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The current work is motivated by the need for robust statistical methods for precision medicine; as such, we address the need for statistical methods that provide actionable inference for a single unit at any point in time. We aim to learn…

Statistics Theory · Mathematics 2021-07-02 Ivana Malenica , Aurelien Bibaut , Mark J. van der Laan

This paper focuses on the problem of modeling and estimating interaction effects between covariates and a continuous treatment variable on an outcome, using a single-index regression approach. The primary motivation is to estimate an…

Methodology · Statistics 2021-02-02 Hyung Park , Eva Petkova , Thaddeus Tarpey , R. Todd Ogden

The ability to predict individualized treatment effects (ITEs) based on a given patient's profile is essential for personalized medicine. We propose a hypothesis testing approach to choosing between two potential treatments for a given…

Methodology · Statistics 2020-08-11 Tianxi Cai , Tony Cai , Zijian Guo

Single-subject health data are becoming increasingly available thanks to advances in self-tracking technology (e.g., wearable devices, mobile apps, sensors, implants). Many users and health caregivers seek to use such observational time…

Applications · Statistics 2019-01-23 Eric Jay Daza

Meta-analysis is a well-established tool used to combine data from several independent studies, each of which usually compares the effect of an experimental treatment with a control group. While meta-analyses are often performed using…

Methodology · Statistics 2025-06-02 Phuc Thien Tran , Long-Hao Xu , Christian Röver , Tim Friede

The primary analysis in two-arm clinical trials usually involves inference on a scalar treatment effect parameter; e.g., depending on the outcome, the difference of treatment-specific means, risk difference, risk ratio, or odds ratio. Most…

Methodology · Statistics 2022-04-25 Anastasios A. Tsiatis , Marie Davidian

Current methods for pattern analysis in time series mainly rely on statistical features or probabilistic learning and inference methods to identify patterns and trends in the data. Such methods do not generalize well when applied to…

Artificial Intelligence · Computer Science 2023-05-01 Yushan Huang , Yuchen Zhao , Alexander Capstick , Francesca Palermo , Hamed Haddadi , Payam Barnaghi

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

Tests for paired censored outcomes have been extensively studied, with some justified in the context of randomization-based inference. These tests are primarily designed to detect an overall treatment effect across the entire follow-up…

Methodology · Statistics 2025-06-10 Sangjin Lee , Kwonsang Lee

Difference-in-differences is based on a parallel trends assumption, which states that changes over time in average potential outcomes are independent of treatment assignment, possibly conditional on covariates. With time-varying treatments,…

Methodology · Statistics 2024-06-25 Nicholas Illenberger , Iván Díaz , Audrey Renson

Measuring the effect of patient safety improvement efforts is needed to determine their value but is difficult due to the inherent complexities of hospital operations. In this paper, we show by case study how interrupted time series design…

Applications · Statistics 2018-06-28 Diego A. Martinez , Mehdi Jalalpour , David T. Efron , Scott R. Levin

Time-series experiments, also called switchback experiments or N-of-1 trials, play increasingly important roles in modern applications in medical and industrial areas. Under the potential outcomes framework, recent research has studied…

Methodology · Statistics 2025-10-28 Zhexiao Lin , Peng Ding

This paper proposes a practical yet novel solution to a longstanding statistical testing problem regarding single subject design. In particular, we aim to resolve an important clinical question: does a new patient behave the same as one…

Applications · Statistics 2016-02-12 Ying Lu , Marc Scott , Preeti Raghavan

The delivery and assessment of quality health care is complex with many interacting and interdependent components. In terms of research design and statistical analysis, this complexity and interdependency makes it difficult to assess the…

Methodology · Statistics 2018-12-03 Maricela Cruz , Daniel L. Gillen , Miriam Bender , Hernando Ombao

Based on technological advances in sensing modalities, randomized trials with primary outcomes represented as high-dimensional vectors have become increasingly prevalent. For example, these outcomes could be week-long time-series data from…

Methodology · Statistics 2024-10-16 Yujin Jeong , Emily Fox , Ramesh Johari

We consider the problem of efficient inference of the Average Treatment Effect in a sequential experiment where the policy governing the assignment of subjects to treatment or control can change over time. We first provide a central limit…

Machine Learning · Statistics 2024-03-05 Thomas Cook , Alan Mishler , Aaditya Ramdas

We provide an approach to exploratory data analysis in matched observational studies with a single intervention and multiple endpoints. In such settings, the researcher would like to explore evidence for actual treatment effects among these…

Methodology · Statistics 2025-12-10 Mengqi Lin , Colin Fogarty

Estimation and evaluation of individualized treatment rules have been studied extensively, but real-world treatment resource constraints have received limited attention in existing methods. We investigate a setting in which treatment is…

Methodology · Statistics 2022-11-24 Hongxiang Qiu , Marco Carone , Alex Luedtke

We present a computational evaluation of three hypotheses about sources of deficit in sentence comprehension in aphasia: slowed processing, intermittent deficiency, and resource reduction. The ACT-R based Lewis and Vasishth (2005) model is…

Computation and Language · Computer Science 2017-06-01 Paul Mätzig , Shravan Vasishth , Felix Engelmann , David Caplan

Targeted maximum likelihood estimation is a general methodology combining flexible ensemble learning and semiparametric efficiency theory in a two-step procedure for estimation of causal parameters. Proposed targeted maximum likelihood…

Methodology · Statistics 2021-06-22 Helene Charlotte Wiese Rytgaard , Frank Eriksson , Mark van der Laan
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