相关论文: Adding new experimental arms to randomised clinica…

The traditional model specification of stepped-wedge cluster-randomized trials assumes a homogeneous treatment effect across time while adjusting for fixed-time effects. However, when treatment effects vary over time, the constant effect…

Measuring disease progression in clinical trials for testing novel treatments for multifaceted diseases as Progressive Supranuclear Palsy (PSP), remains challenging. In this study we assess a range of statistical approaches to compare…

We propose a multiple cohort expansion (MUCE) approach as a design or analysis method for phase 1b multiple expansion cohort trials, which are novel first-in-human studies conducted following phase 1a dose escalation. The MUCE design is…

Testing whether a variable of interest affects the outcome is one of the most fundamental problem in statistics and is often the main scientific question of interest. To tackle this problem, the conditional randomization test (CRT) is…

Bayesian sample size calculations in clinical trials usually rely on complex Monte Carlo simulations in practice. Obtaining bounds on Bayesian notions of the false-positive rate and power often lack closed-form or approximate numerical…

Multi-arm bandits are gaining popularity as they enable real-world sequential decision-making across application areas, including clinical trials, recommender systems, and online decision-making. Consequently, there is an increased desire…

Covariate-adjusted randomization (CAR) can reduce the risk of covariate imbalance and, when accounted for in analysis, increase the power of a trial. Despite CAR advances, stratified randomization remains the most common CAR method. Matched…

The principle of allocating an equal number of patients to each arm in a randomized controlled trial remains widely believed to be optimal for maximising statistical power. However, this long-held belief only holds true if the treatment…

This paper studies inference on the average treatment effect in experiments in which treatment status is determined according to "matched pairs" and it is additionally desired to adjust for observed, baseline covariates to gain further…

After rejecting the null hypothesis in the analysis of variance, the next step is to make the pairwise comparisons to find out differences in means. The purpose of this paper is threefold. The foremost aim is to suggest expression for…

This paper presents the foundations of a computer oriented approach for preparing a list of random treatment assignments to be adopted in randomised controlled trials. Software is presented which can be applied in the earliest stage of…

In this paper,our main focus is to obtain an asymptotic bound on the family wise error rate (FWER) for Bonferroni-type procedure in the simultaneous hypotheses testing problem when the observations corresponding to individual hypothesis are…

In this paper we consider online multiple testing with familywise error rate (FWER) control, where the probability of committing at least one type I error shall remain under control while testing a possibly infinite sequence of hypotheses…

Understanding treatment effect heterogeneity has become increasingly important in many fields. In this paper we study distributions and quantiles of individual treatment effects to provide a more comprehensive and robust understanding of…

The majority of response-adaptive randomisation (RAR) designs in the literature rely on efficacy data to guide dynamic patient allocation. However, their applicability becomes limited in settings where efficacy outcomes, such as survival,…

The recently published ICH E9 addendum on estimands in clinical trials provides a framework for precisely defining the treatment effect that is to be estimated, but says little about estimation methods. Here we report analyses of a clinical…

In this paper, we consider the problem of simultaneously testing many two-sided hypotheses when rejections of null hypotheses are accompanied by claims of the direction of the alternative. The fundamental goal is to construct methods that…

A common concern in non-inferiority (NI) trials is that non adherence due, for example, to poor study conduct can make treatment arms artificially similar. Because intention to treat analyses can be anti-conservative in this situation, per…

While randomized trials may be the gold standard for evaluating the effectiveness of the treatment intervention, in some special circumstances, single-arm clinical trials utilizing external control may be considered. The causal treatment…

In randomized trials, repeated measures of the outcome are routinely collected. The mixed model for repeated measures (MMRM) leverages the information from these repeated outcome measures, and is often used for the primary analysis to…