doesn't approve drugs--when the FDA approves a
drug, it doesn't mean the drug is safe. It just means that the
drugs benefits outweigh its risks, and we have a lot more to
learn about those risks once the drug hits the market.
Mr. Carter. Now, I am not going to dispute what you just
said, but, again, as a practicing pharmacist for over 30 years,
I can tell you, I have witnessed where no matter how long the
review is, you are still going to have those instances, and I
do think that the risk does outweigh--or the benefits do
outweigh the risks in this particular case. That is why I do
hope that the program is permanently renewed, and that we can
move on from there.
And Madam Chair, I see I am out of time, and I will yield
back. Thank you both. Thank you all.
Ms. Eshoo. The gentleman yields back. Thank you for your
good questions. A pleasure to recognize the gentlewoman from
Delaware, Ms. Blunt Rochester, and thank you for your patience.
Ms. Blunt Rochester. Thank you. Thank you, Chairwoman, and
thank you so much to all of our witnesses today.
As a cosponsor of H.R. 2075, the School-Based Health
Centers Reauthorization Act, H.R. 4078, the EARLY Act
Reauthorization, and H.R. 4439, the Creating Hope
Reauthorization Act, I am glad that the subcommittee is taking
the opportunity to discuss legislation that would reauthorize a
number of critically important public health programs.
Delaware leads the Nation in rates of triple-negative
breast cancer, an aggressive form of cancer that
disproportionately affects young African American women. In
fact, a 2019 study found that more than 21 percent of Black
women were diagnosed with triple-negative cancer, compared to
11 percent for all other types of breast cancer, and women
under the age of 40 had twice the odds of a triple-negative
breast cancer diagnosis than women aged 50 to 64 years.
Ms. Blount, what are the unique challenges that young,
Black women face in finding out about this type of cancer and
what their risks might be?
Ms. Blount. Thank you, Congresswoman. You actually
highlight a very serious concern that we have in breast cancer
as we think about what we can do to reduce our risk. The number
one talent is information, is understanding their own risk,
understanding their family history, understanding the need to
go in for a screening mammography, and being in a system where
their providers are likely to ask certain questions, and then
make those recommendations. So this is really critical.
The other really important point is, we don't quite
understand why Black women have twice the rates in some
States--as you mentioned in Delaware and others--nationally
about 30 percent more triple-negative breast cancer. We need to
do a large study to understand that, and I understand a large
randomized control trial will take years. We need to do that,
but in the meantime, what we need to make sure is that Black
women get screened early. Because we know even if it is triple-
negative breast cancer, if we catch it in its earliest stages,
Black women can have wonderful outcomes and die of old age, and
not from breast cancer. So the important thing is to make sure
that they know to get in and get screened early.
Ms. Blunt Rochester. Thank you so much. And in your
testimony, you also talked about access to the latest digital
breast screening technologies. How does the EARLY Act improve
access to better diagnostic care, and what more can Congress do
to reduce disparities in access, in addition to the incredible
work that you are doing?
Ms. Blount. Well, the EARLY Act makes sure that women
understand their risk, understand the need for screening
mammography gets to providers so providers understand that, but
the important thing is, Black women tend to have dense breast
tissue, and so, if they have a lesion, 2D or standard film
mammography is less likely to pick it up as compared with 3D or
tomosynthesis.
So as a part of that education program for providers and
for women, to help them understand that if they have a choice,
try to find a facility that will provide 3D mammography is
going to be much better for them, more likely to pick up their
cancer, more likely to pick it up early when it can be treated.
Ms. Blunt Rochester. Thank you. Thank you so much for your
work.
Ms. Blount. Thank you.
Ms. Blunt Rochester. I want to get to my questions to Dr.
Boyd. I had the opportunity to serve over almost 30 years ago
as a policy adviser to our current Senator, Tom Carper, who, at
the time, was our governor, and he had a vision for making sure
that every school in our State had a wellness center. We
started off with all of the high schools, and now today, I can
report that every public non-charter high school operates a
wellness center in the State, and is looking to expand to
elementary and middle schools.
Dr. Boyd, how do early intervention services provided by
elementary school wellness centers help improve health outcomes
throughout a child's life and save healthcare dollars?
Mr. Boyd. Great question, and Delaware has an extraordinary
program. I am a former Delaware resident, so I am very
appreciative of it.
First, it gets the students used to understanding it is
just health. Tearing down the barriers so that they are
comfortable and confident in interacting with healthcare
professionals, and learning to look after their health and
speak up when there are issues addressing those.
