The role of machine learning in clinical research: transforming the future of evidence generation.



Interest in the application of machine learning (ML) to the design, conduct, and analysis of clinical trials has grown, but the evidence base for such applications has not been surveyed. This manuscript reviews the proceedings of a multi-stakeholder conference to discuss the current and future state of ML for clinical research. Key areas of clinical trial methodology in which ML holds particular promise and priority areas for further investigation are presented alongside a narrative review of evidence supporting the use of ML across the clinical trial spectrum.


Conference attendees included stakeholders, such as biomedical and ML researchers, representatives from the US Food and Drug Administration (FDA), artificial intelligence technology and data analytics companies, non-profit organizations, patient advocacy groups, and pharmaceutical companies. ML contributions to clinical research were highlighted in the pre-trial phase, cohort selection and participant management, and data collection and analysis. A particular focus was paid to the operational and philosophical barriers to ML in clinical research. Peer-reviewed evidence was noted to be lacking in several areas.


ML holds great promise for improving the efficiency and quality of clinical research, but substantial barriers remain, the surmounting of which will require addressing significant gaps in evidence.





Published Version (Please cite this version)


Publication Info

Weissler, E Hope, Tristan Naumann, Tomas Andersson, Rajesh Ranganath, Olivier Elemento, Yuan Luo, Daniel F Freitag, James Benoit, et al. (2021). The role of machine learning in clinical research: transforming the future of evidence generation. Trials, 22(1). p. 537. 10.1186/s13063-021-05489-x Retrieved from

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E. Hope Weissler

House Staff

Erich Senin Huang

Adjunct Assistant Professor in the Department of Surgery

Former Chief Data Officer for Quality, Duke Health
Former Director of Duke Forge
Former Director of Duke Crucible
Former Assistant Dean for Biomedical Informatics

Dr. Huang is currently Chief Science & Innovation Officer for Onduo by Verily, and Head of Clinical Informatics at Verily (Google's life sciences subsidiary), and is now adjunct faculty at Duke. Dr. Huang’s research interests span applied machine learning, research provenance and data infrastructure. Projects include building data provenance tools funded by the NIH’s Big Data to Knowledge program, regulatory science funded by the Burroughs Wellcome Foundation. Applied machine learning applications include “Deep Care Management” a highly interdisciplinary project with Duke Connected Care, Duke’s Accountable Care Organization, that integrates claims and EHR data for predicting unplanned admissions and risk stratifying patients for case management; CALYPSO, a collaboration with the Department of Surgery for utilizing machine learning to predict surgical complications. My team is also building the data platform for the Department of Surgery's "1000 Patients Project" an intensive biospecimen and biomarker study based around patients undergoing the controlled injury of surgery.

As Director of Duke Forge, Dr. Huang is working to build a data science culture and infrastructure across Duke University that focuses on actionable health data science. The Forge emphasizes scientific rigor, awareness that technology does not supersede clinicians’ responsibilities and human relationship with their patients, and the role of data science in society.

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