Eculizumab (trade named, Soliris®) is the only treatment for PNH that effectively slows the rate of haemolysis and thereby significantly reduces the development of life-threatening complications such as blood clots (strokes, heart attacks) and kidney failure.
Eculizumab is administered via an intravenous infusion once weekly for the first four weeks of treatment, and fortnightly thereafter.
The Therapeutic Goods Administration approved Eculizumab for use in Australia on 17 February 2009. However, its costly price tag – approximately AU$480,000 per patient, per year – renders the treatment cost prohibitive for the patients who rely on it to ease their symptoms, improve their quality of life and increase their life expectancy.
In Australia, the Federal Government’s Life Saving Drugs Program (LSDP) may fund high cost drugs for very rare genetic diseases, known as “orphan” drugs.
In March 2009, the Pharmaceutical Benefits Advisory Committee (PBAC) considered an application submitted by the manufacturer of Soliris ®, Alexion Pharmaceuticals Inc, for Eculizumab to be listed on the Pharmaceutical Benefits Scheme (PBS) or alternatively, on the LSDP. The PBAC rejected Alexion’s application to list the treatment on the PBS, but recommended that it be referred to the LSDP. Full details of the PBAC’s decision can be found here.
The PNHSAA has been campaigning the Government since June 2009 for Australian PNH patients to gain access to Eculizumab. Despite assurances in September 2009 from the Health Minster, The Hon. Nicola Roxon MP that the matter would be progressed in a timely manner, patients are still awaiting treatment.
The criteria under which treatments are funded via the LSDP was changed on 10 May 2010.
To date, Eculizumab is yet to be funded on the LSDP, having been overlooked in the 2010-2011 Federal Budget announced on 11 May 2010. On 20 May 2010, the Health Minister requested the PBAC undertake a further review of the treatment against new LSDP criteria. That review is currently underway.
Approximately 23 Australian PNH patients are receiving treatment free of charge directly from the manufacturer, as part of programmes extending from the global clinical trials of Eculizumab commenced in 2005. That supply will stop in May 2011.
How You Can Help
The procedure for funding through the LSDP is complex and time consuming and is ultimately decided by the Federal Government. The PNHSAA has lent its support to the cause, requesting assistance from all Senators and MPs around Australia to urge the Government to urgently fund Eculizumab. We believe it is imperative that Australian patients be given access to this life-saving treatment as soon as possible.
Please write to your local Federal MP requesting they write to the Health Minister seeking urgent action to fund Soliris through the Life Saving Drugs Program to give Australian PNH patients access to this life-saving treatment. Check the AEC’s website if you’re unsure of your local Member: www.aec.gov.au
Sign our petition and ask your friends and family to do the same here.