2023
DOI: 10.1101/2023.07.11.23292507
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Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies

Ryan Fischer,
Pat Furlong,
Annie Kennedy
et al.

Abstract: BACKGROUND: Traditional value assessment frameworks are challenged in comprehensively assessing the societal value new therapies bring to individuals with rare, progressive, genetic, fatal, neuromuscular diseases such as Duchenne muscular dystrophy (DMD). OBJECTIVE: To identify how value assessment frameworks may need to be adapted to measure the value to society of DMD therapies. METHODS: Three groups of stakeholders (patient advocates, clinicians, health economists) participated in semi-structured interviews… Show more

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