But two, that CDC guide I pointed to, we know that it has
significant impact on the academic performance of the students.
Time on task. It keeps them in front of the classroom longer
than not having to leave school, and it solves the problem for
the parent, often women, often low-income, that have to leave
an hourly job to go take that child to a doctor. Nine times out
of ten it doesn't happen.
Ms. Blunt Rochester. Thank you so much. Thank you so much,
Dr. Boyd. And also the mental health aspect of it is so
important, especially now more than ever. Thank you.
I yield back, Madam Chairwoman.
Ms. Eshoo. The gentlewoman yields back. It is a pleasure to
recognize the gentleman from Illinois, Mr. Rush, for his 5
minutes of questions. You need to unmute.
Mr. Rush. And thank you for that gentle reminder to unmute.
Ms. Goler Blount, I want to thank you for the work that your
organization does for Black women and girls all around this
Nation. As a cancer survivor myself, it is heartening to hear
the progress that is being made to prevent unnecessary deaths
among Black women from cancer.
As I listen to your testimony, the parallels to heart
disease were overwhelming. As I am sure you know, more than 60
percent of Black women are not aware that heart disease is
their number one health threat.
I recently wrote to the National Institutes of Health,
asking that they create a Black women's healthy heart program,
which would focus directly on increasing awareness among Black
women about the risk of and the prevention to the variables for
heart disease.
What best practices, Ms. Goler Blount, from your work on
raising breast cancer awareness could be applied to heart
disease awareness, specifically for Black women?
Ms. Blount. Well, thank you, Congressman Rush. You raise a
very important point. Heart disease is the number one killer,
and a lot of us don't know that. And heart disease is an issue
of both lifestyle and circumstances. So the things that we
know--for example, my organization, the Black Women's Health
Imperative, has a program called Change Your Lifestyle, Change
Your Life, which was originally conceived as diabetes
prevention, but the risk factors for diabetes and heart disease
and hypertension are the same. And so, we start with managing
stress and how you eat, why you eat, what emotionally you bring
to eating, and active living, moving around.
We know if women can manage their stress--particularly
Black women--manage their stress, and have access to fresh
fruits and vegetables, and can get physical activity, this can
significantly reduce their rates for all chronic diseases, and
we also know that about 70 percent of chronic disease mortality
is completely avoidable.
So what we need are programs like this program implemented
across the country--and we are in 12 States right now--and to
partner with the CDC on its chronic disease awareness program,
and to make sure women understand what they can do, and that,
in fact, they can do things.
And to your point, if women can manage their stress and
their diet and their physical activity, that also lowers their
risk for breast cancer.
Mr. Rush. Right. Mr. Boyd, and also Ms. Goler Blount, this
pandemic, the coronavirus, in my opinion, gives us an
extraordinary opportunity to completely reimagine, and
revitalize, and re-create a permanent health system. Do you
agree, and what are some of the things that this Congress
should be doing in order to really just create a whole new
perspective, reimagining what a public health system could mean
to all Americans, particularly the least of these?
Ms. Blount. Yes. I agree with you. Our public health
response was not, in any way, sufficient. What we need to do is
listen to scientists. What we could have done to prevent where
we are now with COVID-19 is to reflect back 40 years with HIV.
We needed to do testing. We needed to do contact tracing and
follow-up.
We can still do that now, but what we have got to do is
make sure that people have access to resources, that we are
reaching out to people with the kinds of information and
resources they need----
Mr. Rush. Thank you.
Ms. Blount [continue]. And make sure our providers and
researchers are involved in the conversation.
Mr. Rush. Thank you.
Mr. Boyd, can you chime in with the few seconds that I have
remaining?
Mr. Boyd. Yes, Congressman. Access, access, access. People
of low income don't have access to adequate care. It is that
simple.
Mr. Rush. Thank you very much.
Madam Chair, I yield back the balance of my time.
Ms. Eshoo. The gentleman yields back and thank him for his
excellent questions.
It is a pleasure to recognize the gentlewoman from
California, very patient, Ms. Barragan, for your 5 minutes of
questions.
Ms. Barragan. Thank you, Madam Chairwoman, and thank you to
all our panelists today. I appreciate the conversation and want
to go back to continue to highlight the issue of breast cancer
disparities that we continue to see.
As with many other health conditions plaguing our
communities, there are significant and unacceptable health
disparities when it comes to breast cancer. My own sister got
breast cancer at an early age, so it continues to be a concern,
and in our communities, it is something that I don't hear of
talked about a lot.
