Off-label use of drugs and medical devices: Managing the medico-legal risks

Using medications or medical devices in a manner that might be considered off-label can benefit patients, but it can also lead to medico-legal risks for physicians. These risks include uncertainty about the efficacy of the drug or product, and possible adverse reactions.

Here is our guidance for physicians considering prescribing drugs or using medical devices in a way that might be considered off-label.

What does “off-label use” mean?

Off-label use refers to using licensed medications or medical devices outside of the condition or indication for which the licence was issued. This differs from administering medications or using devices that have never been considered for a licence.

Off-label use can also include prescribing different dosages or frequencies, lengthening or shortening the duration of treatments, or using different routes of administration than indicated on the drug label. New off-label uses are often developed in the context of research studies.

Health Canada does not regulate the off-label use of medications or devices and takes the position that physicians' prescribing practices fall under the jurisdiction of provincial and territorial medical regulatory authorities (Colleges).

Examples of off-label drug prescription and use

Some examples of off-label use include:

• prescribing tricyclic antidepressants for patients with chronic pain or fibromyalgia;
• using antipsychotic medications to treat agitation in dementia patients;
• using a balloon catheter to dilate peripheral arteries in the legs;
• using a dose of a medication above the designated upper limit.

The use of psychedelic medications like ketamine, which is primarily approved for anesthesia and procedural sedation, may also be considered off-label by regulators or courts. Common off-label uses of psychedelic drugs (which are not officially approved indications) include the treatment of chronic pain, post-traumatic stress disorder, and treatment-resistant depression.

Note that off-label prescribing may be more frequent in the pediatric population, since most drugs are approved on the basis of clinical trials conducted with adults.

Minimizing the risk of liability when using off-label medications

Here are some steps physicians can take when prescribing off-label use to patients.

• Determine if the proposed use of the medication or device really does constitute an off-label use.
• Consider if there is sufficient support from the medical literature (e.g. guidelines from medical specialty organizations) for the off-label use of the medication or product. Is the use in keeping with the present standards of practice?
• Also consider checking with the medical regulatory authority (College) regarding standards within your province or territory.
• Ensure compliance with any hospital policy before using off-label medications or products in a hospital setting.
• Take reasonable precautions to ensure that the prescription or use of the drug or device is the appropriate treatment for the patient's condition.
• Obtain a detailed history from patients and examine them to determine if they have a condition that would place them at increased risk of potential side-effects from the off-label use of the drug or device.
• Obtain and document patients' informed consent when appropriate. A greater degree of disclosure is generally required for off-label treatments, which includes disclosure of all associated risks and information about the medication or device being used in an off-label fashion. Advise patients that a drug or device is not approved for their particular condition.
• Document the rationale for off-label use of the medication or device, as well as any questions asked by patients and the answers provided.
• Carefully monitor patients for side-effects during or following an off-label treatment.

CMPA members with further questions on this challenging topic are encouraged to contact CMPA for advice.

Additional reading

• CMPA article: Alternative medicine – What are the medico-legal concerns?
• CMPA article: Suspect unlawful activity with prescriptions or medications? Here’s how to respond
• CMPA Good practices: Medication safety

DISCLAIMER: This content is for general informational purposes and is not intended to provide specific professional medical or legal advice, nor to constitute a "standard of care" for Canadian healthcare professionals. These resources are offered in accordance with CMPA's Terms of Use. AI tools may be used in limited ways, but human subject matter experts always provide oversight and final approval for all CMPA content.