Sex representation in trials relative to indication-specific disease burden in FDA-approved drugs (2015–2023)

Abstract

Abstract Sex-based disparities in disease burden and therapeutic response motivate efforts to prioritize women’s health in drug development. We analyzed 195 drugs across 98 indications approved by the United States Food and Drug Administration (FDA) between 2015 and 2023 to assess whether industry focus and clinical trial enrollment reflect disease prevalence among males and females. Here, we show that therapies for female-predominant indications receive 1.5 times more approvals than male-predominant indications. Additionally, among trials leading to approval, female participation aligns with or exceeds disease prevalence in 67% of cases. Alignment is strongest in oncology, whereas cardiovascular and autoimmune diseases most often under-enroll women, with no improvement over time. We discuss that gains in female enrollment have plateaued and that further progress will require advances in diagnostics, greater use of objective endpoints, and improved tools to assess risks for women of reproductive potential.