The first Australian Rare Disease Research Network (ARDRN) virtual meeting for 2026 was held in May. The ARDRN has grown to over 120 members, including rare disease researchers from a range of jurisdictions, disciplines and career stages. Importantly, there are members with research policy expertise, including representatives from Genomics Australia and Research Australia.
Rare Voices Australia (RVA) thanks the 56 ARDRN members who joined the May meeting. Additionally, we thank ARDRN members, Dr May Aung-Htut, Dr Cherylea Browne, Dr Laura Croft and Associate Professor Honey Heussler, for sharing their research and ARDRN co-chairs, Clin/Prof Gareth Baynam and Dr Lisa Ewans, for co-chairing the meeting.
The focus of the meeting was Priority 1 of Australia’s Top 10 Rare Disease Research Priorities, ‘Development of, and access to, the best treatments and cures, including clinical trials’. This priority directly aligns with several areas in the Australian Government’s National Strategic Action Plan for Rare Diseases, including Priority 2.4, ‘Enable all Australians to have access to the best available health technology’.
During the meeting, discussions highlighted both the impressive strength and breadth of Australia’s rare disease research efforts under Priority 1 and the urgent reforms needed to deliver fair and equitable access at scale. ARDRN member contributions reflected a research community with deep expertise, strong leadership, and a clear commitment to collaboration, innovation and reducing duplication.
Australia Has the Research Capability to Become a Global Leader in Rare Disease Therapeutics
Many ARDRN members are actively inviting collaboration and offering their tools, knowledge and expertise to support shared progress across the sector. Many attendees expressed the need for major reform to deliver on Priority 1, including:
- More sustainable funding—current progress is dependent on philanthropy, internal institutional support and family-led fundraising.
- Stronger national infrastructure including registries, biobanks, and shared patient-derived materials.
- Better visibility of who is working on what to reduce duplication and support national coordination.
- Clinical trial access reforms—sustainable funding for trial delivery, including access to hospital resources such as beds, theatres and equipment. Researchers are currently negotiating with sites and sponsors to fund this important work.
- Positioning trials as core activities of the health system.
- Post-trial access and reimbursement reforms for long-term therapy beyond a clinical trial—Australians currently wait between two to four years longer to access therapies than comparable countries due to delays in funding decisions. ARDRN members are concerned that this is likely to worsen as more advanced therapies become available.
The next ARDRN meeting is scheduled for Thursday, 25 June 2026 between 12pm and 1pm AEST. If you are a rare disease researcher, affiliated with a university or research institution and would like to join the ARDRN, please complete the registration form below.
