{"title":"Elastic integrative analysis of randomised trial and real-world data for treatment heterogeneity estimation.","authors":"Shu Yang, Chenyin Gao, Donglin Zeng, Xiaofei Wang","doi":"10.1093/jrsssb/qkad017","DOIUrl":"10.1093/jrsssb/qkad017","url":null,"abstract":"<p><p>We propose a test-based elastic integrative analysis of the randomised trial and real-world data to estimate treatment effect heterogeneity with a vector of known effect modifiers. When the real-world data are not subject to bias, our approach combines the trial and real-world data for efficient estimation. Utilising the trial design, we construct a test to decide whether or not to use real-world data. We characterise the asymptotic distribution of the test-based estimator under local alternatives. We provide a data-adaptive procedure to select the test threshold that promises the smallest mean square error and an elastic confidence interval with a good finite-sample coverage property.</p>","PeriodicalId":49982,"journal":{"name":"Journal of the Royal Statistical Society Series B-Statistical Methodology","volume":"85 3","pages":"575-596"},"PeriodicalIF":5.8,"publicationDate":"2023-04-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10376438/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10035092","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Tyler J VanderWeele’s contribution to the Discussion of “Vintage Factor Analysis with Varimax Performs Statistical Inference” by Rohe & Zeng","authors":"","doi":"10.1093/jrsssb/qkad045","DOIUrl":"https://doi.org/10.1093/jrsssb/qkad045","url":null,"abstract":"","PeriodicalId":49982,"journal":{"name":"Journal of the Royal Statistical Society Series B-Statistical Methodology","volume":"7 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2023-04-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"76307049","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Seconder of the vote of thanks to Rohe & Zeng and contribution to the Discussion of “Vintage Factor Analysis with Varimax Performs Statistical Inference”","authors":"M. Pensky","doi":"10.1093/jrsssb/qkad031","DOIUrl":"https://doi.org/10.1093/jrsssb/qkad031","url":null,"abstract":"","PeriodicalId":49982,"journal":{"name":"Journal of the Royal Statistical Society Series B-Statistical Methodology","volume":"13 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2023-04-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"90038425","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Tao Wang’s contribution to the Discussion of “Vintage Factor Analysis with Varimax Performs Statistical Inference” by Rohe & Zeng","authors":"T. VanderWeele","doi":"10.1093/jrsssb/qkad046","DOIUrl":"https://doi.org/10.1093/jrsssb/qkad046","url":null,"abstract":"","PeriodicalId":49982,"journal":{"name":"Journal of the Royal Statistical Society Series B-Statistical Methodology","volume":"10 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2023-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"84755440","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Yang Liu’s contribution to the Discussion of “Vintage Factor Analysis with Varimax Performs Statistical Inference” by Rohe & Zeng","authors":"Y. Liu","doi":"10.1093/jrsssb/qkad042","DOIUrl":"https://doi.org/10.1093/jrsssb/qkad042","url":null,"abstract":"","PeriodicalId":49982,"journal":{"name":"Journal of the Royal Statistical Society Series B-Statistical Methodology","volume":"439 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2023-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"91081087","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Mark Pilling's contribution to the Discussion of “Vintage Factor Analysis with Varimax Performs Statistical Inference” by Rohe & Zeng","authors":"M. Pilling","doi":"10.1093/jrsssb/qkad044","DOIUrl":"https://doi.org/10.1093/jrsssb/qkad044","url":null,"abstract":"","PeriodicalId":49982,"journal":{"name":"Journal of the Royal Statistical Society Series B-Statistical Methodology","volume":"36 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2023-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"73894761","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Rungang Han and Anru R Zhang’s contribution to the Discussion of “Vintage Factor Analysis with Varimax Performs Statistical Inference” by Rohe & Zeng","authors":"Anru R. Zhang","doi":"10.1093/jrsssb/qkad034","DOIUrl":"https://doi.org/10.1093/jrsssb/qkad034","url":null,"abstract":"","PeriodicalId":49982,"journal":{"name":"Journal of the Royal Statistical Society Series B-Statistical Methodology","volume":"25 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2023-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"90079238","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Junhui Cai, Dan Yang, Linda Zhao and Wu Zhu’s contribution to the Discussion of “Vintage Factor Analysis with Varimax Performs Statistical Inference” by Rohe & Zeng","authors":"Junhui Cai, Dan Yang, Linda H. Zhao, Wu Zhu","doi":"10.1093/jrsssb/qkad038","DOIUrl":"https://doi.org/10.1093/jrsssb/qkad038","url":null,"abstract":"","PeriodicalId":49982,"journal":{"name":"Journal of the Royal Statistical Society Series B-Statistical Methodology","volume":"35 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2023-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"77102617","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Kaizheng Wang’s contribution to the Discussion of “Vintage Factor Analysis with Varimax Performs Statistical Inference” by Rohe & Zeng","authors":"Kaizheng Wang","doi":"10.1093/jrsssb/qkad033","DOIUrl":"https://doi.org/10.1093/jrsssb/qkad033","url":null,"abstract":"","PeriodicalId":49982,"journal":{"name":"Journal of the Royal Statistical Society Series B-Statistical Methodology","volume":"102 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2023-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"73516499","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Xiaoyue Niu’s contribution to the Discussion of “Vintage Factor Analysis with Varimax Performs Statistical Inference” by Rohe & Zeng","authors":"Xiaoyue Niu","doi":"10.1093/jrsssb/qkad043","DOIUrl":"https://doi.org/10.1093/jrsssb/qkad043","url":null,"abstract":"","PeriodicalId":49982,"journal":{"name":"Journal of the Royal Statistical Society Series B-Statistical Methodology","volume":"30 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2023-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"76994770","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}