[Congressional Record Volume 170, Number 148 (Monday, September 23, 2024)]
[House]
[Pages H5582-H5587]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]
GIVE KIDS A CHANCE ACT OF 2024
Mr. BUCSHON. Mr. Speaker, I move to suspend the rules and pass the
bill (H.R. 3433) to amend the Federal Food, Drug, and Cosmetic Act with
respect to molecularly targeted pediatric cancer investigations, and
for other purposes, as amended.
The Clerk read the title of the bill.
The text of the bill is as follows:
H.R. 3433
Be it enacted by the Senate and House of Representatives of
the United States of America in Congress assembled,
SECTION 1. SHORT TITLE; TABLE OF CONTENTS.
(a) Short Title.--This Act may be cited as the ``Give Kids
a Chance Act of 2024''.
(b) Table of Contents.--The table of contents for this Act
is as follows:
Sec. 1. Short title; table of contents.
TITLE I--GIVE KIDS A CHANCE
Sec. 101. Research into pediatric uses of drugs; additional authorities
of Food and Drug Administration regarding molecularly
targeted cancer drugs.
Sec. 102. Ensuring completion of pediatric study requirements.
Sec. 103. FDA report on PREA enforcement.
Sec. 104. Extension of authority to issue priority review vouchers to
encourage treatments for rare pediatric diseases.
Sec. 105. Limitations on exclusive approval or licensure of orphan
drugs.
Sec. 106. Program for pediatric studies of drugs.
TITLE II--UNITED STATES-ABRAHAM ACCORDS COOPERATION AND SECURITY
Sec. 201. Establishment of Abraham Accords Office within Food and Drug
Administration.
TITLE III--ORGAN PROCUREMENT AND TRANSPLANTATION NETWORK
Sec. 301. Registration fees.
TITLE I--GIVE KIDS A CHANCE
SEC. 101. RESEARCH INTO PEDIATRIC USES OF DRUGS; ADDITIONAL
AUTHORITIES OF FOOD AND DRUG ADMINISTRATION
REGARDING MOLECULARLY TARGETED CANCER DRUGS.
(a) In General.--
(1) Additional active ingredient for application drug;
limitation regarding novel-combination application drug.--
Section 505B(a)(3) of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 355c(a)(3)) is amended--
(A) by redesignating subparagraphs (B) and (C) as
subparagraphs (C) and (D), respectively; and
(B) by striking subparagraph (A) and inserting the
following:
[[Page H5583]]
``(A) In general.--For purposes of paragraph (1)(B), the
investigation described in this paragraph is (as determined
by the Secretary) a molecularly targeted pediatric cancer
investigation of--
``(i) the drug or biological product for which the
application referred to in such paragraph is submitted; or
``(ii) such drug or biological product in combination
with--
``(I) an active ingredient of a drug or biological
product--
``(aa) for which an approved application under section
505(j) under this Act or under section 351(k) of the Public
Health Service Act is in effect; and
``(bb) that is determined by the Secretary to be the
standard of care for treating a pediatric cancer; or
``(II) an active ingredient of a drug or biological
product--
``(aa) for which an approved application under section
505(b) of this Act or section 351(a) of the Public Health
Service Act to treat an adult cancer is in effect and is held
by the same person submitting the application under paragraph
(1)(B); and
``(bb) that is directed at a molecular target that the
Secretary determines to be substantially relevant to the
growth or progression of a pediatric cancer.
``(B) Additional requirements.--
``(i) Design of investigation.--A molecularly targeted
pediatric cancer investigation referred to in subparagraph
(A) shall be designed to yield clinically meaningful
pediatric study data that is gathered using appropriate
formulations for each age group for which the study is
required, regarding dosing, safety, and preliminary efficacy
to inform potential pediatric labeling.
``(ii) Limitation.--An investigation described in
subparagraph (A)(ii) may be required only if the drug or
biological product for which the application referred to in
paragraph (1)(B) contains either--
``(I) a single new active ingredient; or
``(II) more than one active ingredient, if an application
for the combination of active ingredients has not previously
been approved but each active ingredient has been previously
approved to treat an adult cancer.
``(iii) Results of already-completed preclinical studies of
application drug.--The Secretary may require that reports on
an investigation required pursuant to paragraph (1)(B)
include the results of all preclinical studies on which the
decision to conduct such investigation was based.
``(iv) Rule of construction regarding inactive
ingredients.--With respect to a combination of active
ingredients referred to in subparagraph (A)(ii), such
subparagraph shall not be construed as addressing the use of
inactive ingredients with such combination.''.
