Clinical trials: Kids are not just little people

Clinical trials are the backbone of modern evidence-based medicine. They are the vital bridge between research-based discovery and cutting edge patient care. Randomized, controlled clinical trials are the gold standard of medical research, providing a method for evaluation and discovery of novel therapies that improve and even save lives. Despite an increase in the number of pediatric and adult clinical trials over recent decades, this growth has not been equal among these populations. The volume of clinical trials involving children lags substantially behind their adult counterparts. Children are not simply little adults. As a result, extrapolating results from adult clinical trials to the treatment of children may be inappropriate and, possibly, harmful. In this review, we discuss the intricacies of performing clinical trials in all patients and stress the unique distinguishing characteristics of pediatric clinical trials.