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A new matching method is proposed for the estimation of the average treatment effect of social policy interventions (e.g., training programs or health care measures). Given an outcome variable, a treatment and a set of pre-treatment…

Statistics Theory · Mathematics 2007-06-13 Stefano Iacus , Giuseppe Porro

Combination drug therapies hold significant promise for enhancing treatment efficacy, particularly in fields such as oncology, immunotherapy, and infectious diseases. However, designing clinical trials for these regimens poses unique…

Methodology · Statistics 2025-06-04 Nan Miles Xi , Man Mandy Jin , Lin Wang , Xin Huang

In biomedical Subgroup Discovery, practitioners are interested in discovering interpretable and homogeneous subgroups within a group of patients. In this paper, assuming that healthy subjects (i.e., controls) share common but irrelevant…

Machine Learning · Computer Science 2026-05-21 Robin Louiset , Edouard Duchesnay , Benoit Dufumier , Antoine Grigis , Pietro Gori

Platform trials have gained a lot of attention recently as a possible remedy for time-consuming classical two-arm randomized controlled trials, especially in early phase drug development. This short article illustrates how to use the…

This paper develops a unified framework for partial identification and inference in stratified experiments with attrition, accommodating both equal and heterogeneous treatment shares across strata. For equal-share designs, we apply recent…

Econometrics · Economics 2026-01-21 Bruno Ferman , Davi Siqueira , Vitor Possebom

In many clinical trials, individuals in different subgroups have experience differential treatment effects. This leads to individualized differences in treatment benefit. In this article, we introduce the general concept of predictive…

Methodology · Statistics 2018-07-11 Debashis Ghosh , Youngjoo Cho

Rerandomization is an experimental design technique that repeatedly randomizes treatment assignments until covariates are balanced between treatment groups. Rerandomization in the design stage of an experiment can lead to many asymptotic…

Methodology · Statistics 2026-04-10 Antônio Carlos Herling Ribeiro Junior

Phase I early-phase clinical studies aim at investigating the safety and the underlying dose-toxicity relationship of a drug or combination. While little may still be known about the compound's properties, it is crucial to consider…

Methodology · Statistics 2022-09-13 Christian Röver , Moreno Ursino , Tim Friede , Sarah Zohar

We propose a frequentist adaptive phase 2 trial design to evaluate the safety and efficacy of three treatment regimens (doses) compared to placebo for four types of helminth (worm) infections. This trial will be carried out in four…

In big data analysis, a simple task such as linear regression can become very challenging as the variable dimension $p$ grows. As a result, variable screening is inevitable in many scientific studies. In recent years, randomized algorithms…

Methodology · Statistics 2019-02-13 Yu-Hsiang Cheng , Tzee-Ming Huang , Su-Yun Huang

The identification of predictive biomarkers from a large scale of covariates for subgroup analysis has attracted fundamental attention in medical research. In this article, we propose a generalized penalized regression method with a novel…

Methodology · Statistics 2019-04-29 Chong Ma , Wenxuan Deng , Shuangge Ma , Ray Liu , Kevin Galinsky

This paper addresses the problem of deciding whether the dose response relationships between subgroups and the full population in a multi-regional trial are similar to each other. Similarity is measured in terms of the maximal deviation…

Statistics Theory · Mathematics 2024-04-30 Holger Dette , Lukas Koletzko , Frank Bretz

Part-based representation has been proven to be effective for a variety of visual applications. However, automatic discovery of discriminative parts without object/part-level annotations is challenging. This paper proposes a discriminative…

Computer Vision and Pattern Recognition · Computer Science 2017-05-30 Xiaopeng Zhang , Hongkai Xiong , Weiyao Lin , Qi Tian

This paper presents Sparse Partitioning, a Bayesian method for identifying predictors that either individually or in combination with others affect a response variable. The method is designed for regression problems involving binary or…

Quantitative Methods · Quantitative Biology 2011-08-31 Doug Speed , Simon Tavaré

Drug combination trials are increasingly common nowadays in clinical research. However, very few methods have been developed to consider toxicity attributions in the dose escalation process. We are motivated by a trial in which the…

Methodology · Statistics 2018-08-23 Jose L. Jimenez , Mourad Tighiouart , Mauro Gasparini

Diffusion models have emerged as a leading framework in generative modeling, poised to transform the traditionally slow and costly process of drug discovery. This review provides a systematic comparison of their application in designing two…

Machine Learning · Computer Science 2025-11-27 Yiquan Wang , Yahui Ma , Yuhan Chang , Jiayao Yan , Jialin Zhang , Minnuo Cai , Kai Wei

We consider design issues for toxicology studies when we have a continuous response and the true mean response is only known to be a member of a class of nested models. This class of non-linear models was proposed by toxicologists who were…

Statistics Theory · Mathematics 2010-11-29 Holger Dette , Andrey Pepelyshev , Piter Shpilev , Weng Kee Wong

An important objective in the development of targeted therapies is to identify the populations where the treatment under consideration has positive benefit risk balance. We consider pivotal clinical trials, where the efficacy of a treatment…

The concept of personalised medicine in cancer therapy is becoming increasingly important. There already exist drugs administered specifically for patients with tumours presenting well-defined mutations. However, the field is still in its…

Biomolecules · Quantitative Biology 2024-08-26 Abbi Abdel-Rehim , Oghenejokpeme Orhobor , Gareth Griffiths , Larisa Soldatova , Ross D. King

It is recognised that treatment-related clustering should be allowed for in the sample size and analyses of individually-randomised parallel-group trials that evaluate therapist-delivered interventions such as psychotherapy. Here,…

Methodology · Statistics 2026-02-03 Rebecca EA Walwyn , Rosemary A Bailey , Arpan Singh , Neil Corrigan , Steven G Gilmour