Priority Review Voucher - Extension To Rare Pediatric Diseases

Extension To Rare Pediatric Diseases

In 2012, President Obama signed into law the FDA Safety and Innovation Act which includes Section 908 the "Rare Pediatric Disease Priority Review Voucher Incentive Program". The act extends the voucher program to rare pediatric diseases, but only on a trial basis. One year after the third pediatric voucher is awarded, no other pediatric vouchers may be awarded. The extension to rare pediatric diseases was championed by Nancy Goodman of Kids v Cancer.

The pediatric voucher program includes several changes that had been sought for the neglected-disease voucher program, but apply only to pediatric vouchers. First, the pediatric treatment developer can ask the FDA in advance for an indication of whether the disease qualifies as a rare, pediatric disease. Second, the pediatric voucher can be transferred an unlimited number of times, whereas the neglected-disease voucher can only be transferred once. Third, the pediatric voucher user needs to notify FDA 90 days prior to using the voucher, rather than 1 year for the neglected-disease voucher. Fourth, the pediatric voucher winner risks having the voucher revoked if the treatment is not marketed within a year. Fifth, the pediatric voucher winner must report to FDA about use of the pediatric treatment within five years of approval.