Estimands for overall survival in clinical trials with treatment switching in oncology

医学 临床试验 肿瘤科 指南 内科学 心理学 医学物理学 重症监护医学 病理
作者
Juliane Manitz,Natalia Kan‐Dobrosky,Hannes Buchner,Marie‐Laure Casadebaig,Evgeny Degtyarev,Jyotirmoy Dey,Vincent Haddad,Jie Fei,Emily Martin,Mindy Mo,Kaspar Rufibach,Yue Shentu,Viktoriya Stalbovskaya,Rui Tang,Godwin Yung,Jiangxiu Zhou
出处
期刊:Pharmaceutical Statistics [Wiley]
卷期号:21 (1): 150-162 被引量:13
标识
DOI:10.1002/pst.2158
摘要

An addendum of the ICH E9 guideline on Statistical Principles for Clinical Trials was released in November 2019 introducing the estimand framework. This new framework aims to align trial objectives and statistical analyses by requiring a precise definition of the inferential quantity of interest, that is, the estimand. This definition explicitly accounts for intercurrent events, such as switching to new anticancer therapies for the analysis of overall survival (OS), the gold standard in oncology. Traditionally, OS in confirmatory studies is analyzed using the intention-to-treat (ITT) approach comparing treatment groups as they were initially randomized regardless of whether treatment switching occurred and regardless of any subsequent therapy (treatment-policy strategy). Regulatory authorities and other stakeholders often consider ITT results as most relevant. However, the respective estimand only yields a clinically meaningful comparison of two treatment arms if subsequent therapies are already approved and reflect clinical practice. We illustrate different scenarios where subsequent therapies are not yet approved drugs and thus do not reflect clinical practice. In such situations the hypothetical strategy could be more meaningful from patient's and prescriber's perspective. The cross-industry Oncology Estimand Working Group (www.oncoestimand.org) was initiated to foster a common understanding and consistent implementation of the estimand framework in oncology clinical trials. This paper summarizes the group's recommendations for appropriate estimands in the presence of treatment switching, one of the key intercurrent events in oncology clinical trials. We also discuss how different choices of estimands may impact study design, data collection, trial conduct, analysis, and interpretation.
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