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Practitioners conducting adaptive experiments often encounter two competing priorities: maximizing total welfare (or `reward') through effective treatment assignment and swiftly concluding experiments to implement population-wide…

Machine Learning · Computer Science 2024-07-31 Chao Qin , Daniel Russo

Background: Well-designed phase II trials must have acceptable error rates relative to a pre-specified success criterion, usually a statistically significant p-value. Such standard designs may not always suffice from a clinical perspective…

Applications · Statistics 2020-02-10 Satrajit Roychoudhury , Nicolas Scheuer , Beat Neuenschwander

Randomized Controlled Trials (RCTs) represent the gold standard for causal inference yet remain a scarce resource. While large-scale observational data is often available, it is utilized only for retrospective fusion, and remains discarded…

Machine Learning · Statistics 2026-03-05 Erdun Gao , Liang Zhang , Jake Fawkes , Aoqi Zuo , Wenqin Liu , Haoxuan Li , Mingming Gong , Dino Sejdinovic

We consider adaptive designs for a trial involving N individuals that we follow along T time steps. We allow for the variables of one individual to depend on its past and on the past of other individuals. Our goal is to learn a mean…

Statistics Theory · Mathematics 2021-01-20 Aurelien Bibaut , Maya Petersen , Nikos Vlassis , Maria Dimakopoulou , Mark van der Laan

Precision medicine aims to tailor treatment decisions according to patients' characteristics. G-estimation and dynamic weighted ordinary least squares (dWOLS) are double robust statistical methods that can be used to identify optimal…

Methodology · Statistics 2021-11-10 Denis Talbot , Erica EM Moodie , Caroline Diorio

We consider conditional exact tests of factor effects in designed experiments for discrete response variables. Similarly to the analysis of contingency tables, a Markov chain Monte Carlo method can be used for performing exact tests, when…

Statistics Theory · Mathematics 2009-11-20 Satoshi Aoki , Akimichi Takemura

Adaptive therapy improves cancer treatment by controlling the competition between sensitive and resistant cells through treatment holidays. This study highlights the critical role of treatment-holiday thresholds in adaptive therapy for…

Other Quantitative Biology · Quantitative Biology 2025-07-15 Lanfei Sun , Haifeng Zhang , Kai Kang , Xiaoxin Wang , Leyi Zhang , Yanan Cai , Changjing Zhuge , Lei Zhang

Modern randomization methods in clinical trials are invariably adaptive, meaning that the assignment of the next subject to a treatment group uses the accumulated information in the trial. Some of the recent adaptive randomization methods…

Methodology · Statistics 2024-02-12 Alan R. Vazquez , Weng Kee Wong

Randomized controlled trials (RCTs) are the accepted standard for treatment effect estimation but they can be infeasible due to ethical reasons and prohibitive costs. Single-arm trials, where all patients belong to the treatment group, can…

Real-world experiments involve batched & delayed feedback, non-stationarity, multiple objectives & constraints, and (often some) personalization. Tailoring adaptive methods to address these challenges on a per-problem basis is infeasible,…

Machine Learning · Computer Science 2024-11-11 Ethan Che , Daniel R. Jiang , Hongseok Namkoong , Jimmy Wang

Adaptive designs dynamically update treatment probabilities using information accumulated during the experiment. Existing theory for causal inference from adaptive experiments primarily assumes the superpopulation framework with independent…

Methodology · Statistics 2026-02-26 Xinran Li , Anqi Zhao

An early phase clinical trial is the first step in evaluating the effects in humans of a potential new anti-disease agent or combination of agents. Usually called "phase I" or "phase I/II" trials, these experiments typically have the…

Methodology · Statistics 2010-12-01 Peter F. Thall

Randomized Controlled Trials (RCTs) are the gold standard for evaluating the effect of new medical treatments. Treatments must pass stringent regulatory conditions in order to be approved for widespread use, yet even after the regulatory…

Machine Learning · Statistics 2025-03-13 Omer Noy Klein , Alihan Hüyük , Ron Shamir , Uri Shalit , Mihaela van der Schaar

Various adaptive randomization procedures (adaptive designs) have been proposed to clinical trials. This paper discusses several broad families of procedures, such as the play-the-winner rule and Markov chain model, randomized…

Statistics Theory · Mathematics 2009-08-30 Li-Xin Zhang

Making good predictions of a physical system using a computer code requires the inputs to be carefully specified. Some of these inputs called control variables have to reproduce physical conditions whereas other inputs, called parameters,…

Computation · Statistics 2018-04-04 Guillaume Damblin , Pierre Barbillon , Merlin Keller , Alberto Pasanisi , Eric Parent

In a completely randomized experiment, the variances of treatment effect estimators in the finite population are usually not identifiable and hence not estimable. Although some estimable bounds of the variances have been established in the…

Statistics Theory · Mathematics 2022-09-20 Ruoyu Wang , Qihua Wang , Wang Miao , Xiaohua Zhou

In statistical process control, procedures are applied that require relatively strict conditions for their use. If such assumptions are violated, these methods become inefficient, leading to increased incidence of false signals. Therefore,…

Other Statistics · Statistics 2019-01-15 Gejza Dohnal

We study the fundamental tradeoffs between computational tractability and statistical accuracy for a general family of hypothesis testing problems with combinatorial structures. Based upon an oracle model of computation, which captures the…

Machine Learning · Statistics 2015-12-31 Zhaoran Wang , Quanquan Gu , Han Liu

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…

Over time, clinical trials have increasingly incorporated complex design and analysis elements such as interim analyses, adaptations, multiple endpoints, and sophisticated multiplicity schemes for multiple endpoints and/or treatment arms…