Johannesen Kasper M, Claxton Karl, Sculpher Mark J, Wailoo Allan J
Division of Health Care Analysis, Department of Medical and Health Sciences, Linköping University, Linköping, Sweden.
Centre for Health Economics, University of York, York, UK.
Health Econ. 2018 Feb;27(2):e41-e54. doi: 10.1002/hec.3561. Epub 2017 Aug 22.
This paper presents a conceptual framework to analyse the design of the cost-effectiveness appraisal process of new healthcare technologies. The framework characterises the appraisal processes as a diagnostic test aimed at identifying cost-effective (true positive) and non-cost-effective (true negative) technologies. Using the framework, factors that influence the value of operating an appraisal process, in terms of net gain to population health, are identified. The framework is used to gain insight into current policy questions including (a) how rigorous the process should be, (b) who should have the burden of proof, and (c) how optimal design changes when allowing for appeals, price reductions, resubmissions, and re-evaluations. The paper demonstrates that there is no one optimal appraisal process and the process should be adapted over time and to the specific technology under assessment. Optimal design depends on country-specific features of (future) technologies, for example, effect, price, and size of the patient population, which might explain the difference in appraisal processes across countries. It is shown that burden of proof should be placed on the producers and that the impact of price reductions and patient access schemes on the producer's price setting should be considered when designing the appraisal process.
本文提出了一个概念框架，用于分析新型医疗技术成本效益评估过程的设计。该框架将评估过程描述为一种诊断测试，旨在识别具有成本效益的（真阳性）和不具有成本效益的（真阴性）技术。利用该框架，确定了就人群健康净收益而言影响开展评估过程价值的因素。该框架用于深入了解当前的政策问题，包括（a）该过程应有多严格，（b）应由谁承担举证责任，以及（c）在允许上诉、降价、重新提交和重新评估时，最佳设计如何变化。本文表明不存在单一的最佳评估过程，该过程应随着时间推移并根据所评估的特定技术进行调整。最佳设计取决于（未来）技术的特定国家特征，例如效果、价格和患者群体规模，这可能解释了各国评估过程的差异。结果表明，举证责任应落在生产商身上，并且在设计评估过程时应考虑降价和患者准入计划对生产商定价的影响。
