Analysis Examines First 10 Years of Rare Pediatric Disease Priority Review Voucher Program

Rare Daily Staff

A timely analysis in the Orphanet Journal of Rare Disease offers a ten-year analysis of the U.S. Food and Drug Administration’s Rare Pediatric Disease Priority Review Voucher program, an effort to provide a financial incentive to encourage drug developers to pursue treatments for rare pediatric diseases.

The analysis, conducted by staff of the FDA’s Office of Orphan Products Development, comes as the program is set to expire September 30, 2024, unless Congress reauthorizes it. Congress enacted the Rare Pediatric Disease Priority Review Voucher program in 2012. Under the program, the FDA grants Rare Pediatric Disease designation to new treatments for serious or life-threatening diseases that primarily affect children ages 18 years or younger with fewer than 200,000 people affected in the United States.

The program allows for a sponsor who receives an approval to qualify for a priority review voucher that can be redeemed to receive an expedited six-month priority review for any subsequent marketing application or may be sold or transferred.

The vouchers have been an important source of non-dilutive funding to rare disease drug developers. Companies have typically been able to sell these vouchers for around $100 million, although prices have ranged from $65 million to $350 million.

The study found that a total of 569 therapies won Rare Pediatric Disease Designation between 2013 and 2022. The top indications were neurology (26 percent) metabolism (23 percent), and oncology (18 percent). There was a five-fold surge in designations in 2020 when the designations were last set to expire.

The study found a total of 245 unique diseases were represented in the Rare Pediatric Disease designations. A total of 26 diseases had five or more Rare Pediatric Disease designations associated with them. Duchenne muscular dystrophy, neuroblastoma, and sickle cell disease were the indications with the most therapies to receive the designation. Only 6 percent of the designations were for therapies that targeted neonatal indications.

A little more than half of the designations were for drugs. Gene therapies account for 28 percent of the designations.

While 38 vouchers have been awarded through the program, the long development time for therapeutics means it will be sometime before it is clear what percentage of these therapies reach market. The authors, which relied on an internal database for their study, did not seek to gather data on the sales of these vouchers. That, though, would speak to a very concrete benefit of these designations.