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Using Medications Without FDA Approval

'Off-label' drugs have been and can be safely prescribed to children, but care is still needed

en Español

If you have a child struggling with anxiety, depression, or some other mental illness, your doctor may prescribe a medication that does not have FDA approval for use in children. Let’s take a look at what that means.

When drug companies develop a new medicine, they need to do a series of tests to make sure it is safe and effective before it can be sold and prescribed. This process is called US Food and Drug Administration (FDA) approval, and it is required of all new drugs.

FDA approval

But the FDA only “approves” the medication for the particular use the company made it for, and for a specific group of patients. Drugs can be approved for:

  • A specific disease or diseases
  • A specific age group
  • A specific problem (like pain)
  • A specific health condition, like weight or pregnancy

So while a drug might be “approved” for certain people, that doesn’t mean it is approved for everyone to use, because it hasn’t been tested on everyone. But that doesn’t mean that doctors can’t prescribe it—or that it might not be helpful.

Once a medicine is FDA approved for one use, doctors can decide whether it is right for another patient to use. This is called prescribing medication “off-label.” It’s called “off label” because the label on a medication indicates what it’s approved for by the FDA,.

Prescribing medication off-label is fairly common, and there are many off-label uses that most doctors agree are safe and effective. But some of them may never be approved by the FDA, because once a medicine is approved for one purpose, the company often just doesn’t bother to get it approved again, because the process costs a lot of money.

In the meantime, there is a lot of research into how a medicine works for unapproved uses that helps doctors make these decisions. This research is often not done by the drug company itself, but by other scientists and doctors who want to study how a medication works. They publish this research in “peer-reviewed journals,” which means that other scientists look at the work and decide if it is high quality.

Real life examples

What does this mean for parents and caregivers of children on psychiatric medication? Let’s look at two examples of medications:

Because ADHD is mostly identified and treated in childhood or adolescence, drug companies worked to get their medications approved for that age group. But ADHD continues to affect some people into adulthood, and medications used to treat it are still effective. Still, many ADHD medications aren’t FDA approved for adults.

Does this mean the medications aren’t safe and effective? No. It means that doctors who prescribe them to adults must use their medical knowledge to decide if a medication might help symptoms, and they must monitor its effects. This is what doctors should do with all medications.

In the case of antidepressants, many medications approved by the FDA for use in adults aren’t approved for children and adolescents. These medications have proven helpful for treating everything from depression and anxiety to traumatic stress and OCD. There is substantial research showing that they also help kids with these problems.

But they aren’t “approved” for kids. Again, a doctor must use his or her knowledge and experience to decide whether they might help treat a psychiatric disorder.

How are children different?

Whenever physicians prescribe “off-label” to a child they need to think about that child’s body weight and stage of development. This is the same thing a doctor would do with an “approved” medication, but there aren’t specific instructions on the label. The doctor depends on his or her training and the knowledge of other doctors.

Think about aspirin, or acetaminophen. A child gets a child’s dose. It’s the same with prescription medications for things like depression or anxiety.

What you should ask

When treating a child for a psychiatric disorder, you should find an experienced professional who can explain why he or she is prescribing an off-label medication. The doctor should also explain the possible side-effects and what he or she thinks will happen. Like with any medication, you should also ask how long your child will be on the medication.

You can also ask how long the medication has been on the market, and if there have ever been any red flags. One good thing about medications that have been FDA approved for a use (even if it’s not what you’re using it for) is that drug companies are required to tell the FDA whenever someone has a bad experience with the medication, and the FDA posts that information online.

Doctors and patients can and do also report bad experiences. So the FDA never stops watching these medicines, and it can stop them from being sold if there is a problem.

Unapproved medication

There are still some medications that have never been approved by the FDA for any use. No one should take these drugs. If a medication has never been through the FDA approval process, we don’t know enough about it to know if it is safe.

There are other things, like supplements and homeopathic remedies, that don’t have to go through FDA approval because they aren’t “drugs.” You should talk to a trained professional about using things like this to treat your child’s problems.

In the future

The good news is that there is a growing amount of research on the use of medications in kids, even if this isn’t leading towards eventual FDA approval. Companies may not have any incentive to get approval for new uses. A lot of this research is done by independent scientists doing clinical trials to improve care for kids.

For clinicians, this research is an important guide to which medications can be helpful for kids with psychiatric illness.

And remember: FDA approval gives scientists and doctors a lot of information about a medication, including information that isn’t related to the use it is being tested for. And every medication prescribed by a good doctor is FDA approved, even if he or she is prescribing “off-label.”

Frequently Asked Questions

What common drugs are not FDA approved?

Many ADHD drugs are not FDA approved for adults, and some common antidepressant drugs approved for adults are not FDA approved for children and adolescents. Drugs that are not FDA approved for specific groups are still often prescribed off-label under a doctor’s supervision.

This article was last reviewed or updated on December 5, 2023.