The plight of 100 Australians living with Paroxysmal Nocturnal Haemoglobinuria (PNH) – an ultra-rare and life-threatening blood disease – was finally acknowledged by PM Julia Gillard and Health Minister, The Hon. Nicola Roxon MP after representatives of the PNH community attended Question Time in Parliament yesterday(Wednesday, November 17, 2010).
The President and Secretary of the PNH Support Association of Australia (PNHSAA), Ms Linda Charlton and Mrs Jenny Sturrock, were in Canberra this week seeking support for Government-subsidised PNH treatment from Australia’s MPs. Funding for Soliris® – the only effective treatment for PNH – was first recommended by the Pharmaceutical Benefits Advisory Committee (PBAC) in March 2009. However, the Federal Government is yet to approve the subsidy despite a second positive recommendation from the PBAC and 18 months of Government lobbying by the PNHSAA.
Ms Charlton and Ms Sturrock were at last given the opportunity to personally meet separately with Gillard and Roxon yesterday to highlight the importance of affordable access to effective PNH treatment through the Government’s Life Saving Drugs Program(LSDP).
According to Ms Charlton, the subsidy is close, but still out of reach.
“Access to effective PNH treatment has been a long and arduous battle, and in the meantime, Australian lives have hung in the balance.
“The Prime Minister Julia Gillard and the Federal Health Minister, Nicola Roxon, have personally reassured me that the Government is acting with every urgency to grant the PNH community access to Soliris®,” said Ms Charlton.
“I sincerely hope that the only remaining step of Cabinet Approval is expedited so that the PNH community can receive treatment well before Christmas and look forward to a stable future.
“We plan to continue to work every day until this treatment is funded on the LSDP,” Ms Charlton said.
“The PNHSAA is grateful to all Senators and MPs from various parties who have taken the time to meet with us, and support our campaign.”
Soliris is the only effective treatment for the blood disease which is characterised by abnormal and uncontrolled haemolysis(destruction of red blood cells). The treatment acts to suppress the crippling symptoms and complications associated with PNH, and significantly reduces the risk of premature death.
About PNH
PNH is an ultra-rare and life-threatening disease, affecting just 100 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. Symptoms, including abdominal pain, difficulty swallowing, poor physical function, shortness of breath, erectile dysfunction and crippling fatigue, can 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 untreated PNH will die within five years of diagnosis.
About Soliris
Soliris is the only treatment for PNH that reduces the auto-immune destruction of red blood cells.
Soliris stops the immune system 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.
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 funded in more than 30 countries worldwide, including Europe, Eastern Europe, Canada and Brazil, and now Australia.
In 2008, Soliris was awarded the most prestigious global award for biological medicines, the Prix Gallien USA Award, by a panel of seven Nobel Prize Laureates.
