How the TGA regulates gene therapy products
In the Therapeutic Goods Administration’s guidance, human cells that have been genetically modified are biologicals, and GMOs used as medicines, such as viral vectors, come under its rules for prescription medicines. For a biological that is not included in the Australian Register of Therapeutic Goods (ARTG), and not exempt from it, the TGA describes three access pathways: clinical trials, the Special Access Scheme and the authorised prescriber scheme.
General information, not medical advice. The TGA publishes the current rules, and a treating doctor can say whether any pathway applies to a particular patient.
- Regulator
- Therapeutic Goods Administration
- The register
- Australian Register of Therapeutic Goods (ARTG)
- Trial schemes
- Clinical Trial Notification (CTN) and Clinical Trial Approval (CTA)
- Under review
- The CTA scheme
The register, and what “unapproved” means
Generally, a therapeutic good must be included in the ARTG. A product that is not included is called an unapproved therapeutic good, and the TGA says it has not evaluated such a product, so its quality, safety and effectiveness have not been assessed. Biologicals on the ARTG, by contrast, have been evaluated for quality, safety and efficacy.
The ARTG can be searched on the TGA’s website, filtered by product type, including biologicals.
Four classes of biological
The TGA classifies biologicals by the level of risk to patients, which depends on how much the product is processed, what it is used for, and how much outside governance and clinical oversight it has. Read from the foot of the ladder up:
- Class 1Low risk, with outside governance and clinical oversight
When the TGA published its guidance, only faecal microbiota transplant products, under certain conditions, were Class 1.
- Class 2Low risk
When the guidance was published, only medicinal maggots used in maggot debridement therapy, under certain conditions, were Class 2.
- Class 3Medium risk
Includes products prepared with more than minimal manipulation, and products for non-homologous use, as the TGA defines those terms. Stem cell products other than pluripotent ones will generally be treated as Class 3.
- Class 4High risk
Includes human cells or tissues modified to carry out a function they did not have when collected, pluripotent stem cells and products made from them, and products containing live animal cells, tissues or organs. The TGA’s examples of modification that puts a product here include CAR T cells and induced pluripotent stem cells.
Summarised from the TGA’s guidance on classifying biologicals, last updated 8 September 2026.
Some products that meet the definition of a biological are regulated another way. The TGA lists vaccines, recombinant products and plasma-derived products among the goods declared not to be biologicals, and regulates them as prescription medicines. For a GMO used as a medicine, the TGA requires sponsors to consult the OGTR about their obligations under the Gene Technology Act, including for a clinical trial, and the TGA must itself tell the OGTR about applications to supply therapeutic goods that contain GMOs.
Trials: notify the TGA, or apply to it
A clinical trial in Australia that uses an unapproved therapeutic good must either be notified to the TGA under the Clinical Trial Notification (CTN) scheme, or approved under the Clinical Trial Approval (CTA) scheme. Either way, a Human Research Ethics Committee (HREC) must approve the trial before the unapproved good is supplied.
| Question | CTN: notification | CTA: approval |
|---|---|---|
| Does the TGA look at the data? | No. The TGA does not evaluate any data about the trial when it is notified. | Yes. The TGA evaluates scientific data about the product, including quality, preclinical and early clinical safety data, and decides whether to approve supply before the trial starts. |
| Who decides? | Approval comes from the HREC and from the institution where the trial runs. The TGA’s acknowledgement is not needed before recruiting begins. | A senior medical officer of the TGA decides whether to approve supply; the HREC reviews and approves the protocol and monitors the trial. |
| When is it used? | When the requirements of the CTA scheme do not apply. | It is mandatory for a Class 4 biological, unless evidence from earlier clinical use supports the biological, or a regulator with comparable requirements has approved a trial for an equivalent indication. |
For trials that are not of a Class 4 biological, the sponsor chooses the scheme first, then the HREC that approves the protocol; the HREC weighs whether it has the scientific and technical expertise to assess the product’s safety. The TGA also inspects trial sites under its Good Clinical Practice inspection program.
Under review. The TGA has committed to review the CTA process, in response to a report by MTP Connect on the Cell, Gene and Tissue Regulatory Framework in Australia, and says its page will be updated as the review progresses; that page was last updated on 1 September 2024. Check the TGA’s CTA review page before relying on the process described here.
Outside a trial: one patient, or a few
Two other pathways let a practitioner obtain a biological that is not on the ARTG. Under the Special Access Scheme, any regulated therapeutic good can be used for a single patient, case by case. Under the authorised prescriber scheme, a medical practitioner may be authorised to prescribe a specified unapproved biological to particular patients, or a class of patients, in their immediate care. An authorised prescriber needs approval from an HREC or endorsement from a specialist college, and must have the TGA’s approval letter before supply.
Most of these pathways need a medical or dental practitioner to make the notification or request, and patients cannot make them themselves. The pathways cannot be used to make commercial supply easier, and unapproved biologicals supplied through them must generally be made to appropriate good manufacturing practice, though unapproved biologicals used in first-in-human clinical trials are exempt from being made in a GMP-accredited facility.
On cost, the TGA’s consumer page says unapproved products are generally not covered by the Pharmaceutical Benefits Scheme, so the full price is paid, and the TGA has no control over the price companies charge.
Cell treatments offered outside the register
Some products made from a patient’s own cells are excluded from TGA regulation, but only when all three of these hold: the cells are collected from a patient under the clinical care of a registered medical or dental practitioner; they are made by that practitioner, or under their supervision in a hospital, for that patient of that hospital; and the product is not advertised to consumers. If any one is not met, advertising to consumers included, the TGA regulates it.
The TGA’s consumer guide to stem cell treatments, published on 11 September 2019, says haematopoietic stem cell transplantation, with stem cells taken from bone marrow or cord blood, for blood and immune system disorders such as leukaemia, is the only stem cell treatment in Australia proven to be safe and effective, and that no other stem cell treatment has been shown to be safe and effective. It warns that an unproven stem cell treatment may be unsafe and pose serious risks to health, including infection, allergic reactions and the development of cancer, with complications that can be fatal, and that having one can disqualify a person from a registered clinical trial. It also notes that the TGA does not regulate medical practice.
Reporting a side effect
A consumer can report a side effect from a medicine or vaccine to the TGA online, and a GP, nurse practitioner, midwife or community pharmacist can help with the report. Inside a clinical trial the route is different: the Australian Clinical Trials website asks participants to report side effects to the researcher, not to the TGA. Rung 03 on clinical trials sets out who to contact, in order, and rung 05 covers the separate licence a trial of a GMO may need.