{"title":"Prior effective sample size in phase II clinical trials with mixed binary and continuous responses","authors":"Meghna Bose, J. Angers, A. Biswas","doi":"10.1111/stan.12283","DOIUrl":null,"url":null,"abstract":"The problem of finding Effective Sample Size (ESS) in Phase II clinical trials where toxicity and efficacy are the two components of the treatment response vector is considered. In particular, one of the components is assumed to be binary and the other is assumed to be continuous. The case of binary safety and continuous efficacy is studied for different prior distributions under different set up. Theoretical expressions are obtained in various situations. The methods are evaluated and compared by simulation studies. The proposed method is then illustrated by using some real life data on a phase II vaccine trial for Covid‐19.","PeriodicalId":51178,"journal":{"name":"Statistica Neerlandica","volume":"30 1","pages":"233 - 248"},"PeriodicalIF":0.8000,"publicationDate":"2022-11-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Statistica Neerlandica","FirstCategoryId":"100","ListUrlMain":"https://doi.org/10.1111/stan.12283","RegionNum":3,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"STATISTICS & PROBABILITY","Score":null,"Total":0}
引用次数: 0
Abstract
The problem of finding Effective Sample Size (ESS) in Phase II clinical trials where toxicity and efficacy are the two components of the treatment response vector is considered. In particular, one of the components is assumed to be binary and the other is assumed to be continuous. The case of binary safety and continuous efficacy is studied for different prior distributions under different set up. Theoretical expressions are obtained in various situations. The methods are evaluated and compared by simulation studies. The proposed method is then illustrated by using some real life data on a phase II vaccine trial for Covid‐19.
期刊介绍：
Statistica Neerlandica has been the journal of the Netherlands Society for Statistics and Operations Research since 1946. It covers all areas of statistics, from theoretical to applied, with a special emphasis on mathematical statistics, statistics for the behavioural sciences and biostatistics. This wide scope is reflected by the expertise of the journal’s editors representing these areas. The diverse editorial board is committed to a fast and fair reviewing process, and will judge submissions on quality, correctness, relevance and originality. Statistica Neerlandica encourages transparency and reproducibility, and offers online resources to make data, code, simulation results and other additional materials publicly available.
京公网安备 11010802042870号
京ICP备2023020795号-1
分享
求助内容：
应助结果提醒方式：
扫码关注我们