Ms. Blount, as you have mentioned in your testimony, you
know, African American women are almost 40 percent more likely
to die from breast cancer compared to non-Hispanic, White
women. In part, I think that is because they are screened less
frequently, they are more likely to have advanced disease when
a diagnosis is made, and have less access to medical care.
You just mentioned something that I don't think a lot of
people hear about, the 3D mammography availability. Is that
something that a community clinic would have available if,
let's say, I told my constituents to ask about it, or is that
something they would have to have more advanced access to a
different level of healthcare?
Ms. Blount. Yes. Thank you for that question,
Congresswoman. Right now, most 3D mammography is available in
upper-income areas, I will just be honest with you, in suburban
areas. And we need to make sure that all women have access to
3D mammography. That is critically--particularly Black women,
Latinas, particularly women who are going to have dense breast
tissue. We have to make it more available. As Mr. Boyd said,
access is critical.
I do want to highlight one point, that screening rates,
actually between Black and White women, are about the same,
both annually and biannually. The problem is, Black women tend
to get their cancers detected much later. So while about 60
percent of us are screening every year, our cancers don't get
diagnosed until much later, because we tend to wait later. So
if we had access and we were screening at the same rates, our
outcomes would be much better.
Ms. Barragan. Well, thanks for pointing that out because I
think what you just said about what is available in the upper-
income areas versus the lower-income areas goes to the
disparities that we face, and really the different access to
care that people have in this country.
Can you elaborate on how the EARLY Act may help reduce the
intolerable disparities that we are seeing in this area?
Ms. Blount. I can. We have seen over the last ten years,
particularly among younger women, a slight uptick in screening
rates. I mean, we have gone from about--annual--from about 58
percent to 62 percent, so I am going to declare success. But
what the Act will do is make sure women understand the
importance of screening mammography, and most importantly, that
they don't need to be afraid of it.
You know, women aren't being recommended, but also women
are afraid to be screened. They are afraid, what happens if I
have a breast cancer, and the fact is, you know, screening
doesn't impact whether or not you are going to develop breast
cancer, but it can impact how well you will do if a breast
cancer is detected.
So the EARLY Act is critical to help women understand that
they need to get in early, starting at age 40, screen every
year, and that if it so happens a breast cancer gets detected,
you have had this experience, it can be treated in its earlier
stages when outcomes are excellent.
Ms. Barragan. Great. Thank you so much.
Ms. Blount. Thank you.
Ms. Barragan. The National Cancer Institute has also found
that social economic status factors, like access to education,
access to health insurance, living conditions, including
exposure to environmental toxins, are associated with an
individual's, or a group's risk of developing and surviving
cancer.
I have been advocating for investments in these social
determinants of health, including introducing a bill called the
Improving Social Determinants of Health Act which would give
the CDC resources to invest in communities to tackle these
issues. This would help lower health--would actually help
improve health outcomes, especially for those who are low
income and living in underserved communities.
Can you discuss how investing in social determinants of
health can help reduce the rate of breast cancer in underserved
communities and also lead to better outcomes?
Ms. Blount. Yes, I can. Thank you. And that is an important
point. Social determinants covers a variety of things from
community, to neighborhoods, to food, to transportation,
housing. One of the things we can do is by focusing on social
determinants is actually lower our risk. And another issue is
to understand how environmental factors impact our--whether or
not we are going to develop disease, and, critically, and I
have to come back to this, access. Social determinants of
health are the leading impediment to access to healthcare of
any kind.
Ms. Barragan Great. Thank you.
Madam Chairwoman, I yield back.
Ms. Blount. Thank you.
Ms. Eshoo. The gentlewoman yields back.
It is a pleasure to recognize the gentleman from Ohio, Mr.
Johnson, who has, I believe, waived on to our subcommittee
today, and one of the authors of the bills that we are taking
up.
Mr. Johnson. Well, thank you, Madam Chairwoman and Mr.
Ranking Member for the opportunity to waive on to the Health
Subcommittee today in support of H.R. 5373, the United States
Anti-Doping Agency Reauthorization Act.
Mr. Tygart, it is good to see you again as well. Thanks for
your decades of tireless work to make USADA the gold standard
across the world for its anti-doping efforts. You know, this
bipartisan legislation, which I am proud to sponsor, along with
my colleague, Congresswoman DeGette, will reauthorize funding
for USADA, improve education to youth sports, and for both
programs and coaches, and includes provisions to direct Federal
law enforcement to coordinate with USADA to combat the
trafficking and illicit use of performance enhancing drugs.
