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Explanatory studies, such as randomized controlled trials, are targeted to extract the true causal effect of interventions on outcomes and are by design adjusted for covariates through randomization. On the contrary, observational studies…

Methodology · Statistics 2022-05-02 Riddhiman Adib , Sheikh Iqbal Ahamed , Mohammad Adibuzzaman

When drawing causal inferences about the effects of multiple treatments on clustered survival outcomes using observational data, we need to address implications of the multilevel data structure, multiple treatments, censoring and unmeasured…

Methodology · Statistics 2022-02-18 Liangyuan Hu , Jiayi Ji , Ronald D. Ennis , Joseph W. Hogan

Imbalance in covariate distributions leads to biased estimates of causal effects. Weighting methods attempt to correct this imbalance but rely on specifying models for the treatment assignment mechanism, which is unknown in observational…

Methodology · Statistics 2022-05-13 Eric Dunipace

Investigating the causal relationship between exposure and the time-to-event outcome is an important topic in biomedical research. Previous literature has discussed the potential issues of using the hazard ratio as a marginal causal effect…

Methodology · Statistics 2022-05-05 Zihan Lin , Ai Ni , Bo Lu

Clustering and dependence are common in trials. For example, in some cluster randomized trials (CRTs), pre-existing clusters are enrolled, randomized, and serve as the basis of intervention delivery. Such CRTs are "fully clustered":…

Randomized clinical trials (RCTs) are widely considered the gold standard for evaluating the effectiveness of new treatments or interventions in drug development. Still, they may not be feasible in certain cases, such as with rare diseases…

Methodology · Statistics 2025-08-05 Di Ran , Fanni Zhang , Sima Shahsavari , Kristine Broglio , Alasdair Henderson , Binbing Yu

Methods for estimating heterogeneous treatment effect in observational data have largely focused on continuous or binary outcomes, and have been relatively less vetted with survival outcomes. Using flexible machine learning methods in the…

Applications · Statistics 2021-07-09 Liangyuan Hu , Jiayi Ji , Fan Li

Causal mediation analysis in cluster-randomized trials (CRTs) is essential for explaining how cluster-level interventions affect individual outcomes, yet it is complicated by interference, post-treatment confounding, and hierarchical…

Methodology · Statistics 2026-03-25 Yuki Ohnishi , Michael J. Daniels , Lei Yang , Fan Li

Utilizing non-concurrent control data (NCC) in the analysis of late-entering arms in platform trials has recently received considerable attention. While incorporating NCC can lead to increased power and lower sample sizes, it might…

Methodology · Statistics 2025-09-12 Pavla Krotka , Martin Posch , Mohamed Gewily , Günter Höglinger , Marta Bofill Roig

Randomized controlled trials (RCTs) frequently utilize covariate-adaptive randomization (CAR) (e.g., stratified block randomization) and commonly suffer from imperfect compliance. This paper studies the identification and inference for the…

Econometrics · Economics 2025-05-02 Federico A. Bugni , Mengsi Gao , Filip Obradovic , Amilcar Velez

Randomized Controlled Trials (RCT) are the current gold standards to empirically measure the effect of a new drug. However, they may be of limited size and resorting to complementary non-randomized data, referred to as observational, is…

Methodology · Statistics 2025-06-11 Ahmed Boughdiri , Julie Josse , Erwan Scornet

Typically, case-control studies to estimate odds-ratios associating risk factors with disease incidence from logistic regression only include cases with newly diagnosed disease. Recently proposed methods allow incorporating information on…

Methodology · Statistics 2020-10-19 Soutrik Mandal , Jing Qin , Ruth M. Pfeiffer

Ensuring constraint satisfaction is a key requirement for safety-critical systems, which include most robotic platforms. For example, constraints can be used for modeling joint position/velocity/torque limits and collision avoidance.…

Robotics · Computer Science 2025-09-04 Elias Fontanari , Gianni Lunardi , Matteo Saveriano , Andrea Del Prete

In cluster-randomized trials (CRTs), there is emerging interest in exploring the causal mechanism in which a cluster-level treatment affects the outcome through an intermediate outcome. The majority of existing causal mediation methods are…

Methodology · Statistics 2026-01-12 Chao Cheng , Fan Li

Detecting coordinated inauthentic behavior on social media remains a critical and persistent challenge, as most existing approaches rely on superficial correlation analysis, employ static parameter settings, and demand extensive and…

Artificial Intelligence · Computer Science 2026-01-05 Weng Ding , Yi Han , Mu-Jiang-Shan Wang

Platform trials are randomized clinical trials that allow simultaneous comparison of multiple interventions, usually against a common control. Arms to test experimental interventions may enter and leave the platform over time. This implies…

Methodology · Statistics 2024-04-12 Marta Bofill Roig , Ekkehard Glimm , Tobias Mielke , Martin Posch

Machine learning (ML) models are increasingly used as decision-support tools in high-risk domains. Evaluating the causal impact of deploying such models can be done with a randomized controlled trial (RCT) that randomizes users to ML vs.…

Methodology · Statistics 2025-07-17 Jacob M. Chen , Michael Oberst

Effective decision making from randomised controlled clinical trials relies on robust interpretation of the numerical results. However, the language we use to describe clinical trials can cause confusion both in trial design and in…

Randomized controlled trials (RCTs) are the gold standard for estimating heterogeneous treatment effects, yet they are often underpowered for detecting effect heterogeneity. Large observational studies (OS) can supplement RCTs for…

Machine Learning · Computer Science 2026-04-07 Amir Asiaee , Samhita Pal

This paper addresses the problem of identifying and estimating the causal effect of a treatment in the presence of unmeasured confounding and various types of right-censoring. Examples of these censoring mechanisms are administrative…

Statistics Theory · Mathematics 2025-03-19 Ilias Willems , Sara Rutten , Gilles Crommen , Ingrid Van Keilegom