Physicians continue to prescribe off-label drugs for children, with rates increasing over a 10-year period from 2006 to 2015, according to findings from a new study.
The increase occurred despite recent legislation aimed at encouraging pediatric clinical trials, with the intention of improving the “quality of evidence and the number of drugs approved for children,” Divya Hoon of Rutgers University in New Brunswick, N.J., and colleagues wrote in Pediatrics.
“[Our] results can help inform ongoing education, research, and policies around efficacious, effective, and safe use of medications in children,” the researchers said.
To determine trends in, and categories of, drugs prescribed off label, the researchers used data from the National Ambulatory Medical Care Surveys for all pediatric visits and subsequent drug orders from 2006 to 2015. They focused on 141 drugs that are predominantly or exclusively used in systemic formulations and that had been ordered at least 30 times.
At least one off-label systemic drug order occurred at 18.5% of the 1.74 billion estimated ambulatory pediatric visits (95% confidence interval, 17.7%-19.3%), totaling 41.2 million off-label orders per year. The primary reason for a drug being considered off label was that it was for an unapproved condition (74.6%), followed by patient age (17.6%) and weight (0.6%). Absolute and relative rates of off-label ordering increased throughout the study, especially in regard to antihistamines and psychotropic drugs, the investigators said.
In an accompanying editorial, Katelyn Yackey, MD, of the University of Kentucky Children’s Hospital, Lexington, and Rachel Stanley, MD, of Nationwide Children’s Hospital, Columbus, Ohio, stated that “off label is not synonymous with off evidence” and emphasized the need for more clinical trials of medications for children (Pediatrics. 2019 Sep 16. doi: 10.1542/peds.2019-1571).
“Although drugs are often used off label, there may be sufficient preliminary research about a medical condition and particular drugs to support their use,” they wrote. While recognizing that evaluating medications in pediatric patients has been challenging, they added that “children continue to receive medications off label and for unapproved conditions,” so studies that evaluate “safety, efficacy, pharmacokinetics, and optimal dosing in pediatric patients” remain a necessity.
Though the research featured a long study period and large study population, the authors recognized its possible limitations, including the exclusion of less commonly ordered drugs, the inability to determine drug formulation or dosage, and the fact that the survey data captured only ordered medicines and not whether they were actually dispensed or consumed.
The study was funded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases and the Rutgers Robert Wood Johnson Medical School Summer Research Fellowship. The authors reported no conflicts of interest.
SOURCE: Hoon D et al. Pediatrics. 2019 Sep 16. doi: 10.1542/peds.2019-0896.