HR-1262 : Still Just a Bill

The "Give Kids a Chance Act of 2025" (H.R. 1262) amends the Federal Food, Drug, and Cosmetic Act with the goal of improving research and development of treatments for pediatric cancer and other rare diseases. Key provisions include:

• Molecularly Targeted Pediatric Cancer Investigations: Modifies requirements for drug applications to include investigations into pediatric uses of molecularly targeted cancer drugs, including combinations with other drugs that are part of the standard of care for pediatric cancer treatment.
• Ensuring Completion of Pediatric Study Requirements: Strengthens accountability for completing required pediatric studies by clarifying enforcement actions and introducing a "due diligence" standard.
• FDA Report on PREA Enforcement: Requires the FDA to include in its reports a listing of penalties, settlements, or payments for failure to comply with pediatric study requirements.
• Priority Review Vouchers: Extends the authority to issue priority review vouchers for rare pediatric disease treatments until September 30, 2029. It also includes a GAO study on the effectiveness of these vouchers in incentivizing drug development.
• Orphan Drug Exclusivity: Limits exclusive approval or licensure of orphan drugs to the specific "approved use or indication" within a rare disease, rather than the entire disease.
• Pediatric Studies of Drugs Program: Provides \$25,000,000 for each of fiscal years 2025 through 2027.
• Organ Procurement and Transplantation Network: Encourages integration of electronic health records and considers establishing a dashboard to display transplant statistics. The bill also allows the Secretary to collect registration fees from members of the Organ Procurement and Transplantation Network.
• Abraham Accords Office: Establishes an Abraham Accords Office within the FDA to provide technical assistance to regulatory partners in Abraham Accords countries and facilitate interactions between the FDA and interested parties in those countries.

In short, this bill aims to encourage the development of new treatments for pediatric cancer and rare diseases, ensure compliance with pediatric study requirements, and improve the organ transplantation system.