[House Report 118-700]
[From the U.S. Government Publishing Office]
118th Congress } { Report
HOUSE OF REPRESENTATIVES
2d Session } { 118-700
======================================================================
GIVE KIDS A CHANCE ACT OF 2024
_______
September 20, 2024.--Committed to the Committee of the Whole House on
the State of the Union and ordered to be printed
_______
Mrs. Rodgers of Washington, from the Committee on Energy and Commerce,
submitted the following
R E P O R T
[To accompany H.R. 3433]
The Committee on Energy and Commerce, to whom was referred
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, having
considered the same, reports favorably thereon with an
amendment and recommends that the bill as amended do pass.
CONTENTS
Page
Purpose and Summary.............................................. 7
Background and Need for Legislation.............................. 8
Committee Action................................................. 8
Committee Votes.................................................. 9
Oversight Findings and Recommendations........................... 11
New Budget Authority, Entitlement Authority, and Tax Expenditures 11
Congressional Budget Office Estimate............................. 11
Federal Mandates Statement....................................... 11
Statement of General Performance Goals and Objectives............ 11
Duplication of Federal Programs.................................. 11
Related Committee and Subcommittee Hearings...................... 11
Committee Cost Estimate.......................................... 12
Earmark, Limited Tax Benefits, and Limited Tariff Benefits....... 12
Advisory Committee Statement..................................... 12
Applicability to Legislative Branch.............................. 12
Section-by-Section Analysis of the Legislation................... 12
Changes in Existing Law Made by the Bill, as Reported............ 13
The amendment is as follows:
Strike all after the enacting clause and insert the
following:
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:
``(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--
(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.--The Secretary may distribute such fees
among 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.''.
Purpose and Summary
H.R. 3433 provides the Food and Drug Administration (FDA)
with additional authority to require pediatric cancer trials
for combination therapies. The bill also authorizes the FDA to
take enforcement action against companies that do not conduct
required pediatric trials under the Pediatric Research Equity
Act (PREA) and requires the FDA to report on enforcement of
PREA. Additionally, the bill reauthorizes the FDA rare
pediatric disease priority review voucher (PRV) program through
fiscal year 2029, and limits orphan drug exclusivity to the
approved indication, rather than the potentially broader
designation. Lastly, the bill requires the FDA to establish an
office in an Abraham Accord country and allows the Secretary of
Health and Human Services to collect registration fees and
distribute these fees to support the operation of OPTN.
Background and Need for Legislation
Today, it is estimated that over 80 percent of children
diagnosed with cancer will be cured.\1\ However, the rate of
improvement in survival has more recently slowed and more than
one-half of pediatric cancer survivors experience serious long-
term effects of their cancer and its therapy.\2\ Combination
therapy, a treatment method that utilizes two or more
therapeutic agents, is considered a cornerstone of cancer
therapy.\3\ Combination therapies are often more effective than
single agent therapy and demonstrate lower levels of toxicity
during long-term treatment.\4\ Currently, the FDA is only
authorized to require pediatric trials of single drug cancer
treatments. By extending this FDA authority to include studies
of combination therapies, as well as clarifying that Orphan
Drug Exclusivity allows approvals for other uses of a drug for
a disease or condition to permit pediatric indications,
reauthorizing the rare pediatric disease priority review
voucher (PRV) program and establishing an FDA office in an
Abraham Accords country to spur innovation, the Give Kids a
Chance Act of 2024 seeks to accelerate the development of
novel, safe, and effective treatments and improve outcomes for
pediatric cancer and rare pediatric diseases. Further, allowing
the Department of Health and Human Services (HHS) to collect
and distribute registration fees to support the OPTN operations
will continue our shared mission to increase organ transplant
rates and ultimately improve health outcomes.
---------------------------------------------------------------------------
\1\Erin Butler et al., ``Recent progress in the treatment of cancer
in children,'' American Cancer Society Journals, 2021, https://
acsjournals.onlinelibrary.wiley.com/doi/10.3322/caac.21665.
\2\Id.
\3\Rexa Bayat Mokhtari et al., ``Combination therapy in combating
cancer,'' Oncotarget, 2017, https://www.ncbi.nlm.nih.gov/pmc/articles/
PMC5514969/.
\4\Wang, Yiling and Audrey Minden, ``Current Molecular Combination
Therapies Used for the Treatment of Breast Cancer,'' International
Journal of Molecular Sciences, 2022, https://www. ncbi.nlm.nih.gov/pmc/
articles/PMC9569555/#::text=Recently%2C%20combination%20therapies%
20%28in%20which%20two%20or%20more,demonstrate%20lower%20levels%20of%20to
xicity%20 during%20long-term%20treatment.
---------------------------------------------------------------------------
Committee Action
On February 29, 2024, the Subcommittee on Health held a
hearing on several bills, including H.R. 3433, as well as H.R.
7384 and H.R. 7383, which are included with slight modification
in the amended text of H.R. 3433. The title of the hearing was
``Legislative Proposals to Support Patients with Rare
Diseases.'' The Subcommittee received testimony from:
Terence Flotte, MD, Provost and Dean of
UMass Chan Medical School, Vice President of American
Society of Gene and Cell Therapy;
Alexander Bassuk, MD, PhD, Physician-in-
Chief, University of Iowa Stead Family Children's
Hospital; Chair and Professor, Stead Family Department
of Pediatrics;
Aaron Kesselheim, MD, JD, MPH, Professor of
Medicine, Harvard Medical School; Director, Program On
Regulation, Therapeutics, And Law (PORTAL) at Brigham
and Women's Hospital;
Jeromie Ballreich, PhD, Associate Research
Professor, Johns Hopkins Bloomberg School of Public
Health;
Alice Chen, PhD, Senior Fellow, USC
Schaeffer Center for Health Policy and Economics;
Associate Professor and Vice Dean for Research, USC Sol
Price School of Public Policy; and,
Khrystal Davis, JD, Founding President,
Texas Rare Alliance.
On May 16, 2024, the Subcommittee on Health met in open
markup session and forwarded H.R. 3433, as amended, to the full
Committee by a vote of 16 yeas and 11 nays.
On September 18, 2024, the full Committee on Energy and
Commerce met in open markup session and ordered H.R. 3433, as
amended, favorably reported to the House by a recorded vote of
43 yeas and 0 nays.
Committee Votes
Clause 3(b) of rule XIII requires the Committee to list the
record votes on the motion to report legislation and amendments
thereto. The following reflects the record votes taken during
the Committee consideration:
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Oversight Findings and Recommendations
Pursuant to clause 2(b)(1) of rule X and clause 3(c)(1) of
rule XIII, the Committee held a hearing and made findings that
are reflected in this report.
New Budget Authority, Entitlement Authority,
and Tax Expenditures
Pursuant to clause 3(c)(2) of rule XIII, the Committee
finds that H.R. 3433 would result in no new or increased budget
authority, entitlement authority, or tax expenditures or
revenues.
Congressional Budget Office Estimate
Pursuant to clause 3(c)(3) of rule XIII, at the time this
report was filed, the cost estimate prepared by the Director of
the Congressional Budget Office pursuant to section 402 of the
Congressional Budget Act of 1974 was not available.
Federal Mandates Statement
The Committee adopts as its own the estimate of Federal
mandates prepared by the Director of the Congressional Budget
Office pursuant to section 423 of the Unfunded Mandates Reform
Act.
Statement of General Performance Goals and Objectives
Pursuant to clause 3(c)(4) of rule XIII, the general
performance goal or objective of this legislation is to promote
the development of new and effective treatments for pediatric
cancer and rare pediatric diseases.
Duplication of Federal Programs
Pursuant to clause 3(c)(5) of rule XIII, no provision of
H.R. 3433 is known to be duplicative of another Federal
program, including any program that was included in a report to
Congress pursuant to section 21 of Public Law 111-139 or the
most recent Catalog of Federal Domestic Assistance.
Related Committee and Subcommittee Hearings
Pursuant to clause 3(c)(6) of rule XIII, the following
related hearing was used to develop or consider H.R. 3433:
On February 29, 2024, the Subcommittee on
Health held a hearing on H.R. 3433, as well as H.R.
7384 and H.R. 7383, which are included with slight
modification in the amended text of H.R. 3433. The
title of the hearing was ``Legislative Proposals to
Support Patients with Rare diseases.'' The Subcommittee
received testimony from:
Terence Flotte, MD, Provost and Dean of
UMass Chan Medical School, Vice President of American
Society of Gene and Cell Therapy;
Alexander Bassuk, MD, PhD, Physician-in-
Chief, University of Iowa Stead Family Children's
Hospital; Chair and Professor, Stead Family Department
of Pediatrics;
Aaron Kesselheim, MD, JD, MPH, Professor
of Medicine, Harvard Medical School; Director, Program
On Regulation, Therapeutics, And Law (PORTAL) at
Brigham and Women's Hospital;
Jeromie Ballreich, PhD, Associate
Research Professor, Johns Hopkins Bloomberg School of
Public Health;
Alice Chen, PhD, Senior Fellow, USC
Schaeffer Center for Health Policy and Economics;
Associate Professor and Vice Dean for Research, USC Sol
Price School of Public Policy; and,
Khrystal Davis, JD, Founding President,
Texas Rare Alliance.
Committee Cost Estimate
Pursuant to clause 3(d)(1) of rule XIII, the Committee
adopts as its own the cost estimate prepared by the Director of
the Congressional Budget Office pursuant to section 402 of the
Congressional Budget Act of 1974. At the time this report was
filed, the estimate was not available.
Earmark, Limited Tax Benefits, and Limited Tariff Benefits
Pursuant to clause 9(e), 9(f), and 9(g) of rule XXI, the
Committee finds that H.R. 3433 contains no earmarks, limited
tax benefits, or limited tariff benefits.
Advisory Committee Statement
No advisory committees within the meaning of section 5(b)
of the Federal Advisory Committee Act were created by this
legislation.
Applicability to Legislative Branch
The Committee finds that the legislation does not relate to
the terms and conditions of employment or access to public
services or accommodations within the meaning of section
102(b)(3) of the Congressional Accountability Act.
Section-by-Section Analysis of the Legislation
Section 1. Short title
Section 1 provides that the Act may be cited as the ``Give
Kids a Chance Act of 2024''.
TITLE I--GIVE KIDS A CHANCE
Section 101. Research into pediatric uses of drugs; additional
authorities of Food and Drug Administration regarding
molecularly targeted cancer drugs
Section 101 provides the Food and Drug Administration (FDA)
the authority to require pediatric cancer trials for new drugs
that are used in combination with active ingredients that meet
the standard of care for targeting pediatric cancer or have
been approved to treat adult cancer and are directed at
molecular targets for pediatric cancer. It would also require
the Government Accountability Office to conduct a study and
report to Congress on the effectiveness of the requirements
outlined in this section in the development of drugs and
biological products for pediatric cancer indications.
Section 102. Ensuring completion of pediatric study requirements
Section 102 provides the FDA the authority to enforce
against companies that fail to meet pediatric study
requirements. The Secretary of the Department of Health and
Human Services shall perform due diligence before concluding
failure to meet requirements.
Section 103. FDA report on PREA enforcement
Section 103 requires the FDA to report on enforcement of
the Pediatric Research Equity Act (PREA).
Section 104. Extension of authority to issue priority review vouchers
to encourage treatments for rare pediatric diseases
Section 104 extends the FDA priority review voucher program
from fiscal year 2024 through fiscal year 2029, to incentivize
the development of drugs for rare pediatric diseases. It also
requires a study from the Government Accountability Office on
the effectiveness of the pediatric PRV program.
Section 105. Limitations on exclusive approval or licensure of orphan
drugs
Section 105 introduced as H.R. 7383, the ``Retaining Access
and Restoring Exclusivity (RARE) Act,'' by Rep. Doris Matsui
(D-CA), clarifies that orphan drug exclusivity applies to the
approved indication, rather than the potentially broader
designation, in alignment with the FDA's interpretation.
Section 106. Program for pediatric studies of drugs
Section 106 updates authority for the National Institutes
of Health (NIH) to fund studies of drugs in children, to better
reflect how it is currently funded.
TITLE II--UNITED STATES-ABRAHAM ACCORDS COOPERATION AND SECURITY
Section 201. Establishment of Abraham Accords office within Food and
Drug Administration
Section 201 would require the Food and Drug Administration
to establish an office in an Abraham Accords country to enhance
facilitation with the agency and require the Secretary of
Health and Human Services to submit a report to Congress 3
years after the date of enactment of this Act to evaluate the
office's progress.
