Children are a vulnerable population who cannot consent for themselves and therefore require additional protections when participating in clinical research. The National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research met in the late 1970s and made recommendations that led to the establishment of protections for children promulgated in federal regulations. These regulations require that, with rare exceptions, children must have some prospect of direct benefit when participating in research that involves more than a low level of risk. Over the last 40 years, medical science has evolved and pediatric drug development programs have become more complex. An increasing social and legal imperative exists to include children in research to generate an evidence base for their treatment.
This expert workshop, convened under a cooperative agreement with the U.S. Food and Drug Administration, will discuss how direct benefit should be defined in the context of contemporary clinical trials. Concepts such as pediatric extrapolation, the appropriate use of non-human data, applicability of biomarkers to reflect clinical benefit, and the ethics of conducting studies in children when alternative strategies like modeling and simulation are applicable will be explored. The goal of this meeting is to formulate expert opinion on a definition of prospect of direct benefit that includes relevant practical considerations to ensure that pediatric clinical research is ethical and collects adequate data to inform the use of therapeutic products in the pediatric population.