On April 29, 2026, the U.S. Food and Drug Administration (“FDA” or the “Agency”) issued a Request for Information (“RFI”) seeking stakeholder input on a proposed pilot program evaluating the use of artificial intelligence (“AI”) to optimize early-phase clinical trials.
The initiative is intended to assess how AI-enabled technologies “can improve efficiency, speed and quality of decision-making” in Phase I and early Phase II clinical trial development, including through enhanced patient selection, dose optimization, safety monitoring and earlier go/no-go determinations. The FDA frames early-phase trials as a persistent and “critical bottleneck” in drug development, characterized by small patient populations, high uncertainty and resource-intensive decision pathways.
Importantly, the Agency emphasizes that the pilot will be designed to evaluate AI use under “trustworthy AI” principles and systems with attention to transparency, accountability, fairness and data integrity.
The RFI signals regulatory and governance considerations that will shape future FDA expectations for AI in clinical development. These considerations include, without limitation, validation standards for AI-enabled tools and requirements for documenting algorithmic inputs and outputs. The RFI also raises broader questions regarding implications for and adequacy of existing good clinical practice requirements and clinical trial governance structures when AI is embedded into trial design and execution.
Practical Takeaways
The FDA is specifically seeking input on pilot design (e.g., scope and focus), participant selection, collaboration models (e.g., effective partnerships and knowledge sharing), operational structure (e.g., scope of FDA support), timeline and milestones, and evaluation metrics (e.g., trial efficiency, decision quality, participant safety, data integrity, AI system performance and trustworthiness). The breadth of these questions suggests the Agency is still in an exploratory phase.
Comments on the RFI are due May 29, 2026.
If you have any questions or require additional information on this topic, or if you would like assistance with submitting comments, please contact:
- Rebecca Merrill at rmerrill@hallrender.com or (984) 244-5383;
- Summer Martin at smartin@hallrender.com or (984) 244-5382; or
- Your primary Hall Render contact.
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