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We consider the problem of estimating a dose-response curve. Continuous treatments arise often in practice, e.g. in the form of time spent on an operation, distance traveled to a location or dosage of a drug. Letting $A$ denote a continuous…

Methodology · Statistics 2026-04-14 Matteo Bonvini , Edward H. Kennedy

Designing compounds with desired properties is a key element of the drug discovery process. However, measuring progress in the field has been challenging due to the lack of realistic retrospective benchmarks, and the large cost of…

Biomolecules · Quantitative Biology 2023-06-16 Tobiasz Cieplinski , Tomasz Danel , Sabina Podlewska , Stanislaw Jastrzebski

Currently, knowledge discovery in databases is an essential step to identify valid, novel and useful patterns for decision making. There are many real-world scenarios, such as bankruptcy prediction, option pricing or medical diagnosis,…

Artificial Intelligence · Computer Science 2018-11-20 José-Ramón Cano , Pedro Antonio Gutiérrez , Bartosz Krawczyk , Michał Woźniak , Salvador García

No unmeasured confounding is a common assumption when reasoning about counterfactual outcomes, but such an assumption may not be plausible in observational studies. Sensitivity analysis is often employed to assess the robustness of causal…

Methodology · Statistics 2025-08-20 Abhinandan Dalal , Eric J. Tchetgen Tchetgen

We propose a new modeling and estimation approach to select the optimal treatment regime from different options through constructing a robust estimating equation. The method is protected against misspecification of the propensity score…

Methodology · Statistics 2022-11-15 Trinetri Ghosh , Yanyuan Ma , Wensheng Zhu , Yuanjia Wang

We study the problem of finding the optimal dosage in early stage clinical trials through the multi-armed bandit lens. We advocate the use of the Thompson Sampling principle, a flexible algorithm that can accommodate different types of…

Machine Learning · Statistics 2020-04-09 Maryam Aziz , Emilie Kaufmann , Marie-Karelle Riviere

Large language model (LLM) evaluation is increasingly costly, prompting interest in methods that speed up evaluation by shrinking benchmark datasets. Benchmark prediction (also called efficient LLM evaluation) aims to select a small subset…

Machine Learning · Computer Science 2025-06-10 Guanhua Zhang , Florian E. Dorner , Moritz Hardt

Model-assisted designs have garnered significant attention in recent years due to their high accuracy in identifying the maximum tolerated dose (MTD) and their operational simplicity. To identify the MTD, they employ estimated dose limiting…

Applications · Statistics 2025-08-19 Rentaro Wakayama , Tomotaka Momozaki , Shuji Ando

Scharfstein et al. (2021) developed a sensitivity analysis model for analyzing randomized trials with repeatedly measured binary outcomes that are subject to nonmonotone missingness. Their approach becomes computationally intractable when…

This paper focuses on the Bayesian Network Propensity Score (BNPS), a novel approach for estimating treatment effects in observational studies characterized by unknown (and likely unbalanced) designs and complex dependency structures among…

Randomised field experiments, such as A/B testing, have long been the gold standard for evaluating the value that new software brings to customers. However, running randomised field experiments is not always desired, possible or even…

Software Engineering · Computer Science 2022-07-04 Yuchu Liu , David Issa Mattos , Jan Bosch , Helena Holmström Olsson , Jonn Lantz

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

In observational studies, potential confounders may distort the causal relationship between an exposure and an outcome. However, under some conditions, a causal dose-response curve can be recovered using the G-computation formula. Most…

Methodology · Statistics 2019-12-18 Ted Westling , Peter Gilbert , Marco Carone

Label noise is a common problem in real-world datasets, affecting both model training and validation. Clean data are essential for achieving strong performance and ensuring reliable evaluation. While various techniques have been proposed to…

Machine Learning · Computer Science 2025-10-21 Henrique Pickler , Jorge K. S. Kamassury , Danilo Silva

We propose an adaptive design for early phase drug combination cancer trials with the goal of estimating the maximum tolerated dose (MTD). A nonparametric Bayesian model, using beta priors truncated to the set of partially ordered dose…

Applications · Statistics 2019-10-22 Zahra S. Razaee , Galen Wien-Cook , Mourad Tighiouart

We propose new parametric frameworks of regression analysis with the conditional mode of a bounded response as the focal point of interest. Covariate effects estimation and prediction based on the maximum likelihood method under two new…

Methodology · Statistics 2020-06-22 Haiming Zhou , Xianzheng Huang

Nonlinear regression models addressing both efficacy and toxicity outcomes are increasingly used in dose-finding trials, such as in pharmaceutical drug development. However, research on related experimental design problems for corresponding…

Methodology · Statistics 2016-01-06 Holger Dette , Katrin Kettelhake , Kirsten Schorning , Weng Kee Wong , Frank Bretz

We propose a one-to-many matching estimator of the average treatment effect based on propensity scores estimated by isotonic regression. This approach is predicated on the assumption of monotonicity in the propensity score function, a…

Econometrics · Economics 2025-01-27 Mengshan Xu , Taisuke Otsu

Semiparametric regression offers a flexible framework for modeling non-linear relationships between a response and covariates. A prime example are generalized additive models where splines (say) are used to approximate non-linear functional…

Statistics Theory · Mathematics 2018-10-05 Francis K. C. Hui , Chong You , Han Lin Shang , Samuel Müller

Successful pharmaceutical drug development requires finding correct doses that provide an optimum balance between efficacy and toxicity. Competing responses to dose such as efficacy and toxicity often will increase with dose, and it is…

Applications · Statistics 2024-01-26 A. Lawrence Gould