Caregiver Preferences for Emerging Duchenne Muscular Dystrophy Treatments: A Comparison of Best-Worst Scaling and Conjoint Analysis

Through Patient-Focused Drug Development, the US Food and Drug Administration (FDA) documents the perspective of patients and caregivers and are currently conducting 20 public meetings on a limited number of disease areas. Parent Project Muscular Dystrophy (PPMD), an advocacy organization for Duchenne muscular dystrophy (DMD), has demonstrated a community-engaged program of preference research that would complement the FDA’s approach. Our...