FDA Gene Therapy Head Raises Concern on Accelerated Approval Use (1)

More cell and gene therapies will likely come to market using FDA’s accelerated approval pathway, a shift that raises concerns for the agency’s outgoing gene therapy head since it could take a while to prove definitively that they work.

“If it takes years as suggested to tell if something works, then that means it takes years to tell if it doesn’t work,” Wilson Bryan said at the Biopharma Congress on Monday. The meeting was hosted by Prevision Policy and Friends of Cancer Research.

If five or 10 years after the accelerated approval, the confirmatory evidence either shows the drug doesn’t ...