Related papers: Bayesian sample size determination in basket trial…

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…

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…

This paper explores an approach to Bayesian sample size determination in clinical trials. The approach falls into the category of what is often called "proper Bayesian", in that it does not mix frequentist concepts with Bayesian ones. A…

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 paper develops Bayesian sample size formulae for experiments comparing two groups. We assume the experimental data will be analysed in the Bayesian framework, where pre-experimental information from multiple sources can be represented…

Recent substantial advances of molecular targeted oncology drug development is requiring new paradigms for early-phase clinical trial methodologies to enable us to evaluate efficacy of several subtypes simultaneously and efficiently. The…

Randomized controlled clinical trials provide the gold standard for evidence generation in relation to the efficacy of a new treatment in medical research. Relevant information from previous studies may be desirable to incorporate in the…

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,…

We propose an information borrowing strategy for the design and monitoring of phase II basket trials based on the local multisource exchangeability assumption between baskets (disease types). In our proposed local-MEM framework, information…

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…

Bayesian inference and the use of posterior or posterior predictive probabilities for decision making have become increasingly popular in clinical trials. The current practice in Bayesian clinical trials relies on a hybrid…

Power and sample size analysis comprises a critical component of clinical trial study design. There is an extensive collection of methods addressing this problem from diverse perspectives. The Bayesian paradigm, in particular, has attracted…

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…

Replication studies are essential for assessing the credibility of claims from original studies. A critical aspect of designing replication studies is determining their sample size; a too small sample size may lead to inconclusive studies…

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…

The development of a new diagnostic test ideally follows a sequence of stages which, amongst other aims, evaluate technical performance. This includes an analytical validity study, a diagnostic accuracy study and an interventional clinical…

Utilizing Bayesian methods in clinical trials has become increasingly popular, as they can incorporate historical data and expert opinions into the design and allow for smaller sample sizes to reduce costs while providing reliable and…

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…

For dynamic borrowing to leverage external data to augment the control arm of small RCTs, the key step is determining the amount of borrowing based on the similarity of the outcomes in the controls from the trial and the external data…

Targeted therapies on the basis of genomic aberrations analysis of the tumor have shown promising results in cancer prognosis and treatment. Regardless of tumor type, trials that match patients to targeted therapies for their particular…