danielle chartier

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

NDP calls for reversal on denied drug coverage

Sticky Post By On 9 October, 2015

By François Biber

October 8, 2015 – 6:15pm

New Democratic Party MLA Danielle Chartier is challenging the province to reverse its decision to deny medical coverage for the children of a Saskatoon family with a rare disease known as Morquio A Syndrome.

“The Sask. Party government has denied three children their only hope to stop the progression of a terrible and debilitating disease it’s clear they have used incorrect information to make that decision,” Chartier told reporters in Saskatoon.

Earlier this week Muhammad-Amir Akhter was told by Health Minister Dustin Duncan, the province would not cover his children’s Vimizim treatment, a synthetic enzyme that helps the body break down cellular waste. Duncan added he was seeking out-of-province advice to see if this is the best treatment for Muhammad Abdullah, 12, Khadija Amir, 10 and Sara Amir, 8.

But Chartier said there are already 29 patients in Canada using the treatment, including one in Saskatchewan.

“First of all the government should be doing this on a compassionate basis, this is these children’s only hope, so if the government isn’t going to be compassionate they need to look at the evidence,” Chartier said, adding she believes Duncan’s claim that the drug hasn’t been proven to work for children under the age of five is completely false.

According to Health Canada, which has approved Vimizim, the safety and efficacy of the drug have not been established in children less than five years of age.

However, a common drug review conducted by the Canadian Agency for Drugs and Technologies and Health (CADTH) states in a report that evidence did not support the achievement of outcomes known to be clinically relevant to patients using Vimizim. The report also states the long-term safety profile of Vimizim requires further evaluation.

But for Akhter’s children, the situation isn’t getting any better without treatment.

“It’s heart-breaking. I understand, but we’re not losing our heart. We are staying positive and hopefully my kids will be getting the treatment and we will be able to see them happy and healthy in the future, I’m sure,” he said.

Coming in at around $300,000 per treatment, Akhter said they can’t afford treatment for three children without help from the province.

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)

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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.