Off-label and unlicensed medicine use and adverse drug reactions in children: a narrative review of the literature

Eur J Clin Pharmacol. 2012 Jan;68(1):21-8. doi: 10.1007/s00228-011-1097-1. Epub 2011 Jul 22.


The use of unlicensed and off-label medicines in children is common because trials in children have not usually been performed during the drug development process. Consequently, the information available to paediatricians may not always be as detailed or as robust as that available when prescribing a medicine that is licensed for an approved indication. This has led to concerns that children may be receiving drugs at dosages that either lack efficacy or present safety problems. The latter in particular has received a great deal of attention. In this narrative review, we have evaluated the use of off-label and unlicensed medicines in children and whether and how frequently this predisposes to adverse drug reactions.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Adverse Drug Reaction Reporting Systems
  • Child
  • Drug Eruptions / epidemiology
  • Drug-Related Side Effects and Adverse Reactions / diagnosis
  • Drug-Related Side Effects and Adverse Reactions / epidemiology*
  • Drugs, Investigational / adverse effects
  • Drugs, Investigational / therapeutic use
  • Humans
  • Infant
  • Legislation, Drug
  • Off-Label Use*
  • Practice Patterns, Physicians'


  • Drugs, Investigational