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[Congressional Bills 118th Congress]
[From the U.S. Government Publishing Office]
[S. 4905 Introduced in Senate (IS)]

<DOC>

118th CONGRESS
  2d Session
                                S. 4905

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

_______________________________________________________________________

                   IN THE SENATE OF THE UNITED STATES

                             July 31, 2024

 Mr. Reed (for himself and Mrs. Capito) introduced the following bill; 
     which was read twice and referred to the Committee on Health, 
                     Education, Labor, and Pensions

_______________________________________________________________________

                                 A BILL

 
   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.

    This Act may be cited as the ``Innovation in Pediatric Drugs Act of 
2024''.

SEC. 2. 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) for failures described in 
section 505B(d) of such Act (21 U.S.C. 355c(d)) only if such failures 
occur on or after the date that is 180 days after the date of enactment 
of this Act.

SEC. 3. FDA REPORT ON PREA ENFORCEMENT.

    Section 508(b) of the Food and Drug Administration Safety and 
Innovation Act (21 U.S.C. 355c-1(b)) is amended--
            (1) in paragraph (11), by striking the semicolon at the end 
        and inserting ``, including an evaluation of compliance with 
        deadlines provided for in deferrals and deferral extensions;'';
            (2) in paragraph (15), by striking ``and'' at the end;
            (3) in paragraph (16), by striking the period at the end 
        and inserting ``; and''; and
            (4) by adding at the end the following:
            ``(17) a listing of penalties, settlements, or payments 
        under section 303 of the Federal Food, Drug, and Cosmetic Act 
        (21 U.S.C. 353) for failure to comply with requirements under 
        such section 505B, including, for each penalty, settlement, or 
        payment, the name of the drug, the sponsor thereof, and the 
        amount of the penalty, settlement, or payment imposed.''.

SEC. 4. 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.''.

SEC. 5. PEDIATRIC STUDIES OF ORPHAN DRUGS.

