Centre for Research on Health and Social Care Management, SDA Bocconi, Milan, Lombardia, Italy.
Evidence Synthesis & Modelling for Health Improvement, College of Medicine and Health, University of Exeter, Exeter, Devon, UK.
Health Econ. 2022 Sep;31 Suppl 1(Suppl 1):44-72. doi: 10.1002/hec.4524. Epub 2022 May 24.
In the drive toward faster patient access to treatments, health technology assessment (HTA) agencies and payers are increasingly faced with reliance on evidence based on surrogate endpoints, increasing decision uncertainty. Despite the development of a small number of evaluation frameworks, there remains no consensus on the detailed methodology for handling surrogate endpoints in HTA practice. This research overviews the methods and findings of four empirical studies undertaken as part of COMED (Pushing the Boundaries of Cost and Outcome Analysis of Medical Technologies) program work package 2 with the aim of analyzing international HTA practice of the handling and considerations around the use of surrogate endpoint evidence. We have synthesized the findings of these empirical studies, in context of wider contemporary body of methodological and policy-related literature on surrogate endpoints, to develop a web-based decision tool to support HTA agencies and payers when faced with surrogate endpoint evidence. Our decision tool is intended for use by HTA agencies and their decision-making committees together with the wider community of HTA stakeholders (including clinicians, patient groups, and healthcare manufacturers). Having developed this tool, we will monitor its use and we welcome feedback on its utility.
在努力加快患者获得治疗的速度的过程中，卫生技术评估 (HTA) 机构和支付方越来越依赖基于替代终点的证据，这增加了决策的不确定性。尽管已经制定了少数几个评估框架，但在 HTA 实践中处理替代终点的详细方法学上仍未达成共识。本研究综述了作为 COMED（推动医疗技术成本和结果分析的边界）计划工作包 2 的一部分进行的四项实证研究的方法和结果，旨在分析国际 HTA 实践中对替代终点证据的处理和考虑。我们综合了这些实证研究的结果，并参考了更广泛的关于替代终点的当代方法学和政策相关文献，开发了一个基于网络的决策工具，以支持 HTA 机构和支付方在面临替代终点证据时使用。我们的决策工具旨在供 HTA 机构及其决策委员会以及 HTA 利益相关者（包括临床医生、患者群体和医疗保健制造商）更广泛的社区使用。开发了这个工具后，我们将监测其使用情况，并欢迎对其效用的反馈。
