Off-Label Prescribing in Pediatrics

The Food and Drug Administration approved the use of the antidepressant Prozac (fluoxetine) as a treatment for children and adolescents 7 to 17 years of age with depression (major depressive disorder) or obsessive-compulsive disorder (OCD) in 2003.

That new indication followed studies that showed that Prozac was safe and effective for children with these disorders. But just because a medicine isn’t FDA approved for children and adolescents doesn’t mean that it isn’t safe. It simply means that the company that makes the drug has not applied for FDA approval for children and adolescents. Prozac had been safely used in adults since 1987.​

Mother reviewing prescription for daughter in clinic
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Off-Label Prescribing in Pediatrics

Many drugs are not FDA-approved for use in kids but are still used “off-label,” meaning that they are used in children younger than the approved age or for other conditions that they are not approved for yet. In fact, the American Academy of Pediatrics noted in 2002 that “three-fourths of the prescription drugs currently marketed in the United States lack pediatric use information.”

This doesn’t mean that it is not safe to use these medicines though. When deciding to use a medication off-label, a healthcare provider should be making that decision “based on sound scientific evidence, expert medical judgment, or published literature.” It is not like your practitioner is experimenting with your child.

The use of albuterol to treat children with asthma is a good example of the safe off-label use of a drug. Although commonly used in infants and toddlers, albuterol is only FDA-approved for use in children over 2 years old. Many other asthma inhalers, such as Dulera and Advair, are only FDA-approved for use in children over age 4 or 5.

So why are these medications used if they are not FDA approved?

In the case of these asthma medications, there are no other medications to use and these medicines have been used so much, they are thought to be safe and studies have shown that they work. And the alternative, not treating or preventing children’s asthma symptoms, would not be acceptable.

The same is true for depression and other mental health problems, which are unfortunately common in children and sometimes require off-label prescription drugs.

We are seeing more FDA-approved medicines for children with mental health problems, though, including:

  • Lexapro (escitalopram): ages 12 and over with depression
  • Luvox (fluvoxamine): ages 8 and over with obsessive-compulsive disorder (OCD)
  • Zoloft (sertraline): ages 6 and over with OCD
  • Lithium: ages 12 and over with bipolar disorder
  • Orap (pimozide): ages 12 and over with Tourette’s disorder
  • Risperdal (risperidone): ages 5 and over with autism-related irritability
  • Zyprexa (olanzapine): ages 13 and over with schizophrenia or bipolar disorder; in combination with fluoxetine for ages 10 and over with bipolar disorder-related depression
  • Seroquel (quetiapine): ages 10 and over with bipolar mania
  • Abilify (aripiprazole): ages 6 and over with Tourette’s disorder or autism-related irritability; ages 10 and over with bipolar mania; ages 13 and over with schizophrenia

How do you know if your child has been prescribed an off-label medicine?

You could ask your healthcare provider or pharmacist, or simply check the package insert (PI) that came with the medication. In the PI, look for a section called “Indications and Usage” or “Pediatric Use” and see what ages are listed. Remember that just because your child is younger than the listed age doesn’t mean that the medication isn’t safe and you shouldn’t stop it without talking with your healthcare provider.

Downsides of Off-Label Medicine Use

One big downside is that practitioners sometimes have to estimate the dosage to give younger children based on the commonly used dosage for older children and adults. This might lead to children getting too much or too little medicine.

Another downside is that if your healthcare provider doesn’t feel comfortable using drugs off-label then your child might not be getting the best treatment available. Your practitioner might be using an older medication with more side effects or your child might not be receiving any medication at all.

The Pediatric Research Equity Act was instituted by the FDA to try and get drug companies to test their drugs in children. The Best Pharmaceuticals for Children Act also works “to improve the safety and efficacy of pharmaceuticals for children.” Hopefully, they will lead to more drugs being tested and approved for use in children so that they don’t have to be used off-label anymore.

3 Sources
Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read our editorial process to learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.
  1. Committee on Drugs. Uses of drugs not described in the package insert (off-label uses). Pediatrics. 2002;110(1):181-183. doi:10.1542/peds.110.1.181

  2. Frattarelli DA, Galinkin JL, Green TP, et al. Off-label use of drugs in children. Pediatrics. 2014;133(3):563-567. doi:10.1542/peds.2013-4060

  3. National Institutes of Health. Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).

By Vincent Iannelli, MD
 Vincent Iannelli, MD, is a board-certified pediatrician and fellow of the American Academy of Pediatrics. Dr. Iannelli has cared for children for more than 20 years.