With the summer Olympics in Tokyo next year, and the return
of the Olympics to the United States in 2028 in Los Angeles, it
is critical that USADA has the resources it needs to continue
its work.
So, Mr. Tygart, as we heard in your testimony, it is
essential that America takes the global lead in anti-doping in
clean sports. In the past, this has not always been the case.
Can you explain the benefit to the standing of the United
States abroad when we can demonstrate that our athletes compete
and win the right way?
Mr. Tygart. Thank you, Congresswoman Johnson. I really
appreciate the question. You are absolutely right for the rule
of law. We are a democracy that functions when all of us who
agree to a set of rules that are designed to provide certain
benefits for those that follow the rules and ensure that
equality can be provided in the athletic context for our
athletes who are competing. And so, when the United States
sends athletes around the world and they demonstrate that, I
think there is no better example of the values that this
country stands for that our athletes abide by the rule of
sport. They compete healthy. They compete clean. And you can
actually look at them as the role models that they are, and
that they represent this great country and the values that we
espouse around the world like no other instrument we almost
have. And that is kind----
Mr. Johnson. It is kind of like they are ambassadors,
right?
Mr. Tygart. It is absolutely right. And not just to win,
but importantly win the right way. And that is the difference
between our athletes today and what the world saw back in the
late 1990s, when the speculation--and there was some evidence
to suggest it when our system was so poor that our athletes
weren't winning the right way. Today, they know they are being
held to the highest standards, and that they can upgrade
confidence when they go and win medals at the games and
represent this country that they are doing it to the right way.
Mr. Johnson. Sure. Well, what does it mean for global
sports more broadly to have an organization that does things
the right way, like USADA does, and doesn't operate under
malign influences like we have seen with countries like Russia
and others?
Mr. Tygart. It is critically important. That independent
model has become a beacon to many around the world, and I think
is demonstrated, as my written testimony that I submitted
indicates, has shown. I mean, two whistleblowers inside of
Russia, all benefits of the independent model, and specifically
USADA's work, and it motivated them and said there are people
outside of Russia, despite what they were being told, that do
stand for these values. That the rules matter. That integrity
in sport matters. And it motivated them and inspired them to
come forward and ultimately expose a state that was abusing its
own athletes, as we saw in the state-sponsored scandal that has
been exposed now dealing with Russia.
So, it is a beacon to many around the world that winning
the right way is the only way to play.
Mr. Johnson. OK. Well, I mentioned youth sports. You know,
I have got a teenage son, Nathan, who is a competitive high
school swimmer, and he has his dreams set on swimming in
college and maybe even beyond. He is really very good. I am a
little biased, I am his dad, but I think he is really, really
good. His time sure reflects that. But he looks up to our
incredible Olympians, and is looking forward to watching them
compete in Tokyo next year.
Can you explain how USADA's work is essential for the next
generation of top athletes, why taking early action on
educating them on the importance of competing fairly and
ensuring clean drug-free sports in the future, why that is so
important?
Mr. Tygart. Well, it is critically important, and you
should have confidence that your--he does have a number of role
models that he can watch and be proud of and attempt to
emulate.
And can you imagine a young athlete, you know, first grade,
second grade as they are growing up one day dreaming of making
it to the elite level, only then to realize they have to inject
themselves with dangerous performance enhancing drugs? You
know, nobody goes into sport for that reason. And that is why
to protect the value of sport, whether he ultimately becomes an
Olympic level swimmer, or just goes into another profession,
doing it the right way is the only way. And our program is to
educate, but also provide true role models for him--are
critically important to his future success.
Ms. Eshoo. The gentleman's time has expired.
Mr. Johnson. Thank you, Madam Chairman, I yield.
Ms. Eshoo. Thank you. And we share your pride in your son,
Mr. Johnson.
Mr. Johnson. Thank you.
Ms. Eshoo. It is a pleasure to recognize the gentleman from
Arizona, Mr. O'Halleran, for his 5 minutes of questions. And I
believe he is the last member, unless someone walks out and I
see them on the screen.
So, Mr. O'Halleran, you are recognized.
Mr. O'Halleran. Thank you, Madam Chair, and thank you also
ranking member, for allowing me to waive on. This issue speaks
to a really important issue in rural America, not only in
Arizona, but throughout America. And as school-based health
centers provide a wide variety of potential health services to
students and their families, many of them lack health
insurance, and, otherwise, would have no ability to see a
national provider. School-based health centers can treat
chronic conditions like asthma and provide services like
immunization and physical examination. School-based health
centers play a critical role in providing care to children and
families throughout Arizona.
