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Population-adjusted indirect comparisons estimate treatment effects when access to individual patient data is limited and there are cross-trial differences in effect modifiers. Popular methods include matching-adjusted indirect comparison…

Applications · Statistics 2021-11-05 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

Paired cluster-randomized experiments (pCRTs) are common across many disciplines because there is often natural clustering of individuals, and paired randomization can help balance baseline covariates to improve experimental precision.…

Methodology · Statistics 2024-07-03 Charlotte Z. Mann , Adam C. Sales , Johann A. Gagnon-Bartsch

Clinical AI systems frequently suffer performance decay post-deployment due to temporal data shifts, such as evolving populations, diagnostic coding updates (e.g., ICD-9 to ICD-10), and systemic shocks like the COVID-19 pandemic. Addressing…

Applications · Statistics 2026-01-22 Xin Xiong , Zijian Guo , Haobo Zhu , Chuan Hong , Jordan W Smoller , Tianxi Cai , Molei Liu

Synthetic control (SC) methods have gained rapid popularity in economics recently, where they have been applied in the context of inferring the effects of treatments on standard continuous outcomes assuming linear input-output relations. In…

Methodology · Statistics 2024-02-19 Alicia Curth , Hoifung Poon , Aditya V. Nori , Javier González

We introduce causal inference reasoning to cross-over trials, with a focus on Thorough QT (TQT) studies. For such trials, we propose different sets of assumptions and consider their impact on the modelling strategy and estimation procedure.…

Methodology · Statistics 2022-05-31 Jeppe Ekstrand Halkjær Madsen , Thomas Scheike , Christian Pipper

Accelerated life testing (ALT) is a method of reducing the lifetime of components through exposure to extreme stress. This method of obtaining lifetime information involves the design of a testing experiment, i.e., an accelerated test plan.…

Applications · Statistics 2025-11-10 Owen McGrath , Kevin Burke

Rare events such as conformational changes in biomolecules, phase transitions, and chemical reactions are central to the behavior of many physical systems, yet they are extremely difficult to study computationally because unbiased…

Machine Learning · Statistics 2026-04-16 Yuanqi Du , Jiajun He , Dinghuai Zhang , Eric Vanden-Eijnden , Carles Domingo-Enrich

Disruptions in clinical trials may be due to external events like pandemics, warfare, and natural disasters. Resulting complications may lead to unforeseen intercurrent events (events that occur after treatment initiation and affect the…

Applications · Statistics 2024-08-20 Rachael V. Phillips , Mark J. van der Laan

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

Objective: The objective of this study is to develop a machine learning (ML)-based framework for early risk stratification of clinical trials (CTs) according to their likelihood of exhibiting a high rate of dosing errors, using information…

Machine Learning · Computer Science 2026-02-27 Félicien Hêche , Sohrab Ferdowsi , Anthony Yazdani , Sara Sansaloni-Pastor , Douglas Teodoro

Recent research in causal inference has made important progress in addressing challenges to the external validity of trial findings. Such methods weight trial participant data to more closely resemble the distribution of effect-modifying…

Methodology · Statistics 2024-07-18 Justin M. Clark , Kollin W. Rott , James S. Hodges , Jared D. Huling

Difference-in-Differences (DiD) and Synthetic Control (SC) are widely used methods for causal inference in panel data, each with distinct strengths and limitations. We propose a novel method for short-panel causal inference that integrates…

Econometrics · Economics 2025-09-26 Yixiao Sun , Haitian Xie , Yuhang Zhang

Randomized Controlled Trials (RCTs) are often considered the gold standard for estimating causal effect, but they may lack external validity when the population eligible to the RCT is substantially different from the target population.…

Methodology · Statistics 2023-01-11 Bénédicte Colnet , Julie Josse , Erwan Scornet , Gaël Varoquaux

The key innovation of our analytical method, CaRT, lies in establishing a new hierarchical, distributed architecture to guarantee the safety and robustness of a given learning-based motion planning policy. First, in a nominal setting, the…

Robotics · Computer Science 2023-08-15 Hiroyasu Tsukamoto , Benjamin Rivière , Changrak Choi , Amir Rahmani , Soon-Jo Chung

A practical limitation of cluster randomized controlled trials (cRCTs) is that the number of available clusters may be small, resulting in an increased risk of baseline imbalance under simple randomization. Constrained randomization…

Methodology · Statistics 2022-01-19 Yunji Zhou , Elizabeth L. Turner , Ryan A. Simmons , Fan Li

Tabular synthetic data generators are typically trained to match observational distributions, which can yield high conventional utility (e.g., column correlations, predictive accuracy) yet poor preservation of structural relations relevant…

Machine Learning · Computer Science 2026-03-03 Amir Asiaee , Zhuohui J. Liang , Chao Yan

Causal inference for observational longitudinal studies often requires the accurate estimation of treatment effects on time-to-event outcomes in the presence of time-dependent patient history and time-dependent covariates. To tackle this…

Machine Learning · Statistics 2022-06-17 Jie Zhu , Blanca Gallego

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

Randomized controlled trials (RCTs) are the gold standard for evaluating causal effects but are often costly and difficult to scale; consequently, they are frequently augmented with auxiliary external controls in many applications. Prior…

Methodology · Statistics 2026-05-28 Jiawei Shan , Yiteng Tu , Guanbo Wang , Chao Ying , Jiwei Zhao

There is growing interest in exploring causal effects in target populations via data combination. However, most approaches are tailored to specific settings and lack comprehensive comparative analyses. In this article, we focus on a typical…

Methodology · Statistics 2024-09-17 Peng Wu , Shanshan Luo , Zhi Geng