Mikaela Naylon Give Kids a Chance Act
Mikaela Naylon Give Kids a Chance Act
Plain Language Summary
# Mikaela Naylon Give Kids a Chance Act (HR 1262) – Summary **What It Does:** This bill expands the FDA's power to support and enforce research on rare diseases affecting children. It allows pharmaceutical companies to test new cancer drugs in combination with already-approved medications, gives the FDA authority to punish companies that don't complete required pediatric studies, and extends a "priority review voucher" program through 2029. These vouchers reward drug developers who research treatments for rare pediatric diseases by allowing them to fast-track approval of other drugs.
The bill also reauthorizes pediatric research programs through 2027. **Who It Affects:** Children with rare diseases—particularly cancer—are the primary beneficiaries, as the bill aims to encourage more research into treatments. Pharmaceutical companies are also significantly affected since they face stricter requirements to study drugs in pediatric patients and potential penalties for non-compliance. The FDA gains new enforcement tools to ensure companies follow through on pediatric research commitments. **Current Status:** The bill has already passed the House of Representatives and is moving through the legislative process.
CRS Official 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.)
Latest Action
Received in the Senate.