    (a) Application of Pediatric Research Requirements to Orphan 
Drugs.--
            (1) In general.--Section 505B(k) of the Federal Food, Drug, 
        and Cosmetic Act (21 U.S.C. 355c(k)) is amended to read as 
        follows:
    ``(k) Relation to Orphan Drugs.--
            ``(1) In general.--This section does not apply to a drug or 
        biological product for an indication for which orphan 
        designation has been granted under section 526 unless the 
        Secretary determines that pediatric assessments of such drug or 
        biological product required under this section could represent 
        a meaningful therapeutic benefit as described in subsection 
        (c).
            ``(2) Deferrals and waivers.--Deferrals and waivers under 
        subsections (a)(4) and (a)(5) shall apply to assessments 
        described in this subsection to the same extent and in the same 
        manner as such deferrals and waivers apply with respect to the 
        assessments under subsection (a)(1), and waivers under 
        subsection (b)(2) shall apply to assessments described in this 
        subsection to the same extent and in the same manner as such 
        waivers apply with respect to the assessments required pursuant 
        to subsection (b)(1).''.
            (2) Applicability.--The amendment made by paragraph (1) 
        applies only to applications described in subparagraph (A) or 
        (B) of section 505B(a)(1) of the Federal Food, Drug, and 
        Cosmetic Act (21 U.S.C. 355c(a)(1)) that are submitted on or 
        after the later of--
                    (A) the date that is 18 months after the date of 
                issuance of the final guidance under subsection (b); 
                and
                    (B) such later date as may be specified by the 
                Secretary of Health and Human Services (referred to in 
                this section as the ``Secretary'') by regulation.
    (b) Guidance.--
            (1) Issuance.--The Secretary shall--
                    (A) not later than 1 year after the date of 
                enactment of this Act, issue draft guidance describing 
                how, upon the applicability of the amendment made by 
                subsection (a)(1), the requirements of section 505B of 
                the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
                355c) will apply with respect to any drug or biological 
                product for an indication within a disease or condition 
                for which orphan designation has been granted under 
                section 526 of such Act (21 U.S.C. 360bb); and
                    (B) not later than 18 months after the date of the 
                public meeting required by subsection (c)(1), finalize 
                such draft guidance.
            (2) Contents.--The guidance under subsection (b) shall 
        address the following:
                    (A) Information regarding how full and partial 
                waivers under subsections (a)(5)(A), (a)(5)(B), and 
                (b)(2) of section 505B of the Federal Food, Drug, and 
                Cosmetic Act (21 U.S.C. 355c) for any drug or 
                biological product for an indication within a disease 
                or condition for which orphan designation has been 
                granted under section 526 of such Act (21 U.S.C. 360bb) 
                will be granted.
                    (B) Application of the requirements of section 
                505B(e) of such Act (21 U.S.C. 355c(e)) to drugs or 
                biological products for an indication within a disease 
                or condition for which orphan designation has been 
                granted under section 526 of such Act (21 U.S.C. 
                360bb), including submission and timing of planned 
                requests for full or partial waivers and responses by 
                the Food and Drug Administration to those requests.
                    (C) Rare diseases and conditions (as defined in 
                section 526(a)(2) of such Act (21 U.S.C. 360bb(a)(2)) 
                that should be added to the lists under section 
                505B(a)(5)(E) and 505B(b)(2)(E) of such Act, as added 
                by subsection (f), and a process for regularly updating 
                such lists.
                    (D) Situations where the initial pediatric study 
                plan under section 505B(e) of such Act (21 U.S.C. 
                355c(e)) includes a plan to fulfill the requirements of 
                section 505B(a) of such Act (21 U.S.C. 355c(a)) without 
                requesting waivers in any age group.
                    (E) Consideration of how the Secretary will balance 
                the unique scientific challenges of rare disease drug 
                development with the need for improved pediatric 
                labeling of drugs and biological products for 
                indications within diseases or conditions for which 
                orphan designation has been granted under section 526 
                of such Act (21 U.S.C. 360bb).
                    (F) Considerations of the strengths, weaknesses, 
                appropriateness, and limitations of different types of 
                real-world evidence specific to the fulfillment of 
                requirements under section 505B of such Act (21 U.S.C. 
                355c).
                    (G) Consideration of input received from the public 
                meeting set forth in subsection (c).
    (c) Public Meeting.--The Secretary shall--
            (1) not later than 6 months after the date of issuance of 
        the draft guidance under subsection (b)(1)(A), hold a public 
        meeting to inform the final guidance to be issued under 
        subsection (b)(1)(B); and
            (2) publish prior notice of such meeting in the Federal 
        Register.
    (d) GAO Study.--Not later than 4 years after the applicability date 
described in subsection (a)(2), the Comptroller General of the United 
States shall submit to the Committee on Energy and Commerce and the 
Committee on Ways and Means of the House of Representatives and the 
Committee on Health, Education, Labor, and Pensions of the Senate a 
report that--
            (1) addresses the impacts of this Act on--
                    (A) rare disease drug development in the United 
                States; and
                    (B) the availability of pediatric information on 
                drugs and biological products within diseases or 
                conditions for indications for which orphan designation 
                has been granted under section 526 of the Federal Food, 
                Drug, and Cosmetic Act (21 U.S.C. 360bb); and
            (2) includes--
                    (A) the findings of a survey of companies of 
                varying sizes engaged in the development of orphan 
                drugs, which shall include questions regarding the 
                feasibility and other challenges of conducting 
                pediatric studies for such indications;
                    (B) input from patient groups and medical provider 
                associations; and
                    (C) an assessment of the impact changes to required 
                pediatric studies had on drug development for rare 
                diseases.
    (e) Rule of Construction.--Nothing in this section shall be 
construed to limit requirements for investigations, as described in 
section 505B(a)(3) of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 355c(a)(3)), of molecularly targeted pediatric cancer drugs for 
which orphan designation has been granted under section 526 of such Act 
(21 U.S.C. 360bb).
    (f) Certainty Regarding Waivers.--Section 505B of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 355c) is amended--
            (1) in subsection (a)(5), by adding at the end the 
        following:
                    ``(E) Automatic full waiver list.--The Secretary 
                shall maintain a list, posted on the website of the 
                Food and Drug Administration, of adult-related diseases 
                and conditions--
                            ``(i) with respect to which the necessary 
                        studies are impossible or highly impracticable, 
                        as described in subparagraph (A)(i); or
                            ``(ii) for which a drug or biological 
                        product for such disease or condition otherwise 
                        meets the criteria described in subparagraph 
                        (A).'';
            (2) in subsection (b)(2), by adding at the end the 
        following:
                    ``(E) Automatic full waiver list.--The Secretary 
                shall maintain a list, posted on the website of the 
                Food and Drug Administration, of adult-related diseases 
                and conditions with respect to which the necessary 
                studies would meet the criteria for a full waiver under 
                subparagraph (A).''; and
            (3) in subsection (e)(4), by adding at the end the 
        following: ``If, at the time of an applicant's submission of 
        the initial pediatric study plan, the disease or condition for 
        which the drug is intended to treat appears on the list under 
        subsection (a)(5)(E), then the assessments for such disease or 
        condition shall be waived under subsection (a)(5).''.
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