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引用次数: 0
摘要
随机试验的多阶段设计是为了在研究过程中发现试验组与对照组相比疗效较低或较高时,允许提前终止研究。在这种试验中,提前终止规则会导致治疗效果最大似然估计值出现偏差。我们考虑了关于二分结果(如治疗反应)的多阶段随机试验,并研究了几率比的估计。通常情况下,II 期随机癌症临床试验采用两阶段设计,样本量较小,这使得几率比的估计更具挑战性。本文评估了几种现有的几率比估计方法,并提出了适用于随机多阶段试验(包括随机 II 期癌症临床试验)的偏差校正估计器。通过数值研究表明,所提出的估计方法总体上具有较小的偏差和较小的均方误差。
Estimation of the odds ratio from multi-stage randomized trials.
A multi-stage design for a randomized trial is to allow early termination of the study when the experimental arm is found to have low or high efficacy compared to the control during the study. In such a trial, an early stopping rule results in bias in the maximum likelihood estimator of the treatment effect. We consider multi-stage randomized trials on a dichotomous outcome, such as treatment response, and investigate the estimation of the odds ratio. Typically, randomized phase II cancer clinical trials have two-stage designs with small sample sizes, which makes the estimation of odds ratio more challenging. In this paper, we evaluate several existing estimation methods of odds ratio and propose bias-corrected estimators for randomized multi-stage trials, including randomized phase II cancer clinical trials. Through numerical studies, the proposed estimators are shown to have a smaller bias and a smaller mean squared error overall.
期刊介绍:
Pharmaceutical Statistics is an industry-led initiative, tackling real problems in statistical applications. The Journal publishes papers that share experiences in the practical application of statistics within the pharmaceutical industry. It covers all aspects of pharmaceutical statistical applications from discovery, through pre-clinical development, clinical development, post-marketing surveillance, consumer health, production, epidemiology, and health economics.
The Journal is both international and multidisciplinary. It includes high quality practical papers, case studies and review papers.