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 does not mean that it is not safe. It simply means that the drug company that makes the drug has not applied to the FDA for approval. 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, according to the American Academy of Pediatrics, '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 doctor should be making that decision 'based on sound scientific evidence, expert medical judgment, or published literature.' It is not like your doctor 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 four.

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) kids (12 and over) with depression
  • Luvox (fluvoxamine) kids (8-17) with OCD
  • Zoloft (sertraline) kids (6-17) with OCD
  • Lithium kids (12 and over) with bipolar
  • Orap (pimozide) kids (12 and over) with Tourette's disorder
  • Risperdal (risperidone) autistic kids (5 and over)

Other medications, like Lithium, Zyprexa (olanzapine), Seroquel (quetiapine), and Abilify (aripiprazole) and approved for children with bipolar disorder or schizophrenia.

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

You could ask your doctor 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 doctor.

Downsides of 'Off-Label' Medicine Use

One big downside is that doctors 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 doctor doesn't feel comfortable using drugs 'off-label' then your child might not be getting the best treatment available. Your doctor might be using an older medication with more side effects or your child might not be receiving any medication at all.

The Pediatric Drug Rule 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.

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  • American Academy of Pediatrics Policy Statement. Off-Label Use of Drugs in Children. Pediatrics. March 2014, VOLUME 133 / ISSUE 3