Off-Label Prescribing: What You Need to Know

This is common practice in the field of medicine, and comes with many benefits to the patient in improving access to high-quality, effective care. 

‍What is off-label prescribing?

Off-label prescribing refers to the use of drugs for purposes other than those for which they were originally authorized for sale by Health Canada. In other words, it's when a doctor or nurse practitioner prescribes a medication for a different condition or in a different way than what's been authorized by Health Canada. This practice is common in Canada and around the world — It's estimated that 11% of all prescriptions in Canada are for off-label use. In pediatrics, this number jumps to 75%. This is for a good reason: it can benefit patients by giving them access to effective treatments and — when good clinical judgement is used by prescribers — improve patient outcomes. 

When is something considered “off-label”?

Any time practitioners prescribe a drug for an ailment that does not match the label, which means it is "off-label" use.

Health Canada authorizes drugs based on clinical trials and other evidence of their safety and efficacy for specific conditions. Clinical trials and evidence of efficacy and safety are submitted by the pharmaceutical companies who own the rights to the medication. 

When Health Canada authorizes pharmaceutical products, they give an indication, which is a specific use case for an ailment or group of ailments, for how the drug may be used. This information is then put on the “label” for the drug, so that patients know what it’s used to treat. 

As medical knowledge and understanding of diseases grow, doctors may find that drugs can be used effectively for other conditions (based on research or other evidence of efficacy beyond their indication). Despite the additional evidence from clinical practice, not all pharmaceutical companies seek formal approval for a new indication. 

Benefits and risks

Off-label prescribing can be beneficial for patients in several ways. It can provide access to treatments that are not yet approved for their condition, or that are not otherwise available. It can also allow doctors to personalize treatments based on their patients' individual needs, taking into account factors such as age, weight, and other health conditions. More recently, it has been helpful when drug shortages occur or a pharmaceutical company cannot meet demand for a specific drug, but there is a medication with the same active ingredient that can be prescribed instead.

Despite the many benefits, there are also potential risks associated with off-label prescribing. The safety and efficacy of a drug for a particular condition may not have been fully studied, and there may be a higher risk of side effects or interactions with other medications because of this lack of research. In some cases, off-label prescribing may not be supported by scientific evidence, and there may be little or no data to support its use. 

Insurance coverage of off-label prescriptions

A common question when it comes to off-label prescriptions is, “Will I receive insurance coverage for this medication?” Like coverage of all drugs, the coverage you receive for off-label prescriptions will vary based on your insurance policy. It is always best to speak directly with your insurance provider if you’re concerned about your coverage and share any concerns about treatment cost with your healthcare practitioner. 

If you have any questions or concerns about off-label prescribing, be sure to speak to your healthcare practitioner or pharmacist for more information.