Standard 3: data monitoring committees.

* Abbreviations: DMC — : data monitoring committee 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, monitoring can be performed adequately by investigators and sponsors. However, for some trials, a group of independent experts, typically called a data monitoring committee (DMC) or data and safety monitoring board, takes responsibility for this function. DMCs have been a component of certain clinical trials beginning in the 1960s. Initially used in trials funded by government agencies in which the trial objective was to decrease mortality and/or reduce major morbidity, the DMC’s tasks were to carefully monitor the accruing results to ensure that continued enrollment remained appropriate, to consider whether modifications of trial conduct were needed, and to make recommendations to the researchers regarding continuation of the trial with or without changes.2 It was recognized that there were problems with having the investigators themselves perform this interim monitoring because their approach to recruitment and their adherence to the trial protocol could be influenced by their knowledge of interim data. … Address correspondence to Martin Offringa, MD, PhD, Senior Scientist and Program Head, Child Health Evaluative Sciences, Research Institute, The Hospital for Sick Children, 555 University Ave, Toronto, Ontario, Canada M5G 1X8. E-mail: martin.offringa{at}sickkids.ca