S2996 Ten-Year Trends in Enrollment of Women and Minorities in Pivotal Trials Supporting Recent United States Food and Drug Administration Approval of Therapeutics in Gastroenterology and Hepatology

Introduction: In November 2020, the United States Food and Drug Administration (FDA) released updated guidance to increase the clinical trial enrollment of underrepresented populations, including women and racial/ethnic minorities, to ensure the widespread safety and efficacy of novel therapeutics. This study aims to investigate the 10-year trends in participation of women and racial/ethnic minorities in pivotal trials supporting the approval of new gastrointestinal/hepatobiliary drugs from January 2015 to December 2024. Methods: A list of novel pharmacologic agents approved for conditions within gastroenterology/hepatology was abstracted from publicly available data from Drugs@FDA. Publicly available sex and race/ethnicity data were collected from the individual trials via ClinicalTrials.gov. Linear regression analysis was performed to assess the relation between drug approval year and proportion of women and minorities enrolled. Results: Forty-one (n = 41) novel therapeutics for gastrointestinal/hepatobiliary conditions were approved by the FDA throughout the study period. The median number of participants supporting each drug was 691 (interquartile range, 376–2158). Women represented 57% (n = 36,141) of trial participants (n = 63,242). Men represented the majority in drug trials after 2020 (up to 62% in 2024). There was no significant association between enrollment of women with drug approval year (P = 0.298). Among trial participants, 56% were White, 6% Black, 9% Asian, 7% Hispanic/Latino. There was no significant association between White (P = 0.351), Black (P = 0.697), or Hispanic/Latino (P = 0.927) enrollment with drug approval year. However, the proportion of Asian enrollment increased over time (β = 0.032, 95% CI 0.007–0.057, P = 0.016). Conclusion: From 2015-2024, there has been stable representation of women in clinical trial investigations for novel therapeutic agents within gastroenterology/hepatology. Despite 2020 FDA guidance efforts to increase female representation in clinical trials, men represented the majority from 2021–2024. There was a significant upward trend in Asian enrollment over time. Despite accounting for 14% and 19% of the total U.S. population, Black and Hispanic/Latino patients only accounted for 6% and 7% of the clinical trials population. Gender and racial/ethnicity equity in the participation of clinical trials is fundamental to the assurance of widespread safety and efficacy of novel therapeutics, highlighting the need for continued monitoring of future enrollment trends.