Cluster randomization trials in tropical medicine: a case study

Different methods which can be used to construct tests of the effect of treatment when outcomes are binary (e.g., infected/uninfected) are compared.

Abstract

Field trials in tropical medicine are often designed so that intact social units (e.g., families, schools, communities) rather than independent individuals are randomized to an intervention group. Reasons are diverse, but include administrative convenience, a desire to reduce the effect of treatment contamination, and the need to avoid ethical issues that might otherwise arise. Dependencies among cluster members typical of such designs must be considered when determining sample size and analysing the resulting data. Failure to do so can result in false conclusions that the treatment is effective. The purpose of this paper is to compare different methods which can be used to construct tests of the effect of treatment when outcomes are binary (e.g., infected/uninfected). The discussion will be illustrated using data from a trial which randomly assigned families to either a control group or a screening and treatment programme for imported intestinal parasites.