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Multi-arm multi-stage trial designs can bring notable gains in efficiency to the drug development process. However, for normally distributed endpoints, the determination of a design typically depends on the assumption that the patient…

Methodology · Statistics 2017-10-11 Michael Grayling , James Wason , Adrian Mander

In clinical trials, there is potential to improve precision and reduce the required sample size by appropriately adjusting for baseline variables in the statistical analysis. This is called covariate adjustment. Despite recommendations by…

Methodology · Statistics 2022-06-20 Kelly Van Lancker , Joshua Betz , Michael Rosenblum

The traditional model specification of stepped-wedge cluster-randomized trials assumes a homogeneous treatment effect across time while adjusting for fixed-time effects. However, when treatment effects vary over time, the constant effect…

Interval designs are a class of phase I trial designs for which the decision of dose assignment is determined by comparing the observed toxicity rate at the current dose with a prespecified (toxicity tolerance) interval. If the observed…

Methodology · Statistics 2013-09-20 Suyu Liu , Ying Yuan

In various applications, the potential outcome of a unit may be influenced by the treatments received by other units, a phenomenon known as interference, as well as by prior treatments, referred to as carryover effects. These phenomena…

Methodology · Statistics 2025-01-27 Haoyang Yu , Wei Ma , Hanzhong Liu

Technological advancements in the field of mobile devices and wearable sensors have helped overcome obstacles in the delivery of care, making it possible to deliver behavioral treatments anytime and anywhere. Increasingly the delivery of…

Applications · Statistics 2017-11-13 Walter Dempsey , Peng Liao , Santosh Kumar , Susan A. Murphy

Suppose one wishes to estimate the effect of a binary treatment on a binary endpoint conditional on a post-randomization quantity in a counterfactual world in which all subjects received treatment. It is generally difficult to identify this…

Methodology · Statistics 2019-11-12 Alex Luedtke , Jiacheng Wu

In a sequential multiple-assignment randomized trial (SMART), a sequence of treatments is given to a patient over multiple stages. In each stage, randomization may be done to allocate patients to different treatment groups. Even though…

Methodology · Statistics 2024-01-09 Rik Ghosh , Bibhas Chakraborty , Inbal Nahum-Shani , Megan E. Patrick , Palash Ghosh

The e-value is gaining traction as a robust alternative to p-values and Bayes factors for quantifying statistical evidence. e-values are a promising method for adaptive clinical trials due to their anytime-validity: e-values ensure type I…

Methodology · Statistics 2026-05-28 Stef Baas , Judith ter Schure , Joost van Rosmalen

Triple difference designs have become increasingly popular in empirical economics. The advantage of a triple difference design is that, within a treatment group, it allows for another subgroup of the population -- potentially less impacted…

Econometrics · Economics 2025-06-04 Laura Caron

We present a comprehensive framework of modeling covariance in angular streaking experiments. Within the impulsive streaking regime, the displacement of electron momentum distribution (MD) provides a tight connection between the…

Data Analysis, Statistics and Probability · Physics 2024-12-23 Jun Wang , Zhaoheng Guo , Erik Isele , Philip H. Bucksbaum , Agostino Marinelli , James P. Cryan , Taran Driver

For biological experiments aiming at calibrating models with unknown parameters, a good experimental design is crucial, especially for those subject to various constraints, such as financial limitations, time consumption and physical…

Applications · Statistics 2014-07-22 Xiao Lin , Gabriel Terejanu

The efficiency of an experimental design is ultimately measured in terms of time and resources needed for the experiment. Optimal sequential (multi-stage) design is studied in the situation where each stage involves a fixed cost. The…

Statistics Theory · Mathematics 2012-11-08 Juha Karvanen

Time-series experiments, also called switchback experiments or N-of-1 trials, play increasingly important roles in modern applications in medical and industrial areas. Under the potential outcomes framework, recent research has studied…

Methodology · Statistics 2025-10-28 Zhexiao Lin , Peng Ding

A single neuron is categorized as"multisensory" if there is a statistically significant difference between the response evoked by an audio-visual stimulus combination and that evoked by the most effective of its components individually.…

Neurons and Cognition · Quantitative Biology 2016-08-09 Hans Colonius , Adele Diederich

Randomized experiments are the gold standard for estimating the causal effects of an intervention. In the simplest setting, each experimental unit is randomly assigned to receive treatment or control, and then the outcomes in each treatment…

Methodology · Statistics 2020-06-05 Guillaume Basse , Yi Ding , Panos Toulis

In this paper, a methodology is proposed that enables to analyze the sensitivity of the outcome of a therapy to unavoidable high dispersion of the patient specific parameters on one hand and to the choice of the parameters that define the…

Systems and Control · Electrical Eng. & Systems 2022-05-17 Mazen Alamir

User-randomized A/B testing has emerged as the gold standard for online experimentation. However, when this kind of approach is not feasible due to legal, ethical or practical considerations, experimenters have to consider alternatives like…

Methodology · Statistics 2025-06-17 Paul Missault , Lorenzo Masoero , Christian Delbé , Thomas Richardson , Guido Imbens

We present a multivariate one-sided sensitivity analysis for matched observational studies, appropriate when the researcher has specified that a given causal mechanism should manifest itself in effects on multiple outcome variables in a…

Methodology · Statistics 2021-12-03 Peter L. Cohen , Matt A. Olson , Colin B. Fogarty

We consider the problem of constructing optimal designs for population pharmacokinetics which use random effect models. It is common practice in the design of experiments in such studies to assume uncorrelated errors for each subject. In…

Applications · Statistics 2010-11-16 Holger Dette , Andrey Pepelyshev , Tim Holland-Letz