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Precision medicine promises to transform health care by offering individualised treatments that dramatically improve clinical outcomes. A necessary prerequisite is to identify subgroups of patients who respond differently to different…

Machine Learning · Computer Science 2026-03-03 Adam Marcus , Paul Agapow

Individualizing treatment assignment can improve outcomes for diseases with patient-to-patient variability in comparative treatment effects. When a clinical trial demonstrates that some patients improve on treatment while others do not, it…

Methodology · Statistics 2022-11-02 Nina Galanter , Marco Carone , Ronald C. Kessler , Alex Luedtke

Identifying the mutations that drive cancer growth is key in clinical decision making and precision oncology. As driver mutations confer selective advantage and thus have an increased likelihood of occurrence, frequency-based statistical…

Genomics · Quantitative Biology 2021-05-04 Adnan Akbar , Andrey Solovyev , John W Cassidy , Nirmesh Patel , Harry W Clifford

Despite the availability of large amounts of genomics data, medical treatment recommendations have not successfully used them. In this paper, we consider the utility of high dimensional genomic-clinical data and nonparametric methods for…

Methodology · Statistics 2022-02-22 Zhongyuan Chen , Ziyi Wang , Qifan Song , Jun Xie

We develop and evaluate tolerance interval methods for dynamic treatment regimes (DTRs) that can provide more detailed prognostic information to patients who will follow an estimated optimal regime. Although the problem of constructing…

Methodology · Statistics 2017-04-26 Daniel J. Lizotte , Arezoo Tahmasebi

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

Causal machine learning (ML) offers flexible, data-driven methods for predicting treatment outcomes including efficacy and toxicity, thereby supporting the assessment and safety of drugs. A key benefit of causal ML is that it allows for…

The field of precision medicine aims to tailor treatment based on patient-specific factors in a reproducible way. To this end, estimating an optimal individualized treatment regime (ITR) that recommends treatment decisions based on patient…

Confidence intervals (CIs) are instrumental in statistical analysis, providing a range estimate of the parameters. In modern statistics, selective inference is common, where only certain parameters are highlighted. However, this selective…

Methodology · Statistics 2025-09-17 Tzviel Frostig , Yoav Benjamini , Ruth Heller

Heterogeneous treatment effects can be very important in the analysis of randomized clinical trials. Heightened risks or enhanced benefits may exist for particular subsets of study subjects. When the heterogeneous treatment effects are…

Methodology · Statistics 2025-07-25 Richard A. Berk , Matthew Olson , Andreas Buja , Aurelie Ouss

Estimating individualized treatment rules (ITRs) is crucial for tailoring interventions in precision medicine. Typical ITR estimation methods rely on conditional average treatment effects (CATEs) to guide treatment assignments. However,…

Methodology · Statistics 2025-10-20 Peng Wu , Qing Jiang , Shanshan Luo , Zhi Geng

When making treatment selection decisions, it is essential to include a causal effect estimation analysis to compare potential outcomes under different treatments or controls, assisting in optimal selection. However, merely estimating…

Machine Learning · Statistics 2024-10-08 Sherly Alfonso-Sánchez , Kristina P. Sendova , Cristián Bravo

Treatment effect estimation is essential for informed decision-making in many fields such as healthcare, economics, and public policy. While flexible machine learning models have been widely applied for estimating heterogeneous treatment…

Machine Learning · Computer Science 2025-09-29 Pascal Memmesheimer , Vincent Heuveline , Jürgen Hesser

Contrastive dimension reduction (CDR) methods aim to extract signal unique to or enriched in a treatment (foreground) group relative to a control (background) group. This setting arises in many scientific domains, such as genomics, imaging,…

Methodology · Statistics 2025-10-15 Sam Hawke , Eric Zhang , Jiawen Chen , Didong Li

The causal effect of a treatment can vary from person to person based on their individual characteristics and predispositions. Mining for patterns of individual-level effect differences, a problem known as heterogeneous treatment effect…

Machine Learning · Computer Science 2019-09-04 Christopher Tran , Elena Zheleva

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

Decision curve analysis can be used to determine whether a personalized model for treatment benefit would lead to better clinical decisions. Decision curve analysis methods have been described to estimate treatment benefit using data from a…

We study the problem of learning to choose from m discrete treatment options (e.g., news item or medical drug) the one with best causal effect for a particular instance (e.g., user or patient) where the training data consists of passive…

Machine Learning · Statistics 2017-08-02 Nathan Kallus

In most medical research, the average treatment effect is used to evaluate a treatment's performance. However, precision medicine requires knowledge of individual treatment effects: What is the difference between a unit's measurement under…

Computation · Statistics 2022-08-30 Mingyang Cai , Stef van Buuren , Gerko Vink

Conformal methods provide prediction sets for outcomes with confidence guarantees. We study their use in a selective inference setting, where inference is performed only when the prediction set is informative. The analyst may consider as…

Methodology · Statistics 2026-05-22 Israela Solomon , Etienne Roquain , Saharon Rosset , Ruth Heller