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Statistical approaches to handling uncertainty in health economic evaluation.

机译:处理卫生经济评估中不确定性的统计方法。

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摘要

Not so long ago, uncertainty in economic evaluation was handled almost exclusively using simple one-way sensitivity analysis methods whereby individual parameters of an analysis were varied one-by-one over a range of (often ad-hoc) values. With the increasing use of the clinical trial as a vehicle for economic evaluation, there has been increasing interest in the use of statistical methods for handling uncertainty in patient-level data on both costs and effects. However, even for studies based on secondary data, the use of statistical methods for specifying parameter distributions in so-called probabilistic analyses is becoming increasingly common and is now the method recommended by a number of good-practice guides and regulatory agencies. The aim of this paper is to review these statistical approaches to the handling of uncertainty, both for primary studies where patient level cost and effect information is available and in secondary studies that typically use decision analytical methods to synthesize summary information from the published literature. The approach throughout the paper will be to emphasize the intuition behind the methods rather than the technical details. Although the methodology is generally applicable, examples will be drawn from both primary and secondary economic evaluations in the area of gastroenterology. The first example relates to a primary care trial of dyspepsia management which sought to estimate the cost-effectiveness of managing patients through early endoscopy compared to usual care. The second example is a secondary study of the management of gastro-oesophageal reflux disease symptoms which considered six potential strategies simultaneously.
机译:不久之前,经济评估中的不确定性几乎只能使用简单的单向敏感性分析方法来处理,在这种方法中,分析的各个参数在(通常是)特定值范围内一对一地变化。随着越来越多地使用临床试验作为经济评估的手段,人们越来越关注使用统计方法来处理成本和效果方面的患者水平数据的不确定性。但是,即使对于基于辅助数据的研究,在所谓的概率分析中使用统计方法指定参数分布也变得越来越普遍,并且现在已成为许多良好实践指南和监管机构推荐的方法。本文的目的是回顾这些用于处理不确定性的统计方法,既适用于可获得患者水平成本和效果信息的基础研究,也适用于通常使用决策分析方法从已发表文献中汇总摘要信息的中学研究。贯穿本文的方法将是强调方法背后的直觉而不是技术细节。尽管该方法通常适用,但将从胃肠病学领域的主要和次要经济评估中得出实例。第一个例子涉及消化不良管理的初级保健试验,该试验试图评估与常规护理相比,通过早期内窥镜检查管理患者的成本效益。第二个例子是对胃食管反流疾病症状管理的二次研究,该研究同时考虑了六个潜在策略。

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