Use of off-label and unlicenced drugs in hospitalised paediatric patients: a systematic review

Eur J Clin Pharmacol. 2015 Jan;71(1):1-13. doi: 10.1007/s00228-014-1768-9. Epub 2014 Oct 16.


Purpose: The aim of this review is to assess the extent of the use of off-label and/or unlicenced drugs among hospitalised children.

Methods: A systematic search was made in MEDLINE-PubMED for papers published from 1994 to 2012, addressing the prescription of off-label and/or unlicenced drugs for the paediatric population in hospital care.

Results: Of the 829 studies retrieved, 34 met the inclusion criteria. Prescriptions ranged from 12.2 to 70.6 % for off-label and from 0.2 to 47.9 % for unlicensed drugs. The percentage of children who received at least one off-label and/or unlicensed drug ranged from 42.0 to 100 %, with newborns being the population that received most of such drugs. Off-label prescriptions were essential for dose modification (7.1-73.1 %) and unlicensed prescriptions for formulation modification purposes (3.6-100 %).

Conclusions: These findings show that: (i) off-label and/or an unlicensed prescribing is widespread among the hospitalised paediatric population worldwide, (ii) there is no consensus on a definition of off-label and/or unlicensed drugs and (iii) preterm newborns receive most off-label and/or unlicensed drugs. By shedding new light on off-label and/or unlicensed drug prescribing, these findings will hopefully contribute to generating new, more effective knowledge about the paediatric population's need for quality drugs that are both safe and efficacious.

Publication types

  • Review
  • Systematic Review

MeSH terms

  • Child
  • Drug Approval
  • Drug Utilization / statistics & numerical data*
  • Hospitalization / statistics & numerical data*
  • Humans
  • Off-Label Use / statistics & numerical data*

Associated data

  • EudraCT/2010-023527-18