Sample size calculations for multilevel factorial longitudinal cluster randomised trials

TL;DR

This paper develops sample size calculation methods for complex multilevel factorial longitudinal cluster randomised trials, enabling joint assessment of individual- and cluster-level interventions with continuous outcomes.

Contribution

It introduces new methodology tailored for split-plot factorial longitudinal cluster trials, addressing a gap in existing sample size approaches for such designs.

Findings

Provides formulas for power calculation based on standard trial results.

Demonstrates application to the SharES trial on breast cancer interventions.

Allows assessment of interaction effects between interventions at different levels.

Abstract

Typically, trials investigate the impact of either an individual-level intervention on participant outcomes, or the impact of a cluster-level intervention on participant outcomes. Factorial designs consider two (or more) treatments for each of two (or more) different factors. In factorial trial designs, trial units (individuals or clusters) are each randomised to a level of each of the treatments; these designs allow assessment of the interactions between different interventions. Recently, there has been growing interest in the design of trials that jointly assess the impact of individual- and cluster-level interventions (i.e. multi-level interventions); requiring the development of methodology that accommodates randomisation at multiple levels. While recent work has developed sample size methodology for variants combining standard cluster randomisation and individual randomisation, that work does not apply to longitudinal cluster randomised trial designs such as the stepped wedge design or cluster randomised crossover design. Here we present dedicated sample size methodology for "split-plot factorial longitudinal cluster randomised trials" with continuous outcomes: allowing for joint assessment of individual-level and cluster-level interventions that allows for the impact of the cluster-level intervention to be assessed using any longitudinal cluster randomised trial design. We show how the power to detect given effects of the individual-level intervention, the cluster-level intervention, and the interaction between the two depends on standard results for individually-randomised trials and longitudinal cluster randomised trials. We apply these results to the SharES trial, which considered the effects of a patient- and clinician-level interventions for patients with breast cancer on patient knowledge about the risks and benefits of treatment.