[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[H.R. 5416 Introduced in House (IH)]

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117th CONGRESS
  1st Session
                                H. R. 5416

    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 HOUSE OF REPRESENTATIVES

                           September 29, 2021

Mr. Butterfield (for himself, Mr. McCaul, and Ms. Wild) introduced the 
   following bill; which was referred to the Committee on Energy and 
                                Commerce

_______________________________________________________________________

                                 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''.

SEC. 2. RESEARCH INTO PEDIATRIC USES OF DRUGS; ADDITIONAL AUTHORITIES 
              OF FOOD AND DRUG ADMINISTRATION REGARDING MOLECULARLY 
              TARGETED CANCER DRUGS.

    (a) In General.--
            (1) Authority regarding investigation of combination of 
        active ingredients.--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) the active ingredient or ingredients 
                        of such drug or biological product in 
                        combination with--
                                    ``(I) an active ingredient of a 
                                drug or biological product for which 
                                there is in effect an exemption for 
                                investigational use under section 
                                505(i) that was submitted by the person 
                                submitting the application referred to 
                                in paragraph (1)(B); or
                                    ``(II) an active ingredient of a 
                                drug for which an approval under 
                                section 505 is in effect, or an active 
                                ingredient of a biological product for 
                                which an approval under section 351 of 
                                the Public Health Service Act is in 
                                effect.
                    ``(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.
                            ``(ii) Purpose of investigation.--The 
                        purpose of a molecularly targeted pediatric 
                        cancer investigation referred to in 
                        subparagraph (A) shall be--
                                    ``(I) in the case of such an 
                                investigation conducted with respect to 
                                a drug or biological product referred 
                                to in clause (i) of such subparagraph, 
                                to inform potential pediatric labeling 
                                of the drug or biological product 
                                involved; and
                                    ``(II) in the case of such an 
                                investigation conducted with respect to 
                                a combination of active ingredients 
                                described to in clause (ii) of such 
                                subparagraph, to assist in determining 
                                the relevance of its molecular target 
                                to the growth or progression of a 
                                pediatric cancer.
                            ``(iii) Preclinical data.--The reports on 
                        an investigation required in paragraph (1)(B) 
                        shall 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 may 
                        not be construed as addressing the use of 
                        inactive ingredients with such combination. For 
                        purposes of such subparagraph, the Secretary 
                        may establish such requirements with respect to 
                        inactive ingredients as the Secretary 
                        determines to be appropriate.''.
            (2) Clarifying applicability of certain provisions.--
        Section 505B(a)(3) of the Federal Food, Drug, and Cosmetic Act 
        (21 U.S.C. 355c(a)(3)), as amended by paragraph (1), is amended 
        by adding at the end the following:
                    ``(E) Internal committee review; labeling changes; 
                dissemination of information; adverse events; scope of 
                authority.--Subsections (f) through (j) shall apply 
                with respect to investigations described in this 
                paragraph to the same extent and in the same manner as 
                such subsections apply with respect to the assessments 
                required under paragraph (1)(A), except that subsection 
                (g) does not apply with respect to an investigation 
                referred to in subparagraph (A)(ii).''.
            (3) 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)'';
                    (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)''; 
                and
                    (C) in paragraph (5)(D), by inserting before the 
                period at the end the following: ``, except this 
                subparagraph is not applicable to a drug or biological 
                product that is the subject of an investigation 
                referred to in paragraph (3)(A)(ii)''.
    (b) Authority Regarding Preclinical Studies.--Section 505B(a)(1) of 
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)(1)), as 
amended by subsection (b)(1), is further amended by adding at the end 
the following:
                    ``(D) Preclinical studies generally.--
                            ``(i) In general.--With respect to an 
                        application 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 two 
                        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. 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.''.
    (c) Applicability.--The amendments made by this section apply with 
respect to any application 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 expiration of the 3-year period beginning 
on the date of the enactment of this Act.
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