Vaccine Disease-Prevention Efficacy Studies: Traditional Approaches and New Frontiers

The statistics (and the roles of statisticians) in an event-driven vaccine efficacy clinical trial span a sustained effort containing long times of relative inactivity (besides conducting reviews and completing paperwork) interspersed with moments of extreme, frantic, concentrated activity requiring a great deal of statistical experience and expertise. It is an experience that is like none other in biostatistics, and there is much to be gained no matter whether the outcome of the vaccine pivotal study is positive or negative. This chapter summarizes statistical aspects of study design and sample sizing, analysis planning, and interpretation of vaccine clinical efficacy data. Emerging computer intensive approaches including Bayesian statistics are considered. Worked examples and code are included where appropriate.