{"title":"具有成对零聚类先验的多臂贝叶斯有序结果效用序贯试验","authors":"A. Chapple, Yussef Bennani, Meredith Clement","doi":"10.1214/22-ba1316","DOIUrl":null,"url":null,"abstract":". A multi-armed trial based on ordinal outcomes is proposed that lever-ages a flexible non-proportional odds cumulative logit model and numerical utility scores for each outcome to determine treatment optimality. This trial design uses a Bayesian clustering prior on the treatment effects that encourages the pairwise null hypothesis of no differences between treatments. A group sequential design is proposed to determine which treatments are clinically different with an adaptive decision boundary that becomes more aggressive as the sample size or clinical significance grows, or the number of active treatments decreases. A simulation study is conducted for 3 and 5 treatment arms, which shows that the design has superior operating characteristics (family wise error rate, generalized power, average sample size) compared to utility designs that do not allow clustering, a frequentist proportional odds model, or a permutation test based on empirical mean utilities.","PeriodicalId":55398,"journal":{"name":"Bayesian Analysis","volume":null,"pages":null},"PeriodicalIF":4.9000,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"A Multi-Armed Bayesian Ordinal Outcome Utility-Based Sequential Trial with a Pairwise Null Clustering Prior\",\"authors\":\"A. Chapple, Yussef Bennani, Meredith Clement\",\"doi\":\"10.1214/22-ba1316\",\"DOIUrl\":null,\"url\":null,\"abstract\":\". A multi-armed trial based on ordinal outcomes is proposed that lever-ages a flexible non-proportional odds cumulative logit model and numerical utility scores for each outcome to determine treatment optimality. This trial design uses a Bayesian clustering prior on the treatment effects that encourages the pairwise null hypothesis of no differences between treatments. A group sequential design is proposed to determine which treatments are clinically different with an adaptive decision boundary that becomes more aggressive as the sample size or clinical significance grows, or the number of active treatments decreases. A simulation study is conducted for 3 and 5 treatment arms, which shows that the design has superior operating characteristics (family wise error rate, generalized power, average sample size) compared to utility designs that do not allow clustering, a frequentist proportional odds model, or a permutation test based on empirical mean utilities.\",\"PeriodicalId\":55398,\"journal\":{\"name\":\"Bayesian Analysis\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":4.9000,\"publicationDate\":\"2022-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Bayesian Analysis\",\"FirstCategoryId\":\"100\",\"ListUrlMain\":\"https://doi.org/10.1214/22-ba1316\",\"RegionNum\":2,\"RegionCategory\":\"数学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"MATHEMATICS, INTERDISCIPLINARY APPLICATIONS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Bayesian Analysis","FirstCategoryId":"100","ListUrlMain":"https://doi.org/10.1214/22-ba1316","RegionNum":2,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"MATHEMATICS, INTERDISCIPLINARY APPLICATIONS","Score":null,"Total":0}
A Multi-Armed Bayesian Ordinal Outcome Utility-Based Sequential Trial with a Pairwise Null Clustering Prior
. A multi-armed trial based on ordinal outcomes is proposed that lever-ages a flexible non-proportional odds cumulative logit model and numerical utility scores for each outcome to determine treatment optimality. This trial design uses a Bayesian clustering prior on the treatment effects that encourages the pairwise null hypothesis of no differences between treatments. A group sequential design is proposed to determine which treatments are clinically different with an adaptive decision boundary that becomes more aggressive as the sample size or clinical significance grows, or the number of active treatments decreases. A simulation study is conducted for 3 and 5 treatment arms, which shows that the design has superior operating characteristics (family wise error rate, generalized power, average sample size) compared to utility designs that do not allow clustering, a frequentist proportional odds model, or a permutation test based on empirical mean utilities.
期刊介绍:
Bayesian Analysis is an electronic journal of the International Society for Bayesian Analysis. It seeks to publish a wide range of articles that demonstrate or discuss Bayesian methods in some theoretical or applied context. The journal welcomes submissions involving presentation of new computational and statistical methods; critical reviews and discussions of existing approaches; historical perspectives; description of important scientific or policy application areas; case studies; and methods for experimental design, data collection, data sharing, or data mining.
Evaluation of submissions is based on importance of content and effectiveness of communication. Discussion papers are typically chosen by the Editor in Chief, or suggested by an Editor, among the regular submissions. In addition, the Journal encourages individual authors to submit manuscripts for consideration as discussion papers.