(2) Determination of applicable requirements.--Section
505B(e)(1) of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 355c(e)(1)) is amended by adding at the end the
following: ``The Secretary shall determine whether
subparagraph (A) or (B) of subsection (a)(1) shall apply with
respect to an application before the date on which the
applicant is required to submit the initial pediatric study
plan under paragraph (2)(A).''.
(3) Clarifying applicability.--Section 505B(a)(1) of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)(1))
is amended by adding at the end the following:
``(C) Rule of construction.--No application that is subject
to the requirements of subparagraph (B) shall be subject to
the requirements of subparagraph (A), and no application (or
supplement to an application) that is subject to the
requirements of subparagraph (A) shall be subject to the
requirements of subparagraph (B).''.
(4) Conforming amendments.--Section 505B(a) of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)) is amended--
(A) in paragraph (3)(C), as redesignated by paragraph
(1)(A) of this subsection, by striking ``investigations
described in this paragraph'' and inserting ``investigations
referred to in subparagraph (A)''; and
(B) in paragraph (3)(D), as redesignated by paragraph
(1)(A) of this subsection, by striking ``the assessments
under paragraph (2)(B)'' and inserting ``the assessments
required under paragraph (1)(A)''.
(b) Guidance.--The Secretary of Health and Human Services,
acting through the Commissioner of Food and Drugs, shall--
(1) not later than 12 months after the date of enactment of
this Act, issue draft guidance on the implementation of the
amendments made by subsection (a); and
(2) not later than 12 months after closing the comment
period on such draft guidance, finalize such guidance.
(c) Applicability.--The amendments made by this section
apply with respect to any application under section 505(b) of
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(b))
and any application under section 351(a) of the Public Health
Service Act (42 U.S.C. 262(a)), that is submitted on or after
the date that is 3 years after the date of enactment of this
Act.
(d) Reports to Congress.--
(1) Secretary of health and human services.--Not later than
2 years after the date of enactment of this Act, the
Secretary of Health and Human Services shall submit to the
Committee on Energy and Commerce of the House of
Representatives and the Committee on Health, Education,
Labor, and Pensions of the Senate a report on the Secretary's
efforts, in coordination with industry, to ensure
implementation of the amendments made by subsection (a).
(2) GAO study and report.--
(A) Study.--Not later than 3 years after the date of
enactment of this Act, the Comptroller General of the United
States shall conduct a study of the effectiveness of
requiring assessments and investigations described in section
505B of the Federal Food, Drug, and Cosmetic Act (21
U.S.C.355c), as amended by subsection (a), in the development
of drugs and biological products for pediatric cancer
indications.
(B) Findings.--Not later than 7 years after the date of
enactment of this Act, the Comptroller General shall submit
to the Committee on Energy and Commerce of the House of
Representatives and the Committee on Health, Education,
Labor, and Pensions of the Senate a report containing the
findings of the study conducted under subparagraph (A).
SEC. 102. ENSURING COMPLETION OF PEDIATRIC STUDY
REQUIREMENTS.
(a) Equal Accountability for Pediatric Study
Requirements.--Section 505B(d) of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 355c(d)) is amended--
(1) in paragraph (1), by striking ``Beginning 270'' and
inserting ``Noncompliance letter.--Beginning 270'';
(2) in paragraph (2)--
(A) by striking ``The drug or'' and inserting ``Effect of
noncompliance.--The drug or''; and
(B) by striking ``(except that the drug or biological
product shall not be subject to action under section 303)''
and inserting ``(except that the drug or biological product
shall be subject to action under section 303 only if such
person demonstrated a lack of due diligence in satisfying the
applicable requirement)''; and
(3) by adding at the end the following:
``(3) Limitation.--The Secretary shall not issue
enforcement actions under section 303 for failures under this
subsection in the case of a drug or biological product that
is no longer marketed.''.
(b) Due Diligence.--Section 505B(d) of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 355c(d)), as amended by
subsection (a), is further amended by adding at the end the
following:
``(4) Due diligence.--Before the Secretary may conclude
that a person failed to submit or otherwise meet a
requirement as described in the matter preceding paragraph
(1), the Secretary shall--
``(A) issue a noncompliance letter pursuant to paragraph
(1);
``(B) provide such person with a 45-day period beginning on
the date of receipt of such noncompliance letter to respond
in writing as set forth in such paragraph; and
``(C) after reviewing such written response, determine
whether the person demonstrated a lack of due diligence in
satisfying such requirement.''.
