Design and analytic considerations for using patient-reported health data in pragmatic clinical trials: report from an NIH Collaboratory roundtable.
Abstract
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.
Type
Journal articleSubject
electronic health recordspatient reported outcome measures
pragmatic clinical trials as topic
randomized controlled trials as topic, patient-generated health data
Permalink
https://hdl.handle.net/10161/20274Published Version (Please cite this version)
10.1093/jamia/ocz226Publication Info
Rockhold, Frank W; Tenenbaum, Jessica D; Richesson, Rachel; Marsolo, Keith A; & O'Brien,
Emily C (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.This is constructed from limited available data and may be imprecise. To cite this
article, please review & use the official citation provided by the journal.
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Show full item recordScholars@Duke
Keith Allen Marsolo
Associate Professor in Population Health Sciences
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
Emily O'Brien
Associate Professor in Population Health Sciences
I am an epidemiologist and health services researcher at the Duke Clinical Research
Institute. My research focuses on comparative effectiveness, patient-centered outcomes,
and pragmatic health services research in cardiovascular and pulmonary disease.Areas
of expertise: Epidemiology, Health Services Research, and Clinical Decision Sciences
Rachel L Richesson
Associate Professor in the School of Nursing
Rachel Richesson, PhD, MPH, a noted informaticist, joined the DUSON faculty in December
2011. Dr. Richesson earned her BS (Biology) at the University of Massachusetts in
1991, and holds graduate degrees in Community Health (MPH, 1995) and Health Informatics
(MS, 2000 and PhD, 2003) from the University of Texas Health Sciences Center in Houston.
Her dissertation involved the integration of heterogeneous data from multiple emergency
departments. Dr. Richesson spent 7 years as at the University
Frank Wesley Rockhold
Professor of Biostatistics & Bioinformatics
Frank is a full time Professor of Biostatistics and Bioinformatics and Faculty Director
for Biostatistics at Duke University Medical Center, Affiliate Professor of Biostatistics
at Virginia Commonwealth University, and Strategic Consultant at Hunter Rockhold,
Inc. His 40+-year career includes senior research positions at Lilly, Merck, and
GlaxoSmithKline, where he retired as Chief Safety Officer and Senior Vice President
of Global Clinical Safety and Pharmacovigilance. He h
Jessica Dale Tenenbaum
Associate Professor of Biostatistics & Bioinformatics
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 precision medicine; 2. Mental health informatics; 3.
Infrastructure and standards to enable research collaboration and integrative data
analysis; and 4. Ethical, legal, and social issues that arise in translational research,
direct to consumer genetic testing, and data sharing. Cu
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