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Off-label use

What is off-label use? 

Off-label use refers to the unapproved use of an FDA-approved drug.  

When a drug is submitted for FDA review by a company, the manufacturer submits clinical data and other information showing the drug is safe and effective for its intended use, which usually includes specific diseases and conditions. It is for these medical concerns that the drug is then approved to treat. However, a drug may show benefits for those who have different medical concerns, leading a doctor to prescribe the medication “off-label.”  

Some examples of off-label use include:  

  • Giving a drug in a different way (e.g., when the drug is approved as a capsule but given in an oral solution)
  • Using the drug for a medical condition or disease that it is not approved to treat  
  • Giving the drug in a different dose than what is approved  

Why do physicians prescribe drugs off-label?

There are many reasons why a healthcare provider may prescribe a drug off-label. For example, there may not be an approved drug available to treat a patient’s disease or medical condition, so the provider instead prescribes something unapproved that affects the body or bodily processes in a way that will help the patient. Similarly, if there are approved medications for a specific disease or medical condition, but the patient has seen no benefits, their doctor may prescribe another drug off-label.  

In general, healthcare providers prescribe off-label drugs to meet an area of need within healthcare, such as inadequate or nonexistent treatments. Furthermore, when prescribed to be taken in a different way, it may be easier for a patient to take their prescriptions.