Monday, 22 October 2018
Private Members' Business
That this House:
(1) notes that:
(a) 7 to 13 October 2018 is Bleeding Disorders Awareness Week;
(b) haemophilia, the most common bleeding disorder, affects more than 2,700 Australians, almost all of them boys and men;
(c) haemophilia is a genetic disease where a lack of a protein in the blood needed for clotting leads to excessive bleeding, often into joints and muscles;
(d) haemophilia impacts every aspect of day-to-day life—activities we all take for granted—as a result of hospitalisations, pain, accumulated disability and psychological impacts;
(e) treatment for bleeding disorders is delivered through specialised haemophilia treatment centres across the country and is jointly funded by the Commonwealth, state and territory governments through the National Blood Agreement; and
(f) the Haemophilia Foundation of Australia delivers vital support to Australians with bleeding disorders through advocacy, education and promotion of research;
(2) recognises that:
(a) while haemophilia treatment has improved significantly in recent years, there is still a significant need for improved treatments to deliver a better quality of life;
(b) reforms to the Therapeutic Goods Administration process to evaluate new medicines now allows for expedited priority review of new treatments for serious conditions such as haemophilia; and
(c) however, the road to fund new bleeding disorder treatments remains complex; and
(3) calls on the Government to continue working constructively with state and territory governments to ensure all Australians with bleeding disorders receive timely access to the treatments they need.
In moving this very important motion I speak in support of the more than 2,700 Australians, mostly boys and men, living with haemophilia. I acknowledge also the members of the Haemophilia Foundation Australia and thank them for not only raising awareness of this insidious disease during Bleeding Disorders Awareness Week but also the work they do delivering vital support to Australians with bleeding disorders through advocacy, education and promotional research. I also acknowledge my parliamentary colleagues, the member for La Trobe, for seconding this motion in a show of bipartisanship, and the member for Macarthur, who, as a doctor, has treated many sufferers of haemophilia, understands the importance of this motion and thanked me for raising it in the House.
Haemophilia is a rare and lifelong bleeding disorder that is complex to diagnose and difficult to manage. Australians who suffer from haemophilia lack a clotting factor and therefore suffer from bleeding due to injury, surgery or, sadly, for no apparent reason at all. It's the quality of life available for those Australians who live with haemophilia that concerns me, and that is what has prompted me to move this motion. Australians living with haemophilia face physical, financial, employment and psychological challenges throughout their lives.
Pain, patience and perseverance are the three words my Jacana constituent, David Cunningham, uses when asked to describe his life and living with haemophilia: pain because people are in pain all the time or, as he says, 'at least I am'—and that the pain of a major bleed is a nine out of 10 and can last for days on end—patience to get through the ongoing hospital visits during a lifetime of living with haemophilia; and perseverance to:
Keep going, don't give up, don't sit around and do nothing.
David is 63 years old and lives with haemophilia A. He was first diagnosed as a young boy. With no known family history of the bleeding disorder, there were limited people around David who understood the impact haemophilia was having on his life. Growing up in the sixties and seventies, David remembers that there wasn't a lot of support available to allow him to independently manage his condition. Even though he acknowledges that patients today have much greater flexibility and choice, he says there's still more to do and adds that, unless there is a cure, people like him with haemophilia still need support and relief from the disease.
David, like many others who have been living with haemophilia all their lives, has had a chance to reflect and recount what life is really like for sufferers. Even though his bleeds lessened as he got older, when he did bleed, he'd have to take a week at a time off work to recover. With sick leave used up, he resorted to unpaid leave. Then, when his condition worsened, work became untenable. David retired forcibly at 45, after 20 years in a profession he loved, including teaching the young minds at Broadmeadows West Technical School. As a result of haemophilia A and other health complications such as type 2 diabetes, David had to go on the disability support pension, and he's still on it today. This is a man who loved to play football and cricket but had to stop because of ongoing bleeding into his joints and muscles. An avid sports fan and a one-eyed Collingwood supporter, because of mobility issues he can't go to the games anymore and can only watch his beloved team on television.
He says haemophilia is an isolating disease, even more so for him. The bleeds to his knees and ankles have taken their toll, and now David has limited capacity and walks with crutches. He says:
I don't get out as much as I used to because it's hard for me to get out, sit down and then get up, so I just stay home.
David wishes that he was able to enjoy his retirement more but says living with haemophilia has made this impossible:
You get used to it—life goes on and you learn to accept it.
Haemophilia sufferers shouldn't have to get used to this quality of life that sees them retire in their prime, being unable to live an active and healthy life, suffer from financial distress and the list goes on for many of them. More needs to be done to find a cure to this lifelong disease so that we can give Australians who suffer from haemophilia the quality of life they deserve. More investment needs to go into research and development for this disease. I'd like to call on the government to work even more closely and constructively with the state and territory governments to ensure all Australians with bleeding disorders receive timely access to the treatment they need. This will ensure that people like David can finally get the care, treatment and quality of life that should be afforded to all Australians, which David says will be lifesaving for the young Australians who suffer in silence because of this disease.
I rise in support of this motion. Haemophilia and bleeding disorders affect far more Australians than people are aware. Everyone is familiar with the story of haemophilia affecting the royal families, and that is the nub of the problem: the genes are the cause of these unfortunate disorders. But there are other bleeding disorders besides haemophilia. There are various forms—one milder form called Christmas disease, another one called Von Willebrand disease, and there are other very rare blood-coagulation-factor disorders that cause problems. There are these pesky little things call platelets which run around and plug up the holes when you cut yourself, and there are disorders of platelets as well. But the coalition government—as governments before us have done and governments in the future will do—administers and delivers probably the best blood product and treatment system in the world for people afflicted with bleeding disorders. I don't know of a better system. We spend about $1.2 billion on the National Blood Agreement, where the federal government kicks in that amount of money and shares the costs with the states around all of the state based haemophilia and other blood-disorder treatment centres.
