Children are generally regarded as “vulnerable individuals.” There has long been reluctance to perform research in children except in limited settings such as pediatric cancer and vaccine development. However, avoidance of trials in children has resulted in uncertainty regarding efficacy, safety, appropriate formulations, and dosages of treatments in pediatric populations, a situation now recognized as problematic.1 As more pediatric clinical trials are undertaken, it is timely to focus on special issues in their design and conduct. One such issue is the monitoring of accumulating data from ongoing clinical trials.

Monitoring of safety outcomes during any trial is always required. In some trials, monitoring for efficacy is also an essential component of safety assurance, such as when efficacy is measured based on an unfavorable outcome or if a treatment is quickly discovered to be a major advance in terms of saving lives or preventing another serious outcome. In many cases,...

You do not currently have access to this content.