Below is the official letter addressed to key leaders in the U.S. Senate and House of Representatives urging support for the Give Kids a Chance Act and the reauthorization of the Rare Pediatric Priority Review Voucher program.
September 10, 2025
Dear Majority Leader Thune, Minority Leader Schumer, Speaker Johnson, and Minority Leader
Jeffries, Chair Guthrie, and Ranking Member Pallone,
On behalf of the 30 million Americans living with a rare disease, the undersigned 190 organizations write to express strong support for the Give Kids a Chance Act of 2025 (H.R. 1262/S.932) and urge its swift passage by Congress. This urgent legislation would reauthorize the highly effective Rare Pediatric Disease Priority Review Voucher (PRV) program for five years. As many as half of the individuals living with a rare disease are children, and this program offers a crucial incentive to develop therapies for this particularly challenging-to-study patient population living with devastating and often life-threatening rare conditions.
Nearly 70% of rare diseases start in childhood1 and most have no approved treatments. The Rare Pediatric Disease PRV program, created by Congress in 2012, has been a vital catalyst for developing therapies for these vulnerable populations. Thanks to this program, treatments have reached children suffering from nearly 40 rare diseases, many of which previously had no FDA-approved options and led to severe disability or death before adulthood.2
The program’s impact is accelerating – more than half of all Rare Pediatric Disease designations occurred in just the past four years,3 demonstrating its growing importance in addressing urgent unmet medical needs. Yet despite this progress, over 95% of rare diseases still lack an FDA approved therapy.
The Rare Pediatric Disease PRV program’s authorization expired on December 20, 2024. Without reauthorization, newly designated investigational therapies are ineligible for PRVs. Those promising therapies that received Rare Pediatric Designation before the December 20th expiration, but do not receive FDA-approval before September 30, 2026 are also ineligible for a PRV. Allowing this program to lapse not only eliminates a proven pathway for future innovation and hope, but it also creates uncertainty for scientific work currently underway that will not meet the September 30, 2026 deadline.
Importantly, the PRV program is a market-based incentive that comes at no cost to taxpayers. The data shows that the Rare Pediatric Disease PRV program is working to address unmet needs and will continue to do so for years to come when reauthorized.
We urge Congress to act quickly and pass the Give Kids a Chance Act of 2025 (H.R. 1262/S.932) to restore this life-saving program and ensure continued progress in rare pediatric drug development. Our communities are counting on you. We look forward to working with you on this important issue.
For any questions or concerns, please contact Allison Herrity at the National Organization for Rare Disorders, at aherrity@rarediseases.org and Jamie Sullivan at the EveryLife Foundation for Rare Diseases, at jsullivan@everylifefoundation.org.
Thank you for your leadership and commitment to improving the lives of children with rare diseases.
This letter was signed by 190 patient advocacy organizations.
Footnotes:
1. Nguengang Wakap S, Lambert DM, Olry A, Rodwell C, Gueydan C, Lanneau V, et al. Estimating cumulative point
prevalence of rare diseases: analysis of the Orphanet database. Eur J Hum Genet. 2020;28(2):165–73.
https://www.nature.com/articles/s41431-019-0508-0
2. See: https://rarediseases.org/wp-content/uploads/2024/05/NORD_PRV-white-paper_FINAL.pdf
3. Mease, C., Miller, K. L., Fermaglich, L. J., Best, J., Liu, G., & Torjusen, E. (2024). Analysis of the first ten years of
FDA’s rare pediatric disease priority review voucher program: designations, diseases, and drug
development. Orphanet J Rare Dis. 2024;19(1):131. https://pubmed.ncbi.nlm.nih.gov/38403586/
