A novel approach to assessing disparity in representativeness of clinical trial participants within a large midwestern healthcare system.

Background: Representativeness in clinical trials (CT) serves as a metric of access to healthcare and reflects differences that may determine differential efficacy of medical interventions; thus, quantifying representativeness in CT participation is critical.

Methods: This retrospective, descriptive study utilized patient demographic data extracted from the largest Midwestern non-profit healthcare system. Using data between January 1, 2019 and December 31, 2021, a CT Participant Sample of 4,537 system patients who were active CT participants was compared to a CT Patient Population of 195,726 system patients receiving care by the PI of active CTs, which represented the target population. Chi-square goodness-of-fit tests were used to test differences in distributions of demographic variables between groups, indicating disparity in CT participation. Two metrics adapted from literature - participation incidence disparity (PID) and participation incidence ratio (PIR) - were calculated to quantify absolute and relative disparity in representativeness proportions, respectively. Descriptive approaches to assessing representativeness are also provided.

Results: Results showed significant differences by race/ethnicity (χ2 = 50.64; p < 0.0001), age categories (χ2 = 56.64; p < 0.0001), and insurance (χ2 = 41.29; p < 0.0001). PID and PIR metrics revealed reduced CT participation among non-White racial/ethnic groups and increased CT participation among White Non-Hispanic patients. Further, CT participants ≥80 or Worker's Compensation were underrepresented while those with Self-Pay insurance were overrepresented as CT participants.

Conclusions: Despite progress, continued efforts to not only enroll participants into CTs that are representative of the healthcare system and region, but also to better assess representativeness quantitatively are still needed.