[Congressional Record Volume 171, Number 200 (Monday, December 1, 2025)]
[House]
[Pages H4929-H4935]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]
MIKAELA NAYLON GIVE KIDS A CHANCE ACT
Mr. CARTER of Georgia. Mr. Speaker, I move to suspend the rules and
pass the bill (H.R. 1262) 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. 1262
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 ``Mikaela
Naylon Give Kids a Chance Act''.
(b) Table of Contents.--The table of contents for this Act
is as follows:
Sec. 1. Short title; table of contents.
Sec. 2. Research into pediatric uses of drugs; additional authorities
of Food and Drug Administration regarding molecularly
targeted cancer drugs.
Sec. 3. Ensuring completion of pediatric study requirements.
Sec. 4. FDA report on PREA enforcement.
Sec. 5. Extension of authority to issue priority review vouchers to
encourage treatments for rare pediatric diseases.
Sec. 6. Limitations on exclusive approval or licensure of orphan drugs.
Sec. 7. Program for pediatric studies of drugs.
Sec. 8. Organ Procurement and Transplantation Network.
Sec. 9. Establishment of Abraham Accords Office within Food and Drug
Administration.
Sec. 10. Increasing transparency in generic drug applications.
Sec. 11. Medicare Improvement Fund.
SEC. 2. 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:
``(A) In general.--For purposes of paragraph (1)(B), the
investigation described in this paragraph is 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 used 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, after
consultation with the applicant, to be part of 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
[[Page H4930]]
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 is in a drug product
that has been previously approved to treat an adult cancer.
``(iii) Results of already-completed preclinical studies of
application drug.--With respect to an investigation required
pursuant to paragraph (1)(B), the Secretary may require the
results of any completed preclinical studies relevant to the
initial pediatric study plan be submitted to the Secretary at
the same time that the initial pediatric study plan required
under subsection (e)(1) is submitted.
``(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) applies 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
6 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 8 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, including consideration of any benefits to, or
burdens on, pediatric cancer drug development.
(B) Findings.--Not later than 10 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. 3. 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. 4. 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.''.
SEC. 5. 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 ``December 20, 2024, unless'' and all
that follows through the period at the end and inserting
``September 30, 2029.''.
(b) User Fee Payment.--Section 529(c)(4) of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C. 360ff(c)(4)) is
amended by striking subparagraph (A) and inserting the
following:
``(A) In general.--The priority review user fee required by
this subsection shall be due upon the submission of a human
drug application under section 505(b)(1) or section 351(a) of
the Public Health Service Act for which the priority review
voucher is used. All other user fees associated with the
human drug application shall be due as required by the
Secretary or under applicable law.''.
(c) 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--
(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.
[[Page H4931]]
(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. 6. 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. 7. PROGRAM FOR PEDIATRIC STUDIES OF DRUGS.
Section 409I(d)(1) of the Public Health Service Act (42
U.S.C. 284m(d)(1)) is amended by striking ``section,'' and
all that follows through the period at the end and inserting
``section, $25,000,000 for each of fiscal years 2026 through
2028.''.
SEC. 8. ORGAN PROCUREMENT AND TRANSPLANTATION NETWORK.
Section 372 of the Public Health Service Act (42 U.S.C.
274) is amended--
(1) in subsection (b)(2)--
(A) by moving the margins of subparagraphs (M) through (O)
2 ems to the left;
(B) in subparagraph (A)--
(i) in clause (i), by striking ``, and'' and inserting ``;
and''; and
(ii) in clause (ii), by striking the comma at the end and
inserting a semicolon;
(C) in subparagraph (C), by striking ``twenty-four-hour
telephone service'' and inserting ``24-hour telephone or
information technology service'';
(D) in each of subparagraphs (B) through (M), by striking
the comma at the end and inserting a semicolon;
(E) in subparagraph (N), by striking ``transportation,
and'' and inserting ``transportation;'';
(F) in subparagraph (O), by striking the period and
inserting a semicolon; and
(G) by adding at the end the following:
``(P) encourage the integration of electronic health
records systems through application programming interfaces
(or successor technologies) among hospitals, organ
procurement organizations, and transplant centers, including
the use of automated electronic hospital referrals and the
grant of remote, electronic access to hospital electronic
health records of potential donors by organ procurement
organizations, in a manner that complies with the privacy
regulations promulgated under the Health Insurance
Portability and Accountability Act of 1996, at part 160 of
title 45, Code of Federal Regulations, and subparts A, C, and
E of part 164 of such title (or any successor regulations);
and
``(Q) consider establishing a dashboard to display the
number of transplants performed, the types of transplants
performed, the number and types of organs that entered the
Organ Procurement and Transplantation Network system and
failed to be transplanted, and other appropriate statistics,
which should be updated more frequently than annually.''; and
(2) 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 be collected and
distributed only 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 awardees described in subsection (b)(1)(A).
