Health Economics Research Group, Brunel University, Uxbridge, UK.
Health Technol Assess. 2013 Dec;17(58):v-vi, 1-192. doi: 10.3310/hta17580.
National Institute for Health and Care Excellence (NICE) clinical guidelines (CGs) make recommendations across large, complex care pathways for broad groups of patients. They rely on cost-effectiveness evidence from the literature and from new analyses for selected high-priority topics. An alternative approach would be to build a model of the full care pathway and to use this as a platform to evaluate the cost-effectiveness of multiple topics across the guideline recommendations.
In this project we aimed to test the feasibility of building full guideline models for NICE guidelines and to assess if, and how, such models can be used as a basis for cost-effectiveness analysis (CEA).
A 'best evidence' approach was used to inform the model parameters. Data were drawn from the guideline documentation, advice from clinical experts and rapid literature reviews on selected topics. Where possible we relied on good-quality, recent UK systematic reviews and meta-analyses.
Two published NICE guidelines were used as case studies: prostate cancer and atrial fibrillation (AF). Discrete event simulation (DES) was used to model the recommended care pathways and to estimate consequent costs and outcomes. For each guideline, researchers not involved in model development collated a shortlist of topics suggested for updating. The modelling teams then attempted to evaluate options related to these topics. Cost-effectiveness results were compared with opinions about the importance of the topics elicited in a survey of stakeholders.
The modelling teams developed simulations of the guideline pathways and disease processes. Development took longer and required more analytical time than anticipated. Estimates of cost-effectiveness were produced for six of the nine prostate cancer topics considered, and for five of eight AF topics. The other topics were not evaluated owing to lack of data or time constraints. The modelled results suggested 'economic priorities' for an update that differed from priorities expressed in the stakeholder survey.
We did not conduct systematic reviews to inform the model parameters, and so the results might not reflect all current evidence. Data limitations and time constraints restricted the number of analyses that we could conduct. We were also unable to obtain feedback from guideline stakeholders about the usefulness of the models within project time scales.
Discrete event simulation can be used to model full guideline pathways for CEA, although this requires a substantial investment of clinical and analytic time and expertise. For some topics lack of data may limit the potential for modelling. There are also uncertainties over the accessibility and adaptability of full guideline models. However, full guideline modelling offers the potential to strengthen and extend the analytical basis of NICE's CGs. Further work is needed to extend the analysis of our case study models to estimate population-level budget and health impacts. The practical usefulness of our models to guideline developers and users should also be investigated, as should the feasibility and usefulness of whole guideline modelling alongside development of a new CG.
This project was funded by the Medical Research Council and the National Institute for Health Research through the Methodology Research Programme [grant number G0901504] and will be published in full in Health Technology Assessment; Vol. 17, No. 58. See the NIHR Journals Library website for further project information.
英国国家卫生与临床优化研究所(NICE)临床指南(CGs)针对广泛的患者群体,为复杂的大护理途径提供了建议。它们依赖于文献和针对选定高优先级主题的新分析的成本效益证据。另一种方法是构建完整的护理途径模型,并将其用作评估指南建议中多个主题的成本效益的平台。
在本项目中,我们旨在测试为 NICE 指南构建完整指南模型的可行性,并评估此类模型是否以及如何可以用作成本效益分析(CEA)的基础。
采用“最佳证据”方法为模型参数提供信息。数据来自指南文件、临床专家的建议以及选定主题的快速文献综述。在可能的情况下,我们依赖于高质量的、最近的英国系统评价和荟萃分析。
使用了两项已发表的 NICE 指南作为案例研究:前列腺癌和心房颤动(AF)。离散事件模拟(DES)用于模拟推荐的护理途径,并估计相应的成本和结果。对于每个指南,未参与模型开发的研究人员都会整理出一份建议更新的主题清单。然后,建模团队试图评估与这些主题相关的选项。成本效益结果与利益相关者调查中得出的对主题重要性的意见进行了比较。
建模团队开发了指南途径和疾病过程的模拟。开发工作比预期花费的时间更长,需要更多的分析时间。对前列腺癌考虑的九个主题中的六个进行了成本效益评估,对房颤考虑的八个主题中的五个进行了评估。由于缺乏数据或时间限制,其他主题没有进行评估。建模结果表明,更新的“经济优先事项”与利益相关者调查中表达的优先事项不同。
我们没有进行系统评价来为模型参数提供信息,因此结果可能无法反映所有当前证据。数据限制和时间限制限制了我们可以进行的分析数量。我们也无法在项目时间范围内从指南利益相关者那里获得有关模型实用性的反馈。
离散事件模拟可用于 CEA 对完整的指南途径进行建模,尽管这需要大量的临床和分析专业知识。对于某些主题,缺乏数据可能会限制建模的潜力。指南模型的可访问性和适应性也存在不确定性。然而,完整的指南建模有可能增强和扩展 NICE 指南的分析基础。需要进一步工作来扩展我们的案例研究模型的分析,以估计人群层面的预算和健康影响。还应研究我们的模型对指南制定者和用户的实际用途,以及在制定新 CG 时同时进行整个指南建模的可行性和实用性。
该项目由英国医学研究理事会和国家卫生研究院通过方法学研究计划[赠款号 G0901504]资助,并将在卫生技术评估中全文发表;第 17 卷,第 58 期。有关该项目的更多信息,请访问 NIHR 期刊库网站。