Mikaela Naylon Give Kids a Chance Act

The "Give Kids a Chance Act of 2025" seeks to enhance the development of pediatric drugs, particularly for cancer, by amending the Federal Food, Drug, and Cosmetic Act. It expands the scope of required molecularly targeted pediatric cancer investigations to include combination therapies, ensuring studies yield clinically meaningful data for pediatric labeling. The bill also clarifies and strengthens the enforcement mechanisms for pediatric study requirements, mandating a due diligence process before penalties can be imposed for non-compliance. Furthermore, the legislation extends the authority to issue rare pediatric disease priority review vouchers until September 30, 2029, and modifies the user fee payment structure for these vouchers. It also narrows the scope of orphan drug exclusivity , applying it to a "same approved use or indication within such rare disease or condition" rather than the broader disease or condition, which could encourage more treatments for different indications within the same rare disease. The bill authorizes $25 million annually for fiscal years 2025 through 2027 for pediatric drug studies. Beyond drug development, the Act introduces reforms to the Organ Procurement and Transplantation Network (OPTN). These reforms encourage the integration of electronic health records among hospitals, organ procurement organizations, and transplant centers, and mandate consideration of a public dashboard for transplant statistics. The Secretary is also authorized to collect registration fees from OPTN members to support network operations, with transparency requirements and a sunset clause for this authority. Finally, the bill establishes an Abraham Accords Office within the Food and Drug Administration. This office will provide technical assistance to regulatory partners in Abraham Accords countries, focusing on strengthening oversight and converging regulatory requirements, and will facilitate interactions between the FDA and stakeholders in these nations.