Research to Accelerate Cures and Equity for Children Act

Summary: Research to Accelerate Cures and Equity for Children Act or the RACE for Children Act This bill amends the Federal Food, Drug, and Cosmetic Act to expand Food and Drug Administration (FDA) requirements for sponsors of certain drugs and biological products for adult cancer to assess the use of their medications in pediatric populations. (Currently, applications for FDA approval of new medications or new uses of medications must include pediatric assessments of safety and effectiveness for claimed indications, with exceptions.) The pediatric assessment for medications, including orphan drugs, that are used to treat cancer in adults and target a molecule germane to pediatric cancer must assess the safety and effectiveness of the medication for pediatric cancer. The bill limits waivers of pediatric assessments for medications that target a molecule germane to a pediatric cancer for which there is a need for additional treatment options. The FDA may require the sponsor of an approved medication that targets a molecule germane to pediatric cancer to complete a pediatric assessment if: (1) the medication is used for a substantial number of pediatric cancer patients, or (2) there is reason to believe the medication would have a meaningful therapeutic benefit over existing therapies for pediatric cancer patients. The FDA committee that reviews requests for pediatric studies must implement a plan to achieve earlier submission of pediatric studies. (Currently, completion of pediatric clinical studies requested by the FDA extends the patents or marketing exclusivity period for a medication by six months, with exceptions.) The FDA must act within 120 days on proposed pediatric study requests and proposed amendments to requests.        

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Research to Accelerate Cures and 
Equity for Children Act'' or the ``RACE for Children Act''.

SEC. 2. DRUG DEVELOPMENT FOR PEDIATRIC CANCER.

    (a) Molecular Targets Regarding Cancer Drugs.--Section 505B of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c) is amended--
            (1) in subsection (a)(2)(A)(i) by striking ``product for 
        the claimed indications in all relevant pediatric 
        subpopulations; and'' and inserting ``product in all relevant 
        pediatric subpopulations--
                                    ``(I) for the claimed indications; 
                                or'';
                                    ``(II) for a pediatric cancer 
                                indication, if the drug is intended for 
                                the treatment of an adult cancer and is 
                                directed at a molecular target 
                                considered to be germane to the growth 
                                and progression of such pediatric 
                                cancer; and'';
            (2) in subsection (b)(1)--
                    (A) by amending subparagraph (A)(i) to read as 
                follows:
                    ``(A)(i) the drug or biological product is used for 
                a substantial number of pediatric patients--
                            ``(I) for the labeled indications; or
                            ``(II) for a pediatric cancer indication, 
                        if the drug is intended for the treatment of an 
                        adult cancer and is directed at a molecular 
                        target considered to be germane to the growth 
                        and progression of such pediatric cancer; 
                        and''; and
                    (B) by amending subparagraph (B) to read as 
                follows:
                    ``(B) there is reason to believe that the drug or 
                biological product would represent a meaningful 
                therapeutic benefit over existing therapies for 
                pediatric patients--
                            ``(i) for one or more of the claimed 
                        indications; or
                            ``(ii) for a pediatric cancer indication, 
                        if the drug is intended for the treatment of an 
                        adult cancer and is directed at a molecular 
                        target considered to be germane to the growth 
                        and progression of such pediatric cancer; or''; 
                        and
            (3) by amending paragraph (2) of subsection (c) to read as 
        follows:
            ``(2) the drug or biological product is in a class of 
        products, is for an indication, or is directed at a specific 
        molecular target in an adult cancer and such molecular target 
        is germane to the growth or progression of cancer in a 
        pediatric cancer, for which there is need for additional 
        options.''.
    (b) Early Meeting on Pediatric Study Plan.--
            (1) In general.--Clause (i) of section 505B(e)(2)(C) of the 
        Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(e)(2)(C)) 
        is amended to read as follows:
                            ``(i) shall meet with the applicant--
                                    ``(I) if requested by the applicant 
                                with respect to a drug 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 312.47(b) of title 21, Code 
                                of Federal Regulations, or successor 
                                regulations) or within 30 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 any scientific 
                                or operational challenges that may be 
                                the basis of a deferral under 
                                subsection (a)(3) or a full or partial 
                                waiver under subsection (a)(4);''.
            (2) Conforming changes.--Section 505B(e) of the Federal 
        Food, Drug, and Cosmetic Act (21 U.S.C. 355c(e)) is amended--
                    (A) in the heading of paragraph (2), by striking 
                ``meeting'' and inserting ``meetings'';
                    (B) in the heading of paragraph (2)(C), by striking 
                ``Meeting'' and inserting ``Meetings'';
                    (C) in clauses (ii) and (iii) of paragraph (2)(C), 
                by striking ``no meeting'' each place it appears and 
                inserting ``no meeting under clause (i)(II)''; and
                    (D) in paragraph (3) by striking ``meeting under 
                paragraph (2)(C)(i)'' and inserting ``meeting under 
                paragraph (2)(C)(i)(II)''.
    (c) Orphan Drugs.--Section 505B(k) of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 355c(k)) is amended by inserting ``except in 
the case of a drug or biological product that is intended for the 
treatment of an adult cancer and is directed at a molecular target 
considered to be germane to the growth and progression of a pediatric 
cancer,'' after ``regulation,''.
    (d) Guidance.--Not later than 1 year after the date of enactment of 
this Act, the Secretary of Health and Human Services, acting through 
the Commissioner of Food and Drugs, shall issue guidance on the 
implementation of the amendments to section 505B of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 355c) made by this section, 
including--
            (1) study designs;
            (2) molecular targets considered to be germane to the 
        growth and progression present in one or more cancers in 
        pediatric populations that may be appropriate for assessment 
        under such section 505B, as so amended; and
            (3) considerations for implementation of such section 505B, 
        as so amended, and waivers of the requirements of such section 
        505B with regard to molecular targets for which several drugs 
        may be under investigation.
    (e) Applicability.--This section and the amendments made by this 
section apply with respect to applications for a drug submitted under 
section 505 of the Federal Food, Drug, or Cosmetic Act (21 U.S.C. 355) 
or section 351 of the Public Health Service Act (42 U.S.C. 262) on or 
after the date that is 18 months after the date of enactment of this 
Act.
    (f) Report to Congress.--Section 508(b) of the FDA Safety and 
Innovation Act (21 U.S.C. 355c-1(b)) is amended--
            (1) in paragraph (10), by striking ``; and'' and inserting 
        ``;''; and
            (2) by striking paragraph (11) and inserting the following:
            ``(11) an assessment of the impact of the amendments to 
        such section 505B made by the RACE for Children Act on 
        pediatric labeling of drugs and pediatric labeling of 
        molecularly targeted drugs for the treatment of cancer;
            ``(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, 
        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)) before studies conducted pursuant to such 
                sections 505A or 505B 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 prior to the submission 
                of an application under section 505(b)(1) of the 
                Federal Food, Drug, and Cosmetic Act; and
                    ``(E) the average number, and range of numbers, of 
                amendments to written requests issued;
            ``(13) a list of sponsors of applications or holders of 
        approved applications who received exclusivity under such 
        section 505A after receiving a letter issued under such section 
        505B(d)(1) and before the studies referred to in such letter 
        were completed and submitted; and
            ``(14) a list of assessments required under subsection 
        (a)(2)(A)(i)(II), and (b)(1)(B)(ii) of section 505B.''.
    (g) Rule of Construction.--Nothing in this section, including the 
amendments made by this section, shall limit the authority of the 
Secretary of Health and Human Services to issue written requests under 
section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
355a).

