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[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[S. 4215 Introduced in Senate (IS)]

<DOC>

117th CONGRESS
  2d Session
                                S. 4215

    To amend the Federal Food, Drug, and Cosmetic Act to establish 
 additional authorities of the Food and Drug Administration regarding 
 the conduct of pediatric investigations of molecularly targeted drugs 
                to treat cancer, and for other purposes.

_______________________________________________________________________

                   IN THE SENATE OF THE UNITED STATES

                              May 12, 2022

 Mr. Rubio (for himself and Mr. Bennet) 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 to establish 
 additional authorities of the Food and Drug Administration regarding 
 the conduct of pediatric investigations of molecularly targeted drugs 
                to treat cancer, 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 ``Give Kids a Chance Act of 2022''.

SEC. 2. 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 for which an approved application 
                                under section 505(j) is in effect or an 
                                active ingredient of a biological 
                                product for which an approved 
                                application under section 351(k) of the 
                                Public Health Service Act is in effect, 
                                which drug or biological product is 
                                determined by the Secretary to be the 
                                standard of care for treating a 
                                pediatric cancer;
                                    ``(II) an active ingredient of a 
                                drug for which an approved application 
                                under section 505(b) is in effect to 
                                treat an adult cancer, or an active 
                                ingredient of a biological product for 
                                which an approved application under 
                                section 351(a) of the Public Health 
                                Service Act is in effect to treat an 
                                adult cancer, which approved 
                                application is held by the same person 
                                submitting the application; or
                                    ``(III) an active ingredient of a 
                                drug or biological product for which 
                                there is in effect an exemption for 
                                investigational use under section 
                                505(i), which drug or biological 
                                product is under such exemption being 
                                studied jointly by the person 
                                submitting the application referred to 
                                in paragraph (1)(B) and by another 
                                person pursuant to an agreement between 
                                such persons.
                    ``(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, 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.--Studies 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) Preclinical data.--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) 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)(i)''; 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) Authority Regarding Preclinical Studies.--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) Preclinical studies generally.--
                            ``(i) In general.--With respect to a 
                        submission for an exemption for investigational 
                        use under section 505(i) for a drug or 
                        biological product that is intended for the 
                        treatment of an adult cancer, the Secretary may 
                        require, as a condition of permitting the 
                        exemption to go into effect, that the sponsor 
                        involved enter into an agreement with the 
                        Secretary to conduct not more than 2 
                        preclinical studies of the drug or biological 
                        product in order to assist in determining the 
                        relevance of its molecular target to the growth 
                        or progression of a pediatric cancer.
                            ``(ii) Timeframe for preclinical studies.--
                        With respect to the drug or biological product 
                        involved, an agreement under clause (i) for a 
                        preclinical study shall specify the date by 
                        which an initial plan for the study will be 
                        submitted to the Secretary except that the 
                        Secretary may not require the submission of 
                        such plan any earlier than 90 days after the 
                        exemption referred to in clause (i) goes into 
                        effect. The results of the preclinical study 
                        shall be submitted to the Secretary in 
                        accordance with a timeframe to which the 
                        Secretary and the sponsor involved have agreed. 
                        Such timeframe shall provide for deferrals 
                        equivalent to deferrals under paragraphs (4) 
                        and (5).
                            ``(iii) Use of preclinical study results.--
                        The Secretary may not use the results of the 
                        preclinical studies under clause (i) to require 
                        additional clinical studies under subparagraph 
                        (B) other than the pediatric cancer studies 
                        specified in the agreement under clause (i).''.
    (c) Applicability.--The amendments made by this section apply with 
respect to any submission under section 505(i) of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 355(i)), any application under 
section 505 of such Act (21 U.S.C. 355), and any application under 
section 351(a) of the Public Health Service Act (42 U.S.C. 262), that 
is submitted on or after the date that is 2 years after the date of 
enactment of this Act.
    (d) Report to Congress.--Not later than 1 year after the date of 
enactment of this Act, the Secretary of Health and Human Services shall 
submit a report to Congress on the Secretary's efforts, in coordination 
with industry, to ensure implementation of the amendments made by 
subsections (a) and (b) by the date that is 2 years after the date of 
enactment of this Act.
                                 <all>
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