title: Patient-centered benefit-risk assessment in duchenne muscular dystrophy.
abstract: This study quantified caregiver and patient preferences for a therapeutic agent with demonstrated pulmonary benefits for Duchenne muscular dystrophy (DMD). Caregiver and patient differences were also explored.
  
  
  
  A best-worst scaling survey (BWS) was administered to caregivers and patients. Across 9 profiles, respondents selected the best and worst attributes. Utility scores were estimated using mixed logistic regression.
  
  
  
  Respondents indicated greatest preference for therapies that maintain their current level of cough strength for 10 years or for 2 years. Preference scores for risks were low: 50% chance of diarrhea and 4 additional blood draws per year.
  
  
  
  There is a strong preference for pulmonary benefit and willingness to trade off risks and burden to achieve these benefits. In exchange for maintaining cough strength for 10 years, respondents were willing to tolerate high probabilities of diarrhea and additional blood draws. Muscle Nerve 55: 626-634, 2017.
authors.affiliation: Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland, USA.
authors.forename: Ilene L
authors.identifier: 0000-0003-1405-8687
authors.initials: IL
authors.lastname: Hollin
authors.affiliation: RTI International, Research Triangle Park, North Carolina, USA.|Parent Project Muscular Dystrophy, Hackensack, New Jersey, USA.
authors.forename: Holly L
authors.initials: HL
authors.lastname: Peay
authors.affiliation: Department of Rehabilitation Medicine, Seattle Children's Hospital and University of Washington, Seattle, Washington, USA.
authors.forename: Susan D
authors.initials: SD
authors.lastname: Apkon
authors.affiliation: Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland, USA.
authors.forename: John F P
authors.initials: JFP
authors.lastname: Bridges
mesh_terms: D000293:Adolescent; D000328:Adult; D017028:Caregivers; D002648:Child; D000066491:Clinical Decision-Making; D005260:Female; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy*; D010358:Patient Participation; D018570:Risk Assessment; D011795:Surveys and Questionnaires; D055815:Young Adult
keywords: Duchenne/Becker muscular dystrophy; best-worst scaling; patient preferences; patient-centered benefit-risk assessment; quality of life; regulatory review