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arXiv · 2503.08987

Multilevel Primary Aim Analyses of Clustered SMARTs: With Applications in Health Policy

Gabriel Durham·Anil Battalahalli·Amy Kilbourne·Andrew Quanbeck·Wenchu Pan·Tim Lycurgus·Daniel Almirall
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Abstract

In many health policy settings, adaptive interventions target a population of clusters (e.g., schools), with the ultimate intent of impacting outcomes at the level of individuals within the clusters. Health policy researchers can use clustered, sequential, multiple assignment, randomized trials (SMARTs) to answer important scientific questions concerning clustered adaptive interventions. A common primary aim is to compare the mean of a nested, end-of-study outcome between two clustered adaptive interventions. However, existing methods are not suitable when the primary outcome in a clustered SMART is nested and longitudinal (e.g., repeated outcome measures nested within mental healthcare providers, and mental healthcare providers nested within schools). This manuscript proposes a three-level marginal mean modeling and estimation approach for comparing adaptive interventions in a clustered SMART. The proposed method enables policy analysts to answer a wider array of scientific questions in the marginal comparison of clustered adaptive interventions. Further, relative to using an existing two-level method with a nested end-of-study outcome, the proposed method benefits from improved statistical efficiency. With this approach, we examine longitudinal comparisons of adaptive interventions for improving school-based mental healthcare and contrast its performance with existing approaches for studying static end-of-study outcomes. Methods were motivated by the Adaptive School-Based Implementation of CBT (ASIC) study, a clustered SMART designed to construct an adaptive health policy to improve the adoption of evidence-based CBT by mental healthcare professionals in high schools across Michigan.

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Gabriel Durham, Anil Battalahalli, Amy Kilbourne, Andrew Quanbeck, Wenchu Pan, Tim Lycurgus, Daniel Almirall. 2025-03-12. Multilevel Primary Aim Analyses of Clustered SMARTs: With Applications in Health Policy. https://arxiv.org/abs/2503.08987

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