Give Kids a Chance Act of 2024

IIB 118TH CONGRESS 2D SESSION H. R. 3433 IN THE SENATE OF THE UNITED STATES SEPTEMBER 24, 2024 Received; read twice and referred to the Committee on Health, Education, Labor, and Pensions AN ACT To amend the Federal Food, Drug, and Cosmetic Act with respect to molecularly targeted pediatric cancer investiga- tions, and for other purposes. Be it enacted by the Senate and House of Representa- 1 tives of the United States of America in Congress assembled, 2 VerDate Sep 11 2014 03:03 Sep 25, 2024 Jkt 049200 PO 00000 Frm 00001 Fmt 6652 Sfmt 6201 E:\BILLS\H3433.RFS H3433 ssavage on LAPJG3WLY3PROD with BILLS 2 HR 3433 RFS SECTION 1. SHORT TITLE; TABLE OF CONTENTS. 1 (a) SHORT TITLE.—This Act may be cited as the 2 ‘‘Give Kids a Chance Act of 2024’’. 3 (b) TABLE OF CONTENTS.—The table of contents for 4 this Act is as follows: 5 Sec. 1. Short title; table of contents. TITLE I—GIVE KIDS A CHANCE Sec. 101. Research into pediatric uses of drugs; additional authorities of Food and Drug Administration regarding molecularly targeted can- cer drugs. Sec. 102. Ensuring completion of pediatric study requirements. Sec. 103. FDA report on PREA enforcement. Sec. 104. Extension of authority to issue priority review vouchers to encourage treatments for rare pediatric diseases. Sec. 105. Limitations on exclusive approval or licensure of orphan drugs. Sec. 106. Program for pediatric studies of drugs. TITLE II—UNITED STATES-ABRAHAM ACCORDS COOPERATION AND SECURITY Sec. 201. Establishment of Abraham Accords Office within Food and Drug Ad- ministration. TITLE III—ORGAN PROCUREMENT AND TRANSPLANTATION NETWORK Sec. 301. Registration fees. TITLE I—GIVE KIDS A CHANCE 6 SEC. 101. RESEARCH INTO PEDIATRIC USES OF DRUGS; AD- 7 DITIONAL AUTHORITIES OF FOOD AND DRUG 8 ADMINISTRATION REGARDING MOLECU- 9 LARLY TARGETED CANCER DRUGS. 10 (a) IN GENERAL.— 11 (1) ADDITIONAL ACTIVE INGREDIENT FOR AP- 12 PLICATION DRUG; LIMITATION REGARDING NOVEL- 13 COMBINATION APPLICATION DRUG.—Section 14 VerDate Sep 11 2014 03:03 Sep 25, 2024 Jkt 049200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\H3433.RFS H3433 ssavage on LAPJG3WLY3PROD with BILLS 3 HR 3433 RFS 505B(a)(3) of the Federal Food, Drug, and Cos- 1 metic Act (21 U.S.C. 355c(a)(3)) is amended— 2 (A) by redesignating subparagraphs (B) 3 and (C) as subparagraphs (C) and (D), respec- 4 tively; and 5 (B) by striking subparagraph (A) and in- 6 serting the following: 7 ‘‘(A) IN GENERAL.—For purposes of para- 8 graph (1)(B), the investigation described in this 9 paragraph is (as determined by the Secretary) 10 a molecularly targeted pediatric cancer inves- 11 tigation of— 12 ‘‘(i) the drug or biological product for 13 which the application referred to in such 14 paragraph is submitted; or 15 ‘‘(ii) such drug or biological product 16 in combination with— 17 ‘‘(I) an active ingredient of a 18 drug or biological product— 19 ‘‘(aa) for which an approved 20 application under section 505(j) 21 under this Act or under section 22 351(k) of the Public Health 23 Service Act is in effect; and 24 VerDate Sep 11 2014 03:03 Sep 25, 2024 Jkt 049200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\H3433.RFS H3433 ssavage on LAPJG3WLY3PROD with BILLS 4 HR 3433 RFS ‘‘(bb) that is determined by 1 the Secretary to be the standard 2 of care for treating a pediatric 3 cancer; or 4 ‘‘(II) an active ingredient of a 5 drug or biological product— 6 ‘‘(aa) for which an approved 7 application under section 505(b) 8 of this Act or section 351(a) of 9 the Public Health Service Act to 10 treat an adult cancer is in effect 11 and is held by the same person 12 submitting the application under 13 paragraph (1)(B); and 14 ‘‘(bb) that is directed at a 15 molecular target that the Sec- 16 retary determines to be substan- 17 tially relevant to the growth or 18 progression of a pediatric cancer. 