What Is Off-Label Drug Use?

Prescription drugs and the pharmaceutical industry as a whole are heavily regulated. There is a stringent process put in place by organizations like the FDA to ensure drugs are safe before consumers can use them. Even so, there are often adverse effects and deaths due to prescription drugs.

Doctors have to be careful about how they prescribe medication because if there are bad outcomes, they can be liable rather than the drug company being legally responsible. Sometimes both could be found responsible in court.

One area that gets tricky for doctors is called off-label prescribing. As a patient and general consumer, it's helpful to understand what this means and what the pros and cons can be.

What Is Off-Label Prescribing?

Off-label prescribing refers to prescribing a drug for a different condition or perhaps at a different dose than the FDA approves it for. It's not uncommon. Around 20% of all prescriptions in the U.S. are off-label.

For FDA approval, the company that makes a drug has to provide clinical trial information on the conditions it can treat.

Other information the FDA requires include the safe dosage, the populations the drug is tested in, and evidence showing it's safe and effective. The company also has to include side effects or adverse events the drug could lead to, even if they're very rare.

Once a drug receives FDA approval, a doctor can prescribe it, but they don't only have to prescribe for the particular purpose it's approved for.

There are a variety of reasons a doctor might prescribe an off-label drug, including:

• There isn't currently an FDA-approved drug that would treat a patient's condition.
• Other drugs have already been tried and aren't currently effective.
• The FDA's approved drug for a condition may not be accessible or could be too expensive.
• There are side effects or interactions with the FDA-approved drug.
• The doctor may have personal experience or could have seen other evidence that a specific drug will work for a particular off-label application.

An example of a common off-label prescription is a beta-blocker. These drugs have FDA-approved uses for treating conditions like high blood pressure and coronary heart disease, but off-label they are used for the treatment of anxiety.

The Risks vs. the Benefits

Often even when a drug is being prescribed for off-label use, clinical trials are going on that show it works well for the purpose.

Sometimes a drug company might not pursue other approvals because it's too expensive, or they're currently working on an FDA approval that's not yet final.

While it can be very helpful for treatment, there are also risks of off-label prescribing. For example, in a 2009 review, researchers found off-label use puts patients at risk of getting not only ineffective but potentially harmful treatment. Doctors have to carefully weigh whether the benefits will outweigh the risks each time they prescribe, and they should rely on evidence.

What to Know As a Patient

As a patient, it's vital that you're empowered and have a basic understanding of what's going on with your healthcare. This includes off-label prescribing.

There are two scenarios here. In one, you may have independently done the research and found a drug you think could work for you. In this case, you might bring it to your doctor's attention. In the other scenario, your doctor might introduce the idea of an off-label treatment to you.

Either way, you should recognize the FDA hasn't investigated how well this medicine works for your condition. There may not be medical evidence to support its use for your condition, and you could experience unique side effects while taking it.

Doctors aren't required to tell you if they're prescribing something off-label. Each time your health care provider talks to you about potentially prescribing you a new drug, you should ask what it's approved for.

Talk to your doctor about the benefits of taking it and the evidence that shows it could benefit you. You should also ask about side effects and any potential drug interactions.

Prescribing drugs off-label is legal in the U.S., and it's not an inherently ethical or unethical practice. Instead, it depends on the situation and why your doctor is prescribing it.

There can be benefits, such as the opportunity to try something distinctively effective for your condition. However, since there are risks, you should also go into the situation fully understanding what those are and making sure your health care provider has evidence to back up their prescribing recommendation.