The rare disease community realized an important policy victory as Congress approved a four year extension of the Rare Pediatric Disease Priority Review Voucher (PRV) Program, capping off a year of community driven advocacy for the Creating Hope Reauthorization Act. The EveryLife Foundation for Rare Diseases applauds the critical Congressional action that led to the extension of this innovative and cost-effective program. The PRV Program has been a powerful incentive for the development of treatments for rare diseases and has enabled life-saving treatments to reach children with rare diseases faster without adding new costs to taxpayers. We are thankful for the work of the Creating Hope Reauthorization Act’s co-sponsors, G.K. Butterfield, Senator Bob Casey and Senator Susan Collins. The PRV extension was included in the package of COVID-19 relief and government funding bills passed by Congress and sent to the President on December 21.
Children and families with devastating and life-threatening rare diseases have long supported the PRV program. Approximately 93% of the 7,000 rare diseases have no treatment options yet advances in novel technology platforms for therapy development mean that for many, a treatment could be within reach if the right incentives and regulatory pathways exist to make it financially viable for companies. Earlier this month, EveryLife sent a letter to Congressional leadership urging them to complete work on the four year extension and sharing with them just one example of the PRV’s transformative potential for future rare disease treatment development.
The EveryLife Foundation is also grateful for the leadership of our partners, Kids v. Cancer, who never let up the fight to ensure an extension was secured and we are grateful to all the rare disease advocates who placed calls, sent emails and held meetings during Rare Across America in August to bring attention to this issue. We encourage you to send an email letting your members of Congress know that you are grateful for their support of the PRV extension. This RDLA Action Alert makes it simple to do.
We know that the PRV extension is only one piece of the puzzle and many pieces still remain in order to meet the needs of the millions of rare disease patients in the US. As a challenging year comes to a close, we are pleased to see this victory and we look forward to working with Congress, the incoming Administration and the rare disease community to advance more solutions in the new year.
