Using an early outcome as the sole source of information of interim decisions regarding treatment effect on a long-term endpoint: The non-Gaussian case.

IF 1.3 4区 医学 Q4 PHARMACOLOGY & PHARMACY
Leandro Garcia Barrado, Tomasz Burzykowski
{"title":"Using an early outcome as the sole source of information of interim decisions regarding treatment effect on a long-term endpoint: The non-Gaussian case.","authors":"Leandro Garcia Barrado, Tomasz Burzykowski","doi":"10.1002/pst.2398","DOIUrl":null,"url":null,"abstract":"<p><p>In randomized clinical trials that use a long-term efficacy endpoint, the follow-up time necessary to observe the endpoint may be substantial. In such trials, an attractive option is to consider an interim analysis based solely on an early outcome that could be used to expedite the evaluation of treatment's efficacy. Garcia Barrado et al. (Pharm Stat. 2022; 21: 209-219) developed a methodology that allows introducing such an early interim analysis for the case when both the early outcome and the long-term endpoint are normally-distributed, continuous variables. We extend the methodology to any combination of the early-outcome and long-term-endpoint types. As an example, we consider the case of a binary outcome and a time-to-event endpoint. We further evaluate the potential gain in operating characteristics (power, expected trial duration, and expected sample size) of a trial with such an interim analysis in function of the properties of the early outcome as a surrogate for the long-term endpoint.</p>","PeriodicalId":19934,"journal":{"name":"Pharmaceutical Statistics","volume":null,"pages":null},"PeriodicalIF":1.3000,"publicationDate":"2024-06-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pharmaceutical Statistics","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/pst.2398","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
引用次数: 0

Abstract

In randomized clinical trials that use a long-term efficacy endpoint, the follow-up time necessary to observe the endpoint may be substantial. In such trials, an attractive option is to consider an interim analysis based solely on an early outcome that could be used to expedite the evaluation of treatment's efficacy. Garcia Barrado et al. (Pharm Stat. 2022; 21: 209-219) developed a methodology that allows introducing such an early interim analysis for the case when both the early outcome and the long-term endpoint are normally-distributed, continuous variables. We extend the methodology to any combination of the early-outcome and long-term-endpoint types. As an example, we consider the case of a binary outcome and a time-to-event endpoint. We further evaluate the potential gain in operating characteristics (power, expected trial duration, and expected sample size) of a trial with such an interim analysis in function of the properties of the early outcome as a surrogate for the long-term endpoint.

将早期结果作为临时决定对长期终点治疗效果的唯一信息来源:非高斯情况
在采用长期疗效终点的随机临床试验中,观察终点所需的随访时间可能会很长。在此类试验中,一个有吸引力的选择是考虑仅根据早期结果进行中期分析,以加快疗效评估。Garcia Barrado 等人(Pharm Stat. 2022; 21: 209-219)开发了一种方法,可以在早期结果和长期终点均为正态分布连续变量的情况下引入这种早期中期分析。我们将该方法扩展到早期结果和长期终点类型的任何组合。举例来说,我们考虑了二元结果和时间到事件终点的情况。我们将根据作为长期终点替代物的早期结果的特性,进一步评估采用这种中期分析的试验在运行特性(功率、预期试验持续时间和预期样本量)方面的潜在收益。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
Pharmaceutical Statistics
Pharmaceutical Statistics 医学-统计学与概率论
CiteScore
2.70
自引率
6.70%
发文量
90
审稿时长
6-12 weeks
期刊介绍: Pharmaceutical Statistics is an industry-led initiative, tackling real problems in statistical applications. The Journal publishes papers that share experiences in the practical application of statistics within the pharmaceutical industry. It covers all aspects of pharmaceutical statistical applications from discovery, through pre-clinical development, clinical development, post-marketing surveillance, consumer health, production, epidemiology, and health economics. The Journal is both international and multidisciplinary. It includes high quality practical papers, case studies and review papers.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信