Advocates Increase Focus on PRV as Deadline to Reauthorize Approaches

Back in February, rare disease organizations applauded the introduction of legislation to reauthorize the Rare Pediatric Priority Review Voucher program and urged its prompt passage. The current program is set to expire September 30, 2024.

If the program fails to win reauthorization, no new drugs will be able to be designated a rare pediatric disease therapy after that date, and priority review vouchers will cease being awarded to such designees as of September 30, 2026.

A bill in the House would extend the current program an additional four years. A Senate version of the legislation would extend the program to 2030.

At a time when drug developers have faced a challenging environment to secure funding, the program has proved to be not just a welcome incentive to drug developers, but a powerful source of capital to advance rare disease drug development.

Congress created the Rare Pediatric Disease Priority Review Voucher program in 2012. It established an incentive for pharmaceutical companies to develop novel treatments for rare pediatric diseases. Companies that won approval for a rare pediatric disease therapy are eligible for a priority review voucher that can be used to reduce the time on any new drug application to six months from the standard 10-month review.

Because the vouchers are transferable, they are potentially lucrative. Companies that have received priority review vouchers have sold them for between $67.5 million and $350 million, although they have typically sold for around $100 million each. Though such sales are reported at the discretion of companies, that’s potentially proved to be a source of billions of dollars to drug developers that can, in turn, use it to help drive the development of new therapies.

The Rare Pediatric Disease Priority Review Voucher program has been reauthorized with bipartisan support twice since its inception. Since 2012, this program has awarded 53 vouchers for 39 rare pediatric diseases that have led to innovations benefitting over 200,000 patients. Some 36 of the rare pediatric diseases had no previously approved therapies on the market at the time of approval.

As we move toward the other side of Labor Day, efforts around the renewal will likely pick up as advocates and industry fight to get the program reauthorized. Already, op-eds have started to crop up, and the National Organization for Rare Disorders just issued a new report on the Rare Pediatric Disease Priority Review Voucher Program.

The program has done a lot of good to incentivize the pursuit of rare disease therapies. At a time when funding has become harder for companies to access, the vouchers not only provide an incentive but an important source of capital. It’s not just a reward to successful companies that bring these drugs to market, but critical funding to enable them to pursue additional treatments.