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There has been an increasing use of master protocols in oncology clinical trials because of its efficiency and flexibility to accelerate cancer drug development. Depending on the study objective and design, a master protocol trial can be a…

Applications · Statistics 2023-11-13 Jie Chen , Xiaoyun , Li , Chengxing , Lu , Sammy Yuan , Godwin Yung , Jingjing Ye , Hong Tian , Jianchang Lin

Preferably in two- or three-arm randomized clinical trials, a few (2,3) correlated multiple primary endpoints are considered. In addition to the closed testing principle based on different global tests, two max(maxT) tests are compared with…

Applications · Statistics 2021-03-16 Ludwig A. Hothorn , Siegfried Kropf

Clinical trials are indispensable for medical research and the development of new treatments. However, clinical trials often involve thousands of participants and can span several years to complete, with a high probability of failure during…

Machine Learning · Computer Science 2024-07-02 Yue Wang , Tianfan Fu , Yinlong Xu , Zihan Ma , Hongxia Xu , Yingzhou Lu , Bang Du , Honghao Gao , Jian Wu

Data from both a randomized trial and an observational study are sometimes simultaneously available for evaluating the effect of an intervention. The randomized data typically allows for reliable estimation of average treatment effects but…

Methodology · Statistics 2021-12-01 David Cheng , Tianxi Cai

In a clinical trial, the random allocation aims to balance prognostic factors between arms, preventing true confounders. However, residual differences due to chance may introduce near-confounders. Adjusting on prognostic factors is…

Methodology · Statistics 2024-11-18 Joe de Keizer , Rémi Lenain , Raphaël Porcher , Sarah Zoha , Arthur Chatton , Yohann Foucher

Investigating the causal relationship between exposure and the time-to-event outcome is an important topic in biomedical research. Previous literature has discussed the potential issues of using the hazard ratio as a marginal causal effect…

Methodology · Statistics 2022-05-05 Zihan Lin , Ai Ni , Bo Lu

Estimating heterogeneous treatment effects is central to data-driven decision-making, yet industrial applications often face a fundamental tension between limited randomized controlled trial (RCT) budgets and abundant but biased…

We consider the design of a two-arm superiority cluster randomised controlled trial (RCT) with a continuous outcome. We detail Bayesian inference for the analysis of the trial using a linear mixed-effects model. The treatment is compared to…

Methodology · Statistics 2022-08-29 Kevin J Wilson

In modern randomized experiments, large-scale data collection increasingly yields rich baseline covariates and auxiliary information from multiple sources. Such information offers opportunities for more precise treatment effect estimation,…

Methodology · Statistics 2026-03-10 Wei Ma , Zeqi Wu , Zheng Zhang

We consider the challenges associated with causal inference in settings where data from a randomized trial is augmented with control data from an external source to improve efficiency in estimating the average treatment effect (ATE).…

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

Loss of power and clear description of treatment differences are key issues in designing and analyzing a clinical trial where non-proportional hazard is a possibility. A log-rank test may be very inefficient and interpretation of the hazard…

Applications · Statistics 2021-01-13 Satrajit Roychoudhury , Keaven M Anderson , Jiabu Ye , Pralay Mukhopadhyay

Indirect experiments provide a valuable framework for estimating treatment effects in situations where conducting randomized control trials (RCTs) is impractical or unethical. Unlike RCTs, indirect experiments estimate treatment effects by…

Machine Learning · Computer Science 2023-12-06 Yash Chandak , Shiv Shankar , Vasilis Syrgkanis , Emma Brunskill

The matched case-control design, up until recently mostly pertinent to epidemiological studies, is becoming customary in biomedical applications as well. For instance, in omics studies, it is quite common to compare cancer and healthy…

Methodology · Statistics 2024-02-05 Vera Djordjilović , Erica Ponzi , Therese Haugdahl Nøst , Magne Thoresen

Multi-arm designs provide an effective means of evaluating several treatments within the same clinical trial. Given the large number of treatments now available for testing in many disease areas, it has been argued that their utilisation…

Computation · Statistics 2019-06-24 Michael J Grayling , James MS Wason

Cluster-randomized trials (CRTs) are widely used to evaluate group-level interventions and increasingly collect multiple outcomes capturing complementary dimensions of benefit and risk. Investigators often seek a single global summary of…

Methodology · Statistics 2026-01-22 Xinyuan Chen , Fan Li

Matching patients to clinical trials demands a systematic and reasoned interpretation of documents which require significant expert-level background knowledge, over a complex set of well-defined eligibility criteria. Moreover, this…

Computation and Language · Computer Science 2024-10-01 Mael Jullien , Alex Bogatu , Harriet Unsworth , Andre Freitas

We consider how increasingly available observational data can be used to improve the design of randomized controlled trials (RCTs). We seek to design a prospective RCT, with the intent of using an Empirical Bayes estimator to shrink the…

Methodology · Statistics 2022-04-15 Evan T. R. Rosenman , Luke Miratrix

Supervised open-loop training has been widely adopted for training traffic simulation models; however, it fails to capture the inherently dynamic, multi-agent interactions common in complex driving scenarios. We introduce RLFTSim, a…

Training robots for operation in the real world is a complex, time consuming and potentially expensive task. Despite significant success of reinforcement learning in games and simulations, research in real robot applications has not been…

Artificial Intelligence · Computer Science 2017-09-28 Markus Wulfmeier , Ingmar Posner , Pieter Abbeel