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受调查抽样理论启发构建有效临床试验的若干方法

Some Solutions Inspired by Survey Sampling Theory to Build Effective Clinical Trials

International Statistical Review · 2022
被引 3
ABS 3

中文导读

本文展示了调查抽样中的平衡抽样和校准技术如何用于临床试验设计,提出结合立方体法与多元匹配的新方法,并通过模拟评估其效果,对试验设计者有用。

Abstract

Summary 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.

临床试验设计调查抽样平衡设计校准匹配方法