6. Responsibilities of the prescribing doctor

6.1. Doctor-patient discussion in off-label use

Legal and ethical principles require doctors to inform patients about risks of medical treatments. While off-label prescriptions are common and sometimes necessary, they also present significant risks. Given that off-label prescribing of medicines may expose patients to unnecessary risks, it is ethically required for doctors to disclose off-label prescribing to patients and seek their consent to the off-label use.

This includes discussing with the patient/parents/carer the reason for using the medicine, possible alternative therapies (in case there are any) and potential side effects.

Items to discuss could be:

  • Whether the medicine is approved for treatment of the particular condition or would be used off-label.
  • Whether there is an approved alternative.
  • Whether the off-label treatment has advantages compared to approved alternatives.
  • Whether credible research supports the off-label use.
  • Whether using the medicine off-label could carry additional risks.

Additional information about any uncertainties associated with the use off-label should be given. Patients should ask for additional information to address specific concerns.

Documentation of the consent process is recommended and, in some cases, obtaining written consent may be appropriate.

The informed consent should be expanded to require disclosure of off-label prescribing where the medicine has not been proven safe and effective for the specific condition, especially where scientific evidence is inadequate and risks are substantial or unknown.

Requiring disclosure protects patient autonomy and educates patients about alternatives and risks, leading to improved healthcare decisions.