The Coming AI Revolution in Clinical Trials

Abstract

Section snippets

Digital Strategy and Data Infrastructure

Current systems for data storage and analysis are siloed, limiting the potential for AI to function efficiently across data networks. Without interoperable data systems, even the most advanced AI tools will struggle to deliver real impact. We need a unified data strategy—how we manage, integrate, and safeguard data across platforms—focused on a patient-centered data ecosystem that facilitates information flow across EHRs, health system applications, personal records, and clinical trial data

Business Model Innovation

Current clinical trial business models are designed around trial sites, not participants. They involve processes that are labor-intensive, duplicative, and burdened by high fixed costs for recruitment, site management, and data collection. Data are often collected redundantly, despite also being obtained for routine clinical care, with gaps in collection due to episodic follow-up instead of continuous monitoring. AI has the potential to streamline certain processes, shorten timelines, and lower

Regulatory Modernization and AI Evaluation

Regulatory bodies are crucial in ensuring the safety and efficacy of AI-augmented clinical trials, yet the current regulatory framework was not designed to accommodate the rapid advancements in AI technology or the unique risks of AI use in RCTs. Rather than relying on traditional linear approval pathways, regulators should embrace iterative approaches that incorporate regular feedback. Participants in RCTs that use AI tools for operations are a potentially vulnerable population in which there

Funding Support and Author Disclosures

Dr Jain has received consulting fees from Bristol Myers Squibb, ARTIS Ventures, and Broadview Ventures outside of the submitted work. Dr Sarraju holds stock in Medeloop. Dr Ashley is a founder of Personalis, DeepCell, and Svexa; is a founding advisor of Nuevocor; is a nonexecutive director at AstraZeneca; and has served as an advisor for SequenceBio, Novartis, Medical Excellence Capital, Foresite Capital, and Third Rock Ventures. Dr Harrington has served on the Board of Directors for the