Though legal, off-label drug use refers to prescribing a drug to treat a condition or age group it is not approved for. It may also mean taking the medication in a dosage or form that is not FDA-approved. This is a fairly common practice among doctors with 1 in 5 prescriptions for off-label use.
While off-label drug use is beneficial for treating some unapproved conditions, there are also many cases where there is a lack of evidence to support off-label drug use. This can create dangerous situations for patients, who many times are unaware they have been prescribed a drug for off-label use.
Weighing the Dangers of Off-Label Drug Use
Children, pregnant women, and seniors are especially vulnerable to the risks that come along with off-label drugs use. Because the drug is being used for purposes other than what the FDA cleared it for, there is usually a lack of research and evidence to support off-label use.
Even if there are studies surrounding the off-label use of a drug, they typically don’t expand those trials to include kids, pregnant women, or the elderly. This means the drugs may not be as effective in those groups or could have worse or produce different side effects. Without studying these double duty drugs, patient safety is at risk.
Most patients are unaware that they are taking a prescription for off-label use because doctors are not required to share that information. According to a 2006 JAMA study, more than 80 percent of off-label prescribing by doctors lacked strong scientific evidence. Furthermore, patients taking prescriptions off-label were 54 percent more likely to be harmed when compared to people taking the same drug for an approved use.
Senior Danger
For example, in nursing homes, sometimes antipsychotic drugs (usually used to treat schizophrenia) are given to dementia patients to help control their agitation. The seniors that took these off-label drugs were found to have a higher risk of death due to heart failure or an infection like pneumonia. Though the FDA has warned against it, there is no way to stop the practice of off-label drug use.
These results are alarming and even more so for seniors who are likely to be taking several medications at the same time. Even though there are dangers associated with off-label drug use, there are reasons why prescriptions for off-label use are permitted.
The Benefits of Off-Label Drug Use
With the dangers of off-label drug use, you might wonder why this practice is allowed in the first place. There are many times when off-label drug use is supported by evidence, even if the FDA has not approved it.
Cancer treatments can also involve off-label chemotherapy drug use. Chemotherapy drugs are typically approved for one type of cancer, but especially in situations where there are no other options, they can be used to treat other kinds of tumors.
Cardiologists use beta-blockers, which have been approved for treatment of high blood pressure, as the standard of care for heart failure. Tricyclic antidepressants have been known to successfully help treat chronic pain, and some antipsychotics have been used to treat ADHD.
Many times, off-label use eventually gets FDA approval anyway. However, the practice is now almost too common and doctors don’t even tell their patients they are being prescribed a drug off-label.
Patient Safety with Off-Label Drug Use
If a doctor prescribes you medication, they are not required to disclose if they are prescribing it off-label. This is why it is important for you to take an active role in your treatment plan. The next time you go to you doctor and you are prescribed a new drug, here are some things you should ask:
- Is the medication for on- or off-label drug use?
- What evidence does your doctor use to support his or her decision to prescribe the drug?
- What benefits are you likely to gain?
- What are the risk or the side effects of taking the drug?
- Are there clinical trials that you may be eligible for regarding the off-label drug use?
- What are your other options besides an off-label drug?
- Will your insurance cover the off-label drug?
If you are already on medications and want to know if your doctor prescribed you off-label medications, you can visit the National Institute of Health’s DailyMed website where you can search for a drug and find their approved uses.
The most important thing is to make sure your doctor is fully explaining the benefits and risks associated with these drugs. Your doctor may not have to tell you that they are prescribing a medication for off-label use, but you can empower yourself for your well being and safety.
