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Related papers: Interpretable Subgroup Discovery in Treatment Effe…

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Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

Methodology · Statistics 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

Identifying subgroups, which respond differently to a treatment, both in terms of efficacy and safety, is an important part of drug development. A well-known challenge in exploratory subgroup analyses is the small sample size in the…

Computation · Statistics 2016-06-28 Marius Thomas , Björn Bornkamp

Deep learning models have demonstrated promising results in estimating treatment effects (TEE). However, most of them overlook the variations in treatment outcomes among subgroups with distinct characteristics. This limitation hinders their…

Machine Learning · Computer Science 2024-01-31 Seungyeon Lee , Ruoqi Liu , Wenyu Song , Ping Zhang

We introduce an algorithm for identifying interpretable subgroups with elevated treatment effects, given an estimate of individual or conditional average treatment effects (CATE). Subgroups are characterized by ``rule sets'' --…

Machine Learning · Statistics 2025-07-15 Albert Chiu

Recent work has made important contributions in the development of causally-interpretable meta-analysis. These methods transport treatment effects estimated in a collection of randomized trials to a target population of interest. Ideally,…

Methodology · Statistics 2023-02-08 Justin M. Clark , Kollin W. Rott , James S. Hodges , Jared D. Huling

Opioid addiction has become a global epidemic and a national health crisis in recent years, with the number of opioid overdose fatalities steadily increasing since the 1990s. In contrast to the dynamics of a typical illicit drug or disease…

Populations and Evolution · Quantitative Biology 2019-05-03 Nicholas A. Battista , Leigh B. Pearcy , W. Christopher Strickland

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

The opioid epidemic in the United States claims over 40,000 lives per year, and it is estimated that well over two million Americans have an opioid use disorder. Over-prescription and misuse of prescription opioids play an important role in…

Quantitative Methods · Quantitative Biology 2019-05-03 Jen J. Gong , Abigail Z. Jacobs , Toby E. Stuart , Mathijs de Vaan

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

In this study, we present a novel clinical decision support system and discuss its interpretability-related properties. It combines a decision set of rules with a machine learning scheme to offer global and local interpretability. More…

Methodology · Statistics 2021-07-16 Francisco Valente , Simão Paredes , Jorge Henriques

Meta-analysis is commonly used to combine results from multiple clinical trials, but traditional meta-analysis methods do not refer explicitly to a population of individuals to whom the results apply and it is not clear how to use their…

Biomarker measurements can be relatively easy and quick to obtain and they are useful to investigate whether a compound works as intended on a mechanistic, pharmacological level. In some situations, it is realistic to assume that patients,…

Methodology · Statistics 2018-06-26 Björn Bornkamp , Georgina Bermann

Assessing causal effects in the presence of unobserved confounding is a challenging problem. Existing studies leveraged proxy variables or multiple treatments to adjust for the confounding bias. In particular, the latter approach attributes…

Methodology · Statistics 2023-10-17 Yong Wu , Mingzhou Liu , Jing Yan , Yanwei Fu , Shouyan Wang , Yizhou Wang , Xinwei Sun

Machine learning has shown much promise in helping improve the quality of medical, legal, and financial decision-making. In these applications, machine learning models must satisfy two important criteria: (i) they must be causal, since the…

Machine Learning · Computer Science 2021-10-12 Carolyn Kim , Osbert Bastani

The call for patient-focused drug development is loud and clear, as expressed in the 21st Century Cures Act and in recent guidelines and initiatives of regulatory agencies. Among the factors contributing to modernized drug development and…

Estimating heterogeneous treatment effect (HTE) for survival outcomes has gained increasing attention, as it captures the variation in treatment efficacy across patients or subgroups in delaying disease progression. However, most existing…

Methodology · Statistics 2025-11-27 Na Bo , Ying Ding

It has recently become popular to define treatment effects for subsets of the target population characterized by variables not observable at the time a treatment decision is made. Characterizing and estimating such treatment effects is…

Statistics Theory · Mathematics 2007-08-30 Marshall M. Joffe , Dylan Small , Chi-Yuan Hsu

Understanding prescription patterns have relied largely on aggregate statistical measures. Evidence of doctor-shopping, inappropriate prescribing, drug diversion and patient seeking prescription drugs across multiple prescribers demand…

Applications · Statistics 2018-11-27 Radhakrishnan Nagarajan , Jeffery Talbert

Machine-learning models are ubiquitous. In some domains, for instance, in medicine, the models' predictions must be interpretable. Decision trees, classification rules, and subgroup discovery are three broad categories of supervised…

Machine Learning · Computer Science 2022-04-29 Vadim Arzamasov , Benjamin Jochum , Klemens Böhm

A key issue for all observational causal inference is that it relies on an unverifiable assumption - that observed characteristics are sufficient to proxy for treatment confounding. In this paper we argue that in medical cases these…

Applications · Statistics 2023-03-14 Alexander Peysakhovich , Yin Aphinyanaphongs
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