Understanding Unapproved & Off-Label Therapies: What Patients Need to Know
Introduction
When exploring treatment options, you may hear terms like off-label, unapproved, or unapproved product. These terms can be confusing, and it’s important for patients to understand what they mean, how they differ, and why some therapies require additional safeguards and regulatory processes.
What does approved mean?
In Australia, medicines are evaluated by the Therapeutic Goods Administration (TGA). If a medicine is approved, it means:
The TGA has assessed it for quality, safety and efficacy for specific uses
This is the standard regulatory pathway for most medicines patients encounter.
What is off-label use?
A medicine is used off-label when it is prescribed in a way that differs from the TGA-approved Product Information. This may relate to:
Off-label use is common and legal in Australia when supported by clinical judgement and appropriate safeguards.
However, off-label use still involves a medicine that is TGA-approved—only the use is different.
An unapproved therapy refers to a medicine, formulation or product that is not included in the Australian Register of Therapeutic Goods (ARTG). Unapproved therapies may include:
What is an unapproved therapy?
Because they are not listed on the ARTG, they cannot be supplied directly to patients without a special regulatory pathway or exemption.
How can unapproved therapies be prescribed in Australia?
Australian doctors can prescribe unapproved therapies using specific TGA mechanisms, most commonly:
1. SAS-B (Special Access Scheme – Category B): Allows a doctor to apply to the TGA for permission to prescribe an unapproved therapy for an individual patient, with detailed clinical justification.
2. Authorised Prescriber pathways: Allows certain doctors to supply an unapproved therapy to patients under their care once approved by a Human Research Ethics Committee (HREC) or the TGA.
3. Compounding: In some cases, pharmacists may prepare customised formulations when commercially available alternatives are not suitable. These still count as unapproved products, even if made by a licensed compounding facility.
Safeguards for unapproved or off-label therapies
1. Comprehensive Assessment: Thorough clinical assessment, review of investigations, and evaluation of other options.
2. Clinical Justification: The doctor must document why the therapy is being considered, why approved alternatives may not be suitable, and what the theoretical rationale is.
3. Detailed Informed Consent: Patients must understand that the therapy is unapproved, its use is off-label, risks, uncertainties, alternatives, monitoring and when it will be stopped.
4. Structured Monitoring: Scheduled reviews, pathology where appropriate, symptom tracking and escalation processes.
5. Communication With Your GP: With your consent, your GP or specialist is informed.
6. Documentation & Audit: All decisions, discussions and monitoring are recorded for safety and accountability.
Because these therapies have not been evaluated by the TGA, doctors must follow robust clinical and ethical processes, including:
Does unapproved mean unsafe?
No—but it does mean more information is needed.
Unapproved therapies may:
This is why Australian regulations mandate careful oversight, consent and documentation.
Unapproved or Off-Label Use at Regen Institute
At Regen Institute, any consideration of unapproved or off-label therapy follows a strict governance framework, including: