On September 14, a bipartisan coalition of Representatives introduced the Priority for Pediatric Cures Act, which would make the Rare Pediatric Disease Priority Review Voucher (PRV) Program permanent. Currently, the PRV Program will expire in September 2029. This bill eliminates the expiration date, ensuring that the Rare Pediatric Disease PRV Program is protected and established as a permanent program.
The Rare Pediatric Disease Priority Review Voucher (PRV) Program incentivizes pharmaceutical companies to develop treatments for rare pediatric diseases. After a treatment with a Rare Pediatric Designation is approved by the FDA, the company is issued a PRV that can be used to obtain priority review for a treatment that wouldn’t otherwise qualify. Priority review means a treatment is generally reviewed by the FDA within a 6-month period rather than the standard 10-month period.
About 70% of rare diseases are exclusively of pediatric onset, and overall, 95% of rare diseases have no approved treatments. Developing drugs for rare pediatric diseases is particularly challenging due to the small populations affected, difficulties associated with conducting clinical trials for children, delays in diagnosis, and more. The Rare Pediatric Disease PRV Program is essential for children with rare diseases. Since the program’s creation in 2012, over 70 Rare Pediatric Disease PRVs have been issued for innovative treatments in over 60 diseases.
The lack of permanence for the Rare Pediatric Disease PRV Program has lasting consequences. Recently, the Program’s authorization lapsed, forcing some developers to delay and/or halt their clinical trials. Though the Program was reauthorized by Congress at the beginning of this year, the lapse highlighted the need for a permanent program.
Creating permanence removes politics from a program that has wide bipartisan support, ensuring its reauthorization doesn’t get caught up in delays or disagreements over other issues. It also brings the Rare Pediatric Disease PRV Program in alignment with the Tropical Disease PRV Program, which has been permanent since its origin in 2007. However, permanence does not mean there won’t be opportunities for Congressional oversight in the future, a common reason why some programs require regular reauthorization.
It can take up to 15 years to develop a new treatment, but decisions about investments that enable clinical trials are made very early in the process. Making the PRV Program permanent will ensure these decisions are made, knowing the PRV Program will be intact if they reach approval. After almost 15 years of the program, we know it works, and constant reauthorizations and lapses are inefficient for advocates, Congress, and developers.
The RARE Foundation would like to thank Rare Disease Congressional Caucus Co-Chair, Representative Gus Bilirakis (R-FL), as well as Representatives Nanette Barragán (D-CA), Kat Cammack (R-FL), Jake Auchincloss (D-MA), Tom Kean, Jr. (R-NJ), and Kevin Mullin (D-CA) for leading this bill. Learn more about the Rare Pediatric Disease PRV Program on our website.
For families facing a rare pediatric disease, time isn't a luxury. The Rare Pediatric Disease Priority Review Voucher Program has proven to be one of the most effective tools we have for enabling companies to translate promising science into new treatments. Developing a treatment for a devastating pediatric disease takes years, sometimes decades. Making the PRV permanent gives drug developers and the patient communities counting on them the certainty to make that long-term bet. The RARE Foundation, formerly known as the EveryLife Foundation for Rare Diseases, is grateful to Representatives Bilirakis, Barragán, Cammack, Auchincloss, Kean, and Mullin for introducing the Priority for Pediatric Cures Act so that we can build on the incredible momentum of the first 14 years of the PRV Program, which has enabled over 70 new treatments for pediatric rare diseases
Annie Kennedy
Chief Mission Officer, RARE Foundation
