New draft guidance for regenerative medicine

[GH253]

https://www.fda.gov/downloads/Biolo...uidances/CellularandGeneTherapy/UCM585414.pdf

CBER lays out three expedited approval programs for regenerative medicine therapies: Fast Track, Breakthrough Therapy, and the new Regenerative Medicine Advanced Therapy (RMAT). As I understand it, the RMAT designation is the only one that makes a product eligible for accelerated approval based on the use of surrogate or intermediate endpoints.

The document specifically lists a stem cell therapy intended to treat OA as a "hypothetical" example of what the agency would consider for the Breakthrough Therapy designation. The only explicit criterial difference between Breakthrough and RMAT is that to qualify for the latter, the therapy must address an “unmet medical need”—are they saying they don’t consider OA an unmet medical need?

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[drusso]

https://www.fda.gov/downloads/Biolo...uidances/CellularandGeneTherapy/UCM585414.pdf

CBER lays out three expedited approval programs for regenerative medicine therapies: Fast Track, Breakthrough Therapy, and the new Regenerative Medicine Advanced Therapy (RMAT). As I understand it, the RMAT designation is the only one that makes a product eligible for accelerated approval based on surrogate or intermediate endpoints.

The document specifically lists a stem cell therapy intended to treat OA as a "hypothetical" example of what the agency would consider for the Breakthrough Therapy designation. The only explicit criterial difference between Breakthrough and RMAT is that to qualify for the latter, the therapy must address an “unmet medical need”—are they saying they don’t consider OA an unmet medical need?

F.D.A. Speeds Review of Gene Therapies, Vowing to Target Rogue Clinics

"But the message seemed mixed. Dr. Gottlieb and Dr. Marks also said that orthopedic procedures — injecting the fat-derived cells into joints — were of lower priority than treatments that involved injecting or infusing the cells into the central nervous system or bloodstream.

“There are hundreds and hundreds of these clinics,” Dr. Marks said. “We simply don’t have the bandwidth to go after all of them at once.”

Not everyone agrees that orthopedic clinics should be lower on the priority list. Leigh Turner, an associate professor at the University of Minnesota Center for Bioethics, School of Public Health and College of Pharmacy, who studies cellular therapies, said the use of stem cell therapy in athletic clinics must be scrutinized.

“Sport clinics that use stem cells are one of the largest components of the U.S. marketplace,” she said. “They’ve just been told that there is a three-year period in which they don’t have to be worried about the F.D.A.”