Clinical Trials in Children

Abstract

Clinical trials in children are important and challenging. Many advances in pediatric health have occurred as a result of clinical trials, ranging from the polio vaccine to chemotherapy for childhood leukemia. Nevertheless, the majority of medications prescribed to children have never been evaluated in them. Relatively recent programs to encourage testing of drugs in children have yielded important new information about pharmacokinetics and pharmacodynamics in pediatric populations, often contrary to what might have been expected. Trials are required, therefore, in order to determine not only the effects of drugs but also safe doses of medications in light of developmental changes throughout childhood. In addition, clinical trials are necessary in children because they have conditions that adults may not have. Finally, as in the case of early treatment for childhood leukemia, trials in children can help inform treatment in adults with similar conditions. In the current era, there are multiple regulations and strategies to optimize safety of pediatric participants in trials. Trial design in children follows the same principles as any other trial, although particular attention needs to be paid to burden on the child and family and understanding parental perceptions of clinical trials. Pediatric drug formulations and devices may be particularly challenging to develop.