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FOR IMMEDIATE RELEASE: MINISTER ORDERS REVIEW OF DECISION TO DENY LIFE-SAVING DRUG.
TREATMENT REQUIRED IMMEDIATELY BY 3 SASKATOON SIBLINGS; ADVOCATES CALL ON PROVINCE TO ALLOW IMMEDIATE ACCESS WHILE REVIEW TAKES PLACE
“We’re calling on Minister Duncan to tell the public the truth about this drug. His statements so far have been misleading and, frankly, categorically untrue.”– Andrew McFadyen, The Isaac Foundation
The Saskatchewan Minister of Health has ordered a full review of his recent decision to deny access to a life-saving medication for 3 siblings from Saskatoon. 8 year-old Sara Amir, along with her siblings Khadija, 10, and Muhammad, 12, suffer from MPS IVA (Morquio Syndrome) and require the life-saving treatment immediately in order to halt further progression of their devastating disease. On Friday, October 2nd, the Minister denied funding for the treatment. After meeting with the children’s father, Amir Akhter, and members of The Isaac Foundation and the Canadian MPS Society, he agreed to revisit the decision and consult International experts that deal first-hand with the disease and the drug treatment.
Andrew McFadyen, Executive Director of The Isaac Foundation, an advocacy, research, and family support organization that specializes in MPS related diseases, was happy with the news. “We are very pleased with this development and hope that this review takes a serious look at the vast amount of available evidence that exists both in this country and Internationally. The reality here is that Saskatchewan is denying access for these kids at a time when almost every other developed nation in the world are approving access for their kids. There is an incredible disconnect that exists between the initial decision from the Minister and what is happening in other jurisdictions and throughout the rest of the world.”
While pleased with the developments, McFadyen is frustrated with the misinformation being provided by the Minster of Health regarding the impact this treatment has on patients. “The Minister continues to say that there isn’t evidence to show this treatment works in patients over the age of 5. The real truth is that there are 29 patients receiving this drug in Canada. 27 of those patients are over the age of 5 and the vast majority of those patients are over the age of 13. Every one of these patients is seeing improvements. All of them. I’ve told Minister Duncan this on numerous occasions so either the Minster is being fed misinformation from the bureaucrats in his ministry or he is deliberately misleading the public to help justify his cruel decision to deny these kids the help they need. Either way, the evidence just doesn’t support the statements he is making and I think the public and this family deserve to know the truth.”
Jamie Myrah, Executive Director of the Canadian MPS Society, is calling on Health Minister Dustin Duncan and Premier Brad Wall to allow the siblings to begin treatment immediately while the review of their applications takes place. “These kids have waited long enough and they need to begin therapy now. We can’t afford to wait any longer.”
With respect to the review, McFadyen hopes it will be thorough and transparent, and free from Ministry bias and interference. “I’ve sent a message to the Minister asking for a fair, objective, and transparent review of these applications. It is our hope that the true experts –experts from around the world that deal with this disease, experts that treat this disease on a daily basis – have an opportunity to see the baseline data and weigh in on the merits of providing therapy for these children. We also feel like it’s imperative that the reviewers are identified to the public, again so we can be sure the process is as transparent as possible. We want to make certain they are using the true experts to look at this, unlike the initial review that took place.” Thus far, McFadyen hasn’t received a response from the Ministry but adds, “I’m hopeful to hear something today.”
In the meantime, he says he will be in attendance during Question Period when the Legislature resumes sitting next week. “The lives of these kids depend on this drug, and I won’t sit idly by and watch them to die. I’ll be in there every day until a decision is rendered, and I’ll be readily available to the Minister and his office if they need more help and information along the way.”
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For more information about this topic, or to schedule an interview with Andrew McFadyen, please call Andrew at 613-328-9136 or email Andrew at mcfadyena@me.com. The Isaac Foundation can also arrange interviews with parents of patients currently receiving this treatment in Canada.
Backgrounder
Sufferers of Morquio Syndrome lack an enzyme in their blood that breaks down cellular waste in the body. This waste builds up in the bones, tissues, organs, and muscles of affected individuals and leads to many devastating symptoms including heart and airway disease, corneal clouding, stiffening of the joints, shortened stature, and premature death.
