Drug funding refusal for three children afflicted with rare disease to be reviewed

Approval ‘the hope of our life’

11416994Muhammad Amir Akhter helps his children, from left, Khadija, Sara and Muhammad Abdullah put their shoes on before heading to school at River Heights. The provincial health ministry is reconsidering drug funding for the children, who have an enzyme disorder.

Photograph by: Gord Waldner, The Starphoenix , The Starphoenix

The father of three children afflicted with a rare enzyme disease remains hopeful the province will pay for a costly drug therapy, despite receiving word last week that the Health Ministry had rejected his application.

Health Minister Dustin Duncan met Monday in Regina with Muhammed Akhter and his children and agreed to have the ministry seek a third opinion on whether Vimizim will be effective in treating their cases of a debilitating enzyme deficiency, Morquio syndrome or mucopolysaccaridosis IVA (MPS).

“While the answer last week was no, that doesn’t mean it’s no forever,” Duncan said Monday.

“Experts so far say there really isn’t efficacy when it comes to halting progression of the disease as children get older, but I want to have a second look at it.”

Akhter called the possibility of a positive result “the hope of our life.”

Vimizim, which can halt progression of the disease, can cost up to $460,000 per year.

Duncan said the children were denied approval based on the “Do not list” recommendation by the federal Common Drug Review (CDR) and on the opinion of an out-of-province specialist, who questions the effectiveness of the drug in children older than five.

The CDR considers how a new drug compares with alternatives, which patients will benefit and whether it will deliver value for money.

The Akhter children, who are eight, 10 and 12 years old, all began showing symptoms around age four or five, but doctors were unable to diagnose the problem until 2012, when all three were diagnosed in Winnipeg, their father said.

An advocate says the ministry’s decision was based on the drug cost rather than evidence about its effectiveness.

“We are really pushing the minister to make the decision based on the clinical data that shows the earlier treatment begins, the better, but there is no age limitations on which treatment can be beneficial,” said Jaime Myrah, executive director of the Canadian MPS society.

“All of the clinical trials took place in patients above the age of five, so there is actually no clinical data that suggests the treatment is more effective in children under the age of five. We do believe this is really a decision that is being made by financial considerations,” Myrah said.

The disease is estimated to appear in only one out of every 200,000 to 300,000 births.

The Akhter children’s bones and joints are weak, as are their hearts and lungs, and they have all suffered vision and hearing loss. They have also stopped growing.

Akhter wasn’t expecting the refusal to fund, given that the treatment is covered for one other child in Saskatchewan.

“My wife and I, we were very sad,” he said. “We are trying our best to get that medicine, to see them happy, healthy.”

Doctors have told Akhter the children can expect a lifespan of 20 to 30 years – and they’ll become more dependent as the disease continues to take its toll.

Myrah said patients have also been approved in Ontario, Quebec and soon Alberta. Saskatchewan and other provinces have approved treatments similar to Vimizim, she said.

With Betty Ann Adam files