PMU46 TRIAL SEPARATION - IS THERE A TRUE GENDER DISPARITY IN CLINICAL TRIALS?

Abstract

The gender imbalance in clinical studies has been widely discussed with women underrepresented in trial populations and becoming a growing concern. However, there may be several mitigating factors such as ability to recruit, gender-specific diseases, incidence variations and genetic predispositions. To drive a change in gender bias in this aspect of clinical research, more incisive analyses are required to define the true difference and understand the root causal relationship. This research aims to assess the current state of the gender disparity in pivotal clinical trials supporting recent FDA (Food and Drug Administration) CDER (Center for Drug Evaluation and Research) novel drug approvals. Publicly-available FDA CDER Drug Trial Snapshots were used to identify the gender split in participants of key trials supporting novel drug approvals between 01/01/2014-31/12/2018. For the 172 novel drugs approved by the FDA between 2014 to 2018, there were 106,894 (49.4%) male subjects, and 109,600 (50.6%) female subjects in total. In terms of specific therapy areas, for the 50 novel oncology drugs, there were 9,201 (45.4%) male subjects and 11,066 (54.6%) female subjects. For the 9 novel cardiovascular drugs, there were 33,721 (55.8%) male subjects, and 26,683 (44.2%) female subjects. However, there are a number of women-specific diseases which had zero male subjects in the trials such as ovarian cancer and bacterial vaginosis. There are also rare hereditary genetic diseases which had zero female trial subjects such as Duchenne muscular dystrophy and hemophilia A. Despite the inherent mitigating factors, the gender disparity remains unclear with women well-represented, underrepresented, or overrepresented in trials of novel drugs depending on the therapy area. Recruitment and retention of women in clinical studies is a key challenge requiring further research to defining the true gender differences in clinical trials and also to develop strategies to overcome these differences.