[Congressional Bills 118th Congress]
[From the U.S. Government Publishing Office]
[H.R. 3433 Engrossed in House (EH)]

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118th CONGRESS
  2d Session
                                H. R. 3433

_______________________________________________________________________

                                 AN ACT


 
   To amend the Federal Food, Drug, and Cosmetic Act with respect to 
  molecularly targeted pediatric cancer investigations, and for other 
                               purposes.

    Be it enacted by the Senate and House of Representatives of the 
United States of America in Congress assembled,

SECTION 1. SHORT TITLE; TABLE OF CONTENTS.

    (a) Short Title.--This Act may be cited as the ``Give Kids a Chance 
Act of 2024''.
    (b) Table of Contents.--The table of contents for this Act is as 
follows:

Sec. 1. Short title; table of contents.
                      TITLE I--GIVE KIDS A CHANCE

Sec. 101. Research into pediatric uses of drugs; additional authorities 
                            of Food and Drug Administration regarding 
                            molecularly targeted cancer drugs.
Sec. 102. Ensuring completion of pediatric study requirements.
Sec. 103. FDA report on PREA enforcement.
Sec. 104. Extension of authority to issue priority review vouchers to 
                            encourage treatments for rare pediatric 
                            diseases.
Sec. 105. Limitations on exclusive approval or licensure of orphan 
                            drugs.
Sec. 106. Program for pediatric studies of drugs.
    TITLE II--UNITED STATES-ABRAHAM ACCORDS COOPERATION AND SECURITY

Sec. 201. Establishment of Abraham Accords Office within Food and Drug 
                            Administration.
        TITLE III--ORGAN PROCUREMENT AND TRANSPLANTATION NETWORK

Sec. 301. Registration fees.

                      TITLE I--GIVE KIDS A CHANCE

SEC. 101. RESEARCH INTO PEDIATRIC USES OF DRUGS; ADDITIONAL AUTHORITIES 
              OF FOOD AND DRUG ADMINISTRATION REGARDING MOLECULARLY 
              TARGETED CANCER DRUGS.

    (a) In General.--
            (1) Additional active ingredient for application drug; 
        limitation regarding novel-combination application drug.--
        Section 505B(a)(3) of the Federal Food, Drug, and Cosmetic Act 
        (21 U.S.C. 355c(a)(3)) is amended--
                    (A) by redesignating subparagraphs (B) and (C) as 
                subparagraphs (C) and (D), respectively; and
                    (B) by striking subparagraph (A) and inserting the 
                following:
                    ``(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.--Any amounts collected under this 
        subsection shall--
                    ``(A) be credited to the currently applicable 
                appropriation, account, or fund of the Department of 
                Health and Human Services as discretionary offsetting 
                collections; and
                    ``(B) be available, only to the extent and in the 
                amounts provided in advance in appropriations Acts, to 
                distribute such fees among 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.''.

            Passed the House of Representatives September 23, 2024.

            Attest:

                                                                 Clerk.
118th CONGRESS

  2d Session

                               H. R. 3433

_______________________________________________________________________

                                 AN ACT

   To amend the Federal Food, Drug, and Cosmetic Act with respect to 
  molecularly targeted pediatric cancer investigations, and for other 
                               purposes.