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Statistics in Medicine
Article . 2019 . Peer-reviewed
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Article . 2020
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https://dx.doi.org/10.48550/ar...
Article . 2019
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Novel methods for the analysis of stepped wedge cluster randomized trials

Authors: Lee Kennedy‐Shaffer; Victor de Gruttola; Marc Lipsitch;

Novel methods for the analysis of stepped wedge cluster randomized trials

Abstract

Stepped wedge cluster randomized trials (SW‐CRTs) have become increasingly popular and are used for a variety of interventions and outcomes, often chosen for their feasibility advantages. SW‐CRTs must account for time trends in the outcome because of the staggered rollout of the intervention. Robust inference procedures and nonparametric analysis methods have recently been proposed to handle such trends without requiring strong parametric modeling assumptions, but these are less powerful than model‐based approaches. We propose several novel analysis methods that reduce reliance on modeling assumptions while preserving some of the increased power provided by the use of mixed effects models. In one method, we use the synthetic control approach to find the best matching clusters for a given intervention cluster. Another method makes use of within‐cluster crossover information to construct an overall estimator. We also consider methods that combine these approaches to further improve power. We test these methods on simulated SW‐CRTs, describing scenarios in which these methods have increased power compared with existing nonparametric methods while preserving nominal validity when mixed effects models are misspecified. We also demonstrate theoretical properties of these estimators with less restrictive assumptions than mixed effects models. Finally, we propose avenues for future research on the use of these methods; motivation for such research arises from their flexibility, which allows the identification of specific causal contrasts of interest, their robustness, and the potential for incorporating covariates to further increase power. Investigators conducting SW‐CRTs might well consider such methods when common modeling assumptions may not hold.

Keywords

FOS: Computer and information sciences, Cross-Over Studies, synthetic control, cluster randomized trials, stepped wedge, Applications of statistics to biology and medical sciences; meta analysis, Methodology (stat.ME), permutation tests, Research Design, Cluster Analysis, Humans, mixed effects models, Statistics - Methodology, Randomized Controlled Trials as Topic

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    19
    popularity
    This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
    Top 10%
    influence
    This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
    Top 10%
    impulse
    This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
    Top 10%
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selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
19
Top 10%
Top 10%
Top 10%
Green
bronze