Macquarie University, Macquarie University Centre for the Health Economy, Macquarie Park, NSW, Australia.
Macquarie Business School, Macquarie University, Macquarie Park, Australia.
Pharmacoeconomics. 2024 Nov;42(11):1287-1300. doi: 10.1007/s40273-024-01420-9. Epub 2024 Aug 24.
Published literature has levied criticism against the cost-minimisation analysis (CMA) approach to economic evaluation over the past two decades, with multiple papers declaring its 'death'. However, since introducing the requirements for economic evaluations as part of health technology (HTA) decision-making in 1992, the cost-minimisation analysis (CMA) approach has been widely used to inform recommendations about the public subsidy of medicines in Australia. This research aimed to highlight the breadth of use of CMA in Australia and assess the influence of preconditions for the approach on subsidy recommendations METHODS: Relevant information was extracted from Public Summary Documents of Pharmaceutical Benefits Advisory Committee (PBAC) meetings in Australia considering submissions for the subsidy of medicines that included a CMA and were assessed between July 2005 and December 2022. A generalised linear model was used to explore the relationship between whether medicines were recommended and variables that reflected the primary preconditions for using CMA set out in the published PBAC Methodology Guidelines. Other control variables were selected through the Bolasso Method. Subgroup analysis was undertaken which replicated this modelling process.
While the potential for inferior safety or efficacy reduced the likelihood of recommendation (p < 0.01), the effect sizes suggest that the requirements for CMA were not requisite for recommendation.
The Australian practice of CMA does not strictly align with the PBAC Methodology Guidelines and the theoretically appropriate application of CMA. However, within the confines of a deliberative HTA decision-making process that balances values and judgement with available evidence, this may be considered acceptable, particularly if stakeholders consider the current approach delivers sufficient clarity of process and enables patients to access medicines at an affordable cost.
在过去的二十年里,已有文献对成本最小化分析(CMA)方法在经济评价中的应用提出了批评,多份文献宣称其“已死”。然而,自 1992 年将经济评价的要求纳入健康技术(HTA)决策以来,成本最小化分析(CMA)方法已广泛用于为澳大利亚药品的公共补贴提供建议。本研究旨在强调 CMA 在澳大利亚的广泛应用,并评估该方法的前提条件对补贴建议的影响。
从澳大利亚药品福利咨询委员会(PBAC)会议的公共摘要文件中提取相关信息,这些文件考虑了药品补贴申请,其中包括 CMA,并在 2005 年 7 月至 2022 年 12 月之间进行了评估。使用广义线性模型来探索药品是否被推荐与反映 PBAC 方法学指南中规定的 CMA 主要前提条件的变量之间的关系。通过 Bolasso 方法选择了其他控制变量。进行了亚组分析,该分析复制了这一建模过程。
虽然安全性或疗效降低的可能性降低了推荐的可能性(p<0.01),但效应大小表明 CMA 的要求并非推荐的必要条件。
澳大利亚 CMA 的实践并不完全符合 PBAC 方法学指南和理论上适当的 CMA 应用。然而,在权衡价值观、判断和现有证据的审议性 HTA 决策过程中,这可能被认为是可以接受的,特别是如果利益相关者认为当前的方法提供了足够的过程透明度,并使患者能够以负担得起的成本获得药品。