TORONTO ON –The Canadian Association of PNH Patients applauds governments across Canada for their landmark agreement to provide public access to Soliris (eculizumab), a life-saving treatment for the ultra-rare blood disease known as paroxysmal nocturnal haemoglobinuria (PNH). With this agreement, Canada joins the vast majority of jurisdictions in the developed world that ensure access to Soliris for their citizens through government funding.
Currently, there are fewer than 90 people living with PNH in Canada, and many among them are awaiting publicly-funded access to Soliris. In November 2010, Lucas Maciesza, 26, was near death in a London, Ontario hospital when he was given temporary access to Soliris, a treatment he requires for the rest of his life. Today, Lucas and his new wife, Amanda, along with their families and supporters are overjoyed 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,” says Lucas. “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. A long-term funding solution for Soliris was sought by Canadian PNH patient and physician advocates, who appealed to their own provincial governments for support.
“The provinces are to be commended for their commitment to providing equitable access to the care that PNH patients require and deserve. Under Ontario’s leadership, the cooperation and dedication they have shown in supporting patients with this rare disease is truly groundbreaking,” says Barry Katsof, Founder and President of the Canadian Association of PNH Patients. “This news is welcomed with great relief by our community of patients and their loved ones. We thank you for giving us our lives back.”
PNH is a very 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 diagnosis1.
“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,” says Lucas. “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.”
With this funding announcement, the public drug programs have aligned themselves with the global medical community, standing solidly behind this treatment and supporting mounting evidence that Soliris saves lives. In June 2011, Blood, the authoritative peer-reviewed journal of the American Society of Hematology, published a study that resoundingly proved the efficacy of Soliris (eculizumab) for the treatment of PNH[i]. The results show that long-term treatment with Soliris allows a patient’s life expectancy to return to that of a healthy person1. Other studies have shown that treatment with Soliris protects against PNH complications including blood clots[ii], impaired kidney function and pulmonary hypertension.
About the Canadian Association of PNH Patients
The Canadian Association of PNH Patients is a not-for-profit Canadian organization formed in 2009. The mission of the organization is to connect Canadians affected by Paroxysmal Nocturnal Haemoglobinuria and advocate best possible care for patients. The organization also provides support to caregivers, and works to increase awareness and understanding of PNH.