Exploring the Landscape of Patient-Reported Outcomes in PCOS Clinical Trials
Author(s)
Rinchen Doma, BA, BS, MPH1, Naomi Suminski, MS2, Mahak Jain, MSc3.
1Clinical Outcomes Assessment Senior Associate, Parexel, Durham, NC, USA, 2Parexel International, San Diego, CA, USA, 3Parexel International, Mumbai, India.
1Clinical Outcomes Assessment Senior Associate, Parexel, Durham, NC, USA, 2Parexel International, San Diego, CA, USA, 3Parexel International, Mumbai, India.
Presentation Documents
OBJECTIVES: Polycystic ovarian syndrome (PCOS) affects approximately 8-13% of women of reproductive age and is a leading cause of infertility. While PCOS is known for affecting reproductive health, it is also associated with other health issues that affect patients’ physical health (such as acne/skin issues and excessive hair growth) and emotional health (such as anxiety and depression). This review aimed to evaluate the landscape of patient reported outcomes (PROs) and concepts measured in PCOS clinical trials.
METHODS: The terms “Polycystic Ovary Syndrome” and “PCOS” were searched in ClinicalTrials.gov to identify Phase 2-4 active or completed interventional trials. Endpoints from resulting studies were extracted and each study’s primary, secondary, and exploratory outcome data was reviewed. Studies including PROs were identified and trends in PRO use were analyzed.
RESULTS: 172 total studies were included in the review, of which only 43 included PROs. Most endpoints measured clinical markers of reproductive health (pregnancy/ovulation rate or hormone levels) or BMI/weight. The most frequently utilized PRO in trials was unspecified menstruation questionnaires (n=30), followed by the PCOS-QOL (n=6); other studies referenced quality of life (QoL) instruments, such as SF-36 (n=2), ChiQoL (n=1), FertiQoL (n=1), QoL VAS (n=1), or mentioned QoL PROs but were not specific (n=3). Among QoL questionnaires, most contained items measuring emotional function (n=4). Other PROs identified assessed signs/symptoms (n=7), especially weight/BMI (n=3), menstrual characteristics (n=2), bodily pain (n=2), and treatment satisfaction (n=3). Most PROs supported primary and/or secondary endpoints (n=16 and n=27 respectively).
CONCLUSIONS: Less than half of the PCOS trials included PROs, among which the majority focus solely on menstrual characteristics. There was either a lack of clarity or consistency among the PROs that were incorporated. As treatment and management of PCOS extends beyond just reproductive health, further efforts need to be made to incorporate a comprehensive assessment of the patient experience.
METHODS: The terms “Polycystic Ovary Syndrome” and “PCOS” were searched in ClinicalTrials.gov to identify Phase 2-4 active or completed interventional trials. Endpoints from resulting studies were extracted and each study’s primary, secondary, and exploratory outcome data was reviewed. Studies including PROs were identified and trends in PRO use were analyzed.
RESULTS: 172 total studies were included in the review, of which only 43 included PROs. Most endpoints measured clinical markers of reproductive health (pregnancy/ovulation rate or hormone levels) or BMI/weight. The most frequently utilized PRO in trials was unspecified menstruation questionnaires (n=30), followed by the PCOS-QOL (n=6); other studies referenced quality of life (QoL) instruments, such as SF-36 (n=2), ChiQoL (n=1), FertiQoL (n=1), QoL VAS (n=1), or mentioned QoL PROs but were not specific (n=3). Among QoL questionnaires, most contained items measuring emotional function (n=4). Other PROs identified assessed signs/symptoms (n=7), especially weight/BMI (n=3), menstrual characteristics (n=2), bodily pain (n=2), and treatment satisfaction (n=3). Most PROs supported primary and/or secondary endpoints (n=16 and n=27 respectively).
CONCLUSIONS: Less than half of the PCOS trials included PROs, among which the majority focus solely on menstrual characteristics. There was either a lack of clarity or consistency among the PROs that were incorporated. As treatment and management of PCOS extends beyond just reproductive health, further efforts need to be made to incorporate a comprehensive assessment of the patient experience.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
PCR41
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Diabetes/Endocrine/Metabolic Disorders (including obesity), SDC: Reproductive & Sexual Health