Understanding “Off Label” Prescribing and Compounded Medicines
Patients are sometimes surprised to learn that a medicine may be prescribed for a use that does not appear in its official approved indications. This is commonly referred to as off label prescribing.
In Australia, medicines are regulated by the Therapeutic Goods Administration (TGA). When a prescription medicine is approved, the TGA assesses evidence relating to its quality, safety and efficacy for particular uses. These approved uses are listed in the medicine’s Product Information (PI), which doctors can access through clinical prescribing software and reference systems.
Most prescription medicines approved for supply in Australia display AUST R followed by a number on the packaging. This indicates inclusion on the Australian Register of Therapeutic Goods (ARTG).
What does “off label” mean?
Off label prescribing occurs when a registered medicine is prescribed:
- for a condition not specifically listed in the Product Information
- at a different dose
- in a different age group
- by a different route of administration
- or otherwise outside the approved Australian indications.
This does not automatically mean the treatment is inappropriate. In some areas of medicine, off label use is common and supported by specialist experience or published evidence.
Where compounded medicines fit in
There can be overlap between compounded medicines and off label prescribing.
A compounded medicine is typically prepared by a compounding pharmacy to create a customised formulation, dose, strength, or combination that is not readily available as a standard commercial product. This may occur where a patient has special requirements, allergies, swallowing difficulties, or needs a formulation not otherwise available.
Sometimes compounded medicines are used as part of an off label treatment strategy, particularly when no standard Australian product exists in the required form. However, compounding and off label use are not identical concepts:
- A medicine can be off label without being compounded.
- A compounded medicine may or may not involve off label use.
Where a pharmacy compounds a medicine, responsibility rests with the manufacturer/compounder to ensure appropriate standards of quality, safety and efficacy.
Confusion from overseas internet information
Confusion can arise when patients research medicines online.
A medicine found on overseas websites may have different approved uses, strengths, formulations, brand names, or regulatory status in another country. Some medicines approved overseas may not be approved in Australia for the same indication, and some products sold online may not meet Australian regulatory standards.
For this reason, internet information should be interpreted cautiously. What is approved in the United States, United Kingdom or elsewhere does not necessarily mean the same approval applies in Australia.
My practice approach
At this clinic, my usual approach is to require a written recommendation from an appropriate specialist who has experience in the proposed off label use of the medication in question.
This helps ensure:
- the treatment is clinically justified
- recognised alternatives have been considered
- benefits and risks are clearly assessed
- monitoring arrangements are appropriate
- prescribing remains evidence-informed and patient-centred.
The key principle
Whether a medicine is standard, compounded, or proposed for off label use, the central questions remain:
- Is the product of appropriate quality?
- Is it likely to be safe?
- Is there sound evidence it will help?
- Is it suitable for the individual patient?
Careful prescribing decisions are best made through discussion between patient, GP, and where appropriate, the relevant specialist.
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