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The use of patient-level information from previous studies, registries, and other external datasets can support the analysis of single-arm and randomized controlled trials to evaluate and test experimental treatments. However, the…

Methodology · Statistics 2025-10-23 Gopal Kotecha , Daniel E. Schwartz , Steffen Ventz , Lorenzo Trippa

Comparing survival experiences of different groups of data is an important issue in several applied problems. A typical example is where one wishes to investigate treatment effects. Here we propose a new Bayesian approach based on…

Methodology · Statistics 2024-07-17 Alan Riva-Palacio , Fabrizio Leisen , Antonio Lijoi

A common statistical problem in econometrics is to estimate the impact of a treatment on a treated unit given a control sample with untreated outcomes. Here we develop a generative learning approach to this problem, learning the probability…

Machine Learning · Statistics 2019-10-17 Chirag Modi , Uros Seljak

This paper presents an automatic formal controller synthesis method for nonlinear sampled-data systems with safety and reachability specifications. Fundamentally, the presented method is not restricted to polynomial systems and controllers.…

Systems and Control · Computer Science 2018-12-07 Cees F. Verdier , Manuel Mazo

The synthetic control method (SCM) is widely used for causal inference with panel data, particularly when the number of treated units is small. It relies on the stable unit treatment value assumption (SUTVA), ruling out spillover effects.…

Econometrics · Economics 2026-03-26 Shosei Sakaguchi , Hayato Tagawa

Cluster randomized trials (CRTs) with multiple unstructured mediators present significant methodological challenges for causal inference due to within-cluster correlation, interference among units, and the complexity introduced by multiple…

Methodology · Statistics 2025-04-21 Yuki Ohnishi , Fan Li

The widespread adoption of electronic health records (EHRs) and subsequent increased availability of longitudinal healthcare data has led to significant advances in our understanding of health and disease with direct and immediate impact on…

Machine Learning · Computer Science 2022-01-21 Simon Bing , Andrea Dittadi , Stefan Bauer , Patrick Schwab

Electronic health records (EHRs) are invaluable for clinical research, yet privacy concerns severely restrict data sharing. Synthetic data generation offers a promising solution, but EHRs present unique challenges: they contain both…

Machine Learning · Computer Science 2026-03-26 Shaonan Liu , Yuichiro Iwashita , Soichiro Nakako , Masakazu Iwamura , Koichi Kise

Access to electronic health records (EHRs) for digital health research is often limited by privacy regulations and institutional barriers. Synthetic EHRs have been proposed as a way to enable safe and sovereign data sharing; however,…

Machine Learning · Computer Science 2026-03-10 Guanglin Zhou , Armin Catic , Motahare Shabestari , Matthew Young , Chaiquan Li , Katrina Poppe , Sebastiano Barbieri

Spontaneous reporting system databases are key resources for post-marketing surveillance, providing real-world evidence (RWE) on the adverse events (AEs) of regulated drugs or other medical products. Various statistical methods have been…

Methodology · Statistics 2026-03-24 Xin-Wei Huang , Saptarshi Chakraborty

This paper presents the link between stochastic approximation and clinical trials based on randomized urn models investigated in Bai and Hu (1999,2005) and Bai, Hu and Shen (2002). We reformulate the dynamics of both the urn composition and…

Probability · Mathematics 2017-01-19 Sophie Laruelle , Gilles Pagès

The estimation of heterogeneous treatment effects in the potential outcome setting is biased when there exists model misspecification or unobserved confounding. As these biases are unobservable, what model to use when remains a critical…

Methodology · Statistics 2024-05-09 Shonosuke Sugasawa , Kosaku Takanashi , Kenichiro McAlinn , Edoardo M. Airoldi

Single-arm trials (SATs) may be used to support regulatory submissions in settings where there is a high unmet medical need and highly promising early efficacy data undermine the equipoise needed for randomization. In this context,…

When it is not feasible to conduct randomized controlled trials (RCTs), the use of external control arms based on real-world data (RWD) may be a viable option. However, challenges arising from data heterogeneity must be addressed to ensure…

Applications · Statistics 2026-03-02 Javier Cabrera , Berhanu Alemayehu , Demissie Alemayehu , Sofia Weigle

Objective. Demographic groups are often represented at different rates in medical datasets. These differences can create bias in machine learning algorithms, with higher levels of performance for better-represented groups. One promising…

Machine Learning · Computer Science 2024-12-24 Daniel Smolyak , Arshana Welivita , Margrét V. Bjarnadóttir , Ritu Agarwal

Wheelchairs and robotic arms enhance independent living by assisting individuals with upper-body and mobility limitations in their activities of daily living (ADLs). Although recent advancements in assistive robotics have focused on…

Robotics · Computer Science 2026-01-30 Guangping Liu , Tipu Sultan , Vittorio Di Giorgio , Nick Hawkins , Flavio Esposito , Madi Babaiasl

Data quality is the key factor for the development of trustworthy AI in healthcare. A large volume of curated datasets with controlled confounding factors can help improve the accuracy, robustness and privacy of downstream AI algorithms.…

Machine Learning · Computer Science 2022-09-21 Xiaodan Xing , Huanjun Wu , Lichao Wang , Iain Stenson , May Yong , Javier Del Ser , Simon Walsh , Guang Yang

In fixed-confidence best arm identification (BAI), the objective is to quickly identify the optimal option while controlling the probability of error below a desired threshold. Despite the plethora of BAI algorithms, existing methods…

Machine Learning · Computer Science 2026-01-05 Brian M. Cho , Nathan Kallus

Platform trials can evaluate the efficacy of several treatments compared to a control. The number of treatments is not fixed, as arms may be added or removed as the trial progresses. Platform trials are more efficient than independent…

Rare variants are hypothesized to be largely responsible for heritability and susceptibility to disease in humans. So rare variants association studies hold promise for understanding disease. Conversely though, the rareness of the variants…

Methodology · Statistics 2021-12-06 Lorenzo Masoero , Joshua Schraiber , Tamara Broderick