What Does "Off-Label" Mean?
In the United States, the Food and Drug Administration (FDA) approves medications for specific uses. If a medication is used for any other use (other than the approved uses), this is considered an "off-label" use. For instance, amitriptyline
) is a prescription medication approved to treat depression
. However, amitriptyline is commonly prescribed to also treat chronic nerve pain, even though it is not approved for this use. Using amitriptyline for pain is an "off-label" use.
Have Off-Label Uses Been Studied?
Many off-label uses of medications have been thoroughly studied. Just because a medication has been thoroughly studied for a specific use does not mean the FDA automatically approves it for this use. A drug company must apply to the FDA to have a new use for a medication approved. Because this process can be quite lengthy and expensive, many companies do not seek approval for off-label uses. If a medication is older (and generic versions are available), it often does not make sense financially to seek approval for off-label uses. However, if a medication is newer, a new use may mean a new patent for the medication (which may allow the drug company to keep the sole rights to market the medication for a longer period of time).
Drug companies cannot advertise off-label uses for their medications. Similarly, there are special rules limiting how representatives of drug companies can discuss and promote off-label uses of medications. However, healthcare providers are not limited in prescribing medications off-label. There are no laws preventing the off-label prescribing of medications. In fact, the off-label use of medications can be very appropriate in many situations. On the other hand, the off-label use of medications can also be inappropriate. Using a medication off-label without scientific evidence to support such use (especially when other alternatives are available) is generally not appropriate.