AHUS and Bianca Scott

24 June 2014

Senator O’SULLIVAN (Queensland) (20:30): I rise tonight to continue a discussion I started in this place last week, regarding a vulnerable group of Australians who suffer from the medical condition of atypical haemolytic uremic syndrome. This condition is a very aggressive medical condition that promotes organ failure in patients who are exposed—in particular renal failure followed by liver failure and in many cases the patients will go on to suffer brain damage. The condition is often measured in a patient’s life in weeks rather than months and certainly not years. For many this condition is ultimately life-threatening and for some fatal.

For those who do not surrender their life to this condition they often confront a lifetime of living with the results of organ failure—many of them not eligible for corresponding live transplant arrangements because of the condition. I said last time and I will continue to repeat each time I take to my feet that I stand here to speak for those who do not have a voice —for the grandparents, parents, brothers and sisters, uncles and aunties and the patients themselves, who have been engaging with me and other senators and members of the House of Representatives.

There are estimated to be 70 Australians who suffer from the condition, with 22 of them in acute stages of this disease. Not only are their voices silent, but there are very few of them to give momentum for those voices to be heard. Without people like myself and others in this parliament putting forward their case, the condition of their lives will remain in the balance. There is a solution and that has to do with a drug called Soliris. It is a very expensive drug; it costs about $. million a year per patient. The trials are yet to determine whether these patients need to remain on the drug for life or whether exposure to the drug over set periods of time is beneficial. Various time iterations have been presented but up to 12 months may tide them through, protect their organs and their lives until the condition goes into some form of remission. The drug was the subject of an application to the former government, but it was rejected on the basis of clinical trials. Another application was lodged in late 2013 and considered by the PBAC in March 2014. The PBAC have in principle approved the drug for use and have broadly agreed, as I understand it, with the cost impost of the drug, but there are some delays due to negotiations between the health department and the company about the terms and conditions of the managed entry scheme that will apply to Australian patients.

Broad trials for these boutique drugs for rare disorders cannot be conducted and so some trial and error is involved. This particular drug has only been used in the treatment of this condition for three and a quarter years internationally and so there is much more to be learnt both about the performance potential of the drug and any long-term negative impacts it might have on patients who are exposed over longer periods. The difficulty is that, whilst the Department of Health has agreed with man of the terms and conditions of the manufacturer from the United States, there is disagreement over the government’s resolve to ensure that this drug is accepted on terms that meet the national interest and the requirements of the patients. I think some of the conditions that the department is negotiating are very fair and reasonable in the circumstances, but they are asking the company to enter into arrangements where if the drug proves to be unsuccessful with particular patients —and I understand patients react differently to the performance of this medicine—then Australia reserves the right to withdraw from the arrangements. I think that is perfectly reasonable.

I am satisfied that great progress has been made in these discussions. I think they are at a very advanced stage. The difficulty now rests in convincing the pharmaceutical company to give access to some of these patients—and there are 11 Australians who are in critical, immediate need of this drug to maintain their health. If negotiations with the PBS are to go on, as I anticipate they might, and are measured in months, then indeed we will have young Australians—beautiful young Australians like Bianca Scott of the Gold Coast in my home state of Queensland—who will suffer from organ failure, from which they might not return. It may well—and I hope I do not bring Bianca or her family any distress in saying this—put her life at risk. In fact, it will put her life at risk.

That family has spent $235,000 with this company to buy the drug, at a personal level, to this date. They have run out of the capacity to do that. I stand here tonight, as I did the other day, and I call upon this pharmaceutical company. This company is engaging with our country, which is giving favourable consideration to entering into arrangements with that company for tens upon tens upon tens of millions of dollars of Australian taxpayers’ money to subsidise and fund this important drug. This is a company that has a turnover of $1.5 billion. This is a company where the CEO has an annual salary of $14 million and $168 million in share options. I call on them tonight to give consideration to extending access to this drug to these 11 Australians who are in critical need of it at the moment, until such time as we have properly and sensibly worked through the issues associated with the terms and conditions for access to this drug.

I say to the CEO of Alexion tonight—who is, as we speak, I suspect, lying warm and fair in his bed in the United States: tomorrow morning he should ask himself a question; he should close his eyes and give consideration to what he would want his company to do in these circumstances if this were his daughter who faced this terrible plight in life. I am prepared, if he does that in a fair manner, to live with the decision that he takes. (Time expired)