The McKell Institute’s report seven years ago, Funding rare disease therapies in Australia – placed the issue of inequities in access to new medicines for the one in eight Australians that live with a rare disease on the national policy agenda. It has helped shape the debate in Australia, culminating in the release of the first ever National Strategic Action Plan for Rare Diseases in 2020 which was a key recommendation of the 2014 report.

Australians are proud of our universal health system, that strives to provide everyone access to the health care they need. Prioritising groups that are disadvantaged or worse-off in the allocation of resources first is the way to move toward a truly universal health system.

Since the 2014 report there have also been reforms to the Life Saving Drugs Program (LSDP) but have not closed the access gap when looking at international comparator countries.

The current review of the National Medicines Policy (NMP) presents an opportunity to embed equity at the heart of the policies and programs that provide access to new therapies in Australia. This is particularly important as new therapies, including cell and gene therapies, become available that will transform the clinical and policy landscape for rare disease therapies.

In this report, the authors provide an update on major developments in the policy landscape over the past seven years, drawing on a number of reviews, white papers and strategies that have informed government policy. They find that there are still significant gaps that need addressing, and that the NMP needs to be reformed to ensure the policies and programs that provide access to new medicines adequately address the needs of people with rare diseases.