(c) Conforming Amendments.--Section 303(f)(4)(A) of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 333(f)(4)(A))
is amended by striking ``or 505-1'' and inserting ``505-1, or
505B''.
(d) Transition Rule.--The Secretary of Health and Human
Services may take enforcement action under section 303 of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 333) only for
failures described in section 505B(d) of such Act (21 U.S.C.
355c(d)) that occur on or after the date that is 180 days
after the date of enactment of this Act.
SEC. 103. FDA REPORT ON PREA ENFORCEMENT.
Section 508(b) of the Food and Drug Administration Safety
and Innovation Act (21 U.S.C. 355c-1(b)) is amended--
(1) in paragraph (11), by striking the semicolon at the end
and inserting ``, including an evaluation of compliance with
deadlines provided for in deferrals and deferral
extensions;'';
(2) in paragraph (15), by striking ``and'' at the end;
(3) in paragraph (16), by striking the period at the end
and inserting ``; and''; and
(4) by adding at the end the following:
``(17) a listing of penalties, settlements, or payments
under section 303 of the Federal Food, Drug, and Cosmetic Act
(21 U.S.C. 353) for failure to comply with requirements under
such section 505B, including, for each penalty, settlement,
or payment, the name of the drug, the sponsor thereof, and
the amount of the penalty, settlement, or payment imposed;
and''.
SEC. 104. EXTENSION OF AUTHORITY TO ISSUE PRIORITY REVIEW
VOUCHERS TO ENCOURAGE TREATMENTS FOR RARE
PEDIATRIC DISEASES.
(a) Extension.--Paragraph (5) of section 529(b) of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff(b)) is
amended by striking ``September 30, 2024, unless'' and all
that follows through the period at the end and inserting
``September 30, 2029.''.
(b) GAO Report on Effectiveness of Rare Pediatric Disease
Priority Voucher Awards in Incentivizing Rare Pediatric
Disease Drug Development.--
(1) GAO study.--
(A) Study.--The Comptroller General of the United States
shall conduct a study of the effectiveness of awarding rare
pediatric disease priority vouchers under section 529 of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff), as
amended by subsection (a), in the development of human drug
products that treat or prevent rare pediatric diseases (as
defined in such section 529).
(B) Contents of study.--In conducting the study under
subparagraph (A), the Comptroller General shall examine the
following:
(i) The indications for each drug or biological product
that--
[[Page H5584]]
(I) is the subject of a rare pediatric disease product
application (as defined in section 529 of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 360ff)) for which a
priority review voucher was awarded; and
(II) was approved under section 505 of the Federal Food,
Drug, and Cosmetic Act (42 U.S.C. 355) or licensed under
section 351 of the Public Health Service Act (42 U.S.C. 262).
(ii) Whether, and to what extent, an unmet need related to
the treatment or prevention of a rare pediatric disease was
met through the approval or licensure of such a drug or
biological product.
(iii) The size of the company to which a priority review
voucher was awarded under section 529 of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 360ff) for such a drug or
biological product.
(iv) The value of such priority review voucher if
transferred.
(v) Identification of each drug for which a priority review
voucher awarded under such section 529 was used.
(vi) The size of the company using each priority review
voucher awarded under such section 529.
(vii) The length of the period of time between the date on
which a priority review voucher was awarded under such
section 529 and the date on which it was used.
(viii) Whether, and to what extent, an unmet need related
to the treatment or prevention of a rare pediatric disease
was met through the approval under section 505 of the Federal
Food, Drug, and Cosmetic Act (42 U.S.C. 355) or licensure
under section 351 of the Public Health Service Act (42 U.S.C.
262) of a drug for which a priority review voucher was used.
(ix) Whether, and to what extent, companies were motivated
by the availability of priority review vouchers under section
529 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
360ff) to attempt to develop a drug for a rare pediatric
disease.
(x) Whether, and to what extent, pediatric review vouchers
awarded under such section were successful in stimulating
development and expedited patient access to drug products for
treatment or prevention of a rare pediatric disease that
wouldn't otherwise take place without the incentive provided
by such vouchers.
(xi) The impact of such priority review vouchers on the
workload, review process, and public health prioritization
efforts of the Food and Drug Administration.
(xii) Any other incentives in Federal law that exist for
companies developing drugs or biological products described
in clause (i).
