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跨越国界:肿瘤学中真实世界证据可转移性的实证证据需求

Crossing borders: the need for empirical evidence of real-world evidence transportability in oncology.

作者信息

Clunie-O'Connor Caitlin, Thuresson Per-Olof, Masters Elizabeth, Taylor Aliki, Rosenlund Mats, Mpofu Philani, Adamson Blythe

机构信息

Flatiron Health UK, London, UK.

Roche, Basel, Switzerland.

出版信息

J Comp Eff Res. 2025 Aug;14(8):e250053. doi: 10.57264/cer-2025-0053. Epub 2025 Jul 10.

DOI:10.57264/cer-2025-0053
PMID:40637151
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12308535/
Abstract

WHAT IS THIS ARTICLE ABOUT?: This article discusses the challenges of using non local real-world evidence (RWE) in health technology assessments (HTA) when local data are unavailable, insufficient, or inappropriate. HTA organizations often prefer data collected locally or regionally, but the lack of suitable data in many markets has increased interest in understanding data 'transportability' - whether data from one country or population can be used to predict outcomes in another. Established in 2024, the Flatiron Fostering Oncology RWE Use Cases and Methods (FORUM) research consortium is exploring when and how non-local RWE can be effectively applied, with initial work focused on lung cancer, breast cancer and multiple myeloma.

WHAT DOES THE EVIDENCE SUGGEST SO FAR?: Initial studies suggest RWE from the US could predict outcomes in other countries with proper adjustment for population and treatment differences. Recent research in advanced non-small cell lung cancer demonstrated that adjusted US data provided comparable survival to real observed outcomes in Canada and the UK. This limited evidence base indicates that non-local RWE can help inform decision-making when local data is unavailable.

WHAT STUDIES ARE NEEDED NEXT?: The FORUM consortium is expanding research to other cancer types and countries to better understand RWE transportability. Future studies will focus on comparing outcomes across diverse healthcare systems, identifying key variables for adjustment and developing guidelines for when and how non-local data can be used. These efforts aim to create a framework for the use of global RWE in oncology HTA decision-making.

摘要

本文讲了什么?:本文探讨了在当地数据不可用、不足或不适用时,在卫生技术评估(HTA)中使用非本地真实世界证据(RWE)所面临的挑战。HTA组织通常更喜欢本地或区域收集的数据,但许多市场缺乏合适的数据,这增加了人们对理解数据“可转移性”的兴趣——即来自一个国家或人群的数据是否可用于预测另一个国家或人群的结果。Flatiron促进肿瘤学RWE用例和方法(FORUM)研究联盟成立于2024年,正在探索何时以及如何有效地应用非本地RWE,初期工作集中在肺癌、乳腺癌和多发性骨髓瘤方面。

目前证据表明了什么?:初步研究表明,来自美国的RWE在对人群和治疗差异进行适当调整后,可以预测其他国家的结果。最近对晚期非小细胞肺癌的研究表明,经调整的美国数据与加拿大和英国实际观察到的生存率相当。这一有限的证据基础表明,当本地数据不可用时,非本地RWE可以帮助为决策提供信息。

接下来需要进行哪些研究?:FORUM联盟正在将研究扩展到其他癌症类型和国家,以更好地理解RWE的可转移性。未来的研究将集中在比较不同医疗保健系统的结果、确定调整的关键变量以及制定关于何时以及如何使用非本地数据的指南。这些努力旨在创建一个在肿瘤学HTA决策中使用全球RWE的框架。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5ae0/12308535/d97355948399/cer-14-250053-g2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5ae0/12308535/636b7489a0b4/cer-14-250053-g1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5ae0/12308535/d97355948399/cer-14-250053-g2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5ae0/12308535/636b7489a0b4/cer-14-250053-g1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5ae0/12308535/d97355948399/cer-14-250053-g2.jpg

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