{"title":"多地区临床试验中剂量反应相似性的检验。","authors":"Holger Dette, Lukas Koletzko, Frank Bretz","doi":"10.1002/sim.70255","DOIUrl":null,"url":null,"abstract":"<p><p>This article addresses the problem of determining whether the dose response relationships between subgroups and the full population in a multiregional trial are similar. Similarity is assessed in terms of the maximal deviation between the dose response curves. We consider a parametric framework and develop two powerful bootstrap tests: one for assessing the similarity between the dose response curves of a single subgroup and that of the full population, and another for comparing the dose response curves of multiple subgroups with that of the full population. We prove the validity of these tests, investigate their finite sample properties through a simulation study and illustrate the methodology with a case study.</p>","PeriodicalId":21879,"journal":{"name":"Statistics in Medicine","volume":"44 20-22","pages":"e70255"},"PeriodicalIF":1.8000,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12417559/pdf/","citationCount":"0","resultStr":"{\"title\":\"Testing for Similarity of Dose Response in Multiregional Clinical Trials.\",\"authors\":\"Holger Dette, Lukas Koletzko, Frank Bretz\",\"doi\":\"10.1002/sim.70255\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>This article addresses the problem of determining whether the dose response relationships between subgroups and the full population in a multiregional trial are similar. Similarity is assessed in terms of the maximal deviation between the dose response curves. We consider a parametric framework and develop two powerful bootstrap tests: one for assessing the similarity between the dose response curves of a single subgroup and that of the full population, and another for comparing the dose response curves of multiple subgroups with that of the full population. We prove the validity of these tests, investigate their finite sample properties through a simulation study and illustrate the methodology with a case study.</p>\",\"PeriodicalId\":21879,\"journal\":{\"name\":\"Statistics in Medicine\",\"volume\":\"44 20-22\",\"pages\":\"e70255\"},\"PeriodicalIF\":1.8000,\"publicationDate\":\"2025-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12417559/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Statistics in Medicine\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1002/sim.70255\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"MATHEMATICAL & COMPUTATIONAL BIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Statistics in Medicine","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/sim.70255","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"MATHEMATICAL & COMPUTATIONAL BIOLOGY","Score":null,"Total":0}
Testing for Similarity of Dose Response in Multiregional Clinical Trials.
This article addresses the problem of determining whether the dose response relationships between subgroups and the full population in a multiregional trial are similar. Similarity is assessed in terms of the maximal deviation between the dose response curves. We consider a parametric framework and develop two powerful bootstrap tests: one for assessing the similarity between the dose response curves of a single subgroup and that of the full population, and another for comparing the dose response curves of multiple subgroups with that of the full population. We prove the validity of these tests, investigate their finite sample properties through a simulation study and illustrate the methodology with a case study.
期刊介绍：
The journal aims to influence practice in medicine and its associated sciences through the publication of papers on statistical and other quantitative methods. Papers will explain new methods and demonstrate their application, preferably through a substantive, real, motivating example or a comprehensive evaluation based on an illustrative example. Alternatively, papers will report on case-studies where creative use or technical generalizations of established methodology is directed towards a substantive application. Reviews of, and tutorials on, general topics relevant to the application of statistics to medicine will also be published. The main criteria for publication are appropriateness of the statistical methods to a particular medical problem and clarity of exposition. Papers with primarily mathematical content will be excluded. The journal aims to enhance communication between statisticians, clinicians and medical researchers.
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