Will FDA's Accelerated Approval Pathway Facilitate Access to Gene Therapies as Applications Roll In?

Brigid Bondoc spoke to Genomeweb about the head of the U.S. Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research expressing a desire to shepherd more gene therapies for rare diseases to market through the accelerated approval pathway.

“Understanding [the FDA’s] long-term safety monitoring expectations is something that the field has always struggled with,” Brigid said. But this is precisely the promise of gene therapies, which are expected to have long-term, durable, and curative benefits. These therapies could, theoretically, be on the market for many years before confirmatory data are available, which Brigid noted is a common criticism of the accelerated approval pathway.

Brigid added: “[The FDA] will be under a lot of pressure to approve the therapies, but public trust is also on the line. I don’t envy the position that they’re in.”

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