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制定医疗保健决策中结构化专家 elicitation 的参考协议:混合方法研究。

Developing a reference protocol for structured expert elicitation in health-care decision-making: a mixed-methods study.

机构信息

Centre for Health Economics, University of York, York, UK.

Department of Management Science, University of Strathclyde, Glasgow, UK.

出版信息

Health Technol Assess. 2021 Jun;25(37):1-124. doi: 10.3310/hta25370.

Abstract

BACKGROUND

Many decisions in health care aim to maximise health, requiring judgements about interventions that may have higher health effects but potentially incur additional costs (cost-effectiveness framework). The evidence used to establish cost-effectiveness is typically uncertain and it is important that this uncertainty is characterised. In situations in which evidence is uncertain, the experience of experts is essential. The process by which the beliefs of experts can be formally collected in a quantitative manner is structured expert elicitation. There is heterogeneity in the existing methodology used in health-care decision-making. A number of guidelines are available for structured expert elicitation; however, it is not clear if any of these are appropriate for health-care decision-making.

OBJECTIVES

The overall aim was to establish a protocol for structured expert elicitation to inform health-care decision-making. The objectives are to (1) provide clarity on methods for collecting and using experts' judgements, (2) consider when alternative methodology may be required in particular contexts, (3) establish preferred approaches for elicitation on a range of parameters, (4) determine which elicitation methods allow experts to express uncertainty and (5) determine the usefulness of the reference protocol developed.

METHODS

A mixed-methods approach was used: systemic review, targeted searches, experimental work and narrative synthesis. A review of the existing guidelines for structured expert elicitation was conducted. This identified the approaches used in existing guidelines (the 'choices') and determined if dominant approaches exist. Targeted review searches were conducted for selection of experts, level of elicitation, fitting and aggregation, assessing accuracy of judgements and heuristics and biases. To sift through the available choices, a set of principles that underpin the use of structured expert elicitation in health-care decision-making was defined using evidence generated from the targeted searches, quantities to elicit experimental evidence and consideration of constraints in health-care decision-making. These principles, including fitness for purpose and reflecting individual expert uncertainty, were applied to the set of choices to establish a reference protocol. An applied evaluation of the developed reference protocol was also undertaken.

RESULTS

For many elements of structured expert elicitation, there was a lack of consistency across the existing guidelines. In almost all choices, there was a lack of empirical evidence supporting recommendations, and in some circumstances the principles are unable to provide sufficient justification for discounting particular choices. It is possible to define reference methods for health technology assessment. These include a focus on gathering experts with substantive skills, eliciting observable quantities and individual elicitation of beliefs. Additional considerations are required for decision-makers outside health technology assessment, for example at a local level, or for early technologies. Access to experts may be limited and in some circumstances group discussion may be needed to generate a distribution.

LIMITATIONS

The major limitation of the work conducted here lies not in the methods employed in the current work but in the evidence available from the wider literature relating to how appropriate particular methodological choices are.

CONCLUSIONS

The reference protocol is flexible in many choices. This may be a useful characteristic, as it is possible to apply this reference protocol across different settings. Further applied studies, which use the choices specified in this reference protocol, are required.

FUNDING

This project was funded by the NIHR Health Technology Assessment programme and will be published in full in ; Vol. 25, No. 37. See the NIHR Journals Library website for further project information. This work was also funded by the Medical Research Council (reference MR/N028511/1).

摘要

背景

许多医疗保健决策旨在最大化健康,需要对可能具有更高健康效果但可能产生额外成本的干预措施进行判断(成本效益框架)。用于确定成本效益的证据通常是不确定的,因此重要的是要描述这种不确定性。在证据不确定的情况下,专家的经验至关重要。以定量方式正式收集专家意见的过程是结构化专家评估。在医疗保健决策中使用的现有方法存在异质性。有许多指南可用于结构化专家评估;然而,尚不清楚这些指南是否适用于医疗保健决策。

目的

本研究的总体目标是制定用于医疗保健决策的结构化专家评估的方案。目标是(1)明确收集和使用专家判断的方法,(2)考虑在特定情况下是否需要替代方法,(3)为一系列参数的评估确定首选方法,(4)确定哪些评估方法允许专家表达不确定性,以及(5)确定所制定的参考方案的有用性。

方法

采用混合方法:系统评价、有针对性的搜索、实验工作和叙述性综合。对现有的结构化专家评估指南进行了综述。这确定了现有指南中使用的方法(“选择”),并确定是否存在主导方法。进行了有针对性的审查搜索,以选择专家、评估水平、拟合和聚合、评估判断的准确性以及启发式和偏见。为了筛选可用的选择,使用从有针对性的搜索中生成的证据、要评估的数量以及医疗保健决策中的约束来定义支撑在医疗保健决策中使用结构化专家评估的一套原则。这些原则,包括适用性和反映个人专家的不确定性,被应用于选择集,以建立参考方案。还对所制定的参考方案进行了应用评估。

结果

对于结构化专家评估的许多要素,现有的指南之间缺乏一致性。几乎在所有选择中,都缺乏支持建议的经验证据,在某些情况下,这些原则无法为排除特定选择提供充分的理由。为医疗技术评估定义参考方法是可能的。这些方法包括关注具有实质性技能的专家、评估可观察数量以及单独评估信念。对于医疗技术评估以外的决策者,例如在地方一级,或者对于早期技术,可能需要额外的考虑。专家的获取可能受到限制,在某些情况下,可能需要小组讨论来生成分布。

局限性

目前工作中的主要限制不在于所采用的方法,而在于与特定方法选择的适当性相关的更广泛文献中的可用证据。

结论

参考方案在许多选择中具有灵活性。这可能是一个有用的特征,因为可以在不同的环境中应用此参考方案。需要进一步的应用研究,使用本参考方案中指定的选择。

资金

该项目由英国国家卫生与保健优化研究所(NIHR)的健康技术评估计划资助,将全文发表在;第 25 卷,第 37 期。有关该项目的更多信息,请访问 NIHR 期刊图书馆网站。这项工作还得到了英国医学研究理事会(MR/N028511/1)的资助。

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