Off‑label and unapproved pediatric drug utilization: A meta‑analysis

Area of Science:

• Pediatric Pharmacology
• Global Health
• Drug Safety

Background:

• Off-label and unapproved drug use in pediatric populations remains a significant concern despite regulatory efforts.
• Ensuring safe and effective medication use in children is critical for their health outcomes.

Purpose of the Study:

• To determine the global prevalence of off-label and unlicensed drug prescriptions among hospitalized children.
• To analyze geographical variations in pediatric off-label and unapproved drug use.

Main Methods:

• A comprehensive meta-analysis was conducted on studies published between 1990 and 2023.
• Searches were performed across major scientific databases including PubMed, Scopus, EMBASE, Web of Science, and Google Scholar.
• Data from 45 eligible studies reporting pediatric off-label and unlicensed drug prescriptions were synthesized.

Main Results:

• The global prevalence of off-label and unlicensed drug prescriptions in hospitalized children was found to be 56%.
• Prevalence rates varied geographically, with Africa reporting the highest rate (66%), followed by Asia, South America, North America, Australia, and Europe.
• Included studies encompassed a wide range of patient sample sizes and total prescriptions issued.

Conclusions:

• Off-label and unlicensed drug prescriptions in pediatric patients are highly prevalent and geographically widespread.
• There is a critical need for drug authorities to establish standardized pediatric prescription guidelines to improve patient safety.