5 November, 2015 mcfadyena

'It's a life-changing decision': Saskatoon kids to get enzyme treatment after all


When Muhammed Akhter got the phone call saying his three sick children would be getting their critical treatment after all, he had to ask the ministry representative three times be certain.

“It’s a life-changing decision,” he said.

After an external review, the provincial government has decided to cover the cost of Vimizim, an expensive enzyme replacement therapy used to treat the rare Morquio syndrome. This reverses the health ministry’s previous decision not to pay for the drug, which can cost up to $460,000 per year.

The information that went through the initial Common Drug Review process was “fairly limited” in terms of whether Vimizim was effective, Health Minister Dustin Duncan said.

“I’m comfortable with going to essentially a second opinion for myself and having outside experts weigh in and say there is, perhaps, some benefit for these particular patients based on their current conditions.”

Health ministers across the country are facing similar decisions due to different medical opinions, he said.

“In a lot of cases, tretments are changing perhaps more quickly than drug plans and provinces can keep up with them, but as well the medical community.”

The funding is for one year of treatment, after which the children will be examined to see how well it’s working.

The Akhter children, who are eight, 10 and 12 years old, all began showing symptoms around age four or five. They all have vision and hearing loss, weak bones and joints, and stunted growth.

Akhter is hopeful – in most cases Vimizim works, with some children even ridding themselves of their wheelchairs, he said.

“The patients who are taking that medicine, they are getting really better.”

Andrew McFadyen, executive director of advocacy group The Isaac Foundation, praised the decision.

“We’re elated, this is incredible news. At the same time, though, it really just brings us in line with what the expert opinion says.”

Difficulties exist not just in Saskatchewan but across the country when it comes to treatments for rare diseases, he said.

In fact, he’s headed to New Brunswick where another patient has been denied coverage for Vimizim.

“I’m hopeful the news out of Saskatchewan will prompt minters and ministries to take a step back and put in (place) a fair review system that gives access to these patients when they need it most.”



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