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Clinical trials are typically run in order to understand the effects of a new treatment on a given population of patients. However, patients in large populations rarely respond the same way to the same treatment. This heterogeneity in…

Machine Learning · Statistics 2024-02-12 Alihan Hüyük , Zhaozhi Qian , Mihaela van der Schaar

This paper deals exclusively with crossover designs for the purpose of comparing t test treatments with a control treatment when the number of periods is no larger than t+1. Among other results it specifies sufficient conditions for a…

Statistics Theory · Mathematics 2007-06-13 A. S. Hedayat , Min Yang

Applied researchers are increasingly interested in whether and how treatment effects vary in randomized evaluations, especially variation not explained by observed covariates. We propose a model-free approach for testing for the presence of…

Methodology · Statistics 2014-12-17 Peng Ding , Avi Feller , Luke Miratrix

Randomized saturation designs are a family of designs which assign a possibly different treatment proportion to each cluster of a population at random. As a result, they generalize the well-known (stratified) completely randomized designs…

Methodology · Statistics 2022-03-21 Chencheng Cai , Jean Pouget-Abadie , Edoardo M. Airoldi

Completely randomized experiments have been the gold standard for drawing causal inference because they can balance all potential confounding on average. However, they may suffer from unbalanced covariates for realized treatment…

Statistics Theory · Mathematics 2022-10-18 Yuhao Wang , Xinran Li

We study the temporal robustness of temporal logic specifications and show how to design temporally robust control laws for time-critical control systems. This topic is of particular interest in connected systems and interleaving processes…

Systems and Control · Electrical Eng. & Systems 2022-07-26 Alëna Rodionova , Lars Lindemann , Manfred Morari , George J. Pappas

Pragmatic randomized trials are designed to provide evidence for clinical decision-making rather than regulatory approval. Common features of these trials include the inclusion of heterogeneous or diverse patient populations in a wide range…

Methodology · Statistics 2019-11-20 Eleanor J. Murray , Sonja A. Swanson , Miguel A. Hernán

We study causal inference in randomized experiments (or quasi-experiments) following a $2\times 2$ factorial design. There are two treatments, denoted $A$ and $B$, and units are randomly assigned to one of four categories: treatment $A$…

Econometrics · Economics 2024-12-12 Mate Kormos , Robert P. Lieli , Martin Huber

Individualizing treatment assignment can improve outcomes for diseases with patient-to-patient variability in comparative treatment effects. When a clinical trial demonstrates that some patients improve on treatment while others do not, it…

Methodology · Statistics 2022-11-02 Nina Galanter , Marco Carone , Ronald C. Kessler , Alex Luedtke

Randomized experiments often need to be stopped prematurely due to the treatment having an unintended harmful effect. Existing methods that determine when to stop an experiment early are typically applied to the data in aggregate and do not…

Methodology · Statistics 2023-10-31 Hammaad Adam , Fan Yin , Huibin , Hu , Neil Tenenholtz , Lorin Crawford , Lester Mackey , Allison Koenecke

This paper studies the validity of nonparametric tests used in the regression discontinuity design. The null hypothesis of interest is that the average treatment effect at the threshold in the so-called sharp design equals a pre-specified…

Methodology · Statistics 2016-11-16 Vishal Kamat

We examine study designs for extending (generalizing or transporting) causal inferences from a randomized trial to a target population. Specifically, we consider nested trial designs, where randomized individuals are nested within a sample…

Cluster-level dynamic treatment regimens can be used to guide sequential, intervention or treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level DTR, the…

Methodology · Statistics 2016-07-15 Timothy NeCamp , Amy Kilbourne , Daniel Almirall

Background. The DURATIONS design has been recently proposed as a practical alternative to a standard two-arm non-inferiority design when the goal is to optimise some continuous aspect of treatment administration, e.g. duration or frequency,…

Applications · Statistics 2023-04-20 Matteo Quartagno , Ehsan Ghorani , Tim P Morris , Michael J Seckl , Mahesh KB Parmar

According to recent results, convergence in a prespecified or prescribed finite time can be achieved under extreme model uncertainty if control is applied continuously over time. This paper shows that this extreme amount of uncertainty…

Systems and Control · Electrical Eng. & Systems 2023-06-27 Hernan Haimovich , Rodrigo Aldana-Lopez , Richard Seeber , David Gomez-Gutierrez

In a tie-breaker design (TBD), subjects with high values of a running variable are given some (usually desirable) treatment, subjects with low values are not, and subjects in the middle are randomized. TBDs are intermediate between…

Methodology · Statistics 2025-05-12 Tim P. Morrison , Art B. Owen

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

We propose a two-stage design for a clinical trial with an early stopping rule for safety. We use different criteria to assess early stopping and efficacy. The early stopping rule is based on a criteria that can be determined more quickly…

Methodology · Statistics 2013-07-25 Daniel Zelterman

Many applied decision-making problems have a dynamic component: The policymaker needs not only to choose whom to treat, but also when to start which treatment. For example, a medical doctor may choose between postponing treatment (watchful…

Methodology · Statistics 2020-05-01 Xinkun Nie , Emma Brunskill , Stefan Wager

To generalize inferences from a randomized trial to the target population of all trial-eligible individuals, investigators can use nested trial designs, where the randomized individuals are nested within a cohort of trial-eligible…

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