When a child gets sick, doctors are increasingly relying on what’s known as “off-label” use of medications, a new study says.
Off-label use of a drug means that it hasn’t been specifically studied and approved for the condition, age group or weight of the person getting the prescription.
For example, kids with asthma may be prescribed antihistamines (approved for allergies, but not specifically for asthma), because they may have allergies that trigger their wheezing, the researchers noted.
The study found that doctors prescribed one or more off-label drugs for children in almost 1 out of every 5 office visits.
“Off-label doesn’t mean a drug is harmful. There’s often good evidence behind the use of off-label drugs,” said study senior author Dr. Daniel Horton. He’s an assistant professor of pediatrics and epidemiology at the Rutgers Robert Wood Johnson Medical School in New Brunswick, N.J.
“But sometimes there isn’t good evidence, so it’s important for parents to discuss with the child’s doctor what is known and not known about off-label drugs, so they can try to ensure that the benefits of a drug outweigh the risks,” Horton said.
Doctors often turn to off-label drug use because no alternative exists, the authors said.
“Historically, children have been excluded from clinical trials of medications. This left a large gap in knowledge. Over time, legislation and policies to require more research have been developed,” Horton said.
The study is being published in the October issue of the journal Pediatrics, available online Sept. 16.
Dr. Rudolph Valentini, chief medical officer at Children’s Hospital of Michigan in Detroit, agreed that drug trials in children can be challenging.
He said sometimes parents don’t want their children involved in drug research trials. Another issue, he noted, is that “some diseases are just less common in children,” such as high blood pressure. Valentini was not involved with the new study.
Horton and his colleagues used nationally representative surveys from doctors’ offices, including about 1.74 billion visits for children under 18. During these visits, 18.5% of children were given a prescription for one or more off-label drugs.
That means more than 41 million off-label drugs were ordered for children each year, the researchers said.
Off-label drug use was most common in teens and least common in newborns. However, when newborns needed medications, they were prescribed an off-label drug 83% of the time.
Girls were more likely to receive off-label prescriptions, as were patients in the South. Children and teens with chronic conditions and those who needed more than one type of medication were also more likely to be given an off-label prescription.
The types of drugs most commonly prescribed off-label to children under 18 were antihistamines (allergy medications), antibiotics and antidepressants. Antihistamines and antibiotics were often prescribed for respiratory infections, the researchers said. Antidepressants were often prescribed to treat attention deficit hyperactivity disorder.
“Pediatricians and other doctors that care for children are doing the best they can with the best evidence available. Almost all of the medications used off-label in children have a long track record in the adult world,” Valentini explained.
And, he said, doctors are prescribing the medications because they believe their benefit outweighs the risk.
For example, if a baby is in intensive care and needs an off-label sedative because of a painful condition, Valentini said doctors are concerned that a drug might affect the growing brain, but not treating the pain wouldn’t be good for the child either.
“Every decision we make weighs risk versus benefits. If it’s not clear, we will have a serious conversation with the family. Parents should always ask questions and be the advocate for their child. Your child’s doctor should be happy to explain why they’ve chosen a particular medicine,” Valentini said.
Horton said the findings highlight the need for more research and more effective policies to encourage that research.
The U.S. Food and Drug Administration explains more about off-label drug use.