Two decades of off-label prescribing in children: a literature review

Shamala Balan, Mohamed Azmi Ahmad Hassali, Vivienne S.L. Mak

Research output: Contribution to journalArticleResearchpeer-review

27 Citations (Scopus)


Background: In the past two decades, many legislative and regulatory initiatives were taken globally to improve drug use in children. However, children are still found to be prescribed with off-label drugs. This study was conducted to provide an overview of the worldwide trend in off-label prescribing in children from the year 1996 to 2016. 

Data sources: The articles published in PubMed, MEDLINE and Google Scholar were searched using text words: off-label, unlicensed, paediatric and children. Additional articles were identified by reviewing the bibliography of the retrieved articles. Full-text articles published in English which reported on the prevalence of off-label prescribing in children between January 1996 and December 2016 were included. 

Results: A total of 101 studies met the inclusion criteria. Off-label prescribing definition included four main categories: age, indication, dose and route of administration. The three most common reference sources used in the studies were summary of product characteristics, national formularies and package inserts. Overall, the off-label prescribing rates in children ranged from 1.2 to 99.7%. The most common category of off-label prescribing in children was dose and age. 

Conclusions: This review highlighted that off-label prescribing in children was found to be highly prevalent throughout the past two decades, persistently in the neonatal intensive care units. This suggests that besides legislative and regulatory initiatives, behavioural, knowledge aspects and efforts to integrate evidence into practice related to off-label prescribing also need to be evaluated and consolidated as part of the concerted efforts to narrow the gaps in prescribing for children.

Original languageEnglish
Pages (from-to)528-540
Number of pages13
JournalWorld Journal of Pediatrics
Issue number6
Publication statusPublished - 2018


  • Children
  • Dose
  • Indication
  • Off-label
  • Pediatric

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