Mikaela Naylon Give Kids a Chance Act of 2025

This bill would authorize clinical trials for pediatric cancer that investigate combinations of new drugs and existing treatments, including those currently used as the standard of care or approved for adult cancers. It would also renew the Food and Drug Administration's authority to award priority review vouchers to developers of treatments for rare pediatric diseases through September 30, 2030. Additionally, the legislation would require the Government Accountability Office to report on the effectiveness of these drug development programs and the resulting impact on patient access.

This bill affects pediatric cancer patients and individuals with rare pediatric diseases who may benefit from new drug combinations and treatments. It also impacts pharmaceutical sponsors of new products for rare pediatric diseases who become eligible for priority review vouchers. Additionally, the legislation directs the Food and Drug Administration, the Government Accountability Office, and Congress to oversee and report on the implementation and effectiveness of these pediatric drug development programs.

• Expansion of molecularly targeted pediatric cancer investigations. The bill authorizes clinical trials for pediatric cancer that involve combinations of new drugs and previously approved active ingredients, provided the ingredients are part of the standard of care or target specific molecular markers.
• Renewal of the Rare Pediatric Disease Priority Review Voucher program. The legislation extends the Food and Drug Administration's authority to award priority review vouchers to sponsors of new treatments for rare pediatric diseases through September 30, 2030.
• FDA guidance and reporting requirements. The Food and Drug Administration is required to issue implementation guidance for the new clinical trial provisions and submit a report to Congress regarding its efforts to ensure these changes are implemented.
• GAO evaluations of drug development effectiveness. The Government Accountability Office must conduct studies on the effectiveness of the bill's changes to pediatric cancer drug development and the overall success of the Rare Pediatric Disease PRV program in promoting patient access.

The bill renews the Food and Drug Administration’s authority to issue priority review vouchers for rare pediatric disease products through September 30, 2030.

This bill renews the Food and Drug Administration’s authority to award priority review vouchers for rare pediatric diseases through September 30, 2030, reviving a program that expired in December 2024. Additionally, it modifies existing requirements for molecularly targeted pediatric cancer investigations to allow for clinical trials involving combinations of new drugs and previously approved active ingredients.

The bill aims to advance the development of pediatric cancer treatments by authorizing targeted clinical trials for drug combinations and renewing the Food and Drug Administration's authority to award priority review vouchers for rare pediatric disease products through September 30, 2030.