What does "off-label use" of a medication refer to?

"Off-label use" of a medication refers to the practice of using a drug for indications, routes of administration, or patient populations that have not been specifically approved by regulatory authorities, such as the FDA. This can occur when healthcare providers prescribe a medication based on clinical evidence, previous research, or professional experience that supports its efficacy for other uses not officially sanctioned.

The term "off-label" itself highlights the fact that the drug carries labeling that does not include the particular use for which it is being prescribed. This practice is quite common in medicine, especially in areas like pediatrics, where medications may not have been tested thoroughly in children, yet healthcare providers find them beneficial based on adult studies or anecdotal evidence.

In contrast, using a medication at a different dosage focuses solely on dosage adjustments rather than applications outside of approved uses. The prescription of discontinued medications refers to drugs that are no longer available due to various reasons, such as lack of efficacy or safety concerns, while using medications as part of a clinical trial pertains to investigational uses, which are either in a formal study or part of research meant to evaluate the drug's safety and efficacy.