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Uncertainty quantification of causal effects is crucial for safety-critical applications such as personalized medicine. A powerful approach for this is conformal prediction, which has several practical benefits due to model-agnostic…

Machine Learning · Computer Science 2026-02-04 Maresa Schröder , Dennis Frauen , Jonas Schweisthal , Konstantin Heß , Valentyn Melnychuk , Stefan Feuerriegel

We consider the problem of efficient inference of the Average Treatment Effect in a sequential experiment where the policy governing the assignment of subjects to treatment or control can change over time. We first provide a central limit…

Machine Learning · Statistics 2024-03-05 Thomas Cook , Alan Mishler , Aaditya Ramdas

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

This paper provides a statistical method to test whether a system that performs a binary sequential hypothesis test is optimal in the sense of minimizing the average decision times while taking decisions with given reliabilities. The…

Information Theory · Computer Science 2018-01-08 Meik Dörpinghaus , Izaak Neri , Édgar Roldán , Heinrich Meyr , Frank Jülicher

Incorporating historical data or real-world evidence has a great potential to improve the efficiency of phase I clinical trials and to accelerate drug development. For model-based designs, such as the continuous reassessment method (CRM),…

Methodology · Statistics 2020-06-11 Yanhong Zhou , J. Jack Lee , Shunguang Wang , Stuart Bailey , Ying Yuan

Applied researchers in biomedicine and related fields are often interested in estimating the causal effect of a treatment or intervention. Although randomized clinical trials are considered the gold standard for establishing causal effects,…

Purpose: To identify the most informative methods for reporting results of treatment planning comparisons. Methods: Seven papers from the past year of International Journal of Radiation Oncology Biology Physics reported on comparisons of…

Medical Physics · Physics 2012-02-20 Mark Phillips , Clay Holdsworth

Post-randomization events, also known as intercurrent events, such as treatment noncompliance and censoring due to a terminal event, are common in clinical trials. Principal stratification is a framework for causal inference in the presence…

Methodology · Statistics 2023-01-19 Bo Liu , Lisa Wruck , Fan Li

This technical report includes an assortment of technical details and extended discussions related to paper "Should we sample a time series more frequently? Decision support via multirate spectrum estimation (with discussion)", which…

Methodology · Statistics 2016-04-25 Guy P. Nason , Ben Powell , Duncan Elliott , Paul A. Smith

Bridging the gap between internal and external validity is crucial for heterogeneous treatment effect estimation. Randomised controlled trials (RCTs), favoured for their internal validity due to randomisation, often encounter challenges in…

Test-time adaptation (TTA) is an emerging paradigm that addresses distributional shifts between training and testing phases without additional data acquisition or labeling cost; only unlabeled test data streams are used for continual model…

Machine Learning · Computer Science 2023-01-12 Taesik Gong , Jongheon Jeong , Taewon Kim , Yewon Kim , Jinwoo Shin , Sung-Ju Lee

This paper extends my research applying statistical decision theory to treatment choice with sample data, using maximum regret to evaluate the performance of treatment rules. The specific new contribution is to study as-if optimization…

Econometrics · Economics 2021-10-05 Charles F. Manski

As standards of care advance, patients are living longer and once-fatal diseases are becoming manageable. Clinical trials increasingly focus on reducing disease burden, which can be quantified by the timing and occurrence of multiple…

In this study, we consider sequences drawn from time-homogeneous Markov chains and introduce a novel approach for estimating first hitting-time distributions to specified terminal states. Our method- ology is based on the…

Statistics Theory · Mathematics 2026-05-19 Mikael Escobar-Bach , Alexandre Popier , Malo Sahin

Test-Time Adaptation (TTA) has recently emerged as a promising approach for tackling the robustness challenge under distribution shifts. However, the lack of consistent settings and systematic studies in prior literature hinders thorough…

Machine Learning · Computer Science 2023-06-07 Hao Zhao , Yuejiang Liu , Alexandre Alahi , Tao Lin

Automatically discovering personalized trajectories (i.e., sequential event patterns) from longitudinal EHR data is crucial for enabling precision medicine in clinical research, yet it remains a formidable challenge even for contemporary AI…

Machine Learning · Computer Science 2026-05-28 Jia Li , Yu Hou , Rui Zhang

In this commentary, we highlight the importance of: (1) carefully considering and clarifying whether a marginal or conditional treatment effect is of interest in a population-adjusted indirect treatment comparison; and (2) developing…

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

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…

The ICH E9(R1) addendum provides guidelines on accounting for intercurrent events in clinical trials using the estimands framework. However, there has been limited attention to the estimands framework for meta-analysis. Using treatment…

Clinical trials involving novel immuno-oncology (IO) therapies frequently exhibit survival profiles which violate the proportional hazards assumption due to a delay in treatment effect, and in such settings, the survival curves in the two…

Methodology · Statistics 2021-02-02 Nicholas C. Henderson , Kijoeng Nam , Dai Feng
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