The Rare Disease Company Coalition (RDCC), with support from IQVIA, investigated the history and impact of the rare pediatric disease priority review voucher (PRV) program. The findings clearly demonstrate that this program is a proven incentive that has spurred the development of new innovations that benefit more than 200,000 patients across 47 rare pediatric indications. But while there remains an urgent unmet medical need for new treatments for rare pediatric diseases, the future of this vital tool is uncertain.
Without Congressional action, the program will begin to expire on September 30, 2024. Expiration of the rare pediatric disease PRV program would result in reduced incentives for the discovery of new treatments, jeopardize programs already under development, and destabilize investment in new treatments. Preservation is necessary to further investment in and development of new rare disease treatments.
The RDCC applauds Congress for the significant progress in improving the lives of children with rare disease through the creation and subsequent reauthorization of the Rare Pediatric Disease PRV program in 2012, 2016 and 2020, respectively. A long-term extension of the PRV program will support the stability of research & development and ultimately lead to new treatment advances for rare pediatric diseases.