Was that medicine you just gave your toddler tested on children? This is a question that few parents ask themselves, but probably should. A large new study found that doctors are increasingly prescribing “off-label” drugs to kids, medications that the FDA has either not approved for children or not approved for the specific condition being treated. A whopping one in five visits to a pediatrician resulted in an off-label drug being prescribed. This means that if your toddler needed medicine — from antibiotics to antihistamines — there’s a better than 20% chance it was a drug meant for, and tested on, adults.
Why could this be a problem? Because children are not just small adults. They weigh a lot less, yes, but also, their body and brain makeup differs, meaning they metabolize medicines differently and might experience different side effects. These factors might not matter — regardless of physiological differences, a drug may be just as effective for a child as an adult — but unless a medication is put through a clinical trial of children specifically, we can’t know that for sure.
To be clear, off-label drug ordering is perfectly legal and doctors have been doing it for decades. In some cases, especially when faced with serious or life-threatening medical conditions, an off-label drug may be the best or only option. However, this practice always carries some degree of risk, and many parents have no idea it’s even happening, let alone that it’s is on the rise.
41 Million Off-Label Prescriptions Every Year
For the new study published in the American Academy of Pediatrics’ journal Pediatrics, researchers pored over 1.7 billion pediatric office visits over a ten-year period. They found that nearly one in five visits resulted in kids being given one or more off-label drug. And among visits with at least one medication ordered, the rates of ordering off-label increased from 42 percent in 2006 to 47 percent in 2015. Therefore, as it stands now, more than 41 million off-label meds are ordered for children every year.
A big reason why this so widespread is there just aren’t enough drugs that have been rigorously tested in children, a requirement for FDA approval. “Traditionally, children were excluded from most clinical trials on the premise that they are a vulnerable population that needs protection from the risks,” says Dr. Daniel Horton, senior author of the study and assistant professor of pediatric rheumatology at Rutgers Robert Wood Johnson Medical School. “The irony is that excluding children has made them even more vulnerable to the use of untested medicines.”
That said, Horton insists that lack of FDA approval doesn’t always mean that a drug has undergone zero research on children or that doctors are taking a total shot in the dark. “Many drugs given off-label to children are in fact supported by either some scientific evidence or collective clinical experience of treating kids with these meds — or both,” he explains. “Their use is often because there are no approved options available or because approved alternatives did not work. To be honest, sometimes the only way children can get treated for conditions is to use one of these medications off-label.”
As a pediatric rheumatologist, Horton sees many children with diseases that fit this profile. “I commonly prescribe kids off-label drugs when I think the benefits should outweigh the risks,” he says. “Also, I am a father of two young kids, and if they needed treatment for something with no approved options, I’d certainly want them treated with whatever is thought to work.”
The Risks of Off-Label Prescriptions
In many off-label scenarios, there is not sufficient evidence that a drug works — and there is plenty suggesting they can cause problems for children. “For instance, medications that treat widespread pain such as fibromyalgia have been shown to be not as effective in teens and kids,” Horton says. “And since these meds have a lot of known side effects, in the absence of known benefits, they are almost always not worth the risk for children.”
Another example is acid-suppressive medications, used to treat gastric reflux. “We found that babies with gastroesophageal reflux are commonly treated off-label with acid blockers, but most studies have shown that these medications don’t work well for them,” Horton says. “Although acid-suppressive drugs may be generally safe, recent literature suggests they could carry harms for kids, such as more fractures or the development of allergic diseases.” Given a lack of clear benefit plus the documented downsides, giving acid blockers to babies seems unnecessarily risky.
The study also found an increase in off-label usage of antidepressants. In this case, Horton says both scenarios are happening: Kids with depression are being treated with antidepressants that are approved for adults but not children, and kids who do not have depression, but instead have ADHD or obsessive-compulsive disorder, are being treated with antidepressants. The latter circumstance is a double-whammy because not only are these the wrong drugs, they also haven’t been approved for their age or their condition.
“Antidepressants carry many side effects, including the rare but serious suicidal ideations and suicidality, particularly in young people,” Horton says. “Therefore, it is important that we generate better evidence about these medications for children.”
What Parents Can Do About Off-Label Drugs
As it stands, parents are no informed about the evidence or lack of evidence whenever their kid is prescribed an off-label medicine. That’s why it’s up to parents to ask. Is this off-label drug? Is there an alternative? And what are the risks? Once you have all the info, then you can weigh whether the risks versus rewards, and have a serious discussion with your pediatrician.
“The gravity and severity of the condition influences how we think about risks and benefits of medications,” Horton says. “When a child’s life or quality of life is being threatened, then parents are generally more willing to tolerate greater chances.” In the case of allergies, for example, some kids may not be bothered by them at all, so the risk of off-label medications may seem too great for that family. For others, allergies are hugely impactful to a kid’s life — making it much worse and even retarding their development. This kid’s parents would probably take the chance and give it a shot, with close monitoring. It turns out a bit of parental oversight is just what the doctor ordered.