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In a sequential multiple-assignment randomized trial (SMART), a sequence of treatments is given to a patient over multiple stages. In each stage, randomization may be done to allocate patients to different treatment groups. Even though…

Methodology · Statistics 2024-01-09 Rik Ghosh , Bibhas Chakraborty , Inbal Nahum-Shani , Megan E. Patrick , Palash Ghosh

Dynamic treatment regimes (DTRs) are sequences of decision rules that recommend treatments based on patients' time-varying clinical conditions. The sequential multiple assignment randomized trial (SMART) is an experimental design that can…

Methodology · Statistics 2024-05-14 Xinru Wang , Nina Deliu , Yusuke Narita , Bibhas Chakraborty

The sequential multiple assignment randomized trial (SMART) is the gold standard trial design to generate data for the evaluation of multi-stage treatment regimes. As with conventional (single-stage) randomized clinical trials, interim…

Methodology · Statistics 2023-09-13 Cole Manschot , Eric Laber , Marie Davidian

Cluster-level dynamic treatment regimens can be used to guide sequential, intervention or treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level DTR, the…

Methodology · Statistics 2016-07-15 Timothy NeCamp , Amy Kilbourne , Daniel Almirall

A sequential multiple assignment randomized trial (SMART) facilitates comparison of multiple adaptive treatment strategies (ATSs) simultaneously. Previous studies have established a framework to test the homogeneity of multiple ATSs by a…

Methodology · Statistics 2022-11-04 Liwen Wu , Junyao Wang , Abdus S. Wahed

Sequential, multiple assignment randomized trials (SMARTs), which assist in the optimization of adaptive interventions, are growing in popularity in education and behavioral sciences. This is unsurprising, as adaptive interventions reflect…

Methodology · Statistics 2023-09-26 Timothy Lycurgus , Amy Kilbourne , Daniel Almirall

Response-adaptive randomization (RAR) has been studied extensively in conventional, single-stage clinical trials, where it has been shown to yield ethical and statistical benefits, especially in trials with many treatment arms. However, RAR…

Methodology · Statistics 2024-01-09 Peter Norwood , Marie Davidian , Eric Laber

A clustered adaptive intervention (cAI) is a pre-specified sequence of decision rules that guides practitioners on how best - and based on which measures - to tailor cluster-level intervention to improve outcomes at the level of individuals…

Methodology · Statistics 2025-05-05 Yao Song , Kelly Speth , Amy Kilbourne , Andrew Quanbeck , Daniel Almirall , Lu Wang

Multistage design has been used in a wide range of scientific fields. By allocating sensing resources adaptively, one can effectively eliminate null locations and localize signals with a smaller study budget. We formulate a…

Methodology · Statistics 2024-06-17 Weinan Wang , Bowen Gang , Wenguang Sun

The I-SPY2 phase 2 clinical trial is a long-running platform trial that evaluates neoadjuvant treatments for locally advanced breast cancer, assigning subjects to novel agents using response-adaptive randomization. Recently, I-SPY2 was…

Health policy decisions regarding patient treatment strategies require consideration of both treatment effectiveness and cost. Optimizing treatment rules with respect to effectiveness may result in prohibitively expensive strategies; on the…

Methodology · Statistics 2021-10-20 Nicholas Illenberger , Andrew J. Spieker , Nandita Mitra

The sequential multiple assignment randomized trial (SMART) is the ideal study design for the evaluation of multistage treatment regimes, which comprise sequential decision rules that recommend treatments for a patient at each of a series…

Methodology · Statistics 2024-05-15 Anastasios A. Tsiatis , Marie Davidian

We develop methodology for a multistage decision problem with flexible number of stages in which the rewards are survival times that are subject to censoring. We present a novel Q-learning algorithm that is adjusted for censored data and…

Statistics Theory · Mathematics 2012-05-31 Yair Goldberg , Michael R. Kosorok

A dynamic treatment regime is a sequence of decision rules in which each decision rule recommends treatment based on features of patient medical history such as past treatments and outcomes. Existing methods for estimating optimal dynamic…

Methodology · Statistics 2015-05-22 Kristin A. Linn , Eric B. Laber , Leonard A. Stefanski

One of the main goals of sequential, multiple assignment, randomized trials (SMART) is to find the most efficacious design embedded dynamic treatment regimes. The analysis method known as multiple comparisons with the best (MCB) allows…

Methodology · Statistics 2020-08-07 William J. Artman , Ashkan Ertefaie , Kevin G. Lynch , James R. McKay

Clinicians and researchers alike are increasingly interested in how best to personalize interventions. A dynamic treatment regimen (DTR) is a sequence of pre-specified decision rules which can be used to guide the delivery of a sequence of…

Adaptive interventions (AIs) are increasingly becoming popular in medical and behavioral sciences. An AI is a sequence of individualized intervention options that specify for whom and under what conditions different intervention options…

Applications · Statistics 2018-12-18 Palash Ghosh , Inbal Nahum-Shani , Bonnie Spring , Bibhas Chakraborty

Adaptive interventions, aka dynamic treatment regimens, are sequences of pre-specified decision rules that guide the provision of treatment for an individual given information about their baseline and evolving needs, including in response…

Methodology · Statistics 2024-05-02 Wenchu Pan , Daniel Almirall , Amy M. Kilbourne , Andrew Quanbeck , Lu Wang

A dynamic treatment regimen (DTR) is a pre-specified sequence of decision rules which maps baseline or time-varying measurements on an individual to a recommended intervention or set of interventions. Sequential multiple assignment…

Methodology · Statistics 2019-10-23 Brook Luers , Min Qian , Inbal Nahum-Shani , Connie Kasari , Daniel Almirall

Precision medicine aims to tailor therapeutic decisions to individual patient characteristics. This objective is commonly formalized through dynamic treatment regimes, which use statistical and machine learning methods to derive sequential…

Machine Learning · Statistics 2026-03-23 Sophia Yazzourh , Erica E. M. Moodie
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