SEC. 3. IMPROVING THE TIMELINESS OF PEDIATRIC STUDIES.

    (a) Informing Internal Review Committee.--Section 505A(f) of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a(f)) is amended by 
adding at the end the following:
            ``(7) Informing internal review committee.--The Secretary 
        shall provide to the committee referred to in paragraph (1) any 
        response issued to an applicant or holder with respect to a 
        proposed pediatric study request.''.
    (b) Action on Submissions.--Section 505A(d) of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 355a(d)) is amended--
            (1) by redesignating paragraphs (3) through (5) as 
        paragraphs (4) through (6), respectively; and
            (2) by inserting after paragraph (2) the following:
            ``(3) Action on submissions.--The Secretary shall review 
        and act upon a submission of a proposed pediatric study request 
        or a sponsor's proposed amendment to a written request for 
        pediatric studies within 120 days of the submission.''.
    (c) Study.--The Secretary of Health and Human Services, acting 
through the internal review committee established under section 505C of 
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355d) shall, not 
later than one year after the date of enactment of this Act, develop 
and implement a plan to achieve, when appropriate, earlier submission 
of pediatric studies under section 505A of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 355a). Such plan shall include recommendations 
to achieve--
            (1) earlier discussion of proposed pediatric study requests 
        and written requests with sponsors, and if appropriate, at the 
        meeting required under section 505B(e)(2)(C) of the Federal 
        Food, Drug, and Cosmetic Act (21 U.S.C. 355c(e)(2)(C)), as 
        amended by section 2;
            (2) earlier issuance of written requests for a pediatric 
        study under such section 505A, including for investigational 
        new drugs prior to the submission of an application under 
        section 505(b)(1) of the Federal Food, Drug, and Cosmetic Act 
        (21 U.S.C. 355(b)(1)); and
            (3) shorter timelines, when appropriate, for the completion 
        of studies pursuant to a written request under such section 
        505A.

SEC. 4. NEONATOLOGY EXPERTISE.

    Section 6(d) of the Best Pharmaceuticals for Children Act (21 
U.S.C. 393a(d)) is amended by striking ``For the 5-year period 
beginning on the date of enactment of this subsection, at'' and 
inserting ``At''.