Patient-centered benefit-risk assessment in duchenne muscular dystrophy

Ilene L. Hollin, Holly L. Peay, Susan D. Apkon, John F P Bridges

Research output: Contribution to journalArticle

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.

Original languageEnglish (US)
JournalMuscle and Nerve
DOIs
StateAccepted/In press - 2017

Fingerprint

Duchenne Muscular Dystrophy
Caregivers
Cough
Diarrhea
Lung
Patient Preference
Logistic Models
Surveys and Questionnaires
Therapeutics

Keywords

  • Best-worst scaling
  • Duchenne/Becker muscular dystrophy
  • Patient preferences
  • Patient-centered benefit-risk assessment
  • Quality of life
  • Regulatory review

ASJC Scopus subject areas

  • Physiology
  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)

Cite this

Hollin, I. L., Peay, H. L., Apkon, S. D., & Bridges, J. F. P. (Accepted/In press). Patient-centered benefit-risk assessment in duchenne muscular dystrophy. Muscle and Nerve. https://doi.org/10.1002/mus.25411

Patient-centered benefit-risk assessment in duchenne muscular dystrophy. / Hollin, Ilene L.; Peay, Holly L.; Apkon, Susan D.; Bridges, John F P.

In: Muscle and Nerve, 2017.

Research output: Contribution to journalArticle

Hollin, Ilene L. ; Peay, Holly L. ; Apkon, Susan D. ; Bridges, John F P. / Patient-centered benefit-risk assessment in duchenne muscular dystrophy. In: Muscle and Nerve. 2017.
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