医疗利益相关者对杜氏肌萎缩症治疗价值评估方法的看法

Ryan Fischer, Pat Furlong, Annie Kennedy, Kelly Maynard, Marissa Penrod, Debra Miller, Chamindra G Laverty, Linda Lowes, Nancy L Kuntz, Perry B Shieh, Jane Kondejewski, Peter J Neumann, Jason Shafrin, Richard J Willke
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引用次数: 0

摘要

背景:传统的价值评估框架在全面评估新疗法对罕见、进行性、遗传性、致命性神经肌肉疾病(如杜氏肌营养不良症(DMD))患者带来的社会价值方面受到挑战。目的:确定如何调整价值评估框架来衡量DMD治疗的社会价值。方法:三组利益相关者(患者倡导者、临床医生、卫生经济学家)参加了围绕国际药物经济学和结果研究学会价值花的半结构化访谈,其中包括可在医疗保健技术价值评估中考虑的价值要素。结果:所有利益相关者一致认为,基于质量调整生命年(QALY)的传统价值评估框架是狭隘的,并且会低估新的DMD疗法。所有利益攸关方都表达了一定程度的关切,认为使用质量aly作为衡量价值的关键指标是对患有严重进行性疾病和残疾的患者的歧视。一些利益攸关方认为,如果方法适当,在资源有限的环境中使用质量aly进行跨疾病比较是有价值的。所有利益相关者都建议在围绕新的DMD疗法的决策中考虑其他有价值的因素。这些因素反映了:杜氏症患者、护理人员和家庭所产生的经济和人文成本,如间接自付费用、生产力损失和家庭溢出效应;对残疾人和高度未满足需求的个人有意义的属性,如疾病的严重程度、希望的价值和实物期权价值;以及有助于改善人口健康的因素,如保险价值、公平和科学溢出效应。结论:这些发现强调了在评估新的DMD疗法的社会价值时,需要扩展传统的价值评估框架,并采取一种综合个人、护理人员、临床医生和健康经济学家观点的整体方法。扩大价值评估将防止Duchenne患者限制或延迟获得治疗。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies
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 around the International Society for Pharmacoeconomics and Outcomes Researchs Value Flower, which includes elements of value that can be considered 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 use of 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: the economic and humanistic costs incurred by patients, caregivers, and families with Duchenne, such as indirect out-of-pocket costs, lost productivity, and family spillovers; the attributes that are meaningful 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. CONCLUSIONS: 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 heath 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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