Off-Label Drugs: What You Need to Know

By Carolyn M. Clancy, M.D.

April 21, 2009

What should you do if your doctor prescribes a medicine for you and you find out that the medicine is not normally used to treat your condition?

Ask your doctor. It’s possible you’ve been prescribed a medicine for an "off-label" use.

Off-label prescribing is when a physician gives you a drug that the U.S. Food and Drug Administration (FDA) has approved to treat a condition that is different than yours. This practice is legal and common. In fact, one out of every five prescriptions written today is for off-label use.

Under Federal law, the FDA must approve all new prescription drugs using evidence that the medicine is safe and effective for a particular condition. This allows a drug maker to market a drug for the use the FDA agreed it works. While a company is not allowed to market an approved drug for other purposes, the law does let physicians prescribe the medication to treat a condition for which it is not approved.

Are off-label drugs safe?

This is a good question to ask your doctor. Most doctors only prescribe off label when they are confident the medicine will work well for treating a condition. Off-label drugs can help patients when approved treatments aren’t working or when patients have rare conditions that don’t have approved treatments.

Heart medicines, antipsychotics, and antibiotics are commonly prescribed off label. Beta blockers, for example, were first approved for treating high blood pressure but have since been found to be good for treating heart failure and migraines. Some medicines designed to treat depression also are used to treat chronic pain.

All drugs carry risks, however. Treatment decisions involve weighing possible risks against possible benefits. Sometimes the risks outweigh the benefits. When it comes to off-label drugs, sometimes there isn’t enough reliable evidence to make informed decisions.

Research funded by my agency, the Agency for Healthcare Research and Quality (AHRQ), shows that some newer antipsychotic drugs developed to treat schizophrenia and bipolar disorders are prescribed to millions of Americans who suffer from depression, dementia, and other conditions. But there’s no evidence that these drugs work to treat them. That is a problem because another AHRQ study found that adults who took certain antipsychotic drugs had a higher risk of sudden death from heart disease than patients who did not take them. This research should help doctors and patients weigh the risks and benefits of these drugs before prescribing them for depression or for other off-label uses.

Talk with your doctor if you have concerns about any medicine or treatment, particularly if it may be off label. Here are several questions to ask:

• Is this the approved use of the medicine? You may not know if the use is off label. This question can help you start the conversation with your doctor about your medicines.
• Is the off-label use of this drug likely to be more effective than one approved to treat my illness? This is important because the off-label drug may not be as well tested for your condition.
• What evidence shows that this off-label drug can treat my condition?
• What are the risks and benefits of off-label treatment with this drug?
• Will my health insurance cover off-label treatment with this drug?

As we like to say at AHRQ, "Questions are the answer," meaning that it’s important to ask your doctor plenty of questions about any medicines he or she prescribes. When you understand why you are taking certain medicines - including off-label drugs - you are more likely to take them correctly.

I’m Dr. Carolyn Clancy, and that’s my opinion on how to navigate the health care system.

More Information

Agency for Healthcare Research and Quality
Effective Health Care
Efficacy and Comparative Effectiveness of Off-Label Use of Atypical Antipsychotics
http://effectivehealthcare.ahrq.gov/healthInfo.cfm?infotype=rr&ProcessID=5&DocID=64

Agency for Healthcare Research and Quality
Use of Atypical Antipsychotic Drugs Increases Risk of Sudden Cardiac Death in Adults
http://www.ahrq.gov/news/press/pr2009/antipsychpr.htm

Agency for Healthcare Research and Quality
Questions are the Answer: Get More Involved With Your Health Care
http://www.ahrq.gov/questionsaretheanswer/

U.S. Food and Drug Administration
Information for Consumers
http://www.fda.gov/cder/info/consumer.htm

National Cancer Institute
Q&A: Off-Label Drugs
http://www.cancer.gov/clinicaltrials/learning/approval-process-for-cancer-drugs/page5

Consumer Reports Best Buy Drugs
Off-Label Drug Use-The Pros and Cons
http://www.consumerreports.org/health/best-buy-drugs/index.htm

Current as of April 2009

Internet Citation:

Off-Label Drugs: What You Need to Know. Navigating the Health Care System: Advice Columns from Dr. Carolyn Clancy, April 21, 2009. Agency for Healthcare Research and Quality, Rockville, MD. http://www.ahrq.gov/consumer/cc/cc042109.htm