CBER director Peter Marks says FDA is growing to keep up with cell and gene therapies
SOMERVILLE, MA — CBER director Peter Marks spelled out FDA’s plans to keep up with the fast-growing cell and gene therapy space on Tuesday, while also advocating for the use of accelerated approvals.
“This area has grown so much over the past decade that we have not grown with it,” Marks said at Charles River’s Cell & Gene Summit Tour just outside of Boston. That’s about to change, he added, as the FDA officially transitioned on Monday from its Office of Tissues and Advanced Therapies (OTAT) to the Office of Therapeutic Products (OTP).
Six new offices live under the OTP, including the Office of Gene Therapy Chemistry, Manufacturing and Controls (CMC), and the Office of Cellular Therapy and Human Tissue CMC. The move should boost the agency’s focus on manufacturing and “help get us to better timeliness of response and, importantly, also better consistency of response,” Marks said.
“It’s fair criticism that we have not given timely feedback,” he added. With a new communications pilot, Marks said “the idea is to take something that’s early in their clinical development that’s showing promise for a disease that is life-threatening, and see if we give it that same Warp Speed-like attention, can we get it over the finish line faster?”
He also spoke in favor of the FDA’s accelerated approval process for certain rare or serious diseases. The pathway allows regulators to issue a faster approval based on a surrogate endpoint while also requiring drugmakers to prove benefit in a confirmatory trial before conversion to a full approval.
Outgoing OTAT director Wilson Bryan, who recently announced plans to retire, expressed concern last month that confirmatory studies could take years to confirm benefit in the cell and gene space.
“We’re going to get into trouble with accelerated approval for gene therapies if it takes years to tell us if something works,” he said. “Because then that means it takes years to tell us if it doesn’t work.”
Marks acknowledged similar concerns on Tuesday, but argued that “in the cell and gene therapy space, if we don’t make use of accelerated approval more rapidly, we’re not going to actually treat people who could benefit.”
“Does it mean that we’re going to always get it right? No, but if we get it right 90, 95% of the time, that’s probably going to mean that we’re going to have people alive who would not otherwise be,” he said.
Accelerated approval reforms made it into Congress’ year-end spending package in December, after the agency’s top leaders called for tighter controls on confirmatory trials and an easier process for withdrawing dangling approvals that failed their confirmatory trials.
As part of those reforms, FDA will have to create an intra-agency coordinating council on accelerated approvals, including Marks, CDER director Patrizia Cavazzoni, OCE head Richard Pazdur and OND director Peter Stein, according to the bill.