相关论文: Statistical Design and Planning of an Adaptive Tri…

Composite endpoints are widely used in cardiovascular clinical trials. In recent years, hierarchical composite endpoints-particularly the win ratio approach and its predecessor, the Finkelstein-Schoenfeld (FS) test, also known as the…

Win statistics have gained increasing popularity as primary analysis methods for clinical trials with hierarchical endpoints (HEs) as primary endpoints. However, existing sample size and power calculation approaches in trial design still…

The Win Ratio has gained significant traction in cardiovascular trials as a novel method for analyzing composite endpoints (Pocock and others, 2012). Compared with conventional approaches based on time to the first event, the Win Ratio…

Adaptive sample size re-estimation (SSR) is a well-established strategy for improving the efficiency and flexibility of clinical trials. Its central challenge is determining whether, and by how much, to increase the sample size at an…

For randomized clinical trials where a single, primary, binary endpoint would require unfeasibly large sample sizes, composite endpoints are widely chosen as the primary endpoint. Despite being commonly used, composite endpoints entail…

Composite endpoints are increasingly used in clinical trials to capture treatment effects across multiple or hierarchically ordered outcomes. Although inference procedures based on win statistics, such as the win ratio, win odds, and net…

Adaptive subgroup enrichment design is an efficient design framework that allows accelerated development for investigational treatments while also having flexibility in population selection within the course of the trial. The adaptive…

Composite endpoints consisting of both terminal and non-terminal events, such as death and hospitalization, are frequently used in cardiovascular clinical trials. The Finkelstein-Schoenfeld (FS) test provides a way to employ a hierarchical…

Composite endpoints are widely used in cardiovascular clinical trials to improve statistical efficiency while preserving clinical relevance. The Win Ratio (WR) measure and more general frameworks of Win Statistics have emerged as…

Composite endpoints are widely used as primary endpoints in clinical trials. Designing trials with time-to-event endpoints can be particularly challenging because the proportional hazard assumption usually does not hold when using a…

Adaptive sample size re-estimation, early stopping, and trial re-design at interim analyses can reduce expected sample sizes in randomised trials. Cluster randomised trials, in which groups of participants are randomly allocated to…

Adaptive designs have been proposed for clinical trials in which the nuisance parameters or alternative of interest are unknown or likely to be misspecified before the trial. Whereas most previous works on adaptive designs and mid-course…

The win ratio offers a flexible approach to incorporate the hierarchy of clinical outcomes into the analysis of a composite endpoint, enabling simultaneous consideration of multiple outcome types, unlike traditional time-to-first-event…

2-in-1 design (Chen et al. 2018) is becoming popular in oncology drug development, with the flexibility of using different endpoints at different decision time. Based on the observed interim data, sponsors choose either to seamlessly…

In clinical trials, there is potential to improve precision and reduce the required sample size by appropriately adjusting for baseline variables in the statistical analysis. This is called covariate adjustment. Despite recommendations by…

A new approach to adaptive design of clinical trials is proposed in a general multiparameter exponential family setting, based on generalized likelihood ratio statistics and optimal sequential testing theory. These designs are easy to…

Conventional methods for analyzing composite endpoints in clinical trials often only focus on the time to the first occurrence of all events in the composite. Therefore, they have inherent limitations because the individual patients' first…

Summary points: - This article considers the combination of two binary or two time-to-event endpoints to form the primary composite endpoint for leading a trial. - It discusses the relative efficiency of choosing a composite endpoint over…

The development of targeted therapies, which benefit only a subgroup of patients treated for a given type of cancer, has been extremely attractive to many investigators. Adaptive seamless phase II/III designs in oncology clinical trials…

In a randomised clinical trial, when the result of the primary endpoint shows a significant benefit, the secondary endpoints are scrutinised to identify additional effects of the treatment. However, this approach entails a risk of…