II 117th CONGRESS 2d Session S. 4215 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.

1. Short title This Act may be cited as the Give Kids a Chance Act of 2022 . 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.