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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 Bayesian nonparametric inference, random discrete probability measures are commonly used as priors within hierarchical mixture models for density estimation and for inference on the clustering of the data. Recently, it has been shown…

Statistics Theory · Mathematics 2012-11-26 Stefano Favaro , Antonio Lijoi , Igor Prünster

Win statistics, including the win ratio, net benefit, and win odds, summarize treatment effects on hierarchical composite endpoints by sequentially comparing patient pairs on component outcomes ordered by clinical importance, proceeding to…

Methodology · Statistics 2026-05-27 Xi Fang , Fan Li

Our Bayesian approach to Mendelian Randomisation uses multiple instruments to assess the putative causal effect of an exposure on an outcome. The approach is robust to violations of the (untestable) Exclusion Restriction condition, and…

Statistics Theory · Mathematics 2017-02-01 Carlo Berzuini , Hui Guo , Stephen Burgess , Luisa Bernardinelli

We propose a novel Bayesian model selection technique on linear mixed-effects models to compare multiple treatments with a control. A fully Bayesian approach is implemented to estimate the marginal inclusion probabilities that provide a…

Applications · Statistics 2015-09-28 Lei Gong , James M. Flegal , Stephen R. Spindler , Patricia L. Mote

Central to several objective approaches to Bayesian model selection is the use of training samples (subsets of the data), so as to allow utilization of improper objective priors. The most common prescription for choosing training samples is…

Statistics Theory · Mathematics 2007-06-13 James O. Berger , Luis R. Pericchi

Effective and rapid decision-making from randomized controlled trials (RCTs) requires unbiased and precise treatment effect inferences. Two strategies to address this requirement are to adjust for covariates that are highly correlated with…

Methodology · Statistics 2024-02-29 Alyssa M. Vanderbeek , Arman Sabbaghi , Jon R. Walsh , Charles K. Fisher

When the sample size is not too small, M-estimators of regression coefficients are approximately normal and unbiased. This leads to the familiar frequentist inference in terms of normality-based confidence intervals and p-values. From a…

Methodology · Statistics 2018-10-19 Erik van Zwet

The development of high-throughput sequencing and targeted therapies has led to the emergence of personalized medicine: a patient's molecular profile or the presence of a specific biomarker of drug response will correspond to a treatment…

Applications · Statistics 2020-05-27 Jonas Béal , Aurélien Latouche

In adaptive clinical trials, the conventional end-of-trial point estimate of a treatment effect is prone to bias, that is, a systematic tendency to deviate from its true value. As stated in recent FDA guidance on adaptive designs, it is…

Bayesian predictive inference provides a coherent description of entire predictive uncertainty through predictive distributions. We examine several widely used sparsity priors from the predictive (as opposed to estimation) inference…

Statistics Theory · Mathematics 2024-06-03 Veronika Rockova

Bayesian Optimization (BO) is a standard tool for hyperparameter tuning thanks to its sample efficiency on expensive black-box functions. While most BO pipelines begin with uniform random initialization, default hyperparameter values…

Machine Learning · Computer Science 2026-02-10 Nicolás Villagrán Prieto , Eduardo C. Garrido-Merchán

Unmeasured confounding is a key challenge for causal inference. In this paper, we establish a framework for unmeasured confounding adjustment with negative control variables. A negative control outcome is associated with the confounder but…

Methodology · Statistics 2024-09-09 Wang Miao , Xu Shi , Yilin Li , Eric Tchetgen Tchetgen

Treatment effect estimates are often available from randomized controlled trials as a single average treatment effect for a certain patient population. Estimates of the conditional average treatment effect (CATE) are more useful for…

Methodology · Statistics 2023-09-12 Wouter A. C. van Amsterdam , Rajesh Ranganath

It has long been noticed that the efficacy observed in small early phase studies is generally better than that observed in later larger studies. Historically, the inflation of the efficacy results from early proof-of-concept studies is…

Methodology · Statistics 2020-06-11 Yongming Qu , Yu Du , Ying Zhang , Lei Shen

Background: We aimed to design a Bayesian adaption trial through extensive simulations to determine values for key design parameters, demonstrate error rates, and establish the expected sample size. The complexity of the proposed outcome…

Disease models are used to examine the likely impact of therapies, interventions and public policy changes. Ensuring that these are well calibrated on the basis of available data and that the uncertainty in their projections is properly…

Computation · Statistics 2025-01-24 Daria Semochkina , Cathal Walsh

We explore the effects of architecture and training objective choice on amortized posterior predictive inference in probabilistic conditional generative models. We aim this work to be a counterpoint to a recent trend in the literature that…

Machine Learning · Computer Science 2020-10-09 Saeid Naderiparizi , Kenny Chiu , Benjamin Bloem-Reddy , Frank Wood

Model selection and order selection problems frequently arise in statistical practice. A popular approach to addressing these problems in the frequentist setting involves information criteria based on penalised maxima of log-likelihoods for…

Statistics Theory · Mathematics 2025-10-29 Hien Duy Nguyen , Mayetri Gupta , Jacob Westerhout , TrungTin Nguyen

Let $X_1,\ldots,X_n$ be a random sample from an unknown probability distribution $P$ on the sample space ${\cal X}$, and let $\theta=\theta(P)$ be a parameter of interest. The present paper proposes a nonparametric `Bayesian bootstrap'…

Statistics Theory · Mathematics 2026-05-13 Nils Lid Hjort
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