EASING GENE THERAPIES — The FDA is taking multiple approaches aimed at getting innovative gene therapies to more rare disease patients faster and helping biotechs recoup their research and development costs, a senior agency official said Monday. Manufacturing challenges and clinical development timelines have prevented biotechs making gene therapy candidates from “quick wins,” Dr. Peter Marks, director of the agency’s Center for Biologics Evaluation and Research, said at an Alliance for a Stronger FDA meeting. The group advocates for congressional appropriations to the FDA. The small patient pool for many gene therapies constrains drug companies’ ability to profit from their innovations. Marks said the FDA is working with other public and private partners to make developing and manufacturing gene therapies more efficient. Agency interaction: The FDA aims to have regular discussions with gene therapy manufacturers about products in development. In January, the agency will request companies to participate in a pilot program, with CBER working on three products targeting rare diseases. “We will give these products the same type of service that we did during [the pandemic’s] Operation Warp Speed,” Marks said. The agency is also considering ways to collaborate with its counterparts in other high-income locations, such as in Europe, to harmonize their approaches to gene therapies, Marks said. Regulatory red tape in certain countries has kept some companies from seeking approvals, he said, which limits their market and makes it harder to recoup costs. “You might be able to bring benefit to more people globally” and help biopharma drug developers if regulatory work didn’t have to be duplicated across borders, Marks said. GENE THERAPY FEEDBACK — Senate HELP Committee ranking member Bill Cassidy (R-La.) will request information today on bolstering access to gene therapies, Ben reports. The move comes as the FDA is expected to decide by the end of the week whether to approve the nation's first gene-editing therapy. Cassidy wants feedback from industry players for crafting future legislation to ensure commercial insurance can cover the pricey treatments. What’s next: Cassidy will take comments through Jan. 22. DRUG SHORTAGES IN THE SPOTLIGHT — Pharmaceutical supply experts and generic manufacturers will testify before the Senate Finance Committee today about drug shortages, which have struck treatments for a number of conditions, including cancer, diabetes and ADHD over the past year. “Shortages disproportionately affect low-cost therapeutics, which operate in a largely different — and increasingly challenging — economic environment,” said ranking member Mike Crapo (R-Idaho) in opening remarks. “The average shortage affects at least half a million consumers, forcing them to scramble for viable alternatives or else forgo treatment entirely.”
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