TITLE III--ORGAN PROCUREMENT AND TRANSPLANTATION NETWORK
Section 301. Registration fees
Section 301 allows the Secretary of Health and Human
Services to collect registration fees from any member of the
Organ Procurement Transplantation Network (OPTN) for each
transplant candidate such member places on the list and
distribute these fees to support the operation of OPTN.
Changes in Existing Law Made by the Bill, as Reported
In compliance with clause 3(e) of rule XIII of the Rules of
the House of Representatives, changes in existing law made by
the bill, as reported, are shown as follows (existing law
proposed to be omitted is enclosed in black brackets, new
matter is printed in italics, and existing law in which no
change is proposed is shown in roman):
FEDERAL FOOD, DRUG, AND COSMETIC ACT
* * * * * * *
CHAPTER III--PROHIBITED ACTS AND PENALTIES
* * * * * * *
penalties
Sec. 303. (a)(1) Any person who violates a provision of
section 301 shall be imprisoned for not more than one year or
fined not more than $1,000, or both.
(2) Notwithstanding the provisions of paragraph (1) of this
section, if any person commits such a violation after a
conviction of him under this section has become final, or
commits such a violation with the intent to defraud or mislead,
such person shall be imprisoned for not more than three years
or fined not more than $10,000 or both.
(b)(1) Notwithstanding subsection (a), any person who
violates section 301(t) by--
(A) knowingly importing a drug in violation of
section 801(d)(1),
(B) knowingly selling, purchasing, or trading a drug
or drug sample or knowingly offering to sell, purchase,
or trade a drug or drug sample, in violation of section
503(c)(1),
(C) knowingly selling, purchasing, or trading a
coupon, knowingly offering to sell, purchase, or trade
such a coupon, or knowingly counterfeiting such a
coupon, in violation of section 503(c)(2), or
(D) knowingly distributing drugs in violation of
section 503(e)(1),
shall be imprisoned for not more than 10 years or fined not
more than $250,000, or both.
(2) Any manufacturer or distributor who distributes drug
samples by means other than the mail or common carrier whose
representative, during the course of the representative's
employment or association with that manufacturer or
distributor, violated section 301(t) because of a violation of
section 503(c)(1) or violated any State law prohibiting the
sale, purchase, or trade of a drug sample subject to section
503(b) or the offer to sell, purchase, or trade such a drug
sample shall, upon conviction of the representative for such
violation, be subject to the following civil penalties:
(A) A civil penalty of not more than $50,000 for each
of the first two such violations resulting in a
conviction of any representative of the manufacturer or
distributor in any 10-year period.
(B) A civil penalty of not more than $1,000,000 for
each violation resulting in a conviction of any
representative after the second conviction in any 10-
year period.
For the purposes of this paragraph, multiple convictions of one
or more persons arising out of the same event or transaction,
or a related series of events or transactions, shall be
considered as one violation.
(3) Any manufacturer or distributor who violates section
301(t) because of a failure to make a report required by
section 503(d)(3)(E) shall be subject to a civil penalty of not
more than $100,000.
(4)(A) If a manufacturer or distributor or any representative
of such manufacturer or distributor provides information
leading to the institution of a criminal proceeding against,
and conviction of, any representative of that manufacturer or
distributor for a violation of section 301(t) because of a
sale, purchase, or trade or offer to purchase, sell, or trade a
drug sample in violation of section 503(c)(1) or for a
violation of State law prohibiting the sale, purchase, or trade
or offer to sell, purchase, or trade a drug sample, the
conviction of such representative shall not be considered as a
violation for purposes of paragraph (2).
(B) If, in an action brought under paragraph (2) against a
manufacturer or distributor relating to the conviction of a
representative of such manufacturer or distributor for the
sale, purchase, or trade of a drug or the offer to sell,
purchase, or trade a drug, it is shown, by clear and convincing
evidence--
(i) that the manufacturer or distributor conducted,
before the institution of a criminal proceeding against
such representative for the violation which resulted in
such conviction, an investigation of events or
transactions which would have led to the reporting of
information leading to the institution of a criminal
proceeding against, and conviction of, such
representative for such purchase, sale, or trade or
offer to purchase, sell, or trade, or
(ii) that, except in the case of the conviction of a
representative employed in a supervisory function,
despite diligent implementation by the manufacturer or
distributor of an independent audit and security system
designed to detect such a violation, the manufacturer
or distributor could not reasonably have been expected
to have detected such violation,
the conviction of such representative shall not be considered
as a conviction for purposes of paragraph (2).
(5) If a person provides information leading to the
institution of a criminal proceeding against, and conviction
of, a person for a violation of section 301(t) because of the
sale, purchase, or trade of a drug sample or the offer to sell,
purchase, or trade a drug sample in violation of section
503(c)(1), such person shall be entitled to one-half of the
criminal fine imposed and collected for such violation but not
more than $125,000.
(6) Notwithstanding subsection (a), any person who is a
manufacturer or importer of a prescription drug under section
804(b) and knowingly fails to comply with a requirement of
section 804(e) that is applicable to such manufacturer or
importer, respectively, shall be imprisoned for not more than
10 years or fined not more than $250,000, or both.
(7) Notwithstanding subsection (a)(2), any person that
knowingly and intentionally adulterates a drug such that the
drug is adulterated under subsection (a)(1), (b), (c), or (d)
of section 501 and has a reasonable probability of causing
serious adverse health consequences or death to humans or
animals shall be imprisoned for not more than 20 years or fined
not more than $1,000,000, or both.
(8) Notwithstanding subsection (a), any person who violates
section 301(i)(3) by knowingly making, selling or dispensing,
or holding for sale or dispensing, a counterfeit drug, or who
violates section 301(fff)(3) by knowingly making, selling or
dispensing, or holding for sale or dispensing, a counterfeit
device, shall be imprisoned for not more than 10 years or fined
in accordance with title 18, United States Code, or both.
(c) No person shall be subject to the penalties of subsection
(a)(1) of this section, (1) for having received in interstate
commerce any article and delivered it or proffered delivery of
it, if such delivery or proffer was made in good faith, unless
he refuses to furnish on request of an officer or employee duly
designated by the Secretary the name and address of the person
from whom he purchased or received such article and copies of
all documents, if any there be, pertaining to the delivery of
the article to him; or (2) for having violated section 301(a)
or (d), if he establishes a guaranty or undertaking signed by,
and containing the name and address of, the person residing in
the United States from whom he received in good faith the
article, to the effect, in case of an alleged violation of
section 301(a), that such article is not adulterated or
misbranded, within the meaning of this Act, designating this
Act, or to the effect, in case of an alleged violation of
section 301(d), that such article is not an article which may
not, under the provisions of section 404 or 505, be introduced
into interstate commerce; or (3) for having violated section
301(a), where the violation exists because the article is
adulterated by reason of containing a color additive not from a
batch certified in accordance with regulations promulgated by
the Secretary under this Act, if such person establishes a
guaranty or undertaking signed by, and containing the name and
address of, the manufacturer of the color additive, to the
effect that such color additive was from a batch certified in
accordance with the applicable regulations promulgated by the
Secretary under this Act; or (4) for having violated section
301 (b), (c), or (k) by failure to comply with section 502(f)
in respect to an article received in interstate commerce to
which neither section 503(a) nor section 503(b)(1) is
applicable, if the delivery or proffered delivery was made in
good faith and the labeling at the time thereof contained the
same directions for use and warning statements as were
contained in the labeling at the time of such receipt of such
article; or (5) for having violated section 301(i)(2) if such
person acted in good faith and had no reason to believe that
use of the punch, die, plate, stone, or other thing involved
would result in a drug being a counterfeit drug, or for having
violated section 301(i)(3) if the person doing the act or
causing it to be done acted in good faith and had no reason to
believe that the drug was a counterfeit drug; or (6) for having
violated section 301(fff)(2) if such person acted in good faith
and had no reason to believe that use of the punch, die, plate,
stone, or other thing involved would result in a device being a
counterfeit device, or for having violated section 301(fff)(3)
if the person doing the act or causing it to be done acted in
good faith and had no reason to believe that the device was a
counterfeit device.
(d) No person shall be subject to the penalties of subsection
(a)(1) of this section for a violation of section 301 involving
misbranded food if the violation exists solely because the food
is misbranded under section 403(a)(2) because of its
advertising.
(e)(1) Except as provided in paragraph (2), whoever knowingly
distributes, or possesses with intent to distribute, human
growth hormone for any use in humans other than the treatment
of a disease or other recognized medical condition, where such
use has been authorized by the Secretary of Health and Human
Services under section 505 and pursuant to the order of a
physician, is guilty of an offense punishable by not more than
5 years in prison, such fines as are authorized by title 18,
United States Code, or both.
(2) Whoever commits any offense set forth in paragraph (1)
and such offense involves an individual under 18 years of age
is punishable by not more than 10 years imprisonment, such
fines as are authorized by title 18, United States Code, or
both.
(3) Any conviction for a violation of paragraphs (1) and (2)
of this subsection shall be considered a felony violation of
the Controlled Substances Act for the purposes of forfeiture
under section 413 of such Act.
(4) As used in this subsection the term ``human growth
hormone'' means somatrem, somatropin, or an analogue of either
of them.
(5) The Drug Enforcement Administration is authorized to
investigate offenses punishable by this subsection.
(f)(1)(A) Except as provided in subparagraph (B), any person
who violates a requirement of this Act which relates to devices
shall be liable to the United States for a civil penalty in an
amount not to exceed $15,000 for each such violation, and not
to exceed $1,000,000 for all such violations adjudicated in a
single proceeding. For purposes of the preceding sentence, a
person accredited under paragraph (2) of section 704(g) who is
substantially not in compliance with the standards of
accreditation under such section, or who poses a threat to
public health or fails to act in a manner that is consistent
with the purposes of such section, shall be considered to have
violated a requirement of this Act that relates to devices.
(B) Subparagraph (A) shall not apply--
(i) to any person who violates the requirements of
section 519(a) or 520(f) unless such violation
constitutes (I) a significant or knowing departure from
such requirements, or (II) a risk to public health,
(ii) to any person who commits minor violations of
section 519(e) or 519(g) (only with respect to
correction reports) if such person demonstrates
substantial compliance with such section, or
(iii) to violations of section 501(a)(2)(A) which
involve one or more devices which are not defective.
(2)(A) Any person who introduces into interstate commerce or
delivers for introduction into interstate commerce an article
of food that is adulterated within the meaning of section
402(a)(2)(B) or any person who does not comply with a recall
order under section 423 shall be subject to a civil money
penalty of not more than $50,000 in the case of an individual
and $250,000 in the case of any other person for such
introduction or delivery, not to exceed $500,000 for all such
violations adjudicated in a single proceeding.
(B) This paragraph shall not apply to any person who grew the
article of food that is adulterated. If the Secretary assesses
a civil penalty against any person under this paragraph, the
Secretary may not use the criminal authorities under this
section to sanction such person for the introduction or
delivery for introduction into interstate commerce of the
article of food that is adulterated. If the Secretary assesses
a civil penalty against any person under this paragraph, the
Secretary may not use the seizure authorities of section 304 or
the injunction authorities of section 302 with respect to the
article of food that is adulterated.
(C) In a hearing to assess a civil penalty under this
paragraph, the presiding officer shall have the same authority
with regard to compelling testimony or production of documents
as a presiding officer has under section 408(g)(2)(B). The
third sentence of paragraph (5)(A) shall not apply to any
investigation under this paragraph.
(3)(A) Any person who violates section 301(jj) shall be
subject to a civil monetary penalty of not more than $10,000
for all violations adjudicated in a single proceeding.
(B) If a violation of section 301(jj) is not corrected within
the 30-day period following notification under section
402(j)(5)(C)(ii), the person shall, in addition to any penalty
under subparagraph (A), be subject to a civil monetary penalty
of not more than $10,000 for each day of the violation after
such period until the violation is corrected.
(4)(A) Any responsible person (as such term is used in
section 505-1) that violates a requirement of section 505(o),
505(p), [or 505-1] 505-1, or 505B shall be subject to a civil
monetary penalty of--
(i) not more than $250,000 per violation, and not to
exceed $1,000,000 for all such violations adjudicated
in a single proceeding; or
(ii) in the case of a violation that continues after
the Secretary provides written notice to the
responsible person, the responsible person shall be
subject to a civil monetary penalty of $250,000 for the
first 30-day period (or any portion thereof) that the
responsible person continues to be in violation, and
such amount shall double for every 30-day period
thereafter that the violation continues, not to exceed
$1,000,000 for any 30-day period, and not to exceed
$10,000,000 for all such violations adjudicated in a
single proceeding.
