Planning and Monitoring Equitable Clinical Trial Enrollment Using Goal Programming

Area of Science:

• Clinical Trials Methodology
• Health Equity Research
• Health Services Research

Background:

• Randomized clinical trials (RCTs) are crucial for evaluating treatment efficacy but often lack diverse patient representation.
• Enrollment plans may underrepresent patient groups with protected attributes (gender, race, ethnicity), limiting generalizability.
• Ensuring equitable participation in clinical trials is a key concern for major health organizations and policymakers.

Purpose of the Study:

• To propose a novel goal-programming approach for designing equitable enrollment plans in randomized clinical trials.
• To integrate measurable enrollment goals addressing patient representativeness and cost-efficiency.
• To enhance the validity of scientific analysis and subgroup disparity evaluations.

Main Methods:

• Developed a goal-programming model to create equitable enrollment plans for RCTs.
• Evaluated the model using enrollment data from the Systolic Blood Pressure Intervention Trial (SPRINT).
• Assessed model performance in single and multisite trial settings, including site selection strategies.

Main Results:

• The model successfully generated equitable enrollment plans satisfying multiple objectives, including sample representativeness and cost.
• The approach demonstrated the ability to detect and correct deviations from enrollment targets during the trial.
• Site selection strategies within the model showed potential for achieving nationally representative study populations.

Conclusions:

• The proposed goal-programming model offers a robust method for prospectively designing and retrospectively evaluating equitable RCT enrollment.
• This approach can improve the generalizability of trial findings and support the analysis of subgroup disparities.
• Implementing such models can lead to more inclusive and representative clinical research, ultimately benefiting diverse patient populations.