Rare Pediatric Disease & Priority Review Vouchers
BY JAMES ROMANO
What is the Future of the Rare Pediatric Disease Designation and Priority Review Vouchers?
On December 21st , 2024, Congress finalized, and Former President Biden signed into law, the Continuing Resolution to fund the government of the United States until March 20th, 2025.
During the lame-duck session, Congress needed to reauthorize several health care programs that were set to expire. The bill that was crafted by the House and Senate was then torpedoed by President-elect Trump and Elon Musk. To avoid a government shut down, Congress passed a three-month Continuing Resolution funding agencies at the existing levels until the budget is negotiated and passed in hopefully March. However, legislation that needed to be re-authorized like the Rare Pediatric Disease Priority Review Vouchers (PRV) were not included and are in danger of closing.
The RPV Program works to incentivize treatment development for rare diseases focused on children. Created in the bipartisan 21st Century Cures Act (Public Law 114-255), the program allows manufacturers who have taken treatment through the clinical trial process to approval to qualify for a voucher that can be redeemed to receive priority review at the Food and Drug Administration (FDA) for a different product. The FDA awards these rare disease vouchers on pediatric disease products that are approved.
The Voucher allows the manufacturer to fast-track the FDA review process for a different drug application by the holder. A manufacturer must request a voucher prior to submitting their application for its drug. Manufacturers are allowed to sell or transfer the vouchers. Recently Acadia Pharmaceuticals sold their PRV for $150 million.
Since the program was not renewed in December, currently the program is expired. Unless it is included in the budget negotiations for the FY 2025, appropriations the program will come to an end. This will remove a program that is dedicated to incentivizing and assisting in the research of treatments for children with rare diseases. You can reach out to your Member of Congress and ask them to include the reauthorization of the Rare Pediatric Disease Priority Review Vouchers (PRVs) in the final budget.