Presentation (CTI)

OBJECTIVES: Pain is a prevalent, debilitating symptom across women’s health conditions, often significantly impacting quality of life. This study examined how pain is assessed in clinical trials, with a focus on exploring the prioritisation of pain within endpoint hierarchies, and in drug labels in women’s health.
METHODS: The ClinicalTrials.gov database was searched to identify phase 2 and 3 interventional trials in select women’s health conditions (e.g., dysmenorrhoea and endometriosis). Product labelling of treatments for pain associated with women’s health conditions were collected from the Drugs@FDA database. Searches were conducted in June 2025. Data on trial design, intervention, indication/s, endpoints, clinical outcome assessments (COAs) and label language were extracted.
RESULTS: A total of 535 trials were identified; endometriosis trials were most frequent (n=151/535, 28.2%), followed by fibroids and dysmenorrhoea. Fewer than half of the trials specifically referenced ‘pain’ in primary, secondary or other endpoint wording (n=205/535, 38.3%). Standalone visual analogue scales, numerical or verbal rating scales, disease-specific (e.g., Dysmenorrhea Daily Diary), and generic (e.g., short-form McGill Pain Questionnaire) COAs supported primary endpoints of pain. In the past 15 years, only two FDA-approved drugs for pain associated with a women’s health condition were identified, both for endometriosis; the Endometriosis Daily Pain Impact Scale, daily disease-specific numeric rating scales and the Endometriosis Health Profile pain domain supported co-primary/secondary endpoints and were included in product labelling. Other drugs for pain were approved prior to the FDA Patient-Reported Outcome guidance (2009) or for conditions generally also affecting males (e.g., fibromyalgia).
CONCLUSIONS: Despite a high volume of interventional trials in women’s health, particularly for endometriosis, less than half referenced assessments of pain in the endpoint wording, and few recent FDA-approved drugs for pain in women’s health conditions were identified. This underscores the unmet need for both pain-targeting therapies and prioritising appropriate assessments of pain in COA strategies within women’s health.