There are 29 patients receiving therapy in Canada. 2 patients are under the age of 5 while 27 are older. The vast majority of patients are over the age of 13 and the oldest patient receiving therapy is 39.
While not a cure for Morquio Syndrome, the necessary Enzyme-Replacement Therapy (ERT) is designed to provide patients with a synthetic version of the enzyme they are lacking by infusing small doses into the patient’s bloodstream on a weekly basis. The treatment slows down or halts progression of the disease in patients, improves endurance, walking distance, breathing problems, and provides other benefits to sufferers that dramatically improve their quality and length of life. International experts and a Canadian Panel of Genetics Specialists have all recommended Vimizim as the front-line treatment for Morquio Syndrome. Vimizim was approved by Health Canada in July 2014, and is currently being reimbursed for use by patients in Saskatchewan, Ontario, and Quebec. Recently, the National Institute for Health and Care Excellence (NICE) recommended reimbursement for all 88 patients suffering from Morquio Syndrome throughout the UK.
The therapy has been recommended by a Canadian Expert Panel comprising of MPS experts from BC, Alberta, Saskatchewan, Ontario, and Quebec, and International Treatment Guidelines for Morquio Syndrome highlight this treatment as the gold standard of care for affected patients.
Drug funding refusal for three children afflicted with rare disease to be reviewed
Approval ‘the hope of our life’
Muhammad 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
Health Ministry denies funding for Sask. siblings with rare genetic disease
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.
IS IS THE LAST HOPE.
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.
SASKATOON FAMILY DENIED FUNDING FOR TREATMENT
It’s a parent’s worst nightmare; watching your child lose a battle to a devastating disease. Now imagine having to suffer through it multiple times.Muhammad, Sara and Kadija Akhter suffer from a rare but crippling genetic disease called Morquio Syndrome that’s changed their childhood.“This syndrome is progressive. So every day they are losing something … they’re losing more and more. So it’s getting tougher for the family as well as the kids,” said father Amir Akhter. Story continues below
Rare genetic disease diagnosed in 3 kids from same Saskatoon family
The siblings all have the disease which occurs when people are missing a genetic enzyme. It’s twisting their joints, seriously impacting their vision and hearing and they can hardly walk or run.
There is no cure, but until recently, there was still hope. The family was anxiously hoping the province could pay for a treatment called Vimizim. The therapy could help them live longer and could even halt the progression of their disease.The therapy costs hundreds of thousands of dollars.On Monday, there was more heartbreak. The family was denied funding for the treatment.“I should say that we are although walking and eating…we sometimes feel that we are not alive. And we have many challenges. And there is no hope from any end,” said Amir Akhter.
It’s something Amir Akhter would never say in front of them but without therapy, Muhammad, Sara and Kadija could die. Still, they’re hopeful.
They spent Monday in Regina, urging the Saskatchewan Ministry of Health and the premier to reverse the province’s decision.“Hopefully if we get that treatment … there is a new life, new whole life, new world, everything for us, for our family,” said Amir Akhter.
But Saskatchewan Health Minister Dustin Duncan argues that because the kids are past the age of five, this particular treatment may not even be effective. “The health world is so complex and there are so many diseases and so many drugs. You want to be able to say yes to everyone but … healthcare dollars as wisely as possible,” Duncan said.
Still, the minister says he will have a second look. On the other hand, others argue that there’s no time for that and that this therapy is their last shot at survival. “They deserve a lifeline. They deserve some hope. And the potential of halting this disease is so critically important to this eight-year-old, this 10-year-old this 13-year-old and of course, this family,” said Saskatchewan NDP deputy leader Trent Wotherspoon. Although a decision has already been made, they are expected to hear back about a reversal in the next 10 days. Until then, the family will hold on to all they have, their children and hope.
© Shaw Media, 2015
SOURCE: http://or-politics.com/canadahealth/saskatoon-family-denied-funding-for-treatment/102383/
Funding For Rare Disease Treatment Denied, But Health Ministry Will Reconsider
- Written by Carol Thomson
- Category: Local News
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.
SOURCE: http://www.saskatoonhomepage.ca/index.php?option=com_content&view=article&id=69454:funding-for-rare-disease-treatment-denied-but-health-ministry-will-reconsider&catid=61&Itemid=179
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.
