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Platform trials are multi-arm designs that simultaneously evaluate multiple treatments for a single disease within the same overall trial structure. Unlike traditional randomized controlled trials, they allow treatment arms to enter and…

Methodology · Statistics 2024-12-16 Michele Santacatterina , Federico Macchiavelli Giron , Xinyi Zhang , Ivan Diaz

In clinical trials, response-adaptive randomization (RAR) has the appealing ability to assign more subjects to better-performing treatments based on interim results. The traditional RAR strategy alters the randomization ratio on a…

Methodology · Statistics 2021-10-01 David Merrell , Thevaa Chandereng , Yeonhee Park

Machine learning is increasingly used to select which individuals receive limited-resource interventions in domains such as human services, education, development, and more. However, it is often not apparent what the right quantity is for…

Machine Learning · Computer Science 2025-03-20 Vibhhu Sharma , Bryan Wilder

Randomization is a common technique used in clinical trials to eliminate potential bias and confounders in a patient population. Equal allocation to treatment groups is the standard due to its optimal efficiency in many cases. However, in…

Applications · Statistics 2020-04-09 Thevaa Chandereng , Xiaodan Wei , Rick Chappell

We investigate experimental design for randomized controlled trials (RCTs) with both equal and unequal treatment-control assignment probabilities. Our work makes progress on the connection between the distributional discrepancy minimization…

Methodology · Statistics 2025-03-20 Anup B. Rao , Peng Zhang

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

In placebo-controlled randomized trials, the post-randomization use of concomitant medications may be higher in the placebo arm than in the treatment arm. This may dilute the full benefits of the randomized drug as estimated by the…

Modern multi-centre randomized controlled trials (MCRCTs) collect massive amounts of tabular data, and are monitored intensively for irregularities by humans. We began by empirically evaluating 6 modern machine learning-based outlier…

Machine Learning · Computer Science 2023-11-10 Walter Nelson , Jonathan Ranisau , Jeremy Petch

Benkeser et al. demonstrate how adjustment for baseline covariates in randomized trials can meaningfully improve precision for a variety of outcome types. Their findings build on a long history, starting in 1932 with R.A. Fisher and…

Methodology · Statistics 2026-03-03 Laura B. Balzer , Erica Cai , Lucas Godoy Garraza , Pracheta Amaranath

Consider an experiment consisting of a set of independent trials for comparing a set of treatments. In each trial, one treatment is chosen and the mean response of the trial is equal to the effect of the chosen treatment. We examine the…

Statistics Theory · Mathematics 2016-12-21 Samuel Rosa

While randomised controlled trials (RCTs) are the gold standard for estimating causal treatment effects, their limited sample sizes and restrictive criteria make it difficult to extrapolate to a broader population. Observational data, while…

Methodology · Statistics 2025-09-09 Stephanie Riley , Ricardo Silva , Matthew Sperrin

Background: When planning a cluster randomized trial, evaluators often have access to an enumerated cohort representing the target population of clusters. Practicalities of conducting the trial, such as the need to oversample clusters with…

Methodology · Statistics 2024-09-19 Sarah E. Robertson , Jon A. Steingrimsson , Issa J. Dahabreh

The subject of this work is two treatment groups random coefficient regression models, in which observational units receive some group-specific treatments. We provide A- and D-optimality criteria for the estimation of the fixed parameter…

Statistics Theory · Mathematics 2020-08-11 Maryna Prus

Randomized experimentation (also known as A/B testing or bucket testing) is widely used in the internet industry to measure the metric impact obtained by different treatment variants. A/B tests identify the treatment variant showing the…

We study randomized experiments in bipartite systems where only a subset of treatment-side units are eligible for assignment while all units continue to interact, generating interference. We formalize eligibility-constrained bipartite…

Methodology · Statistics 2025-11-17 Albert Tan , Mohsen Bayati , James Nordlund , Roman Istomin

This paper studies inference for the local average treatment effect in randomized controlled trials with imperfect compliance where treatment status is determined according to "matched pairs." By "matched pairs," we mean that units are…

Econometrics · Economics 2024-06-28 Yuehao Bai , Hongchang Guo , Azeem M. Shaikh , Max Tabord-Meehan

We consider learning personalized assignments to one of many treatment arms from a randomized controlled trial. Standard methods that estimate heterogeneous treatment effects separately for each arm may perform poorly in this case due to…

Machine Learning · Statistics 2023-11-02 Rahul Ladhania , Jann Spiess , Lyle Ungar , Wenbo Wu

Given a dataset of individuals each described by a covariate vector, a treatment, and an observed outcome on the treatment, the goal of the individual treatment effect (ITE) estimation task is to predict outcome changes resulting from a…

Machine Learning · Computer Science 2024-06-07 Lokesh Nagalapatti , Pranava Singhal , Avishek Ghosh , Sunita Sarawagi

This paper illustrates asymptotic properties for a response-adaptive design generated by a two-color, randomly reinforced urn model. The design considered is optimal in the sense that it assigns patients to the best treatment, with…

Statistics Theory · Mathematics 2009-04-03 Caterina May , Nancy Flournoy

The estimation of average treatment effects (ATEs), defined as the difference in expected outcomes between treatment and control groups, is a central topic in causal inference. This study develops semiparametric efficient estimators for ATE…

Machine Learning · Computer Science 2025-05-29 Masahiro Kato , Fumiaki Kozai , Ryo Inokuchi
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