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In this paper we study the problems of estimating heterogeneity in causal effects in experimental or observational studies and conducting inference about the magnitude of the differences in treatment effects across subsets of the…

Machine Learning · Statistics 2022-06-08 Susan Athey , Guido Imbens

In the fight against hard-to-treat diseases such as cancer, it is often difficult to discover new treatments that benefit all subjects. For regulatory agency approval, it is more practical to identify subgroups of subjects for whom the…

Methodology · Statistics 2014-10-09 Wei-Yin Loh , Xu He , Michael Man

The appearance of a new dangerous and contagious disease requires the development of a drug therapy faster than what is foreseen by usual mechanisms. Many drug therapy developments consist in investigating through different clinical trials…

Quantitative Methods · Quantitative Biology 2020-03-31 Ezequiel Alvarez , Federico Lamagna , Manuel Szewc

Pharmaceutical companies continue to seek innovative ways to explore whether a drug under development is likely to be suitable for all or only an identifiable stratum of patients in the target population. The sooner this can be done during…

Methodology · Statistics 2019-12-10 Thomas O. Jemielita , Devan V. Mehrotra

Precision oncology aims to prescribe the optimal cancer treatment to the right patients, maximizing therapeutic benefits. However, identifying patient subgroups that may benefit more from experimental cancer treatments based on randomized…

Methodology · Statistics 2026-01-06 Xingyu Li , Qing Liu , Tony Jiang , Hong Amy Xia , Peng Wei , Brian P. Hobbs

We propose a multi-threshold change plane regression model which naturally partitions the observed subjects into subgroups with different covariate effects. The underlying grouping variable is a linear function of covariates and thus…

Methodology · Statistics 2018-08-03 Jialiang Li , Yaguang Li , Baisuo Jin

Clinical trials are an instrument for making informed decisions based on evidence from well-designed experiments. Here we consider adaptive designs mainly from the perspective of multi-arm Phase II clinical trials, in which one or more…

Methodology · Statistics 2021-08-31 Elja Arjas , Dario Gasbarra

In medical research, it is often needed to obtain subgroups with heterogeneous survivals, which have been predicted from a prognostic factor. For this purpose, a binary split has often been used once or recursively; however, binary…

Applications · Statistics 2014-11-04 Soo-Heang Eo , Hyo Jeong Kang , Seung-Mo Hong , HyungJun Cho

In a Phase II dose-finding study with a placebo control, a new drug with several dose levels is compared with a placebo to test for the effectiveness of the new drug. The main focus of such studies often lies in the characterization of the…

Methodology · Statistics 2020-07-14 Saswati Saha , Werner Brannath

Precise estimation of treatment effects is crucial for evaluating intervention effectiveness. While deep learning models have exhibited promising performance in learning counterfactual representations for treatment effect estimation (TEE),…

Machine Learning · Computer Science 2024-01-24 Seungyeon Lee , Ruoqi Liu , Wenyu Song , Lang Li , Ping Zhang

We consider applying multi-armed bandits to model-assisted designs for dose-finding clinical trials. Multi-armed bandits are very simple and powerful methods to determine actions to maximize a reward in a limited number of trials. Among the…

Methodology · Statistics 2022-01-17 Masahiro Kojima

Subgroup analysis is a frequently used tool for evaluating heterogeneity of treatment effect and heterogeneity in treatment harm across observed baseline patient characteristics. While treatment efficacy and adverse event measures are often…

Applications · Statistics 2018-08-14 Nicholas C. Henderson , Ravi Varadhan

Partitioning a set of elements into an unknown number of mutually exclusive subsets is essential in many machine learning problems. However, assigning elements, such as samples in a dataset or neurons in a network layer, to an unknown and…

Machine Learning · Computer Science 2023-11-10 Thomas M. Sutter , Alain Ryser , Joram Liebeskind , Julia E. Vogt

A common problem in Phase II clinical trials is the comparison of dose response curves corresponding to different treatment groups. If the effect of the dose level is described by parametric regression models and the treatments differ in…

Statistics Theory · Mathematics 2016-03-16 Chrystel Feller , Kirsten Schorning , Holger Dette , Georgina Bermann , Björn Bornkamp

Identifying and making statistical inferences on differential treatment effects (commonly known as subgroup analysis in clinical research) is central to precision health. Subgroup analysis allows practitioners to pinpoint populations for…

Machine Learning · Statistics 2026-02-05 Zhongming Xie , Joseph Giorgio , Jingshen Wang

A key challenge in sequential decision making is optimizing systems safely under partial information. While much of the literature has focused on the cases of either partially known states or partially known dynamics, it is further…

Optimization and Control · Mathematics 2023-04-21 Qinyang He , Yonatan Mintz

Dose-finding studies are frequently conducted to evaluate the effect of different doses or concentration levels of a compound on a response of interest. Applications include the investigation of a new medicinal drug, a herbicide or…

Applications · Statistics 2011-08-01 Björn Bornkamp , Frank Bretz , Holger Dette , José Pinheiro

Many phase II clinical trials have used survival outcomes as the primary endpoints in recent decades. Suppose the radiotherapy is evaluated in a phase II trial using survival outcomes. In that case, the competing risk issue often arises…

Applications · Statistics 2022-03-15 Jina Park , Wenjing Hu , Ick Hoon Jin , Hao Liu , Yong Zang

In modern drug development, the broader availability of high-dimensional observational data provides opportunities for scientist to explore subgroup heterogeneity, especially when randomized clinical trials are unavailable due to cost and…

Methodology · Statistics 2021-02-24 Xinzhou Guo , Linqing Wei , Chong Wu , Jingshen Wang

We consider the optimal design problem for identifying effective dose combinations within drug combination studies where the effect of the combination of two drugs is investigated. Drug combination studies are becoming increasingly…