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Is my mom using a drug "off-label"? What does this mean, and should I be concerned?

Q: My mom was prescribed an antipsychotic drug for sleep, but her support worker is concerned it’s being used off-label. Can you explain what that means and should I be concerned? 

Health Canada approves drugs by examining clinical trial data that a pharmaceutical company submits for review. If the data shows that the drug is safe and effective, it will be approved for the specific medical condition, dosage, and patient group studied in the clinical trials. If a drug is used differently than specified by Health Canada’s approval, it’s called “off-label” use. But once the drug is approved, there’s nothing to stop doctors from prescribing it off-label – it’s not illegal or anything like that.

Off-label drug use is relatively uncommon in Canada and most off-label uses lack strong scientific evidence that they actually work. In addition, there can be an increased risk of serious side-effects with off-label use. However, there are some situations in which off-label use can be appropriate, such as when treating rare diseases where there aren't many (or any) treatment options available or when approved drugs have been tried and haven’t been effective.

Should you be concerned about your mom? Perhaps. Medications that affect the central nervous system, including antipsychotics, are among the drugs most often prescribed for off-label use. In this case, the Canadian Psychiatric Association (CPA), the Canadian Academy of Child and Adolescent Psychiatry (CACAP), and the Canadian Academy of Geriatric Psychiatry (CAGP) have made recommendations against the routine use of antipsychotics to treat primary insomnia in any age group.

At GSC, we are big fans of Choosing Wisely Canada, an organization that aims to educate physicians and patients about waste in the health care system and help them make smart and effective choices to ensure high-quality care. They have endorsed the recommendations stated above and instead advise that a thorough assessment be conducted to eliminate possible causes of insomnia, good sleep hygiene be implemented, and if medication is necessary, one of the approved drugs to treat insomnia be used instead.

A decision to use a drug off-label should be made between the physician and the patient (or their caregiver) together. To help you make an informed decision and determine whether this is the most appropriate treatment choice for you mom, you may want to have a discussion with her doctor. Here are the questions I recommend anyone asks about off-label use:

  • What is the drug approved for?
  • What scientific studies are available to support the use of this drug to treat my disease or medical condition?
  • Are there other drugs or therapies approved to treat my disease or medical condition?
  • Is it likely that this drug will work better to treat my disease or medical condition than using an approved treatment?
  • What are the potential benefits and risks of treating my disease or medical condition with this drug?
  • Will my health plan cover treatment with this drug for my disease or medical condition?
  • Are there any clinical trials studying the use of this drug for my disease or medical condition that I could enrol in?