Julie Owens (Parramatta, Australian Labor Party, Shadow Parliamentary Secretary for Small Business) Share this | Hansard source

I move:

That this House:

(1) notes:

  (a) the devastating effects of atypical Haemolytic Uraemic Syndrome (HUS) and its long term impact on the lives of the sufferer, as well as their friends and family;

  (b) that atypical HUS is a genetic disease of excessive immune dysfunction that affects people of all ages, with symptoms including heart failure, pulmonary edema, clotting in the lungs, blurred vision and kidney failure; and

  (c) that the current treatment regimen of plasma exchange and/or dialysis has significant risks and can result in a further reduction in the quality of life for the patient;

(2) acknowledges the community advocacy work that has brought this condition to the attention of the House, such as the work by Ms Jeanette Daher who seeks a listing of the drug under the Government’s Life Saving Drugs Program;

(3) notes that the Pharmaceutical Benefits Advisory Committee (PBAC) is due to make a recommendation on the use of the drug Soliris (Eculizumab), which has shown to put the disease into remission; and

(4)urges the Minister for Health to carefully consider any favourable recommendation of the PBAC as a matter of urgency.

A few months ago a wonderful woman named Jeanette Daher came to see me in my office. Her sister, Marie Taouk, had a very rare disease—in fact it is known as an ultra-rare disease—known as atypical haemolytic uraemic syndrome. This disease affects two people in one million. In Australia, we probably have 60 to 70 sufferers. In fact, out of the 150 members in the House, no more than half of us would have one person in our electorate who suffers from this disease. But, rare as it is, it is also a particularly ugly disease. It is caused by genetic deficiency in one or more complementary regulatory genes and it can lead to a condition known as thrombotic microangiopathy, TMA, which is the formation of very small blood clots in the smaller blood vessels throughout the body. It can happen at any time and there can be a sudden onset.

When a person suffers from TMA, the effect on the body is quite devastating. It will lead to life-threatening damage to vital organs, including the kidneys, the heart and the brain. Nearly two-thirds of patients with atypical haemolytic uraemic syndrome will die or require kidney dialysis or have permanent renal damage or life-threatening damage to their heart or brain within one year of diagnosis. So it is a devastating disease.

Jeanette came to see me because there is a drug called Soliris, which is available in 40 other countries and can have a dramatic impact on a person with this particular disease. She was lobbying for this drug to be included on the PBS. Of course, PBS decisions are made at arm's length from government, but I thought it was worth putting a motion before the House that acknowledged the work of Jeanette and many others around the country who have been lobbying for this drug and asking the parliament to consider it should the Pharmaceutical Benefits Advisory Committee recommend it. Fortunately, since this motion was put on the Notice Paper, the PBAC have considered listing Soliris for the treatment of this disease. In their March 2014 meeting they considered that the medicine could be cost-effective if the sponsor rebated part of or all of the price of the drug. They have, essentially, recommended that a working party be put together to work out the conditions of a prescription. This is truly wonderful.

The stakeholder meeting was held on 24 June. It included representatives from the Australian and New Zealand Paediatric Nephrology Association, the Haematology Society of Australia and New Zealand, Kidney Health Australia, the AHUS Patient Support Group Australia, Rare Voices Australia, Alexion and the PBAC. There was very, very constructive discussion that worked towards finalising the prescribing criteria for listing the product on the PBS, in recognition of the high clinical need for an effective treatment for patients.

The results of tests have also been incredibly good. In the tests, which the PBAC reviewed, there were, for example, 40 patients who were on dialysis that had recently been commenced—that is, within four months of the trial entry. In the results, it was noted that 33 of these 40 patients, 82.5 per cent, were able to cease the use of short-term dialysis during the treatment with Soliris and they remained dialysis free through two years of treatment. Again, it is an incredibly effective drug which will change the lives of people with atypical haemolytic uraemic syndrome profoundly and, of course, the lives of their families.

I, along with Jeanette and the many others who have been working so hard to have this drug listed, urge the Minister for Health, should the PBAC make the recommendation, to consider the recommendation very quickly and, hopefully, make this drug available to the 60 or 70 people in Australia who suffer from this appalling disease.

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