Flinders Health and Medical Research Institute, Flinders University, Bedford Park, SA, Australia.
Centre for Health Economics, University of York, Heslington, York, UK.
Pharmacoeconomics. 2022 Jan;40(1):31-43. doi: 10.1007/s40273-021-01087-6. Epub 2021 Sep 29.
Many health technology assessment committees have an explicit or implicit reference value (often referred to as a 'threshold') below which new health technologies or interventions are considered value for money. The basis for these reference values is unclear but one argument is that it should be based on the health opportunity costs of funding decisions. Empirical estimates of the marginal cost per unit of health produced by a healthcare system have been proposed to capture the health opportunity costs of new funding decisions. Based on a systematic search, we identified eight studies that have sought to estimate a reference value through empirical estimation of the marginal cost per unit of health produced by a healthcare system for England, Spain, Australia, The Netherlands, Sweden, South Africa and China. We review these eight studies to provide an overview of the key methodological approaches taken to estimate the marginal cost per unit of health produced by the healthcare system with the aim to help inform future estimates for additional countries. The lead author for each of these papers was invited to contribute to the current paper to ensure all the key methodological issues encountered were appropriately captured. These included consideration of the key variables required and their measurement, accounting for endogeneity of spending to health outcomes, the inclusion of lagged spending, discounting and future costs, the use of analytical weights, level of disease aggregation, expected duration of health gains, and modelling approaches to estimating mortality and morbidity effects of health spending. Subsequent research estimates for additional countries should (1) carefully consider the specific context and data available, (2) clearly and transparently report the assumptions made and include stakeholder perspectives on their appropriateness and acceptability, and (3) assess the sensitivity of the preferred central estimate to these assumptions.
许多健康技术评估委员会都有明确或隐含的参考值（通常称为“阈值”），低于该值，新的健康技术或干预措施被认为具有成本效益。这些参考值的依据尚不清楚，但有一种观点认为，它应该基于为资金决策付出的健康机会成本。有人提出，应该用医疗体系每产生一个单位健康所产生的边际成本的经验估计值来捕捉新资金决策的健康机会成本。我们通过系统搜索，确定了八项试图通过对英国、西班牙、澳大利亚、荷兰、瑞典、南非和中国的医疗体系每产生一个单位健康所产生的边际成本进行经验估计来确定参考值的研究。我们对这八项研究进行了回顾，以便对医疗体系每产生一个单位健康所产生的边际成本的估计所采用的关键方法进行概述，目的是帮助为其他国家的未来估计提供信息。我们邀请了这些论文的主要作者为当前的论文做出贡献，以确保适当涵盖所有遇到的关键方法问题。这些问题包括考虑所需的关键变量及其测量方法、考虑支出对健康结果的内生性、包括滞后支出、折现和未来成本、使用分析权重、疾病聚集程度、健康收益的预期持续时间以及用于估计健康支出的死亡率和发病率影响的建模方法。随后，为其他国家进行的研究估计应该（1）仔细考虑具体的背景和可用数据，（2）清楚透明地报告所做的假设，并包括利益相关者对其适当性和可接受性的看法，以及（3）评估首选中心估计对这些假设的敏感性。