``(4) Transparency.--The Secretary shall--
``(A) promptly post on the website of the Organ Procurement
and Transplantation 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.
``(6) Sunset.--The authority to collect registration fees
under paragraph (1) shall expire on the date that is 3 years
after the date of enactment of the Mikaela Naylon Give Kids a
Chance Act.''.
SEC. 9. 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; and
``(B) facilitate interactions between the Food and Drug
Administration and interested parties in Abraham Accords
countries, including by sharing relevant information
[[Page H4932]]
regarding United States regulatory pathways with such
parties, and facilitate feedback on the research,
development, and manufacturing of products regulated in
accordance with this Act; 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.
``(e) National Security.--Nothing in this section shall be
construed to require any action inconsistent with a national
security recommendation provided by the Federal
Government.''.
(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 section
1015(c)(1)(B) 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)).
SEC. 10. INCREASING TRANSPARENCY IN GENERIC DRUG
APPLICATIONS.
(a) In General.--Section 505(j)(3) of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 355(j)(3)) is amended by
adding at the end the following:
``(H)(i) Upon request (in controlled correspondence or an
analogous process) by a person that has submitted or intends
to submit an abbreviated application under this subsection
for a drug that is required by regulation to contain one or
more of the same inactive ingredients in the same
concentrations as the listed drug referred to, or for which
the Secretary determines there is a scientific justification
for an approach that is in vitro, in whole or in part, to be
used to demonstrate bioequivalence for a drug if such a drug
contains one or more of the same inactive ingredients in the
same concentrations as the listed drug referred to, the
Secretary shall inform the person whether such drug is
qualitatively and quantitatively the same as the listed drug.
The Secretary may also provide such information to such a
person on the Secretary's own initiative during the review of
an abbreviated application under this subsection for such
drug.
``(ii) Notwithstanding section 301(j), if the Secretary
determines that such drug is not qualitatively or
quantitatively the same as the listed drug, the Secretary
shall identify and disclose to the person--
``(I) the ingredient or ingredients that cause such drug
not to be qualitatively or quantitatively the same as the
listed drug; and
``(II) for any ingredient for which there is an identified
quantitative deviation, the amount of such deviation.
``(iii) If the Secretary determines that such drug is
qualitatively and quantitatively the same as the listed drug,
the Secretary shall not change or rescind such determination
after the submission of an abbreviated application for such
drug under this subsection unless--
``(I) the formulation of the listed drug has been changed
and the Secretary has determined that the prior listed drug
formulation was withdrawn for reasons of safety or
effectiveness; or
``(II) the Secretary makes a written determination that the
prior determination must be changed because an error has been
identified.
``(iv) If the Secretary makes a written determination
described in clause (iii)(II), the Secretary shall provide
notice and a copy of the written determination to the person
making the request under clause (i).
``(v) The disclosures authorized under clauses (i) and (ii)
are disclosures authorized by law, including for purposes of
section 1905 of title 18, United States Code. This
subparagraph shall not otherwise be construed to authorize
the disclosure of nonpublic qualitative or quantitative
information about the ingredients in a listed drug, or to
affect the status, if any, of such information as trade
secret or confidential commercial information for purposes of
section 301(j) of this Act, section 552 of title 5, United
States Code, or section 1905 of title 18, United States
Code.''.
(b) Guidance.--
(1) In general.--Not later than one year after the date of
enactment of this Act, the Secretary of Health and Human
Services shall issue draft guidance, or update guidance,
describing how the Secretary will determine whether a drug is
qualitatively and quantitatively the same as the listed drug
(as such terms are used in section 505(j)(3)(H) of the
Federal Food, Drug, and Cosmetic Act, as added by subsection
(a)), including with respect to assessing pH adjusters.
(2) Process.--In issuing guidance under this subsection,
the Secretary of Health and Human Services shall--
(A) publish draft guidance;
(B) provide a period of at least 60 days for comment on the
draft guidance; and
(C) after considering any comments received and not later
than one year after the close of the comment period on the
draft guidance, publish final guidance.