(2) Report on findings.--Not later than 5 years after the
date of the enactment of this Act, the Comptroller General of
the United States shall submit to the Committee on Energy and
Commerce of the House of Representatives and the Committee on
Health, Education, Labor, and Pensions of the Senate a report
containing the findings of the study conducted under
paragraph (1).
SEC. 105. LIMITATIONS ON EXCLUSIVE APPROVAL OR LICENSURE OF
ORPHAN DRUGS.
(a) In General.--Section 527 of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 360cc) is amended--
(1) in subsection (a), in the matter following paragraph
(2), by striking ``same disease or condition'' and inserting
``same approved use or indication within such rare disease or
condition'';
(2) in subsection (b)--
(A) in the matter preceding paragraph (1), by striking
``same rare disease or condition'' and inserting ``same
approved use or indication for which such 7-year period
applies to such already approved or licensed drug''; and
(B) in paragraph (1), by inserting ``, relating to the
approved use or indication,'' after ``the needs'';
(3) in subsection (c)(1), by striking ``same rare disease
or condition as the already approved drug'' and inserting
``same use or indication for which the already approved or
licensed drug was approved or licensed''; and
(4) by adding at the end the following:
``(f) Approved Use or Indication Defined.--In this section,
the term `approved use or indication' means the use or
indication approved under section 505 of this Act or licensed
under section 351 of the Public Health Service Act for a drug
designated under section 526 for a rare disease or
condition.''.
(b) Application of Amendments.--The amendments made by
subsection (a) shall apply with respect to any drug
designated under section 526 of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 360bb), regardless of the date on
which the drug was so designated, and regardless of the date
on which the drug was approved under section 505 of such Act
(21 U.S.C. 355) or licensed under section 351 of the Public
Health Service Act (42 U.S.C. 262).
SEC. 106. PROGRAM FOR PEDIATRIC STUDIES OF DRUGS.
Section 409I(d) of the Public Health Service Act (42 U.S.C.
284m(d)) is amended to read as follows:
``(d) Funding.--Of the amount made available for pediatric
research to each national research institute and national
center under this title for each of fiscal years 2025, 2026,
and 2027, the Director of NIH is authorized to make available
up to one percent of such amount for pediatric research under
this section.''.
TITLE II--UNITED STATES-ABRAHAM ACCORDS COOPERATION AND SECURITY
SEC. 201. ESTABLISHMENT OF ABRAHAM ACCORDS OFFICE WITHIN FOOD
AND DRUG ADMINISTRATION.
(a) In General.--Chapter X of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 391 et seq.) is amended by adding at
the end the following:
``SEC. 1015. ABRAHAM ACCORDS OFFICE.
``(a) In General.--The Secretary, acting through the
Commissioner of Food and Drugs, shall establish within the
Food and Drug Administration an office, to be known as the
Abraham Accords Office, to be headed by a director.
``(b) Office.--Not later than two years after the date of
enactment of this section, the Secretary shall--
``(1) in consultation with the governments of Abraham
Accords countries, as well as appropriate United States
Government diplomatic and security personnel--
``(A) select the location of the Abraham Accords Office in
an Abraham Accords country; and
``(B) establish such office; and
``(2) assign to such office such personnel of the Food and
Drug Administration as the Secretary determines necessary to
carry out the functions of such office.
``(c) Duties.--The Secretary, acting through the Director
of the Abraham Accords Office, shall--
``(1) after the Abraham Accords Office is established--
``(A) as part of the Food and Drug Administration's work to
strengthen the international oversight of regulated
commodities, provide technical assistance to regulatory
partners in Abraham Accords countries on strengthening
regulatory oversight and converging regulatory requirements
for the oversight of regulated products, including good
manufacturing practices and other issues relevant to
manufacturing medical products that are regulated by the Food
and Drug Administration;
``(B) facilitate interactions between the Food and Drug
Administration and interested parties in Abraham Accords
countries, including by sharing relevant information
regarding United States regulatory pathways with such
parties; and
``(C) facilitate feedback between the Food and Drug
Administration and such parties located within Abraham
Accords countries prior to submission of an application under
section 505(b), 505(j), or 515 of this Act or section 351(a)
or 351(k) of the Public Health Service Act, or a notification
under section 510(k) of this Act, such as feedback on
research, development, and manufacturing of drugs, biologics,
and medical devices; and
``(2) carry out other functions and activities as the
Secretary determines to be necessary to carry out this
section.
