Gene therapy in Australia, explained carefully
Healthdirect Australia, the national public health information service, describes gene therapy as a medical treatment that aims to treat or prevent genetic conditions by delivering a corrected gene into a person’s cells. It also says gene therapy is mostly still in research and clinical trials, and is not generally available in Australia. This guide, published by Dotto, follows a gene therapy through the rules it meets on the way: the regulators, the trials, the ethics review and the consent.
General information, not medical advice. Your own doctor or specialist is the person to talk to about any treatment, and healthdirect’s guide to genetic disorders is a good official place to start reading.
Six rungs, one question each
Each rung answers one question from the pages of the body that is responsible for it. Start anywhere; each one links to the others.
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Rung 01
What gene therapy is
How healthdirect, the TGA and the Gene Technology Regulator describe it, and the laws on embryo research and mitochondrial donation.
Written from: healthdirect, TGA, OGTR
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Rung 02
How the TGA regulates it
The four classes of biological, notifying or applying for a trial, and the pathways for unapproved products.
Written from: Therapeutic Goods Administration
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Rung 03
Clinical trials, and finding one
The phases of a trial, searching the national registry, who signs off a gene therapy trial, and who to contact with a concern.
Written from: Australian Clinical Trials
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Rung 04
Consent and research ethics
The values an ethics committee applies, who sits on one, and what consent involves when research touches genetic information.
Written from: NHMRC
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Rung 05
The Gene Technology Regulator
Why a viral vector is a GMO, which trials need a licence, how long a decision takes, and the rules now under review.
Written from: Office of the Gene Technology Regulator
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Rung 06
Questions to ask a doctor
Questions to take to an appointment about a genetic condition and its treatment, set out from healthdirect’s guidance.
Written from: healthdirect
Who looks after what
Several bodies share the oversight of gene therapy research and products in Australia. In one line each, from their own pages:
| Body | What it looks after | Read more |
|---|---|---|
| Therapeutic Goods Administration (TGA) | Therapeutic goods, including medicines and biologicals. A clinical trial that uses an unapproved therapeutic good must be notified to the TGA or approved by it. | Rung 02: how the TGA regulates it |
| Gene Technology Regulator, supported by the OGTR | Dealings with genetically modified organisms (GMOs), to protect the health and safety of people and the environment. A human clinical trial of a GMO needs the Regulator’s licence, unless it is a trial of patients’ own cells, modified and returned to them, that meets set requirements. | Rung 05: the Gene Technology Regulator |
| Human Research Ethics Committees (HRECs) | Reviewing research with human participants to check that it is ethically acceptable. For a trial of an unapproved good, the HREC approves the protocol and monitors the trial. | Rung 04: consent and research ethics |
| National Health and Medical Research Council (NHMRC) | The National Statement on Ethical Conduct in Human Research, developed jointly with the Australian Research Council and Universities Australia, and a registration scheme for HRECs. | NHMRC: the National Statement |
| Australian Clinical Trials website | Information about clinical trials and a search of trials listed in the Australian New Zealand Clinical Trials Registry. The Australian Government does not refer people to individual trials. | Rung 03: clinical trials, and finding one |
| State and territory health complaints bodies | Complaints about healthcare providers and registered health professionals, which they have formal powers to investigate. | Australian Clinical Trials: the list of complaints bodies |
Three lines the official sources draw
Worth holding in mind before reading anything else about gene therapy, including advertising.
- Where it mostly is
Healthdirect says gene therapy is mostly still in research and clinical trials, and is not generally available in Australia.
- What “unapproved” means
The TGA says a product that is not in the Australian Register of Therapeutic Goods has not been evaluated by it, so its quality, safety and effectiveness have not been assessed.
- Embryo research and cloning
The NHMRC explains that the Research Involving Human Embryos Act 2002, together with the Prohibition of Human Cloning for Reproduction Act 2002, prohibit human cloning for reproductive purposes and regulate research that uses human embryos, with strong penalties for breaking them.
About this guide
genetherapy.com.au is a concept website: a guide to gene therapy in Australia in plain words, published by Dotto. It is not part of the TGA, the Office of the Gene Technology Regulator, the NHMRC or any health service, and has no connection with them.
Each rung is written from the official pages of the body responsible for it, and every page lists its sources at its foot. It describes the rules and how the processes work. Whether a treatment or a trial is right for a particular person is a matter for that person and their own doctor.
Rules change, and three of the schemes described here are under review as this guide is written: the TGA’s Clinical Trial Approval scheme, the national gene technology scheme and parts of the National Statement. Where this guide and an official page differ, the official page is right.