(c) Applicability.--Section 505(j)(3)(H) of the Federal
Food, Drug, and Cosmetic Act, as added by subsection (a),
applies beginning on the date of enactment of this Act,
irrespective of the date on which the guidance required by
subsection (b) is finalized.
SEC. 11. MEDICARE IMPROVEMENT FUND.
Section 1898(b)(1) of the Social Security Act (42 U.S.C.
1395iii(b)(1)) is amended by striking ``$1,403,000,000'' and
inserting ``$2,622,000,000''.
The SPEAKER pro tempore. Pursuant to the rule, the gentleman from
Georgia (Mr. Carter) and the gentlewoman from Michigan (Mrs. Dingell)
each will control 20 minutes.
The Chair recognizes the gentleman from Georgia.
General Leave
Mr. CARTER of Georgia. Mr. Speaker, I ask unanimous consent that all
Members may have 5 legislative days in which to revise and extend their
remarks and insert extraneous material in the Record on the bill.
The SPEAKER pro tempore. Is there objection to the request of the
gentleman from Georgia?
There was no objection.
Mr. CARTER of Georgia. Mr. Speaker, I yield 3 minutes to the
gentleman from Florida (Mr. Bilirakis), a very valuable member of the
Committee on Energy and Commerce.
Mr. BILIRAKIS. Mr. Speaker, I am rising in strong support of H.R.
1262, the Mikaela Naylon Give Kids a Chance Act, led by my friend, the
gentleman from Texas, Congressman McCaul.
This piece of legislation is comprised of bipartisan solutions to
increase access to innovative treatments for those living with rare
diseases, particularly children and adolescents.
Ten years ago, Mr. Speaker, the idea that we would be able to not
just treat but cure sickle cell disease sounded like a dream that was
too far out of reach. Today, we are curing sickle cell disease. We are
curing spinal muscular atrophy. We are curing hemophilia and blood
cancers. These cures are just a few of the success stories attributable
to the FDA's Rare Pediatric Disease Priority Review Voucher Program.
The PRV program is a powerful incentive that spurs the development of
treatments for rare pediatric diseases, providing a lifeline for
patients who require specialized treatments.
I also want to highlight the other provisions in this bill that seek
to bolster important components of our healthcare system.
This legislation supports the improvement of the Organ Procurement
and Transplantation Network by allowing the Secretary of HHS to collect
member registration fees to support the operation of the OPTN.
This bill also helps secure our medical product supply chain by
requiring 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
work to facilitate operations between the FDA and regulatory
authorities and innovators in Abraham Accords countries.
Last but not least, this bill also includes a provision championed by
Representatives Dunn and Mullin that will ensure more affordable drug
options are available to patients faster rather than being delayed by
unnecessary red tape.
Mr. Speaker, this bill has one of the largest totals of bipartisan
cosponsors of any legislation we will see this Congress.
One of the reasons for that is the tireless work of the patient
advocates. They come to our office on a regular basis and tell us their
personal stories. It makes such a difference, Mr. Speaker.
This bill now bears the name of one of those heroic kids, Mikaela
Naylon, who passed away earlier this year after battling cancer for
nearly 6 years. May her memory be eternal. She was 16 years old, and I
will never forget the videos.
Throughout her battle, Mikaela was a fierce advocate of this bill to
create
[[Page H4933]]
hope for a cure, not just for herself but for the thousands of kids
around the country who are fighting the same fight.
This is a very important bill, and I want to get it to the Senate as
soon as possible so it can become law.
Mr. Speaker, I encourage my colleagues to support this bill.
Mrs. DINGELL. Mr. Speaker, I yield such time as he may consume to the
gentleman from New Jersey (Mr. Pallone), the distinguished ranking
member of the Energy and Commerce Committee.
Mr. PALLONE. Mr. Speaker, I thank the sponsor of this legislation on
the Democratic side, the gentlewoman from Michigan (Mrs. Dingell), for
yielding.
I rise today in support of this legislation, the Mikaela Naylon Give
Kids a Chance Act, sponsored by Representatives Dingell and McCaul.
This bill includes several strong, bipartisan pieces of legislation
to help expand access to care for patients with rare diseases and
complex medical needs. It increases research and development into rare
pediatric diseases, improves regulatory certainty, strengthens the
medical product supply chain, and enhances patient access to lower-cost
medicines.
This bill will help in our efforts to bring new treatments to
pediatric patients with more advanced forms of cancer. It also includes
important enforcement authority for the Food and Drug Administration to
take action against companies that do not conduct required studies for
pediatric patients.
