What does it mean when a drug is off-label?
Off-label drug use is the use of medications for an indication or in an unapproved age group, dosage, or route of administration that has not been approved by the U.S. Food and Drug Administration (FDA). Both prescription drugs and over-the-counter drugs (OTCs) can be used in off-label ways, although most studies of off-label use focus on prescription drugs.
Off-label drug use is very common and generally legal unless it violates ethical guidelines or safety regulations. Off-label drug use can be motivated by several factors. First, a medication may not have been studied and approved for a specific population (eg, pediatric, geriatric, or pregnant patients). Second, a life-threatening or terminal medical condition may motivate a health care professional to give any treatment that is logical and available, whether approved by the FDA or not.
Third, if one medication from a class of drugs has FDA approval, physicians commonly use other medications in the same class without specific FDA approval for that use for the same indication. In addition, if the pathologic or physiologic features of 2 conditions are similar, a physician may use a medication approved for 1 of these conditions for both (eg, diabetes and metabolic syndrome; psychiatric diseases such as anxiety and posttraumatic stress disorder).
What is the difference between off-label and unlicensed?
Yes, unlicensed or unapproved drug use refers to the use of a medication that is legally approved or licensed in other countries, but not the US or UK. Most medications that fall under this category have to be imported because it is not available. Most times, these medicines are used for treating rare illnesses. The manufacturer may have decided that it was too expensive to carry out the clinical trials or it would be difficult to find enough patients for the clinical trials needed to secure approval or license.
What are the benefits of off-label drug use?
A drug is granted a license for use after a thorough assessment of risks and benefits based on high-quality scientific proof of its efficacy and safety. Off-label prescribing makes clinical sense only in exceptional circumstances. If reasonable evidence suggests that the benefit of off-label use will outweigh the risks, that declining to treat the condition poses even greater dangers than the off-label prescription does, and that there is no adequate alternative therapy, then off-label prescribing can benefit patients.
Prescribing off-label drugs gives medical practitioners more treatment options if one medication doesn’t work for you. It’s especially useful for rare conditions that have limited treatment options. For example, Off-label drug use makes it possible for chemotherapy approved to treat one type of cancer, to be used by healthcare providers to treat a different type of cancer.
How do I know that an “off-label” or unlicensed medicine is safe and will work?
Unlicensed and “off-label” medicines are only prescribed after careful consideration of other options available. Your doctors will have read information from medical publications supporting its use and may have taken advice from other experts. An unlicensed or “off-label” medicine will only be used if it is the most appropriate medicine for you and your illness or condition.
How do I know whether a medicine is unlicensed or being used “off-label”?
Your doctor or pharmacist will tell you that the medicine is not licensed or is being used off-label. The leaflet or insert that comes with the medicine may not say anything about how you should use it or may say that the medicine is not suitable for being used in the way we are prescribing it. This does not mean that it cannot be used safely to treat your condition – it means that the drug company does not have a license for using it in this way and so is not allowed to recommend this use.
What are the risks of off-label prescriptions?
Prescriptions for off-label uses of FDA-approved drugs are made without the benefit of an FDA-reviewed analysis of safety and effectiveness data. Physicians may resort to such prescribing to take advantage of new ideas and treatment approaches when available information to support them is inadequate. However, despite the potential risks associated with off-label uses, efforts to prohibit such uses might hurt the public. Some off-label prescribing may result because manufacturers have chosen not to invest the resources needed to have FDA add indications to the drug’s approval and labeling. A worst-case scenario for the nation’s health would be the widespread acceptance of a drug for off-label use that sufficient research would have revealed to be ineffective, unsafe or both.
Aside from the drug’s direct harm, the time spent waiting to see whether it worked would have been time not spent exploring other treatment options. Unchecked off-label prescribing may also threaten the FDA gold standard of drug approval. If clinicians had already accepted a new use into practice through off-label prescribing, a manufacturer may choose to not invest resources to go through clinical trials and the FDA process to win approval. Although manufacturers do share information on off-label uses, courts have sometimes found they had overstepped allowable bounds.
How is Off-label drug regulated in the United States?
In the United States, once a drug has been approved for sale for one purpose, physicians are free to prescribe it for any other purpose that in their professional judgment is both safe and effective, and are not limited to official, FDA-approved indications. Pharmaceutical companies are not allowed to promote a drug for any other purpose without formal FDA approval. Marketing information for the drug will list one or more indications, that is, illnesses or medical conditions for which the drug has been shown to be both safe and effective.
This off-label prescribing is most commonly done with older, generic medications that have found new uses but have not had the formal (and often costly) applications and studies required by the FDA to formally approve the drug for these new indications. However, there is often extensive medical literature to support the off-label use.
A leading example of how regulatory agencies approach off-label use is provided by the FDA’s Center for Drug Evaluation and Research, which reviews a company’s New Drug Application (NDA) for clinical trial data to see if the results support the drug for a specific use or indication. If satisfied that the drug is safe and effective, the drug’s manufacturer and the FDA agree on specific language describing dosage, route of administration, and other information to be included on the drug’s label. More detail is included in the drug’s package insert.
