FDA Promises Flexibility for Stem Cell Trials

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

In a final guidance, 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.