Baie Sainte Anne Boy Needs Drug To Manage Rare Disease

December 9, 2015

A young Baie Sainte Anne boy’s plight made it to the floor of the provincial legislature this morning.

Morgan Doucet, 10, is currently the only person in New Brunswick suffering from morquio syndrome.

On Wednesday morning, Southwest Miramichi-Bay du Vin MLA Jake Stewart called on the province reverse its decision not to fund a Health Canada approved treatment for Doucet’s illness.

According to the U.S. National Library of Medicine, morquio syndrome is an inherited disease of metabolism in which the body is missing or doesn’t have enough of a substance needed to break down long chains of sugar molecules called glycosaminoglycans (formerly called mucopolysaccharides).

Symptoms include:

– Abnormal development of bones, including the spine
– Bell-shaped chest with ribs flared out at the bottom
– Coarse facial features
– Hypermobile joints
– Knock-knees
– Large head (macrocephaly)
-Short stature with a particularly short trunk
– Widely spaced teeth

While Stewart didn’t name the treatment in question, the only Health Canada approved drug for the disease is Vimizim, which was approved last year.

Annual treatment can cost upwards of $100,000.

Stewart said Doucet needs treatment.

“Recently access to this lifesaving medication for Morgan Doucet was denied by the minister of health and the department, even though it is the gold standard of care as recommended by the international treatment guidelines, has been recommended by the Canadian Expert Panel on Morquio Syndrome and has been prescribed by Morgan’s genetic specialist at the IWK ” Stewart said in the legislature.

He accused the government of overruling the experts and said there is no process for appeal.

Health Minister Victor Boudreau said he could not discuss the specifics of the case on the floor of the legislature, citing privacy rules, but said their is a process in place for situations like Doucet’s.

“Because we do not have the level of expertise on these rare diseases and rare drugs here in the province of New Brunswick we rely on the province of Ontario and this is a process that was put in place by the member for Rothesay, the former minister,” Boudreau said.

He explained that the file is sent to experts in Ontario and they get back to the New Brunswick government with a recommendation.
But Stewart wasn’t happy with that reply.

“The drug that Morgan requires is currently being funded by Saskatchewan, Ontario and Quebec and in most developed countries.  Morgan’s application was denied by a single reviewer from Ontario, a reviewer who no longer practices medicine and has never used this treatment,” Stewart said.

He also said Saskatchewan found flaws with the review process and with that reviewer. He said that province now deals directly with the experts on specific diseases.

“I ask the minister of health and the premier, will they show the same leadership and do the right thing by ordering a new review of Morgan’s file and provide immediate access to this life saving treatment while that review is taking place?” Stewart asked.

Boudreau said these are very tough situations.

“Everybody in this legislature has a heart and understands that these decisions are difficult to make,” Boudreau said.

“I can tell you that across the country, and in New Brunswick, some cases get approved, some cases don’t. We follow a process, Mr. Speaker. I am not an expert, and we don’t even have those experts within our department or within our province,” he added, saying the process was followed.

NDP calls for reversal on denied drug coverage

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.

Health Ministry denies funding for Sask. siblings with rare genetic disease

Lasia Kretzel

 October 5, 2015 – 5:20pm

Muhammad Akhter spent 12 hours trying to figure out how to tell his wife some terrible news.

The couple’s three children have been denied treatment coverage from the province for their rare genetic disease, but Health Minister Dustin Duncan said he will seek a second opinion.

Muhammad Abdullah, 12, Khadija Amir, 10 and Sara Amir, 8 have an enzyme-related disease called Morquio A Syndrome (also called MPS IV type A). Their bodies lack an enzyme in their blood that breaks down cellular waste in the body. Without treatment it can lead to multiple diseases and premature death. The children have already experienced growth problems and stiff joints. Two of the them already use wheelchairs.

There is no cure, but treatment with a synthetic enzyme called Vimizim could slow down the disease and potentially extend the life of the children. The treatment would cost hundreds of thousands of dollars a year for each child.

“I was not expecting to be denied from the (common drug review’s) side because this is the last resort. This is the last hope,” Akhter said. “It’s a feeling, it’s a pain from the inside. It’s pretty hard.”

The Ministry of Health sought consultation from experts outside the province and provided information from the family and their physicians. Health Minister Dustin Duncan said they were told the drug wouldn’t be as effective on the children because they were older than five years.

“The longer it takes for this drug to be administered, the disease progresses to a point where it just becomes a question of whether or not there is efficacy to administering this drug,” Duncan said. “You would like to be able to say yes to everybody, but you want to ensure that for the sustainability of the system that you’re using the health care dollars as wisely as possible.”

Morquio A Syndrome only affects an estimated 100 people in Canada right now, according to the group Morquio in Canada.