(B) In determining the amount of a civil penalty under
subparagraph (A)(ii), the Secretary shall take into
consideration whether the responsible person is making efforts
toward correcting the violation of the requirement of section
505(o), 505(p), or 505-1 for which the responsible person is
subject to such civil penalty.
(5)(A) A civil penalty under paragraph (1), (2), (3), (4), or
(9) shall be assessed, or a no-tobacco-sale order may be
imposed, by the Secretary by an order made on the record after
opportunity for a hearing provided in accordance with this
subparagraph and section 554 of title 5, United States Code.
Before issuing such an order, the Secretary shall give written
notice to the person to be assessed a civil penalty, or upon
whom a no-tobacco-sale order is to be imposed, under such order
of the Secretary's proposal to issue such order and provide
such person an opportunity for a hearing on the order. In the
course of any investigation, the Secretary may issue subpoenas
requiring the attendance and testimony of witnesses and the
production of evidence that relates to the matter under
investigation.
(B) In determining the amount of a civil penalty, or the
period to be covered by a no-tobacco-sale order, the Secretary
shall take into account the nature, circumstances, extent, and
gravity of the violation or violations and, with respect to the
violator, ability to pay, effect on ability to continue to do
business, any history of prior such violations, the degree of
culpability, and such other matters as justice may require. A
no-tobacco-sale order permanently prohibiting an individual
retail outlet from selling tobacco products shall include
provisions that allow the outlet, after a specified period of
time, to request that the Secretary compromise, modify, or
terminate the order.
(C) The Secretary may compromise, modify, or remit, with or
without conditions, any civil penalty which may be assessed
under paragraph (1), (2), (3), (4), or (9). The amount of such
penalty, when finally determined, or the amount agreed upon in
compromise, may be deducted from any sums owing by the United
States to the person charged.
(D) The Secretary may compromise, modify, or terminate, with
or without conditions, any no-tobacco-sale order.
(6) Any person who requested, in accordance with paragraph
(5)(A), a hearing respecting the assessment of a civil penalty
or the imposition of a no-tobacco-sale order and who is
aggrieved by an order assessing a civil penalty or the
imposition of a no-tobacco-sale order may file a petition for
judicial review of such order with the United States Court of
Appeals for the District of Columbia Circuit or for any other
circuit in which such person resides or transacts business.
Such a petition may only be filed within the 60-day period
beginning on the date the order making such assessment was
issued, or on which the no-tobacco-sale order was imposed, as
the case may be.
(7) If any person fails to pay an assessment of a civil
penalty--
(A) after the order making the assessment becomes
final, and if such person does not file a petition for
judicial review of the order in accordance with
paragraph (6), or
(B) after a court in an action brought under
paragraph (6) has entered a final judgment in favor of
the Secretary,
the Attorney General shall recover the amount assessed (plus
interest at currently prevailing rates from the date of the
expiration of the 60-day period referred to in paragraph (6) or
the date of such final judgment, as the case may be) in an
action brought in any appropriate district court of the United
States. In such an action, the validity, amount, and
appropriateness of such penalty shall not be subject to review.
(8) If the Secretary finds that a person has committed
repeated violations of section 906(d)(5) or of restrictions
promulgated under section 906(d) at a particular retail outlet
then the Secretary may impose a no-tobacco-sale order on that
person prohibiting the sale of tobacco products in that outlet.
A no-tobacco-sale order may be imposed with a civil penalty
under paragraph (1). Prior to the entry of a no-sale order
under this paragraph, a person shall be entitled to a hearing
pursuant to the procedures established through regulations of
the Food and Drug Administration for assessing civil money
penalties, including at a retailer's request a hearing by
telephone, or at the nearest regional or field office of the
Food and Drug Administration, or at a Federal, State, or county
facility within 100 miles from the location of the retail
outlet, if such a facility is available.
(9) Civil Monetary Penalties for Violation of Tobacco Product
Requirements.--
(A) In general.--Subject to subparagraph (B), any
person who violates a requirement of this Act which
relates to tobacco products shall be liable to the
United States for a civil penalty in an amount not to
exceed $15,000 for each such violation, and not to
exceed $1,000,000 for all such violations adjudicated
in a single proceeding.
(B) Enhanced penalties.--
(i) Any person who intentionally violates a
requirement of section 902(5), 902(6), 904,
908(c), or 911(a), shall be subject to a civil
monetary penalty of--
(I) not to exceed $250,000 per
violation, and not to exceed $1,000,000
for all such violations adjudicated in
a single proceeding; or
(II) in the case of a violation that
continues after the Secretary provides
written notice to such person, $250,000
for the first 30-day period (or any
portion thereof) that the person
continues to be in violation, and such
amount shall double for every 30-day
period thereafter that the violation
continues, not to exceed $1,000,000 for
any 30-day period, and not to exceed
$10,000,000 for all such violations
adjudicated in a single proceeding.
(ii) Any person who violates a requirement of
section 911(g)(2)(C)(ii) or 911(i)(1), shall be
subject to a civil monetary penalty of--
(I) not to exceed $250,000 per
violation, and not to exceed $1,000,000
for all such violations adjudicated in
a single proceeding; or
(II) in the case of a violation that
continues after the Secretary provides
written notice to such person, $250,000
for the first 30-day period (or any
portion thereof) that the person
continues to be in violation, and such
amount shall double for every 30-day
period thereafter that the violation
continues, not to exceed $1,000,000 for
any 30-day period, and not to exceed
$10,000,000 for all such violations
adjudicated in a single proceeding.
(iii) In determining the amount of a civil
penalty under clause (i)(II) or (ii)(II), the
Secretary shall take into consideration whether
the person is making efforts toward correcting
the violation of the requirements of the
section for which such person is subject to
such civil penalty.
(g)(1) With respect to a person who is a holder of an
approved application under section 505 for a drug subject to
section 503(b) or under section 351 of the Public Health
Service Act, any such person who disseminates or causes another
party to disseminate a direct-to-consumer advertisement that is
false or misleading shall be liable to the United States for a
civil penalty in an amount not to exceed $250,000 for the first
such violation in any 3-year period, and not to exceed $500,000
for each subsequent violation in any 3-year period. No other
civil monetary penalties in this Act (including the civil
penalty in section 303(f)(4)) shall apply to a violation
regarding direct-to-consumer advertising. For purposes of this
paragraph: (A) Repeated dissemination of the same or similar
advertisement prior to the receipt of the written notice
referred to in paragraph (2) for such advertisements shall be
considered one violation. (B) On and after the date of the
receipt of such a notice, all violations under this paragraph
occurring in a single day shall be considered one violation.
With respect to advertisements that appear in magazines or
other publications that are published less frequently than
daily, each issue date (whether weekly or monthly) shall be
treated as a single day for the purpose of calculating the
number of violations under this paragraph.
(2) A civil penalty under paragraph (1) shall be assessed by
the Secretary by an order made on the record after providing
written notice to the person to be assessed a civil penalty and
an opportunity for a hearing in accordance with this paragraph
and section 554 of title 5, United States Code. If upon receipt
of the written notice, the person to be assessed a civil
penalty objects and requests a hearing, then in the course of
any investigation related to such hearing, the Secretary may
issue subpoenas requiring the attendance and testimony of
witnesses and the production of evidence that relates to the
matter under investigation, including information pertaining to
the factors described in paragraph (3).
(3) The Secretary, in determining the amount of the civil
penalty under paragraph (1), shall take into account the
nature, circumstances, extent, and gravity of the violation or
violations, including the following factors:
(A) Whether the person submitted the advertisement or
a similar advertisement for review under section 736A.
(B) Whether the person submitted the advertisement
for review if required under section 503B.
(C) Whether, after submission of the advertisement as
described in subparagraph (A) or (B), the person
disseminated or caused another party to disseminate the
advertisement before the end of the 45-day comment
period.
(D) Whether the person incorporated any comments made
by the Secretary with regard to the advertisement into
the advertisement prior to its dissemination.
(E) Whether the person ceased distribution of the
advertisement upon receipt of the written notice
referred to in paragraph (2) for such advertisement.
(F) Whether the person had the advertisement reviewed
by qualified medical, regulatory, and legal reviewers
prior to its dissemination.
(G) Whether the violations were material.
(H) Whether the person who created the advertisement
or caused the advertisement to be created acted in good
faith.
(I) Whether the person who created the advertisement
or caused the advertisement to be created has been
assessed a civil penalty under this provision within
the previous 1-year period.
(J) The scope and extent of any voluntary, subsequent
remedial action by the person.
(K) Such other matters, as justice may require.
(4)(A) Subject to subparagraph (B), no person shall be
required to pay a civil penalty under paragraph (1) if the
person submitted the advertisement to the Secretary and
disseminated or caused another party to disseminate such
advertisement after incorporating each comment received from
the Secretary.
(B) The Secretary may retract or modify any prior comments
the Secretary has provided to an advertisement submitted to the
Secretary based on new information or changed circumstances, so
long as the Secretary provides written notice to the person of
the new views of the Secretary on the advertisement and
provides a reasonable time for modification or correction of
the advertisement prior to seeking any civil penalty under
paragraph (1).
(5) The Secretary may compromise, modify, or remit, with or
without conditions, any civil penalty which may be assessed
under paragraph (1). The amount of such penalty, when finally
determined, or the amount charged upon in compromise, may be
deducted from any sums owed by the United States to the person
charged.
(6) Any person who requested, in accordance with paragraph
(2), a hearing with respect to the assessment of a civil
penalty and who is aggrieved by an order assessing a civil
penalty, may file a petition for de novo judicial review of
such order with the United States Court of Appeals for the
District of Columbia Circuit or for any other circuit in which
such person resides or transacts business. Such a petition may
only be filed within the 60-day period beginning on the date
the order making such assessments was issued.
(7) If any person fails to pay an assessment of a civil
penalty under paragraph (1)--
(A) after the order making the assessment becomes
final, and if such person does not file a petition for
judicial review of the order in accordance with
paragraph (6), or
(B) after a court in an action brought under
paragraph (6) has entered a final judgment in favor of
the Secretary,
the Attorney General of the United States shall recover the
amount assessed (plus interest at currently prevailing rates
from the date of the expiration of the 60-day period referred
to in paragraph (6) or the date of such final judgment, as the
case may be) in an action brought in any appropriate district
court of the United States. In such an action, the validity,
amount, and appropriateness of such penalty shall not be
subject to review.
* * * * * * *
CHAPTER V--DRUGS AND DEVICES
Subchapter A--Drugs and Devices
* * * * * * *
SEC. 505B. RESEARCH INTO PEDIATRIC USES FOR DRUGS AND BIOLOGICAL
PRODUCTS.
(a) New Drugs and Biological Products.--
(1) In general.--
(A) General requirements.--Except with
respect to an application for which
subparagraph (B) applies, a person that
submits, on or after the date of the enactment
of the Pediatric Research Equity Act of 2007,
an application (or supplement to an
application) for a drug--
(i) under section 505 for a new
active ingredient, new indication, new
dosage form, new dosing regimen, or new
route of administration; or
(ii) under section 351 of the Public
Health Service Act (42 U.S.C. 262) for
a new active ingredient, new
indication, new dosage form, new dosing
regimen, or new route of
administration,
shall submit with the application the
assessments described in paragraph (2).
(B) Certain molecularly targeted cancer
indications.--A person that submits, on or
after the date that is 3 years after the date
of enactment of the FDA Reauthorization Act of
2017, an original application for a new active
ingredient under section 505 of this Act or
section 351 of the Public Health Service Act,
shall submit with the application reports on
the investigation described in paragraph (3) if
the drug or biological product that is the
subject of the application is--
(i) intended for the treatment of an
adult cancer; and
(ii) directed at a molecular target
that the Secretary determines to be
substantially relevant to the growth or
progression of a pediatric cancer.
(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).
(2) Assessments.--
(A) In general.--The assessments referred to
in paragraph (1)(A) shall contain data,
gathered using appropriate formulations for
each age group for which the assessment is
required, that are adequate--
(i) to assess the safety and
effectiveness of the drug or the
biological product for the claimed
indications in all relevant pediatric
subpopulations; and
(ii) to support dosing and
administration for each pediatric
subpopulation for which the drug or the
biological product is safe and
effective.