The bill also includes critical language to clarify the FDA's long-
held treatment of orphan drug exclusivity and bring new treatments to
more patients suffering from rare diseases. It will also allow for
disclosure of certain information related to drug formulations to
generic drug manufacturers to bring lower-cost medications to patients
more quickly.
These provisions all advance our goal of accelerating the development
of novel, safe, effective, and affordable treatments while improving
outcomes for patients.
I am also pleased that the legislation provides the Health Resources
and Services Administration, or HRSA, with the authority to modernize
the organ transplant system. This language will allow HRSA to collect
registration fees and distribute them among the multiple contractors
that will be supporting the OPTN. The system is lifesaving for
Americans, and I am glad that we can continue to support these much-
needed reform efforts.
Mr. Speaker, this bill also 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.
As a whole, this bill is going to make a difference, but I continue
to believe that additional guardrails are needed for the PRV program to
function the way Congress intended it to.
Previous reports from the Government Accountability Office on the
program and continued research from academics show that there is little
evidence that it incentivizes the products that Congress intended to
help come to market.
This is especially concerning as the FDA has introduced a new
priority review program with potentially massive financial benefits for
pharmaceutical companies without any transparency or congressional
authorization. That is why I am pleased that this bill includes a
requirement for the GAO to conduct a new study and report on the
effectiveness of the PRV program in achieving our original intent.
Let us not overstate the impact of this bill, however. While we are
working together to advance the bill forward on incremental but
important bipartisan legislation to advance both drug discovery and
drug affordability, the Trump administration continues its devastating
attacks on public health, including in cancer research.
{time} 1610
The Trump administration's substantial cuts to research funding for
the biomedical community is chilling the innovation that we are trying
to promote through this bill.
The Trump administration is decimating research and endangering the
pipeline of new cancer therapies that could come to FDA for approval.
Their actions are putting patients at risk and leaving pediatric
patients without the treatments that we are trying to ensure can be
tested and brought to them.
Meanwhile, the Trump administration is using the FDA as a tool to
provide lucrative gifts to drugmakers and allies of President Trump,
undermining the Agency's science-based review process, and endangering
the health of the American public with potentially unsafe and rushed
drug reviews.
The Trump administration is also decimating our vaccine
infrastructure, from the programs that cover our vaccines and ensure
that patients can afford them to destroying the pipeline to future
vaccines through their ideological, pseudoscience agenda.
Mr. Speaker, one day, I hope that my Republican colleagues will join
us in calling on the Trump administration to reverse these terrible
policies that are only harming the public health.
Mr. CARTER of Georgia. Mr. Speaker, I yield myself such time as I may
consume.
Mr. Speaker, I rise today in strong support of the bipartisan Give
Kids a Chance Act, which supports research and development of drugs for
children with cancer and other rare diseases.
Nearly 70 percent of rare diseases start in childhood, and most still
have no approved treatments. The Rare Pediatric Disease Priority Review
Voucher Program has been a catalyst for developing therapies for these
vulnerable populations.
Thanks to this program, treatments have reached children who are
suffering from nearly 40 rare diseases, many of which previously had no
FDA-approved options and often led to severe disability or death before
adulthood. Since 2012, the Rare Pediatric Disease PRV Program has
helped bring more than 60 new treatments to market and has driven
investment in hundreds of additional therapies for rare pediatric
diseases that would otherwise be financially unviable.
The Give Kids a Chance Act restores this critical program, ensuring
that we continue to spur innovation, improve outcomes for young
patients, and close gaps in pediatric drug research.
Mr. Speaker, I encourage my colleagues to support this bill.
Mr. Speaker, I reserve the balance of my time.
Mrs. DINGELL. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, I rise today to share my strong support for H.R. 1262,
the Mikaela Naylon Give Kids a Chance Act. I am proud to co-lead this
important, bipartisan legislation alongside Representatives Michael
McCaul, Dr. Kim Schrier, and Gus Bilirakis.
This bill will accelerate pediatric cancer treatments and expand
access to lifesaving therapies for children battling rare diseases.
It is impossible to fully express the pain and the devastation that a
family experiences when their child is diagnosed with cancer.
One of the bill's advocates was one that we are all going to talk
about, Mikaela Naylon, a 16-year-old girl in Colorado who battled bone
cancer. She was a fierce fighter for this bill. She talked to all of
us, and we all sent videos to her. Unfortunately, she died on October
30.
