Participant Engagement and Preference Study for Clinical Outcomes Associated With Atrial Fibrillation: The PEARL-AF Study.

Background: Quantifying patients' preferences for health outcomes associated with atrial fibrillation (AF) and its treatments offers a replicable approach to considering the patient perspective in regulatory decision-making.

Objective: The authors conducted a preference survey to estimate the relative importance of AF-related events for use in clinical trial analyses to estimate net health benefits with anticoagulants.

Methods: The survey included nontechnical descriptions of three severities of stroke, systemic embolism, myocardial infarction (MI) with or without subsequent heart failure (HF), major bleeding, clinically relevant nonmajor bleeding, and death. A best-worst scaling question format was used in which patients were shown 10 sets of four events and asked to select what they considered to be most and least serious.

Results: One thousand twenty-eight patients, mean age 69.2 years, 40.4% female, completed the survey. Best-worst scaling importance weights were significantly different across all events except between major bleeding and MI with HF. Death was considered the most serious (reweighted to 1), followed by severe disabling stroke (0.83), then major bleeding (0.53) or MI with HF (0.50), moderate-severity stroke (0.28) and systemic embolism (0.13). Clinically relevant nonmajor bleeding, MI without HF, and minor stroke (0.10, 0.06, and 0.04, respectively) were considered least serious. Events ordered by importance were consistent across age, sex, and race, but relative weights across events varied by sex and race.

Conclusions: Patients expressed relatively high levels of concern about major bleeding compared to moderate-severity stroke or systemic embolism, endpoints frequently used in AF trials. Estimated weights could be used in patient-centered net-benefit determinations for AF therapies.