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Simulation-Free Bayesian Power and Sample Size Calculations for Bayes Factors in Single-Arm Phase II Trials with Binary Endpoints

二元终点单臂二期试验中贝叶斯因子功效与样本量的免模拟计算

Bayes factors provide a coherent Bayesian measure of evidence for competing hypotheses and have recently been used as the basis for single-arm phase II trial designs with binary endpoints. In contrast to classical power analyses based on test statistics and p-values, Bayes-factor based sample size calculations target high probabilities of obtaining compelling evidence either for a relevant treatment effect or for the null hypothesis, given pre-specified Bayes-factor…

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Practically significant differences between conditional distribution functions

条件分布函数之间具有实际意义的差异

In the framework of semiparametric distribution regression, we consider the problem of comparing the conditional distribution functions corresponding to two samples. In contrast to testing for exact equality, we are interested in the (null) hypothesis that the $L^2$ distance between the conditional distribution functions does not exceed a certain threshold in absolute value. The consideration of these hypotheses is motivated by the observation that in applications, it is…

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A nonparametric relative treatment effect for direct comparisons of censored paired survival outcomes

用于右删失配对生存结局直接比较的非参数相对治疗效应

A very classical problem in statistics is to test the stochastic superiority of one distribution to another. However, many existing approaches are developed for independent samples and, moreover, do not take censored data into account. We develop a new estimand-driven method to compare the effectiveness of two treatments in the context of right-censored survival data with matched pairs. With the help of competing risks techniques, the so-called relative treatment effect is…

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A non-parametric proportional risk model to assess a treatment effect in time-to-event data

评估事件发生时间数据中治疗效应的非参数比例风险模型

Time-to-event analysis often relies on prior parametric assumptions, or, if a non-parametric approach is chosen, Cox's model. This is inherently tied to the assumption of proportional hazards, with the analysis potentially invalidated if this assumption is not fulfilled. In addition, most interpretations focus on the hazard ratio, that is often misinterpreted as the relative risk. In this paper, we introduce an alternative to current methodology for assessing a treatment…

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