S. 3302 — Mikaela Naylon Give Kids a Chance Act of 2025

This section revises the Pediatric Research Equity Act (PREA, 21 U.S.C. 355c)—which requires sponsors of certain new drug (under FD&C Act §505(b)) and biologics license (under PHS Act §351(a)) applications to assess pediatric safety and effectiveness for drugs that may treat pediatric diseases—by expanding the scope of required molecularly targeted pediatric cancer investigations to include (1) the applicant’s drug or biological product alone or (2) such product in combination with an active ingredient that is either part of the pediatric cancer standard of care (with an approved abbreviated application) or from the same sponsor’s approved adult cancer application targeting a substantially relevant molecular target for pediatric cancer. (Thus, combinations are limited to those with a single new active ingredient or a novel combination where each ingredient was previously approved for adult cancer; investigations must be designed to produce age-appropriate data on dosing, safety, and preliminary efficacy; and sponsors must submit relevant completed preclinical study results with the initial pediatric study plan.) The section further requires FDA to determine applicable PREA requirements before the initial pediatric study plan deadline; clarifies that the new combination provision supersedes the prior single-ingredient requirement (with no dual applicability); and makes conforming amendments. Additionally, this section directs FDA to issue draft implementation guidance within 12 months of enactment and finalize it within another 12 months; applies the amendments to applications submitted 3 years after enactment; and requires (1) an HHS report to Congress within 6 years on implementation efforts and (2) a GAO study and report within 8 years on the effectiveness of the required assessments and investigations.

This section (1) extends the authority to award priority review vouchers for FDA-approved drugs treating rare pediatric diseases—serious or life-threatening rare diseases (i.e., affecting fewer than 200,000 people in the United States) primarily in individuals from birth to 18 years—through September 30, 2030 (from December 20, 2024); (2) revises the timing of priority review user fees to require payment upon submission of the drug application using the voucher, with all other user fees due as otherwise required; and (3) directs the Government Accountability Office to study the effectiveness of the program in incentivizing drug development, including analyses of approved products, company sizes, voucher values and usage, unmet needs addressed, and FDA workload impacts, and to submit a report to specified congressional committees not later than five years after enactment.