Father fights for access to life-saving drug

A YENDON father-of-two faces a $20,000-a-month bill if the federal government does not fund a drug to fight a rare life-threatening illness.Mark Filmer, 36, was diagnosed nine years ago with Paroxysmal Nocturnal Hemoglobinuria (PNH), a debilitating blood condition.For the last five years he has been participating in a trial by a US pharmaceutical company of the drug Soliris, the only treatment for PNH.In the recent budget, the Australian government refused to fund the drug under its Life Saving Drugs Program and last week Alexion, the pharmaceutical company conducting the trials, said it was withdrawing its supply of Soliris to all participating Australian patients in May next year. The drug had been supplied to Australian patients under Alexion's compassionate use program.Now Mr Filmer and his wife, Jasmine, are pleading for people to sign an online petition to lobby the federal government for funding to help Mark and the 69 other Australians suffering PNH. JOIN THE PETITION HERE "The federal government has stepped away from its commitment to fund the drug for us,'' said Mr Filmer."This federal government's decision has effectively made it impossible for those waiting treatment to access it and has prompted the manufacturer to announce a withdrawal of their compassionate use program to those currently receiving the drug."This decision will end the lives of a number of PNH sufferers. It will condemn the rest of us to a short life of pain, blood transfusions and welfare dependence. We will be relying on our loved ones to exist as carers and our children to grow up without the input of one parent.''Ballarat MP Catherine King pushed the issue with Health Minister Nicola Roxon, who will now make an urgent request for further advice from the Pharmaceuticals Benefits Advisory Committee on the potential benefits of adding Soliris to the government's Life Saving Drugs Program.The only other options for Mr Filmer, father of Max, 6, and Tess, 2, is either regular blood transfusions or a bone marrow transplant, from which he has only a 60 per cent chance of surviving due to his low health."The (drug) trials have been a success and the condition has been managed. Without this drug my quality of life will be greatly impaired and I will have a limited life expectancy,'' the part-time youth justice worker said."All I want to do is kick the footy and run around with the kids. I want to be allowed to live a normal life.''Of the 70 PNH sufferers in Australia, 23 are currently on the US drug trial. Mr Filmer believes he is the only PNH sufferer in Ballarat.Mr Filmer was a fit, healthy man in 2000. Not long after, he began suffering from flu-like symptoms and was unable to get off the couch.After enduring debilitating symptoms for 12 months, he was iagnosed with PNH."Unfortunately my condition saw my health deteriorate rapidly, to the point where before beginning the first drug trial in 2005, I was dependant on blood transfusions to give me the energy to live ... I felt like I was living from transfusion to transfusion. At times, walking from one end of the house to the other left me exhausted and breathless,'' he said.One-in-three people living with PNH will die within five years of diagnosis. PNH involves the destruction of red blood cells, which occurs when the cells lack a protein that protects them from attack by the immune system. Since starting the trials of Soliris, Mr Filmer's blood count is normal and he no longer experiences most PNH symptoms. The drug stops the immune system from attacking red blood cells by selectively blocking the proteins that cause the attack and reducing the symptoms associated with the disease. "Soliris allows people like me to return to a normal life,'' Mr Filmer said."As the cost of developing this drug is very expensive and given the extremely small proportion of people with PNH, the high cost of this drug when it comes on the market prohibits patients like me from being able to afford it.'' Ms King yesterday said she appreciated both the challenging nature of Mr Filmer's condition and circumstances and the fact the Soliris was one of the world's most expensive drugs."I met with Mr Filmer in April last year and subsequently made a follow-up submission to Health Minister Nicola Roxon on the issue,'' Ms King said."I'm pleased to report that Minister Roxon has made an urgent request for further advice on the potential benefits of adding Soliris to the government's Life Saving Drugs Program."There are apparently a number of uncertainties in the evidence about the drug. The government certainly understands that this is an important matter and have asked the committee to respond as quickly as possible."www .gopetition.com/online/36580.html