AABB Urges Congress to Reauthorize Rare Pediatric Disease Priority Review Voucher Program

AABB joined a coalition of health care organizations in urging Congressional leaders to advance the Mikaela Naylon Give Kids a Chance Act (S.932/H.R.1262), which would reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program to support the development of innovative therapies for children with serious and rare diseases.

In a letter to Senate leadership, coalition partners emphasized that the Food and Drug Administration program is a proven and vital incentive for the commercial development of therapies for children who currently have few or no therapeutic options. They noted that it has been particularly effective as a pathway to encourage private investment in early-stage cell and gene therapy (CGT) programs, with 63 PRVs awarded across 47 rare pediatric diseases. Prior to the creation of the program, only four of those diseases had an FDA-approved treatment.

AABB and its coalition partners also highlighted that the program has broad stakeholder support, including FDA, advocacy organizations, academia and therapeutic developers, and underscored that the PRV program is a strong example of a policy that aligns incentives to advance therapies for children while strengthening the broader research and development ecosystem.