S. 932 — Give Kids a Chance Act of 2025

This section revises section 505B of the Federal Food, Drug, and Cosmetic Act (i.e., Pediatric Research Equity Act, which requires sponsors of certain new drug and biologics license applications to assess pediatric uses, including formulations, studies, or investigations) as follows: (1) expands the scope of required molecularly targeted pediatric cancer investigations under subsection (a)(3)(A) to include the sponsor's drug or biological product, alone or in combination with (A) an active ingredient in an approved abbreviated application that is part of the standard of care for pediatric cancer or (B) an active ingredient in the sponsor's approved adult cancer application targeting a molecular target substantially relevant to pediatric cancer growth or progression (with requirements for study design to produce age-appropriate data on dosing, safety, and efficacy; submission of relevant completed preclinical results with the initial pediatric study plan; limitations to single new active ingredients or novel combinations of previously approved adult cancer active ingredients; and a rule of construction excluding inactive ingredients); (2) requires the Secretary, under subsection (e)(1), to determine before the initial pediatric study plan deadline whether subsection (a)(1)(A) or (B) applies; (3) clarifies under subsection (a)(1)(C) that no application subject to (a)(1)(B) is subject to (a)(1)(A), and vice versa; and (4) makes conforming amendments to subsection (a)(3)(C) and (D). Directs the FDA Commissioner to issue draft guidance on these amendments within 12 months of enactment and finalize it within 12 months thereafter. Specifies that the amendments apply to applications under FFDCA section 505(b) or PHSA section 351(a) submitted 3 years after enactment. Requires (1) an HHS report to Congress on implementation efforts, due 6 years after enactment, and (2) a GAO study and report on the effectiveness of required assessments and investigations, due 8 years after enactment.

This section (1) extends authority for FDA to award priority review vouchers for sponsors of rare pediatric disease product applications (i.e., new drugs or biological products for serious or life-threatening diseases primarily affecting individuals from birth to 18 years that are also rare diseases) through September 30, 2029 (from December 20, 2024); (2) revises the timing of the priority review user fee to require payment upon submission of the human drug application for which the voucher is used, with all other user fees due as otherwise required; and (3) directs the Comptroller General to study the effectiveness of the program in incentivizing development of treatments for rare pediatric diseases—including factors such as unmet needs addressed, company sizes, voucher values and usage, and FDA workload impacts—and to report findings to the House Energy and Commerce Committee and Senate Health, Education, Labor, and Pensions Committee not later than five years after enactment. (As background, the program awards vouchers entitling holders to FDA priority review—i.e., action within six months—of a single drug application, which may be transferred or sold to expedite review of unrelated drugs.)