Palmetto State News

U.S. Senator Tim Scott, alongside Representative Danny Davis, has addressed a letter to FDA Commissioner Robert Califf concerning the agency's interpretation of "active ingredient" in awarding priority review vouchers (PRVs). This interpretation could potentially affect drug development for rare diseases such as sickle cell disease.

The letter highlights concerns regarding the FDA's Rare Pediatric Priority Review Voucher program and its application to gene therapies. “We write regarding the administration of the Food and Drug Administration’s (FDA’s) Rare Pediatric Priority Review Voucher (PRV) program, particularly as it applies to innovative treatments like gene therapies,” stated Scott and Davis.

They further expressed concern over the FDA's recent decisions within this program. “However, we are concerned that FDA’s recent administration of the program may not be fulfilling the program’s promise, original vision, and intent. Specifically, we understand that FDA has made an initial decision to narrowly interpret the definition of 'active ingredient' in the PRV statute, resulting in the unexpected denial of at least one pediatric PRV.”

Scott and Davis argued that interpreting "active ingredient" is more about policy than science. They noted Congress had not specified how to determine if two active ingredients in an ex-vivo gene therapy are different or identical but intended to incentivize treatment development for underserved patient populations. “By interpreting this statutory requirement in a narrower way than the statute requires – and one that flies directly against Congressional intent – we risk disrupting this delicate ecosystem," they warned.

The lawmakers acknowledged that FDA might reconsider its interpretation. “We understand that FDA may be in the process of reconsidering its interpretation. This decision is critical for the future of rare disease drug development and rare disease patients," they concluded.