Medication Information

Standard and off-label use of medication 

Medications may be prescribed for standard or off-label uses. What is the difference?

An infographic comparing standard medication to off-label medication. Standard medication is used in a way approved by Health Canada, safety tested in large research studies. An example standard medication is baclofen for muscle spasticity. Off-label medication is used in a way not approved by Health Canada, but safety is supported by some research studies and use is legal and common. An example of off-label medication is gabapentin for neuropathic pain.

Standard use of medication

Standard use of medication Using the medication according to the instructions provided by the healthcare provider and manufacturer. is when it is prescribed for and used based on specific conditions (e.g., indication, population/ condition, dosage/ dosage form) approved by regulatory bodies such as Health Canada.

These specific conditions are based on clinical trial A research study that tests the safety and effectiveness of medical treatments, drugs, or devices on humans. data, which are research studies involving human participants. A clinical trial A research study that tests the safety and effectiveness of medical treatments, drugs, or devices on humans. is conducted to understand and assess the safety and efficacy The ability of a treatment, medication, or intervention to produce a desired result in ideal or controlled conditions such as in a clinical trial. of a medication.

Standard use of medication Using the medication according to the instructions provided by the healthcare provider and manufacturer. follows guidelines that are found in a medication’s prescribing information.

Off-label use of medication

Off-label use of medication Prescribing a medicine to treat a condition or symptom that has not been approved for that use by regulatory authorities. is when it is prescribed for or used in a way that was not approved by regulatory bodies (e.g., Health Canada). For example, the medication may be used for a different indication, in a different population, or in a different dosage or dosage form (e.g., capsule, liquid).  

Off-label use of medication Prescribing a medicine to treat a condition or symptom that has not been approved for that use by regulatory authorities. is typically supported by some research, but often not large trials like those conducted for standard use of medication Using the medication according to the instructions provided by the healthcare provider and manufacturer. . However, off-label use of medication Prescribing a medicine to treat a condition or symptom that has not been approved for that use by regulatory authorities. is still considered a legal and common practice.  

Your healthcare provider may prescribe a medication for an off-label use for various reasons. For example, you may have tried all standard use medications without seeing any benefits, or there may not be a standard use medication to treat your medical condition. Taking depression medication for pain management would be considered an example of off-label use. 

Questions to ask

If your healthcare provider is thinking about prescribing an “off-label” medication, consider asking them the following questions:  

  • What is the medication approved for?  
  • Are there other medications that are approved to treat my medical condition?  
  • Is there evidence to support that this medication can treat my medical condition?  
  • Will this medication work better to treat my medical condition than using an approved treatment?  
  • What are the potential risks for taking “off-label” medications?  
  • Will my health insurance cover “off-label” medication use?  

Examples of off-label medications

Some examples of common off-label medications, sometimes taken by individuals with SCI/D Spinal cord injury or dysfunction. , include: 

  • Gabapentin (Neurontin) – approved for treating seizures (antiepileptic), but often used for neuropathic pain A type of pain caused by nerve damage or dysfunction. It can feel like burning, tingling, or shooting sensations and is often chronic (long-lasting). management 
  • Amitriptyline (Elavil)– approved for managing depression (antidepressant), but often used for neuropathic pain A type of pain caused by nerve damage or dysfunction. It can feel like burning, tingling, or shooting sensations and is often chronic (long-lasting). management 

References

Canadian Agency for Drugs and Technologies in Health. (2017). Off-label use of drugs. https://www.cadth.ca/sites/default/files/pdf/off_label_use_of_drugs_pro_e.pdf  

Commissioner, F. and D. A. (2017, February 5). Understanding unapproved use of approved drugs “Off label.” https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/understanding-unapproved-use-approved-drugs-label  

Wittich, C. M., Burkle, C. M., & Lanier, W. L. (2012). Ten common questions (and their answers) about off-label drug use. Mayo Clinic Proceedings, 87(10), 982–990. https://doi.org/10.1016/j.mayocp.2012.04.017  

Note: Some of the information included above is also based on lived experiences.