Andronis L, Barton P, Bryan S
Department of Health Economics, University of Birmingham, UK.
Health Technol Assess. 2009 Jun;13(29):iii, ix-xi, 1-61. doi: 10.3310/hta13290.
To determine how we define good practice in sensitivity analysis in general and probabilistic sensitivity analysis (PSA) in particular, and to what extent it has been adhered to in the independent economic evaluations undertaken for the National Institute for Health and Clinical Excellence (NICE) over recent years; to establish what policy impact sensitivity analysis has in the context of NICE, and policy-makers' views on sensitivity analysis and uncertainty, and what use is made of sensitivity analysis in policy decision-making.
Three major electronic databases, MEDLINE, EMBASE and the NHS Economic Evaluation Database, were searched from inception to February 2008.
The meaning of 'good practice' in the broad area of sensitivity analysis was explored through a review of the literature. An audit was undertaken of the 15 most recent NICE multiple technology appraisal judgements and their related reports to assess how sensitivity analysis has been undertaken by independent academic teams for NICE. A review of the policy and guidance documents issued by NICE aimed to assess the policy impact of the sensitivity analysis and the PSA in particular. Qualitative interview data from NICE Technology Appraisal Committee members, collected as part of an earlier study, were also analysed to assess the value attached to the sensitivity analysis components of the economic analyses conducted for NICE.
All forms of sensitivity analysis, notably both deterministic and probabilistic approaches, have their supporters and their detractors. Practice in relation to univariate sensitivity analysis is highly variable, with considerable lack of clarity in relation to the methods used and the basis of the ranges employed. In relation to PSA, there is a high level of variability in the form of distribution used for similar parameters, and the justification for such choices is rarely given. Virtually all analyses failed to consider correlations within the PSA, and this is an area of concern. Uncertainty is considered explicitly in the process of arriving at a decision by the NICE Technology Appraisal Committee, and a correlation between high levels of uncertainty and negative decisions was indicated. The findings suggest considerable value in deterministic sensitivity analysis. Such analyses serve to highlight which model parameters are critical to driving a decision. Strong support was expressed for PSA, principally because it provides an indication of the parameter uncertainty around the incremental cost-effectiveness ratio.
The review and the policy impact assessment focused exclusively on documentary evidence, excluding other sources that might have revealed further insights on this issue. In seeking to address parameter uncertainty, both deterministic and probabilistic sensitivity analyses should be used. It is evident that some cost-effectiveness work, especially around the sensitivity analysis components, represents a challenge in making it accessible to those making decisions. This speaks to the training agenda for those sitting on such decision-making bodies, and to the importance of clear presentation of analyses by the academic community.
确定我们如何定义一般敏感性分析尤其是概率敏感性分析(PSA)中的良好实践,以及近年来为英国国家卫生与临床优化研究所(NICE)进行的独立经济评估在多大程度上遵循了该定义;确定敏感性分析在NICE背景下的政策影响、政策制定者对敏感性分析和不确定性的看法,以及敏感性分析在政策决策中的用途。
检索了三个主要电子数据库,即MEDLINE、EMBASE和英国国家医疗服务体系经济评估数据库,检索时间从建库至2008年2月。
通过文献综述探讨敏感性分析广泛领域中“良好实践”的含义。对NICE最近的15项多项技术评估判断及其相关报告进行了审核,以评估独立学术团队为NICE进行敏感性分析的方式。对NICE发布的政策和指导文件进行综述,旨在评估敏感性分析尤其是PSA的政策影响。还分析了作为早期研究一部分收集的NICE技术评估委员会成员的定性访谈数据,以评估为NICE进行的经济分析中敏感性分析部分的价值。
所有形式的敏感性分析,尤其是确定性和概率性方法,都有其支持者和反对者。单变量敏感性分析的实践差异很大,在使用的方法和所采用范围的依据方面存在很大的不明确性。关于PSA,类似参数所使用的分布形式差异很大,而且很少给出此类选择的理由。几乎所有分析都未考虑PSA中的相关性,这是一个令人担忧的领域。NICE技术评估委员会在做出决策的过程中明确考虑了不确定性,并表明高度不确定性与负面决策之间存在相关性。研究结果表明确定性敏感性分析具有相当大的值。此类分析有助于突出哪些模型参数对推动决策至关重要。对PSA表示了强烈支持,主要是因为它提供了增量成本效益比周围参数不确定性的指标。
综述和政策影响评估仅侧重于文献证据,排除了可能揭示该问题更多见解的其他来源。在寻求解决参数不确定性时,应同时使用确定性和概率性敏感性分析。显然,一些成本效益工作,尤其是围绕敏感性分析部分,在使其对决策者可用方面构成挑战。这涉及到此类决策机构成员的培训议程,以及学术界清晰呈现分析的重要性。