Be Aware: Study Shows Increased Use of Off-label Antipsychotic Drugs in Children

Sixty-five percent of children prescribed antipsychotics in 2007 were using the drugs “off-label,” or without FDA safety and efficacy data to support their use to treat young patients.

A national study conducted by researchers at The Children’s Hospital of Philadelphia (CHOP) shows increased use of powerful antipsychotic drugs to treat publicly insured children over the last decade. The study, published today in the journal Health Services Research, found a 62 percent increase in the number of Medicaid-enrolled children ages 3 to 18 taking antipsychotics, reaching a total of 354,000 children by 2007.

Increased use of antipsychotic drugs in children seen across a wide range of mental health diagnoses

Increased antipsychotic use was observed across a wide range of mental health diagnoses, and was particularly high for children with ADHD or conduct disorder, although the FDA has not approved the drugs to treat these conditions in children. In total, 65 percent of children prescribed antipsychotics in 2007 were using the drugs “off-label,” or without FDA safety and efficacy data to support their use to treat young patients. The CHOP study is the second released this month that focuses on the use of antipsychotic drug use in children and is largest of its kind, representing 35 percent of children in the country.

Antipsychotic drug use in children a pediatric research priority for National Institutes of Health

“Given the significant proportion of off-label use of antipsychotics in children, it is reassuring that these drugs have been recognized as a priority for pediatric research by the National Institutes of Health,” said David M. Rubin, MD, MSCE, a senior author of the study, attending pediatrician, and co-director of CHOP’s PolicyLab. “If a child is prescribed an antipsychotic, it’s important for doctors to inform parents and caregivers if the drug is being prescribed off-label, of potential side effects, and of counseling therapies that might be offered as an alternative to medication.” The frequent off-label use of antipsychotics has raised concern among many healthcare providers, especially in light of evidence linking antipsychotics with an increased risk of serious metabolic side effects in children, including weight gain and diabetes.

Increase in use partly due to increase in mental health diagnoses in children

The researchers note that the increase in antipsychotic use is due to in part to an overall increase in the number of mental health diagnoses assigned to children. Researchers found a 28 percent increase in the number of children with a mental health diagnosis, but this alone did not account for the spike in prescriptions. “We knew that the number of children prescribed antipsychotics had grown steadily over the past two decades, particularly among children with public insurance,” said Meredith Matone, MHS, the study’s lead author and a researcher at PolicyLab. “With this study, we wanted to learn more about why these drugs are being used so often, what diagnoses they’re being used to treat, and how prescribing patterns changed over the course of the last decade.”

While schizophrenia, bipolar disorder and autism were the most likely diagnoses to result in an antipsychotic prescription, children with these disorders did not make up the majority of antipsychotic users. Children with ADHD and those who were diagnosed with three or more concurrent mental health disorders made up the largest group of children taking antipsychotics. In 2007, 50 percent of children taking antipsychotics had a diagnosis of ADHD, and 14 percent had ADHD as their only diagnosis.

“The fact that we see an uptick in prescribing antipsychotics for many diagnoses tells us that antipsychotics are likely being used to treat specific behaviors, like aggression, that are shared among a variety of mental health diagnoses,” explained Matone. “Insights like this are only available by conducting very large-scale studies like this one. Reaching an average of 15 million children a year provided the needed national perspective on medication use. Continuing to conduct population-based, public health studies is crucial to inform policies and guidelines for the use of antipsychotics for children.”

Press release The Children’s Hospital of Philadelphia