Give Kids a Chance Act of 2025

Improved Pediatric Cancer Treatment Data: The bill requires investigations to be designed to yield clinically meaningful data regarding dosing, safety, and preliminary efficacy for different age groups (Section 2(a)(1)(B)(i)).

Expanded Research Scope: Investigations can now include a drug or biological product used in combination with an active ingredient that is part of the standard of care for treating a pediatric cancer (Section 2(a)(1)(A)(ii)(I)(bb)).

Targeted Adult-to-Pediatric Research: Investigations may involve an active ingredient of a drug used to treat an adult cancer, provided it is directed at a molecular target relevant to the growth or progression of a pediatric cancer (Section 2(a)(1)(A)(ii)(II)(aa) and (bb)).

Encouragement of Rare Disease Treatments: The bill extends the authority to issue priority review vouchers, which is intended to encourage the development of treatments for rare pediatric diseases (Section 3(a)).

Potential Burdens on Drug Development: The Government Accountability Office (GAO) is tasked with studying whether these requirements create burdens on pediatric cancer drug development (Section 2(d)(2)(A)).

FDA Workload Concerns: The GAO study will examine the impact of priority review vouchers on the workload and review process of the Food and Drug Administration (Section 3(c)(1)(B)(xi)).