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Appl Health Econ Health Policy. 2019 Dec;17(6):771-780. doi: 10.1007/s40258-019-00513-3.
Cost-effectiveness analysis provides information on the potential value of new cancer treatments, which is particularly pertinent for decision makers as demand for treatment grows while healthcare budgets remain fixed. A range of decision-analytic modelling approaches can be used to estimate cost effectiveness. This study summarises the key modelling approaches considered in oncology, alongside their advantages and limitations. A review was conducted to identify single technology appraisals (STAs) submitted to the National Institute for Health and Care Excellence (NICE) and published papers reporting full economic evaluations of cancer treatments published within the last 5 years. The review was supplemented with the existing methods literature discussing cancer modelling. In total, 100 NICE STAs and 124 published studies were included. Partitioned-survival analysis (n = 54) and discrete-time state transition structures (n = 41) were the main structures submitted to NICE. Conversely, the published studies reported greater use of discrete-time state transition models (n = 102). Limited justification of model structure was provided by authors, despite an awareness in the existing literature that the model structure should be considered thoroughly and can greatly influence cost-effectiveness results. Justification for the choice of model structure was limited and studies would be improved with a thorough rationale for this choice. The strengths and weaknesses of each approach should be considered by future researchers. Alternative methods (such as multi-state modelling) are likely to be utilised more frequently in the future, and so justification of these more advanced methods is paramount to their acceptability to inform healthcare decision making.
成本效益分析提供了有关新癌症治疗方法潜在价值的信息,对于决策者来说尤为重要,因为治疗需求不断增长,而医疗保健预算却保持不变。可以使用一系列决策分析模型方法来估计成本效益。本研究总结了肿瘤学中考虑的主要模型方法,以及它们的优缺点。进行了一项综述,以确定提交给英国国家卫生与保健优化研究所(NICE)的单一技术评估(STA)和在过去 5 年内报告癌症治疗全经济评估的已发表论文。综述还补充了讨论癌症模型的现有方法文献。总共纳入了 100 项 NICE STA 和 124 项已发表的研究。分区生存分析(n=54)和离散时间状态转移结构(n=41)是提交给 NICE 的主要结构。相反,已发表的研究报告显示,离散时间状态转移模型的使用更为广泛(n=102)。尽管现有文献中意识到模型结构应进行彻底考虑,并且可能会极大地影响成本效益结果,但作者并未对模型结构提供充分的合理性证明。尽管如此,作者对模型结构的选择缺乏充分的论证,因此如果选择这种方法,研究将得到改进。未来的研究人员应考虑每种方法的优缺点。未来可能会更频繁地使用替代方法(如多状态建模),因此,对这些更先进方法的合理性证明对于将其用于医疗保健决策至关重要。