19 ‘‘(B) ADDITIONAL REQUIREMENTS.— 20 ‘‘(i) DESIGN OF INVESTIGATION.—A 21 molecularly targeted pediatric cancer inves- 22 tigation referred to in subparagraph (A) 23 shall be designed to yield clinically mean- 24 ingful pediatric study data that is gathered 25 VerDate Sep 11 2014 03:03 Sep 25, 2024 Jkt 049200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\H3433.RFS H3433 ssavage on LAPJG3WLY3PROD with BILLS 5 HR 3433 RFS using appropriate formulations for each 1 age group for which the study is required, 2 regarding dosing, safety, and preliminary 3 efficacy to inform potential pediatric label- 4 ing. 5 ‘‘(ii) LIMITATION.—An investigation 6 described in subparagraph (A)(ii) may be 7 required only if the drug or biological 8 product for which the application referred 9 to in paragraph (1)(B) contains either— 10 ‘‘(I) a single new active ingre- 11 dient; or 12 ‘‘(II) more than one active ingre- 13 dient, if an application for the com- 14 bination of active ingredients has not 15 previously been approved but each ac- 16 tive ingredient has been previously ap- 17 proved to treat an adult cancer. 18 ‘‘(iii) RESULTS OF ALREADY-COM- 19 PLETED PRECLINICAL STUDIES OF APPLI- 20 CATION DRUG.—The Secretary may re- 21 quire that reports on an investigation re- 22 quired pursuant to paragraph (1)(B) in- 23 clude the results of all preclinical studies 24 VerDate Sep 11 2014 03:03 Sep 25, 2024 Jkt 049200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\H3433.RFS H3433 ssavage on LAPJG3WLY3PROD with BILLS 6 HR 3433 RFS on which the decision to conduct such in- 1 vestigation was based. 2 ‘‘(iv) RULE OF CONSTRUCTION RE- 3 GARDING INACTIVE INGREDIENTS.—With 4 respect to a combination of active ingredi- 5 ents referred to in subparagraph (A)(ii), 6 such subparagraph shall not be construed 7 as addressing the use of inactive ingredi- 8 ents with such combination.’’. 9 (2) DETERMINATION OF APPLICABLE REQUIRE- 10 MENTS.—Section 505B(e)(1) of the Federal Food, 11 Drug, and Cosmetic Act (21 U.S.C. 355c(e)(1)) is 12 amended by adding at the end the following: ‘‘The 13 Secretary shall determine whether subparagraph (A) 14 or (B) of subsection (a)(1) shall apply with respect 15 to an application before the date on which the appli- 16 cant is required to submit the initial pediatric study 17 plan under paragraph (2)(A).’’. 18 (3) CLARIFYING APPLICABILITY.—Section 19 505B(a)(1) of the Federal Food, Drug, and Cos- 20 metic Act (21 U.S.C. 355c(a)(1)) is amended by 21 adding at the end the following: 22 ‘‘(C) RULE OF CONSTRUCTION.—No appli- 23 cation that is subject to the requirements of 24 subparagraph (B) shall be subject to the re- 25 VerDate Sep 11 2014 03:03 Sep 25, 2024 Jkt 049200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\H3433.RFS H3433 ssavage on LAPJG3WLY3PROD with BILLS 7 HR 3433 RFS quirements of subparagraph (A), and no appli- 1 cation (or supplement to an application) that is 2 subject to the requirements of subparagraph 3 (A) shall be subject to the requirements of sub- 4 paragraph (B).’’. 5 (4) CONFORMING AMENDMENTS.—Section 6 505B(a) of the Federal Food, Drug, and Cosmetic 7 Act (21 U.S.C. 355c(a)) is amended— 8 (A) in paragraph (3)(C), as redesignated 9 by paragraph (1)(A) of this subsection, by 10 striking ‘‘investigations described in this para- 11 graph’’ and inserting ‘‘investigations referred to 12 in subparagraph (A)’’; and 13 (B) in paragraph (3)(D), as redesignated 14 by paragraph (1)(A) of this subsection, by 15 striking ‘‘the assessments under paragraph 16 (2)(B)’’ and inserting ‘‘the assessments re- 17 quired under paragraph (1)(A)’’. 