AI In Clinical Trials: Key Insights From Industry Experts

The January 23, 2025 Clinical Leader Live webinar, AI in Action: Transforming Clinical Trials, explored how AI-driven protocol optimization and proactive FDA guidance are reshaping trial design and execution. FDA Associate Director for Data Science and AI Policy Tala Fakhouri and industry consultant Ella Sinclair discussed AI’s ability to rapidly process vast datasets, identify patterns, and improve trial outcomes—particularly by reducing costly protocol amendments and enhancing indication selection.

The FDA has adopted a progressive, innovation-friendly approach, avoiding prohibitions on tools such as large language models and instead emphasizing transparency, risk-based validation, and early engagement. Sponsors are encouraged to document the context of use, validation methods, and integration with other evidence sources, ensuring AI outputs are traceable and regulatory expectations are met. Internationally, regulatory bodies are moving toward harmonized AI guidance, while techniques like federated learning address data privacy challenges without compromising model performance. The FDA’s stance allows companies to integrate AI earlier in the trial lifecycle, enabling faster starts, reduced costs, and higher probabilities of success. With only 15–20% of organizations currently adopting AI at scale, those who act align with FDA’s proactive framework now can secure a competitive advantage in the evolving clinical research landscape.