Rare Conversations 

In late July, we attended the Rare Conversations event, hosted by Patient Voice Initiative and Alexion Pharmaceuticals.

The event brought together individuals from patient organisations, clinical practice, as well as industry and policy backgrounds, to explore how Australia can improve access to new medicines for people living with a rare disease.

This gathering showcased lived experience voices and panelist conversations as well as the launch of IQVIA's new white paper, All Too Rare: The PBS Access Gap for the Two Million Australians with a Rare Disease. 

Within the report, Australia was ranked second to last for funding rare disease medicines against 14 comparable countries.

Discussions centred around Health Technology Assessment (HTA) processes and the need for Pharmaceutical Benefits Scheme (PBS) reform.

Panelists included:

• Dr Gordon Reid Federal Member for Robertson Member of the House of Representatives health committee and emergency doctor., 
• Ann Single CEO, Patient Voice & President of HTAi, 
• Timothy Fulton Inaugural Ambassador, Rare Voices Australia, Founder, Running Rare, 
• Dr Andrew Wilson Co-Director, Leeder Centre for Health Policy, Economics and Data Professor, Public Health in School of Public Health, University of Sydney Former Chair, Pharmaceutical Benefits Advisory Committee (PBAC) 
• Dr Danny Hsu Director, Immune Haematology, Obstetric Haematology, Haematology Clinical Trials Unit in South West Sydney Local Health District
• President, Thrombosis Haemostasis Society of Australia and New Zealand (THANZ).

It was an important day to meet with like-minded individuals and organisations seeking greater access and equity and treatment pathways for rare diseases. We look forward to being part of ongoing conversations.