Mandelblatt Jeanne S, Ramsey Scott D, Lieu Tracy A, Phelps Charles E
Department of Oncology, Georgetown University Medical Center, Washington, DC, USA; Lombardi Comprehensive Cancer Center, Washington, DC, USA.
Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
Value Health. 2017 Feb;20(2):185-192. doi: 10.1016/j.jval.2016.11.013.
The recent acceleration of scientific discovery has led to greater choices in health care. New technologies, diagnostic tests, and pharmaceuticals have widely varying impact on patients and populations in terms of benefits, toxicities, and costs, stimulating a resurgence of interest in the creation of frameworks intended to measure value in health. Many of these are offered by providers and/or advocacy organizations with expertise and interest in specific diseases (e.g., cancer and heart disease). To help assess the utility of and the potential biases embedded in these frameworks, we created an evaluation taxonomy with seven basic components: 1) define the purpose; 2) detail the conceptual approach, including perspectives, methods for obtaining preferences of decision makers (e.g., patients), and ability to incorporate multiple dimensions of value; 3) discuss inclusions and exclusions of elements included in the framework, and whether the framework assumes clinical intervention or offers alternatives such as palliative care or watchful waiting; 4) evaluate data sources and their scientific validity; 5) assess the intervention's effect on total costs of treating a defined population; 6) analyze how uncertainty is incorporated; and 7) illuminate possible conflicts of interest among those creating the framework. We apply the taxonomy to four representative value frameworks recently published by professional organizations focused on treatment of cancer and heart disease and on vaccine use. We conclude that each of these efforts has strengths and weaknesses when evaluated using our taxonomy, and suggest pathways to enhance the utility of value-assessing frameworks for policy and clinical decision making.
近期科学发现的加速发展带来了更多的医疗保健选择。新技术、诊断测试和药物在益处、毒性和成本方面对患者及人群有着广泛不同的影响，这激发了人们对创建旨在衡量健康价值的框架的兴趣再度兴起。其中许多框架由对特定疾病（如癌症和心脏病）有专业知识和兴趣的医疗服务提供者和/或倡导组织提供。为了帮助评估这些框架的效用以及其中潜在的偏差，我们创建了一个包含七个基本组成部分的评估分类法：1）明确目的；2）详细说明概念方法，包括观点、获取决策者（如患者）偏好的方法以及纳入多维度价值的能力；3）讨论框架中所包含元素的纳入和排除情况，以及该框架是假定临床干预还是提供诸如姑息治疗或观察等待等替代方案；4）评估数据来源及其科学有效性；5）评估干预措施对特定人群治疗总成本的影响；6）分析不确定性是如何纳入的；7）阐明框架创建者之间可能存在的利益冲突。我们将该分类法应用于专业组织最近发布的四个分别关注癌症和心脏病治疗以及疫苗使用的代表性价值框架。我们得出结论，当使用我们的分类法进行评估时，这些努力中的每一项都有优点和缺点，并提出了提高价值评估框架对政策和临床决策效用的途径。