There is good news. The former speaker mentioned the desire for a cure. The good news is there is a cure on the way. There are cases of gene therapy where the gene for producing factor IX is introduced into the person whose genes don't make it, and they have been cured, basically just with gene therapy.
Like CAR T therapy for lymphomas and leukaemias, it's being researched around the world as we speak. All those doctors, scientists and biochemical wizzes are working on gene therapy for factor VIII haemophilia, which is the more commonly known and severe haemophilia. We also have clever biochemists and scientists who've developed recombinant factor VIII, factor IX and various other replacement therapies so that you don't have to rely on blood products that are pooled from multiple donations, which is what was the case when I was working in the haemophilia centre at Royal Prince Alfred Hospital and Westmead Children's Hospital.
There was one stage in the eighties, before HIV was understood and hepatitis C was understood when, unfortunately, whole cohorts of children with haemophilia got infected because, if your products were pooled from 100 donors, only one of them had to have hepatitis C or the at-that-time-unknown HIV for the person to be infected. It was a tragedy. But recombinant technology has saved that, and gene therapy is on the way.
Parents do have a lifelong struggle. Many of them adjust, but for the child or young adult it really does mean a different life. But the wonders of modern medicine are making huge gains in the quality of life. Instead of big haemorrhages and massive bruises—which you couldn't imagine if you had normal clotting—distorting and damaging joints, in the future, hopefully, those days will be gone. There are other treatments that they can give—sprays into the nose, under the skin or infusions—which will increase the amount of factor VIII.
So there is light at the end of the tunnel for many of these people who, through no fault of their own—it just runs in their family; it's recessive and then it pops up mainly in boys. There is really amazing technology, and Professor Rasko at RPA and other people overseas have been instrumental in this initial trial of gene therapy with factor IX. It's Australian scientists who are really at the cutting edge. The European body that provides certification for these therapies and the FDA in America are both fast-tracking their assessment of this. The Medical Services Advisory Committee in Australia, which is like the PBAC that approves drugs, is looking at these new therapies as we speak.
I thoroughly support this motion because it is a dreadful impost on unlucky people, but we have a great system in Australia.
I congratulate the member for Calwell for bringing this motion. It really is very apposite. Last week, 7 to 13 October, was Bleeding Disorders Awareness Week, and it's very important that the Australian community is able to access the best treatment for these complex and fascinating disorders. Bleeding disorders are more than just haemophilia. You may be aware that newborn babies in most developed countries now get vitamin K injections to prevent what was a very severe and sometimes fatal bleeding disorder of newborns called haemorrhagic disease of the newborn due to vitamin K deficiency. Australia led the world in introducing this many years ago.
Haemophilia is a disorder that most of us recognise as the most significant bleeding disorder, but it's not the most common. Haemophilia A, or factor VIII deficiency, is the most common severe bleeding disorder. Its counterpart, haemophilia B, or factor IX deficiency, is rarer and generally less severe but can be quite severe. But the commonest bleeding disorder was mentioned by the member for Lyne: von Willebrand disease. There are over 200,000 Australians who have von Willebrand disease—usually fairly mild and unlikely to cause a problem unless there's severe injury et cetera but occasionally quite severe.
These disorders are genetic. Haemophilia A and B are what are called X-linked. In other words, the mutation's carried on the X chromosome. Females have two X chromosomes, so they don't express the disorder; males, who have one X chromosome and one Y chromosome, will express the disorder if the genetic abnormality is on their X chromosome, inherited from their mother—although some can be mutations. The English royal family were afflicted by haemophilia B—not A, but B. The defective gene was carried by Queen Victoria, and she caused several of her descendants to have severe haemophilia B.
I've looked after many kids with bleeding disorders, particularly the haemophilias and Von Willebrand disease. The treatment used to be essentially that of replacement therapy, using factor VIII in the case of haemophilia A when bleeding occurred, derived from pooled blood products. As the member for Lyne has explained, many of these children developed HIV, hepatitis B and hepatitis C. Thankfully we now use recombinant clotting factors VIII and IX. This is bioengineered; it's not from human blood products and is much safer. We now have treatments, very effective treatments, for hepatitis C.
The central problem with haemophilia though is spontaneous bleeding, often presenting not in the newborn period, but when a child first starts to mobilise, starts to walk. The usual story is you see a child coming in covered in multiple bruises but who is otherwise well when he's started to walk. Kids, as we all know, when they start to walk, they have lots of falls and get lots of bruising. Sometimes it's a pretty traumatic time waiting to get the blood test results when you see a child like this. Is it leukaemia or is it a bleeding disorder such as haemophilia?
A diagnosis of haemophilia is quite a difficult one, because these children have spontaneous bleeding often to their joints, and this can lead to a lot of scarring inside the joint and joint deformity. Many of the older people with haemophilia in particular are left with quite significant long-term joint deformities. These days we're much better at treatment. We offer prophylactic treatment to try to prevent bruising and bleeding, to prevent ongoing joint damage. And now, just around the corner, is gene therapy. This is a fantastic new treatment. There's a new medication called emicizumab, which is a gene therapy given subcutaneously on a weekly basis that can really prevent long-term damage. So it's a wonderful new therapy, which is just starting to become available. It's a wonderful new development that I'm very pleased to see. I hope that we can provide this treatment for our children with haemophilia in the future, and we'll prevent long-term damage. (Time expired)