Program boosting rare disease treatments set to expire

The Food and Drug Administration’s (FDA) Rare Pediatric Disease Priority Review Voucher (RPD PRV) program awards companies that develop rare pediatric disease therapies vouchers for quicker FDA review of any new product. The program has been pivotal in encouraging the development of new therapies for primary immunodeficiency (PI) and will expire on September 30, 2024, unless Congress passes the Creating Hope Reauthorization Act.

Legislative history and challenges

Congress first established the priority review voucher program in 2007 to encourage companies to create new treatments for neglected tropical diseases like malaria and tuberculosis. Based on a 2006 Health Affairs paper, the program awarded developers of treatments for tropical diseases with a "priority review voucher." PRVs speed up the FDA’s review of any future medical treatment or device, including those outside the scope of the voucher program. According to the Congressional Research Service Report, on average, vouchers shorten the time it takes to receive the FDA’s approval decision by four months. Once obtained, PRVs can also be sold to a third party at an average price of $100 million based on an Orphanet Journal of Rare Diseases study.

In 2012, the Creating Hope Act expanded the program to include rare pediatric disease therapies, given the lack of existing treatments for children affected by rare diseases. The FDA defines a rare pediatric disease as a serious or life-threatening condition that affects 200,000 or fewer individuals in the U.S., primarily from birth to the age of 18. A condition is also eligible for the RPD PRV program if it affects more than 200,000 people, but there is no reasonable expectation that the cost of developing a treatment will be recovered from sales of the treatment.

Since it began, the RPD PRV program has been reauthorized in 2016 and 2020 with bipartisan support. However, unless Congress reauthorizes the Creating Hope Act again, the program will expire on September 30, 2024.

The main obstacle to reauthorization is the limited time remaining in 2024 to pass bills. Some legislators concerned about renewing the bill argue that companies should not benefit from subsidy programs funded with taxpayer dollars, given recent record profits in the pharmaceutical industry. In addition, critics cite a 2019 Government Accountability Office report on the FDA’s PRV programs questioning their effectiveness based on data collected during RPD PRV's first seven years. However, drugs in development often take 12 years or more to earn FDA approval. A recent National Organization for Rare Disorders (NORD) study that uses data through April 2024 shows that the program’s impact has sharply increased over time, with more than half of the designations, awards, and redemptions occurring after 2017.