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Longitudinal cohort studies, which follow a group of individuals over time, provide the opportunity to examine causal effects of complex exposures on long-term health outcomes. Utilizing data from multiple cohorts has the potential to add…

In causal inference, the correct formulation of the scientific question of interest is a crucial step. Here we apply the estimand framework to a comparison of the outcomes of patient-level clinical trials and observational data to help…

There is increasing interest in combining information from experimental studies, including randomized and single-group trials, with information from external experimental or observational data sources. Such efforts are usually motivated by…

Methodology · Statistics 2026-04-23 Lawson Ung , Guanbo Wang , Sebastien Haneuse , Miguel A. Hernán , Issa J. Dahabreh

Causal inference is the goal of randomized trials and many observational studies. The first step in a formal causal inference framework is to define the causal estimand, and in both types of study this can be intuitively defined as the…

Methodology · Statistics 2025-09-09 Margarita Moreno-Betancur , Rushani Wijesuriya , John B. Carlin

We take steps towards causally interpretable meta-analysis by describing methods for transporting causal inferences from a collection of randomized trials to a new target population, one-trial-at-a-time and pooling all trials. We discuss…

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…

Investigators are increasingly using novel methods for extending (generalizing or transporting) causal inferences from a trial to a target population. In many generalizability and transportability analyses, the trial and the observational…

Methodology · Statistics 2022-09-20 Yu-Han Chiu , Issa J. Dahabreh

While standard meta-analysis pools the results from randomized trials that compare two treatments, network meta-analysis aggregates the results of randomized trials comparing a wider variety of treatment options. However, it is unclear…

Methodology · Statistics 2016-08-15 Mireille E. Schnitzer , Russell J. Steele , Michèle Bally , Ian Shrier

We present methods for causally interpretable meta-analyses that combine information from multiple randomized trials to estimate potential (counterfactual) outcome means and average treatment effects in a target population. We consider…

A randomized trial and an analysis of observational data designed to emulate the trial sample observations separately, but have the same eligibility criteria, collect information on some shared baseline covariates, and compare the effects…

Methodology · Statistics 2022-03-29 Issa J. Dahabreh , Jon A. Steingrimsson , James M. Robins , Miguel A. Hernán

Randomized trials are considered the gold standard for estimating causal effects. Trial findings are often used to inform policy and programming efforts, yet their results may not generalize well to a relevant target population due to…

Observational studies provide the only evidence on the effectiveness of interventions when randomized controlled trials (RCTs) are impractical due to cost, ethical concerns, or time constraints. While many methodologies aim to draw causal…

Background: When planning a cluster randomized trial, evaluators often have access to an enumerated cohort representing the target population of clusters. Practicalities of conducting the trial, such as the need to oversample clusters with…

Methodology · Statistics 2024-09-19 Sarah E. Robertson , Jon A. Steingrimsson , Issa J. Dahabreh

The target trial framework enables causal inference from longitudinal observational data by emulating randomized trials initiated at multiple time points. Precision is often improved by pooling information across trials, with standard…

Methodology · Statistics 2026-01-08 Edoardo Efrem Gervasoni , Liesbet De Bus , Stijn Vansteelandt , Oliver Dukes

Clinical trials are indispensable in developing new treatments, but they face obstacles in patient recruitment and retention, hindering the enrollment of necessary participants. To tackle these challenges, deep learning frameworks have been…

Machine Learning · Computer Science 2023-03-27 Chia-Yuan Chang , Jiayi Yuan , Sirui Ding , Qiaoyu Tan , Kai Zhang , Xiaoqian Jiang , Xia Hu , Na Zou

When assessing causal effects, determining the target population to which the results are intended to generalize is a critical decision. Randomized and observational studies each have strengths and limitations for estimating causal effects…

Methodology · Statistics 2022-10-21 Irina Degtiar , Sherri Rose

As clinical decision-making increasingly moves toward individualized and context-specific treatment recommendations, reliance on any single evidence source, randomized or observational, may be insufficient. Principled integration of…

Randomized clinical trials (RCTs) are ideal for estimating causal effects, because the distributions of background covariates are similar in expectation across treatment groups. When estimating causal effects using observational data,…

Methodology · Statistics 2019-02-27 Anthony D. Scotina , Roee Gutman

When making treatment selection decisions, it is essential to include a causal effect estimation analysis to compare potential outcomes under different treatments or controls, assisting in optimal selection. However, merely estimating…

Machine Learning · Statistics 2024-10-08 Sherly Alfonso-Sánchez , Kristina P. Sendova , Cristián Bravo

Undertaking causal inference with observational data is incredibly useful across a wide range of tasks including the development of medical treatments, advertisements and marketing, and policy making. There are two significant challenges…

Machine Learning · Statistics 2022-01-19 Matthew James Vowels , Necati Cihan Camgoz , Richard Bowden
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