FDA Explores AI to Accelerate Early-Phase Clinical Trials in Pilot Program
The U.S. Food and Drug Administration (FDA) is advancing the use of AI and data science to accelerate clinical trial reporting. The recent announcement highlighted two proof-of-concept real-time clinical trials initiated by AstraZeneca and Amgen, along with a Request for Information on AI-enabled technologies for optimizing a proposed early-phase trial pilot program set to begin this summer.
The FDA identified persistent challenges in early-phase trials, including inefficient progression decisions, lengthy timelines, limited patient populations, and uncertainty. To address these issues, the pilot program will evaluate AI-based tools to improve the efficiency, speed, and quality of safety monitoring, dose selection, and regulatory decision-making. The agency emphasized the use of trustworthy AI in alignment with the National Institute of Standards and Technology's AI Risk Management Framework and existing FDA draft guidances.
The Request for Information also outlined key questions to guide pilot development, including which trial types may benefit most from AI applications, and how to measure success in both AI implementation and early-phase trials.
Comments on the Request for Information will be accepted through May 29, 2026. The FDA plans to release selection criteria in July, with pilot selections expected by August.
For real-time clinical trials, the FDA is partnering with Paradigm Health to deploy a platform that provides real-time insights from the study into key safety and efficacy signals. Paradigm Health develops AI-based platforms for the clinical research ecosystem. The company said the platform created specifically for the collaboration with the FDA integrates data from electronic health records and other sources, analyzes data points and reporting criteria algorithmically, and transmits critical information to both the FDA and the trial sponsor.
“For 60 years, we've been conducting clinical trials in the same way, where key data signals can take years to reach the FDA. The lag time can delay regulatory decisions unnecessarily and slow down the drug development timeline,” stated Marty Makary, MD, MPH, FDA Commissioner. “We are boldly advancing a modern approach whereby FDA scientists can view safety signals and endpoints in real time as a trial progresses. This will help us accelerate promising therapies, and build toward our ultimate goal of running real-time, continuous trials across all phases of drug development.”
ASCO AI in Oncology is published by Conexiant under a license arrangement with the American Society of Clinical Oncology, Inc. (ASCO®). The ideas and opinions expressed in ASCO AI in Oncology do not necessarily reflect those of Conexiant or ASCO. For more information, see Policies.