The Canadian Association of PNH Patients applauds governments across Canada for their landmark agreement to provide public access to soliris, a life-saving treatment for a rare blood disease.
It is known as paroxysmal nocturnal haemoglobinuria. With the agreement, Canada joins a majority of jurisdictions that ensure access to Soliris for citizens through government funding.
In the past year, the high cost of soliris came to light because a Mount Forest man needed it to stay alive. The government was funding the drug only intermittently, and Lucas Maciesza nearly died. Doctors in London provided it to him without guarantee of payment. The provincial government later agreed to fund the life-saving treatment.
Currently, there are fewer than 90 people living with PNH in Canada, and many are awaiting publicly-funded access to soliris.
Maciesza, 26, who now lives in Guelph was near death in a London hospital when he was given temporary access to soliris, a treatment he requires for the rest of his life.
Maciesza and his new wife, Amanda, along with their families and supporters are pleased that a long-term funding solution has been found.
“We are so thrilled to receive the incredible News today that Canadians living with PNH will now receive government funding for this miracle treatment that saved my life,” said Maciesza.
“I can’t begin to express my gratitude to the Ontario government for coming to my rescue at my family’s greatest time of need, and to all the incredible people who supported us along the way.”
Since the treatment was approved by Health Canada in early 2009, the issue of inequitable access to Soliris across Canada raised a question of ethics among patients and medical experts. Those without private insurance were unable to receive the treatment through their provincial drug plan, while some patients had access to the drug through private coverage and others did not.
The premiers agreed at a recent provincial conference to pay for the drug.
Barry Katsof, founder and president of the Canadian Association of PNH Patients, said, “The provinces are to be commended for their commitment to providing equitable access to the care that PNH patients require and deserve.”
He added, “This News is welcomed with great relief by our community of patients and their loved ones.”
PNH is a rare, progressive, and fatal acquired disease that affects the blood and major organs. It develops without warning, usually when patients are in the prime of their lives. It is characterized by haemolysis (the destruction of red blood cells) which can lead to life-threatening blood clots, kidney disease, pulmonary hypertension, and other catastrophic consequences.
Without treatment, approximately one?third of PNH patients do not survive more than five years and about half die within 10 years from the time of diagnosis.
“Receiving access to Soliris means we can stop living in fear of dying from a blood clot or organ failure, and start living again and planning for our future,” said Maciesza. “This funding decision gives me faith that things are changing for people living with rare diseases. I think it’s a sign that we will be better supported by our governments in the future; this is the Canada I grew up to believe in.”
In June, Blood, the authoritative peer-reviewed journal of the American Society of Hematology, published a study that resoundingly proved the efficacy of soliris for the treatment of PNH. The results show that long-term treatment with soliris allows a patient’s life expectancy to return to that of a healthy person. Other studies have shown that treatment with soliris protects against PNH complications including blood clots, impaired kidney function and pulmonary hypertension.
Arnott pleased
Wellington-Halton Hills MPP Ted Arnott expressed appreciation to the provincial premiers of Canada for agreeing to provide funding for soliris.
“This is great News for Lucas Maciesza and his family, who fought long and hard to get this medication approved and save Lucas’ life,” Arnott said.
Last November, Arnott was contacted by Rick Maciesza, Lucas’ father.
Arnott said, “When I heard that Lucas Maciesza might die without soliris, we went to work. I repeatedly raised the issue with the Minister of Health, including questions in the legislature,” Arnott recalled. “I am so pleased that this story has had a happy ending.” The premiers of Canada have agreed to band together to bulk-buy Soliris. It is hoped that kind of cooperation can reduce the cost of medication for rare diseases, including PNH.
Arnott said, “The premiers deserve credit, but most of all, the credit should go to Lucas, his father Rick and the others, like Tanya Talaga, of the Toronto Star, who advocated so passionately, and in the end successfully,” Arnott concluded.
