In the last 4 years only two new therapies have been approved under Australia’s current program for rare disease therapies. Aside from these products, no new treatments for rare disease have successfully navigated the entire process for funding rare disease therapies since the reforms in 2010. Australians are generally waiting from 2 to 4 years longer for access to rare disease therapies available in comparable countries like the United Kingdom, Canada, Germany and the Netherlands. Some medications remain unavailable 8 years after becoming available overseas.

The Therapeutic Goods Act includes a limit of 2,000 patients for the registration of orphan drugs – the equivalent of approximately 1 patient in 10,000 persons. This definition captures fewer rare diseases than in comparable countries. In the United States the definition is 1 in 1,500, in Canada and the European Union it is 1 in 2,000 and in South Korea it is 1 in 2,500.

This report explains how we can reform our system to bring better health care to more Australians suffering from rare diseases.