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Explainer: why are off-label medicines prescribed?

Explainer: why are off-label medicines prescribed?

I expect many of us have heard of the term 'Off-label', but aren't quite sure what it means. Medical drugs are approved for a specific use by government agencies, e.g. the Therapeutic Goods Administration (TGA) in Australia, the Food and Drug Administration (FDA) in the United States and the European Medicines Agency in the European Union. To gain this approval, "The drug company has to specify the health conditions the medicine will be used for (also called “indications”), the doses, the routes of administration (tablet, injection, lotion, for instance) and the types of patients who will use the medicine.


If a prescription medicine is used for a different reason, at a different dose or route of administration, or in different patient groups from those approved by the TGA (in Australia), then this is referred to as “off-label” use."

Here's an excellent explanation of why medicines are prescribed off label, along with the associated risks and benefits by Lisa Kalisch Ellett, Research Fellow, Quality Use of Medicines and Pharmacy Research Centre at University of South Australia:

As Lisa concludes, Off-label use of medicines can play an important role in health care, particularly when this is the only treatment option for patients. But it’s important to remember that we still need evidence to show the medicine works for the off-label condition and that the benefits of using the medicine outweigh the risks."


Photo: Spot the impostor; a royal spoonbill mixes with some ibis, hoping they won't notice.

2 Replies

Brad7 Neil could you give some indication of what drugs would be avalable in Australia as a first treatment after W&W had ended,


Hi Brad,

That's a good question to which I don't have an answer, only to say that our choices are less than in Canada, the UK or the USA. Given you, like me are still CLL treatment naïve, I hope that there will be more choices if we get to that point. For those that have had their first treatment, there are more clinical trial opportunities to access drugs still unapproved in Australia and if available treatments are unsuitable, it may be possible to gain compassionate access to newer drugs, including non-chemo options like Ibrutinib, Idelalisib and Venetoclax (ABT-199). Unfortunately that can be a matter of timing, location, etc all working in your favour.

The Australian Leukaemia Foundation plan to have a document 'Accessing Blood Cancer Drugs in Australia' available for download sometime this October:

Keep an eye out for it.


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