The U.S. FDA has finalized guidance for drug sponsors interested in studying multiple versions of a cellular or gene therapy product in an early-phase clinical trial for a single disease.
In contrast to traditional trial designs where only one product is evaluated in a single clinical trial, 'umbrella' trials use a single trial infrastructure, design, and master protocol to simultaneously evaluate multiple products for a specific disease or condition, allowing for more efficient product development.
In the new guidance — which finalizes draft guidance of the same title from September 2021— the agency provides recommendations for studies that evaluate multiple versions of a cellular or gene therapy product, including how to organize and structure the INDs, submit new information and report adverse events.
According to the FDA, "the potential benefits of this type of umbrella trial include flexibility and efficiency in product development. Instead of an iterative approach to clinical studies, multiple versions of a cellular or gene therapy product can be studied in parallel, which may streamline early clinical development by expeditiously identifying alternative versions of a product that may be safer or more effective."
