Design and analytic considerations for using patient-reported health data in pragmatic clinical trials: report from an NIH Collaboratory roundtable.
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2020-02-06
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Pragmatic clinical trials often entail the use of electronic health record (EHR) and claims data, but bias and quality issues associated with these data can limit their fitness for research purposes particularly for study end points. Patient-reported health (PRH) data can be used to confirm or supplement EHR and claims data in pragmatic trials, but these data can bring their own biases. Moreover, PRH data can complicate analyses if they are discordant with other sources. Using experience in the design and conduct of multi-site pragmatic trials, we itemize the strengths and limitations of PRH data and identify situational criteria for determining when PRH data are appropriate or ideal to fill gaps in the evidence collected from EHRs. To provide guidance for the scientific rationale and appropriate use of patient-reported data in pragmatic clinical trials, we describe approaches for ascertaining and classifying study end points and addressing issues of incomplete data, data alignment, and concordance. We conclude by identifying areas that require more research.
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Rockhold, Frank W, Jessica D Tenenbaum, Rachel Richesson, Keith A Marsolo and Emily C O'Brien (2020). Design and analytic considerations for using patient-reported health data in pragmatic clinical trials: report from an NIH Collaboratory roundtable. Journal of the American Medical Informatics Association : JAMIA, 27(4). pp. 634–638. 10.1093/jamia/ocz226 Retrieved from https://hdl.handle.net/10161/20274.
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Jessica Dale Tenenbaum
Dr. Tenenbaum is a faculty member in the Division of Translational Biomedical Informatics in the Department of Biostatistics and Bioinformatics. Her primary research interests are 1. Informatics to enable whole person health, including mental health and social determinants of health (SDOH); 2. Infrastructure and data governance to enable collaboration and integrative data analysis; and 3. Ethical, legal, and social issues in biomedicine. She is also Special Advisor for Research Data Innovation in Duke's Office of Research & Innovation.
From 2019 to 2024, Dr. Tenenbaum served as Chief Data Officer for North Carolina's Department of Health and Human Services (NC DHHS). She continues to consult with state government, serving as Senior Data Strategist for NC HealthConnex, North Carolina's statewide Health Information Exchange (HIE).
Nationally Dr. Tenenbaum has served as an Associate Editor for the Journal of Biomedical Informatics, an elected member of AMIA's Board of Directors, and on the Board of Scientific Counselors for the National Library of Medicine. She is also an elected Fellow and President-Elect of the American College of Medical Informatics.
Keith Allen Marsolo
Dr. Marsolo is a faculty member in the Department of Population Health Sciences (DPHS) and a member of the Duke Clinical Research Institute (DCRI). His current research focuses on infrastructure to support the use of electronic health records (EHRs) and other real-world data sources in observational and comparative effectiveness research and public health surveillance, as well as standards and architectures for multi-center learning health systems. He serves as faculty advisor to the DPHS DataShare Shared Facility and faculty lead for the Pragmatic Health Services Research (PHSR) functional group within the DCRI. Dr. Marsolo received his PhD in Computer Science from The Ohio State University, with a dissertation on data mining, specifically the modeling and classification of biomedical data.
Prior to joining DPHS, Dr. Marsolo was an an Associate Professor in the Division of Biomedical Informatics (BMI) at Cincinnati Children’s Hospital Medical Center (CCHMC). While at CCHMC, Dr. Marsolo served as faculty advisor for BMI Data Services, a shared facility that supported distributed data sharing networks and also developed registry platforms to support learning networks. These included a configurable system for capturing summary or practice-level measures, and a “data-in-once” architecture that allowed information to be collected in the EHR and then be automatically transferred to a registry in order to support chronic care management, quality improvement and research.Area of Expertise: Informatics, Data Quality, Common Data Models, Data Standards and Data Harmonization
Emily O'Brien
Dr. Emily O’Brien is Associate Professor in Population Health Sciences, Associate Professor in Neurology, Core Faculty Member at Duke-Margolis Center for Health Policy, and Co-Director of Population Health Sciences at the Duke Clinical Research Institute. Her research focuses on comparative effectiveness, patient-centered outcomes, and pragmatic health systems research in cardiovascular and pulmonary disease. Her areas of expertise include: Epidemiology, Pragmatic Clinical Trials, and Clinical Decision Sciences. Dr. O’Brien received her PhD in Epidemiology from the University of North Carolina in Chapel Hill. As principal investigator for projects funded by the FDA, NIH, and PCORI, she has extensive experience working with diverse data sources including registries, epidemiologic cohorts, electronic health records, and administrative claims data. Dr. O’Brien teaches Analytic Methods in the Department of Population Health Sciences PhD program and has co-authored over 160 manuscripts in peer-reviewed journals on topics ranging from epidemiologic methods, comparative effectiveness, and pragmatic clinical trials. She is an associate editor for Circulation: Cardiovascular Quality and Outcomes, Chair of the AHA QCOR Scientific & Clinical Education Lifelong Learning Committee, social media editor for the Journal of the American Heart Association, and a fellow of the American Heart Association.
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