Race and ethnicity reporting and representation in hemophilia clinical trials

• Published in: Blood advances 2024-05, Vol.8 (10), p.2351-2360
• Main Authors: Fedewa, Stacey A., Valentino, Leonard A., Koo, Andee, Cafuir, Lorraine, Tran, Duc Q., Antun, Ana, Kempton, Christine L.
• Format: Article
• Language: English
• Subjects: Adult; Asian; Asian People; Black or African American; Black People; Canada; Clinical Trials as Topic; Cross-Sectional Studies; Ethnicity; Hemophilia A - ethnology; Hemophilia A - therapy; Hispanic or Latino; Humans; Racial Groups; United Kingdom; United States; White; White People
• ISSN: 2473-9529; 2473-9537; 2473-9537
• DOI: 10.1182/bloodadvances.2024012862

•Despite increases in the reporting of race/ethnicity, Black and Hispanic persons were consistently underrepresented in hemophilia trials.•Strategies must be developed to overcome the underrepresentation of historically marginalized populations in interventional trials. [Display omitted] Racial and ethnic representativeness in clinical trials is crucial to mitigate disparities in outcomes; however, diversity among hemophilia trials is unknown. The aim of this study is to examine the reporting and representation of race and ethnicity in trials of people with hemophilia (PwH). In this cross-sectional study, the ClinicalTrials.gov database was queried in April 2023 for interventional clinical trials involving PwH between 2007 and 2022. The distribution of participants (observed) was compared with expected proportions based on US Hemophilia Treatment Center (HTC) and country-specific census data with observed-to-expected ratios (OERs). Of 129 trials included, 94.6% were industry sponsored, with a mean of 62 participants and mean age of 26.8 years. Overall, 52.0% (n = 66) of trials reported data on race and ethnicity, increasing from 13.9% in 2007-2012 to 22.5% in 2013-2016 to 100% in 2017-2022 (P = .001). Among these 66 trials, 65.8%, 22.8%, 5.1%, 3.9% of participants were White, Asian, Hispanic, and Black, respectively. OERs were 10% to 20% lower for White participants vs US HTC, and US, UK, and Canadian census populations and ∼75% lower for Black or Hispanic participants when compared with US HTC and US census population. OERs for Asian participants were 1.6 to 3 times higher than Canada, US, and UK census populations. The reporting of race and ethnicity in hemophilia trials has drastically improved; however, Black and Hispanic PwH remain especially underrepresented. To address these disparities, stakeholders across the clinical trial enterprise need to implement strategies to ensure equitable participation.