Navigating Health Care: Prescribing Drugs for Off-Label Use is Common

Rand: Your doctor prescribes a medication you’ve never had before and you decide to do a little research. You find out that the medication is designed for a condition that’s different from the one for which she’s treating you. Did she make a mistake? Talk with your doctor. She’s probably giving you the medication in an off-label use, which means that the drug has been approved to treat one condition but is being used to treat another. It’s a common practice, and AHRQ Director Dr. Carolyn Clancy is with us to help explain why. Dr. Clancy, thank you so much for joining us.

Dr. Clancy: Thank you.

Rand: So, off-label prescribing is common, but is it safe?

Dr. Clancy: People shouldn’t be concerned if a doctor prescribed a medicine for an off-label use. Frankly, many medicines are used off-label and work very effectively. And most doctors only prescribe a medication when they feel confident that a drug has been shown to work well at treating that condition. However, I encourage consumers to talk with their clinicians if they do have concerns over any medicine or treatment. It’s always a good idea to know all the risks and benefits.

Rand: Are there specific conditions that are more commonly treated with off-label drugs?

Dr. Clancy: Well, there are many types of off-label use, and they vary widely. For example, medicines called beta blockers were initially approved for treating high blood pressure and have since been found to treat heart failure. And some medicines designed to treat depression are also used to relieve chronic pain.

Rand: So then what should patients ask their clinicians if they’re prescribed an off-label drug?

Dr. Clancy: First, you may not know that your doctor is prescribing an off-label medication. So when you get a prescription, you may want to start by asking, "Is this the primary use of that medication?" If it turns out that your doctor has given you an off-label drug, you should ask your doctor if the off-label drug is likely to work better than an approved treatment. This is very important, because the off-label medication may not have been as well tested for your condition as it has been for the condition it was designed to treat. For example, a study by AHRQ found that some newer antipsychotic medications are being prescribed for other conditions, but have not shown good results. The take-home message is that you should also talk with your clinician about the potential side effects and other possible risks. You should also ask for information showing how well it works. This information should help you engage in a healthy discussion with your doctor about which treatment choice is right for you. I’m Dr. Carolyn Clancy and that’s my advice for navigating the health care system.

Rand: For more on medication safety and other information, visit ahrq.gov/consumer.