The Isaac Foundation said another Saskatchewan child has already received funding for the treatment, and Duncan said he only knew of one child being approved for therapy in recent memory.

The family, with the support of the foundation, met with the minister Monday to plead him to reconsider.

Duncan said because another child was approved for the drug before and because he feels there is debate in the medical community about the effectiveness of the drug at certain ages, he would like to review the siblings’ case.

“I’m going to ask the ministry to consult further with some other out-of-province experts to give me a second opinion on this,” Duncan said. “There’s not a lot of options for these patients so we want to make sure we give a full look before we close the door.”

Akhter said the family is now waiting anxiously, adding they aren’t sure what they will do if the second answer is still no.

“It’s like night and night; no shining,” he said.

  Twitter  @lkretzel

Funding For Rare Disease Treatment Denied, But Health Ministry Will Reconsider

The executive director of an advocacy group for rare diseases like Morquio Syndrome is puzzled as to why access for treatment has been denied for 3 Saskatoon siblings.

Andrew McFadyen, from the Isaac Foundation, along with the children and their father Amir Akhter spoke with Saskatchewan’s Health Minister this morning (Mon) in hopes of changing his mind.

McFadyen says the file on this case was sent to Ontario for review by only one person with no opportunity for different points of view, so today, he took the opportunity to speak to Dustin Duncan about why he should reverse the decision.

He adds that the Akhter family first requested access to provincial funding in March of 2014 and although the treatment wouldn’t cure the children, it could dramatically halt progression of the disease.

Dustin Duncan has said he will have another look at the case, but no date has been set for a decision.

The treatment is expensive at approximately $300,000 per year per child, but McFadyen says you could also argue that the cost of dealing with the effects of the disease when not treated could be similar when considering hip surgeries, corneal replacements, medical appointments, and other costs.

Duncan says the treatment costs $500,000 per patient.

There is a 2 year old in the province that has funding for treatment, which Duncan says is because the belief is that it’s more effective for children under 5.

McFadyen disputes that saying there is no data that actually proves that to be true.


Sask Health Minister wants second opinion on treatment for three Saskatoon children

Saskatchewan Health Minister Dustin Duncan is asking for a second opinion on an unproven drug to treat three young Saskatoon children.

Initially coverage for the drug was denied last week.

The drug is been used to treat a very rare and fatal blood disease called Morquio Syndrome.

Health Minister Duncan says he wants to make sure they do their due diligence.

There is one child in the province who is on the drug to treat the same disease. However studies seem to show it’s effective for those under the age of five.

But in this case the three children are eight or older.

The drug costs five hundred thousand dollars a year per patient.


Saskatchewan health minister to seek second opinion for family denied drugs

at 18:36 on October 05, 2015, EDT.

The Canadian Press

REGINA – Saskatchewan’s health minister says he will ask for a second opinion on an unproven drug for three Saskatoon children with a rare and often fatal blood disease.

The Akhter family has met with Dustin Duncan to ask that the government cover the expensive treatment for Morquio syndrome.

It would cost $500,000 a year per child and the family’s funding request was turned down by ministry officials last week.

The drug is not a cure, although studies indicate it is effective in slowing down the disease in children under five.

In this case, the three children are eight, 10 and 12.

Duncan says he wants to make sure the department does its due diligence.

“It’s a very difficult situation, so I’m going to ask the ministry to consult further with some other out-of-province experts to give me a second opinion on this,” Duncan said Monday.

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

There is one child in the province who is on the drug.

Duncan said it’s important to get as much information as possible through the drug review process and from the manufacturers.

NDP Opposition critic Trent Wotherspoon said he feels the family should get coverage on compassionate grounds, even if the drug is not 100 per cent proven.

“They deserve a lifeline. They deserve some hope,” Wotherspoon said. “The potential of halting this degenerative disease and the progression of that disease is so critically important (to these children).”

Morquio syndrome is a hereditary disease in which the blood lacks a certain enzyme. The syndrome is characterized by skeletal defects such as stunted growth, deformity of the spine and chest, short neck and loose and enlarged joints. It can also lead to thin tooth enamel and corneal clouding.

The Isaac Foundation, an organization that funds research projects aimed at finding a cure for Morquio syndrome, is advocating for the Akhters.

Executive director Andrew McFadyen said the decision to deny funding was made on scant evidence.

“This was only looked at by one reviewer in Ontario, who clearly disregraded the international treatment guidelines, who clearly ignored the Canadian expert opinion on treating this disease,” McFadyen said.

The ministry has suggested the family take a long-term palliative approach to care for the children.

McFadyen suggested that may prove even more costly to the health-care system.

“When we look at the total cost of that drug, it’s often noted that the cost to not treat these children may be the same … when you look at interventions, medical appointments, hip replacements, corneal transplants, etc.”

(CKRM, CJWW, The Canadian Press)

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