Her mother told all of us, the sponsors and the cosponsors of this
bill, that we gave her reason to live, and she hopes that other
children will not die at her age and that this bill will help save
lives.
She supported and spoke on the importance of funding cancer research
to be able to help other kids like herself. She was a fighter her
entire life, and this lifesaving bill has been renamed in her honor.
There are too many others facing the fight, as Mikaela has, each
year. Nearly 16,000 children in just the United States are diagnosed
with cancer each year.
Mr. Speaker, 1 in every 285 children in the U.S. will be diagnosed
with cancer before they reach 20 years of age. In fact, children
comprise as many as half of those living with rare diseases. Yet, here
is the reality for those children: Treatment options for children
remain extremely limited compared to those for adults.
We know that children respond to cancer treatments differently, but
[[Page H4934]]
there is limited research on how these therapies impact them. We owe
our kids better than that, and their families deserve more.
We need this bill to offer more children a fighting chance. The Give
Kids a Chance Act would reauthorize the Food and Drug Administration
Priority Review Voucher Program to allow pharmaceutical companies to
expedite FDA review of more profitable drugs in return for developing
treatments for rare pediatric diseases.
Since 2011, 53 PRVs have been awarded for 35 different rare pediatric
diseases. Additionally, thousands of successful drug combination
therapies are now being studied and developed for adults but not for
kids; not for our children.
The Give Kids a Chance Act also authorizes the FDA to direct
companies to study a combination of cancer drugs and therapies in
pediatric trials, as well. Together, we can show these young children
and their families that they are not alone in this fight.
Mr. Speaker, I do thank the 313 cosponsors of this bill for their
support, along with Chairman Guthrie and Ranking Member Pallone for
their assistance and support for this important bill. It has taken too
long to get here today, but we are here. Let's show the kids in this
country that we care.
Mr. Speaker, I urge my colleagues to vote ``yes,'' and I reserve the
balance of my time.
Mr. CARTER of Georgia. Mr. Speaker, I yield such time as he may
consume to the gentleman from Texas (Mr. McCaul), the esteemed Member
who has announced that this will be his last session and who has had a
stellar career in this august body and who has led in many, many ways,
in many committees, and whose career we all want to emulate.
Mr. McCAUL. Mr. Speaker, I thank my dear friend from Georgia (Mr.
Carter) for those kind words. I thank Chairman Guthrie, Gus Bilirakis,
Mr. Pallone, and, especially, Chairwoman Dingell for their steadfast
advocacy for these kids. I can't think of anything more important in my
22 years.
As chairman of the Childhood Cancer Caucus, I host a yearly childhood
cancer summit. Mrs. Dingell is a co-chair. It is a time for pediatric
cancer patients, survivors, and advocates to rally together around
these precious children, the most precious thing we have, and to spread
hope to them.
Each year, I meet a child who impacts me in a very strong way. This
year, I was blessed to get to know this beautiful, beautiful young
woman by the name of Mikaela Naylon. I could see in her eyes that she
was at the height of her struggle with cancer. It took so much strength
for her to come to the summit, but she believed that she was meant to
be there. She wanted her presence and her voice to help shape our
policies and change our world for the better.
We got her into a joint clinical trial at MD Anderson and Texas
Children's Hospital, but, unfortunately, it was too late. She had to be
put on a ventilator 3 weeks after our summit, and she passed away.
As Congresswoman Dingell mentioned, we sent videos to her to remind
her of how important she is. In her final moments on this Earth, I
reached out to her and told her that her story would not be forgotten
and that her legacy would continue to help thousands of other children.
I wanted her to know that her impact would outlive all of us.
Mr. Speaker, today, in her honor, the United States Congress will
vote on the Mikaela Naylon Give Kids a Chance Act.
Mr. Speaker, 16 years ago, I founded the Childhood Cancer Caucus
because I saw that children with cancer did not have a voice here in
Washington. We cared about adult treatments, but no one was paying
attention to the precious children who are battling this heartbreaking
disease. In fact, they called it rare orphan diseases.
{time} 1620
Since then, we have made a change for the better, and today is proof
of that. Put simply, this bill gives kids the same chance to beat
cancer that adults already have.
Right now, thousands of successful drug combination therapies are
being studied and developed for adults, but not for children. The Give
Kids a Chance Act will change that. It authorizes the FDA to direct
companies to study those same drugs and therapies in children, as well.
It will also reauthorize my Creating Hope Reauthorization Act, which
created a pediatric review voucher program within the FDA. It was the
first-ever market incentive for pharmaceutical companies to develop
childhood cancer treatments. Prior to that, there were none.
