Department of Health and Human Services

Patient Network

Bringing Your Voice to Drug and Device Approval and Safety

Off-Label Use of Approved Drugs

The drug label of FDA-approved drugs gives information about the drug, including the approved doses and how it's to be given to treat the medical condition for which it was approved. When a drug is used in a way that is different from that described in the FDA-approved drug label, it is said to be an "off-label" use. This can mean that the drug is:

  • Used for a different disease or medical condition.
  • Given in a different way (such as by a different route).
  • Given in a different dose.

For example, when a chemotherapy drug is approved for treating one type of cancer, but is used to treat a different cancer, it is off-label use.

Off-label is also called "non-approved" or "unapproved" use of a drug. New uses for these drugs may have been found, and often medical evidence supports the new use. But the makers of the drugs have not put them through the formal, lengthy, and often costly studies required by FDA to officially approve the drug for new uses.

What Questions Should I Ask my Healthcare Provider About Off-Label Use?

If you and your Healthcare Provider are considering off-label use of an approved drug, consider asking these questions before deciding whether it is right for you.

  • Is there information available to support the off-label use of this drug to treat my condition?
  • Is this off-label drug likely to work better than an approved drug?
  • What are the risks and benefits of off-label treatment with this drug?
  • Will my health insurance cover off-label treatment with this drug?