Definition of Off-Label Use

Off-Label UseOff-Label Use – Taking a drug for a purpose other than that for which it has received regulatory approval.

In the United States the Food and Drug Administration (FDA) requires pharmaceutical manufacturers to demonstrate the safety and efficacy of a drug before approving it for use.

Once a drug receives FDA approval, however, doctors may legally prescribe it for uses that are consistent with current standards of care. The FDA does not regulate how doctors prescribe or individuals take approved drugs.

Additional beneficial effects of a drug often emerge after the drug has been in use for some time and doctors begin to notice those effects. For some of these drugs the additional effects are so significant that prescribing the drug for them subsequently becomes an approved use. In other situations the drug becomes widely known for its additional effects but the manufacturer does not conduct further studies or seek FDA approval for them.

Off-label use is most common when treating conditions for which conventional therapies are limited or unsuccessful, especially when the condition is progressive or chronic such as multiple sclerosis, parkinson’s disease, cancer, and chronic pain syndromes.

Doctors may also turn to off-label use when prescribing medications for children because many drugs receive approval without having been tested for safety and efficacy in children. A doctor’s decision to prescribe a drug offlabel draws from available clinical study results, clinical observations, and best practices standards.

It is important for a person considering off-label use of a drug to fully understand the potential benefits and risks of such use as well as the drug’s possible side effects and adverse reactions.


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