Patient‐centered benefit–risk assessment in duchenne muscular dystrophy

• Published in: Muscle & nerve Vol. 55; no. 5; pp. 626 - 634
• Main Authors: Hollin, Ilene L., Peay, Holly L., Apkon, Susan D., Bridges, John F.P.
• Format: Journal Article
• Language: English
• Published: United States Wiley Subscription Services, Inc 01.05.2017
• Subjects: Adolescent; Adult; best–worst scaling; Caregivers; Child; Clinical Decision-Making; Duchenne/Becker muscular dystrophy; Female; Humans; Male; Muscular dystrophy; Muscular Dystrophy, Duchenne - drug therapy; Patient Participation; patient preferences; patient‐centered benefit–risk assessment; Preferences; quality of life; regulatory review; Risk Assessment; Surveys and Questionnaires; Young Adult; regulatory review; Duchenne/Becker muscular dystrophy; quality of life; best-worst scaling; patient preferences; patient-centered benefit-risk assessment

ABSTRACT Introduction 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. Methods A best–worst scaling survey (BWS) was administered to caregi...