Cohort versus cross-sectional design in large field trials: precision, sample size, and a unifying model.

Abstract

In planning large longitudinal field trials, one is often faced with a choice between a cohort design and a cross-sectional design, with attendant issues of precision, sample size, and bias. To provide a practical method for assessing these trade-offs quantitatively, we present a unifying statistical model that embraces both designs as special cases. The model takes account of continuous and discrete endpoints, site differences, and random cluster and subject effects of both a time-invariant and a time-varying nature. We provide a comprehensive design equation, relating sample size to precision for cohort and cross-sectional designs, and show that the follow-up cost and selection bias attending a cohort design may outweigh any theoretical advantage in precision. We provide formulae for the minimum number of clusters and subjects. We relate this model to the recently published prevalence model for COMMIT, a multi-site trial of smoking cessation programmes. Finally, we tabulate parameter estimates for some physiological endpoints from recent community-based heart-disease prevention trials, work an example, and discuss the need for compiling such estimates as a basis for informed design of future field trials.