Australians living with ultra-rare blood disease facing uncertain future
Desperate Australians living with Paroxysmal Nocturnal Haemoglobinuria (PNH) – an ultra-rare,
life-threatening condition that causes red blood cells to explode – have been denied medical funding in this year’s Federal Budget.
Soliris® (eculizumab) is the only treatment for PNH that reduces the rate of blood cell destruction and
has been proven to work immediately and sustainably in almost 100 per cent of patients.
The treatment is available in more than 30 countries worldwide.
According to Professor Jeffrey Szer, Director of the Department of Clinical Haematology & Bone Marrow
Transplant Service, Royal Melbourne Hospital, the Government’s failure to provide funding for PNH
treatment will undoubtedly cost lives.
“One-in-three people living with PNH will die within five years of diagnosis, if they do not have access to
effective treatment.
“For others, PNH spells the onset of many, varied health complications including fatal blood clots;
chronic kidney disease; anaemia; heart problems; stroke and other damage to the brain, liver or
stomach; disabling fatigue and poor physical function,” said Prof Szer.
“If PNH struck Australians at the same rate diabetes or heart disease do, the Government would not
hesitate to open its wallet and fund the necessary medical care. I see no reason for people living with a
rare disease to be ignored, simply because there are so few of them.”
Newly-wed, Jenny Sturrock, 28, was diagnosed with PNH in January 2010 after a sudden collapse and
subsequent emergency medical treatment. Since diagnosis, Jenny’s PNH has progressed rapidly; she
now experiences severe chest pains, frequent vomiting, sleepless nights and must undergo regular
blood transfusions.
Jenny says that PNH has filled her life with fear and doubt. She can no longer work full-time and can’t
start planning the family she and her new husband had wanted until she has access to more effective
treatment, which she relies on the Government to help fund.
“I’m angry and disheartened by the Government’s decision not to fund this treatment because the
entire PNH community had been led to believe that the funding would be provided.
“I’ve never broken a law, I’m a hard worker, I’m honest and caring and the one time that I have relied on
the Government to help me out, I’ve been put on the backburner,” Jenny said.
“I’m fearful, not just for myself – with perhaps another 10 years to live – but for other people with PNH
who may only have months to live, without treatment.”
President of the PNH Support Association of Australia (PNHSAA), Ms Linda Charlton said the fact that
some of Australia’s most vulnerable people have been stripped of the hope provided by the
Government in the lead up to the Federal Budget is nothing short of a national disgrace.
“The small group of Australians living with PNH has been left with a proven and effective treatment
completely out of its grasp. This adds a tremendous amount of stress and anxiety – not only to those
already living with ongoing pain, poor quality of life and significantly reduced life expectancy – but to
their partners and families as well.
“How can the Government put a monetary value on someone’s life? Surely a life inflicted by PNH is
worth no less than one inflicted by a more common medical condition,” Ms Charlton said.
About PNH
PNH is an ultra-rare, progressive and life-threatening disease, believed to affect just 70 Australians. PNH
is characterised by haemolysis, or the destruction of red blood cells, which can cause a variety of major
health problems such as the formation of blood clots, high blood pressure and damage or failure of
organs such as the brain, liver, gastro-intestinal system and kidneys. Varied symptoms, including
abdominal pain, difficulty swallowing, poor physical function, shortness of breath, erectile dysfunction
and debilitating fatigue, can also interfere with the quality of life of people living with PNH.
PNH develops without warning and can strike men and women of all races, backgrounds and ages. On
average, the disease affects people in their mid-30s, however, 10 per cent of all people living with PNH
start developing symptoms before they’re 21.
PNH often goes unrecognised, with diagnosis sometimes taking more than 10 years. It is estimated that
approximately one-third of patients with PNH do not survive more than five years from the time of
diagnosis.
About Soliris
Soliris is the only treatment for PNH that reduces the destruction of red blood cells caused by a person’s
own immune system.
Soliris stops the immune system from attacking itself by selectively blocking the proteins responsible for
the attack, thereby reducing the symptoms associated with the disease and allowing the patient to
return to their normal life. In 2008, Soliris was awarded the most prestigious award possible for
biological medicines, the Prix Gallien USA Award, by a panel of seven Nobel Prize Laureates.
Studies show that Soliris immediately and sustainably reduces the destruction of red blood cells in
almost 100 per cent of patients living with PNH. This reduction has been clinically proven to reduce the
rate of blood clot formation and the need for blood transfusions, as well as improve energy levels and
overall quality of life.
The cost of Soliris to the Government is equivalent to all other medications currently funded under the
Commonwealth Government’s Life Saving Drugs Program. PNH sufferers in the US have had access to
Soliris since March 2007 and treatment is now funded in more than 30 countries worldwide, including
Europe, Eastern Europe, Canada and Brazil.