SearcharxivSearch

arXiv · 2606.04637

Optimal designs for incomplete stepped wedge trials

Abstract

Background: Stepped wedge trials are longitudinal randomised evaluations, usually cluster-randomised, in which the experimental intervention is introduced in a staggered fashion. Incomplete stepped wedge designs focus the effort of data collection on particular periods in particular sequences. Methods: We suppose there is a cost for every period in every cluster where we collect data, and that there are a fixed number of individuals, m, with data available in each period in each cluster. If we are willing to pay the cost of data collection in that cluster-period then we collect the data on all m individuals, and if we are not willing to pay the cost then we collect no data in that cluster-period. We consider the problem of designing a trial to minimise the total number of cluster-periods of data collection needed to achieve given precision for the treatment effect estimator, or equivalently, to maximise precision for a given number of cluster-periods of data collection. Results: We present the solution for two-period trials, which has two distinct forms, depending on the correlation between two cluster-period means from the same cluster in different periods. We also present a conjecture on the form of the solution for multi-period trials, informed by results from a greedy search of the design space. Conclusions: A real-life stepped wedge design problem will involve trading off the costs of various design elements subject also to constraints on the scale of data collection. Nevertheless, the solutions to the problem considered here add significantly to our understanding of the optimal design of incomplete stepped wedge trials.

Explore related subjects

Keep this discovery

Explore connections, maps & timelines

BibTeXRIS

Richard Hooper, Alan Girling. 2026-06-03. Optimal designs for incomplete stepped wedge trials. https://arxiv.org/abs/2606.04637

Cite the original work for its findings. Save a collection to share your selection of sources.

KEEP EXPLORING

Related papers

Privacy-Preserving Causal Meta-Mediation Analysis with Survival Outcomes

Privacy and data-governance constraints often prevent pooling individual-level data across studies, limiting the use of conventional approaches for causal media- tion analysis in multicenter settings. We propose a federated causal meta-mediation framework for right-censored time-to-event outcomes that enables collaborative es- timation without sharing individual-level data. Our framework targets natural indirect effects in a prespecified population by combining information on mediator and outcome mechanisms across distributed data sources. A site-by-site identifi- cation strategy further allows heterogeneity across data sources to be character- ized, with a variance decomposition separating outcome-related, mediator-related, and interaction components. We develop federated one-step and targeted maxi- mum likelihood estimators that accommodate data-adaptive and machine-learning methods for nuisance-function estimation. The finite-sample performance of the proposed estimators is evaluated through numerical simulations. To illustrate the practical utility of the framework, we apply it on data from the French National Health Data System to evaluate the role of methotrexate coprescription in explain- ing the effect of TNFi versus IL-12/23 inhibitor therapy on treatment persistence among psoriatic patients.

stat.AP

Geospatial Foundation Models Capture Health-Relevant Dimensions of Place Beyond Conventional Social Risk Indices

Area-based social risk indices summarize residents' socioeconomic conditions but incompletely capture physical features of place that may affect health. We evaluated whether numerical representations of physical place produced by four geospatial foundation model families from 2022 satellite data explained residual variance in tract-level associations between the Area Deprivation Index, Social Deprivation Index, and Social Vulnerability Index with health outcomes. We used LightGBM to predict variables from the American Community Survey and 40 chronic disease and health-behavior outcomes from CDC PLACES across 82,646 census tracts in the contiguous United States, evaluating performance across 10 held-out states. Among survey variables, models were moderately predictive of some variables including housing type (R-squared up to 0.54) but weak for disability, unemployment, and income disparity. For health outcomes, models explained up to 54% of variance left unexplained by social risk indices, with the largest gains for annual checkups, arthritis, and high blood pressure. Mean total variance explained by geospatial foundation models across the 40 health-related outcomes increased from 0.31 in the smallest tract-size decile to 0.39 in the largest. Geospatial foundation models capture health-relevant features of place not represented by conventional social risk indices and may usefully augment them in epidemiological analyses.

stat.AP

A spatiotemporal negative binomial model with dynamic dispersion: An application to Tuberculosis infections

Tuberculosis (TB) remains a critical public health concern in Brazil, characterized by pronounced spatial heterogeneity and fluctuating temporal volatility. In this paper, we study monthly TB notifications across 61 microregions of Sao Paulo state from 2001 to 2024. To do this, we introduce a negative binomial spatial integer-valued generalized autoregressive conditional heteroskedastic (INGARCH) model featuring jointly dynamic conditional means and time-varying dispersion. To capture inter-regional spillovers, we incorporate both discrete adjacency structures and a novel continuous distance-based formulation leveraging the Matern correlation function. Parameter estimation via conditional maximum likelihood employs a two-step profile-likelihood iterative scheme, demonstrating solid finite-sample performance in simulation studies. Applied to the Sao Paulo TB surveillance data, the framework substantially outperforms standard Poisson and fixed-dispersion spatiotemporal baselines in empirical fit and uncertainty quantification, maintaining nominal 95% predictive coverage across both dense metropolitan centers and rural microregions. Our results reveal marked spatial heterogeneity in baseline incidence, dynamic overdispersion driven by localized outbreaks, and short-range spatial interaction decay. By accurately modeling spatiotemporal volatility, the proposed methodology provides a robust statistical tool to support public health surveillance, policy-making, and resource allocation.

stat.AP