Optimal allocation in stratified cluster‐based outcome‐dependent sampling designs

Abstract

In public health research, finite resources often require that decisions be made at the study design stage regarding which individuals to sample for detailed data collection. At the same time, when study units are naturally clustered, as patients are in clinics, it may be preferable to sample clusters rather than the study units, especially when the costs associated with travel between clusters are high. In this setting, aggregated data on the outcome and select covariates are sometimes routinely available through, for example, a country's Health Management Information System. If used wisely, this information can be used to guide decisions regarding which clusters to sample, and potentially obtain gains in efficiency over simple random sampling. In this article, we derive a series of formulas for optimal allocation of resources when a single-stage stratified cluster-based outcome-dependent sampling design is to be used and a marginal mean model is specified to answer the question of interest. Specifically, we consider two settings: (i) when a particular parameter in the mean model is of primary interest; and, (ii) when multiple parameters are of interest. We investigate the finite population performance of the optimal allocation framework through a comprehensive simulation study. Our results show that there are trade-offs that must be considered at the design stage: optimizing for one parameter yields efficiency gains over balanced and simple random sampling, while resulting in losses for the other parameters in the model. Optimizing for all parameters simultaneously yields smaller gains in efficiency, but mitigates the losses for the other parameters in the model.