April 2015

This week, the ATS joined the National Organization for Rare Diseases (NORD) and other organizations in a letter urging Congress to renew a program to incentivize pediatric rare disease research, called the Rare Pediatric Disease Priority Review Voucher Program (RPDPRVP).

The RPDPRVP program was created in 2012 to encourage development of new therapies for children with rare diseases. Under the program, upon FDA approval of a new rare pediatric disease treatment, a biopharmaceutical manufacturer has the opportunity to receive a voucher guaranteeing a six month priority review of a New Drug Application (NDA) or Biologic License Application (BLA) for another product. Since its creation, the RPDPRVP has awarded three vouchers for new therapies, showing that it is an effective incentive for the development of new treatments for rare pediatric diseases. The RPDPRVP must be renewed by Congress before it expires in March 2016.