As part of its ongoing effort to promote complex innovative trial designs (CIDs), the U.S. Food and Drug Administration (FDA) just unveiled an expansive pilot meeting program designed to let sponsors discuss specifics with agency experts.
The CID pilot meeting program is for innovative designs that require simulations. There are several parameters, however. For example, false positives, or Type 1 errors, are generally deal-breakers.
To be eligible for the program, sponsors filing Investigational New Drug (IND) applications must have a pre-IND or IND number for the medical product(s) included in the proposal, and the proposed CID should be intended to provide “substantial” evidence of effectiveness to support regulatory approval of the product.
The trial cannot be a first-in-human study. There must also be “sufficient clinical information” available to inform the proposed CID, the agency said in the notice it posted on August 30.
The agency said meeting requests should include key features of the statistical analysis plan, as well as the simulation plan and components of the study design the sponsor believes are non-disclosable. The sponsor and FDA must be able to reach agreement on the trial design information to be publicly disclosed.
The agency said it will select requests based on innovative features of the trial design, particularly if the innovations may provide “advantages over alternative approaches.” Initially, priority will be given to trial designs for which analytically derived properties, such as Type 1 errors, may not be feasible and simulations are necessary to determine operating characteristics. Requests will also generally move to the end of the line if they demonstrate a therapeutic need (e.g., therapies being developed for use in disease areas for which there are no or limited treatments).
The CID pilot meeting program is scheduled to run through September 30, 2022. Sponsors may submit meeting requests for the pilot program through June 30, 2022. It’s a joint program administered by offices within the FDA’s Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research.
Author: Michael Causey