10 Years on From the FDA’s Women’s Health Research Roadmap: A Landscape Review of Female-Specific Clinical Outcome Assessments (COA), Health Agency Recommendations and Drug Label Claims With COA Related to Women’s Health
Author(s)
Stott T1, Lien S2, Desvignes-Gleizes C3, Bothorel S3, Sherafat-Kazemzadeh R3
1Mapi Research Trust, Caluire et Cuire, 69, France, 2Mapi Research Trust, Lyon, France, 3Mapi Research Trust, Lyon, Rhone, France
Presentation Documents
OBJECTIVES: The FDA’s 2015 Women’s Health Research Roadmap advocated outcomes research into conditions solely or disproportionately affecting women and conditions which manifest differently in women compared to men.
As the Roadmap’s 10th birthday approaches, the objective was to assess its progress with a landscape review of female-specific COAs as well as women’s health-related FDA and EMA recommendations and endpoints regarding COAs in drug labels.METHODS: The term “females” was filtered for within the PROQOLIDTM database to identify COAs developed in specific female populations. From the female-specific COAs found, a list of conditions was generated to identify guidelines in PROINSIGHTTM and approved drugs in PROLABELSTM.
RESULTS: 72 COAs developed for female-specific conditions were identified (e.g. endometriosis) and 67 for conditions affecting men and women (e.g. urinary incontinence (UI)); however, these latter conditions disproportionately affect women (e.g. breast cancer (n=13 COAs) or osteoporosis (n=6 COAs)).
EMA issued 3 guidelines with COA recommendations for premenstrual dysphoric disorder (Daily Record of Severity of Problems (DRSP)), UI (Incontinence Stress Index/Incontinence Impact Questionnaire-Short Form), and menopause (Kupperman Index). Conversely, FDA issued 1 for psychological sexual dysfunction (Female Sexual Function Index (FSFI)/Female Sexual Distress Scale (FSDS)). Of COAs included in female condition-specific labels, only 35% (n=24) were developed in a female-specific population. 3 of the 6 female-specific EMA- and FDA-recommended COAs were claimed in FDA labels (DRSP, FSDS, FSFI) - none in EMA labels.CONCLUSIONS: Importantly, COAs are being developed for female-specific conditions, some of which are represented in EMA and FDA guidelines. Whilst these results are limited to conditions reflected in COAs developed with a female-specific population, the findings show these COAs are rarely included in female disease specific labels, underlining The Roadmap’s ongoing relevance. To ensure fit-for-purpose endpoint and outcome measure selection, women’s unique experiences should be incorporated into COA development and regulatory guidance for diseases affecting women and men.
Conference/Value in Health Info
Value in Health, Volume 27, Issue 12, S2 (December 2024)
Code
PCR69
Topic
Clinical Outcomes, Health Policy & Regulatory, Patient-Centered Research
Topic Subcategory
Approval & Labeling, Clinical Outcomes Assessment, Health Disparities & Equity, Instrument Development, Validation, & Translation
Disease
No Additional Disease & Conditions/Specialized Treatment Areas