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While sample sizes in randomized clinical trials are large enough to estimate the average treatment effect well, they are often insufficient for estimation of treatment-covariate interactions critical to studying data-driven precision…

机器学习 · 统计学 2020-04-22 Steve Yadlowsky , Fabio Pellegrini , Federica Lionetto , Stefan Braune , Lu Tian

It is increasingly common to augment randomized controlled trial with external controls from observational data, to evaluate the treatment effect of an intervention. Traditional approaches to treatment effect estimation involve ambiguous…

统计方法学 · 统计学 2025-03-28 Bo Liu , Laine Thomas , Rury R. Holman , Fan Li

Population-adjusted indirect comparisons (PAICs) are widely used to synthesize evidence when randomized controlled trials enroll different patient populations and head-to-head comparisons are unavailable. Although PAICs adjust for observed…

统计方法学 · 统计学 2026-02-20 Conor Chandler , Jack Ishak

Longitudinal data tracking repeated measurements on individuals are highly valued for research because they offer controls for unmeasured individual heterogeneity that might otherwise bias results. Random effects or mixed models approaches,…

应用统计 · 统计学 2009-09-29 J. R. Lockwood , Daniel F. McCaffrey

While randomized controlled trials (RCTs) are critical for establishing the efficacy of new therapies, there are limitations regarding what comparisons can be made directly from trial data. RCTs are limited to a small number of comparator…

Violations of the parallel trends assumption pose significant challenges for causal inference in difference-in-differences (DiD) studies, especially in policy evaluations where pre-treatment dynamics and external shocks may bias estimates.…

统计方法学 · 统计学 2025-08-06 Seong Woo Han , Nandita Mitra , Gary Hettinger , Arman Oganisian

Randomized experimentation (also known as A/B testing or bucket testing) is widely used in the internet industry to measure the metric impact obtained by different treatment variants. A/B tests identify the treatment variant showing the…

统计方法学 · 统计学 2020-12-23 Ye Tu , Kinjal Basu , Cyrus DiCiccio , Romil Bansal , Preetam Nandy , Padmini Jaikumar , Shaunak Chatterjee

An important task in drug development is to identify patients, which respond better or worse to an experimental treatment. Identifying predictive covariates, which influence the treatment effect and can be used to define subgroups of…

统计方法学 · 统计学 2018-11-27 Marius Thomas , Björn Bornkamp , Katja Ickstadt

We studied how lagged linear regression can be used to detect the physiologic effects of drugs from data in the electronic health record (EHR). We systematically examined the effect of methodological variations ((i) time series…

统计方法学 · 统计学 2018-01-29 Matthew E. Levine , David J. Albers , George Hripcsak

This paper studies semiparametric Bayesian inference for the average treatment effect on the treated (ATT) within the difference-in-differences (DiD) research design. We propose two new Bayesian methods with frequentist validity. The first…

计量经济学 · 经济学 2025-06-17 Christoph Breunig , Ruixuan Liu , Zhengfei Yu

Inference in hierarchical nonlinear models needs careful consideration about targeting parameters that have either a conditional or population-average interpretation. For the special case of mixed-effects nonlinear sigmoidal models we…

应用统计 · 统计学 2017-07-11 Daniel Gerhard , Christian Ritz

In many practical situations, randomly assigning treatments to subjects is uncommon due to feasibility constraints. For example, economic aid programs and merit-based scholarships are often restricted to those meeting specific income or…

统计方法学 · 统计学 2025-04-25 Kevin Christian Wibisono , Debarghya Mukherjee , Moulinath Banerjee , Ya'acov Ritov

We propose principled prediction intervals to quantify the uncertainty of a large class of synthetic control predictions (or estimators) in settings with staggered treatment adoption, offering precise non-asymptotic coverage probability…

计量经济学 · 经济学 2025-02-04 Matias D. Cattaneo , Yingjie Feng , Filippo Palomba , Rocio Titiunik

The conditional average treatment effect (CATE) is frequently estimated to refute the homogeneous treatment effect assumption. Under this assumption, all units making up the population under study experience identical benefit from a given…

This chapter covers different approaches to policy evaluation for assessing the causal effect of a treatment or intervention on an outcome of interest. As an introduction to causal inference, the discussion starts with the experimental…

计量经济学 · 经济学 2019-10-03 Martin Huber

Randomized clinical trials (RCTs) are widely considered the gold standard for evaluating the effectiveness of new treatments or interventions in drug development. Still, they may not be feasible in certain cases, such as with rare diseases…

统计方法学 · 统计学 2025-08-05 Di Ran , Fanni Zhang , Sima Shahsavari , Kristine Broglio , Alasdair Henderson , Binbing Yu

The positivity assumption is central in the identification of a causal effect, and especially the stochastic variant is an issue many applied researchers face, yet is rarely discussed, especially in conjunction with continuous treatments or…

应用统计 · 统计学 2026-02-03 Katharina Ring , Michael Schomaker

We propose a difference-in-differences (DiD) framework designed for time-varying continuous treatments across multiple periods. Specifically, we estimate the average treatment effect on the treated (ATET) by comparing distinct non-zero…

计量经济学 · 经济学 2026-01-16 Michel F. C. Haddad , Martin Huber , José Eduardo Medina-Reyes , Lucas Z. Zhang

Randomized trials are viewed as the benchmark for assessing causal effects of treatments on outcomes of interest. Nonetheless, challenges such as measurement error can undermine the standard causal assumptions for randomized trials. In…

统计方法学 · 统计学 2025-08-27 Dane Isenberg , Nandita Mitra , Steven C. Marcus , Rinad S. Beidas , Kristin A. Linn

Trial engagement effects are effects of trial participation on the outcome that are not mediated by treatment assignment. Most work on extending (generalizing or transporting) causal inferences from a randomized trial to a target population…

统计方法学 · 统计学 2024-07-23 Lawson Ung , Tyler J. VanderWeele , Issa J. Dahabreh
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