II 

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. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Give Kids a Chance 

5 Act of 2022’’. 


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1 SEC. 2. RESEARCH INTO PEDIATRIC USES OF DRUGS; ADDI2 TIONAL AUTHORITIES OF FOOD AND DRUG 

3 ADMINISTRATION REGARDING MOLECU4 LARLY TARGETED CANCER DRUGS. 

5 (a) IN GENERAL.— 

6 (1) ADDITIONAL ACTIVE INGREDIENT FOR AP7 PLICATION DRUG; LIMITATION REGARDING NOVEL8 COMBINATION APPLICATION DRUG.—Section 

9 505B(a)(3) of the Federal Food, Drug, and Cos10 metic Act (21 U.S.C. 355c(a)(3)) is amended— 

11 (A) by redesignating subparagraphs (B) 

12 and (C) as subparagraphs (C) and (D), respec13 tively; and 

14 (B) by striking subparagraph (A) and in15 serting the following: 

16 ‘‘(A) IN GENERAL.—For purposes of para17 graph (1)(B), the investigation described in this 

18 paragraph is (as determined by the Secretary) 

19 a molecularly targeted pediatric cancer inves20 tigation of— 

21 ‘‘(i) the drug or biological product for 

22 which the application referred to in such 

23 paragraph is submitted; or 

24 ‘‘(ii) such drug or biological product 

25 in combination with— 


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1 ‘‘(I) an active ingredient of a 

2 drug for which an approved applica3 tion under section 505(j) is in effect 

4 or an active ingredient of a biological 

5 product for which an approved appli6 cation under section 351(k) of the 

7 Public Health Service Act is in effect, 

8 which drug or biological product is de9 termined by the Secretary to be the 

10 standard of care for treating a pedi11 atric cancer; 

12 ‘‘(II) an active ingredient of a 

13 drug for which an approved applica14 tion under section 505(b) is in effect 

15 to treat an adult cancer, or an active 

16 ingredient of a biological product for 

17 which an approved application under 

18 section 351(a) of the Public Health 

19 Service Act is in effect to treat an 

20 adult cancer, which approved applica21 tion is held by the same person sub22 mitting the application; or 

23 ‘‘(III) an active ingredient of a 

24 drug or biological product for which 

25 there is in effect an exemption for inVerDate Sep 11 2014 22:29 May 17, 2022 Jkt 029200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\S4215.IS S4215

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1 vestigational use under section 505(i), 

2 which drug or biological product is 

3 under such exemption being studied 

4 jointly by the person submitting the 

5 application referred to in paragraph 

6 (1)(B) and by another person pursu7 ant to an agreement between such 

8 persons. 

9 ‘‘(B) ADDITIONAL REQUIREMENTS.— 

10 ‘‘(i) DESIGN OF INVESTIGATION.—A 

11 molecularly targeted pediatric cancer inves12 tigation referred to in subparagraph (A) 

13 shall be designed to yield clinically mean14 ingful pediatric study data, gathered using 

15 appropriate formulations for each age 

16 group for which the study is required, re17 garding dosing, safety, and preliminary ef18 ficacy to inform potential pediatric label19 ing. 

20 ‘‘(ii) LIMITATION.—Studies described 

21 in subparagraph (A)(ii) may be required 

22 only if the drug or biological product for 

23 which the application referred to in para24 graph (1)(B) contains either— 


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1 ‘‘(I) a single new active ingre2 dient; or 

3 ‘‘(II) more than one active ingre4 dient, if an application for the com5 bination of active ingredients has not 

6 previously been approved but each ac7 tive ingredient has been previously ap8 proved to treat an adult cancer. 

9 ‘‘(iii) PRECLINICAL DATA.—The Sec10 retary may require that reports on an in11 vestigation required pursuant to paragraph 

12 (1)(B) include the results of all preclinical 

13 studies on which the decision to conduct 

14 such investigation was based. 