(B) Similar course of disease or similar
effect of drug or biological product.--
(i) In general.--If the course of the
disease and the effects of the drug are
sufficiently similar in adults and
pediatric patients, the Secretary may
conclude that pediatric effectiveness
can be extrapolated from adequate and
well-controlled studies in adults,
usually supplemented with other
information obtained in pediatric
patients, such as pharmacokinetic
studies.
(ii) Extrapolation between age
groups.--A study may not be needed in
each pediatric age group if data from
one age group can be extrapolated to
another age group.
(iii) Information on extrapolation.--
A brief documentation of the scientific
data supporting the conclusion under
clauses (i) and (ii) shall be included
in any pertinent reviews for the
application under section 505 of this
Act or section 351 of the Public Health
Service Act (42 U.S.C. 262).
(3) Molecularly targeted pediatric cancer
investigation.--
[(A) In general.--With respect to a drug or
biological product described in paragraph
(1)(B), the investigation described in this
paragraph is a molecularly targeted pediatric
cancer investigation, which shall be designed
to yield clinically meaningful pediatric study
data, 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.]
(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.
[(B)] (C) Extrapolation of data.--Paragraph
(2)(B) shall apply to [investigations described
in this paragraph] investigations referred to
in subparagraph (A) to the same extent and in
the same manner as paragraph (2)(B) applies
with respect to the assessments required under
paragraph (1)(A).
[(C)] (D) Deferrals and waivers.--Deferrals
and waivers under paragraphs (4) and (5) shall
apply to investigations described in this
paragraph to the same extent and in the same
manner as such deferrals and waivers apply with
respect to [the assessments under paragraph
(2)(B)] the assessments required under
paragraph (1)(A).
(4) Deferral.--
(A) In general.--On the initiative of the
Secretary or at the request of the applicant,
the Secretary may defer submission of some or
all assessments required under paragraph (1)(A)
or reports on the investigation required under
paragraph (1)(B) until a specified date after
approval of the drug or issuance of the license
for a biological product if--
(i) the Secretary finds that--
(I) the drug or biological
product is ready for approval
for use in adults before
pediatric studies are complete;
(II) pediatric studies should
be delayed until additional
safety or effectiveness data
have been collected; or
(III) there is another
appropriate reason for
deferral; and
(ii) the applicant submits to the
Secretary--
(I) certification of the
grounds for deferring the
assessments or reports on the
investigation;
(II) a pediatric study plan
as described in subsection (e);
(III) evidence that the
studies are being conducted or
will be conducted with due
diligence and at the earliest
possible time; and
(IV) a timeline for the
completion of such studies.
(B) Deferral extension.--
(i) In general.--On the initiative of
the Secretary or at the request of the
applicant, the Secretary may grant an
extension of a deferral approved under
subparagraph (A) for submission of some
or all assessments required under
paragraph (1)(A) or reports on the
investigation required under paragraph
(1)(B) if--
(I) the Secretary determines
that the conditions described
in subclause (II) or (III) of
subparagraph (A)(i) continue to
be met; and
(II) the applicant submits a
new timeline under subparagraph
(A)(ii)(IV) and any significant
updates to the information
required under subparagraph
(A)(ii).
(ii) Timing and information.--If the
deferral extension under this
subparagraph is requested by the
applicant, the applicant shall submit
the deferral extension request
containing the information described in
this subparagraph not less than 90 days
prior to the date that the deferral
would expire. The Secretary shall
respond to such request not later than
45 days after the receipt of such
letter. If the Secretary grants such an
extension, the specified date shall be
the extended date. The sponsor of the
required assessment under paragraph
(1)(A) or reports on the investigation
under paragraph (1)(B) shall not be
issued a letter described in subsection
(d) unless the specified or extended
date of submission for such required
studies has passed or if the request
for an extension is pending. For a
deferral that has expired prior to the
date of enactment of the Food and Drug
Administration Safety and Innovation
Act or that will expire prior to 270
days after the date of enactment of
such Act, a deferral extension shall be
requested by an applicant not later
than 180 days after the date of
enactment of such Act. The Secretary
shall respond to any such request as
soon as practicable, but not later than
1 year after the date of enactment of
such Act. Nothing in this clause shall
prevent the Secretary from updating the
status of a study or studies publicly
if components of such study or studies
are late or delayed.
(C) Annual review.--
(i) In general.--On an annual basis
following the approval of a deferral
under subparagraph (A), the applicant
shall submit to the Secretary the
following information:
(I) Information detailing the
progress made in conducting
pediatric studies.
(II) If no progress has been
made in conducting such
studies, evidence and
documentation that such studies
will be conducted with due
diligence and at the earliest
possible time.
(III) Projected completion
date for pediatric studies.
(IV) The reason or reasons
why a deferral or deferral
extension continues to be
necessary.
(ii) Public availability.--Not later
than 90 days after the submission to
the Secretary of the information
submitted through the annual review
under clause (i), the Secretary shall
make available to the public in an
easily accessible manner, including
through the Internet Web site of the
Food and Drug Administration--
(I) such information;
(II) the name of the
applicant for the product
subject to the assessment or
investigation;
(III) the date on which the
product was approved; and
(IV) the date of each
deferral or deferral extension
under this paragraph for the
product.
(5) Waivers.--
(A) Full waiver.--On the initiative of the
Secretary or at the request of an applicant,
the Secretary shall grant a full waiver, as
appropriate, of the requirement to submit
assessments or reports on the investigation for
a drug or biological product under this
subsection if the applicant certifies and the
Secretary finds that--
(i) necessary studies are impossible
or highly impracticable (because, for
example, the number of patients is so
small or the patients are
geographically dispersed);
(ii) there is evidence strongly
suggesting that the drug or biological
product would be ineffective or unsafe
in all pediatric age groups; or
(iii) the drug or biological
product--
(I) does not represent a
meaningful therapeutic benefit
over existing therapies for
pediatric patients; and
(II) is not likely to be used
in a substantial number of
pediatric patients.
(B) Partial waiver.--On the initiative of the
Secretary or at the request of an applicant,
the Secretary shall grant a partial waiver, as
appropriate, of the requirement to submit
assessments or reports on the investigation for
a drug or biological product under this
subsection with respect to a specific pediatric
age group if the applicant certifies and the
Secretary finds that--
(i) necessary studies are impossible
or highly impracticable (because, for
example, the number of patients in that
age group is so small or patients in
that age group are geographically
dispersed);
(ii) there is evidence strongly
suggesting that the drug or biological
product would be ineffective or unsafe
in that age group;
(iii) the drug or biological
product--
(I) does not represent a
meaningful therapeutic benefit
over existing therapies for
pediatric patients in that age
group; and
(II) is not likely to be used
by a substantial number of
pediatric patients in that age
group; or
(iv) the applicant can demonstrate
that reasonable attempts to produce a
pediatric formulation necessary for
that age group have failed.
(C) Pediatric formulation not possible.--If a
partial waiver is granted on the ground that it
is not possible to develop a pediatric
formulation, the waiver shall cover only the
pediatric groups requiring that formulation. An
applicant seeking such a partial waiver shall
submit to the Secretary documentation detailing
why a pediatric formulation cannot be developed
and, if the waiver is granted, the applicant's
submission shall promptly be made available to
the public in an easily accessible manner,
including through posting on the Web site of
the Food and Drug Administration.
(D) Labeling requirement.--If the Secretary
grants a full or partial waiver because there
is evidence that a drug or biological product
would be ineffective or unsafe in pediatric
populations, the information shall be included
in the labeling for the drug or biological
product.
(b) Marketed Drugs and Biological Products.--
(1) In general.--The Secretary may (by order in the
form of a letter) require the sponsor or holder of an
approved application for a drug under section 505 or
the holder of a license for a biological product under
section 351 of the Public Health Service Act to submit
by a specified date the assessments described in
subsection (a)(2), if the Secretary finds that--
(A)(i) the drug or biological product is used
for a substantial number of pediatric patients
for the labeled indications; and
(ii) adequate pediatric labeling could confer
a benefit on pediatric patients;
(B) there is reason to believe that the drug
or biological product would represent a
meaningful therapeutic benefit over existing
therapies for pediatric patients for 1 or more
of the claimed indications; or
(C) the absence of adequate pediatric
labeling could pose a risk to pediatric
patients.
(2) Waivers.--
(A) Full waiver.--At the request of an
applicant, the Secretary shall grant a full
waiver, as appropriate, of the requirement to
submit assessments under this subsection if the
applicant certifies and the Secretary finds
that--
(i) necessary studies are impossible
or highly impracticable (because, for
example, the number of patients in that
age group is so small or patients in
that age group are geographically
dispersed); or
(ii) there is evidence strongly
suggesting that the drug or biological
product would be ineffective or unsafe
in all pediatric age groups.
(B) Partial waiver.--At the request of an
applicant, the Secretary shall grant a partial
waiver, as appropriate, of the requirement to
submit assessments under this subsection with
respect to a specific pediatric age group if
the applicant certifies and the Secretary finds
that--
(i) necessary studies are impossible
or highly impracticable (because, for
example, the number of patients in that
age group is so small or patients in
that age group are geographically
dispersed);
(ii) there is evidence strongly
suggesting that the drug or biological
product would be ineffective or unsafe
in that age group;
(iii)(I) the drug or biological
product--
(aa) does not represent a
meaningful therapeutic benefit
over existing therapies for
pediatric patients in that age
group; and
(bb) is not likely to be used
in a substantial number of
pediatric patients in that age
group; and
(II) the absence of adequate labeling
could not pose significant risks to
pediatric patients; or
(iv) the applicant can demonstrate
that reasonable attempts to produce a
pediatric formulation necessary for
that age group have failed.
(C) Pediatric formulation not possible.--If a
waiver is granted on the ground that it is not
possible to develop a pediatric formulation,
the waiver shall cover only the pediatric
groups requiring that formulation. An applicant
seeking either a full or partial waiver shall
submit to the Secretary documentation detailing
why a pediatric formulation cannot be developed
and, if the waiver is granted, the applicant's
submission shall promptly be made available to
the public in an easily accessible manner,
including through posting on the Web site of
the Food and Drug Administration.
(D) Labeling requirement.--If the Secretary
grants a full or partial waiver because there
is evidence that a drug or biological product
would be ineffective or unsafe in pediatric
populations, the information shall be included
in the labeling for the drug or biological
product.
(3) Effect of subsection.--Nothing in this subsection
alters or amends section 301(j) of this Act or section
552 of title 5 or section 1905 of title 18, United
States Code.
(c) Meaningful Therapeutic Benefit.--For the purposes of
paragraph (4)(A)(iii)(I) and (4)(B)(iii)(I) of subsection (a)
and paragraphs (1)(B) and (2)(B)(iii)(I)(aa) of subsection (b),
a drug or biological product shall be considered to represent a
meaningful therapeutic benefit over existing therapies if the
Secretary determines that--
(1) if approved, the drug or biological product could
represent an improvement in the treatment, diagnosis,
or prevention of a disease, compared with marketed
products adequately labeled for that use in the
relevant pediatric population; or
(2) the drug or biological product is in a class of
products or for an indication for which there is a need
for additional options.
(d) Submission of Assessments And Reports on the
Investigation.--If a person fails to submit a required
assessment described in subsection (a)(2) or the investigation
described in subsection (a)(3), fails to meet the applicable
requirements in subsection (a)(4), or fails to submit a request
for approval of a pediatric formulation described in subsection
(a) or (b), in accordance with applicable provisions of
subsections (a) and (b), the following shall apply:
(1) [Beginning 270] Noncompliance letter._Beginning
270 days after the date of enactment of the Food and
Drug Administration Safety and Innovation Act, the
Secretary shall issue a non-compliance letter to such
person informing them of such failure to submit or meet
the requirements of the applicable subsection. Such
letter shall require the person to respond in writing
within 45 calendar days of issuance of such letter.
Such response may include the person's request for a
deferral extension if applicable. Such letter and the
person's written response to such letter shall be made
publicly available on the Internet Web site of the Food
and Drug Administration 60 calendar days after
issuance, with redactions for any trade secrets and
confidential commercial information. If the Secretary
determines that the letter was issued in error, the
requirements of this paragraph shall not apply. The
Secretary shall inform the Pediatric Advisory Committee
of letters issued under this paragraph and responses to
such letters.
