Regulating pediatric off-label uses of medicines in the EU and USA: challenges and potential solutions: Comparative regulation framework of off label prescriptions in pediatrics: a review

Benedetta Guidi*, Andrea Parziale, Luca Nocco, Aniello Maiese, Raffaele La Russa, Marco Di Paolo, Emanuela Turillazzi

*Corresponding author for this work

Research output: Contribution to journalEditorialAcademicpeer-review

1 Citation (Web of Science)


Background Off-label uses of medicines are common in pediatrics. The literature reports that at least one-third of children in hospital and up to 90% of newborns in neonatal intensive care units receive off-label prescriptions. Moreover, the lack of data on safety and efficacy in the pediatric population may sometimes increase the risk of adverse drug reactions. Objective This paper aims to (a) map the main gaps in the existing EU and US regulatory frameworks for pediatric drug development and off-label use and (b) propose potential solutions for further discussion. Conclusion The selected jurisdictions aim to limit off-label prescribing, but implementation levels generally seem low, including in pediatric settings. Subject to further research, we propose potential concerted actions and initiatives of international cooperation to fill this gap. In particular, regulators and pediatric societies could strengthen manufacturers' incentives to develop pediatric medicines, pediatricians' information about off-label uses, and patients' and parents' awareness.
Original languageEnglish
Pages (from-to)264-269
Number of pages6
JournalInternational Journal of Clinical Pharmacy
Issue number1
Publication statusPublished - Feb 2022
Externally publishedYes


  • Child
  • Drug-Related Side Effects and Adverse Reactions/epidemiology
  • Humans
  • Infant, Newborn
  • Intensive Care Units, Neonatal
  • Off-Label Use
  • Pediatrics
  • Prescriptions
  • United States
  • Off-patent medicines
  • Off-label prescription
  • European Union
  • Ethical and legal implications
  • Pediatric population

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