Funding therapies for rare diseases: an ethical dilemma with a potential solutionColman Taylor A B C D , Stephen Jan A B and Kelly Thompson A B
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: email@example.com
Australian Health Review - https://doi.org/10.1071/AH16194
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.
References Global Genes. Rare list. 2015. Available at: https://globalgenes.org/rarelist/ [verified February 2016].
 Norman R, Hall J, Street D, Viney R. Efficiency and equity: a stated preference approach. Health Econ 2013; 22 568–81.
| Efficiency and equity: a stated preference approach.CrossRef |
 Department of Health. Public summary documents by product. 2016. Available at: http://www.health.gov.au/internet/main/publishing.nsf/Content/lsdp-criteria [verified February 2016].
 Department of Health. Life saving drugs program criteria and conditions. Available at: http://www.health.gov.au/internet/main/publishingnsf/Content/lsdp-criteria [verified 11 January 2017].
 Newton S, Ellery B, Fischer S, Farah C, Gum D, Liufu Z, Milverton J, Parsons J, Pridam L, Schubert C, Tamblyn D, Merlin T, Boltzmann L. Life Saving Drugs Programme review: technical assessment. April 2015. Available at: http://www.pbs.gov.au/reviews/lsdp-technical-assessment-report/lsdp-review-technical-assessment-april-2015.pdf [verified 11 January 2017].
 National Institute for Health and Care Excellence (NICE). NICE highly specialised technologies guidance. 2016. Available at: https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-highly-specialised-technologies-guidance [verified 11 January 2017].
 Scottish Medicines Consortium. PACE. Medicines for end of life and very rare conditions. Available at: https://www.scottishmedicines.org.uk/Submission_Process/Submission_guidance_and_forms/PACE [verified 11 Janaury 2017].
 Winquist E, Bell C, Clarke J, Evans G, Martin J, Sabharwal M, Gadhok A, Stevenson H, Coyle D. An evaluation framework for funding drugs for rare diseases. Value in Health 2013; 15 982–86.
| An evaluation framework for funding drugs for rare diseases.CrossRef |