Policymakers Urge FDA to Address Gene Therapy Priority Voucher Discrepancy

Sen. Tim Scott (R-S.C.) and Rep. Danny Davis (D-Ill.) recently sent a letter to the Food and Drug Administration Commissioner Robert Califf, MD, urging the agency to reconsider its approach to awarding priority review vouchers (PRVs) under the Rare Pediatric Priority Review Voucher program. The letter highlighted concerns that FDA’s interpretation of “active ingredient” may restrict access to PRVs for treatments such as gene therapies, which could hinder drug development for rare diseases like sickle cell disease (SCD).

Scott and Davis emphasized that FDA’s current interpretation may potentially undermine Congressional intent to incentivize drug development for underserved patient populations. They noted that a narrow interpretation of “active ingredient” recently led to the denial of a pediatric PRV, which could jeopardize future investments in rare disease treatments. The letter expressed appreciation for FDA’s attention to the issue and encouraged the agency to carefully consider the broader implications of its interpretation for SCD patients and the future of rare disease innovation.