``(d) Abraham Accords Country Defined.--In this section,
the term `Abraham Accords country' means a country identified
by the Department of State as having signed the Abraham
Accords Declaration.''.
(b) Report to Congress.--
(1) In general.--Not later than 3 years after the date of
enactment of this Act, the Secretary of Health and Human
Services shall submit to the Congress a report on the Abraham
Accords Office, including--
(A) an evaluation of how the Office has advanced progress
toward conformance with Food and Drug Administration
regulatory requirements by manufacturers in the Abraham
Accords countries;
(B) a numerical count of parties that the Office has helped
facilitate interactions or feedback pursuant to subparagraphs
(B) and (C) of section 1015(c)(1) of the Federal Food, Drug,
and Cosmetic Act (as added by subsection (a));
(C) a summary of technical assistance provided to
regulatory partners in Abraham Accords countries pursuant to
subparagraph (A) of such section 1015(c)(1); and
(D) recommendations for increasing and improving
coordination between the Food and Drug Administration and
entities in Abraham Accords countries.
(2) Abraham accords country defined.--In this subsection,
the term ``Abraham Accords country'' has the meaning given
such term in section 1015(d) of the Federal Food, Drug, and
Cosmetic Act (as added by subsection (a)).
TITLE III--ORGAN PROCUREMENT AND TRANSPLANTATION NETWORK
SEC. 301. REGISTRATION FEES.
Section 372 of the Public Health Service Act (42 U.S.C.
274) is amended by adding at the end the following:
``(d) Registration Fees.--
``(1) In general.--The Secretary may collect registration
fees from any member of the Organ Procurement and
Transplantation Network for each transplant candidate such
member places on the list described in subsection
(b)(2)(A)(i). Such registration fees shall only be collected
and distributed to support the operation of the Organ
Procurement and Transplantation Network. Such registration
fees are authorized to remain available until expended.
``(2) Collection.--The Secretary may collect the
registration fees under paragraph (1) directly or through
awards made under subsection (b)(1)(A).
``(3) Distribution.--Any amounts collected under this
subsection shall--
``(A) be credited to the currently applicable
appropriation, account, or fund of the Department of Health
and Human Services as discretionary offsetting collections;
and
``(B) be available, only to the extent and in the amounts
provided in advance in appropriations Acts, to distribute
such fees among
[[Page H5585]]
the awardees described in subsection (b)(1)(A).
``(4) Transparency.--The Secretary shall--
``(A) promptly post on the Internet website of the Organ
Procurement and Transplant Network--
``(i) the amount of registration fees collected under this
subsection from each member of the Organ Procurement and
Transplantation Network; and
``(ii) a list of activities such fees are used to support;
and
``(B) update the information posted pursuant to
subparagraph (A), as applicable for each calendar quarter for
which fees are collected under paragraph (1).
``(5) GAO review.--Not later than 2 years after the date of
enactment of this subsection, the Comptroller General of the
United States shall, to the extent data are available--
``(A) conduct a review concerning the activities under this
subsection; and
``(B) submit to the Committee on Health, Education, Labor,
and Pensions and the Committee on Finance of the Senate and
the Committee on Energy and Commerce of the House of
Representatives, a report on such review, including related
recommendations, as applicable.''.
The SPEAKER pro tempore. Pursuant to the rule, the gentleman from
Indiana (Mr. Bucshon) and the gentleman from New Jersey (Mr. Pallone)
each will control 20 minutes.
The Chair recognizes the gentleman from Indiana.
General Leave
Mr. BUCSHON. Mr. Speaker, I ask unanimous consent that all Members
may have 5 legislative days in which to revise and extend their remarks
and include extraneous material in the Record on the bill.
The SPEAKER pro tempore. Is there objection to the request of the
gentleman from Indiana?
There was no objection.
Mr. BUCSHON. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, I rise in support of H.R. 3433, the Give Kids a Chance
Act of 2024, and thank Representatives McCaul, Eshoo, and Bilirakis for
their hard work on this important piece of legislation.
Mr. Speaker, I am excited that we have reached broad bipartisan
agreement on this comprehensive bill, which will provide hope to
approximately 30 million Americans affected by rare diseases.
Currently, only about 500 of these diseases have FDA-approved
treatments. This legislation will foster the development of more
pediatric rare disease treatments by strengthening the orphan drug
pathway, ensuring drugs are studied for children battling cancer, and
reauthorizing the rare pediatric disease priority review voucher
program, which is set to expire on September 30.
The PRV program has been a lifeline for patients who require
specialized treatments that would not be developed without the
incentive this program provides.
