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Off-label drug prescribing: What does it mean for you?

Doctors often prescribe medication to treat problems the drugs aren't officially approved for, which can be good—and bad—for patients

Last updated: December 2012

Has your doctor ever prescribed an antidepressant to curb your hot flashes or a blood-pressure pill to calm your stage fright? How about an antipsychotic drug to help you sleep? Like most Americans, you probably assume that when your doctor prescribes a medication, the U.S. Food and Drug Administration has approved it for your specific ailment. But about one in five prescriptions are written “off label”—that is, for a use not approved by the FDA.  While legal and common, drugs prescribed off-label present both opportunities and challenges for consumers—and for doctors. 

Drugmakers benefit, too. They can save millions of dollars by not submitting an application or safety and efficacy testing results to the FDA to get a drug approved for treating a second (or third) condition or for a new group of patients, like children. And they profit from additional sales of a drug prescribed for unapproved uses. A result is that many of those uses have little or no scientific support.

What are the risks?

Doctors will often prescribe a drug off label without realizing it is not approved for that use,  says G. Caleb Alexander, M.D., an associate professor of epidemiology and medicine at Johns Hopkins University. A 2009 survey by Alexander and colleagues of some 1,200 primary-care physicians and psychiatrists found that many of them thought that some common drug treatments were FDA-approved when in fact they weren’t, and that there was little evidence to support the off-label use of those drugs. “That’s particularly of concern where drugs may have specific, noteworthy safety concerns,” Alexander says. 

For instance, newer antipsychotics—such as aripiprazole (Abilify and generic), olanzapine (Zyprexa and generic), quetiapine (Seroquel and generic), and risperidone (Risperdal and generic)—are approved for the treatment of schizophrenia, certain aspects of bipolar disorder, and in the case of Abilify and Zyprexa, as an add-on treatment for depression. But they’re also prescribed off-label for numerous other conditions, including obsessive-compulsive disorder, dementia, anxiety, substance abuse, eating disorders, and insomnia. In a 2011 study, Alexander and colleagues found that their off-label use doubled from 1995 through 2008, particularly in unapproved uses for bipolar disorder

But only only a few of those unapproved uses are supported by evidence, according to a September 2011 review of the drugs that was published in the Journal of the American Medical Association. The review also found that side effects and risks were an issue, including abnormal limb and body movements, sedation, fatigue, weight gain, and a small increased risk of death for seniors with dementia.

Unsafe practices

To avoid exposing large numbers of people to the questionable benefits and potential harms of off-label drug use, the FDA prohibits manufacturers from directly promoting unapproved uses of their drugs. 

Yet such marketing has been common in the U.S. In 2012, the drugmaker GlaxoSmithKline agreed to pay a $3 billion fine in the largest health-care fraud settlement in U.S. history. The charges included promoting the antidepressant Paxil for children and adolescents even though it was not approved for them and studies had found it was ineffective and potentially dangerous for kids. GSK was also charged with marketing the antidepressant Wellbutrin for weight loss and the treatment of sexual dysfunction, substance addictions, and other unapproved uses. Some experts argue that pharmaceutical companies might view such fines, although hefty, as merely a cost of doing business.

When the benefit outweighs the risk

Off-label prescribing can make sense if a new use for a drug emerges after it was approved for another purpose. That way, doctors can take advantage of the drug without having to wait for formal proof, as has been the case with certain medications to treat cance. 

And there are off-label uses that are supported by good scientific evidence. For example, topiramate, an antiseizure drug, has been recommended in national and international guidelines for the off-label treatment of alcohol dependence. And amitriptyline, an older antidepressant, is recognized as a first-line drug for short-term pain relief of fibromyalgia, and it cost much less than approved treatments.

What you can do

How can you protect yourself from the potential hazards of off-label drug use? Our medical consultants recommend the following precautions:

  • When your doctor prescribes a drug, ask if it’s an approved use. If he or she doesn’t know, ask your pharmacist.
  • Check for yourself. Go to DailyMed (dailymed.nlm.nih.gov/) and search for the drug. Then click on the tab for “Indications & Usage” to see if your condition is listed.
  • If it’s an off-label use, ask your doctor if it’s supported by well-designed trials showing significant improvement for people with your condition.
  • Ask your doctor why he or she thinks the drug will work better than approved drugs for your illness.
  • Find out if your health insurer covers payment for the off-label use. Some may require evidence of effectiveness or failure with conventional treatments, especially if the drug is expensive.

Drugs commonly prescribed off-label*

Specific drug, type of drug

Examples of off-label use**
Aripiprazole (Abilify), antipsychotic Dementia, Alzheimer’s disease
Tiagabine (Gabitril), antiseizure Depression
Gabapentin (Neurontin), antiseizure Nerve pain caused by diabetes, migraines, hot flashes
Topiramate (Topamax), antiseizure, in combination with phenteramine for weight loss Bipolar disorder, depression, nerve pain, alcohol dependence, eating disorders
Risperidone (Risperdal), antipsychotic Alzheimer’s disease, dementia, eating disorders, post-traumatic stress disorder 
Trazodone (Desyrel), antidepressant Insomnia, anxiety, bipolar disorder
Propranolol (Inderal), high blood pressure, heart disease Stage fright
Sildenafil (Viagra), erectile dysfunction To enhance sexual performance in people not diagnosed with erectile dysfunction, to improve sexual function in women taking certain antidepressants
Quetiapine (Seroquel), antipsychotic Dementia, Alzheimer’s disease, obsessive-compulsive disorder, anxiety, post-traumatic stress disorder
SSRI antidepressants such as paroxetine (Paxil) and sertraline (Zoloft) Premature ejaculation, hot flashes, tinnitus (ringing in the ears)
Prazosin (Minipress), high blood pressure Post-traumatic stress disorder
Amitriptyline (Elavil), antidepressant Fibromyalgia, migraines, eating disorders, pain after shingles infection
Bevacizumab (Avastin), certain types of cancer Wet age-related macular degeneration (eye disease)
Statins such as atorvastatin (Lipitor), simvastatin (Zocor), high cholesterol in adults, children with an inherited cholesterol condition  Rheumatoid arthritis, to lower cholesterol in children who lack the inherited condition
Clonidine (Catapres), high blood pressure Smoking cessation, hot flashes, attention deficit/hyperactivity disorder (ADHD), Tourette’s syndrome, restless legs syndrome

* Not meant to be a comprehensive list. Many of the drugs listed here are also available as generics.

** Does not imply that use is clinically appropriate or inappropriate, or beneficial or not. 

***To find out if a drug’s off-label use is supported by evidence, click on the medication name. 

Editor's Note: These materials were made possible by a grant from the state Attorney General Consumer and Prescriber Education Grant Program, which is funded by the multi-state settlement of consumer fraud claims regarding the marketing of the prescription drug Neurontin (gabapentin).
   

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