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Background: In settings where proof-of-principle trials have succeeded but the effectiveness of different forms of implementation remains uncertain, trials that not only generate information about intervention effects but also provide…

Quantitative Methods · Quantitative Biology 2017-05-16 Guy Harling , Rui Wang , Jukka-Pekka Onnela , Victor De Gruttola

Sustained treatment strategies are common in many domains, particularly in medicine, where many treatment are delivered repeatedly over time. The effects of adherence to a treatment strategy throughout follow-up are often more relevant to…

Crossover designs are an extremely useful tool to investigators, whilst group sequential methods have proven highly proficient at improving the efficiency of parallel group trials. Yet, group sequential methods and crossover designs have…

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

Recently, methodology was presented to facilitate the incorporation of interim analyses in stepped-wedge (SW) cluster randomised trials (CRTs). Here, we extend this previous discussion. We detail how the stopping boundaries, allocation…

Methodology · Statistics 2018-03-28 Michael Grayling , David Robertson , James Wason , Adrian Mander

Empirical studies using Regression Discontinuity (RD) designs often explore heterogeneous treatment effects based on pretreatment covariates, even though no formal statistical methods exist for such analyses. This has led to the widespread…

Dynamic treatment regimes operationalize the clinical decision process as a sequence of functions, one for each clinical decision, where each function takes as input up-to-date patient information and gives as output a single recommended…

Methodology · Statistics 2012-08-08 Eric B. Laber , Daniel J. Lizotte , Bradley Ferguson

The ability to accurately estimate the sample size required by a stepped-wedge (SW) cluster randomized trial (CRT) routinely depends upon the specification of several nuisance parameters. If these parameters are mis-specified, the trial…

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

We consider the estimation of treatment effects in settings when multiple treatments are assigned over time and treatments can have a causal effect on future outcomes or the state of the treated unit. We propose an extension of the…

Econometrics · Economics 2021-06-18 Greg Lewis , Vasilis Syrgkanis

This paper studies how to design two-wave experiments in the presence of spillovers for precise inference on treatment effects. We consider units connected through a single network, local dependence among individuals, and a general class of…

Econometrics · Economics 2025-11-25 Davide Viviano

Individual participant data (IPD) meta-analyses of randomised trials are considered a reliable way to assess participant-level treatment effect modifiers but may not make the best use of the available data. Traditionally, effect modifiers…

How should a network experiment be designed to achieve high statistical power? Ex- perimental treatments on networks may spread. Randomizing assignment of treatment to nodes enhances learning about the counterfactual causal effects of a…

Methodology · Statistics 2018-04-02 Jake Bowers , Bruce A. Desmarais , Mark Frederickson , Nahomi Ichino , Hsuan-Wei Lee , Simi Wang

The treatment effects of the same therapy observed from multiple clinical trials can often be very different. Yet the patient characteristics accounting for these differences may not be identifiable in real world practice. There needs to be…

Statistics Theory · Mathematics 2015-03-20 Tinghui Yu , Yabing Mai , Sherry Liu , Xiaofei Hu

Sequential, multiple assignment, randomized trial (SMART) designs have become increasingly popular in the field of precision medicine by providing a means for comparing sequences of treatments tailored to the individual patient, i.e.,…

Applications · Statistics 2018-04-13 William J. Artman , Inbal Nahum-Shani , Tianshuang Wu , James R. McKay , Ashkan Ertefaie

A Bayesian multivariate model with a structured covariance matrix for multi-way nested data is proposed. This flexible modeling framework allows for positive and for negative associations among clustered observations, and generalizes the…

Methodology · Statistics 2024-08-27 Stef Baas , Richard J. Boucherie , Jean-Paul Fox

Stochastic differential equations (SDEs) are established tools to model physical phenomena whose dynamics are affected by random noise. By estimating parameters of an SDE intrinsic randomness of a system around its drift can be identified…

Computation · Statistics 2012-05-03 Umberto Picchini , Susanne Ditlevsen

We investigate spike-timing dependent plasticity (STPD) in the case of a synapse connecting two neural cells. We develop a theoretical analysis of several STDP rules using Markovian theory. In this context there are two different…

Neurons and Cognition · Quantitative Biology 2021-11-16 Philippe Robert , Gaëtan Vignoud

The two-stage preference design (TSPD) enables the inference for treatment efficacy while allowing for incorporation of patient preference to treatment. It can provide unbiased estimates for selection and preference effects, where a…

Methodology · Statistics 2023-11-28 Ruyi Liu , Fan Li , Denise Esserman , Mary M. Ryan

A common problem faced in clinical studies is that of estimating the effect of the most effective (e.g., the one having the largest mean) treatment among $k~(\geq2)$ available treatments. The most effective treatment is adjudged based on…

Statistics Theory · Mathematics 2022-09-20 Masihuddin , Neeraj Misra

Randomized controlled trials (RCTs) can be used to generate guarantees on treatment effects. However, RCTs often spend unnecessary resources exploring sub-optimal treatments, which can reduce the power of treatment guarantees. To address…

Computers and Society · Computer Science 2024-10-16 Santiago Cortes-Gomez , Naveen Raman , Aarti Singh , Bryan Wilder

We consider experiments for comparing treatments using units that are ordered linearly over time or space within blocks. In addition to the block effect, we assume that a trend effect influences the response. The latter is modeled as a…

Statistics Theory · Mathematics 2008-12-18 Dibyen Majumdar , John Stufken
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