Case Studies

In previous chapters, various innovative study designs and analyses are discussed focusing on small population of pediatric and rare diseases. The chapter illustrates three drug approvals that use different innovative designs and analyses that establish the substantial evidence of efficacy and safety in the small population. Case study of Tocilizumab utilized retrospective analysis that led to the new indication approval for treatment of Chimeric Antigen Receptor (CAR) T Cell-induced severe or life-threatening Cytokine Release Syndrome (CRS) in pediatric patients 2 years and older. Second case study uses natural history as a comparator that led to Prograf (Tacrolimus) new indication approval to prevent rejection in adults and pediatric patients who receive lung transplants. The third case is Belimumab’s approval that has applied Bayesian analysis to borrow adult data into pediatric to support approval in children with system lupus erythematosus (SLE).