Jill Hall (Shortland, Australian Labor Party) Share this | Hansard source

Firstly I would like to congratulate the member for Parramatta for bringing to the parliament this important motion on atypical haemolytic uraemic syndrome. It is a motion that shows that this parliament is interested in providing treatments, medications and research in the area of rare diseases. aHUS is a rare disease. Only 60 to 70 Australians are living with this disease. It is a genetic disease that causes abnormal blood clots to form in small blood vessels. It can severely damage vital organs, such as the kidneys, the heart and the brain.

There are three major problems: there is progressive renal failure, problems associated with red blood cells and platelet counts and problems that occur in the vascular system. It is life threatening. It is an ultrarare disease. It is caused by a genetic deficiency in one or more of the complementary regulatory genes. It is definitely a life-threatening disease and people living with it have their life very much modified. aHUS is, as I said, a rare, life-threatening, progressive disease that frequently causes very, very severe impacts on those people that are living with it. It is in most cases caused by the chronic, uncontrolled activation of a branch of the body's immune system that destroys and removes foreign particles.

The PBAC met recently and considered the listing of Soliris. It is a medication that can be used and used very successfully to treat aHUS. When it met it made the recommendation that this drug should be listed. The public summary documents outlined in detail the PBAC's recommendations for Soliris. That happened at its March 2014 meeting. The Department of Health is currently working with the medicine's sponsors, Alexion Pharmaceuticals Australasia Pty Ltd, to progress the PBAC recommendation. A stakeholder meeting was held on 24 June 2014. It included representatives from a number of organisations, including the Australian and New Zealand Paediatric Nephrology Association, the Haematology Society of Australia & New Zealand, Kidney Health Australia, aHUS Patient Support Group, rare voices Australia. I just need to emphasise again the wonderful work that rare voices does. It is not only aHUS but it is also any number of rare diseases where there is only a small number of patients that are actually living with that disease. They get in there and they advocate very strongly for those people living with the disease. It is through their work in a number of areas that there has been some changes and support offered to people who are living with a rare disease.

The next step is for cabinet to consider the listing of this medication. As the member for Parramatta emphasised, a study showed that of the 40 patients on dialysis, 33 no longer received dialysis once they started using the medication Soliris. To my way of thinking that is a cost saving to government. If people no longer needed to undergo constant dialysis and that can be replaced with the medication Soliris, it is a good step—it is a step in the right direction. It not only improves the quality of life of those people that are living with aHUS but it is also a saving to government. I recommend the motion to the House.

Debate adjourned.

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