Solving the rare disease funding dilemma

Funding therapies for rare diseases: an ethical dilemma with a potential solution.

Funding rare disease therapies presents a challenge in Australia, where there is a legislative requirement to consider cost-effectiveness.

Future access

The Life Saving Drugs Programme (LSDP) subsidises access to high-cost therapies for rare, life-threatening conditions. However, the LSDP is currently under review, and future access to rare disease therapies is uncertain. Australians value equal opportunity in relation to health care, especially for those with life-threatening conditions.

There is a clear need to maintain a separate fit-for-purpose framework that draws on overseas best practices to evaluate and fund rare disease therapies, providing certainty for the industry to continue to invest in treatments, and ensuring funding recommendations are reflective of Australian values balanced against financial sustainability.

Funding processes

This paper briefly examines current evaluation and funding processes in Australia, as well several international critical analysis frameworks and assessment processes for rare disease therapies. It maps conditions for a separate fit-for-purpose evaluation and framework, and provides insight into LSPD funding criteria.

​Most critical, it offers a possible solution to the ethical quandary decision makers face when considering rare disease therapies: How does one balance equity considerations and opportunity cost.

How does one balance equity considerations and opportunity cost.

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