(2) [The drug or] Effect of noncompliance._The drug
or biological product that is the subject of an
assessment described in subsection (a)(2) or the
investigation described in subsection (a)(3),
applicable requirements in subsection (a)(4), or
request for approval of a pediatric formulation, may be
considered misbranded solely because of that failure
and subject to relevant enforcement action [(except
that the drug or biological product shall not be
subject to action under section 303)] (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), but such failure shall not be the basis
for a proceeding--
(A) to withdraw approval for a drug under
section 505(e); or
(B) to revoke the license for a biological
product under section 351 of the Public Health
Service Act.
(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.
(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.
(e) Pediatric Study Plans.--
(1) In general.--An applicant subject to subsection
(a) shall submit to the Secretary an initial pediatric
study plan prior to the submission of the assessments
described under subsection (a)(2) or the investigation
described in subsection (a)(3). 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).
(2) Timing; content; meetings.--
(A) Timing.--An applicant shall submit the
initial pediatric study plan under paragraph
(1)--
(i) before the date on which the
applicant submits the assessments under
subsection (a)(2) or the investigation
described in subsection (a)(3); and
(ii) not later than--
(I) 60 calendar days after
the date of the end-of-Phase 2
meeting (as such term is used
in section 312.47 of title 21,
Code of Federal Regulations, or
successor regulations); or
(II) such other time as may
be agreed upon between the
Secretary and the applicant.
Nothing in this section shall preclude the
Secretary from accepting the submission of an
initial pediatric study plan earlier than the
date otherwise applicable under this
subparagraph.
(B) Content of initial pediatric study
plan.--The initial pediatric study plan shall
include--
(i) an outline of the pediatric study
or studies that the applicant plans to
conduct (including, to the extent
practicable study objectives and
design, age groups, relevant endpoints,
and statistical approach);
(ii) any request for a deferral,
partial waiver, or waiver under this
section, if applicable, along with any
supporting information; and
(iii) other information specified in
the regulations promulgated under
paragraph (7).
(C) Meetings.--The Secretary--
(i) shall meet with the applicant--
(I) if requested by the
applicant with respect to a
drug or biological product that
is intended to treat a serious
or life-threatening disease or
condition, to discuss
preparation of the initial
pediatric study plan, not later
than the end-of-Phase 1 meeting
(as such term is used in
section 312.82(b) of title 21,
Code of Federal Regulations, or
successor regulations) or
within 30 calendar days of
receipt of such request,
whichever is later;
(II) to discuss the initial
pediatric study plan as soon as
practicable, but not later than
90 calendar days after the
receipt of such plan under
subparagraph (A); and
(III) to discuss the bases
for the deferral under
subsection (a)(4) or a full or
partial waiver under subsection
(a)(5);
(ii) may determine that a written
response to the initial pediatric study
plan is sufficient to communicate
comments on the initial pediatric study
plan, and that no meeting under clause
(i)(II) is necessary; and
(iii) if the Secretary determines
that no meeting under clause (i)(II) is
necessary, shall so notify the
applicant and provide written comments
of the Secretary as soon as
practicable, but not later than 90
calendar days after the receipt of the
initial pediatric study plan.
(3) Agreed initial pediatric study plan.--Not later
than 90 calendar days following the meeting under
paragraph (2)(C)(i)(II) or the receipt of a written
response from the Secretary under paragraph
(2)(C)(iii), the applicant shall document agreement on
the initial pediatric study plan in a submission to the
Secretary marked ``Agreed Initial Pediatric Study
Plan'', and the Secretary shall confirm such agreement
to the applicant in writing not later than 30 calendar
days of receipt of such agreed initial pediatric study
plan.
(4) Deferral and waiver.--If the agreed initial
pediatric study plan contains a request from the
applicant for a deferral, partial waiver, or waiver
under this section, the written confirmation under
paragraph (3) shall include a recommendation from the
Secretary as to whether such request meets the
standards under paragraphs (3) or (4) of subsection
(a).
(5) Amendments to the agreed initial pediatric study
plan.--At the initiative of the Secretary or the
applicant, the agreed initial pediatric study plan may
be amended at any time. The requirements of paragraph
(2)(C) shall apply to any such proposed amendment in
the same manner and to the same extent as such
requirements apply to an initial pediatric study plan
under paragraph (1). The requirements of paragraphs (3)
and (4) shall apply to any agreement resulting from
such proposed amendment in the same manner and to the
same extent as such requirements apply to an agreed
initial pediatric study plan.
(6) Internal committee.--The Secretary shall consult
the internal committee under section 505C on the review
of the initial pediatric study plan, agreed initial
pediatric study plan, and any significant amendments to
such plans.
(7) Required rulemaking.--Not later than 1 year after
the date of enactment of the Food and Drug
Administration Safety and Innovation Act, the Secretary
shall promulgate proposed regulations and issue
guidance to implement the provisions of this
subsection.
(f) Review of Pediatric Study Plans,Assessments, Deferrals,
Deferral Extensions, and Waivers.--
(1) Review.--Beginning not later than 30 days after
the date of the enactment of the Pediatric Research
Equity Act of 2007, the Secretary shall utilize the
internal committee established under section 505C to
provide consultation to reviewing divisions on initial
pediatric study plans, agreed initial pediatric study
plans, and any significant amendments to such plans,
and assessments prior to approval of an application or
supplement for which a pediatric assessment is required
under this section and all deferral, deferral
extension, and waiver requests granted pursuant to this
section.
(2) Activity by committee.--The committee referred to
in paragraph (1) may operate using appropriate members
of such committee and need not convene all members of
the committee.
(3) Documentation of committee action.--For each drug
or biological product, the committee referred to in
paragraph (1) shall document, for each activity
described in paragraph (4) or (5), which members of the
committee participated in such activity.
(4) Review of pediatric study plans, assessments,
deferrals, deferral extensions, and waivers.--
Consultation on initial pediatric study plans, agreed
initial pediatric study plans, and assessments by the
committee referred to in paragraph (1) pursuant to this
section shall occur prior to approval of an application
or supplement for which a pediatric assessment is
required under this section. The committee shall review
all requests for deferrals, deferral extensions, and
waivers from the requirement to submit a pediatric
assessment granted under this section and shall provide
recommendations as needed to reviewing divisions,
including with respect to whether such a supplement,
when submitted, shall be considered for priority
review.
(5) Retrospective review of pediatric assessments,
deferrals, and waivers.--Not later than 1 year after
the date of the enactment of the Pediatric Research
Equity Act of 2007, the committee referred to in
paragraph (1) shall conduct a retrospective review and
analysis of a representative sample of assessments
submitted and deferrals and waivers approved under this
section since the enactment of the Pediatric Research
Equity Act of 2003. Such review shall include an
analysis of the quality and consistency of pediatric
information in pediatric assessments and the
appropriateness of waivers and deferrals granted. Based
on such review, the Secretary shall issue
recommendations to the review divisions for
improvements and initiate guidance to industry related
to the scope of pediatric studies required under this
section.
(6) Tracking of assessments and labeling changes.--
The Secretary, in consultation with the committee
referred to in paragraph (1), shall track and make
available to the public in an easily accessible manner,
including through posting on the Web site of the Food
and Drug Administration--
(A) the number of assessments conducted under
this section;
(B) the specific drugs and biological
products and their uses assessed under this
section;
(C) the types of assessments conducted under
this section, including trial design, the
number of pediatric patients studied, and the
number of centers and countries involved;
(D) aggregated on an annual basis--
(i) the total number of deferrals and
deferral extensions requested and
granted under this section and, if
granted, the reasons for each such
deferral or deferral extension;
(ii) the timeline for completion of
the assessments;
(iii) the number of assessments
completed and pending; and
(iv) the number of postmarket non-
compliance letters issued pursuant to
subsection (d), and the recipients of
such letters;
(E) the number of waivers requested and
granted under this section and, if granted, the
reasons for the waivers;
(F) the number of pediatric formulations
developed and the number of pediatric
formulations not developed and the reasons any
such formulation was not developed;
(G) the labeling changes made as a result of
assessments conducted under this section;
(H) an annual summary of labeling changes
made as a result of assessments conducted under
this section for distribution pursuant to
subsection (h)(2);
(I) an annual summary of information
submitted pursuant to subsection (a)(4)(C); and
(J) the number of times the committee
referred to in paragraph (1) made a
recommendation to the Secretary under paragraph
(4) regarding priority review, the number of
times the Secretary followed or did not follow
such a recommendation, and, if not followed,
the reasons why such a recommendation was not
followed.
(g) Labeling Changes.--
(1) Dispute resolution.--
(A) Request for labeling change and failure
to agree.--If, on or after the date of the
enactment of the Pediatric Research Equity Act
of 2007, the Commissioner determines that a
sponsor and the Commissioner have been unable
to reach agreement on appropriate changes to
the labeling for the drug that is the subject
of the application or supplement, not later
than 180 days after the date of the submission
of the application or supplement that receives
a priority review or 330 days after the date of
the submission of an application or supplement
that receives a standard review--
(i) the Commissioner shall request
that the sponsor of the application
make any labeling change that the
Commissioner determines to be
appropriate; and
(ii) if the sponsor does not agree
within 30 days after the Commissioner's
request to make a labeling change
requested by the Commissioner, the
Commissioner shall refer the matter to
the Pediatric Advisory Committee.
(B) Action by the pediatric advisory
committee.--Not later than 90 days after
receiving a referral under subparagraph
(A)(ii), the Pediatric Advisory Committee
shall--
(i) review the pediatric study
reports; and
(ii) make a recommendation to the
Commissioner concerning appropriate
labeling changes, if any.
(C) Consideration of recommendations.--The
Commissioner shall consider the recommendations
of the Pediatric Advisory Committee and, if
appropriate, not later than 30 days after
receiving the recommendation, make a request to
the sponsor of the application or supplement to
make any labeling changes that the Commissioner
determines to be appropriate.
(D) Misbranding.--If the sponsor of the
application or supplement, within 30 days after
receiving a request under subparagraph (C),
does not agree to make a labeling change
requested by the Commissioner, the Commissioner
may deem the drug that is the subject of the
application or supplement to be misbranded.
(E) No effect on authority.--Nothing in this
subsection limits the authority of the United
States to bring an enforcement action under
this Act when a drug lacks appropriate
pediatric labeling. Neither course of action
(the Pediatric Advisory Committee process or an
enforcement action referred to in the preceding
sentence) shall preclude, delay, or serve as
the basis to stay the other course of action.
(2) Other labeling changes.--If, on or after the date
of the enactment of the Pediatric Research Equity Act
of 2007, the Secretary makes a determination that a
pediatric assessment conducted under this section does
or does not demonstrate that the drug that is the
subject of such assessment is safe and effective in
pediatric populations or subpopulations, including
whether such assessment results are inconclusive, the
Secretary shall order the labeling of such product to
include information about the results of the assessment
and a statement of the Secretary's determination.
(h) Dissemination of Pediatric Information.--
(1) In general.--Not later than 210 days after the
date of submission of an application (or supplement to
an application) that contains a pediatric assessment
under this section, if the application (or supplement)
receives a priority review, or not later than 330 days
after the date of submission of an application (or
supplement to an application) that contains a pediatric
assessment under this section, if the application (or
supplement) receives a standard review, the Secretary
shall make available to the public in an easily
accessible manner the medical, statistical, and
clinical pharmacology reviews of such pediatric
assessments, and shall post such assessments on the Web
site of the Food and Drug Administration.
(2) Dissemination of information regarding labeling
changes.--Beginning on the date of the enactment of the
Pediatric Research Equity Act of 2007, the Secretary
shall require that the sponsors of the assessments that
result in labeling changes that are reflected in the
annual summary developed pursuant to subsection
(f)(6)(H) distribute such information to physicians and
other health care providers.
(3) Effect of subsection.--Nothing in this subsection
shall alter or amend section 301(j) of this Act or
section 552 of title 5 or section 1905 of title 18,
United States Code.
(i) Adverse Event Reporting.--
(1) Reporting in first 18-month period.--Beginning on
the date of the enactment of the Pediatric Research
Equity Act of 2007, during the 18-month period
beginning on the date a labeling change is made
pursuant to subsection (g), the Secretary shall ensure
that all adverse event reports that have been received
for such drug (regardless of when such report was
received) are referred to the Office of Pediatric
Therapeutics. In considering such reports, the Director
of such Office shall provide for the review of such
reports by the Pediatric Advisory Committee, including
obtaining any recommendations of such committee
regarding whether the Secretary should take action
under this Act in response to such reports.
