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. 2025 Mar;4(3):101593.
doi: 10.1016/j.jacadv.2025.101593. Epub 2025 Feb 8.

Embracing Generative Artificial Intelligence in Clinical Research and Beyond: Opportunities, Challenges, and Solutions

Henry P Foote  1 , Chuan Hong  2 , Mohd Anwar  3 , Maria Borentain  4 , Kevin Bugin  5 , Nancy Dreyer  6 , Josh Fessel  7 , Nitender Goyal  8 , Morgan Hanger  9 , Adrian F Hernandez  10 , Christoph P Hornik  10 , Jennifer G Jackman  10 , Alistair C Lindsay  11 , Michael E Matheny  12 , Kerem Ozer  13 , Jan Seidel  14 , Norman Stockbridge  5 , Peter J Embi  12 , Christopher J Lindsell  15
Affiliations

Embracing Generative Artificial Intelligence in Clinical Research and Beyond: Opportunities, Challenges, and Solutions

Henry P Foote et al. JACC Adv. 2025 Mar.

Abstract

To explore threats and opportunities and to chart a path for safely navigating the rapid changes that generative artificial intelligence (AI) will bring to clinical research, the Duke Clinical Research Institute convened a multidisciplinary think tank in January 2024. Leading experts from academia, industry, nonprofits, and government agencies highlighted the potential opportunities of generative AI in automation of documentation, strengthening of participant and community engagement, and improvement of trial accuracy and efficiency. Challenges include technical hurdles, ethical dilemmas, and regulatory uncertainties. Success is expected to require establishing rigorous data management and security protocols, fostering integrity and trust among stakeholders, and sharing information about the safety and effectiveness of AI applications. Meeting insights point towards a future where, through collaboration and transparency, generative AI will help to shorten the translational pipeline and increase the inclusivity and equitability of clinical research.

Keywords: artificial intelligence; clinical research; generative AI; participant engagement; research ethics.

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Conflict of interest statement

Funding support and author disclosures Funding support for the meeting from which this consensus document was generated was provided through registration fees from the following industry sponsors: Alnylam Pharmaceuticals, Inc (Cambridge, MA, USA); Amgen, Inc (Apex, NC, USA); AstraZeneca (Durham, NC, USA); Bayer Pharma AG (Berlin, Germany); Boehringer Ingelheim Pharma GmbH & Co KG (Ingelheim am Rhein, Germany); Bristol Myers Squibb Company (New York, NY, USA); Intellia Therapeutics, Inc (Cambridge, MA, USA); Janssen Pharmaceuticals (Beerse, Belgium); Companies of Johnson & Johnson (New Brunswick, NJ, USA); Novartis AG (Basel, Switzerland); and Novo Nordisk A/S (Bagsværd, Denmark). No government funds were used for this meeting. The think tank was funded by industry sponsors with representation at the meeting. Dr Foote is supported by the Eunice Kennedy Shriver National Institute of Child Health and Human Development under awards T32HD094671 and T32HD104576. Dr Borentain has stock or stock options with Bayer and is a Bayer employee. Dr Dreyer received travel support for attendance at a meeting by Picnic Health and has stock options from Picnic Health. Dr Fessel received payment for a lecture given at the University for Continuing Education, Kremes, Austria, and received travel support from Boehringer Ingelheim Pharma GmbH & Co KG, Department of Global Biostatistics & Data Sciences. Dr Goyal received travel support from Alnylam Pharmaceuticals for attendance at the think tank meeting and receives stock and stock options for Alnylam Pharmaceuticals as an employee. NG is also the co-founder of the patient recruitment platforms iTrials. Hanger works for the Clinical Trials Transformation Initiative, which is supported primarily by an award from the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS), with an additional 15% from nongovernmental entities (member organizations); also received travel support from the National Academies of Sciences, Engineering, Medicine Forum on Drug Discovery, Development, and Translation. Dr Hernandez has received grants or contracts from Pfizer Inc and Merck and consulting fees from Merck. Dr Lindsay has stock or stock options with GSK and Johnson & Johnson. Dr Hornik has received grants from the National Institutes of Health (award numbers: 5R01-HD106588-02, 1RL1-HD107784-03, 5R33-HL147833-05, 5T32HD104576-03, OT2OD034481, and 1U01-MD018294-01) and Burroughs Wellcome (1020016) and has received consulting fees from Lightship Inc, Cytokinetics, and UCB Pharma; and also has participated on an advisory board for The Emmes Corporation, LLC. Dr Matheny has received consulting fees from the Patient-Centered Outcomes Research Institute (PCORI) trust fund. Dr Ozer has stock with Novo Nordisk and is a Novo Nordisk employee. Dr Lindsell has received grants or contracts from the following, with all payments made to the institution: the National Institutes of Health, the Department of Defense, the U.S. Centers for Disease Control and Prevention, Novartis, AstraZeneca, Cytokinetics, Biomeme, Entegrion Inc, Endpoint Health, and bioMerieux; In addition, patents for risk stratification in sepsis and septic shock were issued to Cincinnati Children's Hospital Medical Center; has also participated in data safety monitoring boards unrelated to the current work and has participated on Clinical and Translational Science Awards (CTSA) external advisory boards; is also an ex officio member of Association for Clinical and Translational Science (ACTS) board, Clinical Research Forum (CRF) board, ACTS Advisory Committee, and the Center for Clinical and Translational Science (CCTS) Executive Committee; and has stock options with Bioscape Digital. In addition, CJL is the Editor-in-Chief of the Journal of Clinical and Translational Science. CH, JJ, KB, MA, NS, and PJE report no conflicts of interest. The first draft of the manuscript was written independently of sponsor input. Industry representatives were able to provide comments on subsequent drafts of the manuscript. The ultimate decision to include these comments rested with the primary and senior authors.

Figures

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Graphical abstract
Figure 1
Figure 1
Summary of the Workshop Session and Topics The sessions were structured around several discussion points: efficiency, engagement, novel endpoints, and overarching impacts. We aimed to cultivate a consensus among the diverse participants on the scope of generative artificial intelligence, the practical and ethical concerns, the required validation, and regulatory landscape within each of the sessions. AI = artificial intelligence.
Central Illustration
Central Illustration
Capacities, Impact, and Strategic Pathways of Generative Artificial Intelligence in Clinical Research Generative artificial intelligence will facilitate both incremental and transformative changes. Collaboration and knowledge sharing will be key to navigating the challenges and ethical considerations inherent in its deployment. AI = artificial intelligence.
See this image and copyright information in PMC

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