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J Am Med Inform Assoc. 2020 Feb 6. pii: ocz226. doi: 10.1093/jamia/ocz226. [Epub ahead of print]

Design and analytic considerations for using patient-reported health data in pragmatic clinical trials: report from an NIH Collaboratory roundtable.

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Duke Clinical Research Institute, Durham, North Carolina, USA.
Department of Biostatistics and Bioinformatics, Duke University School of Medicine, Durham, North Carolina, USA.
Duke University School of Nursing, Durham, North Carolina, USA.
Duke University School of Medicine, Durham, North Carolina, USA.
Population Health Sciences, Duke University School of Medicine, Durham, North Carolina, USA.


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.


electronic health records; patient reported outcome measures; pragmatic clinical trials as topic; randomized controlled trials as topic, patient-generated health data


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