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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

Subject selection plays a critical role in experimental studies, especially ones with human subjects. Anecdotal evidence suggests that many such studies, done at or near university campus settings suffer from selection bias, i.e., the…

Machine Learning · Computer Science 2020-12-21 Tahereh Arabghalizi , Alexandros Labrinidis

An accurate multiclass classification strategy is crucial to interpreting antibody tests. However, traditional methods based on confidence intervals or receiver operating characteristics lack clear extensions to settings with more than two…

Quantitative Methods · Quantitative Biology 2024-05-07 Rayanne A. Luke , Anthony J. Kearsley , Paul N. Patrone

A platform trial with a master protocol provides an infrastructure to ethically and efficiently evaluate multiple treatment options in multiple diseases. Given that certain study drugs can enter or exit a platform trial, the randomization…

Methodology · Statistics 2025-07-15 Tianyu Zhan , Jane Zhang , Lei Shu , Yihua Gu

Effect modification means the magnitude or stability of a treatment effect varies as a function of an observed covariate. Generally, larger and more stable treatment effects are insensitive to larger biases from unmeasured covariates, so a…

Methodology · Statistics 2018-03-12 Kwonsang Lee , Dylan S. Small , Paul R. Rosenbaum

New crop varieties are extensively tested in multi-environment trials in order to obtain a solid empirical basis for recommendations to farmers. When the target population of environments is large and heterogeneous, a division into…

Applications · Statistics 2020-04-14 Maryna Prus , Hans-Peter Piepho

In recent years, bilevel approaches have become very popular to efficiently estimate high-dimensional hyperparameters of machine learning models. However, to date, binary parameters are handled by continuous relaxation and rounding…

Machine Learning · Computer Science 2025-03-20 Sara Venturini , Marianna de Santis , Jordan Patracone , Francesco Rinaldi , Saverio Salzo , Martin Schmidt

This paper combines the idea of a hierarchical distributed genetic algorithm with different inter-agent partnering strategies. Cascading clusters of sub-populations are built from bottom up, with higher-level sub-populations optimising…

Neural and Evolutionary Computing · Computer Science 2010-07-05 Uwe Aickelin , Larry Bull

Practitioners often use data from a randomized controlled trial to learn a treatment assignment policy that can be deployed on a target population. A recurring concern in doing so is that, even if the randomized trial was well-executed…

Econometrics · Economics 2023-04-25 Lihua Lei , Roshni Sahoo , Stefan Wager

Subgroup selection in clinical trials is essential for identifying patient groups that react differently to a treatment, thereby enabling personalised medicine. In particular, subgroup selection can identify patient groups that respond…

Collecting genomics data across multiple heterogeneous populations (e.g., across different cancer types) has the potential to improve our understanding of disease. Despite sequencing advances, though, resources often remain a constraint…

Methodology · Statistics 2024-03-05 Yunyi Shen , Lorenzo Masoero , Joshua G. Schraiber , Tamara Broderick

A treatment regime is a deterministic function that dictates personalized treatment based on patients' individual prognostic information. There is a fast-growing interest in finding optimal treatment regimes to maximize expected long-term…

Statistics Theory · Mathematics 2016-11-25 Runchao Jiang , Wenbin Lu , Rui Song , Marie Davidian

In recent years, real-world external controls have grown in popularity as a tool to empower randomized placebo-controlled trials, particularly in rare diseases or cases where balanced randomization is unethical or impractical. However, as…

Methodology · Statistics 2024-11-14 Chenyin Gao , Shu Yang , Mingyang Shan , Wenyu Ye , Ilya Lipkovich , Douglas Faries

An important goal of precision medicine is to personalize medical treatment by identifying individuals who are most likely to benefit from a specific treatment. The Likely Responder (LR) framework, which identifies a subpopulation where…

Methodology · Statistics 2026-03-13 Annan Deng , Carole Siegel , Hyung G. Park

Large-scale testing is crucial in pandemic containment, but resources are often prohibitively constrained. We study the optimal application of pooled testing for populations that are heterogeneous with respect to an individual's infection…

Computer Science and Game Theory · Computer Science 2023-09-22 Simon Finster , Michelle González Amador , Edwin Lock , Francisco Marmolejo-Cossío , Evi Micha , Ariel D. Procaccia

Achieving covariate balance in randomized experiments enhances the precision of treatment effect estimation. However, existing methods often require heuristic adjustments based on domain knowledge and are primarily developed for binary…

Methodology · Statistics 2025-02-25 Wenxuan Guo , Tengyuan Liang , Panos Toulis

Randomized trials typically estimate average relative treatment effects, but decisions on the benefit of a treatment are possibly better informed by more individualized predictions of the absolute treatment effect. In case of a binary…

Methodology · Statistics 2021-08-20 J Hoogland , J IntHout , M Belias , MM Rovers , RD Riley , FE Harrell , KGM Moons , TPA Debray , JB Reitsma

There are many different notions of optimality even in testing a single hypothesis. In the multiple testing area, the number of possibilities is very much greater. The paper first will describe multiplicity issues that arise in tests…

Statistics Theory · Mathematics 2007-06-13 Juliet Popper Shaffer

Optimizing the allocation of units into treatment groups can help researchers improve the precision of causal estimators and decrease costs when running factorial experiments. However, existing optimal allocation results typically assume a…

Methodology · Statistics 2024-05-31 Arun Ravichandran , Nicole E. Pashley , Brian Libgober , Tirthankar Dasgupta

Non-significant randomized control trials can hide subgroups of good responders to experimental drugs, thus hindering subsequent development. Identifying such heterogeneous treatment effects is key for precision medicine and many post-hoc…

Methodology · Statistics 2024-01-24 Valentine Perrin , Nathan Noiry , Nicolas Loiseau , Alex Nowak