Is It Safe to Use Medications Without FDA Approval?


Is it safe to use medications that don’t have FDA approval for use in children?


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

FDA Approval

When drug companies develop a new medicine, they do a series of tests to make sure it is safe and effective before it can be sold and prescribed. The U.S. Food and Drug Administration (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

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. There are many off-label uses that most doctors agree are safe and effective. But some of these uses may never be approved by the FDA. Once a medicine is approved for one purpose, the company often just doesn’t bother to get it approved again. The process costs a lot of money.

Continued research into how a medicine works for unapproved uses helps doctors make these decisions. This research is often not done by the drug company itself. It’s done by other scientists and doctors who want to study how a medication works. They publish this research in peer-reviewed journals. This 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:

is mostly identified and treated in childhood or adolescence. So drug companies work to get their medications approved for that age group. But ADHD continues to affect some people into adulthood. 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 FDA approved for kids. Again, a doctor must use his or her knowledge and experience to decide whether these drugs might help treat a psychiatric disorder in a child.

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 without approval there aren’t specific instructions on the label. The doctor depends on his or her training and the knowledge of other doctors.

What You Should Ask

If your child is being treated for a psychiatric disorder with an off-label medication, you should be sure the doctor can explain why. The doctor should also explain the possible side effects and what he or she thinks will happen. As 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. The FDA posts that information online.

Doctors and patients can and do report bad experiences. So the FDA never stops watching these medicines. 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 toward 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.

About the author

About the author

Child Mind Institute is dedicated to transforming mental health care for children everywhere.