Make Her Voice Count: Understanding the Use of Patient-Reported Outcomes in Contraceptive Trials

OBJECTIVES: Patient-reported outcomes (PROs) in clinical endpoints have increased following regulatory emphasis on the patient voice supporting drug approval. Although contraceptives continue to be evaluated, many were approved prior to current patient-centered guidance. Contraceptives are used widely for intended (e.g., pregnancy prevention) and off-label purposes (e.g., menstrual pain, acne), and assessment of dimensions beyond pregnancy is crucial to holistically understand patient experiences. This research was conducted to understand PRO inclusion in clinical trials for contraceptives.

METHODS: A search was completed using clinicaltrials.gov to identify Phase 2-4 trials for interventional contraceptive treatments for females. Details extracted included: date of initiation, use of PROs/inclusion of PROs in endpoints, and concepts assessed and by which PROs. Trends over time were also evaluated.

RESULTS: Of trials identified, 219 were included in this review (initiated between 1997 and 2022). Seventy-three included PROs, while the remaining 146 were either unclear about use of PROs (n=52) or no PRO/potential patient-reported concept was mentioned (n=94). Thirty trials included PROs as a primary endpoint and 63 included PROs as a secondary or ‘other’ endpoint. The concepts most commonly assessed by PROs were satisfaction and bleeding/spotting. The PROs used most were questionnaires assessing satisfaction and patient diaries assessing bleeding and/or spotting. All other concepts and PROs were included in <25.0% of relevant trials. Prior to the Food and Drug Administration (FDA) PRO Guidance, 29.5% of the trials specified use of PROs, whereas this rose to 40.5% after it was published.

CONCLUSIONS: While PROs were used more commonly after the FDA PRO Guidance, they were still included in less than half of trials. PROs were not used consistently or clearly detailed, and there was a limited number of concepts frequently assessed, demonstrating restrictions and inconsistencies in capturing the patient voice. Future research for contraceptive efficacy would benefit from a broadened assessment of the patient perspective.