Validation of a study design that promotes diversity in clinical trials.

Abstract

e23000 Background: Diversity and inclusivity in clinical trials are important for generating well-balanced biomedical data for analysis. The ongoing, multi-center FLEX trial (NCT03053193) is a prime example of a study that can capture a diverse patient population. Therefore, a study was designed to validate this proposition and discuss its study design. Methods: A retrospective cohort analysis of breast cancer patients was designed to compare the institution-specific ethnic distribution of FLEX trial enrolled patients against the ethnic distribution of all patients diagnosed with breast cancer from the tumor registry data of the same institution from 2018 to 2022. Results: The tumor registry data revealed a total of 959 breast cancer patients over the specified period. The ethnic distribution was as follows: 687 White patients (71.6%), 141 Black patients (14.7%), 58 Asian patients (6%), and 73 Hispanic-Caribbean patients (7.6%). The FLEX registry data for the same timeframe showed that 134 patients were enrolled, with the following ethnic distribution: 79 White patients (59%), 23 Black patients (17.2%), 2 Asian patients (1.5%), and 30 Hispanic-Caribbean patients (22.4%). Conclusions: Comparison between the FLEX-enrolled patients and the background breast cancer population from the institution’s tumor registry confirmed that the racial distribution of FLEX participants was representative of the overall patient population served by the institution. This demonstrates that a well-designed study can effectively capture the racial diversity of the affected population. To further elaborate on its design, the FLEX study can be considered a variant of a Master Observational trial(MOT), a prospective, observational study with broad eligibility criteria independent of biomarker signature. Although the FLEX trial does not dictate intervention based on biomarker data, it collects data on how the clinician has independently ‘intervened’ based on biomarker data to create prospective analyses. The FLEX study offers a participant-friendly model, without the fear of uncertainty associated with dichotomous randomized clinical trial designs. It facilitates a deeper understanding and acceptance of clinical research by integrating real-world data with biomarker-based decisions, fostering broad inclusivity and equity in research participation. This study shows that reimagining study designs can advance access and equity in cancer care. By embracing these new strategies, we can foster an inclusive research environment, ultimately contributing to effective healthcare solutions for all.