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Data collected in clinical trials are often composed of multiple types of variables. For example, laboratory measurements and vital signs are longitudinal data of continuous or categorical variables, adverse events may be recurrent events,…

Methodology · Statistics 2023-01-12 Tuo Wang , Rachel Zilinskas , Ying Li , Yongming Qu

Causal discovery serves a pivotal role in mitigating model uncertainty through recovering the underlying causal mechanisms among variables. In many practical domains, such as healthcare, access to the data gathered by individual entities is…

Machine Learning · Computer Science 2024-02-13 Amin Abyaneh , Nino Scherrer , Patrick Schwab , Stefan Bauer , Bernhard Schölkopf , Arash Mehrjou

In this paper, we propose a new approach to causal inference with panel data. Instead of using panel data to adjust for differences in the distribution of unobserved heterogeneity between the treated and comparison groups, we instead use…

Econometrics · Economics 2025-12-01 Brantly Callaway , Derek Dyal , Pedro H. C. Sant'Anna , Emmanuel S. Tsyawo

This paper is concerned with identification, estimation, and specification testing in causal evaluation problems when data is selective and/or missing. We leverage recent advances in the literature on graphical methods to provide a unifying…

Econometrics · Economics 2023-08-10 Simon Calmar Andersen , Louise Beuchert , Phillip Heiler , Helena Skyt Nielsen

Estimating causal effects from observational data is not always possible due to confounding. Identifying a set of appropriate covariates (adjustment set) and adjusting for their influence can remove confounding bias; however, such a set is…

Methodology · Statistics 2020-11-19 Sofia Triantafillou , Gregory Cooper

Methods for addressing missing data have become much more accessible to applied researchers. However, little guidance exists to help researchers systematically identify plausible missing data mechanisms in order to ensure that these methods…

Applications · Statistics 2020-07-29 Adam Davey , Ting Dai

We propose a new method to estimate causal effects from nonexperimental data. Each pair of sample units is first associated with a stochastic 'treatment' - differences in factors between units - and an effect - a resultant outcome…

Methodology · Statistics 2022-11-08 Andre F. Ribeiro , Frank Neffke , Ricardo Hausmann

Meta-analysis, by synthesizing effect estimates from multiple studies conducted in diverse settings, stands at the top of the evidence hierarchy in clinical research. Yet, conventional approaches based on fixed- or random-effects models…

Most causal inference methods focus on estimating marginal average treatment effects, but many important causal estimands depend on the joint distribution of potential outcomes, including the probability of causation and proportions…

Methodology · Statistics 2025-10-16 Zach Shahn , David Madigan

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 studies that rely on data from electronic health records (EHRs), unstructured text data such as clinical progress notes offer a rich source of information about patient characteristics and care that may be missing from structured data.…

Computation and Language · Computer Science 2024-05-22 Reagan Mozer , Aaron R. Kaufman , Leo A. Celi , Luke Miratrix

Estimating long-term causal effects by combining long-term observational and short-term experimental data is a crucial but challenging problem in many real-world scenarios. In existing methods, several ideal assumptions, e.g. latent…

Machine Learning · Computer Science 2025-05-12 Ruichu Cai , Junjie Wan , Weilin Chen , Zeqin Yang , Zijian Li , Peng Zhen , Jiecheng Guo

We consider a general statistical estimation problem involving a finite-dimensional target parameter vector. Beyond an internal data set drawn from the population distribution, external information, such as additional individual data or…

Methodology · Statistics 2025-07-31 Guorong Dai , Lingxuan Shao , Jinbo Chen

In recent years, many methods have been developed for detecting causal relationships in observational data. Some of them have the potential to tackle large data sets. However, these methods fail to discover a combined cause, i.e. a…

Artificial Intelligence · Computer Science 2015-10-16 Saisai Ma , Jiuyong Li , Lin Liu , Thuc Duy Le

Detection of interactions between treatment effects and patient descriptors in clinical trials is critical for optimizing the drug development process. The increasing volume of data accumulated in clinical trials provides a unique…

Applications · Statistics 2017-12-25 Baptiste Goujaud , Eric W. Tramel , Pierre Courtiol , Mikhail Zaslavskiy , Gilles Wainrib

In recent years, rather than enclosing data within a single organization, exchanging and combining data from different domains has become an emerging practice. Many studies have discussed the economic and utility value of data and data…

Computers and Society · Computer Science 2020-12-23 Teruaki Hayashi , Hiroki Sakaji , Hiroyasu Matsushima , Yoshiaki Fukami , Takumi Shimizu , Yukio Ohsawa

Randomized trials are considered the gold standard for making informed decisions in medicine, yet they often lack generalizability to the patient populations in clinical practice. Observational studies, on the other hand, cover a broader…

Methodology · Statistics 2026-04-14 Piersilvio De Bartolomeis , Javier Abad , Konstantin Donhauser , Fanny Yang

Suppose we are interested in the effect of a treatment in a clinical trial. The efficiency of inference may be limited due to small sample size. However, external control data are often available from historical studies. Motivated by an…

Methodology · Statistics 2021-12-10 Xinyu Li , Wang Miao , Fang Lu , Xiao-Hua Zhou

To improve confounder adjustments, observational studies are often matched on potential confounders. While matched case-control studies are common and well covered in the literature, our focus here is on matched cohort studies, which are…

The standard methods for detecting differential gene expression are mostly designed for analyzing a single gene expression experiment. When data from multiple related gene expression studies are available, separately analyzing each study is…

Methodology · Statistics 2013-11-07 Yingying Wei , Hongkai Ji