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In adaptive clinical trials, the conventional end-of-trial point estimate of a treatment effect is prone to bias, that is, a systematic tendency to deviate from its true value. As stated in recent FDA guidance on adaptive designs, it is…

Adaptive designs for clinical trials permit alterations to a study in response to accumulating data in order to make trials more flexible, ethical and efficient. These benefits are achieved while preserving the integrity and validity of the…

Bayesian adaptive clinical trials offer a flexible and efficient alternative to traditional fixed-design trials, but their implementation is often hindered by the complexity of Bayesian computations and the need for advanced statistical…

Applications · Statistics 2025-07-04 Krishna Padmanabhan , Danny Baker

Recent FDA guidance on adaptive clinical trial designs defines bias as "a systematic tendency for the estimate of treatment effect to deviate from its true value", and states that it is desirable to obtain and report estimates of treatment…

Randomized Controlled Trials (RCTs) are the gold standard for comparing the effectiveness of a new treatment to the current one (the control). Most RCTs allocate the patients to the treatment group and the control group by uniform…

Machine Learning · Statistics 2018-10-22 Onur Atan , William R. Zame , Mihaela van der Schaar

Test-time adaptation (TTA) adapts the pre-trained models to test distributions during the inference phase exclusively employing unlabeled test data streams, which holds great value for the deployment of models in real-world applications.…

Computer Vision and Pattern Recognition · Computer Science 2023-10-10 Shuang Li , Longhui Yuan , Binhui Xie , Tao Yang

Clinical trials are an instrument for making informed decisions based on evidence from well-designed experiments. Here we consider adaptive designs mainly from the perspective of multi-arm Phase II clinical trials, in which one or more…

Methodology · Statistics 2021-08-31 Elja Arjas , Dario Gasbarra

Adaptive therapy is a dynamic cancer treatment protocol that updates (or "adapts") treatment decisions in anticipation of evolving tumor dynamics. This broad term encompasses many possible dynamic treatment protocols of patient-specific…

Clinical trials are an integral component of medical research. Trials require careful design to, for example, maintain the safety of participants, use resources efficiently and allow clinically meaningful conclusions to be drawn. Adaptive…

Methodology · Statistics 2025-07-17 James M. McGree , Antony M. Overstall , Mark Jones , Robert K. Mahar

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…

Statistics Theory · Mathematics 2011-05-25 Jay Bartroff , Tze Leung Lai

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

Methodology · Statistics 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

The main challenge in domain generalization (DG) is to handle the distribution shift problem that lies between the training and test data. Recent studies suggest that test-time training (TTT), which adapts the learned model with test data,…

Computer Vision and Pattern Recognition · Computer Science 2023-04-18 Liang Chen , Yong Zhang , Yibing Song , Ying Shan , Lingqiao Liu

Clinical trial adaptation refers to any adjustment of the trial protocol after the onset of the trial. The main goal is to make the process of introducing new medical interventions to patients more efficient by reducing the cost and the…

Machine Learning · Computer Science 2014-11-17 Ognjen Arandjelovic

Evidence syntheses and meta-analyses are used to inform clinical practice guidelines and health economic evaluations. However, heterogeneity of treatment effects poses a significant challenge. Conventional meta-analysis addresses…

Continual Test-Time Adaptation (CTTA) is an emerging and challenging task where a model trained in a source domain must adapt to continuously changing conditions during testing, without access to the original source data. CTTA is prone to…

Machine Learning · Computer Science 2024-05-29 Ziqi Shi , Fan Lyu , Ye Liu , Fanhua Shang , Fuyuan Hu , Wei Feng , Zhang Zhang , Liang Wang

The most common objective for response adaptive clinical trials is to seek to ensure that patients within a trial have a high chance of receiving the best treatment available by altering the chance of allocation on the basis of accumulating…

Methodology · Statistics 2020-11-09 Helen Yvette Barnett , Sofia S Villar , Helena Geys , Thomas Jaki

Test-time adaptation (TTA) addresses distribution shifts for streaming test data in unsupervised settings. Currently, most TTA methods can only deal with minor shifts and rely heavily on heuristic and empirical studies. To advance TTA under…

Machine Learning · Computer Science 2024-04-09 Shurui Gui , Xiner Li , Shuiwang Ji

There has been a split in the statistics community about the need for taking covariates into account in the design phase of a clinical trial. There are many advocates of using stratification and covariate-adaptive randomization to promote…

Methodology · Statistics 2011-02-21 William F. Rosenberger , Oleksandr Sverdlov

When individuals participating in a randomized trial differ with respect to the distribution of effect modifiers compared compared with the target population where the trial results will be used, treatment effect estimates from the trial…

Response-adaptive clinical trial designs allow targeting a given objective by skewing the allocation of participants to treatments based on observed outcomes. Response-adaptive designs face greater regulatory scrutiny due to potential type…

Methodology · Statistics 2025-03-19 Stef Baas , Peter Jacko , Sofía S. Villar
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