Duties and responsibilities
Risk management when using drugs or medical devices off-label
Originally published September 2012
Using medications or medical devices off-label may benefit patients, but can lead to medico-legal risks for physicians — risks that include uncertainty about the efficacy of the drug or product, and possible adverse reactions.
The term off-label refers to using licensed medications or medical devices outside of the condition or indication for which the licence was issued. This use differs from administering medications or using devices that have never been considered for a licence. Prescribing a drug in a way that is inconsistent with the label of any product for which a drug identification number has been issued by Health Canada is considered off-label use of the drug. 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. 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 use include prescribing tricyclic antidepressants for patients with chronic pain or fibromyalgia, using antipsychotic medications to treat agitation in dementia patients, or using a dose of a medication above the designated upper limit. 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.
What the CMPA's cases revealed
The CMPA frequently receives calls from members asking for medico-legal advice on the off-label use of medications or devices, and has a number of files on the matter involving legal actions or College complaints.
A review of the cases highlighted the importance of physicians taking the following actions:
- Reviewing the available medical literature (e.g. guidelines from medical specialty organizations) and considering if the proposed off-label use of the medication or product has gained enough acceptance among peers.
- Taking reasonable precautions to ensure that the prescription or use of the drug or device is the appropriate treatment for the patient's condition.
- Advising patients that a drug or device is not approved for their particular condition.
- Obtaining and documenting the appropriate informed consent before using the drug or device off-label.
A 16-year-old male had a history of alcohol dependency, and severe generalized anxiety and oppositional defiance disorder.
The patient's psychiatrist talked to him and his parents about treatment with paroxetine 30 mg OD. The discussion included the material risks of using the medication. The courts consider material risks to be, generally, the ones a reasonable person, in the same circumstances, would want to know before consenting to an investigation or treatment. Following the discussion, the psychiatrist prescribed paroxetine. Based on the product monograph, paroxetine was not indicated for use in patients below the age of 18 years.
The patient experienced good control of his anxiety after he was started on paroxetine. After a pharmaceutical warning about the increased risk of suicide in adolescents on paroxetine was sent to physicians, the patient's psychiatrist made plans to admit him to hospital in this particular case. In hospital the patient's paroxetine was tapered and he was started on olanzapine. He was discharged two months later and committed suicide.
The patient's parents complained to the College that they had not been advised that the use of paroxetine to treat this condition was off-label. The College was supportive of the physician's care, stating that the lack of clinical trials in patients under the age of 18 did not imply that the medication was unsafe for patients under age 18. The College highlighted the importance of obtaining informed consent when prescribing drugs off-label.
A 92-year-old female with a history of dementia was assessed by a psycho-geriatrician, who felt that the patient's depression was affecting her behaviour.
He recommended olanzapine and citalopram to improve her sleep, and day-time orientation. The specialist also recommended an assessment of cardiovascular risk factors before prescribing olanzapine. Based on the specialist's recommended treatment, the patient's family physician started the patient on olanzapine and citalopram. The patient died three months later.
The family complained to College about the family physician's care stating that olanzapine was approved for the treatment of schizophrenia and bipolar disorder and was not indicated for the elderly with dementia, so was being used off-label. The College supported the care provided by the family physician and acknowledged that he had followed the psycho-geriatrician's recommendations. There were no absolute contraindications to the use of olanzapine in an elderly patient with dementia. It also stated that physicians are advised to assess the risks and benefits of the use of olanzapine in elderly patients with dementia.
A 25-year-old female went to her family physician to have a tattoo removed from her arm.
The family physician discussed treatment options, including treatments with a diode laser therapy that was approved only for hair removal. He advised her that there was a risk of poor healing with all techniques, but he did not discuss the specific risks associated with use of laser treatment or that the laser had not been approved for this use.
The patient suffered a second degree burn from the treatment and, as a result, started a legal action. An expert confirmed that the device was not medically approved for this use and said that the use of diode laser for tattoo removal did not conform to the standard of care at the time the patient was treated. In addition, experts in the case criticized the family physician's consent discussion given the device was being used off-label. The matter was settled on behalf of the defendant physician.
How to minimize the risk of liability when using medications or medical devices off-label
- Determine if the proposed use of the medication or device constitutes 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?
- Document the rationale for using the medication or device off-label.
- 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' consent after an appropriate discussion of the potential risks and benefits, and after a discussion about the medication or device being used in an off-label fashion.
- Document any questions asked by patients and the answers provided.
- Carefully monitor patients for side-effects during or following on an off-label treatment.
CMPA members with further questions on this challenging topic are encouraged to contact the CMPA for advice.