The Gene Technology Regulator and gene therapy trials
The Gene Technology Regulator administers the Gene Technology Act 2000, and the TGA explains that in Australia dealings with genetically modified organisms (GMOs) are prohibited unless explicitly authorised under that Act. Putting a GMO into a person in a clinical trial therefore needs the Regulator’s licence, unless the GMO is a modified human somatic cell that meets specific conditions, such as a patient’s own cells modified and returned to them.
General information, not legal or medical advice. The Office of the Gene Technology Regulator publishes the current rules and guidance, and its own guidance encourages applicants to talk to it before applying.
- The law
- Gene Technology Act 2000 and Regulations 2001
- The Regulator
- An independent statutory office holder
- Supported by
- The OGTR, in the Department of Health, Disability and Ageing
- Under review
- The Act and the Regulations
Who the Regulator is
The Gene Technology Regulator is an independent statutory office holder who administers the Gene Technology Act 2000 and the matching state and territory laws, and is appointed by the Governor-General only with the agreement of a majority of all jurisdictions. The Office of the Gene Technology Regulator (OGTR) supports the Regulator and is part of the Department of Health, Disability and Ageing.
The Regulator’s job is to protect the health and safety of people, and the environment, by identifying the risks posed by gene technology and managing them through regulating dealings with GMOs. A dealing covers almost anything done with a GMO, including experimenting with it, making it, importing it, transporting it and disposing of it.
Why there are two regulators
The TGA regulates clinical trials of therapeutic goods under the Therapeutic Goods Act 1989, and its requirements, including approval from a Human Research Ethics Committee, must be met before a trial starts. The Gene Technology Regulator looks at something different: the dealings with the GMO itself. The TGA requires sponsors of a GMO medicine, including in a clinical trial, to consult the OGTR, and must itself inform the OGTR of applications to supply therapeutic goods that contain GMOs. The OGTR does not require the approvals in any particular order; they can run in parallel.
Under the Act an organism includes any biological entity capable of transferring genetic material, which the OGTR reads as covering replication-defective viral vectors. Rung 01 explains what that means for what counts as gene therapy.
Which approval a trial needs
The OGTR’s guidance sorts trials by what the GMO is and what happens to it once it is in a participant.
- If the product isModified human somatic cells
Including a person’s own cells, modified and returned to them. No licence is needed if the cells cannot secrete or produce infectious agents because of the modification, and, where a viral vector was used, the cells have been tested free of other viruses likely to recombine with it and the vector is no longer present.
These are exempt dealings: they must not involve intentional release into the environment, and once the cells are in the person they are no longer regulated under the Act. Making the cells in Australia must itself be authorised.
- If the GMO may be releasedA DIR licence
A licence for dealings involving intentional release, needed where the way it is given releases the GMO, such as an intranasal spray or a nebuliser, or where it may be shed, excreted or passed on to other people or animals during the study.
Trial licences of this kind are usually for a “limited and controlled” release.
- If it is not expected to be releasedA DNIR licence
A licence for dealings not involving intentional release, supported by data showing release is not expected. The OGTR says it considers some AAV-based gene therapy treatments eligible for this kind of licence.
A product made from a GMO but containing no viable GMO, such as a purified recombinant protein or a killed vaccine, needs no authorisation from the Regulator for a trial.
How a licence application runs
The OGTR encourages applicants to contact it before applying. The steps it sets out:
- The application form is completed by the applicant, usually an organisation with a legal presence in Australia, such as an Australian sponsor, a local clinical research organisation, or the site of a single-site trial.
- An Institutional Biosafety Committee (IBC) reviews it and adds supporting information. IBCs include a range of suitable experts and at least one independent person.
- The OGTR receives and screens it for completeness, and gives it an ID number once accepted.
- The OGTR prepares a risk assessment and risk management plan for the application.
- The Regulator decides and notifies the applicant, and the decision goes on the public GMO Record.
| Licence | Working days to decide |
|---|---|
| DNIR | 90 |
| Limited and controlled DIR | 150 |
| Limited and controlled DIR, where significant risk is identified | 170 |
Weekends, ACT public holidays and time spent waiting for the applicant to answer certain requests do not count. The OGTR’s guidance, last updated in September 2022, says there is currently no fee for applications under the Act.
One licence can cover a trial at several sites. Before work with the GMO starts at each site, licence conditions generally require the Regulator to be told the site’s details and given a compliance management plan.
The public GMO Record
The Record of GMO Dealings, running since 2001, gives the community information about GMOs in Australia, and covers every dealing except exempt ones. It includes therapeutic goods containing GM products approved for supply under the Therapeutic Goods Act. The OGTR’s “what we’ve approved” pages are the way in.
Rules under review
Changes to the national gene technology scheme are in progress: the OGTR says implementation of the 27 recommendations of the Third Review of the National Gene Technology Scheme is under way.
So the licence types and timeframes on this page may change. Check the OGTR’s legislative reviews page and the OGTR’s guidance before relying on them. The TGA’s side of a trial is on rung 02, and how trials are found and run is on rung 03.