Abstract 11144: Demographics of Study Participants in Clinical Trials for Cardiovascular Drugs Approved at FDA CDER From 2013 to 2014

Abstract

Introduction: Cardiovascular disease (CVD) is the leading cause of death for women in the United States. To assess the safety and efficacy of CVD drugs in the diverse population of patients who will use them post-approval, the US FDA has implemented guidance and regulation to encourage the inclusion of women and racial/ethnic minorities in drug clinical trials (CTs). This study assessed the participation of women and minorities in CTs for CVD indications for New Drug Applications (NDAs) approved at FDA CDER from 2013-2014. Methods: The sex, race/ethnicity, and age of subjects who participated in CVD drug CTs were assessed from final clinical study reports accessed via internal FDA databases, and were evaluated for the presence of data analysis by sex. The ratio of the percent of women in the CT population was also compared to those in the disease population. Results: Four novel CVD drugs were approved in this time frame, including 2 drugs indicated for pulmonary hypertension (PH) (riociguat, total CT population n=2691; macitentan, n=1884), and 1 drug each for prevention of thrombotic acute CV events (vorapaxar, n=41972) and treatment of neurogenic orthostatic hypotension (NOH) (droxidopa, n=1125). Demographic subgroup analysis indicated that percent female participation was 26% for thrombotic CV events, 40% for NOH, and 47% for PH. Female participation in pivotal trials only (in which benefit-risk was demonstrated) was 24% for thrombotic CV events, 33% for NOH, and 75% for PH. The majority of the trial participants were White (85%), and mean ages were 48 years (PH), 61 years (thrombotic CV events), and 64 years (NOH). Sex-based analysis for both efficacy and safety was presented for all drugs. Three of the NDAs with available disease prevalence data had a ratio of percent female participation to disease prevalence that was between 0.80 and 1.2. Conclusions: Percent female participation varied substantially by disease indication, with PH (a female predominant disease) having the highest female participation. Female participation in the prevention of thrombotic CV events was lower than anticipated based upon disease prevalence in the population. Further exploration of demographic subgroup participation in CTs for CVD drugs in a larger sample size is ongoing.