For Patients

Understanding Unapproved Use of Approved Drugs "Off Label"

Pill Bottle and Prescription Pad

Has your healthcare provider ever talked to you about using a drug to treat your disease or medical condition that has not been studied before?

Have you heard the term “off-label when your healthcare provider prescribed a drug for you?

It is important to know that before a drug can be approved, a company must submit clinical data and other information to FDA for review. The investigational drug must show that it is safe and effective to use. “Safe” does not mean that the drug has no side effects. But the FDA has determined the benefits outweigh the potential risks.

When FDA approves a drug, it will have drug labeling for healthcare providers. Some drugs may also have information for patients such as Medication Guides, Patient Package Inserts and Instructions for use.  The approved drug label for healthcare providers gives key information about the drug that includes. 

  • Specific disease and conditions that the drug can be used for.

  • How the drug can be prescribed and used.

  • Information about the risks of the drug. 

When your healthcare provider prescribes you a drug and uses it the way it was approved for, you can be sure that the:

  •  FDA has conducted an evaluation of its benefits and risks.  

  • Decision to use the drug is supported by strong scientific data.  

When the FDA approves a drug, healthcare providers may prescribed the drug to treat a disease or medical condition that has not been studied if they judge that it is medically appropriate. 

You may be asking yourself why your healthcare provider would give you a drug that has not been approved to treat you. Remember that there might not be an approved drug to treat you or you may have tried all approved treatments. In situations like these, you and your healthcare providers may talk about using an approved drug for an unapproved use to treat your disease or medical condition.

Unapproved use of an approved drug is often called “off-label”. This term can mean that the drug is.

  • Used for a disease or medical condition that was not studied.  For example when a chemotherapy is approved to treat one type of cancer, but healthcare providers use it to treat a different type of cancer.

  • Given in a different way (such as by a different way of taking the drug).

  • Given in a different dose.

If your healthcare provider decides to use an approved drug for an unapproved use to treat you,  remember that FDA has not studied to see if that the drug is safe and effective for the way that you might using it.

Questions You May Want to Consider 

If your healthcare provider is thinking about using an approved drug for an unapproved use, you may want to ask questions like these:

  • Are there other drugs that are approved to treat my disease or medical condition?

  • What is the drug approved for?

  • What scientific studies are available to support the use of this drug to treat my disease or medical condition?

  •  Is it likely that this drug will work better to treat my disease or medical condition?

  • What are the potential benefits and risks of treating my disease or medical condition with this drug? 

  •  Will my health insurance cover treatment of my disease or medical condition with this drug?  

  • Are there any clinical trials studying the use of this drug for my disease or medical condition that I could enroll in?

Page Last Updated: 05/26/2016
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