Tips for Parents: "Off-Label" Prescribing for Children with Mental Health Problems

Feb 11, 2014
By: Tiffany Farchione M.D., Division of Psychiatry Products, Food and Drug Administration

How often have you asked your child's doctor: “Is this medicine approved by the FDA for use in children?”

It’s an important question that is not often raised, but can help parents and caregivers understand how drugs that treat mental health problems are used and developed for children. This question can also help parents understand the significant progress the FDA has made in making medicines safer and more effective for kids.

FDA-approved labeling provides doctors information about how a drug works, conditions for which a drug has been proven effective, what dose to use, and what side effects might occur. These labels help guide doctors' treatment choices.

However, when a medication has not been studied in children, there is no information in the FDA-approved label to guide prescribing for this age group. Instead, doctors need to rely on alternative sources for this information.

This can lead to what is known as “off-label” use of a prescription drug, or prescribing and use of an FDA-approved drug for a purpose FDA has not specifically approved.

Prescribing a drug approved for adults to a child is just one example of off-label use.

Safety, Effectiveness of Off-Label Prescribing

Off-label prescribing is a common part of medical practice. However, prescribers must be aware that the safety and effectiveness of off-label uses are not necessarily established using the same standards as FDA-labeled uses.

Off-label prescribing can be based on incomplete information, such as whether an effective dose has been selected or what side effects the child may experience.

In pediatric psychiatry, some examples of off-label prescribing are the use of:

Antipsychotics to treat aggression;
Antipsychotic or antidepressant medications as sleep aids; and
Drugs at higher than approved doses.

Absent FDA approval, some treatment choices may be made by doctors based on data that suggest a drug may be effective and safe even if the evidence is not conclusive.

Testing Drug Safety for Children

In recent years, new laws have been enacted to encourage specific testing for safety and efficacy in children. For instance, in 2012, Congress permanently reauthorized two important programs; the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA).

BPCA provides an incentive for drug companies to conduct pediatric studies by granting six additional months of marketing exclusivity. PREA requires drug companies to study their products in children under certain circumstances.

Prior to implementation of these two programs, more than 80 percent of drugs approved for adults had no pediatric safety and efficacy data in their labels. Today that number has been reduced to about 50 percent.

While progress has been significant, there is still much work to be done. Off-label prescribing will continue to be part of the overall landscape of drug therapy in children until most drugs are adequately studied in children.

Don't Be Afraid to Ask Questions

It is useful for parents to understand why their children are receiving particular drugs. Don't be afraid to ask your child's doctor if the treatment prescribed is approved by FDA for use in children.

Sometimes a doctor will select an off-label treatment for a child when an FDA-approved option is available.

Off-label prescribing may be an appropriate approach for your child, but doctors should be able to explain their choice of treatment. They can tell you why they are choosing one medication over another, why they are prescribing a medication for an off-label use, and why they believe it will be effective. You have a right to know!

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