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Study: 18.5% of pediatric visits result in off-label drug order :

September 16, 2019

Children were prescribed a drug off-label in about 18.5% of visits to a doctor’s office, and rates have been rising, a new study found.

Laws like the Best Pharmaceuticals for Children Act and Pediatric Research Equity Act have helped improve the information available on prescribing for children, but many drugs still haven’t been studied in young patients.

Researchers from Rutgers University set out to look at trends in off-label prescribing using survey data from a nationally representative group of U.S. office-based physicians treating children from 2006-’15. Investigators included 141 commonly prescribed systemic drugs and considered their use off-label if not based on Food and Drug Administration indications for age, weight and condition.

About 18.5% of pediatric visits included an off-label drug order, equating to about 41.2 million off-label orders per year, according to “Trends in Off-Label Drug Use in Ambulatory Settings: 2006-2015,” (Hoon D, et al. Pediatrics. Sept. 16, 2019,

Across visits with systemic drugs, about 44.5% were off-label, which increased over the course of the study from 42% in 2006-’08 to 47% in 2012-’15, adjusted results show.

About 75% of drug orders that were confirmed off-label or possibly off-label were used to treat unapproved conditions.

Adolescents had the highest rates of off-label drug orders at 322 per 1,000 visits (absolute rates); the most common prescription was nervous system drugs at 123 orders per 1,000 visits.

Across all ages, off-label anti-infectives, respiratory drugs and nervous system drugs were prescribed at the highest absolute rates.

Looking at the rate of off-label orders among all ordered drugs (relative rates), neonates were highest. About 83% of their visits with a drug order included one that was off-label.

Across all ages, female patients, southern clinics, subspecialists and chronic conditions were linked to higher relative rates of off-label drug use.

“Although the prescribing and use of medicines off label is common in pediatrics, off-label drug use is not always off evidence,” authors wrote. “Certain more-common indications we identified as off label were, in fact, supported by high-quality evidence.”

The AAP took a similar stance in its 2014 policy Off-Label Use of Drugs in Children, while continuing its long history of advocating for more research and trials to provide evidence for drug use in children.

Study authors said their findings “can help inform ongoing education, research, and policies around efficacious, effective, and safe use of medications in children.”

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