Ontario to provide 'interim' funding for patients with ultra-rare disease
Marlene Leung, CTVNews.ca
Published Wednesday, February 25, 2015 11:31AM EST
Last Updated Wednesday, February 25, 2015 7:26PM EST
Ontario patients with a rare life-threatening disease will now be provided with interim funding for an expensive drug treatment that can cost hundreds of thousands of dollars a year per patient.
Atypical Hemolytic Uremic Syndrome (aHUS) is a progressive disease that causes the formation of blood clots throughout the body, which can lead to stroke, heart attack and kidney failure. The disease can affect both adults and children, and is often linked to genetics.
The Ontario government announced Wednesday it would begin "interim" funding for patients who meet the clinical criteria for the disease and require the drug Soliris.
"Our government is committed to getting people the drugs they need and the drugs that work for them," the province's health minister Eric Hoskins said in a statement.
"When people have an illness and there is a drug that can help them with that, we do everything we can to get access to those particular drugs."
But Progressive Conservative health critic Christine Elliott called it a "last-minute, half-baked announcement," made right after aHUS Canada raised its concerns at a news conference at the legislature.
"How convenient is it that the announcement was made today," Elliott said. "I guess it takes a little bit of the heat off you, but I think people need to know that this is not going to be funded for all aHUS patients; it's only a one-off strategy.
"Only those who are really, really sick are going to be able to get access to this, not everybody who needs it."
Several studies have found Soliris to be effective in helping to control aHUS. In a small 2013 clinical trial, researchers found the drug was associated with improvement in renal function, as well as quality of life.
aHUS Canada estimates approximately 30 patients in the province could benefit from the drug.
But Soliris, made by Alexion Pharmaceuticals, is very expensive, with some estimates pegging the cost per patient to be as high as $500,000 per year.
Last month, the National Institute for Health and Care Excellence in the U.K. issued a final guidance, approving funding for the drug. In Canada, Quebec also funds Soliris.
Among aHUS patients who have received Soliris is 13-year-old Joshua DeBortoli, who told reporters Wednesday that the drug allowed him to get healthier and return to school.
The boy’s mother, Sonia Girotto, said her son now has a paper route and is able to play soccer and drums.
“For our family, access to Solaris meant that we got our little boy back,” she said.
Last year, Ontario mother and aHUS patient Johann Kerlow was granted temporary funding for Soliris for up to six months through the province's Compassionate Review Policy.
Kerlow, who lives in Markham, Ont., had been hospitalized several times because of the disease.
"I was fortunate to begin receiving Soliris when nothing else was controlling my disease, and I am grateful to be alive, but my kidneys sustained so much damage while I was waiting for access to treatment that I am now fully dependent on dialysis," Johann said in a statement released by aHUS Canada on Wednesday.
"Soliris has certainly changed the quality of my life, and without it, I am fearful that my condition will begin to deteriorate again."
With files from CTV Toronto and The Canadian Press