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Reporting a Medication Error

What Does “Off-Label” Use Mean?

Published May 6, 2024

The off-label use of a medicine means that the US Food and Drug Administration (FDA) has not specifically approved the medicine to be prescribed for the condition it is being used to treat. When the FDA approves a medicine, that approval is based on studies showing that it is safe and effective to treat the specific conditions that were studied. But, many medicines have been used safely and effectively to treat other conditions that were never approved by the FDA. In fact, doctors often prescribe medicines for their off-label uses.  

Off-label use not only includes prescribing a medicine for a different condition, but it can also be for different age groups. There are several medicines that are prescribed off-label for children because the effects of medicines are not often studied in this age group. For example, albuterol nebulized solution, used to treat breathing difficulties such as asthma, is only approved for use in children 12 years and older. However, it is commonly prescribed to treat breathing issues for all ages.

It is important to understand why a medicine is prescribed for you and what it is being used to treat, especially if it is prescribed off-label. The story that follows is a good example of why it is important to know as much as possible about the medicines you take.

A woman went to see her doctor about neck pain that would not go away. The doctor believed her pain was caused by nerve damage. He gave her a prescription for amitriptyline (Elavil),but he did not write the reason for the medicine on the prescription. At the pharmacy, the woman was surprised when she read on the drug information leaflet that the medicine was used to treat depression. She asked the pharmacist about this, and he confirmed that amitriptyline was an antidepressant. The woman became very angry and refused to take the medicine. She later accused her doctor of believing that her neck pain was all "in her head."

Amitriptyline is often used to treat nerve pain, which is an off-label use. Unfortunately, most drug information leaflets that you get from your pharmacy only list the original FDA "approved" uses for the medicine. Thus, the woman did not see "pain" listed on the leaflet and became confused about the medicine she was supposed to take.

Here’s what you can do: Always ask your doctor to write the reason for your medicine on your prescription. This will help your pharmacist provide you with accurate information if your medicine is being used to treat an off-label condition. Listing the reason for your medicine on each prescription will also help your pharmacist choose the right medicine.

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