Congress Must Reauthorize FDA Program for Pediatric Rare Diseases to Save Lives

1. Creating Hope Act: The Creating Hope Act incentivizes the development of new drugs to treat childhood cancers and other rare pediatric diseases. It has led to the development of nearly four dozen pediatric rare disease medicines.
2. Priority Review Voucher Program: The program awards vouchers to companies that successfully develop medicines for rare pediatric diseases. These vouchers can be used to receive priority review for another product or sold to another company to fund further research.
3. Expiration Date: The program is set to expire in September 2024, and without reauthorization, it will no longer be able to issue new vouchers, potentially halting the development of new treatments for rare pediatric diseases.
4. Impact: The program has been instrumental in bringing new treatments to market, with 95% of rare diseases lacking FDA-approved treatments. Reauthorization is crucial to continue this progress.
5. Bipartisan Support: The Creating Hope Reauthorization Act of 2024 has bipartisan support from U.S. Representatives Gus Bilirakis (R-FL), Anna Eshoo (D-CA), Michael McCaul (R-TX), Lori Trahan (D-MA), Michael Burgess (R-TX), and Nanette Barragán (D-CA).
6. Patient Advocacy: Over 130 patient advocacy organizations have urged swift reauthorization of the program to ensure continued hope for children living with rare diseases.