As a parent and advocate for children with rare diseases, I recently traveled to Capitol Hill to urge Congress to reauthorize the FDA’s Priority Review Voucher (PRV) program. This critical initiative incentivizes pharmaceutical companies to develop treatments for pediatric rare diseases by offering a valuable voucher upon approval, which can be used to expedite the review of another drug application.
The PRV program has been instrumental in bringing new therapies to market for children with life-threatening rare conditions. Since its implementation, over 30 vouchers have been awarded, leading to the development of treatments for diseases such as Duchenne muscular dystrophy and spinal muscular atrophy. However, the program is set to expire on September 30, 2024, putting future progress at risk.
Congress must act now to reauthorize the PRV program and ensure that children with rare diseases continue to have hope for new treatments. By extending this vital initiative, we can encourage ongoing investment in pediatric rare disease research and provide a lifeline for families desperately seeking effective therapies for their children.
