[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.
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                            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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