Navigating the Regulation of Cell and Gene Therapies in Australia and New Zealand
Globally, cell and gene therapies are revolutionising modern medicine, offering groundbreaking treatments for life-threatening conditions including cancer, genetic disorders and rare diseases. However, despite the pace of scientific innovation, regulatory frameworks in many countries, including Australia and New Zealand, have struggled to keep up.
Neither country has dedicated regulatory pathways specifically for advanced therapies like cell and gene treatments. Instead, these therapies are regulated under legislation originating in the 1980s, creating challenges for companies entering these markets.
In this blog, we’ll explore the challenging landscape that is, cell and gene therapies regulation in Australia and New Zealand and discuss how a regulatory consultant can help navigate these complex frameworks.
Australia: Regulatory Oversight for Advanced Therapies
In Australia, the Therapeutic Goods Administration (TGA) is the primary body overseeing therapeutic goods, including cell and gene therapies regulation. These products are generally regulated under the Therapeutic Goods Act 1989. The absence of a dedicated regulatory framework for advanced therapies means that cell and gene therapies are regulated as either biologicals or prescription biological medicines.
Somewhat confusingly, biological medicines and biologicals are not regulated in the same way!
Biologicals
Human cell and tissue-based therapeutic goods, as well as live animal cells, tissues and organs are regulated as biologicals. This category includes gene therapies that involve ex-vivo manipulation of human cells such as CAR-T cell therapies.
Biologicals are further classified as classes 1 to 4, based on their risk to patients. Early classification of biologicals is essential for product development planning, as the class determines the requirements for product registration.
Prescription Medicines
Advanced therapies involving in-vivo genetic manipulation (such as monoclonal antibodies, vaccines without human cells, plasma derivatives, and recombinant products) are regulated as prescription medicines. Examples of treatments regulated as medicines include small silencing RNAs, CRISPR and other gene editing technologies, and gene therapies administered by vectors.
Clinical Trials
The TGA offers two primary pathways for approval of clinical trials: the Clinical Trial Notification (CTN) and Clinical Trial Approval (CTA) schemes.
- The CTN pathway is typically used for lower-risk therapies where sufficient preclinical data is available
- The CTA pathway is required for higher-risk or novel treatments like gene therapies, especially where safety data is limited
Gene Technology Approval
Any gene therapy that involves genetic modification must also be approved by the Office of the Gene Technology Regulator (OGTR). OGTR approval is required prior to TGA approval, and OGTR applications should be filed either before or concurrently with ARTG applications.
New Zealand: Regulatory Framework for Advanced Therapies
Like Australia, New Zealand regulates cell and gene therapies under its existing therapeutic goods legislation, notably the Medicines Act 1981. Under this act, advanced therapies are considered medicines, and the traditional medicines registration pathway applies.
Medicines
Advanced therapies in New Zealand must undergo the usual regulatory processes for new medicinal products including submission for approval to Medsafe, the country’s primary regulatory authority for therapeutic goods.
Clinical Trials
All clinical trials in New Zealand must be assessed by Health Research Council of New Zealand (HRC) and approved by Medsafe before commencing. Trials involving gene therapies are specifically reviewed by the Gene Technology Advisory Committee (GTAC), which evaluates the safety and ethical considerations of gene-based interventions.
Genetically Modified Organisms (GMOs)
Products involving genetic modification are also subject to regulation by the Environmental Protection Agency (EPA) under the Hazardous Substances and New Organisms Act 1996 (HAZNO Act). Approval from the EPA is necessary for the import, development, or release of GMOs.
Manufacturing and GMP Requirements
In both Australia and New Zealand, Good Manufacturing Practice (GMP) is essential to ensure the safety and quality of advanced therapies.
Australia
Manufacturing of prescription medicines supplied in Australia must meet the PIC/S Guide to Good Manufacturing Practice (PE009-16), except for some Annexes which are not adopted in Australia. Australian manufacturers will require a TGA licence for the relevant manufacturing steps following an on-site inspection. Overseas manufacturers of prescription medicines undergo a GMP Clearance process to ensure compliance to the PIC/S GMP principles.
If the advanced therapy product is classified as a biological, the manufacturing principles outlined in the Australian code of GMP for human blood and blood components, human tissues and human cellular therapy products must be applied. Since this standard is specific to Australia, TGA inspection of overseas manufacturing sites may be necessary to be able to register the product in Australia.
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New Zealand
Medsafe follows the New Zealand Code of Good Manufacturing Practice which is aligned with the PIC/S Guide to GMP, ensuring that all therapeutic goods manufactured meet internationally recognized quality and safety standards.
GMO Considerations
In addition to the GMP manufacturing requirements, approval may be required to manufacture the product; from OGTR in Australia or EPA in New Zealand. The key consideration for OGTR and EPA is to ensure the health of humans and the environment. In this regard, manufacturers must implement appropriate containment measures to prevent the accidental release of genetically modified cells. This may involve the use of secure facilities, specialised equipment, and strict protocols for handling and disposal of materials.
How Regulatory Consultants Can Support Your Cell and Gene Therapy Development
Given the complexity and nuances of regulatory processes in Australia and New Zealand, partnering with a specialised regulatory consultant can significantly streamline the approval and commercialisation of advanced therapies.
At The Adjutor Group, we specialise in providing regulatory support for companies in the cell and gene therapy sector. Our expert consultants can guide you through every step of the regulatory process, helping you bring your innovative therapies to market safely and efficiently, including:
- Tailored guidance: Our consultants can provide hands-on assistance with all aspects of the process including product classifications, gap analysis of your regulatory dossier, streamlined product registration, sponsorship support, regulatory approvals, and post-approval support (including distribution advice).
- Expertise and knowledge: With in-depth understanding of the complex and ever-changing regulatory landscape, our consultants can help ensure compliance with both local and global standards, reducing risks and building trust and credibility with your key stakeholders.
- Global expansion support: Adjutor, and our partner network, can assist in developing a global strategy for market entry, ensuring that your product complies with the requirements of multiple jurisdictions, enabling faster and more efficient access to new markets.
Australia & NZ
Cell & Gene Therapies Regulation.
For more information regarding advanced therapy regulation in Australia and New Zealand, contact The Adjutor Group for expert advice.