Since its inception in 2012, 63 vouchers have been awarded for
treatments of at least 39 different rare pediatric diseases, 36 of
which previously had no FDA-approved treatments. These diseases
typically lead 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 this voucher program to obtain FDA approval
for a treatment called CAR-T immunotherapy. He received the Nobel Prize
in medicine for it, and this voucher program had everything to do with
the FDA approval of it. This breakthrough treatment could change
everything, and it has changed everything, not just for kids with
cancer, but also for many adults.
This bill will keep this successful voucher program going for another
5 years, fueling more innovation and more hope for children and
families who desperately need it.
Mr. Speaker, I thank all of my friends on the Committee on Energy and
Commerce--I am not on the committee--who helped me get this bill done.
I also want to recognize the childhood cancer survivors who are in the
gallery today.
With 313 cosponsors, this bill has the most bipartisan support in
Congress, and that is because there is nothing political about a child
with cancer. There is nothing more important than saving the lives of
the next generation.
My prayers remain with Mikaela's family, and we are honored to have
them here with us today: her mother, Kassandra; her father, Doug; and
her brother, Ayden. I thank them for sharing Mikaela's beautiful story
with us.
Mikaela represents all the children who have suffered with this
heartbreaking disease. After meeting with many of them over all of
these years, I can tell you these kids are tougher and more resilient
than any adult I have ever seen, and they are truly my biggest
inspiration. Many survive, but many don't, and that needs to change.
Losing these kids has been the hardest part of my career over 22 years.
Today, I am encouraged because Mikaela's legacy and all of their
legacies, all those who have died and survived this horrible disease,
will live on in this bill forever.
Mr. Speaker, it is rare in this place that you can pass a bill that
will end up saving children's lives. I can think of nothing more
important.
I urge my colleagues to do right by our children and pass the Mikaela
Naylon Give Kids a Chance Act without delay.
Mrs. DINGELL. Mr. Speaker, I am ready to close, but before doing so,
I thank my colleague, with whom I have been proud to co-chair the
caucus. We both know too many children who have died, but we know a lot
who we were able to get help for. We make a commitment to all the
families in the gallery, and those who we have not gotten to know, that
no matter where the gentleman is going, he and I are in this fight for
the rest of our lives. I thank him for all of his work. I am going to
miss my colleague.
Mr. Speaker, I yield back the balance of my time and beg everybody to
support this bill.
The SPEAKER pro tempore. The Chair reminds Members not to refer to
persons in the gallery.
Mr. CARTER of Georgia. Mr. Speaker, I apologize. I do have one other
speaker.
Mr. Speaker, I yield such time as he may consume to the gentleman
from California (Mr. Obernolte).
Mr. OBERNOLTE. Mr. Speaker, I thank my friend, the gentleman from
Georgia, for recognizing me.
Mr. Speaker, I rise in strong support as both a member of the Health
Subcommittee and a proud cosponsor of this bill, H.R. 1262, the Give
Kids a Chance Act.
Mr. Speaker, we live in the most technologically advanced society in
the history of human civilization, and modern medicine has recently
come up
[[Page H4935]]
with amazing solutions and cures for many of the physical ailments that
have afflicted humanity.
Unfortunately, Mr. Speaker, we have many rare pediatric diseases for
which there is no cure. While we are researching the cures for those
diseases, thousands and thousands of children continue to suffer.
Mr. Speaker, we need to give every priority and do everything we
possibly can to speed up the delivery of these treatments for rare
pediatric diseases, and that is what this bill is all about. The Give
Kids a Chance Act will reauthorize the FDA's Priority Review Voucher
system to speed the research and development of these technologies and
innovative treatments and bring them to market. It also clarifies the
FDA's authority over expediting these therapies and reauthorizes needed
funding for the NIH to fund research and development into these cures.
Mr. Speaker, this bill will help us finally bring solutions to these
thousands of suffering children.
Mr. Speaker, I thank my bipartisan cosponsors for their hard work on
this bill. I urge this body to pass it this evening and urge its speedy
consideration in the Senate.
Mr. CARTER of Georgia. Mr. Speaker, in closing, I encourage a ``yes''
vote on this bill, and I yield back the balance of my time.
The SPEAKER pro tempore. The question is on the motion offered by the
gentleman from Georgia (Mr. Carter) that the House suspend the rules
and pass the bill, H.R. 1262, 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.
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