I am also supportive of the other provisions in this bill, which
would increase organ transplant rates by strengthening OPTN operations
and require the FDA to establish an office in a country that has signed
the Abraham Accords.
I am grateful to Representatives Harshbarger and Vargas for their
leadership on this effort to facilitate operations between the FDA and
regulatory authorities and innovators in Abraham Accords countries.
There has been a lot of discussion about moving supply chains for
medical products back to the U.S. and our allies, and this office is a
good step to help do just that.
Mr. Speaker, I encourage my colleagues to support this bill, and I
reserve the balance of my time.
Mr. PALLONE. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, I rise today in support of H.R. 3433, the Give Kids a
Chance Act, sponsored by Representatives McCaul and Eshoo.
This bill includes several strong bipartisan pieces of legislation,
and I am pleased that our Republican colleagues agreed to work with us
on advancing important policies.
This legislation will help expand access to care for patients with
rare diseases and complex medical needs, increase research and
development into rare pediatric diseases, and improve regulatory
certainty.
This bill will help in our efforts to bring new treatments to
pediatric patients with more advanced forms of cancer. It also includes
important sections of H.R. 6664, Representative Eshoo's bill, the
Innovation in Pediatric Drugs Act. Specifically, it gives the Food and
Drug Administration new enforcement authority to take action against
companies that do not conduct required studies in pediatric patients.
The bill also includes critical language introduced by Representative
Matsui as part of H.R. 7383, the Retaining Access and Restoring
Exclusivity Act, or RARE Act, and this will clarify FDA's long-held
treatment of orphan drug exclusivity and bring new treatments to more
patients suffering from rare diseases. These provisions all advance our
goals of accelerating the development of novel, safe, and effective
treatments and improving outcomes for patients.
These bills are going to make a difference, but I remain disappointed
that my Republican colleagues have been unwilling to consider necessary
changes to require drug manufacturers to study their approved rare
disease drugs in pediatric populations.
{time} 1545
It is disappointing that my Republican colleagues refuse to hold drug
manufacturers' feet to the fire and have decided to move forward with a
long-term reauthorization of the priority review voucher program, or
PRV, without requiring manufacturers to study rare disease drugs in
pediatric patients.
I have been consistent in my belief that we need reforms to the PRV
program. Unfortunately, we still have not seen the compelling evidence
that this program is functioning how Congress intended it to. That is
why I am pleased that the bill includes a requirement for the
Government Accountability Office to conduct a new study and report on
the effectiveness of the program in attaining our original intent.
Previous reports from GAO on the program, and continued research from
academics, show that there is little evidence that it incentivized the
products Congress intended to help come to market.
I continue to believe that additional guardrails are needed to better
target the intent of the program, but despite these concerns, I am
pleased that this bill includes important provisions to ensure that our
medical product supply chain can be strengthened by working with
international partners that believe in both tolerance and respect for
every person as well as advancement of science and medicine. I look
forward to working with our Republican colleagues to ensure FDA
receives the funding necessary to implement this policy and establish
an FDA office in Israel.
I am also pleased that this legislation provides the Health Resources
and Services Administration the authority to modernize the organ
transplant system. This system is lifesaving for many Americans, and I
am glad that we can continue to support these much-needed reform
efforts.
I believe that we could have achieved more for patients in this
legislation. However, since there are strong bipartisan policies
included here, I encourage all of my colleagues to vote ``yes'' on this
important bill, and I reserve the balance of my time.
Mr. BUCSHON. Mr. Speaker, I yield 5 minutes to the gentleman from
Texas (Mr. McCaul).
Mr. McCAUL. Mr. Speaker, I thank the gentleman from Indiana for
yielding.
Mr. Speaker, as a founder of the Childhood Cancer Caucus 15 years
ago, I have met countless cancer patients and advocates who have asked
me for one thing: to give the kids a chance. They want a chance to
receive better and safer treatments that preserve their quality of
life, a chance to beat childhood cancer, and a chance to eradicate this
disease once and for all.
The Give Kids a Chance Act does just that.
The bill is simple. It directs drug companies researching combination
drug therapies for adults to also research them for children, where
appropriate. This will allow us to treat children the same as we treat
adults.
With 235 cosponsors, this legislation is one of the most bipartisan
bills in Congress because there is nothing partisan or political about
a child with cancer.
