{"title":"调查抽样理论对构建有效临床试验的启示","authors":"Yves Tillé","doi":"10.1111/insr.12498","DOIUrl":null,"url":null,"abstract":"The organisation of a design of experiments, for example, for the realisation of a clinical trial, is crucial. It is often desirable to balance designs so that the means of the covariates are approximately the same in the test and control groups. In survey sampling theory, balanced sampling and calibration are two techniques that improve the precision of estimates. In this paper, we show the links between the two areas. We begin by assessing the gain in precision between a balanced design and a simple random sampling for the least squares estimators and the estimator by differences. We compare rerandomisation techniques and the cube method in order to balance the design. We propose a new method, particularly efficient, which combines the cube method with multivariate matching. A set of simulations is carried out in order to evaluate the different methods. The interest of the calibration is shown even if the design is almost balanced. It is thus shown that tools used by survey statisticians can be useful for experimental designs and clinical trials.","PeriodicalId":1,"journal":{"name":"Accounts of Chemical Research","volume":null,"pages":null},"PeriodicalIF":16.4000,"publicationDate":"2022-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"2","resultStr":"{\"title\":\"Some Solutions Inspired by Survey Sampling Theory to Build Effective Clinical Trials\",\"authors\":\"Yves Tillé\",\"doi\":\"10.1111/insr.12498\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"The organisation of a design of experiments, for example, for the realisation of a clinical trial, is crucial. It is often desirable to balance designs so that the means of the covariates are approximately the same in the test and control groups. In survey sampling theory, balanced sampling and calibration are two techniques that improve the precision of estimates. In this paper, we show the links between the two areas. We begin by assessing the gain in precision between a balanced design and a simple random sampling for the least squares estimators and the estimator by differences. We compare rerandomisation techniques and the cube method in order to balance the design. We propose a new method, particularly efficient, which combines the cube method with multivariate matching. A set of simulations is carried out in order to evaluate the different methods. The interest of the calibration is shown even if the design is almost balanced. It is thus shown that tools used by survey statisticians can be useful for experimental designs and clinical trials.\",\"PeriodicalId\":1,\"journal\":{\"name\":\"Accounts of Chemical Research\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":16.4000,\"publicationDate\":\"2022-04-10\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"2\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Accounts of Chemical Research\",\"FirstCategoryId\":\"100\",\"ListUrlMain\":\"https://doi.org/10.1111/insr.12498\",\"RegionNum\":1,\"RegionCategory\":\"化学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"CHEMISTRY, MULTIDISCIPLINARY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Accounts of Chemical Research","FirstCategoryId":"100","ListUrlMain":"https://doi.org/10.1111/insr.12498","RegionNum":1,"RegionCategory":"化学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CHEMISTRY, MULTIDISCIPLINARY","Score":null,"Total":0}
Some Solutions Inspired by Survey Sampling Theory to Build Effective Clinical Trials
The organisation of a design of experiments, for example, for the realisation of a clinical trial, is crucial. It is often desirable to balance designs so that the means of the covariates are approximately the same in the test and control groups. In survey sampling theory, balanced sampling and calibration are two techniques that improve the precision of estimates. In this paper, we show the links between the two areas. We begin by assessing the gain in precision between a balanced design and a simple random sampling for the least squares estimators and the estimator by differences. We compare rerandomisation techniques and the cube method in order to balance the design. We propose a new method, particularly efficient, which combines the cube method with multivariate matching. A set of simulations is carried out in order to evaluate the different methods. The interest of the calibration is shown even if the design is almost balanced. It is thus shown that tools used by survey statisticians can be useful for experimental designs and clinical trials.
期刊介绍:
Accounts of Chemical Research presents short, concise and critical articles offering easy-to-read overviews of basic research and applications in all areas of chemistry and biochemistry. These short reviews focus on research from the author’s own laboratory and are designed to teach the reader about a research project. In addition, Accounts of Chemical Research publishes commentaries that give an informed opinion on a current research problem. Special Issues online are devoted to a single topic of unusual activity and significance.
Accounts of Chemical Research replaces the traditional article abstract with an article "Conspectus." These entries synopsize the research affording the reader a closer look at the content and significance of an article. Through this provision of a more detailed description of the article contents, the Conspectus enhances the article's discoverability by search engines and the exposure for the research.