English
Related papers

Related papers: Towards Generalizing Inferences from Trials to Tar…

200 papers

Claiming causal inferences in network settings necessitates careful consideration of the often complex dependency between outcomes for actors. Of particular importance are treatment spillover or outcome interference effects. We consider…

Methodology · Statistics 2022-07-18 Duncan A. Clark , Mark S. Handcock

We derive new variance formulas for inference on a general class of estimands of causal average treatment effects in a Randomized Control Trial (RCT). We generalize Robins (1988) and show that when the estimand of interest is the Sample…

Statistics Theory · Mathematics 2017-10-19 Jasjeet S. Sekhon , Yotam Shem-Tov

We describe methods that extend (generalize or transport) causal inferences from cluster randomized trials to a target population of clusters, under a general nonparametric model that allows for arbitrary within-cluster dependence. We…

The validity of medical studies based on real-world clinical data, such as observational studies, depends on critical assumptions necessary for drawing causal conclusions about medical interventions. Many published studies are flawed…

Artificial Intelligence · Computer Science 2024-07-30 Ahmed Alaa , Rachael V. Phillips , Emre Kıcıman , Laura B. Balzer , Mark van der Laan , Maya Petersen

Randomized Controlled Trials are one of the pillars of science; nevertheless, they rely on hand-crafted hypotheses and expensive analysis. Such constraints prevent causal effect estimation at scale, potentially anchoring on popular yet…

Machine Learning · Computer Science 2026-01-07 Tommaso Mencattini , Riccardo Cadei , Francesco Locatello

Causality is a fundamental part of the scientific endeavour to understand the world. Unfortunately, causality is still taboo in much of psychology and social science. Motivated by a growing number of recommendations for the importance of…

Methodology · Statistics 2022-06-27 Matthew J. Vowels

Causal inference from observational data can be viewed as a missing data problem arising from a hypothetical population-scale randomized trial matched to the observational study. This links a target trial protocol with a corresponding…

Methodology · Statistics 2022-07-27 Andrew Yiu , Edwin Fong , Stephen Walker , Chris Holmes

Randomized controlled trials (RCTs) often suffer from limited inferential efficiency in estimating treatment effects due to their small sample sizes. In recent years, incorporating external controls (ECs) has gained increasing attention as…

Methodology · Statistics 2026-04-16 Qinwei Yang , Jingyi Li , Peng Wu , Shu Yang

External controls (ECs) from historical trials or real-world data have gained increasing attention as a way to augment hybrid and single-arm trials, especially when balanced randomization is infeasible. While most existing work has focused…

Methodology · Statistics 2025-12-15 Yujing Gao , Xiang Zhang , Shu Yang

Estimating treatment effects (TE) from observational data is a critical yet complex task in many fields, from healthcare and economics to public policy. While recent advances in machine learning and causal inference have produced powerful…

Machine Learning · Computer Science 2025-08-15 Jeroen Berrevoets , Julianna Piskorz , Robert Davis , Harry Amad , Jim Weatherall , Mihaela van der Schaar

Generalization to novel compound tasks under distribution shift is important for deploying transformer-based language models (LMs). This work investigates Chain-of-Thought (CoT) reasoning as a means to enhance OOD generalization. Through…

Computation and Language · Computer Science 2026-03-31 Ru Wang , Wei Huang , Selena Song , Haoyu Zhang , Qian Niu , Yusuke Iwasawa , Yutaka Matsuo , Jiaxian Guo

A platform trial is an innovative clinical trial design that uses a master protocol to evaluate multiple treatments, where patients are often assigned to different subsets of treatment arms based on individual characteristics, enrollment…

Mendelian randomization (MR) has become an essential tool for causal inference in biomedical and public health research. By using genetic variants as instrumental variables, MR helps address unmeasured confounding and reverse causation,…

Methodology · Statistics 2025-11-04 Minhao Yao , Anqi Wang , Xihao Li , Zhonghua Liu

Typical causal effects are defined based on the marginal distribution of potential outcomes. However, many real-world applications require causal estimands involving the joint distribution of potential outcomes to enable more nuanced…

Methodology · Statistics 2026-04-17 Peng Wu , Xiaojie Mao

Causal effect estimation seeks to determine the impact of an intervention from observational data. However, the existing causal inference literature primarily addresses treatment effects on frequently occurring events. But what if we are…

Machine Learning · Statistics 2025-06-18 Jiyuan Tan , Jose Blanchet , Vasilis Syrgkanis

Generalisability and transportability of clinical prediction models (CPMs) refer to their ability to maintain predictive performance when applied to new populations. While CPMs may show good generalisability or transportability to a…

Methodology · Statistics 2024-12-06 Kritchavat Ploddi , Matthew Sperrin , Glen P. Martin , Maurice M. O'Connell

Causal inference studies whether the presence of a variable influences an observed outcome. As measured by quantities such as the "average treatment effect," this paradigm is employed across numerous biological fields, from vaccine and drug…

Recent developments in causal inference allow us to transport a causal effect of a time-fixed treatment from a randomized trial to a target population across space but within the same time frame. In contrast to transportability across…

Methodology · Statistics 2026-03-11 Laura Forastiere , Fan Li , Michela Baccini

With the growth in experimental studies in education, policymakers and practitioners are interested in understanding not only what works, but for whom an intervention works. This interest in the generalizability of a study's findings has…

Methodology · Statistics 2022-05-02 Wendy Chan , Jimin Oh , Katherine J. Wilson

Objective: The growing availability of large-scale observational clinical datasets and challenges in conducting randomized controlled trials have spurred enthusiasm in using causal machine learning (ML) for causal inference in observational…