(2) Reporting in subsequent periods.--Following the
18-month period described in paragraph (1), the
Secretary shall, as appropriate, refer to the Office of
Pediatric Therapeutics all pediatric adverse event
reports for a drug for which a pediatric study was
conducted under this section. In considering such
reports, the Director of such Office may provide for
the review of such reports by the Pediatric Advisory
Committee, including obtaining any recommendation of
such Committee regarding whether the Secretary should
take action in response to such reports.
(3) Preservation of authority.--Nothing in this
subsection shall prohibit the Office of Pediatric
Therapeutics from providing for the review of adverse
event reports by the Pediatric Advisory Committee prior
to the 18-month period referred to in paragraph (1), if
such review is necessary to ensure safe use of a drug
in a pediatric population.
(4) Effect.--The requirements of this subsection
shall supplement, not supplant, other review of such
adverse event reports by the Secretary.
(j) Scope of Authority.--Nothing in this section provides to
the Secretary any authority to require a pediatric assessment
of any drug or biological product, or any assessment regarding
other populations or uses of a drug or biological product,
other than the pediatric assessments described in this section.
(k) Relation to Orphan Drugs.--
(1) In general; exemption for orphan indications.--
Unless the Secretary requires otherwise by regulation
and except as provided in paragraph (2), this section
does not apply to any drug or biological product for an
indication for which orphan designation has been
granted under section 526.
(2) Applicability despite orphan designation of
certain indications.--This section shall apply with
respect to a drug or biological product for which an
indication has been granted orphan designation under
526 if the investigation described in subsection (a)(3)
applies to the drug or biological product as described
in subsection (a)(1)(B).
(l) New Active Ingredient.--
(1) Non-interchangeable biosimilar biological
product.--A biological product that is biosimilar to a
reference product under section 351 of the Public
Health Service Act, and that the Secretary has not
determined to meet the standards described in
subsection (k)(4) of such section for
interchangeability with the reference product, shall be
considered to have a new active ingredient under this
section.
(2) Interchangeable biosimilar biological product.--A
biological product that is interchangeable with a
reference product under section 351 of the Public
Health Service Act shall not be considered to have a
new active ingredient under this section.
(m) List of Primary Molecular Targets.--
(1) In general.--Within one year of the date of
enactment of the FDA Reauthorization Act of 2017, the
Secretary shall establish and update regularly, and
shall publish on the internet website of the Food and
Drug Administration--
(A) a list of molecular targets considered,
on the basis of data the Secretary determines
to be adequate, to be substantially relevant to
the growth and progression of a pediatric
cancer, and that may trigger the requirements
under this section; and
(B) a list of molecular targets of new cancer
drugs and biological products in development
for which pediatric cancer study requirements
under this section will be automatically
waived.
(2) Consultation.--In establishing the lists
described in paragraph (1), the Secretary shall consult
the National Cancer Institute, members of the internal
committee under section 505C, and the Pediatric
Oncology Subcommittee of the Oncologic Drugs Advisory
Committee, and shall take into account comments from
the meeting under subsection (c).
(3) Rule of construction.--Nothing in paragraph (1)
shall be construed--
(A) to require the inclusion of a molecular
target on the list published under such
paragraph as a condition for triggering the
requirements under subsection (a)(1)(B) with
respect to a drug or biological product
directed at such molecular target; or
(B) to authorize the disclosure of
confidential commercial information, as
prohibited under section 301(j) of this Act or
section 1905 of title 18, United States Code.
* * * * * * *
Subchapter B--Drugs for Rare Diseases or Conditions
* * * * * * *
protection for drugs for rare diseases or conditions
Sec. 527. (a) Except as provided in subsection (b), if the
Secretary--
(1) approves an application filed pursuant to section
505, or
(2) issues a license under section 351 of the Public
Health Service Act
for a drug designated under section 526 for a rare disease or
condition, the Secretary may not approve another application
under section 505 or issue another license under section 351 of
the Public Health Service Act for the same drug for the [same
disease or condition] same approved use or indication within
such rare disease or condition for a person who is not the
holder of such approved application or of such license until
the expiration of seven years from the date of the approval of
the approved application or the issuance of the license.
Section 505(c)(2) does not apply to the refusal to approve an
application under the preceding sentence.
(b) During the 7-year period described in subsection (a) for
an approved application under section 505 or license under
section 351 of the Public Health Service Act, the Secretary may
approve an application or issue a license for a drug that is
otherwise the same, as determined by the Secretary, as the
already approved drug for the [same rare disease or condition]
same approved use or indication for which such 7-year period
applies to such already approved or licensed drug if--
(1) the Secretary finds, after providing the holder
of exclusive approval or licensure notice and
opportunity for the submission of views, that during
such period the holder of the exclusive approval or
licensure cannot ensure the availability of sufficient
quantities of the drug to meet the needs, relating to
the approved use or indication, of persons with the
disease or condition for which the drug was designated;
or
(2) the holder provides the Secretary in writing the
consent of such holder for the approval of other
applications or the issuance of other licenses before
the expiration of such seven-year period.
(c) Condition of Clinical Superiority.--
(1) In general.--If a sponsor of a drug that is
designated under section 526 and is otherwise the same,
as determined by the Secretary, as an already approved
or licensed drug is seeking exclusive approval or
exclusive licensure described in subsection (a) for the
[same rare disease or condition as the already approved
drug] same use or indication for which the already
approved or licensed drug was approved or licensed, the
Secretary shall require such sponsor, as a condition of
such exclusive approval or licensure, to demonstrate
that such drug is clinically superior to any already
approved or licensed drug that is the same drug.
(2) Definition.--For purposes of paragraph (1), the
term ``clinically superior'' with respect to a drug
means that the drug provides a significant therapeutic
advantage over and above an already approved or
licensed drug in terms of greater efficacy, greater
safety, or by providing a major contribution to patient
care.
(3) Applicability.--This subsection applies to any
drug designated under section 526 for which an
application was approved under section 505 of this Act
or licensed under section 351 of the Public Health
Service Act after the date of enactment of the FDA
Reauthorization Act of 2017, regardless of the date on
which such drug was designated under section 526.
(d) Regulations.--The Secretary may promulgate regulations
for the implementation of subsection (c). Beginning on the date
of enactment of the FDA Reauthorization Act of 2017, until such
time as the Secretary promulgates regulations in accordance
with this subsection, the Secretary may apply any definitions
set forth in regulations that were promulgated prior to such
date of enactment, to the extent such definitions are not
inconsistent with the terms of this section, as amended by such
Act.
(e) Demonstration of Clinical Superiority Standard.--To
assist sponsors in demonstrating clinical superiority as
described in subsection (c), the Secretary--
(1) upon the designation of any drug under section
526, shall notify the sponsor of such drug in writing
of the basis for the designation, including, as
applicable, any plausible hypothesis offered by the
sponsor and relied upon by the Secretary that the drug
is clinically superior to a previously approved drug;
and
(2) upon granting exclusive approval or licensure
under subsection (a) on the basis of a demonstration of
clinical superiority as described in subsection (c),
shall publish a summary of the clinical superiority
findings.
(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.
* * * * * * *
SEC. 529. PRIORITY REVIEW TO ENCOURAGE TREATMENTS FOR RARE PEDIATRIC
DISEASES.
(a) Definitions.--In this section:
(1) Priority review.--The term ``priority review'',
with respect to a human drug application as defined in
section 735(1), means review and action by the
Secretary on such application not later than 6 months
after receipt by the Secretary of such application, as
described in the Manual of Policies and Procedures of
the Food and Drug Administration and goals identified
in the letters described in section 101(b) of the
Prescription Drug User Fee Amendments of 2012.
(2) Priority review voucher.--The term ``priority
review voucher'' means a voucher issued by the
Secretary to the sponsor of a rare pediatric disease
product application that entitles the holder of such
voucher to priority review of a single human drug
application submitted under section 505(b)(1) or
section 351(a) of the Public Health Service Act after
the date of approval of the rare pediatric disease
product application.
(3) Rare pediatric disease.--The term ``rare
pediatric disease'' means a disease that meets each of
the following criteria:
(A) The disease is a serious or life-
threatening disease in which the serious or
life-threatening manifestations primarily
affect individuals aged from birth to 18 years,
including age groups often called neonates,
infants, children, and adolescents.
(B) The disease is a rare disease or
condition, within the meaning of section 526.
(4) Rare pediatric disease product application.--The
term ``rare pediatric disease product application''
means a human drug application, as defined in section
735(1), that--
(A) is for a drug or biological product that
is for the prevention or treatment of a rare
pediatric disease;
(B)(i) is for such a drug--
(I) that contains no active moiety
(as defined by the Secretary in section
314.3 of title 21, Code of Federal
Regulations (or any successor
regulations)) that has been previously
approved in any other application under
subsection (b)(1), (b)(2), or (j) of
section 505; and
(II) that is the subject of an
application submitted under section
505(b)(1); or
(ii) is for such a biological product--
(I) that contains no active
ingredient that has been previously
approved in any other application under
section 351(a) or 351(k) of the Public
Health Service Act; and
(II) that is the subject of an
application submitted under section
351(a) of the Public Health Service
Act;
(C) the Secretary deems eligible for priority
review;
(D) that relies on clinical data derived from
studies examining a pediatric population and
dosages of the drug intended for that
population;
(E) that does not seek approval for an adult
indication in the original rare pediatric
disease product application; and
(F) is approved after the date of the
enactment of the Advancing Hope Act of 2016.
(b) Priority Review Voucher.--
(1) In general.--The Secretary shall award a priority
review voucher to the sponsor of a rare pediatric
disease product application upon approval by the
Secretary of such rare pediatric disease product
application.
(2) Transferability.--
(A) In general.--The sponsor of a rare
pediatric disease product application that
receives a priority review voucher under this
section may transfer (including by sale) the
entitlement to such voucher. There is no limit
on the number of times a priority review
voucher may be transferred before such voucher
is used.
(B) Notification of transfer.--Each person to
whom a voucher is transferred shall notify the
Secretary of such change in ownership of the
voucher not later than 30 days after such
transfer.
(3) Limitation.--A sponsor of a rare pediatric
disease product application may not receive a priority
review voucher under this section if the rare pediatric
disease product application was submitted to the
Secretary prior to the date that is 90 days after the
date of enactment of the Prescription Drug User Fee
Amendments of 2012.
(4) Notification.--
(A) Sponsor of a rare pediatric disease
product.--
(i) In general.--Beginning on the
date that is 90 days after the date of
enactment of the Advancing Hope Act of
2016, the sponsor of a rare pediatric
disease product application that
intends to request a priority review
voucher under this section shall notify
the Secretary of such intent upon
submission of the rare pediatric
disease product application that is the
basis of the request for a priority
review voucher.
(ii) Applications submitted but not
yet approved.--The sponsor of a rare
pediatric disease product application
that was submitted and that has not
been approved as of the date of
enactment of the Advancing Hope Act of
2016 shall be considered eligible for a
priority review voucher, if--
(I) such sponsor has
submitted such rare pediatric
disease product application--
(aa) on or after the
date that is 90 days
after the date of
enactment of the
Prescription Drug User
Fee Amendments of 2012;
and
(bb) on or before the
date of enactment of
the Advancing Hope Act
of 2016; and
(II) such application
otherwise meets the criteria
for a priority review voucher
under this section.
(B) Sponsor of a drug application using a
priority review voucher.--
(i) In general.--The sponsor of a
human drug application shall notify the
Secretary not later than 90 days prior
to submission of the human drug
application that is the subject of a
priority review voucher of an intent to
submit the human drug application,
including the date on which the sponsor
intends to submit the application. Such
notification shall be a legally binding
commitment to pay the user fee to be
assessed in accordance with this
section.
(ii) Transfer after notice.--The
sponsor of a human drug application
that provides notification of the
intent of such sponsor to use the
voucher for the human drug application
under clause (i) may transfer the
voucher after such notification is
provided, if such sponsor has not yet
submitted the human drug application
described in the notification.
(5) Termination of authority.--The Secretary may not
award any priority review vouchers under paragraph (1)
after [September 30, 2024, unless the rare pediatric
disease product application--] September 30, 2029.
[(A) is for a drug that, not later than
September 30, 2024, is designated under
subsection (d) as a drug for a rare pediatric
disease; and
[(B) is, not later than September 30, 2026,
approved under section 505(b)(1) of this Act or
section 351(a) of the Public Health Service
Act.]
(c) Priority Review User Fee.--
(1) In general.--The Secretary shall establish a user
fee program under which a sponsor of a human drug
application that is the subject of a priority review
voucher shall pay to the Secretary a fee determined
under paragraph (2). Such fee shall be in addition to
any fee required to be submitted by the sponsor under
chapter VII.
