Wheaton Lorna, Bujkiewicz Sylwia
Biostatistics Research Group, Department of Population Health Sciences, University of Leicester, Leicester, UK.
Int J Technol Assess Health Care. 2025 Feb 19;41(1):e11. doi: 10.1017/S0266462325000017.
Surrogate endpoints, used to substitute for and predict final clinical outcomes, are increasingly being used to support submissions to health technology assessment agencies. The increase in the use of surrogate endpoints has been accompanied by literature describing the frameworks and statistical methods to ensure their robust validation. The aim of this review was to assess how surrogate endpoints have recently been used in oncology technology appraisals by the National Institute for Health and Care Excellence (NICE) in England and Wales.
This article identifies technology appraisals in oncology published by NICE between February 2022 and May 2023. Data are extracted on the use and validation of surrogate endpoints including purpose, evidence base, and methods used.
Of the 47 technology appraisals in oncology available for review, 18 (38 percent) utilized surrogate endpoints, with 37 separate surrogate endpoints being discussed. However, the evidence supporting the validity of the surrogate relationship varied significantly across putative surrogate relationships with 11 providing randomized controlled trial evidence, 7 providing evidence from observational studies, 12 based on the clinical opinion, and 7 providing no evidence for the use of surrogate endpoints.
This review supports the assertion that surrogate endpoints are frequently used in oncology technology appraisals in England and Wales and despite the increasing availability of statistical methods and guidance on appropriate validation of surrogate endpoints, this review highlights that use and validation of surrogate endpoints can vary between technology appraisals, which can lead to uncertainty in decision making.
替代终点用于替代和预测最终临床结局，越来越多地被用于支持向卫生技术评估机构提交的申请。替代终点使用的增加伴随着描述确保其可靠验证的框架和统计方法的文献。本综述的目的是评估替代终点最近在英格兰和威尔士国家卫生与临床优化研究所（NICE）的肿瘤学技术评估中是如何使用的。
本文识别了NICE在2022年2月至2023年5月期间发布的肿瘤学技术评估。提取有关替代终点使用和验证的数据，包括目的、证据基础和使用的方法。
在可供审查的47项肿瘤学技术评估中，18项（38%）使用了替代终点，共讨论了37个不同的替代终点。然而，支持替代关系有效性的证据在不同的假定替代关系中差异很大，11项提供了随机对照试验证据，7项提供了观察性研究的证据，12项基于临床意见，7项没有提供使用替代终点的证据。
本综述支持以下观点，即替代终点在英格兰和威尔士的肿瘤学技术评估中经常被使用，尽管关于替代终点适当验证的统计方法和指南越来越多，但本综述强调替代终点的使用和验证在不同技术评估之间可能有所不同，这可能导致决策的不确定性。
