Evidera, Patient-Centered Research Group, Bethesda, MD, USA.
Evidera, Patient-Centered Research Group, Bethesda, MD, USA.
Value Health. 2021 Jun;24(6):812-821. doi: 10.1016/j.jval.2020.12.017. Epub 2021 May 14.
Health technology assessment agencies often prefer that utilities used to calculate quality-adjusted life years in cost-utility analyses (CUAs) are derived using standardized methods, such as generic preference-based measures completed by patients in clinical trials. However, there are situations when no standardized approach is feasible or appropriate for a specific medical condition or treatment that must be represented in a CUA. When this occurs, vignette-based methods are often used to estimate utilities. A vignette (sometimes called a "scenario," "health state description," "health state vignette," or "health state") is a description of a health state that is valued in a preference elicitation task to obtain a utility estimate. This method is sometimes the only feasible way to estimate utilities representing a concept that is important for a CUA. Consequently, vignette-based studies continue to be conducted and published, with the resulting utilities used in economic models to inform decision making about healthcare resource allocation. Despite the potential impact of vignette-based utilities on medical decision making, there is no published guidance or review of this methodology. This article provides recommendations for researchers, health technology assessment reviewers, and policymakers who may be deciding whether to use vignette-based methods, designing a vignette study, using vignette-based utilities in a CUA, or evaluating a CUA that includes vignette-based utilities. Recommendations are provided on: (A) when to use vignette-based utilities, (B) methods for developing vignettes, (C) valuing vignettes, (D) use of vignette-based utilities in models, and (E) limitations of vignette methods.
卫生技术评估机构通常希望用于计算成本效用分析（CUA）中质量调整生命年的效用值是使用标准化方法得出的，例如通过临床试验中的患者完成的通用偏好量表。然而，在某些情况下，对于特定的医疗状况或治疗方法，没有标准化的方法是可行的或合适的，而这些情况必须在 CUA 中得到体现。当这种情况发生时，通常会使用基于情景的方法来估计效用值。情景（有时称为“情景”、“健康状态描述”、“健康状态情景”或“健康状态描述”）是在偏好 elicitation 任务中对健康状态进行估值以获得效用估计值的描述。对于代表 CUA 中重要概念的效用值，这种方法有时是唯一可行的估计方法。因此，基于情景的研究仍在继续进行和发表，由此产生的效用值用于经济模型中，为医疗资源分配的决策提供信息。尽管基于情景的效用值对医疗决策有潜在影响，但目前没有关于该方法的发表指南或审查。本文为可能决定是否使用基于情景的方法、设计情景研究、在 CUA 中使用基于情景的效用值或评估包含基于情景的效用值的 CUA 的研究人员、卫生技术评估审查人员和政策制定者提供建议。建议涉及：（A）何时使用基于情景的效用值，（B）开发情景的方法，（C）对情景进行估值，（D）在模型中使用基于情景的效用值，以及（E）情景方法的局限性。