(2) Fee amount.--The amount of the priority review
user fee shall be determined each fiscal year by the
Secretary, based on the difference between--
(A) the average cost incurred by the Food and
Drug Administration in the review of a human
drug application subject to priority review in
the previous fiscal year; and
(B) the average cost incurred by the Food and
Drug Administration in the review of a human
drug application that is not subject to
priority review in the previous fiscal year.
(3) Annual fee setting.--The Secretary shall
establish, before the beginning of each fiscal year
beginning after September 30, 2012, the amount of the
priority review user fee for that fiscal year.
(4) Payment.--
(A) In general.--The priority review user fee
required by this subsection shall be due upon
the notification by a sponsor of the intent of
such sponsor to use the voucher, as specified
in subsection (b)(4)(A). All other user fees
associated with the human drug application
shall be due as required by the Secretary or
under applicable law.
(B) Complete application.--An application
described under subparagraph (A) for which the
sponsor requests the use of a priority review
voucher shall be considered incomplete if the
fee required by this subsection and all other
applicable user fees are not paid in accordance
with the Secretary's procedures for paying such
fees.
(C) No waivers, exemptions, reductions, or
refunds.--The Secretary may not grant a waiver,
exemption, reduction, or refund of any fees due
and payable under this section.
(5) Offsetting collections.--Fees collected pursuant
to this subsection for any fiscal year--
(A) shall be deposited and credited as
offsetting collections to the account providing
appropriations to the Food and Drug
Administration; and
(B) shall not be collected for any fiscal
year except to the extent provided in advance
in appropriations Acts.
(d) Designation Process.--
(1) In general.--Upon the request of the manufacturer
or the sponsor of a new drug, the Secretary may
designate--
(A) the new drug as a drug for a rare
pediatric disease; and
(B) the application for the new drug as a
rare pediatric disease product application.
(2) Request for designation.--The request for a
designation under paragraph (1) shall be made at the
same time a request for designation of orphan disease
status under section 526 or fast-track designation
under section 506 is made. Requesting designation under
this subsection is not a prerequisite to receiving a
priority review voucher under this section.
(3) Determination by secretary.--Not later than 60
days after a request is submitted under paragraph (1),
the Secretary shall determine whether--
(A) the disease or condition that is the
subject of such request is a rare pediatric
disease; and
(B) the application for the new drug is a
rare pediatric disease product application.
(e) Marketing of Rare Pediatric Disease Products.--
(1) Revocation.--The Secretary may revoke any
priority review voucher awarded under subsection (b) if
the rare pediatric disease product for which such
voucher was awarded is not marketed in the United
States within the 365-day period beginning on the date
of the approval of such drug under section 505 of this
Act or section 351 of the Public Health Service Act.
(2) Postapproval production report.--The sponsor of
an approved rare pediatric disease product shall submit
a report to the Secretary not later than 5 years after
the approval of the applicable rare pediatric disease
product application. Such report shall provide the
following information, with respect to each of the
first 4 years after approval of such product:
(A) The estimated population in the United
States suffering from the rare pediatric
disease.
(B) The estimated demand in the United States
for such rare pediatric disease product.
(C) The actual amount of such rare pediatric
disease product distributed in the United
States.
(f) Notice and Report.--
(1) Notice of issuance of voucher and approval of
products under voucher.--The Secretary shall publish a
notice in the Federal Register and on the Internet Web
site of the Food and Drug Administration not later than
30 days after the occurrence of each of the following:
(A) The Secretary issues a priority review
voucher under this section.
(B) The Secretary approves a drug pursuant to
an application submitted under section 505(b)
of this Act or section 351(a) of the Public
Health Service Act for which the sponsor of the
application used a priority review voucher
under this section.
(2) Notification.--If, after the last day of the 1-
year period that begins on the date that the Secretary
awards the third rare pediatric disease priority
voucher under this section, a sponsor of an application
submitted under section 505(b) of this Act or section
351(a) of the Public Health Service Act for a drug uses
a priority review voucher under this section for such
application, the Secretary 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 document--
(A) notifying such Committees of the use of
such voucher; and
(B) identifying the drug for which such
priority review voucher is used.
(g) Eligibility for Other Programs.--Nothing in this section
precludes a sponsor who seeks a priority review voucher under
this section from participating in any other incentive program,
including under this Act, except that no sponsor of a rare
pediatric disease product application may receive more than one
priority review voucher issued under any section of this Act
with respect to the drug for which the application is made..
(h) Relation to Other Provisions.--The provisions of this
section shall supplement, not supplant, any other provisions of
this Act or the Public Health Service Act that encourage the
development of drugs for tropical diseases and rare pediatric
diseases.
(i) GAO Study and Report.--
(1) Study.--
(A) In general.--Beginning on the date that
the Secretary awards the third rare pediatric
disease priority voucher under this section,
the Comptroller General of the United States
shall conduct a study of the effectiveness of
awarding rare pediatric disease priority
vouchers under this section in the development
of human drug products that treat or prevent
such diseases.
(B) Contents of study.--In conducting the
study under subparagraph (A), the Comptroller
General shall examine the following:
(i) The indications for which each
rare disease product for which a
priority review voucher was awarded was
approved under section 505 or section
351 of the Public Health Service Act.
(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 of such a
rare disease product.
(iii) The value of the priority
review voucher if transferred.
(iv) Identification of each drug for
which a priority review voucher was
used.
(v) The length of the period of time
between the date on which a priority
review voucher was awarded and the date
on which it was used.
(2) Report.--Not later than 1 year after the date
under paragraph (1)(A), 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 results of the study under paragraph
(1).
* * * * * * *
CHAPTER X--MISCELLANEOUS
* * * * * * *
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.
----------
FOOD AND DRUG ADMINISTRATION SAFETY AND INNOVATION ACT
* * * * * * *
TITLE V--PEDIATRIC DRUGS AND DEVICES
* * * * * * *
SEC. 508. REPORT.
(a) In General.--Not later than four years after the date of
enactment of this Act and every five years thereafter, the
Secretary shall prepare and submit to the Committee on Health,
Education, Labor, and Pensions of the Senate and the Committee
on Energy and Commerce of the House of Representatives, and
make publicly available, including through posting on the
Internet Web site of the Food and Drug Administration, a report
on the implementation of sections 505A and 505B of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C. 355a, 355c).
(b) Contents.--Each report under subsection (a) shall
include--
(1) an assessment of the effectiveness of sections
505A and 505B of the Federal Food, Drug, and Cosmetic
Act in improving information about pediatric uses for
approved drugs and biological products, including the
number and type of labeling changes made since the date
of enactment of this Act and the importance of such
uses in the improvement of the health of children;
(2) the number of required studies under such section
505B that have not met the initial deadline provided
under such section 505B, including--
(A) the number of deferrals and deferral
extensions granted and the reasons such
extensions were granted;
(B) the number of waivers and partial waivers
granted; and
(C) the number of letters issued under
subsection (d) of such section 505B;
(3) an assessment of the timeliness and effectiveness
of pediatric study planning since the date of enactment
of this Act, including the number of initial pediatric
study plans not submitted in accordance with the
requirements of subsection (e) of such section 505B and
any resulting rulemaking;
(4) the number of written requests issued, accepted,
and declined under such section 505A since the date of
enactment of this Act, and a listing of any important
gaps in pediatric information as a result of such
declined requests;
(5) a description and current status of referrals
made under subsection (n) of such section 505A;
(6) an assessment of the effectiveness of studying
biological products in pediatric populations under such
sections 505A and 505B and section 409I of the Public
Health Service Act (42 U.S.C. 284m);
(7)(A) the efforts made by the Secretary to increase
the number of studies conducted in the neonatal
population (including efforts made to encourage the
conduct of appropriate studies in neonates by companies
with products that have sufficient safety and other
information to make the conduct of the studies ethical
and safe); and
(B) the results of such efforts;
(8)(A) the number and importance of drugs and
biological products for children with cancer that are
being tested as a result of the programs under such
sections 505A and 505B and under section 409I of the
Public Health Service Act; and
(B) any recommendations for modifications to such
programs that would lead to new and better therapies
for children with cancer, including a detailed
rationale for each recommendation;
(9) any recommendations for modification to such
programs that would improve pediatric drug research and
increase pediatric labeling of drugs and biological
products;
(10) an assessment of the successes of and
limitations to studying drugs for rare diseases under
such sections 505A and 505B;
(11) an assessment of the impact of the amendments to
such section 505B made by the FDA Reauthorization Act
of 2017 on pediatric research and labeling of drugs and
biological products and pediatric labeling of
molecularly targeted drugs and biological products for
the treatment of cancer[;], including an evaluation of
compliance with deadlines provided for in deferrals and
deferral extensions;
(12) an assessment of the efforts of the Secretary to
implement the plan developed under section 505C-1 of
the Federal Food, Drug, and Cosmetic Act, regarding
earlier submission of pediatric studies under sections
505A and 505B of such Act and section 351(m) of the
Public Health Service Act, including--
(A) the average length of time after the
approval of an application under section
505(b)(1) of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 355(b)(1)) or section
351(a) of the Public Health Service Act (42
U.S.C. 262(a)) before studies conducted
pursuant to such section 505A, 505B, or section
351(m) are completed, submitted, and
incorporated into labeling;
(B) the average length of time after the
receipt of a proposed pediatric study request
before the Secretary responds to such request;
(C) the average length of time after the
submission of a proposed pediatric study
request before the Secretary issues a written
request for such studies;
(D) the number of written requests issued for
each investigational new drug or biological
product prior to the submission of an
application under section 505(b)(1) of the
Federal Food, Drug, and Cosmetic Act or section
351(a) of the Public Health Service Act; and
(E) the average number, and range of numbers,
of amendments to written requests issued, and
the time the Secretary requires to review and
act on proposed amendments to written requests;
(13) a list of sponsors of applications or holders of
approved applications who received exclusivity under
such section 505A or such section 351(m) after
receiving a letter issued under such section 505B(d)(1)
for any drug or biological product before the studies
referred to in such letter were completed and
submitted;
(14) a list of assessments and investigations
required under such section 505B;
(15) how many requests under such section 505A for
molecular targeted cancer drugs, as defined by
subsection (a)(1)(B) of such section 505B, approved
prior to 3 years after the date of enactment of the FDA
Reauthorization Act of 2017, have been issued by the
Food and Drug Administration, and how many such
requests have been completed; [and]
(16) the Secretary's assessment of the overall impact
of the amendments made by section 504 of the FDA
Reauthorization Act of 2017 on the conduct and
effectiveness of pediatric cancer research and the
orphan drug program, as well any subsequent
recommendations[.]; and
(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
(c) Stakeholder Comment.--At least 180 days prior to the
submission of each report under subsection (a), the Secretary
shall consult with representatives of patient groups (including
pediatric patient groups), consumer groups, regulated industry,
academia, and other interested parties to obtain any
recommendations or information relevant to the report including
suggestions for modifications that would improve pediatric drug
research and pediatric labeling of drugs and biological
products.
* * * * * * *
----------
PUBLIC HEALTH SERVICE ACT
TITLE III--GENERAL POWERS AND DUTIES OF PUBLIC HEALTH SERVICE
* * * * * * *
Part H--Organ Transplants
* * * * * * *
organ procurement and transplantation network
Sec. 372. (a) In General--The Secretary shall provide for the
continued operation of an Organ Procurement and Transplantation
Network which meets the requirements of subsection (b). The
Secretary may award grants, contracts, or cooperative
agreements, as the Secretary determines appropriate, for
purposes of carrying out this section.
(b) Composition.--
(1) In general.--The Organ Procurement and
Transplantation Network shall--
(A) be operated through awards to public or
private entities made by the Secretary that are
distinct from the awards made to support the
organization tasked with supporting the board
of directors described in subparagraph (B); and
(B) have a board of directors--
(i) that includes representatives of organ
procurement organizations (including
organizations that have received grants under
section 371), transplant centers, voluntary
health associations, and the general public;
and
(ii) that shall establish an executive
committee and other committees, whose
chairpersons shall be selected to ensure
continuity of leadership for the board.
