Australia has become a leading destination for early-phase and investigator-led clinical trials — thanks to its strong regulatory framework, skilled researchers, and efficient ethics approval processes. However, to conduct a clinical trial successfully, sponsors must understand their responsibilities, key stakeholders, and the pathways available through the Therapeutic Goods Administration (TGA).
1. Setting the Foundation for Successful Trial
Before the first participant is enrolled, the sponsor should have a clear strategy in place.
A successful clinical trial begins with minimum of:
- Well defined clinical trial objectives and research questions.
- Agreements with all collaborators/parties/suppliers involved in the clinical trial.
- Standardized methods/plans for: data management, monitoring, safety oversight and reporting of adverse events.
- Qualified investigators and clinical site(s).
- Indemnity and insurance in accordance with applicable regulatory requirements.
- HREC and institutional governance approvals.
- Quality management system (QMS) with policies and standard operating procedures (SOPs) compliant to the Good Clinical Practice (GCP) guidelines and regulations.
- Quality of the manufactured investigational medicinal product is appropriate to the clinical trial phase that will be conducted.
- Procedures for reporting progress, adverse events to ethics committees (study approving HREC) and regulators (TGA).
- Procedures for collation and retention of trial essential documents.
Every detail matters. Getting these foundations right prevents delays and ensures the trial runs smoothly from start to finish.
2. Who’s Involved in an Australian Clinical Trial?
A clinical trial in Australia involves several key stakeholders:
- TGA – the national regulator ensuring the safety, quality, and compliance of therapeutic goods manufactured and/or used in Australia.
- HRECs – reviewing and approving the ethical and scientific integrity of the proposed study.
- Approving institutions – providing site-specific governance approval.
- Sponsors – ultimately responsible for the conduct and compliance of the clinical trial.
- Investigators and sites – enrolling participants and conducting the trial under GCP principles.
- Volunteers Participants – the heart of every clinical trial.
3. The Role of the TGA
The TGA administers the Therapeutic Goods Act 1989, Therapeutic Goods Regulations 1990, and Therapeutic Goods (Medical Devices) Regulations 2002. Its responsibilities include:
- Inspecting and approving manufacturing standards for investigational products.
- Implementing GCP inspection program to verify compliance.
- Monitoring safety of investigational products (IPs)/investigational medicinal products (IMPs).
- Managing the Australian Register of Therapeutic Goods (ARTG).
4. Clinical Trial Pathways – CTN vs CTA
There are two schemes administered by TGA that can be used to conduct clinical trial in Australia, and these include CTN (Clinical Trial Notification) and CTA (Clinical Trial Approval) scheme. Authorised Prescriber Scheme (AP) and Special Access Scheme (SAS) cannot be used to conduct a clinical trial in Australia (see Australian clinical trial handbook).
The difference between CTN and CTA schemes are summarised below:
| Requirement | CTN | CTA |
| TGA review/approval | No | Yes |
| HREC review/approval | Yes | Yes |
| TGA notification | Yes | Yes |
| Institutional approval | Yes | Yes |
| Typical timelines | 2–10 days | 30–50 days |
| Fee (AUD, July 2025) * | $443 | Medicines: $2,111 (30 days) / $26,240 (50 days) Biologicals: $31,955 |
*Source: TGA fees and charges summary, 1 July 2025
The CTN scheme is the most common route used to conduct a clinical trial. It involves HREC and institutional approval, with subsequent notification to the TGA.
The CTA (Clinical Trial Approval) scheme, is similar to the US IND or EU CTA, and it requires regulatory (TGA) review of product scientific data before trial commencement. Sponsors intending to use CTA are encouraged to contact TGA early.
5. Understanding the HREC Process
All clinical trial in Australia must be approved by a HREC (also known in the US as an Institutional Review Board (IRB)) that is constituted and operating in accordance with the National Statement on Ethical Conduct in Human Research(National Statement). To approve a clinical trial, the HREC will review:
- The scientific justification and available safety data.
- Study design, recruitment, and data collection methods.
- Measures to protect participant welfare.
HREC also provides ongoing oversight throughout the trial and can suspend approval if the study no longer meets ethical standards.
When selecting an HREC, sponsors should ensure that it:
- Is registered with the NHMRC.
- Has experience with the specific IMP type (e.g., biologics, GMO products).
- Possesses the scientific expertise to assess risk and safety.
HREC review fees typically range from $4,000 to $15,000 AUD, depending on the institution and complexity.
6. Choosing the Right Investigator and Site
A qualified PI should:
- Appropriately qualified and hold current medical registration.
- Has relevant specialist credentials (e.g. paediatrician if the trial involves children).
- Free from any disqualification or suspension.
- Has Experience in conduct of clinical trials and compliance with GCP.
- Have prior GCP training and experience with similar studies.
- Have sufficient time and institutional support for the trial.
When choosing a clinical site, sponsors should consider:
- Staff experience and trial capabilities.
- Patient availability and recruitment potential.
- Infrastructure, equipment, and support needs.
- Level of support required from the sponsor or CRO.
Sites with less experience may need additional oversight and support to ensure GCP compliance.
7. Sponsor Oversight and Ongoing Responsibilities
Sponsors are ultimately responsible for GCP and regulatory compliance, even when tasks are delegated to CROs. They must ensure:
- Compliance with the Therapeutic Goods legislation.
- The clinical trial is conducted in accordance with the Declaration of Helsinki and National Statement on Ethical Conduct in Human Research ((the National Statement).
- Applicable commonwealth and/or state and territory legislation requirements have been met.
Sponsors must also:
- Have valid insurance and indemnity arrangements.
- Register the trial as required (e.g., ANZCTR).
- Monitor data quality, protocol and GCP adherence, and safety reporting.
- Notify TGA of any:
- CTN/CTA variations.
- Sponsor changes or transfers.
- Trial completion or extension.
- Adverse event reports.
- Withdrawal of ethics approval.
8. Common Issues Found by the TGA
TGA inspections frequently highlight issues such as:
- Risks associated with the use of the IMP and the proposed study including mitigation strategy not included in the protocol.
- Enrolling ineligible participants.
- Eligibility criteria not compliant with the IMP IB.
- Outdated or incomplete GCP training.
- Poorly defined inclusion/exclusion criteria.
- Unblinding process not clearly described in the protocol.
- No process in place for unblinding in case of clinical emergency.
- Protocol deviations and AEs not investigated and/or reported appropriately.
- Use of uncalibrated equipment.
- Failure to define dose stopping criteria.
- Inadequate communication or safety oversight.
Avoiding these pitfalls starts with strong planning, clear documentation, and proactive communication between the sponsor, CRO, PI and clinical sites, and HREC.
Australia’s clinical-trial environment is efficient and highly regarded, but compliance requires careful planning and ongoing sponsor engagement.
With robust systems, qualified partners, and clear communication, sponsors can ensure their trials are ethical, scientifically sound, and contribute meaningfully to advancing new therapies.
