Final FDA Guidance Includes ASGCT Recommendations, Beneficial Revisions

An FDA draft guidance document finalized last week—Expedited Programs for Regenerative Medicine Therapies for Serious Conditions—includes revisions that address comments from ASGCT.

Definition of Regenerative Medicine Therapy

ASGCT found the wording, “durable modification” to lack clarity in the FDA's statement: “… gene therapies, including genetically modified cells, that lead to a durable modification of cells or tissues may meet the definition of a regenerative medicine therapy.” The agency's changed wording to “sustained effect on cells and tissues” helps clarify the criteria for gene therapies to be considered as regenerative medicine advanced therapies (RMATs) and to be eligible for the RMAT designation.

Innovative Trial Design

The FDA added to the document that historical controls may be considered as appropriate, and that natural history data can be used as the basis of a historical control when the control and treatment populations are adequately matched. Another useful addition is for “trials that compare several different investigational agents to each other and a common control,” each practice needs to meet the BLA requirements, and product manufacturing needs to meet current good manufacturing practices requirements. The FDA intends to issue a guidance this year about this trial design, which may assist small sponsors lacking sufficient scale to conduct a clinical trial on their own.

Another guidance document, Evaluation of Devices Used with Regenerative Medicine Advanced Therapies, was also finalized last week. Both documents are part of the agency’s comprehensive regenerative medicine policy framework.