Off-label Use of Drugs

What is “off-label”? This term refers to when a physician prescribes a drug that the U.S. Food and Drug Administration (FDA) has approved to treat a different condition. This practice is fairly common and is also legal. As a matter of fact, one in five prescriptions written are for off-label use.

Why do physicians prescribe off-label drugs? There may be several answers to this question. One reason may be that there are currently no drugs approved by the FDA for a patient’s condition. Another reason could be that a patient has tried other medications that are approved by the FDA but none of them have been successful in treating the condition. Another form of off-label use is when a drug is given in a different way such as when a drug comes as a capsule, but it is given in an oral solution instead. Furthermore, a drug can be prescribed at a different dose than what clinical trials have exposed patients to. Healthcare providers are legally allowed to prescribe a drug for an unapproved use when they determine that it is medically appropriate and necessary for their patient.

Unfortunately, off-label use comes with a price. In a study, published in JAMA Internal Medicine, it was noted that side effects for off-label drugs were 44% higher than on-label medications.

Of course, use of off label medications has to be medically necessary. Some examples of off-label uses of medications include:

• Prazosin (Minipress): On-label use: treatment of hypertension. Off label- use: nightmares related to post-traumatic stress disorder (PTSD).
• Clomiphene (Clomid) On-label use: treatment of female infertility due to ovulatory disorder. Off label- use: treatment of male infertility
• Memantine (Namenda): On-label use: treatment of Alzheimer’s disease. Off-label use: treatment of obsessive-compulsive disorder (OCD)
• Propranolol (Inderal): On-label use: treatment of hypertension. Off label- use: performance anxiety

Those were just a few examples of common medications that are being used effectively in patients with different conditions than the ones approved by the FDA. It is very important that patients are aware that they are using a drug off-label so they can watch for adverse reactions and contraindications. Patients should keep in mind that FDA has not determined that the drug is safe and effective for the unapproved use.

• Kimberly Parada

References:

10 Surprising Off-Label Uses for Prescription Medications. Pharmacy Times. https://www.pharmacytimes.com/contributor/timothy-o-shea/2016/01/10-surprising-off-label-uses-for-prescription-medications. Accessed May 6, 2020.

Understanding Unapproved Use of Approved Drugs “Off Label”. U.S. Food and Drug Administration. https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/understanding-unapproved-use-approved-drugs-label. Accessed May 6, 2020.