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The standard paradigm for confirmatory clinical trials is to compare experimental treatments with a control, for example the standard of care or a placebo. However, it is not always the case that a suitable control exists. Efficient…

Methodology · Statistics 2024-10-29 Thomas Burnett , Thomas Jaki

Platform trials gained popularity during the last few years as they increase flexibility compared to multi-arm trials by allowing new experimental arms entering when the trial already started. Using a shared control group in platform trials…

A gradual takeover strategy is proposed, in which the dynamic driving privilege assignment in real-time and the driving privilege gradual handover are realized. Firstly, the driving privilege assignment based on the risk level is achieved.…

Systems and Control · Electrical Eng. & Systems 2020-11-13 Rui Liu , Xichan Zhu , Xuan Zhao , Jian Ma

In early phase drug development of combination therapy, the primary objective is to preliminarily assess whether there is additive activity from a novel agent when combined with an established monotherapy. Due to potential feasibility…

Methodology · Statistics 2025-02-24 Zhaohua Lu , John Toso , Girma Ayele , Philip He

Design of experiments has traditionally relied on the frequentist hypothesis testing framework where the optimal size of the experiment is specified as the minimum sample size that guarantees a required level of power. Sample size…

Methodology · Statistics 2025-08-07 Shirin Golchi , Luke Hagar

Cluster randomized trials (CRTs) are popular in public health and in the social sciences to evaluate a new treatment or policy where the new policy is randomly allocated to clusters of units rather than individual units. CRTs often feature…

Methodology · Statistics 2019-08-16 Hyunseung Kang , Luke Keele

Unbiased assessment of the predictivity of models learnt by supervised machine-learning methods requires knowledge of the learned function over a reserved test set (not used by the learning algorithm). The quality of the assessment depends,…

Statistics Theory · Mathematics 2022-07-11 Elias Fekhari , Bertrand Iooss , Joseph Muré , Luc Pronzato , Maria-João Rendas

The question of selecting the "best" amongst different choices is a common problem in statistics. In drug development, our motivating setting, the question becomes, for example: what is the dose that gives me a pre-specified risk of…

Statistics Theory · Mathematics 2018-03-15 Pavel Mozgunov , Thomas Jaki

Accurate estimation of treatment effects is essential for decision-making across various scientific fields. This task, however, becomes challenging in areas like social sciences and online marketplaces, where treating one experimental unit…

Machine Learning · Computer Science 2025-02-04 Mohsen Bayati , Yuwei Luo , William Overman , Sadegh Shirani , Ruoxuan Xiong

Hierarchical random effect models are used for different purposes in clinical research and other areas. In general, the main focus is on population parameters related to the expected treatment effects or group differences among all units of…

Applications · Statistics 2021-04-07 Maryna Prus , Norbert Benda , Rainer Schwabe

In most nonrandomized observational studies, differences between treatment groups may arise not only due to the treatment but also because of the effect of confounders. Therefore, causal inference regarding the treatment effect is not as…

Methodology · Statistics 2018-07-04 Debashis Ghosh

We study the effects of symmetry-breaking defects at continuous quantum transitions (CQTs), which may arise from localized external fields coupled to the order-parameter operator. The problem is addressed within renormalization-group (RG)…

Statistical Mechanics · Physics 2022-05-10 Alessio Franchi , Davide Rossini , Ettore Vicari

Clinical trials with time-to-event endpoints, such as overall survival (OS) or progression-free survival (PFS), are fundamental for evaluating new treatments, particularly in immuno-oncology. However, modern therapies, such as…

Methodology · Statistics 2025-09-10 James Salsbury , Jeremy Oakley , Steven Julious , Lisa Hampson

Several low-dimensional systems show a crossover from diffusive to ballistic heat transport when system size is decreased. Although there is some phenomenological understanding of this crossover phenomena in the coarse grained level, a…

Statistical Mechanics · Physics 2015-05-27 Debarshee Bagchi , P. K. Mohanty

A two-stage collaborative exam is one in which students first complete the exam individually, and then complete the same or similar exam in collaborative groups immediately afterward. To quantify the learning effect from the group component…

Physics Education · Physics 2015-01-14 Joss Ives

If an experimental treatment is experienced by both treated and control group units, tests of hypotheses about causal effects may be difficult to conceptualize let alone execute. In this paper, we show how counterfactual causal models may…

Methodology · Statistics 2012-08-03 Jake Bowers , Mark Fredrickson , Costas Panagopoulos

Response-adaptive designs allow the randomization probabilities to change during the course of a trial based on cumulated response data, so that a greater proportion of patients can be allocated to the better performing treatments. A major…

Methodology · Statistics 2020-06-03 David S. Robertson , James M. S. Wason

This study considers testing the specification of spillover effects in causal inference. We focus on experimental settings in which the treatment assignment mechanism is known to researchers. We develop a new randomization test utilizing a…

Methodology · Statistics 2023-12-27 Tadao Hoshino , Takahide Yanagi

We present a new experimental design procedure that divides a set of experimental units into two groups in order to minimize error in estimating an additive treatment effect. One concern is minimizing error at the experimental design stage…

Methodology · Statistics 2021-02-02 Abba M. Krieger , David Azriel , Adam Kapelner

We consider causal inference in dynamic settings where treatment is assigned by thresholding a state variable that can change over time. There is a large literature on regression-discontinuity methods building on the fact that, in the…

Methodology · Statistics 2026-05-25 Aditya Ghosh , Stefan Wager
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