Regulatory agencies often use caregiver preferences to inform decision making regarding degenerative, rare, pediatric, and other conditions. |
Previous research quantifying preferences in Duchenne muscular dystrophy indicated that caregivers and patients have different preferences, but evidence was limited by small samples and confounded by age and health status. |
This large international study indicates that caregivers and patients have concordant treatment preferences once adjusting for patient age, an indicator of disease progression. |
We present a methodological advancement in comparing preferences across cohorts using matching approaches. |