A guide to handling missing data in cost-effectiveness analysis conducted within randomised controlled trials.
A guide to handling missing data in cost-effectiveness analysis conducted within randomised controlled trials.
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DOI：
10.1007/s40273-014-0193-3
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发表时间：
2014-12
影响因子：
4.4
通讯作者：
White, Ian R.
中科院分区：
文献类型：
--
作者：
Faria, Rita;Gomes, Manuel;Epstein, David;White, Ian R.
在随机对照试验的成本-效果分析（CEA）中，数据缺失是一个常见的问题。处理缺失数据的不当方法可能导致误导性的结果，并最终影响干预措施是否物有所值的决定。本文提供了如何处理试验内CEAs中缺失数据的实用指导，以下是一种原则性方法：(i)分析应基于对缺失数据机制的合理假设，即数据丢失的概率是否独立于或依赖于观察到的和/或未观察到的值；（ii）为基本情况所选择的方法应符合所假定的机制；（iii）进行敏感性分析，探讨结果随着假设的变化有多大程度的变化。该方法的实现分为三个阶段，并对其进行了详细描述：(1)描述性分析，以告知对缺失数据机制的假设；(2)如何在给定基本假设的备选方法之间进行选择；(3)灵敏度分析方法。案例研究说明了如何在实践中应用这种方法，包括软件代码。文章最后提出了实践建议和对未来研究的建议。本文的在线版本（doi:10.1007/s40273-014-0193-3）包含补充资料，仅供授权用户使用。
Missing data are a frequent problem in cost-effectiveness analysis (CEA) within a randomised controlled trial. Inappropriate methods to handle missing data can lead to misleading results and ultimately can affect the decision of whether an intervention is good value for money. This article provides practical guidance on how to handle missing data in within-trial CEAs following a principled approach: (i) the analysis should be based on a plausible assumption for the missing data mechanism, i.e. whether the probability that data are missing is independent of or dependent on the observed and/or unobserved values; (ii) the method chosen for the base-case should fit with the assumed mechanism; and (iii) sensitivity analysis should be conducted to explore to what extent the results change with the assumption made. This approach is implemented in three stages, which are described in detail: (1) descriptive analysis to inform the assumption on the missing data mechanism; (2) how to choose between alternative methods given their underlying assumptions; and (3) methods for sensitivity analysis. The case study illustrates how to apply this approach in practice, including software code. The article concludes with recommendations for practice and suggestions for future research. The online version of this article (doi:10.1007/s40273-014-0193-3) contains supplementary material, which is available to authorized users.
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