相关论文: Frequentist analysis of basket trials with one-sam…

Basket trials are increasingly used for the simultaneous evaluation of a new treatment in various patient subgroups under one overarching protocol. We propose a Bayesian approach to sample size determination in basket trials that permit…

Therapeutic advancements in oncology have shifted towards targeted therapy based on genomic aberrations. This necessitates innovative statistical approaches in clinical trials, particularly in master protocol studies. Basket trials, a type…

Precision medicine has led to a paradigm shift allowing the development of targeted drugs that are agnostic to the tumor location. In this context, basket trials aim to identify which tumor types - or baskets - would benefit from the…

Basket trials have emerged as a new class of efficient approaches in oncology to evaluate a new treatment in several patient subgroups simultaneously. In this paper, we extend the key ideas to disease areas outside of oncology, developing a…

Heterogeneity in efficacy is sometimes observed across baskets in basket trials. In this study, we propose a model-free clustering framework that groups baskets based on transition probabilities derived from the trajectories of treatment…

Research in oncology has changed the focus from histological properties of tumors in a specific organ to a specific genomic aberration potentially shared by multiple cancer types. This motivates the basket trial, which assesses the efficacy…

Basket designs are prospective clinical trials that are devised with the hypothesis that the presence of selected molecular features determine a patient's subsequent response to a particular "targeted" treatment strategy. Basket trials are…

Basket trials have gained increasing attention for their efficiency, as multiple patient subgroups are evaluated simultaneously. Conducted basket trials focus primarily on establishing the early efficacy of a treatment, yet continued…

Basket trials examine the efficacy of an intervention in multiple patient subgroups simultaneously. The division into subgroups, called baskets, is based on matching medical characteristics, which may result in small sample sizes within…

This study examines the application of Bayesian approach in the context of clinical trials, emphasizing their increasing importance in contemporary biomedical research. While conventional frequentist approach provides a foundational basis…

Basket trials can efficiently evaluate a single treatment across multiple diseases with a common shared target. Prior methods for randomized basket trials required baskets to have the same sample and effect sizes. To that end, we developed…

Basket trials test a single therapeutic treatment on several patient populations under one master protocol. A desirable adaptive design feature in these studies may be the incorporation of new baskets to an ongoing study. Limited basket…

We propose a general method to carry out a valid Bayesian analysis of a finite-dimensional `targeted' parameter in the presence of a finite-dimensional nuisance parameter. We apply our methods to causal inference based on estimating…

In basket trials a treatment is investigated in several subgroups. They are primarily used in oncology in early clinical phases as single-arm trials with a binary endpoint. For their analysis primarily Bayesian methods have been suggested,…

Heckman selection model is the most popular econometric model in analysis of data with sample selection. However, selection models with Normal errors cannot accommodate heavy tails in the error distribution. Recently, Marchenko and Genton…

Estimating treatment effects from observational data provides insights about causality guiding many real-world applications such as different clinical study designs, which are the formulations of trials, experiments, and observational…

In this paper, a Bayesian approach is developed for simultaneously comparing multiple experimental treatments with a common control treatment in an exploratory clinical trial. The sample size is set to ensure that, at the end of the study,…

The prediction interval has been increasingly used in meta-analyses as a useful measure for assessing the magnitude of treatment effect and between-studies heterogeneity. In calculations of the prediction interval, although the…

In many epidemiological contexts, disease occurrences and their rates are naturally modelled by counting processes and their intensities, allowing an analysis based on martingale methods. These methods lend themselves to extensions of…

The Mantel-Haenszel (MH) risk difference estimator, commonly used in randomized clinical trials for binary outcomes, calculates a weighted average of stratum-specific risk difference estimators. Traditionally, this method requires the…