A novel approach to reducing disparities in health outcomes by enhancing interpretation of cancer clinical trials for underrepresented patient groups.

ABSTRACT

There has been a growing realization, based on emerging evidence from the point of care, that real-world outcomes of patients with cancer are often inferior to those reported in conventional clinical trials. This phenomenon can be attributed in part to deficits in external validity that are present in many studies. Several factors contribute to external validity deficits, including narrow eligibility criteria; differences between protocol-specified procedures and routine care; and inadequate access to clinical trial participation among underrepresented and socioeconomically disadvantaged groups. As a result, the current body of clinical evidence derived from conventional clinical trials can be inadequate to inform patient-specific treatment decisions at the point of care. Furthermore, lack of practical guidance on how to evaluate the impact of external validity deficits can impede both the design of more generalizable clinical trials and efforts to personalize treatment decisions for individual patients. In this methodological review, we suggest an approach to aid clinicians in such evaluations, providing visual and quantitative methods for assessing the magnitude of, and adjusting for, the impact of external validity deficits in conventional clinical trials. Our methods and visualizations have broad applicability across important areas of real-world medical decision-making and research, providing opportunities to design clinical studies that are more reflective of the diverse needs of patients with cancer, including those excluded from traditional clinical trials due to narrow eligibility criteria, socioeconomic disadvantages, and other systemic barriers to equitable access to healthcare resources.