What the Heck Does “Off-Label” Mean, Anyway?

January 7th, 2010. By Kristine B

One of the phrases we at LawyersandSettlements.com see frequently is “prescribed for off-label uses.” In fact, we see it a lot. But, we realized that not everyone understands what “off-label uses” actually means, or what its implications are. So, this week’s Pleading Ignorance examines the off-label use of drugs. (I promise this won’t be too painful). 

When a drug is approved by the FDA it’s approved for specific circumstances (meaning, for specific health problems) and, often, for specific people (usually defined by an age range, e.g. adults). That’s because the drug has been tested and shown to be beneficial in treating those health problems in that group of people. 

When a drug is prescribed for off-label use, the drug is being used either for a condition that it was not approved to treat or for a person outside the group listed in the prescribing information or both. 

According to a report at USA Today (11/25/08), a study published in Pharmacotherapy listed 14 drugs that should be studied further regarding their off-label use. Those drugs include Seroquel, an antipsychotic medication that is approved to treat schizophrenia and short-term manic or depressive episodes in bipolar disorder. However, according to researchers, in three out of four cases, the drug has been used off-label, for maintenance therapy of bipolar disorder. Other drugs included on the list, as cited at The Wall Street Journal blog (11/25/08) were Coumadin, Lexapro, Risperdal and Singulair. 

It’s not illegal to prescribe drugs for off-label uses. Doctors are allowed to prescribe an FDA-approved drug for circumstances the drug wasn’t previously approved for. However, when a drug is prescribed for off-label use, the drug hasn’t been proven to be safe or effective for that use or in that group of people. Consider that a drug approved for use in adults could be used in children but, because no studies have been done on its safety in children, doctors may have difficulty determining what dose to give or may not know if the drug could affect the child’s development. 

What IS illegal is when pharmaceutical companies market their drugs for off-label uses. Drug companies can only market their drugs for the circumstances that they were approved for. They can’t go around to doctor’s offices saying, “Drug A was approved to treat colds but we have found it can cure cancer, so you really should consider prescribing this drug for cancer patients.” All they can say is, “Drug A has proven to treat colds and you should prescribe it for people with colds.” (Not surprisingly, one company—Allergan, the maker of the Botox brand of botulinum toxin A—has reportedly filed a lawsuit claiming that off-label marketing is protected by freedom of speech, so it should be allowed…)

Unfortunately, drug companies try to get away with promoting drugs for off-label uses. If you follow the news, you’ve probably heard about drug companies facing lawsuits that they marketed their drugs for off-label uses. 

In 2009, Eli Lilly & Co. reportedly settled lawsuits with eight states that alleged that the company promoted Zyprexa for off-label uses. Zyprexa was approved for the treatment of severe bipolar disorder and schizophrenia, but lawsuits alleged Eli Lilly encouraged doctors to prescribe Zyprexa for patients with age-related dementia and patients with mild bipolar disorder who were previously diagnosed with depression.

How do you know whether you’re taking a drug that’s been prescribed for you off-label? Ask your doctor. You can also do your homework by looking up any drug at either drugs.com or rxlist.com.

Got a question about a legal term, phrase or issue? Ask away…drop me a line at

Tags: Drugs, FDA, Off-label, Pleading Ignorance

This was posted on Thursday, January 7th, 2010 at 5:17 pm and is filed under Pleading Ignorance . Feel free to respond, or trackback. Read our comments policy.