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.
最长约 10秒,即可获得该文献文件

科研通智能强力驱动
Strongly Powered by AbleSci AI
更新
PDF的下载单位、IP信息已删除 (2025-6-4)

科研通是完全免费的文献互助平台,具备全网最快的应助速度,最高的求助完成率。 对每一个文献求助,科研通都将尽心尽力,给求助人一个满意的交代。
实时播报
刚刚
dream完成签到 ,获得积分10
2秒前
唐唐发布了新的文献求助10
2秒前
史克珍香完成签到 ,获得积分10
8秒前
晓风完成签到,获得积分10
11秒前
CR完成签到 ,获得积分10
12秒前
mammer应助超帅无色采纳,获得10
13秒前
科研通AI2S应助科研通管家采纳,获得10
13秒前
所所应助科研通管家采纳,获得10
14秒前
Owen应助科研通管家采纳,获得10
14秒前
14秒前
lilylwy完成签到 ,获得积分0
14秒前
li完成签到 ,获得积分10
14秒前
可爱的函函应助唐唐采纳,获得10
19秒前
小石头完成签到,获得积分10
21秒前
量子星尘发布了新的文献求助10
25秒前
xiaoxiaoxingchen完成签到 ,获得积分10
25秒前
laohu完成签到,获得积分10
26秒前
风格完成签到,获得积分10
26秒前
大橙子发布了新的文献求助150
28秒前
八点必起完成签到,获得积分10
29秒前
sduweiyu完成签到 ,获得积分10
30秒前
hyf完成签到 ,获得积分10
31秒前
aldehyde应助芊芊要发SCI采纳,获得10
32秒前
Twinkle完成签到,获得积分10
34秒前
Eureka完成签到,获得积分10
36秒前
40秒前
浮熙完成签到 ,获得积分10
47秒前
笔芯完成签到,获得积分10
50秒前
看文献完成签到,获得积分0
52秒前
爱与感谢完成签到 ,获得积分10
54秒前
华仔应助大橙子采纳,获得10
55秒前
小帅完成签到,获得积分10
55秒前
man完成签到 ,获得积分10
56秒前
biofresh完成签到,获得积分10
58秒前
平凡完成签到,获得积分10
1分钟前
1分钟前
哈利波特完成签到,获得积分10
1分钟前
菓小柒完成签到 ,获得积分10
1分钟前
basil完成签到,获得积分10
1分钟前
高分求助中
【提示信息,请勿应助】关于scihub 10000
Les Mantodea de Guyane: Insecta, Polyneoptera [The Mantids of French Guiana] 3000
徐淮辽南地区新元古代叠层石及生物地层 3000
The Mother of All Tableaux: Order, Equivalence, and Geometry in the Large-scale Structure of Optimality Theory 3000
Handbook of Industrial Diamonds.Vol2 1100
Global Eyelash Assessment scale (GEA) 1000
Picture Books with Same-sex Parented Families: Unintentional Censorship 550
热门求助领域 (近24小时)
化学 材料科学 医学 生物 工程类 有机化学 生物化学 物理 内科学 纳米技术 计算机科学 化学工程 复合材料 遗传学 基因 物理化学 催化作用 冶金 细胞生物学 免疫学
热门帖子
关注 科研通微信公众号,转发送积分 4038157
求助须知:如何正确求助?哪些是违规求助? 3575869
关于积分的说明 11373842
捐赠科研通 3305650
什么是DOI,文献DOI怎么找? 1819255
邀请新用户注册赠送积分活动 892655
科研通“疑难数据库(出版商)”最低求助积分说明 815022