The EveryLife Foundation for Rare Diseases applauds the introduction of The Creating Hope Reauthorization Act of 2024, introduced this week in Congress by Representatives McCaul (TX-10), Bilirakis (FL-12), Burgess (TX-26), Barragán (CA-44), Eshoo (CA-16), and Trahan (MA-3). This critical piece of legislation seeks to extend the Rare Pediatric Priority Review Voucher (PRV) Program for an additional four years, a move that promises to catalyze further advancements in rare disease treatments without imposing any new costs on taxpayers.
The urgency of Congressional action cannot be overstated. With the current Rare Pediatric PRV Program set to expire on September 30, 2024, there is a palpable risk of losing a pivotal incentive mechanism that has encouraged the development of treatments for diseases that predominantly affect children. Given that a mere five percent of rare diseases currently have FDA-approved treatments, the expiration of this program could 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 49 rare PRVs issued as of January 2024, leading to treatments for nearly 40 rare disease communities, its impact is undeniable. Diseases such as spinal muscular atrophy, sickle cell disease, and Rett syndrome, which previously had no treatment options, have seen tangible progress thanks to this program.
In a letter applauding the leadership of the representatives leading the bill, the EveryLife Foundation highlighted the successes achieved thus far and the potential consequences of allowing the program to lapse. The Foundation emphasizes the unique challenges in developing treatments for rare pediatric diseases, including small population sizes, the complexities of clinical trials in children, and the often-delayed diagnosis of these conditions.
By advocating for the extension of the PRV Program, EveryLife Foundation is championing the cause of the 30 million Americans living with a rare disease, particularly the children who face these devastating diagnoses. The Foundation’s proactive engagement with lawmakers reflects its unwavering commitment to advancing solutions that can bring hope and potentially life-saving treatments to the rare disease community.
EveryLife Foundation for Rare Diseases is grateful for the leadership and dedication of the lawmakers who support the Creating Hope Reauthorization Act. As the Foundation prepares to host over 600 rare disease advocates in Washington, DC during Rare Disease Week, we remain hopeful that this vital legislation will be enacted, ensuring the continuation of a program that has already made a profound difference in the lives of many.
Priority Review Voucher – One Pager
