Caregiver preferences for emerging Duchenne muscular dystrophy treatments

Abstract

Background: 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. Objective: Our objective was to compare two stated-preference methods, best-worst scaling (BWS) and conjoint analysis, within a study measuring caregivers’ DMD-treatment preferences. Methods: Within one survey, two preference-elicitation methods were applied to 18 potential treatments incorporating six attributes and three levels. For each treatment profile, caregivers identified the best and worst feature and intention to use the treatment. We conducted three analyses to compare the elicitation methods using parameter estimates, conditional attribute importance and policy simulations focused on the 18 treatment profiles. For each, concordance between the results was compared using Spearman’s rho. Results: BWS and conjoint analysis produced similar parameter estimates (p