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Related papers: Externally Valid Policy Evaluation Combining Trial…

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We present a new approach to the problems of evaluating and learning personalized decision policies from observational data of past contexts, decisions, and outcomes. Only the outcome of the enacted decision is available and the historical…

Machine Learning · Statistics 2019-06-04 Nathan Kallus

In classical study designs, the aim is often to learn about the effects of a treatment or intervention on a single outcome; in many modern studies, however, data on multiple outcomes are collected and it is of interest to explore effects on…

Methodology · Statistics 2017-06-15 Edward H. Kennedy , Shreya Kangovi , Nandita Mitra

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…

We describe how the target trial framework can be used to plan and report analyses that attempt to answer causal questions by combining information from multiple, diverse sources. Such analyses may involve comparisons of treatments…

Methodology · Statistics 2026-05-26 Lawson Ung , Miguel A. Hernán , Issa J. Dahabreh

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…

Econometrics · Economics 2019-10-03 Martin Huber

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

Epidemiologists and applied statisticians often believe that relative effect measures conditional on covariates, such as risk ratios and mean ratios, are ``transportable'' across populations. Here, we examine the identification of causal…

Methodology · Statistics 2022-02-24 Issa J. Dahabreh , Sarah E. Robertson , Jon A. Steingrimsson

Observational data is often readily available in large quantities, but can lead to biased causal effect estimates due to the presence of unobserved confounding. Recent works attempt to remove this bias by supplementing observational data…

In an empirical Bayesian setting, we provide a new multiple testing method, useful when an additional covariate is available, that influences the probability of each null hypothesis being true. We measure the posterior significance of each…

Applications · Statistics 2008-07-30 Egil Ferkingstad , Arnoldo Frigessi , Håvard Rue , Gudmar Thorleifsson , Augustine Kong

In stochastic decision problems, one often wants to estimate the underlying probability measure statistically, and then to use this estimate as a basis for decisions. We shall consider how the uncertainty in this estimation can be…

Statistics Theory · Mathematics 2017-05-24 Samuel N. Cohen

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…

Methodology · Statistics 2024-07-23 Lawson Ung , Tyler J. VanderWeele , Issa J. Dahabreh

Learning about causal effects in target populations and their subsets may be facilitated by combining information from multiple sources. One major class of study designs that combine information involves appending an index study with data…

Predictive models are often introduced to decision-making tasks under the rationale that they improve performance over an existing decision-making policy. However, it is challenging to compare predictive performance against an existing…

Machine Learning · Computer Science 2024-06-13 Luke Guerdan , Amanda Coston , Kenneth Holstein , Zhiwei Steven Wu

Sampling methods such as Stratified Random Sampling can be used to select representative samples of schools for randomized controlled trials of educational interventions. However, these methods may still yield external validity bias when…

Other Statistics · Statistics 2023-04-25 Robert B. Olsen , Maria L. Vasquez-Rossi

When engagement with a randomized trial is driven by factors that affect the outcome or when trial engagement directly affects the outcome independent of treatment, the average treatment effect among trial participants is unlikely to…

Contemporary sample size calculations for external validation of risk prediction models require users to specify fixed values of assumed model performance metrics alongside target precision levels (e.g., 95% CI widths). However, due to the…

Applications · Statistics 2026-02-13 Mohsen Sadatsafavi , Paul Gustafson , Solmaz Setayeshgar , Laure Wynants , Richard D Riley

We consider a general statistical estimation problem involving a finite-dimensional target parameter vector. Beyond an internal data set drawn from the population distribution, external information, such as additional individual data or…

Methodology · Statistics 2025-07-31 Guorong Dai , Lingxuan Shao , Jinbo Chen

To unbiasedly evaluate multiple target policies, the dominant approach among RL practitioners is to run and evaluate each target policy separately. However, this evaluation method is far from efficient because samples are not shared across…

Machine Learning · Computer Science 2024-12-30 Shuze Daniel Liu , Claire Chen , Shangtong Zhang

External validity is often questionable in empirical research, especially in randomized experiments due to the trade-off between internal validity and external validity. To quantify the robustness of external validity, one must first…

Methodology · Statistics 2022-06-20 Tenglong Li

Hybrid randomized controlled trials (hybrid RCTs) integrate external control data, such as historical or concurrent data, with data from randomized trials. While numerous frequentist and Bayesian methods, such as the test-then-pool and…

Methodology · Statistics 2025-10-07 Han Chang Chiam , Franz König , Martin Posch