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Personalized intervention strategies, in particular those that modify treatment based on a participant's own response, are a core component of precision medicine approaches. Sequential Multiple Assignment Randomized Trials (SMARTs) are…

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

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

Sequential Multiple Assignment Randomized Trials (SMARTs) are considered the gold standard for estimation and evaluation of treatment regimes. SMARTs are typically sized to ensure sufficient power for a simple comparison, e.g., the…

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

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

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

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

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…

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

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

The solution here proposed can be used to conduct economic analysis in randomized clinical trials. It is based on a statistical approach and aims at calculating a revised version of the incremental costeffective ratio (ICER) in order to…

Applications · Statistics 2013-06-19 C. D'Urso

Background: Missing data poses an acute threat to sequential multiple assignment randomized trial (SMART) analyses because of the sequential treatment structure and response-dependent re-randomization. Objectives: This study aimed to (1)…

Sequential multiple assignment randomized trials (SMARTs) are used to construct data-driven optimal intervention strategies for subjects based on their intervention and covariate histories in different branches of health and behavioral…

Methodology · Statistics 2022-04-28 Palash Ghosh , Xiaoxi Yan , Bibhas Chakraborty

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

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

The incremental cost-effectiveness ratio (ICER) and incremental net benefit (INB) are widely used for cost-effectiveness analysis. We develop methods for estimation and inference for the ICER and INB which use the semiparametric stratified…

Methodology · Statistics 2025-05-12 Polyna Khudyakov , Li Xu , Ce Yang , Donna Spiegelman , Molin Wang

The optimal prophylaxis, and treatment if the prophylaxis fails, for a disease may be best evaluated using a sequential multiple assignment randomised trial (SMART). A SMART is a multi-stage study that randomises a participant to an initial…

Methodology · Statistics 2022-03-25 Robert K. Mahar , Katherine J. Lee , Bibhas Chakraborty , Agus Salim , Julie A. Simpson

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
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