Building on CDER's efforts to modernize clinical trials, C3TI will support innovative approaches to clinical trial design and conduct.
In April 2024, the U.S. Food and Drug Administration ("FDA") Center for Drug Evaluation and Research ("CDER") announced the launch of the Center for Clinical Trial Innovation ("C3TI") to support innovative approaches to clinical trials designed to improve drug development efficiency through enhanced communication and collaboration.
C3TI will primarily serve as a "central hub" to:
- Facilitate sharing of lessons learned across CDER's existing clinical trial innovation programs.
- Serve as the single CDER point of contact to support non-product-specific questions about clinical trial innovation.
- Support knowledge-sharing through discussion forums, public workshops, communications, and a knowledge repository consolidating information on CDER's clinical trial innovation efforts.
- Host a C3TI demonstration program for sponsors of innovative clinical trials in the pre-investigational new drug application ("pre-IND") or IND phase.
C3TI's demonstration program is open to innovative clinical trial sponsors in three initial demonstration project areas:
- Streamlined trials embedded in clinical practice (i.e., point-of-care or pragmatic trials). Available for trials incorporating pragmatic design elements (e.g., broad eligibility criteria, decentralized procedures, limited safety data collection consistent with ICH E19) to address and resolve issues with respect to statistical analyses, incorporation of real-world data and evidence, endpoint selection, and inspectional approaches.
- Bayesian supplemental analyses.Available for phase 3 efficacy, safety, or non-inferiority standalone trials with a simple non-adaptive design that supplement the primary statistical analyses with Bayesian analyses to further understanding of Bayesian methods and establish processes for future integration.
- Selective safety data collection. Available for late-stage pre- or post-marketing trials on drug products with well-characterized safety profiles, such as trials for an approved drug seeking a new indication in a similar population, to streamline safety data collection, reduce expenses, better allocate resources, and facilitate trial conduct to answer important scientific questions regarding long-term efficacy and safety.
CDER has announced that C3TI's demonstration program will accept up to nine proposals on a rolling basis within a 12-month period. Sponsors will be required to submit proposals to the relevant application (i.e., pre-IND, IND) and email CDERclinicaltrialinnovation@fda.hhs.gov to notify FDA of the submission. Further information can be found here.
Since its launch in April, there have already been several notable C3TI updates, including the rollout of C3TI Compass on May 17, 2024, which is a searchable database that includes a list of CDER's clinical trial innovation activities and experience, such as the recent public workshop, Advancing the Use of Complex Innovative Designs in Clinical Trials: From Pilot to Practice, which discussed using Bayesian methods, external data sources, and simulations in complex innovative trial designs and implementation.
The content of this article is intended to provide a general guide to the subject matter. Specialist advice should be sought about your specific circumstances.