Ontario patients with Paroxysmal Nocturnal Haemoglobinuria (PNH) want the drug Soliris funded through the Ontario Public Drug Program.
Barry Katsof, president of the Canadian Association of PNH Patients, says the group has been waiting for the province to take action on access to the drug 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,” says Katsof. “No one deserves to live with this fear and uncertainty when a proven, effective life-saving treatment exists.”
PNH, which affects about 90 people in Canada, occurs when red blood cells are destroyed by a protein in the body’s immune system, leaving the organs without oxygen. This puts patients at risk for life?threatening blood clots, kidney disease, and organ failure.
In January, on compassionate grounds, the province granted 26-year-old Lucas Maciesza temporary access to Soliris for six months. Maciesza, who was very sick at the time, needs to take the drug regularly, a $500,000-a-year regimen he is unable to afford.
“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,” he says.
Ontario Minister of Health Deb Matthews said in the Legislature last November that a review by her department found that the drug should not receive blanket funding under the provincial drug program.
However, there may be a “small subset of patients for whom Soliris may be effective,” and that evidence is being reviewed, she said.
Soliris, which prevents the destruction of red blood cells, is said to have a high success rate in the majority of patients. Another effective treatment is a bone marrow transplant, but surgery carries a significant risk for those with PNH and it takes a long time to find a matching donor.
The onset of the disease usually occurs in the early 30s. According to the Canadian Association of PNH Patients, without treatment, a third of patients do not survive beyond five years and about half die within 10 years from the time of diagnosis.
The group cites studies showing that long-term treatment with Soliris allows a patient’s life expectancy to return to that of a healthy person. The drug is administered by infusion every 14 days by a licensed health care professional.
There are about 10 PNH patients waiting for publicly-funded access to Soliris in Ontario, one of whom is Hilary Handley, a mother of three from Toronto.
“Without Soliris, our disease could claim our lives at any moment without warning, yet the government will only consider helping us if we survive a catastrophic health event,” Handley said in a press release.
On Feb. 28, Rare Disease Day, the group launched a social media campaign to rally support for access to Soliris.
Meanwhile, Maciesza, who is getting married in the spring, hopes he will soon know if he can continue with the treatment beyond June.
“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.”
