Center Watch Weekly
February 18, 2019

Regulators are promising “flexibility” in clinical trial design for stem cell therapies in rare diseases.

In a final guidance issued last week, the FDA says it “recognizes that, for regenerative medicine therapies for rare diseases, certain aspects of drug development that are feasible for common diseases may not be feasible, and that development challenges can be greater with increasing rarity of the disease.”

“Innovative trial designs, such as trials that compare several different investigational agents to each other and a common control, may be particularly useful in studies of regenerative medicine therapies to treat rare diseases,” the guidance says.

CBER might also be open to historical controls data obtained from natural histories, and regulators might be willing to consider a trial that — unlike the traditional, multi-site study — uses multiple sites that are sharing data on stem cell therapies in a combination to support a BLA.

Sponsors are encouraged “to obtain input from the affected patient communities” when they’re coming up with endpoints for stem cell trials.

The FDA also is promising to be flexible about fast-tracking some potential stem cell treatments. The agency will want clinical data to justify a fast-track application, but the data doesn’t necessarily have to come from prospective clinical trials.

“In some cases, clinical evidence obtained from clinical investigations with appropriately chosen historical controls may provide sufficient preliminary clinical evidence of the potential to address an unmet medical need,” the agency says. “In other cases, preliminary clinical evidence could come from well-designed retrospective studies or clinical case series that provide data systematically collected by treating physicians.”

Read the guidance here: