Holly Peay|Ilene L. Hollin|Ryan Fischer|John F.P. Bridges
A Community-Engaged Approach to Quantifying Caregiver Preferences for the Benefits and Risks of Emerging Therapies for Duchenne Muscular Dystrophy
Parent Project Muscular Dystrophy|Johns Hopkins University|Parent Project Muscular Dystrophy|Johns Hopkins University
backgroundthere growing agreement regulators performing benefitrisk evaluations take patients caregivers preferences consideration patientfocused drug development initiative us food drug administration offers patients caregivers enhanced opportunity contribute regulatory processes offering direct testimonials process may advanced providing scientific evidence regarding treatment preferences engagement broad community patients caregiversobjectivein article demonstrate communityengaged approach measure caregiver preferences potential benefits risks emerging therapies duchenne muscular dystrophy dmdmethodsan advocacy oversight team led communityengaged study caregivers treatment preferences measured using bestworst scaling bws six relevant understandable attributes describing potential benefits risks emerging dmd therapies identified engagement advocates n 5 clinicians n 9 drug developers pharmaceutical companies academic centers n 11 stakeholders n 5 attributes defined across 3 levels included muscle function life span knowledge drug nausea risk bleeds risk arrhythmia cognitive interviewing caregivers n 7 used refine terminology assess acceptability bws instrument study implemented online survey dmd caregivers recruited united states advocacy group snowball sampling caregivers presented 18 treatment profiles identified via maineffect orthogonal experimental design dependent variable respondents judgment best worst feature profile preference weights estimated calculating relative number times feature chosen best worst used estimate relative attribute importanceresultsa total 119 dmd caregivers completed bws instrument predominately biological mothers 672 married 899 white 916 treatment effect muscle function important among experimental attributes 287 followed risk heart arrhythmia 224 risk bleeding 212 additional postapproval data relatively least important attribute 23conclusionswe present model process advocacy organizations aiming promote patientcentered drug development communityengaged approach successfully used develop implement survey measure caregiver preferences caregivers willing accept serious risk balanced noncurative treatment even absent improvement life span preferences inform food drug administrations benefitrisk assessment emerging dmd therapies study highlights synergistic integration traditional advocacy methods scientific approach quantify benefitrisk preferences