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Off-label drug use in neonates and infants in Spain: A five-year observational study

Irene Lizano-Díez1, Joseph Kargodorian1, M Ángeles Piñero-López1

  • 1Clinical Pharmacy and Pharmaceutical Care Unit, Department of Pharmacy and Pharmaceutical Technology, and Physical Chemistry, Faculty of Pharmacy and Food Sciences, University of Barcelona, Barcelona, Spain.

Pharmacoepidemiology and Drug Safety
|September 10, 2021
PubMed

Insights

Off-label drug prescriptions are common in infants up to one year in Spain, with over a third of total prescriptions being off-label. Most off-label use relates to dosage, indications, or contraindications, highlighting a public health concern.

Area of Science:

  • Pediatric Pharmacology
  • Drug Utilization Research
  • Public Health

Background:

  • Off-label prescribing is common in pediatric populations due to limited drug data.
  • Neonates and infants represent a vulnerable group with unique physiological differences impacting drug response.
  • Understanding off-label drug use is crucial for ensuring patient safety and optimizing therapeutic outcomes.

Purpose of the Study:

  • To determine the rate of off-label drug prescriptions in neonates and infants up to one year old in Spain.
  • To analyze the evidence supporting off-label medication use in this pediatric population.
  • To identify patterns and reasons for off-label prescribing in primary healthcare.

Main Methods:

  • An observational, exploratory study was conducted over five years (2015-2019) in Spanish primary healthcare settings.
  • All drug prescriptions for infants aged 0-1 year were analyzed and categorized as on-label or off-label.
  • Off-label prescriptions were cross-referenced with national formularies and databases (SmPC, Pediamécum, BNF, DailyMed) to assess evidence base.

Main Results:

  • An average of 34.50% of all prescriptions were off-label according to the Summary of Product Characteristics (SmPC).
  • 17.93% of prescriptions lacked clinical evidence from key sources.
  • Off-label use most frequently involved dosage (88%), followed by indications (35.20%) and contraindications (24.10%).
  • Salbutamol, topical tobramycin, and colecalciferol were the most commonly prescribed off-label drugs.

Conclusions:

  • Off-label drug prescribing remains a significant public health concern in neonates and infants.
  • Evidence-based off-label prescribing is a stable and widespread practice, offering flexibility to pediatricians.
  • The findings underscore the need for continued monitoring and evidence generation for pediatric drug use.
Abstract

Keywords:
Spaindrug prescriptioninfantsneonatesoff-label useoutpatientprimary health care

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