Rare disease means strict diet, but Alex is ‘OK with it’14 Aprile 2005
MALATTIE METABOLICHE : CONCERTO PER LA RICERCA24 Aprile 2005
“WHITEHORSE – The doctor for a 10-year-old Yukon boy with a rare genetic disorder says Canada needs to develop a mechanism to fund life-saving drugs.
Mackenzie Olsen, who now lives in Alberta, was part of a three-year clinical trial by the drug producer Genzyme, that provided an expensive, but life-prolonging drug for free. That trial ended last month.
Now Health Canada and the province are fighting over who should pay for the Yukon boy’s treatment.
Olsen has about a two-week supply left of the enzyme drug, called Aldurazyme.
It slows the progression of Hurler-Scheie Syndrome, which is caused by the lack of an enzyme called a-L-iduronidase.
Without the drug, Olsen’s health will deteriorate and he will eventually die. His doctor, Robyn Casey says it’s the first time patients with this disease have had any kind of an option.
“I’ve cared for patients with these sorts of disorders for 20 years now,” he says.
“And I’ve had other patients with this condition die, because we’ve had no treatments. Now we do have a treatment, and we can signficantly affect the outcome. But we have to be able to buy the drug to do that.
“That’s where the problem lies right now.”
Part of the funding problem is that Aldurazyme is a relatively new drug, and doesn’t fall under any federal or provincial health insurance plan.
It’s expensive as well. Olson’s parents can’t afford the $17,000 a week it takes to keep his treatment going. Casey says there needs to be a process in place to deal with the funding issues.
“The process that is currently in place and being utilized perhaps does not meet that need,” he says. “There’s a significant cost-benefit aspect to their analysis, which is very hard to achieve for rare diseases.”
Olsen’s parents are frustrated as they watch their child’s drug supply run out.
“It’s been a long few weeks since all this started,” says Raymond Amato, Olsen’s father. “We seem to be getting further ahead, and then with a blink of an eye we seem to be taking two steps back as well. So it’s a journey, that’s for sure.”
Meanwhile, the Assembly of First Nations says it will file a lawsuit against the province of Alberta. They want to force it to pay for Mackenzie’s treatment until the funding dispute between Health Canada and the province is worked out.