SOURCE: http://www.620ckrm.com/ckrm-on-air/ckrm-local-news/12882-sask-health-minister-wants-second-opinion-on-treatment-for-three-saskatoon-children
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) Content Provided By Canadian Press. Source: http://www.cfra.com/HealthCP/Article.aspx?id=481958 |
Treatment funding denied for 3 children with rare genetic disease
SASKATOON – It’s a parent’s worst nightmare; watching your child lose a battle to a devastating disease. Now imagine having to suffer through it multiple times.
Muhammad, Sara and Kadija Akhter suffer from a rare but crippling genetic disease called Morquio Syndrome that’s changed their childhood.
Video – http://globalnews.ca/video/2260432/treatment-funding-denied-for-3-children-with-rare-genetic-disease
“This syndrome is progressive. So every day they are losing something … they’re losing more and more. So it’s getting tougher for the family as well as the kids,” said their father, Amir Akhter.
The therapy costs hundreds of thousands of dollars.
On Monday, there was more heartbreak. The family was denied funding for the treatment.
“I should say that we are although walking and eating…we sometimes feel that we are not alive. And we have many challenges. And there is no hope from any end,” said Akhter.
It’s something Amir Akhter would never say in front of them but without therapy, Muhammad, Sara and Kadija could die. Still, they’re hopeful.
They spent Monday in Regina, urging the Saskatchewan Ministry of Health and the premier to reverse the province’s decision.
“Hopefully if we get that treatment … there is a new life, new whole life, new world, everything for us, for our family,” said Akhter.
READ MORE: Rare genetic disease diagnosed in 3 kids from same Saskatoon family
But Saskatchewan Health Minister Dustin Duncan argues that because the kids are past the age of five, this particular treatment may not even be effective.
“The health world is so complex and there are so many diseases and so many drugs. You want to be able to say yes to everyone but … healthcare dollars as wisely as possible,” Duncan said. Still, the minister says he will have a second look.
On the other hand, others argue that there’s no time for that and that this therapy is their last shot at survival.
“They deserve a lifeline. They deserve some hope. And the potential of halting this disease is so critically important to this eight-year-old, this 10-year-old this 13-year-old and of course, this family,” said Saskatchewan NDP deputy leader Trent Wotherspoon.
Although a decision has already been made, they are expected to hear back about a reversal in the next 10 days.
Until then, the family will hold on to all they have, their children and hope.
© Shaw Media, 2015
Source: http://globalnews.ca/news/2260420/saskatoon-family-denied-funding-for-treatment/
Province says no to medicine for three children, but will review decision
Children suffering from crippling genetic disease
CBC News Posted: Oct 05, 2015 6:43 PM CT Last Updated: Oct 05, 2015 7:00 PM CT
The province’s health ministry says it won’t pay for a costly drug that may help three children with a crippling genetic disease, but Health Minister Dustin Duncan said he will ask for a second opinion.
Muhammad Abdullah, 12, Khadija Amir, 10 and Sara Amir, 8 all have a rare enzyme deficiency called Morquio A Syndrome (also called MPS IV type A).
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.
Muhammad Amir Akhter is the children’s father. He said the disease can be life-threatening.
“Enzyme replacement therapy like Vimizim is the last resort,” Akhter said.
He said despite the current decision, he is staying positive. “It’s pretty hard for us, because this is the last hope here.”
Health minister will get a second opinion
Health Minister Dustin Duncan explained the decision to reject the drug coverage.
“These are very, very complex cases,” said Duncan.
He said in this case there is not enough evidence to prove that the drug will be effective for people over the age of five.
“There’s a lot of opinions on this. And so while we do rely and have relied on a previous case, on an out-of-province expert I’m asking the ministry to consult with others that may have some information on this particular case.”
Duncan said the ministry will review some of the medical debate about the efficacy of the drug, and will invite the Akhter family to submit any further evidence they find.
“While the answer last week was no, it doesn’t mean it’s no forever.”
The Opposition NDP said the children should get the drug since it is their only hope. It added that effectiveness can be monitored while they take it.
CTV NEWS – Family denied funding for life-saving treatment
October 5, 2015
Source: http://saskatoon.ctvnews.ca/video?clipId=720671