Trends in Off-Label Drug Use in Ambulatory Settings: 2006-2015

Area of Science:

• Pediatric Pharmacology
• Drug Utilization Research
• Public Health

Background:

• Off-label drug use in children is prevalent and poses potential risks.
• Previous research often focused on hospitalized children or non-US settings.
• Characterizing ambulatory US settings is crucial for understanding pediatric prescribing patterns.

Purpose of the Study:

• To determine the frequency, trends, and reasons for off-label systemic drug orders in US pediatric ambulatory care.
• To identify factors associated with off-label prescribing in children.
• To inform strategies for improving safe and effective medication use in pediatric populations.

Main Methods:

• Analysis of nationally representative National Ambulatory Medical Care Surveys (2006-2015).
• Inclusion of children aged <18 receiving systemic drugs.
• Assessment of off-label status based on US Food and Drug Administration (FDA) labeling for age, weight, and indication.

Main Results:

• Off-label systemic drug orders occurred at 18.5% of pediatric visits, most commonly for unapproved conditions (74.6%).
• Off-label use was highest in neonates (83%) and substantial in adolescents.
• Rates of off-label orders increased over time, with rising use of antihistamines and psychotropics, and stable/declining antibiotic use.

Conclusions:

• US office-based physicians are increasingly prescribing systemic drugs off-label to children, predominantly for unapproved indications.
• Despite efforts to expand pediatric drug approvals, significant off-label prescribing persists.
• Findings underscore the need for enhanced education, research, and policy development to ensure safe pediatric medication practices.