This module considers statistical issues in the design and analysis of randomized clinical trials for evaluating vaccine efficacy to prevent infection and disease. Topics include:
- vaccine mechanisms of protection and assays for measuring immune response;
- study designs for assessing different types of vaccine efficacy parameters (individual and cluster randomized, test negative, ring stepped wedge);
- definitions and statistical frameworks for assessing immune response biomarkers as surrogate endpoints, mediators, and modifiers of vaccine efficacy;
- statistical methods for assessing immune response biomarkers as correlates and surrogate endpoints using statistical learning techniques such as super-learner;
- statistical methods for assessing immune response biomarker modifiers of vaccine efficacy, including an R practicum on the R package pseval; and
- statistical methods for assessing how vaccine efficacy depends on pathogen genomic sequences (sieve analysis) and accounting for multiple founder infection.
Illustrations include trials of malaria, dengue, HIV, rabies, polio, and Ebola vaccines. Guest lecturers include Erin Gabriel.