119-s932

[Congressional Bills 119th Congress]
[From the U.S. Government Publishing Office]
[S. 932 Introduced in Senate

    (IS) ]

<DOC>

119th CONGRESS
 1st Session
 S. 932

 To amend the Federal Food, Drug, and Cosmetic Act with respect to 
 molecularly targeted pediatric cancer investigations, and for other 
 purposes.

_______________________________________________________________________

 IN THE SENATE OF THE UNITED STATES

 March 11 (legislative day, March 10), 2025

Mr. Mullin (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 with respect to 
 molecularly targeted pediatric cancer investigations, 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 2025''.

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 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 used 
 in combination with--
 ``
            (I) an active ingredient of a 
 drug or biological product--
 ``

    (aa) for which an 
 approved application under 
 

section 505

    (j) under this Act 
 or under 

section 351

    (k) of the 
 Public Health Service Act is in 
 effect; and
 ``

    (bb) that is determined 
 by the Secretary, after 
 consultation with the 
 applicant, to be part of the 
 standard of care for treating a 
 pediatric cancer; or
 ``
            (II) an active ingredient of a 
 drug or biological product--
 ``

    (aa) for which an 
 approved application under 
 

section 505

    (b) of this Act or 
 

section 351

    (a) of the Public 
 Health Service Act to treat an 
 adult cancer is in effect and 
 is held by the same person 
 submitting the application 
 under paragraph

    (1)
        (B) ; and
 ``

    (bb) that is directed at 
 a molecular target that the 
 Secretary determines to be 
 substantially relevant to the 
 growth or progression of a 
 pediatric cancer.
 ``
        (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 that is 
 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.--An investigation 
 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 is in a drug product 
 that has been previously approved to 
 treat an adult cancer.
 ``
            (iii) Results of already-completed 
 preclinical studies of application drug.--With 
 respect to an investigation required pursuant 
 to paragraph

    (1)
        (B) , the Secretary may require 
 the results of any completed preclinical 
 studies relevant to the initial pediatric study 
 plan be submitted to the Secretary at the same 
 time that the initial pediatric study plan 
 required under subsection

    (e)

    (1) is submitted.
 ``
            (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) Determination of applicable requirements.--

Section 505B

    (e)

    (1) of the Federal Food, Drug, and Cosmetic Act (21 
 U.S.C. 355c

    (e)

    (1) ) is amended by adding at the end the 
 following: ``The Secretary shall determine whether subparagraph
        (A) or
        (B) of subsection

    (a)

    (1) applies with respect to an 
 application before the date on which the applicant is required 
 to submit the initial pediatric study plan under paragraph

    (2)
        (A) .''.

    (3) Clarifying applicability.--

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) Rule of construction.--No application that is 
 subject to the requirements of subparagraph
        (B) shall 
 be subject to the requirements of subparagraph
        (A) , and 
 no application (or supplement to an application) that 
 is subject to the requirements of subparagraph
        (A) shall be subject to the requirements of subparagraph
        (B) .''.

    (4) 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) ''; 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) Guidance.--The Secretary of Health and Human Services, acting 
through the Commissioner of Food and Drugs, shall--

    (1) not later than 12 months after the date of enactment of 
 this Act, issue draft guidance on the implementation of the 
 amendments made by subsection

    (a) ; and

    (2) not later than 12 months after closing the comment 
 period on such draft guidance, finalize such guidance.
            (c) Applicability.--The amendments made by this section apply with 
respect to any application under 

section 505

    (b) of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 355

    (b) ) and any application under 

section 351

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

    (a) ), 
that is submitted on or after the date that is 3 years after the date 
of enactment of this Act.
            (d) Reports to Congress.--

    (1) Secretary of health and human services.--Not later than 
 6 years after the date of enactment of this Act, the Secretary 
 of Health and Human Services shall submit to the Committee on 
 Energy and Commerce of the House of Representatives and the 
 Committee on Health, Education, Labor, and Pensions of the 
 Senate a report on the Secretary's efforts, in coordination 
 with industry, to ensure implementation of the amendments made 
 by subsection

    (a) .

    (2) GAO study and report.--
        (A) Study.--Not later than 8 years after the date 
 of enactment of this Act, the Comptroller General of 
 the United States shall conduct a study of the 
 effectiveness of requiring assessments and 
 investigations described in 

section 505B of the Federal 
 Food, Drug, and Cosmetic Act (21 U.S.C. 355c), as 
 amended by subsection

    (a) , in the development of drugs 
 and biological products for pediatric cancer 
 indications, including consideration of any benefits 
 to, or burdens on, pediatric cancer drug development.
        (B) === Findings ===
-Not later than 10 years after the 
 date of enactment of this Act, the Comptroller General 
 shall submit to the Committee on Energy and Commerce of 
 the House of Representatives and the Committee on 
 Health, Education, Labor, and Pensions of the Senate a 
 report containing the findings of the study conducted 
 under subparagraph
        (A) .

