Use of surrogate endpoints in health technology assessment: a review of selected NICE technology appraisals in oncology.

IF 2.6 4区 医学 Q2 HEALTH CARE SCIENCES & SERVICES
Lorna Wheaton, Sylwia Bujkiewicz
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引用次数: 0

Abstract

Objectives: 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.

Methods: 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.

Results: 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.

Conclusions: 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.

替代终点在卫生技术评估中的应用:对选定的NICE肿瘤学技术评估的回顾
目的:替代终点,用于替代和预测最终临床结果,越来越多地被用于支持提交给卫生技术评估机构。代理端点使用的增加伴随着描述框架和统计方法以确保其稳健性验证的文献。本综述的目的是评估英格兰和威尔士国家健康与护理卓越研究所(NICE)最近在肿瘤学技术评估中如何使用替代终点。方法:本文识别了NICE在2022年2月至2023年5月期间发表的肿瘤学技术评价。提取关于替代终点的使用和验证的数据,包括目的、证据基础和使用的方法。结果:在47项肿瘤学技术评估中,18项(38%)使用了替代终点,37项替代终点正在讨论中。然而,支持替代关系有效性的证据在假定的替代关系中存在显著差异,其中11个提供随机对照试验证据,7个提供观察性研究证据,12个基于临床意见,7个没有提供使用替代终点的证据。结论:这篇综述支持了替代终点在英格兰和威尔士肿瘤技术评估中被频繁使用的说法,尽管替代终点的适当验证的统计方法和指南的可用性越来越高,但这篇综述强调了替代终点的使用和验证在不同的技术评估中可能会有所不同,这可能导致决策的不确定性。
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来源期刊
International Journal of Technology Assessment in Health Care
International Journal of Technology Assessment in Health Care 医学-公共卫生、环境卫生与职业卫生
CiteScore
4.40
自引率
15.60%
发文量
116
审稿时长
6-12 weeks
期刊介绍: International Journal of Technology Assessment in Health Care serves as a forum for the wide range of health policy makers and professionals interested in the economic, social, ethical, medical and public health implications of health technology. It covers the development, evaluation, diffusion and use of health technology, as well as its impact on the organization and management of health care systems and public health. In addition to general essays and research reports, regular columns on technology assessment reports and thematic sections are published.
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