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评估成本效益和进一步研究价值的方法,当数据稀疏时:负压伤口疗法治疗严重压疮。

Methods to assess cost-effectiveness and value of further research when data are sparse: negative-pressure wound therapy for severe pressure ulcers.

机构信息

Centre for Health Economics, The University of York, York, UK (MOS, LB, KC)

Department of Health Sciences, The University of York, York, UK (JCD, RLA, CI, DT)

出版信息

Med Decis Making. 2013 Apr;33(3):415-36. doi: 10.1177/0272989X12451058. Epub 2012 Aug 27.

Abstract

Health care resources are scarce, and decisions have to be made about how to allocate funds. Often, these decisions are based on sparse or imperfect evidence. One such example is negative-pressure wound therapy (NPWT), which is a widely used treatment for severe pressure ulcers; however, there is currently no robust evidence that it is effective or cost-effective. This work considers the decision to adopt NPWT given a range of alternative treatments, using a decision analytic modeling approach. Literature searches were conducted to identify existing evidence on model parameters. Given the limited evidence base, a second source of evidence, beliefs elicited from experts, was used. Judgments from experts on relevant (uncertain) quantities were obtained through a formal elicitation exercise. Additionally, data derived from a pilot trial were also used to inform the model. The 3 sources of evidence were collated, and the impact of each on cost-effectiveness was evaluated. An analysis of the value of further information indicated that a randomized controlled trial may be worthwhile in reducing decision uncertainty, where from a set of alternative designs, a 3-arm trial with longer follow-up was estimated to be the most efficient. The analyses presented demonstrate how allocation decisions about medical technologies can be explicitly informed when data are sparse and how this kind of analyses can be used to guide future research prioritization, not only indicating whether further research is worthwhile but what type of research is needed and how it should be designed.

摘要

医疗资源稀缺,因此必须做出资金分配决策。这些决策通常基于稀疏或不完善的证据。负压伤口疗法(NPWT)就是一个例子,它是治疗严重压疮的常用方法;但目前没有强有力的证据表明它是有效或具有成本效益的。本研究考虑了在一系列替代疗法的情况下采用 NPWT 的决策,使用决策分析建模方法。进行了文献检索以确定模型参数的现有证据。鉴于证据基础有限,第二个证据来源是从专家那里得出的信念。通过正式的启发式研究获得了专家对相关(不确定)数量的判断。此外,还利用了一项试点试验得出的数据来为模型提供信息。整理了 3 个证据来源,并评估了每个来源对成本效益的影响。对进一步信息价值的分析表明,随机对照试验可能有助于降低决策不确定性,在一系列替代设计中,估计 3 臂试验具有更长的随访时间,是最有效的。所呈现的分析表明,在数据稀缺的情况下,如何明确告知有关医疗技术的分配决策,以及如何使用这种分析来指导未来的研究优先级,不仅表明进一步研究是否值得,还表明需要什么样的研究以及如何设计研究。

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