Give Kids a Chance Act of 2025

This bill would expand the Food and Drug Administration's authority to require pediatric cancer research on drug combinations and allow for enforcement actions against drug sponsors that fail to meet pediatric study requirements. It proposes to reauthorize the rare pediatric disease priority review voucher program through September 30, 2029, and extend funding for pediatric research at the National Institutes of Health through fiscal year 2027. Additionally, the bill would codify that orphan drug exclusivity applies only to the specific approved use of a drug rather than all potential uses for a rare disease.

This bill affects drug sponsors seeking approval for new products, particularly those developing molecularly targeted pediatric cancer investigations and treatments for rare pediatric diseases. The legislation also impacts the Food and Drug Administration (FDA) and the National Institutes of Health (NIH) by modifying enforcement authorities and reauthorizing research funding. Additionally, the bill affects pharmaceutical companies seeking orphan drug exclusivity by codifying specific market exclusivity limitations for rare disease treatments.

• Expansion of molecularly targeted pediatric cancer investigations. The bill allows for research on new drugs in combination with previously approved active ingredients for pediatric cancer investigations, provided specific conditions are met.
• Enforcement of pediatric study requirements. The FDA is authorized to take enforcement action against drug sponsors that fail to meet pediatric study requirements if the sponsor demonstrates a lack of due diligence.
• Extension of rare pediatric disease priority review vouchers. The authority for the FDA to award priority review vouchers to sponsors of new products for rare pediatric diseases is renewed through September 30, 2029.
• Reauthorization of pediatric research funding. The bill reauthorizes funding through fiscal year 2027 for the National Institutes of Health to support priority pediatric research programs.
• Clarification of orphan drug exclusivity scope. The bill codifies the FDA's interpretation that the seven-year market exclusivity period for orphan drugs applies only to the specific approved use or indication, rather than the entire disease or condition.

The bill reauthorizes funding for priority pediatric research through fiscal year 2027 and extends the FDA's authority to award priority review vouchers for rare pediatric disease treatments through September 30, 2029.

The bill reauthorizes existing FDA programs for rare pediatric disease priority review vouchers and NIH pediatric research funding, while providing statutory authority for the FDA's interpretation of orphan drug exclusivity in response to the Catalyst Pharmaceuticals, Inc. v. Becerra court ruling. Additionally, it expands the FDA's enforcement power regarding existing pediatric study requirements and modifies current regulations concerning molecularly targeted pediatric cancer investigations.

The bill aims to expand the Food and Drug Administration’s authority to facilitate and enforce research on rare pediatric diseases, specifically by authorizing investigations into combination therapies for pediatric cancer and extending programs that incentivize the development of treatments for rare childhood conditions. Additionally, it seeks to codify the scope of market exclusivity for orphan drugs to ensure it applies specifically to the approved use or indication of the drug.