Off Label Use as an Indicator of Therapeutic Need in Pediatrics

Abstract

There are therapeutic areas where the unauthorized use of a medicine is very wide, for example the pediatric field. The spread of off-label use in the pediatric field derives mainly from the difficulty in starting trials in this context, situation which it has long been reflected in a social and ethical paradigm. The pediatric population should be protected from research, but the difficulty in starting trials involving this population makes it “orphan” of authorized therapies. Ethical and, sometimes, methodological and economic issues make the pediatric trials “unattractive”. Furthermore, children are not a homogeneous population; in fact, within this category it is possible to distinguish different groups based on age groups characterized by biological diversity and for which starting a trial would be an expensive and complex process [2].