Australian Health Review Australian Health Review Society
Journal of the Australian Healthcare & Hospitals Association

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

Colman Taylor A B C D , Stephen Jan A B and Kelly Thompson A B

A The George Institute for Global Health, PO Box M201, Missenden Road, Camperdown, NSW 2050, Australia. Email:;

B Sydney Medical School, The University of Sydney, Edward Ford Building A27, The University of Sydney, NSW 2006, Australia.

C Optum, Level 1, 370 Norton Street, Lilyfield, NSW 2040, Australia.

D Corresponding author. Email:

Australian Health Review -
Submitted: 2 September 2016  Accepted: 9 December 2016   Published online: 16 February 2017


Funding rare disease therapies presents a challenge in Australia where there is a legislative requirement to consider cost-effectiveness. Currently the Life Saving Drugs Programme (LSDP) provides subsidised access to high-cost therapies for rare, life-threatening conditions. However the LSDP is currently under review by the Minsiter for Health and future access to rare disease therapies in uncertain. Internationally there is no gold standard model to evaluate and fund rare disease therapies, and considerable variation exists. However, common features of international systems include the opportunity for early stakeholder engagement, flexibility with evidence requirements, cost-effectiveness criteria and transparency in relation to the decision making framework and outcomes. Australians value equality and equal opportunity in relation to health care. To meet these expectations there is a clear need to maintain a separate fit-for-purpose framework to evaluate and fund rare disease therapies drawing on overseas best practice. This will provide certainty for industry to continue to invest in such treatments, as well as ensuring funding recommendations are reflective of Australian values balanced against the need for financial sustainability.


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