Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies
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Le résumé fourni par la source
Purpose: 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). The objective of this study was to identify how value assessment frameworks may need to be adapted to measure the value to society of DMD therapies. Patients and Methods: Three stakeholder groups (6 patient advocates, 4 clinicians, 3 health economists; N = 13) participated in semi-structured interviews around the International Society for Pharmacoeconomics and Outcomes Research's Value Flower, which includes elements to consider within value assessments of healthcare technologies. Results: All stakeholders agreed that traditional value assessment frameworks based on the quality-adjusted life year (QALY) are narrow and will undervalue new DMD therapies. All stakeholders expressed some level of concern that using the QALY as a key metric of value discriminates against patients with severe progressive diseases and disabilities. Some stakeholders saw value in using the QALY for cross-disease comparisons in resource-constrained environments if the methodology was appropriate. All stakeholders recommended considering additional elements of value in decision-making around new DMD therapies. These elements reflect: economic and humanistic costs incurred by patients, caregivers, and families with Duchenne, such as indirect out-of-pocket costs, lost productivity, and family spillovers; meaningful attributes for individuals with disabilities and high unmet need, such as severity of disease, value of hope, and real option value; and factors that contribute to improvements in population health, such as insurance value, equity, and scientific spillovers. Conclusion: These findings highlight the need to expand traditional value assessment frameworks and take a holistic approach that incorporates the perspectives of individuals with Duchenne, caregivers, clinicians, and health economists when assessing the societal value of new DMD therapies. Broadening value assessment will prevent restricted or delayed access to therapies for individuals with Duchenne.
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Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies
- Date Crossref
- 01/08/2024
- Éditeur
- Informa UK Limited
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Où se fait cette recherche
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Parent Project Muscular Dystrophy pays non établi dans la noticeOrganisation à but non lucratif
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Hercules Foundation pays non établi dans la noticeOrganisation à but non lucratif
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Cranbrook Academy of Art pays non établi dans la noticeInstitution
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CureDuchenne pays non établi dans la noticeOrganisation à but non lucratif
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University of California San Diego Department of Neuroscience pays non établi dans la noticeUniversité ou école supérieure
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Nationwide Children's Hospital pays non établi dans la noticeÉtablissement de santé
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The Ohio State University Department of Pediatrics pays non établi dans la noticeUniversité ou école supérieure
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Northwestern University Department of Pediatrics pays non établi dans la noticeUniversité ou école supérieure
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Lurie Children's Hospital Division of Neurology pays non établi dans la noticeÉtablissement de santé
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University of California Department of Neurology and Pediatrics pays non établi dans la noticeUniversité ou école supérieure
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Tufts Medical Center pays non établi dans la noticeÉtablissement de santé
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University of Tennessee at Chattanooga pays non établi dans la noticeUniversité ou école supérieure
Parent Project Muscular Dystrophy, Hercules Foundation et Cranbrook Academy of Art, avec 9 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.