All human clinical trials undertaken in Australia, including trials of gene therapy, must be conducted under either the Clinical Trial Notification (CTN) or Clinical Trial Exemption (CTX) schemes established by the Therapeutic Goods Act 1989 (Commonwealth), and overseen by the Therapeutic Goods Administration (TGA), the competent authority for regulating therapeutic goods in Australia. Further information about clinical trials in Australia is available on the TGA website (www.australianclinicaltrials.gov.au/).

Human Research Ethics Committees (HRECs) play a central role in the Australian system of ethical oversight of research involving humans. HRECs review research proposals involving human participants to ensure that they are ethically acceptable and in accordance with relevant standards and guidelines. HRECs may be established by research organisations (for example hospitals and universities), and are registered with the National Health and Medical Research Council (NHMRC) (www.nhmrc.gov.au).

The choice of which clinical trial scheme to follow (CTN or CTX), lies firstly with the sponsor and then with the HREC that reviews the trial protocol. Under the CTN scheme, the HREC will first review the trial and then notify the TGA; whereas under the CTX scheme, the trial is reviewed by the TGA directly, and may not proceed until approval is granted. The CTX scheme may be more appropriate where the experimental protocol includes new technology, new material, or a new treatment concept which has not been evaluated previously in clinical trials in any country, including ‘first-in-man’ trials. In assessing human clinical trial protocols, including those involving gene therapies, HRECs and the TGA must consider patient safety and efficacy.

In Australia, human clinical trials involving gene therapy may also require approval under the Gene Technology Act 2000 (Commonwealth) (the ‘GT Act’), administered by the Gene Technology Regulator (the Regulator). The GT Act underpins the national scheme for the regulation of live and viable genetically modified organisms (GMOs) in Australia. The object of the GT Act is: “to protect the health and safety of people, and to protect the environment, by identifying risks posed by or as a result of gene technology, and by managing those risks through regulating certain dealings with GMOs.”

Under the Gene Technology Regulations 2001 (Commonwealth), the introduction of a GMO into a human, including human clinical trials involving gene therapy, requires a licence from the Regulator. Further information about the licensing requirements for human gene therapy clinical trials involving GMOs is available from the Office of the Gene Technology Regulator (OGTR) (www.ogtr.gov.au; This email address is being protected from spambots. You need JavaScript enabled to view it.; +61 2 6271 4262).

(Last reviewed by the Competent Authority: 12 July 2010)