The US Food and Drug Administration (FDA) laid out its recommendations for sponsors to study multiple versions of a cellular or gene therapy in a clinical trial for a single disease. The agency said its final guidance should help sponsors conduct umbrella trials of similar products for the same disease more efficiently to bring cell and gene therapies to market sooner.
While clinical trials typically evaluate a single product, umbrella trials use a single-trial infrastructure, design and master protocol to simultaneously evaluate multiple products for a disease or condition, which can be more efficient. (RELATED: For gene therapies, FDA drafts trial guidance, finalizes « sameness » for orphan exclusivity, Regulatory Focus 30 September 2022)
“Sponsors have expressed interest in gathering preliminary evidence of safety and activity using multiple versions of a cellular or gene therapy product in a single clinical trial,” FDA said. “Although multiple versions of a product can be studied together in a single clinical trial, each version of the product is distinct and is generally submitted to FDA in a separate investigational new drug application (IND). The objective of these early-phase clinical studies is to guide which version(s) of the product to pursue for further development in later-phase studies.”…
