On April 15, 2024, the US Food & Drug Administration (FDA) announced the establishment of the Center for Drug Evaluation and Research (CDER) Center for Clinical Trial Innovation (C3TI). C3TI will serve as the central support hub for innovative approaches to clinical trials designed to improve the quality and efficiency of drug development and regulatory decision-making.

IN DEPTH

BACKGROUND

As the division of FDA responsible for regulating drugs and certain biologics, CDER’s authority includes overseeing the development and testing of such products. As technology advancements and challenges and opportunities in drug development evolve, CDER is seeking to cultivate drug development and innovation through novel clinical trial designs, trial execution strategies and drug development tools and by leading or co-leading several efforts to foster improvements across the study design landscape. One such effort included a public solicitation of comments on the barriers of clinical trial designs on October 17, 2023. After discussion of the comments, FDA determined that the establishment of C3TI would enhance CDER’s ability to address those barriers and foster innovation.

C3TI

C3TI is governed by various offices of CDER, including the Office of the Center Director, the Office of Communications, the Office of Compliance, the Office of Medical Policy, the Office of New Drugs and the Office of Translational Sciences. C3TI’s mission is to “promote existing and future CDER clinical trial innovation through enhanced communication and collaboration.” C3TI aims to improve clinical trial efficiency, effectiveness, data quality, innovation and coordination in the hope that such efforts will increase the participation of diverse populations in clinical trials and accelerate the development of safe and effective drugs.

C3TI will:

1. Facilitate the sharing of lessons learned across CDER’s existing clinical trial innovation programs
2. Communicate and collaborate with external parties regarding innovative clinical trials
3. Manage a C3TI Demonstration Program to expand opportunities for sponsors to interact with CDER staff

The C3TI Demonstration Program will initially consist of three project areas under a pre-investigational new drug application (pre-IND) or IND: (1) point-of-care or pragmatic trials, (2) Bayesian analyses and (3) trials using selective safety data collection. Sponsors may submit program proposals via email to CDERclinicaltrialinnovation@fda.hhs.gov. The program will accept up to nine proposals on a rolling basis within a 12-month period. Sponsors selected for the program will engage with CDER staff to serve as case examples and generate lessons for the broader community.

C3TI is under development and will eventually serve as the conduit for internal and external parties to access resources and obtain information regarding clinical trial designs and innovation efforts. Stakeholders including sponsors, contract research organizations and other service providers in the research industry, and entities conducting industry-sponsored research should monitor C3TI’s development and implementation. The learnings that emerge from C3TI’s activities may meaningfully shape clinical trial strategy and related business offerings in the research industry.

Jae Hyun Lee, law clerk in McDermott’s New York office, also contributed to this article.