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We often seek to estimate the impact of an exposure naturally occurring or randomly assigned at the cluster-level. For example, the literature on neighborhood determinants of health continues to grow. Likewise, community randomized trials…

Methodology · Statistics 2021-07-08 Laura B. Balzer , Wenjing Zheng , Mark J. van der Laan , Maya L. Petersen

The cross-entropy (CE) method is a popular stochastic method for optimization due to its simplicity and effectiveness. Designed for rare-event simulations where the probability of a target event occurring is relatively small, the CE-method…

Machine Learning · Computer Science 2020-09-22 Robert J. Moss

A new matching method is proposed for the estimation of the average treatment effect of social policy interventions (e.g., training programs or health care measures). Given an outcome variable, a treatment and a set of pre-treatment…

Statistics Theory · Mathematics 2007-06-13 Stefano Iacus , Giuseppe Porro

We introduce the BREASE framework for the Bayesian analysis of randomized controlled trials with a binary treatment and a binary outcome. Approaching the problem from a causal inference perspective, we propose parameterizing the likelihood…

Methodology · Statistics 2024-11-26 Nicholas J. Irons , Carlos Cinelli

Causal effect estimation (CEE) provides a crucial tool for predicting the unobserved counterfactual outcome for an entity. As CEE relaxes the requirement for ``perfect'' counterfactual samples (e.g., patients with identical attributes and…

Machine Learning · Computer Science 2024-11-19 Hechuan Wen , Tong Chen , Guanhua Ye , Li Kheng Chai , Shazia Sadiq , Hongzhi Yin

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

Randomized controlled trials are the standard method for estimating causal effects, ensuring sufficient statistical power and confidence through adequate sample sizes. However, achieving such sample sizes is often challenging. This study…

Methodology · Statistics 2025-03-28 Keisuke Hanada , Masahiro Kojima

Causal mediation analysis of observational data is an important tool for investigating the potential causal effects of medications on disease-related risk factors, and on time-to-death (or disease progression) through these risk factors.…

We present a unified three-state model (TSM) framework for evaluating treatment effects in clinical trials in the presence of treatment crossover. Researchers have proposed diverse methodologies to estimate the treatment effect that would…

Methodology · Statistics 2024-09-18 Zile Zhao , Ye Li , Xiaodong Luo , Ray Bai

Typically, trials investigate the impact of either an individual-level intervention on participant outcomes, or the impact of a cluster-level intervention on participant outcomes. Factorial designs consider two (or more) treatments for each…

Methodology · Statistics 2026-05-04 Rhys Bowden , Rebecca Walwyn , Jessica Kasza , Andrew Copas , Fan Li , James Wason , Andrew Forbes

Recent methods to improve generalizations from nonrandom samples typically invoke assumptions such as the strong ignorability of sample selection that are often controversial in practice to derive point estimates. Rather than focus on the…

Applications · Statistics 2017-01-06 Wendy Chan

Evaluating quality-of-life (QoL) outcomes in populations with high mortality risk is complicated by truncation by death, since QoL is undefined for individuals who do not survive to the planned measurement time. We propose a framework that…

In this paper, a Bayesian approach is developed for simultaneously comparing multiple experimental treatments with a common control treatment in an exploratory clinical trial. The sample size is set to ensure that, at the end of the study,…

Statistics Theory · Mathematics 2019-11-14 John Whitehead , Faye Cleary , Amanda Turner

Estimating average treatment effects from observational data is challenging under practical violations of the positivity assumption. Targeted Maximum Likelihood Estimators (TMLEs) are widely used because of their double robustness and…

Methodology · Statistics 2026-04-28 Yichen Xu , Susan Gruber , Mark J. van der Laan

We show that causal effects can be identified when there is bunching in the distribution of a continuous treatment variable, without imposing any parametric assumptions. This yields a new nonparametric method for overcoming selection bias…

Econometrics · Economics 2025-07-08 Carolina Caetano , Gregorio Caetano , Leonard Goff , Eric Nielsen

We propose a new method to estimate causal effects from nonexperimental data. Each pair of sample units is first associated with a stochastic 'treatment' - differences in factors between units - and an effect - a resultant outcome…

Methodology · Statistics 2022-11-08 Andre F. Ribeiro , Frank Neffke , Ricardo Hausmann

The existing cross-validated risk scores (CVRS) design has been proposed for developing and testing the efficacy of a treatment in a high-efficacy patient group (the sensitive group) using high-dimensional data (such as genetic data). The…

Methodology · Statistics 2024-02-07 Svetlana Cherlin , James M. S. Wason

Treatment non-compliance, where individuals deviate from their assigned experimental conditions, frequently complicates the estimation of causal effects. To address this, we introduce a novel learning framework based on a mixture of experts…

Methodology · Statistics 2025-06-25 François Grolleau , Céline Béji , Raphaël Porcher , François Petit

This paper proposes a Sequential Monte Carlo approach for the Bayesian estimation of mixed causal and noncausal models. Unlike previous Bayesian estimation methods developed for these models, Sequential Monte Carlo offers extensive…

Econometrics · Economics 2025-01-08 Gianluca Cubadda , Francesco Giancaterini , Stefano Grassi

Objectives: With accurate estimates of expected safety results, clinical trials could be better designed and monitored. We evaluated methods for predicting serious adverse event (SAE) results in clinical trials using information only from…

Computation and Language · Computer Science 2025-08-12 Qixuan Hu , Xumou Zhang , Jinman Kim , Florence Bourgeois , Adam G. Dunn
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