Unlicensed and Off-Label Drug Use in Children Before and After Pediatric Governmental Initiatives

Area of Science:

• Pediatric pharmacology
• Drug utilization research
• Regulatory science

Background:

• Regulatory agencies like the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) introduced policies to enhance pediatric clinical research.
• These initiatives aimed to address the historical lack of data and improve the safe and effective use of medications in children.

Purpose of the Study:

• To quantify the prevalence of unlicensed and off-label drug use in pediatric populations.
• To compare these rates before and after the implementation of major regulatory initiatives in the US and Europe.

Main Methods:

• A comprehensive literature review was conducted using PubMed and Google Scholar, covering studies from 1985 to 2014.
• Articles were selected based on their evaluation of unlicensed/off-label drug use in pediatric populations within defined healthcare settings.
• Included studies were categorized by geographical region (US, Europe, other countries) and by their timing relative to the 2007 regulatory implementation in Europe.

Main Results:

• In Europe, unlicensed drug use rates decreased marginally post-initiative, from 0.2%-36% (inpatients) and 0.3%-16.6% (outpatients) to 11.4% and 1.26%-6.7%, respectively.
• Off-label drug use in Europe also saw a marginal decrease, from 18%-66% (inpatients) and 10.5%-37.5% (outpatients) to 33.2%-46.5% and 3.3%-13.5%, respectively.
• In other countries, reported rates for unlicensed and off-label drug use were 8%-27.3% and 11%-47%, respectively.

Conclusions:

• Governmental initiatives aimed at improving pediatric clinical research appear to have had a limited, albeit positive, effect on reducing unlicensed and off-label drug use in Europe.
• The findings suggest a need for continued monitoring and potentially stronger regulatory actions to further minimize these practices in pediatric care.