Patient‐centered benefit–risk assessment in duchenne muscular dystrophy

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...

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Published inMuscle & 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
LanguageEnglish
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
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Summary: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 caregivers and patients. Across 9 profiles, respondents selected the best and worst attributes. Utility scores were estimated using mixed logistic regression. Results 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. Conclusion 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
Bibliography:Disclosure
H.P. was an employee of the Parent Project Muscular Dystrophy at the time of this research.
This study was supported by the Parent Project Muscular Dystrophy (PPMD, to I.H. and J.B.). PPMD received funding for this project from Santhera Pharmaceuticals. This work was also supported by a Patient‐Centered Outcomes Research Institute (PCORI) Methods Program award (ME‐1303‐5946 to J.B.) and by the Johns Hopkins–FDA Center for Excellence in Regulatory Science and Innovation (1U01FD004977‐01 to J.B.).
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ISSN:0148-639X
1097-4598
DOI:10.1002/mus.25411