ndp

08 Oct 2015
Sticky Post By Posted in Advocacy, Blog Postings, Morquio, MPS IVA, Newspaper, Saskatoon Siblings Permalink

NDP calls for immediate drug funding

Sticky Post By On 8 October, 2015

BY SEAN TREMBATH, THE STARPHOENIX OCTOBER 8, 2015 7:03 PM

Father Muhammad Amir Akhter helps children Muhammad Abdullah, Sara and Khadija on with their shoe before heading off to school at River Heights, September 16, 2015. Th children have a enzyme disorder and hope the province will fund treatment.

Photograph by: Gord Waldner , The StarPhoenix

Saskatchewan’s NDP Opposition is criticizing the provincial government for rejecting a Saskatoon family’s request to fund drug treatment for three children suffering from a rare developmental disease.

“We think the government needs to immediately reverse this decision and get these children the drugs they need,” said NDP health critic Danielle Chartier, MLA for Saskatoon Riversdale.

Muhammad Akhter has three children — Muhammad, 12, Khadija, 10, and Sara, eight — who suffer from Morquio syndrome or mucopolysaccaridosis IVA (MPS). The disease affects many aspects of development. The children have bowed legs, fragile bones, respiratory and heart problems and diminished sight and hearing.

“The symptoms are affecting almost every part of the body,” Akhter said.

Health Canada approved the drug Vimizim, or elosulfase alfa, as a treatment for MPS in 2014. It was also approved by the U.S Food and Drug Administration that year. Vimizim can cost up to $460,000 a year per patient.

Health Canada’s trials found the drug to be safe and effective for people aged five to 65. In particular, a test that measured how far patients could walk over six minutes saw a statistically significant improvement in those who had been given the drug.

“In conclusion, the efficacy of Vimizim (2 mg/kg/week) has been adequately demonstrated,” Health Canada’s report said.

Speaking to media on Monday about the decision to refuse coverage for the drug, Health Minister Dustin Duncan referred to a different study — a common drug review done by the Canadian Agency for Drugs and Technologies in Health (CADTH), a non-profit established by the federal and provincial governments in 1989.

The CADTH’s study looked at Health Canada’s testing and reached a different conclusion. It acknowledged the improvement in walking distance, but said “the clinical relevance of this finding is uncertain.”

When looking at the trials as a whole, the CADTH said there was not enough evidence to say the drug is effective.

“Treatment with elosulfase alfa has not been shown to improve other clinical end points, including reducing pain, fatigue, disease progression, or the need for surgical intervention,” the CADTH report said.

Duncan said the children were also denied approval because an out-of-province specialist questions the drug’s effectiveness in children older that five.

On Thursday, Chartier noted that Saskatchewan is already paying for another child — a girl under five — to receive the treatment.

The Isaac Foundation, a national advocacy group for people with enzyme disorders, released a statement in support of Vimizim treatment on Thursday. It said 29 people in Canada are receiving the treatment, 27 of whom are older than five.

“Every one of these patients is seeing improvements,” the statement said.

Chartier also provided a fact sheet from BioMarin Pharmaceutical Inc., the company that sells Vimizim, that states the drug is safe and effective for people five years and older.

Duncan said Monday that the government would review the denial and seek another professional opinion on the drug. Chartier said the government should reverse the denial immediately.

“These children every day experience issues that get worse. The time to make the decision to change is right now,” she said.

strembath@thestarphoenix.com

twitter.com/strembath

08 Oct 2015
Sticky Post By Posted in Advocacy, Blog Postings, Media, Morquio, MPS IVA, Newspaper, Saskatoon Siblings Permalink

Provincial opposition says drugs for sick kids denied over false information

Sticky Post By On 8 October, 2015

Expert says drug would work well for Akhter children

CBC News Posted: Oct 08, 2015 11:15 AM CT Last Updated: Oct 08, 2015 11:15 AM CT

Sara Amir, 8, Khadija Amir, 10, and Muhammad Abdullah, 12, all suffer from a crippling enzyme deficiency that threatens their lives. (Kathy Fitzpatrick/CBC)

Related Stories

The provincial New Democratic Party says Health Minister Dustin Duncan shouldn’t have refused drug coverage for three children.

Sara, Khadji and Muhammad Akhter all suffer from a degenerative enzyme disease called Morquio A Syndrome.

The province has denied drug coverage to the children, stating that the drug is not effective in children over five.

However, the NDP disagrees, saying that the drug’s manufacturer states that it is safe for children five years and older.

The disease can be life-threatening. Without the missing enzyme, cellular waste builds up in the bones, tissues, organs and muscles.

The condition is stunting the children’s growth, twisting their joints, affecting their eyesight and hearing, and making it tough for them to breathe. Two of them use wheelchairs at school.

Health Minister Dustin Duncan said he is looking for a second opinion on whether the province should pay for the drug. He said the original decision to deny funding the drug was based on medical opinion from the Common National Drug Review and on the recommendation of an out-of-province specialist.

Still, Duncan said he understands that timeliness is a factor, and said the decision will be communicated directly to the Akhter family.

07 Oct 2015
Sticky Post By Posted in Advocacy, Blog Postings, Media, Morquio, MPS IVA, Newspaper, Saskatoon Siblings Permalink

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

Sticky Post By On 7 October, 2015

Approval ‘the hope of our life’

 BY JONATHAN CHARLTON, THE STARPHOENIX OCTOBER 6, 2015
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

jcharlton@thestarphoenix.com

© Copyright (c) The StarPhoenix