U.S. Senator Tim Scott led a letter on Mar. 27 to the Food and Drug Administration (FDA) asking the agency to reconsider its earlier denial of a Pediatric Rare Disease Priority Review Voucher for a gene therapy treating sickle cell disease. Representatives Gus Bilirakis, Mike Kelly, Rick Allen, and Buddy Carter joined Scott in signing the letter.
The lawmakers said their request is important because sickle cell disease affects about 100,000 Americans, with more than 90% being African American. They argued that supporting new treatments could help address this serious health issue.
“We write regarding the administration of the Food and Drug Administration’s (FDA’s) Pediatric Rare Disease Priority Review Voucher (PRV) program, particularly the decision by the previous administration to deny a PRV for a one-time administered novel curative gene therapy that treats sickle cell disease,” they wrote in their letter.
The group continued, “This chronic, debilitating disease causes severe pain episodes often resulting in hospitalization, progressive organ damage, and serious acute events like strokes… The approvals in December 2023 of two curative gene therapies represented a transformative moment for Americans…living with SCD.”
They also criticized how the FDA interpreted eligibility rules for these vouchers: “The previous administration ignored this guidance, inexplicably choosing to treat the modified cell types of patients with Betathalassemia and those with SCD as the same ‘active ingredient.’” The lawmakers warned that upholding this approach “risks jeopardizing the viability of an industry intent on creating novel treatments for pediatric patients with sickle cell disease and other rare, severe genetic diseases.”
Scott grew up in a single-parent household in North Charleston and was raised there as well according to his official website. He obtained his Bachelor of Science degree from Charleston Southern University according to his official website. Before serving as senator, he held positions on Charleston County Council as well as in both state and federal legislatures according to his official website.
In closing their letter about FDA policy on rare pediatric diseases incentives established by Congress in 2012—meant to encourage drug development without taxpayer cost—the lawmakers said reversing this decision would restore those incentives: “Revisiting, and ultimately reversing, this decision… will further support [ongoing efforts to advance gene therapies]…and do so at no cost to the federal government.”
Scott offers constituent services including help with federal agencies and community resources for South Carolina residents according to his official website.
