The Sunsetting Of Rare Pediatric Disease Designation

By Joseph Watson

As with other incentive programs, FDA created the Rare Pediatric Disease (RPD) designation to encourage drug development in products with questionable financial viability; in this case, the treatment of certain rare pediatric diseases.  One of the main benefits of RPD designation is the potential to receive priority review vouchers, which can be used to obtain priority review on a subsequent human drug or biologic application, should the product with RPD designation be approved.  Priority review vouchers have few restrictions, and can be used by the sponsor or sold/transferred to a separate organization.  In some cases, priority review vouchers have sold for over $100 million.