Editorials
Pharmacology
Volume 200 - Issue 2

Off-label prescribing

Author:  J Paul Seale

Med J Aust 2014; 200 (2): 65. || doi: 10.5694/mja13.00184
Published online: 3 February 2014
Off-label prescribing (to patient groups or for indications not listed in a medicine's product information) is okay if supported by evidence
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Prescribing medications “off label” in some settings is appropriate as long as it is evidence-based

Many medication prescriptions are written for approved indications, as listed in the product information (PI) for the drug. “Off-label” prescribing is the term used when a drug is prescribed for an indication, a route of administration, or a patient group that is not included in the approved PI. There are groups of patients who are not included in the clinical trials undertaken for drug registration, and these groups may not be included in the PI. They typically include children, pregnant women, older men and women, and patients with terminal illness. Prescribing the medication for patients in these categories would be off label if they are not listed in the PI.

Based on a drug’s mechanism of action in an approved indication, it may be hypothesised that the drug may be efficacious in treating a medical disorder that is not listed in the PI. It is acceptable to prescribe off label, provided that there is sufficient evidence of efficacy and safety. This situation can arise for medications that have been available for many years, during which well conducted studies in patients with an off-label indication have shown acceptable efficacy and safety. If the drug’s patent has expired, it is unlikely that the pharmaceutical company that sponsors the drug will engage in the process of submitting data to the Therapeutic Goods Administration (TGA) for having the PI revised to include the new indication. The process for assessing the appropriateness of off-label medication use has been well described.1

The prescriber should inform patients that their prescription is off label so that they are not concerned by not finding their condition listed in the Consumer Medicines Information (CMI). Patients should also be informed about the rationale for prescribing the medication for them to increase the likelihood of good adherence to the treatment regimen.

Off-label indications are not covered by the Pharmaceutical Benefits Scheme (PBS), so patients will have to pay the full price for the medication. They need to be informed of this as well. The prescriber needs to monitor the tolerability of the medication and report any significant adverse events to the TGA to help develop a comprehensive profile of the medication prescribed in the off-label indication.

Provenance: Commissioned; not externally peer reviewed.

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