(2) The Organ Procurement and Transplantation Network shall--
(A) establish in one location or through regional
centers--
(i) a national list of individuals who need
organs, and
(ii) a national system, through the use of
computers and in accordance with established
medical criteria, to match organs and
individuals included in the list, especially
individuals whose immune system makes it
difficult for them to receive organs,
(B) establish membership criteria and medical
criteria for allocating organs and provide to members
of the public an opportunity to comment with respect to
such criteria,
(C) maintain a twenty-four-hour telephone service to
facilitate matching organs with individuals included in
the list,
(D) assist organ procurement organizations in the
nationwide distribution of organs equitably among
transplant patients,
(E) adopt and use standards of quality for the
acquisition and transportation of donated organs,
(F) prepare and distribute, on a regionalized basis
(and, to the extent practicable, among regions or on a
national basis), samples of blood sera from individuals
who are included on the list and whose immune system
makes it difficult for them to receive organs, in order
to facilitate matching the compatibility of such
individuals with organ donors,
(G) coordinate, as appropriate, the transportation of
organs from organ procurement organizations to
transplant centers,
(H) provide information to physicians and other
health professionals regarding organ donation,
(I) collect, analyze, and publish data concerning
organ donation and transplants,
(J) carry out studies and demonstration projects for
the purpose of improving procedures for organ
procurement and allocation,
(K) work actively to increase the supply of donated
organs,
(L) submit to the Secretary an annual report
containing information on the comparative costs and
patient outcomes at each transplant center affiliated
with the organ procurement and transplantation network,
(M) recognize the differences in health and in organ
transplantation issues between children and adults
throughout the system and adopt criteria, polices, and
procedures that address the unique health care needs of
children,
(N) carry out studies and demonstration projects for
the purpose of improving procedures for organ donation
procurement and allocation, including but not limited
to projects to examine and attempt to increase
transplantation among populations with special needs,
including children and individuals who are members of
racial or ethnic minority groups, and among populations
with limited access to transportation, and
(O) provide that for purposes of this paragraph, the
term ``children'' refers to individuals who are under
the age of 18.
(3) Clarification.--In adopting and using standards
of quality under paragraph (2)(E), the Organ
Procurement and Transplantation Network may adopt and
use such standards with respect to organs infected with
human immunodeficiency virus (in this paragraph
referred to as ``HIV''), provided that any such
standards ensure that organs infected with HIV may be
transplanted only into individuals who--
(A) are infected with HIV before receiving
such organ; and
(B)(i) are participating in clinical research
approved by an institutional review board under
the criteria, standards, and regulations
described in subsections (a) and (b) of section
377E; or
(ii) if the Secretary has determined under
section 377E(c) that participation in such
clinical research, as a requirement for such
transplants, is no longer warranted, are
receiving a transplant under the standards and
regulations under section 377E(c).
(c) The Secretary shall establish procedures for--
(1) receiving from interested persons critical
comments relating to the manner in which the Organ
Procurement and Transplantation Network is carrying out
the duties of the Network under subsection (b); and
(2) the consideration by the Secretary of such
critical comments.
(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.--The Secretary may distribute such
fees among 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.
* * * * * * *
TITLE IV--NATIONAL RESEARCH INSTITUTES
* * * * * * *
Part B--General Provisions Respecting National Research Institutes
* * * * * * *
SEC. 409I. PROGRAM FOR PEDIATRIC STUDIES OF DRUGS.
(a) List of Priority Issues in Pediatric Therapeutics.--
(1) In general.--Not later than one year after the
date of the enactment of the Best Pharmaceuticals for
Children Act of 2007, the Secretary, acting through the
Director of the National Institutes of Health and in
consultation with the Commissioner of Food and Drugs
and experts in pediatric research, shall develop and
publish a priority list of needs in pediatric
therapeutics, including drugs, biological products, or
indications that require study. The list shall be
revised every three years.
(2) Consideration of available information.--In
developing and prioritizing the list under paragraph
(1), the Secretary--
(A) shall consider--
(i) therapeutic gaps in pediatrics
that may include developmental
pharmacology, pharmacogenetic
determinants of drug response,
metabolism of drugs and biologics in
children, and pediatric clinical
trials;
(ii) particular pediatric diseases,
disorders or conditions where more
complete knowledge and testing of
therapeutics, including drugs and
biologics, and identification of
biomarkers for such diseases,
disorders, or conditions, may be
beneficial in pediatric populations;
and
(iii) the adequacy of necessary
infrastructure to conduct pediatric
pharmacological research, including
research networks and trained pediatric
investigators; and
(B) may consider the availability of
qualified countermeasures (as defined in
section 319F-1), security countermeasures (as
defined in section 319F-2), and qualified
pandemic or epidemic products (as defined in
section 319F-3) to address the needs of
pediatric populations, in consultation with the
Assistant Secretary for Preparedness and
Response, consistent with the purposes of this
section.
(b) Pediatric Studies and Research.--The Secretary, acting
through the National Institutes of Health, shall award funds to
entities that have the expertise to conduct pediatric clinical
trials or other research (including qualified universities,
hospitals, laboratories, contract research organizations,
practice groups, federally funded programs such as pediatric
pharmacology research units, other public or private
institutions, or individuals) to enable the entities to conduct
the drug studies or other research on the issues described in
paragraphs (1) and (2)(A) of subsection (a). The Secretary may
use contracts, grants, or other appropriate funding mechanisms
to award funds under this subsection.
(c) Process for Proposed Pediatric Study Requests and
Labeling Changes.--
(1) Submission of proposed pediatric study request.--
The Director of the National Institutes of Health
shall, as appropriate, submit proposed pediatric study
requests for consideration by the Commissioner of Food
and Drugs for pediatric studies of a specific pediatric
indication identified under subsection (a). Such a
proposed pediatric study request shall be made in a
manner equivalent to a written request made under
subsection (b) or (c) of section 505A of the Federal
Food, Drug, and Cosmetic Act, or section 351(m) of this
Act, including with respect to the information provided
on the pediatric studies to be conducted pursuant to
the request. The Director of the National Institutes of
Health may submit a proposed pediatric study request
for a drug for which--
(A)(i) there is an approved application under
section 505(j) of the Federal Food, Drug, and
Cosmetic Act or section 351(k) of this Act; or
(ii) there is a submitted application that
could be approved under the criteria of such
section; and
(B) there remains no patent listed pursuant
to section 505(b)(1) of the Federal Food, Drug,
and Cosmetic Act, and every three-year and
five-year period referred to in subsection
(c)(3)(E)(ii), (c)(3)(E)(iii), (c)(3)(E)(iv),
(j)(5)(F)(ii), (j)(5)(F)(iii), or (j)(5)(F)(iv)
of section 505 of the Federal Food, Drug, and
Cosmetic Act, or applicable twelve-year period
referred to in section 351(k)(7) of this Act,
and any seven-year period referred to in
section 527 of the Federal Food, Drug, and
Cosmetic Act has ended for at least one form of
the drug; and
(C) additional studies are needed to assess
the safety and effectiveness of the use of the
drug in the pediatric population.
(2) Written request to holders of approved
applications.--The Commissioner of Food and Drugs, in
consultation with the Director of the National
Institutes of Health, may issue a written request based
on the proposed pediatric study request for the
indication or indications submitted pursuant to
paragraph (1) (which shall include a timeframe for
negotiations for an agreement) for pediatric studies
concerning a drug identified under subsection (a) to
all holders of an approved application for the drug.
Such a written request shall be made in a manner
equivalent to the manner in which a written request is
made under subsection (b) or (c) of section 505A of the
Federal Food, Drug, and Cosmetic Act or section 351(m)
of this Act, including with respect to information
provided on the pediatric studies to be conducted
pursuant to the request and using appropriate
formulations for each age group for which the study is
requested.
(3) Requests for proposals.--If the Commissioner of
Food and Drugs does not receive a response to a written
request issued under paragraph (2) not later than 30
days after the date on which a request was issued, the
Secretary, acting through the Director of the National
Institutes of Health and in consultation with the
Commissioner of Food and Drugs, shall publish a request
for proposals to conduct the pediatric studies
described in the written request in accordance with
subsection (b).
(4) Disqualification.--A holder that receives a first
right of refusal shall not be entitled to respond to a
request for proposals under paragraph (3).
(5) Contracts, grants, or other funding mechanisms.--
A contract, grant, or other funding may be awarded
under this section only if a proposal is submitted to
the Secretary in such form and manner, and containing
such agreements, assurances, and information as the
Secretary determines to be necessary to carry out this
section.
(6) Reporting of studies.--
(A) In general.--On completion of a pediatric
study in accordance with an award under this
section, a report concerning the study shall be
submitted to the Director of the National
Institutes of Health and the Commissioner of
Food and Drugs. The report shall include all
data generated in connection with the study,
including a written request if issued.
(B) Availability of reports.--
(i) In general.--Each report
submitted under subparagraph (A) shall
be considered to be in the public
domain (subject to section 505A(d)(4)
of the Federal Food, Drug, and Cosmetic
Act) and not later than 90 days after
submission of such report, shall be--
(I) posted on the internet
website of the National
Institutes of Health in a
manner that is accessible and
consistent with all applicable
Federal laws and regulations,
including such laws and
regulations for the protection
of--
(aa) human research
participants, including
with respect to
privacy, security,
informed consent, and
protected health
information; and
(bb) proprietary
interests, confidential
commercial information,
and intellectual
property rights; and
(II) assigned a docket number
by the Commissioner of Food and
Drugs and made available for
the submission of public
comments.
(ii) Submission of comments.--An
interested person may submit written
comments concerning such pediatric
studies to the Commissioner of Food and
Drugs, and the submitted comments shall
become part of the docket file with
respect to each of the drugs.
(C) Action by commissioner.--The Commissioner
of Food and Drugs shall take action in a timely
and appropriate manner in response to the
reports submitted under subparagraph (A), and
shall begin such action upon receipt of the
report under subparagraph (A), in accordance
with paragraph (7).
(7) Requests for labeling change.--Within the 180-day
period after the date on which a report is submitted
under paragraph (6)(A), the Commissioner of Food and
Drugs shall--
(A) review the report and such other data as
are available concerning the safe and effective
use in the pediatric population of the drug
studied;
(B) negotiate with the holders of approved
applications for the drug studied for any
labeling changes that the Commissioner of Food
and Drugs determines to be appropriate and
requests the holders to make; and
(C)(i) include in the public docket file a
reference to the location of the report on the
internet website of the National Institutes of
Health and a copy of any requested labeling
changes; and
(ii) publish through a posting on the Web
site of the Food and Drug Administration a
summary of the report and a copy of any
requested labeling changes.
(8) Dispute resolution.--
(A) Referral to pediatric advisory
committee.--If, not later than the end of the
180-day period specified in paragraph (7), the
holder of an approved application for the drug
involved does not agree to any labeling change
requested by the Commissioner of Food and Drugs
under that paragraph, the Commissioner of Food
and Drugs shall refer the request to the
Pediatric Advisory Committee.
(B) Action by the pediatric advisory
committee.--Not later than 90 days after
receiving a referral under subparagraph (A),
the Pediatric Advisory Committee shall--
(i) review the available information
on the safe and effective use of the
drug in the pediatric population,
including study reports submitted under
this section; and
(ii) make a recommendation to the
Commissioner of Food and Drugs as to
appropriate labeling changes, if any.
(9) FDA determination.--Not later than 30 days after
receiving a recommendation from the Pediatric Advisory
Committee under paragraph (8)(B)(ii) with respect to a
drug, the Commissioner of Food and Drugs shall consider
the recommendation and, if appropriate, make a request
to the holders of approved applications for the drug to
make any labeling change that the Commissioner of Food
and Drugs determines to be appropriate.
(10) Failure to agree.--If a holder of an approved
application for a drug, within 30 days after receiving
a request to make a labeling change under paragraph
(9), does not agree to make a requested labeling
change, the Commissioner of Food and Drugs may deem the
drug to be misbranded under the Federal Food, Drug, and
Cosmetic Act.
(11) No effect on authority.--Nothing in this
subsection limits the authority of the United States to
bring an enforcement action under the Federal Food,
Drug, and Cosmetic Act when a drug lacks appropriate
pediatric labeling. Neither course of action (the
Pediatric Advisory Committee process or an enforcement
action referred to in the preceding sentence) shall
preclude, delay, or serve as the basis to stay the
other course of action.
[(d) Authorization of Appropriations.--
[(1) In general.--There are authorized to be
appropriated to carry out this section, $5,753,425 for
the period beginning on October 1, 2022 and ending on
December 23, 2022.
[(2) Availability.--Any amount appropriated under
paragraph (1) shall remain available to carry out this
section until expended.]
(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.
* * * * * * *
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