Mount Forest native Lucas Maciesza was one of about 25 PNH patients who were joined by supporters in a demonstration at Queen’s Park on Monday.
The Canadian Association of Paroxysmal Nocturnal Haemoglobinuria (PNH) Patients organized the March 7 demonstration in order to make a direct and urgent appeal to the Premier and Minister of Health for immediate, long-term access to Soliris, the life-saving treatment they need for survival.
Ontario patients with the ultra-rare blood disorder Paroxysmal Nocturnal Haemoglobinuria (PNH), supported by family and friends, pleaded with the government to end their long and needless wait by funding Soliris through the Ontario public drug programs.
“We have been waiting for two years for the government to take action on access to Soliris, since it was approved by Health Canada in 2009. PNH patients continue to face the threat of deadly blood clots with each day they go untreated,” said Barry Katsof, President of the Canadian Association of PNH Patients.
“No one deserves to live with this fear and uncertainty when a proven, effective life-saving treatment exists.”
In January, Maciesza’s battle with PNH – and the media attention it garnered – spurred an eleventh-hour change in Ontario’s compassionate review policy, which granted him temporary access to Soliris for only six months.
“While I am grateful to be alive, I can’t really begin living until I know with certainty that I will have permanent access to Soliris after my funding runs out in June,” said Maciesza.
“I am getting married in May and my fiancée and I need to know if we can start planning the rest of our lives, or if we will be back again fighting for my life and access to this drug.”
Studies show that long-term treatment with Soliris allows a patient’s life expectancy to return to that of a healthy person1. Without access to treatment, PNH patients are still at risk of developing a life-threatening blood clot or other serious complications. In fact, one third of patients do not survive more than five years from the time of diagnosis.
“Unfortunately, June is fast approaching and we may be back to square one,” Rick Maciesza, Lucas’ father, said in an email to the Advertiser.
“Government officials feel they have accomplished their mission, but we are concerned that we will be at their mercy once again as they dither to make a longer term decision.”
Canadian patients living with this rare and debilitating disease remain the last in the developed world with no long-term public access to Soliris.
“PNH experts in Ontario, and indeed around the world, support the call for access to Soliris because they know it works and agree there is simply no excuse for leaving PNH patients to die prematurely,” said Katsof. “The experts have spoken. The government has done its due diligence. We need an immediate and permanent decision to make Soliris accessible to all PNH patients who require this treatment.”
