Comparison of Clinical Characteristics Between Clinical Trial Participants and Nonparticipants Using Electronic Health Record Data.

Importance: Assessing generalizability of clinical trials is important to ensure appropriate application of interventions, but most assessments provide minimal granularity on comparisons of clinical characteristics.

Objective: To assess the extent of underlying clinical differences between clinical trial participants and nonparticipants by using a combination of electronic health record and trial enrollment data.

Design, Setting, And Participants: This cross-sectional study used data obtained from a single academic medical center between September 1996 and January 2019 to identify 1645 clinical trial participants from a diverse set of 202 available trials conducted at the center. Using an aggregated resampling procedure, nonparticipants were matched to participants 1:1 based on trial conditions, number of recent visits to a health care professional, and calendar time.

Exposures: Clinical trial enrollment vs no enrollment.

Main Outcomes And Measures: The primary outcome was standardized differences in clinical characteristics between participants and nonparticipants in clinical trials stratified into the 4 most common disease domains.

Results: This cross-sectional study included 1645 participants from 202 trials (929 [56.5%] male; mean [SD] age, 54.65 [21.38] years) and an aggregated set of 1645 nonparticipants (855 [52.0%] male; mean [SD] age, 57.24 [21.91] years). The most common disease domains for the selected trials were neoplastic disease (86 trials; 737 participants), disorders of the digestive system (31 trials; 321 participants), inflammatory disorders (28 trials; 276 participants), and disorders of the cardiovascular system (27 trials; 319 participants); trials could qualify for multiple disease domains. Among 31 conditions, the percentage of conditions for which the prevalence was lower among participants than among nonparticipants per standardized differences was 64.5% (20 conditions) for neoplastic disease trials, 61.3% (19) for digestive system trials, 58.1% (18) for inflammatory disorder trials, and 38.7% (12) for cardiovascular system trials. Among 17 medications, the percentage of medications for which use was less among participants than among nonparticipants per standardized differences was 64.7% (11) for neoplastic disease trials, 58.8% (10) for digestive system trials, 88.2% (15) for inflammatory disorder trials, and 52.9% (9) for cardiovascular system trials.

Conclusions And Relevance: Using a combination of electronic health record and trial enrollment data, this study found that clinical trial participants had fewer comorbidities and less use of medication than nonparticipants across a variety of disease domains. Combining trial enrollment data with electronic health record data may be useful for better understanding of the generalizability of trial results.