SEC. 3. EXTENSION OF AUTHORITY TO ISSUE PRIORITY REVIEW VOUCHERS TO 
 ENCOURAGE TREATMENTS FOR RARE PEDIATRIC DISEASES.

    (a) Extension.--

Section 529

    (b)

    (5) of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 360ff

    (b)

    (5) ) is amended by striking ``December 
20, 2024, unless'' and all that follows through the period at the end 
and inserting ``September 30, 2029.''.

    (b) User Fee Payment.--Subsection 529
            (c) (4) of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 360ff
            (c) (4) ) is amended by striking 
subparagraph
        (A) and inserting the following:
 ``
        (A) In general.--The priority review user fee 
 required by this subsection shall be due upon the 
 submission of a human drug application under 

section 505

    (b)

    (1) or 

section 351

    (a) of the Public Health 
 Service Act for which the priority review voucher is 
 used. All other user fees associated with the human 
 drug application shall be due as required by the 
 Secretary or under applicable law.''.
            (c) GAO Report on Effectiveness of Rare Pediatric Disease Priority 
Voucher Awards in Incentivizing Rare Pediatric Disease Drug 
Development.--

    (1) GAO study.--
        (A) Study.--The Comptroller General of the United 
 States shall conduct a study of the effectiveness of 
 awarding rare pediatric disease priority vouchers under 
 

section 529 of the Federal Food, Drug, and Cosmetic Act 
 (21 U.S.C. 360ff), as amended by subsection

    (a) , in the 
 development of human drug products that treat or 
 prevent rare pediatric diseases (as defined in such 
 

section 529).
        (B) Contents of study.--In conducting the study 
 under subparagraph
        (A) , the Comptroller General shall 
 examine the following:
            (i) The indications for each drug or 
 biological product that--
            (I) is the subject of a rare 
 pediatric disease product application 
 (as defined in 

section 529 of the 
 Federal Food, Drug, and Cosmetic Act 
 (21 U.S.C. 360ff)) for which a priority 
 review voucher was awarded; and
            (II) was approved under 

section 505 
 of the Federal Food, Drug, and Cosmetic 
 Act (42 U.S.C. 355) or licensed under 
 

section 351 of the Public Health 
 Service Act (42 U.S.C. 262).
            (ii) Whether, and to what extent, an unmet 
 need related to the treatment or prevention of 
 a rare pediatric disease was met through the 
 approval or licensure of such a drug or 
 biological product.
            (iii) The size of the company to which a 
 priority review voucher was awarded under 
 

section 529 of the Federal Food, Drug, and 
 Cosmetic Act (21 U.S.C. 360ff) for such a drug 
 or biological product.
            (iv) The value of such priority review 
 voucher if transferred.
            (v) Identification of each drug for which a 
 priority review voucher awarded under such 
 

section 529 was used.
            (vi) The size of the company using each 
 priority review voucher awarded under such 
 

section 529.
            (vii) The length of the period of time 
 between the date on which a priority review 
 voucher was awarded under such 

section 529 and 
 the date on which it was used.
            (viii) Whether, and to what extent, an 
 unmet need related to the treatment or 
 prevention of a rare pediatric disease was met 
 through the approval under 

section 505 of the 
 Federal Food, Drug, and Cosmetic Act (42 U.S.C. 
 355) or licensure under 

section 351 of the 
 Public Health Service Act (42 U.S.C. 262) of a 
 drug for which a priority review voucher was 
 used.
            (ix) Whether, and to what extent, companies 
 were motivated by the availability of priority 
 review vouchers under 

section 529 of the 
 Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
 360ff) to attempt to develop a drug for a rare 
 pediatric disease.
            (x) Whether, and to what extent, pediatric 
 review vouchers awarded under such section were 
 successful in stimulating development and 
 expedited patient access to drug products for 
 treatment or prevention of a rare pediatric 
 disease that wouldn't otherwise take place 
 without the incentive provided by such 
 vouchers.
            (xi) The impact of such priority review 
 vouchers on the workload, review process, and 
 public health prioritization efforts of the 
 Food and Drug Administration.
            (xii) Any other incentives in Federal law 
 that exist for companies developing drugs or 
 biological products described in clause
            (i) .

    (2) Report on 

=== findings ===
-Not later than 5 years after the 
 date of the enactment of this Act, the Comptroller General of 
 the United States shall submit to the Committee on Energy and 
 Commerce of the House of Representatives and the Committee on 
 Health, Education, Labor, and Pensions of the Senate a report 
 containing the findings of the study conducted under paragraph

    (1) .
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