Sen. Scott Urges FDA to Address Gene Therapy Priority Voucher Discrepancy

WASHINGTON — In order to help increase treatment for sickle cell disease (SCD), U.S. Senator Tim Scott (R-S.C.) penned a letter to the Food and Drug Administration (FDA) Commissioner Robert Califf raising concerns over how the FDA interprets “active ingredient” for awarding priority review vouchers, which could negatively impact drug development for rare diseases like sickle cell disease. Senator Scott was joined on the letter by Representative Danny Davis (D-Ill.).

“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,” Senator Scott and Representative Davis wrote.

They continued, “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.”

“However, the decision whether to narrowly or broadly interpret the definition of “active ingredient” for purposes of awarding a PRV is not so much a question of science as it is one of policy. Congress has not provided guidance as to how FDA should determine if two active ingredients in an ex-vivo gene therapy are the same or different, but the text of the statute and the legislative history make clear that Congress wanted to create an incentive to treat patient populations that might not warrant investment if left simply to ordinary market forces. 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, jeopardizing not only the availability of SCD treatments today, but also investments in complex and life-saving innovation for the future,” stated Scott and Davis.

“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, and we are grateful for the FDA’s close attention to this matter. We hope that you will keep these additional considerations in mind as you conduct your review,” they concluded.

Read the full letter here!

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