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

Journal Article (Journal Article)

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.

Full Text

Duke Authors

Cited Authors

  • Rockhold, FW; Tenenbaum, JD; Richesson, R; Marsolo, KA; O'Brien, EC

Published Date

  • April 1, 2020

Published In

Volume / Issue

  • 27 / 4

Start / End Page

  • 634 - 638

PubMed ID

  • 32027359

Pubmed Central ID

  • PMC7075526

Electronic International Standard Serial Number (EISSN)

  • 1527-974X

Digital Object Identifier (DOI)

  • 10.1093/jamia/ocz226


  • eng

Conference Location

  • England