European Journal of Pediatrics

, Volume 164, Issue 9, pp 552–558

A literature review on off-label drug use in children

Original Paper

DOI: 10.1007/s00431-005-1698-8

Cite this article as:
Pandolfini, C. & Bonati, M. Eur J Pediatr (2005) 164: 552. doi:10.1007/s00431-005-1698-8


The aim was to compare results of studies performed in different settings worldwide and identify common therapeutic areas to allow for focused interventions, because off-label drug use can be a measure of the lack of knowledge concerning paediatric treatments. A secondary objective was to provide a brief review of efforts to date. A literature review of articles on off-label and unlicensed drug use in children involving general prescription samples was performed using Medline and Embase. In all, 30 studies from 1985–2004 were included. Eleven involved paediatric hospital wards, seven neonatal hospital wards, and 12 the community setting. The off-label and unlicensed classification methods varied, making results difficult to compare. In general, off-label/unlicensed prescription rates ranged from 11%–80%, and higher rates were found in younger versus older patients and in the hospital versus community settings. On the paediatric hospital wards, off-label/unlicensed prescriptions ranged from 16%–62% and most often concerned acetaminophen, cisapride, chloral hydrate, and salbutamol. In the neonatal wards, rates ranged from 55%–80% and often involved caffeine. In the community setting, rates ranged from 11%–37% and the most commonly implicated drugs were salbutamol and amoxicillin. Conclusion:A lack of harmonisation between the evidence, the information available to doctors, and its use in clinical practice exists and this is part of the reason off-label therapies are so common. Attempts have been made to improve knowledge concerning paediatric treatments, but more focused interventions are needed, also taking into consideration this lack of harmonisation.


DrugDrug labellingLegislationPaediatricsPrescriptions

Copyright information

© Springer-Verlag 2005

Authors and Affiliations

  1. 1.Laboratory for Mother and Child Health“Mario Negri” Institute for Pharmacological ResearchMilan Italy