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A further understanding of cause and effect within observational data is critical across many domains, such as economics, health care, public policy, web mining, online advertising, and marketing campaigns. Although significant advances…

Machine Learning · Computer Science 2023-04-11 Zhixuan Chu , Sheng Li

In recent years, real-world external controls have grown in popularity as a tool to empower randomized placebo-controlled trials, particularly in rare diseases or cases where balanced randomization is unethical or impractical. However, as…

Methodology · Statistics 2024-11-14 Chenyin Gao , Shu Yang , Mingyang Shan , Wenyu Ye , Ilya Lipkovich , Douglas Faries

Randomized controlled trials (RCTs) are the gold standard for causal inference, yet practical constraints often limit the size of the concurrent control arm. Borrowing control data from previous trials offers a potential efficiency gain,…

Methodology · Statistics 2026-03-17 Linying Yang , Xing Liu , Robin J. Evans

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

In oncology the efficacy of novel therapeutics often differs across patient subgroups, and these variations are difficult to predict during the initial phases of the drug development process. The relation between the power of randomized…

Methodology · Statistics 2025-06-05 Boyu Ren , Federico Ferrari , Sandra Fortini , Steffen Ventz , Lorenzo Trippa

Generalization methods offer a powerful solution to one of the key drawbacks of randomized controlled trials (RCTs): their limited representativeness. By enabling the transport of treatment effect estimates to target populations subject to…

Methodology · Statistics 2025-05-20 Ahmed Boughdiri , Clément Berenfeld , Julie Josse , Erwan Scornet

Despite advances in language modelling, distributional methods that build semantic representations from co-occurrences fail to discriminate between plausible and implausible events. In this work, we investigate how plausibility prediction…

Computation and Language · Computer Science 2025-03-18 Jacob Chmura , Jonah Dauvet , Sebastian Sabry

Data from observational studies (OSs) is widely available and readily obtainable yet frequently contains confounding biases. On the other hand, data derived from randomized controlled trials (RCTs) helps to reduce these biases; however, it…

Methodology · Statistics 2024-10-30 Dong Yao , Caizhi Tang , Qing Cui , Longfei Li

Gathering the most information by picking the least amount of data is a common task in experimental design or when exploring an unknown environment in reinforcement learning and robotics. A widely used measure for quantifying the…

Machine Learning · Statistics 2015-09-17 Johannes Kulick , Robert Lieck , Marc Toussaint

Randomised controlled trials (RCTs) are regarded as the gold standard for estimating causal treatment effects on health outcomes. However, RCTs are not always feasible, because of time, budget or ethical constraints. Observational data such…

Methodology · Statistics 2024-02-20 Li Su , Roonak Rezvani , Shaun R. Seaman , Colin Starr , Isaac Gravestock

In this work, we integrate the predictive capabilities of compartmental disease dynamics models with machine learning ability to analyze complex, high-dimensional data and uncover patterns that conventional models may overlook.…

Numerical Analysis · Mathematics 2025-05-28 Muhammad Awais , Abu Safyan Ali , Giacomo Dimarco , Federica Ferrarese , Lorenzo Pareschi

High dimensional case control studies are ubiquitous in the biological sciences, particularly genomics. To maximise power while constraining cost and to minimise type-1 error rates, researchers typically seek to replicate findings in a…

Methodology · Statistics 2017-07-11 James Liley

We consider the problem of estimating the average treatment effect (ATE) when both randomized control trial (RCT) data and external real-world data (RWD) are available. We decompose the ATE estimand as the difference between a pooled-ATE…

Methodology · Statistics 2025-01-22 Mark van der Laan , Sky Qiu , Jens Magelund Tarp , Lars van der Laan

The growing availability of large health databases has expanded the use of observational studies for comparative effectiveness research. Unlike randomized trials, observational studies must adjust for systematic differences in patient…

Methodology · Statistics 2026-01-21 Haidong Lu , Fan Li , Laine E. Thomas , Fan Li

In many scientific experiments, the data annotating cost constraints the pace for testing novel hypotheses. Yet, modern machine learning pipelines offer a promising solution, provided their predictions yield correct conclusions. We focus on…

Adaptive subgroup enrichment design is an efficient design framework that allows accelerated development for investigational treatments while also having flexibility in population selection within the course of the trial. The adaptive…

Methodology · Statistics 2022-10-21 Liwen Wu , Qing Li , Mengya Liu , Jianchang Lin

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

Our study investigates the impact of data augmentation on the performance of multivariate time series models, focusing on datasets from the UCR archive. Despite the limited size of these datasets, we achieved classification accuracy…

Machine Learning · Computer Science 2024-06-11 Romain Ilbert , Thai V. Hoang , Zonghua Zhang

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…

Covariate adjustment is an important tool in the analysis of randomized clinical trials and observational studies. It can be used to increase efficiency and thus power, and to reduce possible bias. While most statistical tests in randomized…

Methodology · Statistics 2011-08-03 Xiaoru Wu , Zhiliang Ying
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