Australia

Benefits: Australia plays a pivotal role in global clinical research, particularly in rare and genetic diseases, due to its streamlined regulatory processes, world-class research facilities, and patient-centric approach. The country’s review processes enable clinical trials to start quicker than other countries, often within 6-8 weeks, allowing patients faster access to cutting-edge treatments. Additionally, Australia has a strong reputation for high-quality clinical research, with specialized centers in genetics, pediatrics, and rare diseases, making it an attractive destination for international patients seeking innovative therapies.  

Spotlight: Royal Melbourne Hospital (RMH) – Melbourne, Victoria

Key Features:

• Specializations:

  • Neurological rare diseases (ALS, Huntington’s)

  • Lysosomal storage disorders (Gaucher, Fabry)

  • CAR-T cell and gene therapy trials

• Why It Leads?
  Fastest trial approvals (6-8 weeks via Australia’s streamlined system)

   CREDO Network Hub (Australia’s largest rare disease research platform)

   30% international patients (Medical Visa Subclass 602 support)

• Global Partnerships:

  • Collaborates with NIH (USA) and European Reference Networks (ERNs)

• How to access a rare disease clinical trial if you’re a non-Australian national? 

  • The patient or patient team would need to check if the trial of interest is recruiting international patients. 

• How to apply? 

  • Patients would need to apply for a medical treatment visa - Subclass 602

  • If applying from inside Australia

    • Visa costs ~ AUD 370 

  • If applying from outside Australia 

    • Visa is free

• Costs to consider

  • The patient would cover the costs of living expenses in Australia while participating in the trial. 

  • Trial participation can be free. Or, the patient or patient team can apply for support grants or reimbursements through NORD or EURORDIS.

• Check for clinical trials in Australia on the Australian Clinical Trials website.

Notable rare disease clinical trials research centers in Australia