FDA starts pilot to help sponsors use novel trial designs

Acceptance into the program isn’t a guarantee the FDA will sign off on a design. (FDA)

The FDA has initiated a pilot program (PDF) to help drug developers use complex innovative trial designs (CID). Sponsors accepted into the four-year pilot will meet twice with the FDA to discuss regulatory approaches to CID. 

Officials at the FDA committed to help advance the use of complex adaptive, Bayesian and otherwise novel clinical trial designs as part of the latest PDUFA agreement. These designs pose new challenges for sponsors such as the need to use simulations to determine trial operating characteristics, and therefore additional regulatory input is beneficial to ensure they meet the FDA’s expectations.

The pilot program is intended to ensure sponsors can access that input. The FDA is asking sponsors with plans to use CID to file to join the program, although it has only committed to accepting up to two applications a quarter. The agency will prioritize trials that rely on simulations and will test drugs that address unmet needs when choosing which applicants to accept. 

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The resulting meetings will help the FDA take its stance on CID.

“The goal ... is to provide advice on how a proposed CID approach can be used in a specific drug development program and to promote innovation by allowing FDA to publicly present the trial designs considered through the pilot program, including trial designs for drugs that have not yet been approved by FDA,” the agency wrote. 

Sponsors accepted into the program will hold an initial meeting with the FDA to discuss a CID, and then meet again within 120 days to follow up on the same topic. This input could make it easier for the sponsors to adopt CID, but acceptance into the program isn’t a guarantee the FDA will sign off on a design, nor is a rejection from the pilot an unbreachable barrier to a company running a novel trial. 

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