FDA develops pilot to harmonize international gene therapy regulations

The spokesperson said that the program, called the Collaboration on Gene Therapies Global Pilot, is part of the agency’s work toward global regulatory harmonization of these products. The FDA is working with international partners, global regulators and the World Health Organization on these efforts, they said.

While it’s still early on in development, the pilot will explore the possibility for “concurrent collaborative” review of applications alongside global regulatory partners.

“While countries around the world have their own regulatory authorities, there are no uniform global standards for the evaluation and regulation of cell and gene therapy products,” the spokesperson said. “We believe that harmonization efforts in this area can help facilitate more efficient clinical development.”

The FDA has also used this approach for Project Orbis, which it established in 2019 to provide a pathway for concurrent submission and review of oncology products.

The pilot for gene therapy products comes as the agency has tightened its focus on gene therapies and rare disease in recent years.

This past fall, the FDA launched an Operation Warp Speed-like pilot program for rare disease to speed the approval process. FDA’s Peter Marks has also previously said that the agency is pushing the accelerated approval pathway for rare disease products.

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