Introduced this week in Congress by Representatives Michael McCaul (R-TX) and Debbie Dingell (D-MI). This critical piece of bipartisan legislation includes a five-year reauthorization of the Rare Pediatric Priority Review Voucher (PRV) Program and policies to increase access to pediatric cancer clinical trials and clarify the application of the Orphan Drug Act’s exclusivity provisions. The urgency of Congressional action cannot be overstated. In late December 2024, following heroic advocacy efforts by the rare disease and pediatric cancer communities, a bipartisan healthcare policy package, including a five-year reauthorization of the Rare Pediatric Priority Review Voucher (PRV) Program passed by the House in September 2024, was set to be included in the end-of-year government funding extension. Unfortunately, most healthcare policies were excluded from the final bill, including the PRV Program’s reauthorization. As a result, the PRV Program expired on December 20, 2024.
The PRV Program is a pivotal incentive mechanism that has encouraged the development of treatments for rare diseases that predominantly affect children. Given that 70 percent of the 10,000 known rare diseases originate in childhood and a mere five percent of rare diseases currently have FDA-approved treatments, the expiration of this program will significantly hinder progress in a field where the stakes are incredibly high.
The PRV Program has been a beacon of hope for the rare disease community since its expansion to rare pediatric diseases in 2012. With 59 Rare Pediatric Disease PRVs issued to date, leading to treatments for 44 rare disease communities, its impact is undeniable and growing steadily. Over half of all PRVs awarded since 2012 have been in the last five years.
By advocating for the reauthorization of the PRV Program, EveryLife Foundation continues to advocate for the 30 million Americans living with a rare disease, particularly the children who face these devastating diagnoses. Proactively engaging with lawmakers illustrates our mission to advance solutions that can bring hope and potentially life-saving treatments to the rare disease community.
EveryLife Foundation extends our profound appreciation to Representatives McCaul and Dingell and the original co-sponsors who have joined them in introducing this critical legislation, Representatives Gus Bilirakis (R-FL), Doris Matsui (D-CA), Kim Schrier (D-WA), Diana Harshbarger (R-TN), Kathy Castor (D-FL), Mike Kelly (R-PA), Dan Crenshaw (R-TX), Lori Trahan (D-MA), and Randy Weber (R-TX)
As the EveryLife Foundation prepares to host over 800 rare disease advocates in Washington, DC, during Rare Disease Week beginning on February 24, we remain hopeful that Congress will act swiftly to pass the Give Kids a Chance Act, ensuring the continuation of a program that has already made a profound difference in the lives of many without imposing new costs on taxpayers.
