Australian Health Review article prompts Parliamentary questions on funding treatments for rare diseases

Wednesday, March 8, 2017

An article published in the AHHA’s peer-reviewed academic journal, Australian Health Review (AHR), has prompted Senators in the federal Parliament to question how decisions are made in Australia on subsidising treatments for rare diseases.

The line of questioning was directly prompted by the AHR article Funding therapies for rare diseases: an ethical dilemma with a potential solution, by Colman Taylor, Stephen Jan and Kelly Thompson.

AHHA Chief Executive Alison Verhoeven said it was ‘very pleasing to the Association and its members that a well-considered and thoughtful article in the AHHA’s peer reviewed journal could have a direct influence on policy-making in this way’.

The issue of funding rare disease therapies has been brought to light through the plight of a 6 year old boy who has the rare condition Morquio A.

Morquio A affects just 21 Australians, impacting the development of their bones, respiratory system, eyesight, hearing, teeth, liver and other body parts. People with Morquio A live an average of 25 years.

‘The only available treatment is the drug Vimizim’, Ms Verhoeven said.

Vimizim has not, to date, been subsidised under the government’s Pharmaceutical Benefits Scheme as it does not meet cost-effectiveness criteria.

‘It has not been funded under the Life Saving Drugs Program because, like many such drugs, there are difficulties meeting a criterion specifying acceptable evidence that a patient’s lifespan will be “substantially extended”.’

At Senate Estimates hearings last week, Senators asked questions of the Department of Health about subsidising Vimizim. The Department said it was considering the drug for listing on the Life Saving Drugs Program.

The AHR article advocates that the federal government urgently re-assesses how it funds treatments for ultra-rare diseases to reflect ethical and community values as well as commercial considerations:

‘We believe there is a need to maintain a separate fit-for-purpose framework to evaluate and fund rare disease therapies drawing on overseas best practice that incorporates community values in decision making’ the authors said.

‘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.’

The Australian Healthcare and Hospitals Association is the national peak body for public and not-for-profit hospitals, Primary Health Networks, community and primary healthcare services, and advocates for universal, high quality and affordable healthcare to benefit the whole community.

A copy of the Australian Health Review article is below. For more information on AHHA, visit


Media enquiries: Ms Alison Verhoeven, Chief Executive, Australian Healthcare and Hospitals Association, 0403 282 501