A choice of approval procedures for clinical trials in Australia and New Zealand, depending on the degree of risk to participants, would be available under proposed regulatory arrangements for the joint Australia New Zealand Therapeutic Products Authority (ANZTPA).

The clinical trials model is based on the risk-management principles that would generally apply to the regulation of medicines, medical devices and biologicals by the ANZTPA, a consultation paper explains. Studies posing a high level of risk to participants, and requiring prior review by the ANZTPA, would be subject to a Clinical Trial Assessment (CTA) Scheme. All other studies would fall under the less demanding Clinical Trial Certification (CTC) Scheme.

The CTA Scheme would come into play when the ANZTPA or sponsor/investigator had determined that a product might pose a high risk of harm to trial participants, whether due to the nature/toxicity of the product itself or the phase/type of clinical trial. This judgment might also follow the advice of a Human Research Ethics Committee (HERC) in Australia or comparable advisory body in New Zealand.

Specifically, the CTA Scheme would apply to:

- Trials involving the first administration in humans of a “new “ substance; a novel Active Implantable (or organ support) Medical Device; or a hybrid medicine/biological device

- Trials of gene therapy products

- Trials using Class 4 human cells or tissues

- Trials involving xenotransplantation

Any clinical trials approved through the CTA Scheme would require evidence of ethics committee endorsement before the trial could go ahead. To reflect differences in underlying legislation and the ethical review systems in Australia and New Zealand, sponsors would need to apply for separate ethics committee clearance in whichever country/countries the trial was due to take place. Where a data package had been lodged with the ANZTPA, individual trial proposals falling within the usage guidelines submitted as part of the CTA could be presented in parallel to the ethics committee for consideration and approval.

The ANZTPA assessment would include a review of the quality, safety and efficacy of the information supplied. Once a CTA had been cleared, subsequent trials consistent with the approved usage guidelines could proceed via the CTC route in either country.

Clinical Trial Certification>

The risk-managed approach proposed for the CTC Scheme is based on a mixed model of assessment and self-certification by the trial sponsor. It would not routinely involve a full scientific review of the product and its proposed usage by the ANZTPA. Study proposals submitted under the CTC Scheme would, however, first need to obtain ethics committee approval in Australia and/or New Zealand.

Evidence of ethics committee approval would be part of the self-certification requirements for trials using the CTC route. Sponsors would also need to supply evidence that the product involved was manufactured in a Good Manufacturing Practice-licensed or otherwise compliant facility; an undertaking by the chief investigators and sponsors that the proposed study complied with Good Clinical Practice (GCP) requirements as set out in ANZTPA guidelines; and an undertaking from the trial site(s) and ethics committee that officers from the joint authority could inspect the site(s) for compliance with GCP where appropriate.

The following types of study would also require submission to the ANZTPA of basic trial documentation, such as the investigator’s brochure and trial protocol:

- First use of a therapeutic product in a new patient group, for a new indication or via a “substantially different” route of administration

- Trials involving gene therapy, xenotransplantation or use of Class 4 human cells or tissues that had previously been judged by a relevant ethics committee to present a lower-risk profile suitable for the CTC route.

The deadline for comments on the ‘Proposed clinical trial regulatory arrangements under the Australia New Zealand Therapeutic Products Authority’ is 18 April 2007.