18 (b) GUIDANCE.—The Secretary of Health and 19 Human Services, acting through the Commissioner of 20 Food and Drugs, shall— 21 (1) not later than 12 months after the date of 22 enactment of this Act, issue draft guidance on the 23 implementation of the amendments made by sub- 24 section (a); and 25 VerDate Sep 11 2014 03:03 Sep 25, 2024 Jkt 049200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\H3433.RFS H3433 ssavage on LAPJG3WLY3PROD with BILLS 8 HR 3433 RFS (2) not later than 12 months after closing the 1 comment period on such draft guidance, finalize 2 such guidance. 3 (c) APPLICABILITY.—The amendments made by this 4 section apply with respect to any application under section 5 505(b) of the Federal Food, Drug, and Cosmetic Act (21 6 U.S.C. 355(b)) and any application under section 351(a) 7 of the Public Health Service Act (42 U.S.C. 262(a)), that 8 is submitted on or after the date that is 3 years after the 9 date of enactment of this Act. 10 (d) REPORTS TO CONGRESS.— 11 (1) SECRETARY OF HEALTH AND HUMAN SERV- 12 ICES.—Not later than 2 years after the date of en- 13 actment of this Act, the Secretary of Health and 14 Human Services shall submit to the Committee on 15 Energy and Commerce of the House of Representa- 16 tives and the Committee on Health, Education, 17 Labor, and Pensions of the Senate a report on the 18 Secretary’s efforts, in coordination with industry, to 19 ensure implementation of the amendments made by 20 subsection (a). 21 (2) GAO STUDY AND REPORT.— 22 (A) STUDY.—Not later than 3 years after 23 the date of enactment of this Act, the Comp- 24 troller General of the United States shall con- 25 VerDate Sep 11 2014 03:03 Sep 25, 2024 Jkt 049200 PO 00000 Frm 00008 Fmt 6652 Sfmt 6201 E:\BILLS\H3433.RFS H3433 ssavage on LAPJG3WLY3PROD with BILLS 9 HR 3433 RFS duct a study of the effectiveness of requiring 1 assessments and investigations described in sec- 2 tion 505B of the Federal Food, Drug, and Cos- 3 metic Act (21 U.S.C.355c), as amended by sub- 4 section (a), in the development of drugs and bi- 5 ological products for pediatric cancer indica- 6 tions. 7 (B) FINDINGS.—Not later than 7 years 8 after the date of enactment of this Act, the 9 Comptroller General shall submit to the Com- 10 mittee on Energy and Commerce of the House 11 of Representatives and the Committee on 12 Health, Education, Labor, and Pensions of the 13 Senate a report containing the findings of the 14 study conducted under subparagraph (A). 15 SEC. 102. ENSURING COMPLETION OF PEDIATRIC STUDY 16 REQUIREMENTS. 17 (a) EQUAL ACCOUNTABILITY FOR PEDIATRIC STUDY 18 REQUIREMENTS.—Section 505B(d) of the Federal Food, 19 Drug, and Cosmetic Act (21 U.S.C. 355c(d)) is amend- 20 ed— 21 (1) in paragraph (1), by striking ‘‘Beginning 22 270’’ and inserting ‘‘NONCOMPLIANCE LETTER.— 23 Beginning 270’’; 24 (2) in paragraph (2)— 25 VerDate Sep 11 2014 03:03 Sep 25, 2024 Jkt 049200 PO 00000 Frm 00009 Fmt 6652 Sfmt 6201 E:\BILLS\H3433.RFS H3433 ssavage on LAPJG3WLY3PROD with BILLS 10 HR 3433 RFS (A) by striking ‘‘The drug or’’ and insert- 1 ing ‘‘EFFECT OF NONCOMPLIANCE.—The drug 2 or’’; and 3 (B) by striking ‘‘(except that the drug or 4 biological product shall not be subject to action 5 under section 303)’’ and inserting ‘‘(except that 6 the drug or biological product shall be subject 7 to action under section 303 only if such person 8 demonstrated a lack of due diligence in satis- 9 fying the applicable requirement)’’; and 10 (3) by adding at the end the following: 11 ‘‘(3) LIMITATION.—The Secretary shall not 12 issue enforcement actions under section 303 for fail- 13 ures under this subsection in the case of a drug or 14 biological product that is no longer marketed.’’