Pediatric Cancer Drug Research Bill

Official: Mikaela Naylon Give Kids a Chance Act

IntroducedFebruary 12, 2025

HealthInternational law and treatiesCongressional oversightGovernment information and archivesGovernment studies and investigationsComputers and information technologyPerformance measurementChild healthExecutive agency funding and structureHealth information and medical recordsCancerLicensing and registrationsMedical researchPrescription drugsDrug safety, medical device, and laboratory regulationArab-Israeli relationsOrgan and tissue donation and transplantationBahrainFood and Drug Administration (FDA)Israel

Why This Matters

This bill focuses on improving the research and approval process for drugs used to treat pediatric cancers and other rare diseases in children.

If you have a child with cancer, this bill could lead to more effective treatment options becoming available.

Affects: Children with cancer and their families will benefit from better treatment options.

Summary

Give Kids a Chance Act of 2025This bill expands the Food and Drug Administration’s (FDA’s) authority with respect to research on rare pediatric diseases, including by permitting the FDA to take enforcement action against drug sponsors that fail to satisfy pediatric study requirements and by reauthorizing programs that support pediatric research. Specifically, the billmodifies requirements relating to molecularly targeted pediatric cancer investigations to permit research on new drugs in combination with active ingredients that have already been approved, provided certain conditions are met; permits the FDA to take enforcement action against drug sponsors that fail to comply with pediatric study requirements, if such sponsors demonstrated a lack of due diligence in satisfying the requirement; renews the FDA’s authority to award priority review vouchers to sponsors of new products intended to treat rare pediatric diseases through September 30, 2029; andreauthorizes through FY2027 certain funding for the National Institutes of Health to support priority pediatric research. The bill also provides statutory authority for the FDA’s interpretation of the orphan drug exclusivity period. The bill specifies, consistent with FDA regulations, that the seven-year market exclusivity period for drugs for rare diseases or conditions (i. e., orphan drugs) prohibits the approval of the same drug for the same approved use or indication with respect to the disease or condition. (In Catalyst Pharmaceuticals, Inc. v. Becerra, a court rejected the FDA’s interpretation and held that orphan drug exclusivity extends to all uses or indications for the disease or condition.)