Mikaela Naylon Give Kids a Chance Act of 2025

Improved pediatric cancer data: Investigations must be designed to yield "clinically meaningful pediatric study data" regarding dosing, safety, and preliminary efficacy to inform potential pediatric labeling (Section 2(a)(1)(B)(i)).

Targeted treatment options: The bill allows for investigations of drugs or biological products used in combination with ingredients that are part of the "standard of care for treating a pediatric cancer" or directed at a "molecular target that the Secretary determines to be substantially relevant to the growth or progression of a pediatric cancer" (Section 2(a)(1)(A)).

Encouragement of rare disease treatments: The act extends the authority to issue priority review vouchers until September 30, 2030, to encourage the development of treatments for rare pediatric diseases (Section 3(a)).

Accountability through oversight: The bill requires the Secretary of Health and Human Services to submit a report on implementation efforts and mandates the GAO to conduct studies on the effectiveness of these pharmaceutical development programs (Section 2(d)).

Increased requirements for drug applicants: Investigations for certain combination drugs may be required to yield specific study data regarding dosing and safety for each age group (Section 2(a)(1)(B)(i)).

Potential burdens on drug development: The GAO is tasked with studying whether the required assessments and investigations create "burdens on, pediatric cancer drug development" (Section 2(d)(2)(A)).