Mr. Speaker, I thank Mr. Bilirakis from Florida and Ms. Anna Eshoo
from California, my partner in this. I also
[[Page H5586]]
thank Chair Cathy McMorris Rodgers and Ranking Member Frank Pallone for
all of their help in making this come together.
Included in this legislation is another one of my bills, the Creating
Hope Reauthorization Act. We first passed this legislation in 2012 to
create the pediatric priority review voucher program within the FDA
because we found that there was no market incentive for pharmaceutical
companies to develop cancer treatments for children. This program
provides the incentives.
Since its inception in 2012, 53 vouchers have been awarded for
treatments of 39 different rare pediatric diseases, 36 of which
previously had no FDA-approved treatments. These diseases typically led
to death before a child can reach adulthood, but not anymore.
A dear friend of mine, Dr. Allison from MD Anderson in my home State
of Texas, was able to use the voucher program to obtain FDA approval
for a treatment called CAR-T immunotherapy. Immunotherapy is a
breakthrough cancer treatment not just for children but for all adults.
He received the Nobel Prize in medicine for it.
This bill would reauthorize that successful program for another 5
years.
We just had our 15-year summit, and these children, I have to say to
the ranking member and to the chair, who I hope is listening, how much
joy this gave to their lives. These children who have either survived
cancer or are going through cancer, some can make it, some may not, but
this gives them hope.
I founded the caucus to give them a voice in the Congress because
they didn't have one. They didn't have high-powered lobbyists, but they
have us. This is the result of their voices.
We wouldn't be standing here today considering this legislation
without the help of patient advocates, including my dear childhood
cancer friends, Sadie Keller, Sophie Ryan, Brianna Commerford, and
Ailani Myers, and so many other brave children who have joined me in
advocating tirelessly on this important issue.
By the way, Mr. Speaker, these kids are tougher than any adults I
know, and they are my biggest inspiration in life.
Mr. Speaker, I am proud that my colleagues have come together in a
bipartisan spirit to do what is right for our children, which will
influence them for decades to come.
Mr. PALLONE. Mr. Speaker, I have no additional speakers, and I
reserve the balance of my time.
Mr. BUCSHON. Mr. Speaker, I yield 3 minutes to the gentleman from
Florida (Mr. Bilirakis).
Mr. BILIRAKIS. Mr. Speaker, I thank Dr. Bucshon, a great friend of
mine who does an outstanding job on our committee, for yielding.
Mr. Speaker, I rise in strong support of H.R. 3433, the Give Kids a
Chance Act, by Representative McCaul. His legislation strengthens
pathways for treatments and cures for children across the Nation.
The Give Kids a Chance Act would require that new studies of
pediatric cancer trials focus on combination therapies rather than
solely single-use drugs. This bill requires these pathways for children
with cancer in cases where both the drugs are FDA approved and owned by
the same company.
These combination products may help unlock new treatment options for
pediatric cancers and help extend the lives of children battling a rare
cancer.
I am proud to cosponsor this legislation. Furthermore, I am
particularly glad to see that this comprehensive package contains
provisions of two bills I co-lead, the Creating Hope Reauthorization
Act and the RARE Act. These bills will support innovation for pediatric
rare disease treatments and cures.
The Creating Hope Reauthorization Act will extend the FDA's rare
pediatric priority review voucher program for another 5 years, through
fiscal year 2029. This voucher serves as a critical and necessary
incentive for innovators to invest in research and development of
pediatric rare diseases.
Due to their extremely small patient populations, these diseases too
often go unnoticed, unfortunately. However, the voucher program allows
innovative rare disease companies to receive FDA priority review if
they get an approval for a pediatric rare disease. These vouchers can
then be used to expedite consideration of new treatments in the
pipeline, or transferred or sold to another company to recoup
investment losses.
This successful FDA incentive does not cost any taxpayer dollars and
must be extended to continue the pipeline of treatments for children
with rare diseases.
I was proud to lead the effort in the Energy and Commerce Committee
with my good friend and colleague, Anna Eshoo. We are going to miss
her. She is retiring, but hopefully, she will be around to give us some
advice in the future.
Again, we have bipartisan agreement on this. It is so very important
for our children.
Mr. Speaker, I ask my colleagues to join me in supporting H.R. 3433
off the floor today.
Mr. PALLONE. Mr. Speaker, I reserve the balance of my time.
Mr. BUCSHON. Mr. Speaker, I yield 5 minutes to the gentlewoman from
Tennessee (Mrs. Harshbarger).
Mrs. HARSHBARGER. Mr. Speaker, I rise today in strong support of H.R.
