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Precision medicine has led to a paradigm shift allowing the development of targeted drugs that are agnostic to the tumor location. In this context, basket trials aim to identify which tumor types - or baskets - would benefit from the…

Methodology · Statistics 2026-01-05 Marcio A. Diniz , Hulya Kocyigit , Erin Moshier , Madhu Mazumdar , Deukwoo Kwon

Dealing with distribution shifts is one of the central challenges for modern machine learning. One fundamental situation is the covariate shift, where the input distributions of data change from training to testing stages while the…

Machine Learning · Computer Science 2024-05-28 Yu-Jie Zhang , Zhen-Yu Zhang , Peng Zhao , Masashi Sugiyama

Monitoring time between events (TBE) is a critical task in industrial settings. Traditional Statistical Process Monitoring (SPM) methods often assume that TBE variables follow an exponential distribution, which implies a constant failure…

Methodology · Statistics 2025-01-22 Hussam Ahmad , Adel Ahmadi Nadi , Mohammad Amini , Subhabrata Chakraborti

Causal inference with time-to-event outcomes is fundamental in various scientific studies. In a static setup with fitted propensity scores, weighted Kaplan-Meier estimation for survival probabilities and weighted Breslow-Peto estimation for…

Methodology · Statistics 2026-05-18 Wenfu Xu , Yi Zhang , Tobias Gerhard , Zhiqiang Tan

We revisit the problem of estimating the local average treatment effect (LATE) and the local average treatment effect on the treated (LATT) when control variables are available, either to render the instrumental variable (IV) suitably…

Econometrics · Economics 2022-11-16 Tymon Słoczyński , S. Derya Uysal , Jeffrey M. Wooldridge

While randomized trials may be the gold standard for evaluating the effectiveness of the treatment intervention, in some special circumstances, single-arm clinical trials utilizing external control may be considered. The causal treatment…

Methodology · Statistics 2025-05-26 Huan Wang , Fei Wu , Yeh-Fong Chen

In epidemiology, identifying subpopulations that are particularly vulnerable to exposures and those who may benefit differently from exposure-reducing interventions is essential. Factors such as age, gender-specific vulnerabilities, and…

Methodology · Statistics 2024-12-11 David McCoy , Wenxin Zhang , Alan Hubbard , Mark van der Laan , Alejandro Schuler

What can be considered an appropriate statistical method for the primary analysis of a randomized clinical trial (RCT) with a time-to-event endpoint when we anticipate non-proportional hazards owing to a delayed effect? This question has…

Methodology · Statistics 2023-04-18 José L. Jiménez , Isobel Barrott , Francesca Gasperoni , Dominic Magirr

Two-phase sampling is a simple and cost-effective estimation strategy in survey sampling and is widely used in practice. Because the phase-2 sampling probability typically depends on low-cost variables collected at phase 1, naive estimation…

Methodology · Statistics 2025-11-11 Kazuharu Harada , Masataka Taguri

Pretraining on large-scale datasets has been shown to improve transformer generalizability, even for out-of-domain (OOD) modalities and tasks. However, two common assumptions often fail under OOD transfer: that downstream datasets can be…

Individualized treatment regimes (ITRs) aim to improve clinical outcomes by assigning treatment based on patient-specific characteristics. However, existing methods often struggle with high-dimensional covariates, limiting accuracy,…

Machine Learning · Statistics 2026-01-13 Sungtaek Son , Eardi Lila , Kwun Chuen Gary Chan

Marginal structural models (MSMs) with inverse probability weighting offer an approach to estimating causal effects of treatment sequences on repeated outcome measures in the presence of time-varying confounding and dependent censoring.…

Methodology · Statistics 2018-07-02 Sean Yiu , Li Su

Dose-finding trials for oncology studies are traditionally designed to assess safety in the early stages of drug development. With the rise of molecularly targeted therapies and immuno-oncology compounds, biomarker-driven approaches have…

Methodology · Statistics 2025-09-15 Xijin Chen , Pavel Mozgunov , Richard D. Baird , Thomas Jaki

Test-time adaptation (TTA) seeks to tackle potential distribution shifts between training and test data by adapting a given model w.r.t. any test sample. Although recent TTA has shown promising performance, we still face two key challenges:…

Machine Learning · Computer Science 2025-08-27 Mingkui Tan , Guohao Chen , Jiaxiang Wu , Yifan Zhang , Yaofo Chen , Peilin Zhao , Shuaicheng Niu

To use historical controls for indirect comparison with single-arm trials, the population difference between data sources should be adjusted to reduce confounding bias. The adjustment is more difficult for time-to-event data with a cure…

Methodology · Statistics 2023-08-23 Jixian Wang , Hongtao Zhang , Ram Tiwari

The primary objective of phase I cancer clinical trials is to evaluate the safety of a new experimental treatment and to find the maximum tolerated dose (MTD). We show that the MTD estimation problem can be regarded as a level set…

Machine Learning · Statistics 2025-04-15 Keiichiro Seno , Kota Matsui , Shogo Iwazaki , Yu Inatsu , Shion Takeno , Shigeyuki Matsui

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

This paper considers robust modeling of the survival time for cancer patients. Accurate prediction can be helpful for developing therapeutic and care strategies. We propose a unified Expectation-Maximization approach combined with the…

Methodology · Statistics 2019-12-23 Yi Li , Muxuan Liang , Lu Mao , Sijian Wang

We propose a novel multi-task neural network approach for estimating distributional treatment effects (DTE) in randomized experiments. While DTE provides more granular insights into the experiment outcomes over conventional methods focusing…

Machine Learning · Computer Science 2025-07-11 Tomu Hirata , Undral Byambadalai , Tatsushi Oka , Shota Yasui , Shingo Uto

In realistic scenarios, multivariate timeseries evolve over case-by-case time-scales. This is particularly clear in medicine, where the rate of clinical events varies by ward, patient, and application. Increasingly complex models have been…

Machine Learning · Computer Science 2020-03-06 Jacob Deasy , Ari Ercole , Pietro Liò