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. 2022 Mar 4;15(5):577–588. doi: 10.1007/s40271-022-00574-y
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.