3433, the Give Kids a Chance Act, which is bipartisan legislation to
help speed up the process for getting treatments to children with
cancer and other rare diseases.
I am pleased that this bill also includes a modified version of
bipartisan legislation I have been leading with Democratic
Representative Juan Vargas: the United States-Abraham Accords
Cooperation and Security Act.
This section of H.R. 3433 will establish a U.S. Food and Drug
Administration office in the Abraham Accords region, creating a staging
ground for technical assistance and friend-shoring collaboration
between the U.S. and Abraham Accords countries, for development of and
bringing to our markets lifesaving drugs.
I thank House Committee on Energy and Commerce Chair Rodgers and
Ranking Member Pallone for working with me to advance this important
initiative. Chair Rodgers has been a strong ally in developing this.
Our continued dependence on adversarial countries like China for
pharmaceutical products endangers our medical supply chains and
jeopardizes our national security. More than 80 percent of the active
pharmaceutical ingredients for drugs sold in the U.S. are imported from
foreign countries, primarily China and India.
As our country works toward securing critical healthcare supply
chains from reliance on adversarial countries like China, the strategy
of friend-shoring or near-shoring has emerged as part of the solution
for industries where domestic production is not yet fully available.
Leveraging the Abraham Accords countries' robust biopharmaceutical
industries to help diversify and protect our access to crucial medical
supply chains that don't rely on China is a commonsense step to work
with our natural allies in our quest to safeguard our access to vital
medical products.
We need to begin this process of fortifying our medical supply chains
as soon as possible because we have a big hill to climb. With adoption
of H.R. 3433 and establishment of an FDA technical assistance office in
the Abraham Accords region, we begin climbing that hill.
Mr. Speaker, I urge all of my colleagues to support this bipartisan
legislation.
Mr. PALLONE. Mr. Speaker, obviously, this bill does a lot to help
childhood disorders. It is a very important bill, and I urge that it be
passed on a bipartisan basis.
Mr. Speaker, I yield back the balance of my time.
Mr. BUCSHON. Mr. Speaker, in closing, I encourage a ``yes'' vote on
this bill, and I yield back the balance of my time.
Ms. ESHOO. Mr. Speaker, today, the House will vote on my legislation,
H.R. 3433, the Give Kids a Chance Act.
My thanks to Reps. Michael McCaul (R-TX) and Gus Bilirakis (R-FL),
the chairwoman of the Energy and Commerce Committee Cathy McMorris
Rodgers (R-WA) and Ranking Member Frank Pallone (D-NJ) for their very
hard work on this important effort.
H.R. 3433 is the most comprehensive legislation to improve and
address childhood cancer and rare disease in over a decade.
As medicines become more sophisticated and we improve our
understanding of the
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deadliest diseases facing children in our country, our laws must keep
up.
This legislation will ensure children have access to the most
innovative treatments and therapies for cancer and rare disease, and it
does that by doing the following.
It reauthorizes the FDA's pediatric priority voucher program for five
years. The program expires September 23, 2024 and we cannot let
critical tools to encourage the development of drugs for children to
lapse.
It directs companies to conduct pediatric trials with combinations of
drugs. More than 40 combination therapies are approved for adults, but
only 2 are approved for children. This legislation fixes that inequity.
Every member of the Energy and Commerce Committee and 392 Members of
the House voted for this provision as part of the user fees package
last Congress.
It brings the FDA's enforcement capabilities for children on par with
that of adults, giving the FDA new options to ensure pediatric studies
are completed on time. Today, the FDA can only remove a drug from the
market if pediatric studies are not completed. This bill gives the FDA
more flexibility to ensure companies follow the law.
The bill dedicates existing funds for pediatric research through the
NIH's Best Pharmaceuticals for Children Act Program over the next three
years, the program's first funding update since it was authorized in
2002. Zero new funding will be used.
This bill was advanced by the House Energy and Commerce Committee
unanimously last week.
Children are not little adults. They deserve effective medicines,
just as adults do. The Give Kids a Chance Act will get children what
they deserve and save lives.
I urge my colleagues to vote for this critical legislation.
The SPEAKER pro tempore. The question is on the motion offered by the
gentleman from Indiana (Mr. Bucshon) that the House suspend the rules
and pass the bill, H.R. 3433, as amended.
The question was taken; and (two-thirds being in the affirmative) the
rules were suspended and the bill, as amended, was passed.
A motion to reconsider was laid on the table.
____________________