. 15 (b) DUE DILIGENCE.—Section 505B(d) of the Fed- 16 eral Food, Drug, and Cosmetic Act (21 U.S.C. 355c(d)), 17 as amended by subsection (a), is further amended by add- 18 ing at the end the following: 19 ‘‘(4) DUE DILIGENCE.—Before the Secretary 20 may conclude that a person failed to submit or oth- 21 erwise meet a requirement as described in the mat- 22 ter preceding paragraph (1), the Secretary shall— 23 ‘‘(A) issue a noncompliance letter pursuant 24 to paragraph (1); 25 VerDate Sep 11 2014 03:03 Sep 25, 2024 Jkt 049200 PO 00000 Frm 00010 Fmt 6652 Sfmt 6201 E:\BILLS\H3433.RFS H3433 ssavage on LAPJG3WLY3PROD with BILLS 11 HR 3433 RFS ‘‘(B) provide such person with a 45-day 1 period beginning on the date of receipt of such 2 noncompliance letter to respond in writing as 3 set forth in such paragraph; and 4 ‘‘(C) after reviewing such written response, 5 determine whether the person demonstrated a 6 lack of due diligence in satisfying such require- 7 ment.’’. 8 (c) CONFORMING AMENDMENTS.—Section 9 303(f)(4)(A) of the Federal Food, Drug, and Cosmetic Act 10 (21 U.S.C. 333(f)(4)(A)) is amended by striking ‘‘or 505– 11 1’’ and inserting ‘‘505–1, or 505B’’. 12 (d) TRANSITION RULE.—The Secretary of Health 13 and Human Services may take enforcement action under 14 section 303 of the Federal Food, Drug, and Cosmetic Act 15 (21 U.S.C. 333) only for failures described in section 16 505B(d) of such Act (21 U.S.C. 355c(d)) that occur on 17 or after the date that is 180 days after the date of enact- 18 ment of this Act. 19 SEC. 103. FDA REPORT ON PREA ENFORCEMENT. 20 Section 508(b) of the Food and Drug Administration 21 Safety and Innovation Act (21 U.S.C. 355c–1(b)) is 22 amended— 23 (1) in paragraph (11), by striking the semicolon 24 at the end and inserting ‘‘, including an evaluation 25 VerDate Sep 11 2014 03:03 Sep 25, 2024 Jkt 049200 PO 00000 Frm 00011 Fmt 6652 Sfmt 6201 E:\BILLS\H3433.RFS H3433 ssavage on LAPJG3WLY3PROD with BILLS 12 HR 3433 RFS of compliance with deadlines provided for in defer- 1 rals and deferral extensions;’’; 2 (2) in paragraph (15), by striking ‘‘and’’ at the 3 end; 4 (3) in paragraph (16), by striking the period at 5 the end and inserting ‘‘; and’’; and 6 (4) by adding at the end the following: 7 ‘‘(17) a listing of penalties, settlements, or pay- 8 ments under section 303 of the Federal Food, Drug, 9 and Cosmetic Act (21 U.S.C. 353) for failure to 10 comply with requirements under such section 505B, 11 including, for each penalty, settlement, or payment, 12 the name of the drug, the sponsor thereof, and the 13 amount of the penalty, settlement, or payment im- 14 posed; and’’. 15 SEC. 104. EXTENSION OF AUTHORITY TO ISSUE PRIORITY 16 REVIEW VOUCHERS TO ENCOURAGE TREAT- 17 MENTS FOR RARE PEDIATRIC DISEASES. 18 (a) EXTENSION.—Paragraph (5) of section 529(b) of 19 the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 20 360ff(b)) is amended by striking ‘‘September 30, 2024, 21 unless’’ and all that follows through the period at the end 22 and inserting ‘‘September 30, 2029.’’. 23 (b) GAO REPORT ON EFFECTIVENESS OF RARE PE- 24 DIATRIC DISEASE PRIORITY VOUCHER AWARDS IN 25 VerDate Sep 11 2014 03:03 Sep 25, 2024 Jkt 049200 PO 00000 Frm 00012 Fmt 6652 Sfmt 6201 E:\BILLS\H3433.RFS H3433 ssavage on LAPJG3WLY3PROD with BILLS 13 HR 3433 RFS INCENTIVIZING RARE PEDIATRIC DISEASE DRUG DEVEL- 1 OPMENT.— 2 (1) GAO STUDY.— 3 (A) STUDY.—The Comptroller General of 4 the United States shall conduct a study of the 5 effectiveness of awarding rare pediatric disease 6 priority vouchers under section 529 of the Fed- 7 eral Food, Drug, and Cosmetic [Text truncated for display. 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