In fact, according to the Arizona School-Based Health
Alliance, 45 percent of school-based health centers are located
in rural areas. Rural America already is suffering from the
lack of access to high quality and affordable healthcare.
Additionally, 82 percent of the children who use these services
are uninsured. That is why School-Based Health Centers
Reauthorization Act of 2019 is so critical. I fear that if
Congress fails to act, many children will lose access to the
important services provided by these sectors.
The COVID-19 pandemic has exploded, as those of us in rural
America already know. Many of us lack affordable access to
high-quality healthcare, in particular, and concern about the
mental healthcare of students and families whose lives have
been upended and whose social structure has completely changed
in light of COVID-19.
Dr. Boyd, thank you for your testimony here today and all
the work you and your members do. In your testimony, you talked
quite a bit about the importance of reimbursing mental health
of our students and remembering. What changes have you and your
members already seen in the mental health of our students given
the COVID-19 situation? Also, what are the long-term health and
educational paths of these sorts of mental health issues? And
whether the students are going to go back into schools or stay
home and do it virtually, this is having a profound impact on
students across our country.
Dr. Boyd?
Mr. Boyd. This is a great question, Congressman. We are in
process of putting some polls in field, but it is very, very
difficult. As I said earlier, there are estimates that could be
as many as a third of Title 1 students people haven't heard
from. And so, knowing exactly what is happening in those
households, we don't know. We are hopeful that as schools come
back online, you know, after--after this summer break, that
they will be able to give us more information. Our fear is that
enough--we may lose a number of school-based health centers
because their sponsoring agency does not have the resources for
them to reopen.
The impact we believe, along with our colleagues and the
school psychologists and counseling areas and the nurses are
going to be devastating. And students are going to be typecast
when they come back and they show behaviors that to a student
going through trauma is actually normal for a person going
through trauma, but because they are acting out, may then get
typecast and put into the system. That is a problem that we are
really, really concerned about. And I don't think anybody at
this point has good solutions to it. But we just, frankly,
don't know what is happening with a lot of kids at home. And
that is where the telehealth opportunity is great, and support
from the FCC would be great to provide those kinds of platforms
and resources to get out there and find out and communicate
with the kids.
Mr. O'Halleran. And, also, do school-based health centers
look to serve as COVID-19 testing centers?
Mr. Boyd. They would be if they had access to the test,
yes, sir.
Mr. O'Halleran. Madam Chair, I yield.
Ms. Eshoo. Mr. O'Halleran yields back.
I don't see any other members, so I think that we have
heard from subcommittee members from both sides of the aisle,
as well as our colleagues that waived on.
I have documents to submit to the record, and I see my
friend, Mr. Griffith, standing by. I have four pages. So I
would like to ask for a unanimous consent request that all of
the documents that are contained in this stack be entered into
the record.
Mr. Griffith. As long as it has been provided to our
Committee staff as well, no objection.
[The information appears at the conclusion of the hearing.]
Ms. Eshoo. It all has. I haven't added anything to what was
presented to the minority staff, and I thank the gentleman.
Let me close on this note. First of all, our deepest thanks
to each one of you. We have been together for 3 hours and 15
minutes, not that you were counting the seconds or the minutes.
But, boy, was it time well spent. These are such important
programs, and you have taken a very deep dive into why they are
so important, how they work, who has served, and really the
original motivations for these programs. In the case of Ms.
Goodman, it was the tragedy of losing her son.
So I can't thank you enough for enlightening us. And these
reauthorizations are very important. And for the--if anyone is
listening in in the country to this virtual hearing,
reauthorization means that we are going--we are reviewing the
program, we may add, we may subtract, but we are renewing the
contract on it, so to speak, so that the program can keep
running.
So from our children to the diseases that they--the rare
diseases that they have, to saving lives through the--the
donors and cord blood, to doping, and making sure that our
sports are absolutely clean, to our friend from Harvard who has
offered, I think, in a very clear way what his position is on
some of the legislation--I hope I haven't missed anyone--but we
appreciate you. We appreciate you and the testimony that you
have given. Continue to work with us. We always need the expert
advice. We are, bettered as a result of it.
And with that, I will now adjourn the Health Subcommittee.
And thank you all for your participation. The committee is
adjourned.
[Whereupon, at 1:16 p.m., the subcommittee was adjourned.]
[GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
[all]