Presentation (CTI)

OBJECTIVES: Women spend 25% more of their lives in debilitating health than men. To help bridge the women’s health (WH) gap, the FDA’s WH Office updated their Research Roadmap in 2024. Selecting appropriate clinical outcome assessments (COAs) and endpoints was cited again as a key priority area. An earlier version of our WH study took a bottom-up approach, reviewing COAs developed in a female population. This study adopts a top-down approach, using roadmap recommended therapeutic areas (TAs) to explore the WH COA landscape by identifying COA availability, as well as their presence in regulatory guidance and drug label claims.
METHODS: The roadmap outlines 13 priority TAs, which were mapped to Medical Subject Heading therapeutic indications (TI). These were used to search a COA library (PROQOLID^TM), regulatory guidelines database with COA recommendations (PROINSIGHT^TM), and drug label claims database (PROLABELS^TM).
RESULTS: 527 COAs, of which 47 were developed in a female-specific population, were identified covering 254 TIs. The greatest number of COAs developed in a female-specific population were for breast neoplasms (n=13 COAs), osteoporosis (n=9), and pregnancy (n=8). 234 TIs did not have a single COA developed in a female-specific population. 21 guidelines were identified including 18 unique COAs, none were developed in a female-specific population. TIs with the greatest number of guidelines were obesity (n=3 TIs) and diabetes mellitus (n=3).106 labels were retrieved encompassing 48 unique COAs, only 8 developed in a female-specific population. Prevalent TIs were osteoporosis (n=30 TIs), breast neoplasms (n=20) and diabetes mellitus (n=20).
CONCLUSIONS: Certain TAs outlined in the roadmap require further COA research. For example, no guidelines with a named COA were identified for cardiovascular disease and there were limited COAs available. Greater emphasis could also be placed on developing female-specific COAs for indications that impact men and women but whose effects manifest differently, for example musculoskeletal diseases.