15 ‘‘(iv) RULE OF CONSTRUCTION RE16 GARDING INACTIVE INGREDIENTS.—With 

17 respect to a combination of active ingredi18 ents referred to in subparagraph (A)(ii), 

19 such subparagraph shall not be construed 

20 as addressing the use of inactive ingredi21 ents with such combination.’’. 

22 (2) CONFORMING AMENDMENTS.—Section 

23 505B(a) of the Federal Food, Drug, and Cosmetic 

24 Act (21 U.S.C. 355c(a)) is amended— 


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1 (A) in paragraph (3)(C), as redesignated 

2 by paragraph (1)(A) of this subsection, by 

3 striking ‘‘investigations described in this para4 graph’’ and inserting ‘‘investigations referred to 

5 in subparagraph (A)(i)’’; and 

6 (B) in paragraph (3)(D), as redesignated 

7 by paragraph (1)(A) of this subsection, by 

8 striking ‘‘the assessments under paragraph 

9 (2)(B)’’ and inserting ‘‘the assessments re10 quired under paragraph (1)(A)’’. 

11 (b) AUTHORITY REGARDING PRECLINICAL STUD12 IES.—Section 505B(a)(1) of the Federal Food, Drug, and 

13 Cosmetic Act (21 U.S.C. 355c(a)(1)) is amended by add14 ing at the end the following: 

15 ‘‘(C) PRECLINICAL STUDIES GEN16 ERALLY.— 

17 ‘‘(i) IN GENERAL.—With respect to a 

18 submission for an exemption for investiga19 tional use under section 505(i) for a drug 

20 or biological product that is intended for 

21 the treatment of an adult cancer, the Sec22 retary may require, as a condition of per23 mitting the exemption to go into effect, 

24 that the sponsor involved enter into an 

25 agreement with the Secretary to conduct 


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1 not more than 2 preclinical studies of the 

2 drug or biological product in order to as3 sist in determining the relevance of its mo4 lecular target to the growth or progression 

5 of a pediatric cancer. 

6 ‘‘(ii) TIMEFRAME FOR PRECLINICAL

7 STUDIES.—With respect to the drug or bi8 ological product involved, an agreement 

9 under clause (i) for a preclinical study 

10 shall specify the date by which an initial 

11 plan for the study will be submitted to the 

12 Secretary except that the Secretary may 

13 not require the submission of such plan 

14 any earlier than 90 days after the exemp15 tion referred to in clause (i) goes into ef16 fect. The results of the preclinical study 

17 shall be submitted to the Secretary in ac18 cordance with a timeframe to which the 

19 Secretary and the sponsor involved have 

20 agreed. Such timeframe shall provide for 

21 deferrals equivalent to deferrals under 

22 paragraphs (4) and (5). 

23 ‘‘(iii) USE OF PRECLINICAL STUDY

24 RESULTS.—The Secretary may not use the 

25 results of the preclinical studies under 


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1 clause (i) to require additional clinical 

2 studies under subparagraph (B) other than 

3 the pediatric cancer studies specified in the 

4 agreement under clause (i).’’. 

5 (c) APPLICABILITY.—The amendments made by this 

6 section apply with respect to any submission under section 

7 505(i) of the Federal Food, Drug, and Cosmetic Act (21 

8 U.S.C. 355(i)), any application under section 505 of such 

9 Act (21 U.S.C. 355), and any application under section 

10 351(a) of the Public Health Service Act (42 U.S.C. 262), 

11 that is submitted on or after the date that is 2 years after 

12 the date of enactment of this Act. 

13 (d) REPORT TO CONGRESS.—Not later than 1 year 

14 after the date of enactment of this Act, the Secretary of 

15 Health and Human Services shall submit a report to Con16 gress on the Secretary’s efforts, in coordination with in17 dustry, to ensure implementation of the amendments 

18 made by subsections (a) and (b) by the date that is 2 years 

19 after the date of enactment of this Act. 

Æ 


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