Monday, 14 July 2014
Private Members' Business
Atypical Haemolytic Uraemic Syndrome
That this House:
(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.
I second the motion. I add my voice to the statement made by the member for Parramatta. On 28 April 2014, the outcomes of the March 2014 PBAC meeting were made public. At this meeting, the PBAC recommended the drug Soliris for listing on the PBS for the treatment of atypical haemolytic uraemic syndrome, which I will call aHUS. Despite the extremely high price requested, the PBAC decided that the medicine could be most effective if the sponsor agreed to participate in a managed entry scheme. This scheme would require the pharmaceutical company to rebate part of the entire price of the drug depending on how well the patient responded to the treatment. As a result of this important decision, all sufferers of aHUS will be eligible to receive subsidised Soliris on the Pharmaceutical Benefits Scheme.
As the only available treatment that is clinically proven to prevent premature death and vital organ damage for people with aHUS, subsidised Soliris could be life-saving treatment for one of my constituents—like the constituent of the member for Parramatta. This constituent of Reid is a mother of two teenage children who is currently on dialysis and requires a renal transplant as a part of her treatment. For a person suffering from end-stage renal failure, dialysis will only keep them alive temporarily. Undergoing dialysis also entails a substantial reduction in the quality of life, and its outcome is far inferior to that of transplantation.
Professor Steve Chadban is the senior staff nephrologist and transplant physician at the Royal Prince Alfred Hospital. According to the professor, transplantation for patients—
A division having been called in the House of Representatives—
Proceedings suspended from 12:23 to 12:40
Renal transplantation with Soliris offers a high probability of success but ongoing treatment is extremely expensive. The government is currently in negotiations with the manufacturer and sponsor of Soliris, Alexion Pharmaceuticals Australasia Pty Ltd, to reach an equitable price for the drug. Once an agreement has been made, the PBAC will be provided with a price proposal and will conduct the finalisation of the conditions for the listing on the PBS.
Alexion previously estimated a total cost to the PBS of between $100 million and $200 million over the first five years of listing. If a drug costs more than $20 million in any of the first four years of listing, it must be put before cabinet for consideration. This is of course the likely outcome for Soliris. While the cost of Soliris on a per patient case is high, the condition itself is extremely rare, with only two in one million Australians battling this life-threatening blood disease—that is, approximately 44 people across the entire country. Whilst this may at first appear to be a high cost for the benefit of so few people, I submit to the House the following comments made by my constituent's GP prior to the PBAC's positive recommendation:
This is a rare condition affecting people of any age. [My patient] is a mother with teenage children, a wife, a daughter of elderly parents and a potential employee. One does not have to be a health economist to do the calculations [to] realise that the cost to society will be outweighed by the benefits of her health …
Untreated, this syndrome has far further implications than just the physical health of the patient. The lady I have been speaking to in my electorate has described to me in detail the adverse impact her diagnosis with aHUS and the following treatment phase have had on not just her children and husband but also her parents and wider social group. The restrictions of the existing treatment methods have also had an effect on her emotional and mental health. The availability and affordability of Soliris for those suffering aHUS obviously have wider benefits for not just patients but their family and the community.
I congratulate the PBAC for its recommendation to list the treatment on the PBS and for moving us closer to the goal of wider accessibility to Soliris. I also take this opportunity to thank the Minister for Health for all his assistance in this matter. I look forward to seeing this matter finalised and having this life-saving drug more accessible to all Australians who are afflicted with this most horrible condition.
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.