Navigating the risks and rewards of off-label prescribing

Prescribing medications and other treatments in ways that they were not originally intended can have great benefits for patients, but it also comes with some risk, which physicians must be careful to manage.“Many established treatments, particularly in psychiatry, pain medicine, oncology and pediatrics, have off-label uses,” said Dr. Armand Aalamian, executive director of safe medical care and equity, diversity and inclusion at the Canadian Medical Protective Association (CMPA).The CMPA defines off-label use as prescribing a medication or medical device outside of its approved indication, dosage, route of administration, duration or patient population. Common examples include prescribing certain GLP-1 receptor agonists for weight management, tricyclic antidepressants for patients with chronic pain or fibromyalgia, or psychedelics like ketamine for chronic pain, post-traumatic stress disorder, and treatment-resistant depression. It is frequently used in pediatrics, since most drugs are approved based on clinical trials conducted on adults.While off-label treatments can often be effective, they can also be more risky for both the patient and doctor, due to uncertainty about the efficacy of the drug or product, and possible adverse reactions.That’s why it is important, said Dr. Aalamian, when considering off-label uses, that the approach is supported by evidence and is consistent with current standards of practice.“Clinical judgement is always critical,” he said. “Is (the off-label use) really appropriate, and have you taken the time to really get to know your patient, their condition, their confounding factors and co-morbidities?”The evidence supporting off-label treatments often comes from clinical experience on the ground and can eventually grow strong enough to lead to clinical trials and regulatory approval for that use. The use of some GLP-1s for weight management recently followed this route, as did the use of Botox to treat migraines.Clear communication with the patient, and getting informed consent, is always important, said Dr. Aalamian, but “even to a higher degree” when dealing with off-label treatments so that patients really understand why the treatment is being provided off-label and what are the risks and benefits. “That communication has to be really well documented,” he said. “It’s critically important that that documentation really reflects the discussion and the patient’s understanding of what this means and why it’s being used.”CMPA recommends that doctors check with the medical regulatory authority in their province to see if there are standards or guidelines on off-label use. But both the College of Physicians and Surgeons of B.C. and the College of Physicians and Surgeons of Ontario told the Medical Post that they do not have a practice standard specific to off-label prescribing.A spokesperson for CPSBC said in an email statement that licensees “are expected to broadly meet the standards set out in CPSBC's practice standards and the expectations of the profession. This includes prescribing within the limits of their competence, exercising sound clinical judgment, obtaining informed consent, maintaining appropriate documentation, considering the available evidence, and monitoring patients appropriately.”Doctors who are considering off-label treatments should follow the college’s Complementary and Alternative Medicine practice standard, the spokesperson said. In Ontario, off-label prescribing is generally addressed through the broader expectations set out in CPSO’s Prescribing Drugs policy. Since the colleges tend not to have specific advice on particular products or treatments, Dr. Aalamian said CMPA is there to help physicians talk through the decision to go off-label.“If in doubt, we encourage folks to call us and ask,” he said. “We try to provide what they need to look for to safely practice and safely use these off-label medications.”

Navigating the risks and rewards of off-label prescribing

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