MPS IVA

09 Dec 2015
Sticky Post By Posted in Advocacy, Morquio, MPS IVA, Uncategorized Permalink

This Is Not OK – When Answers Aren’t Really Answers

Sticky Post By On 9 December, 2015

To me, this is the most difficult part to accept. No appeal? No opportunity to ensure the decision was rendered correctly before we condemn this little boy to a life of pain and suffering…to death? Prisoners on death row – prisoners there for the heinous crimes they have committed – have multiple opportunities to appeal their decided fate. Before that, their fates get decided by collections of people, people who have the opportunity to hear all viewpoints, all expert testimony, everything. They exhaust all avenues in making death sentence rulings, and then we allow those prisoners the opportunity to appeal and fight their fate. Not so for this little boy, it appears, and that will never be OK with me.  ~Andrew McFadyen, Executive Director, The Isaac Foundation

Late last night, I boarded a plane to Fredericton with hopes of meeting with the Minister of Health, Victor Boudreau, about 10-year-old Morgan Doucet.  Recently, the Minister denied access to the life-saving medication that Morgan needs immediate access to, thereby condemning Morgan to a life of pain and suffering, and immediately signalling that Morgan’s care should enter a palliative approach to his disease.

This is NOT OK, for a whole host of reasons, not least of which is that we CAN help Morgan, we can stop his disease progression, and we can do it quickly with access to the treatment he needs.

This is not OK because the treatment Morgan needs has been approved by Health Canada for almost 2 years now, and it’s being used to help save the lives of  33 patients throughout the country, while being fully reimbursed for use in patients in Saskatchewan, Ontario, and Quebec.  It’s also funded in most developed countries throughout the world – including the UK – where they recently took steps to fund every patient that needs access immediately.

This is not OK because Morgan’s review was done by one person alone, a person who has never used this treatment before and a person who isn’t  a practicing physician anymore.  He’s a bureaucrat, paid by the government of Ontario to review files and render life and death decisions about kids – life and death decisions that come without rigorous debate and input from the true experts in the field.

This is not OK because the overwhelming evidence and recommendations by those true experts in the field all conclude that this drug works and should be given to patients immediately in order to stave off the ravages of the disease.  It has been prescribed by Morgan’s genetics specialist, it has been recommended for use in patients by the Canadian Expert Opinion on Morquio Syndrome and it is recommended as the front line treatment for Morquio Syndrome in the International Treatment Guidelines for Morquio Syndrome.  In fact, it’s considered the gold standard of care for patients suffering from this disease, and it’s inhumane and unethical to deny patients the help they need when they need it most.

This is not OK because we are allowing those bureaucrats and politicians to overrule and contradict medical experts, clinicians, and researchers, regarding best practice and treatment for this little boy suffering from this devastating disease.  In essence, the Minister and his bureaucrats are making clinical decisions where they are not qualified to do so.

And this is not OK because denying life-saving treatment to dying kids is not what our Health Care System is supposed to stand for, it’s not the principles that our country and our Medicare Act were founded on, and it’s not who we want to be as a Canadian People.

With all this on my mind, I took the late flight here for an arrival in New Brunswick in the middle of the night.  I came  to Question Period this morning to hear MLA Jake Stewart ask the hard questions of the Minister that he’s been avoiding answering.  Jake was poignant, thoughtful, and strong in his belief that Morgan should receive access to this drug.  After all, the Liberals themselves said last year that they “can – and MUST – do better” for our kids suffering from rare diseases.

With three questions asked, Minister Boudreau decided not to provide proper answers.  He started by saying there is a process, and that process includes a CDR report and they won’t fund drugs until that process is complete (it is, and was completed a long time ago).  He neglected to mention that no MPS drugs ever get the blessing of the CDR panel, yet New Brunswick is paying for every other drug for MPS diseases that are currently on the market.  He neglected to point out that the only patient with MPS in this province that isn’t receiving the life saving drug they need is Morgan.  He neglected to say that this has less to do with what the experts say and more to do with the cost of the drug, something that should never be a hinderance to proper care in this country.

Minister Boudreau commented on the process used – Morgan’s file was sent to a reviewer in Ontario to make a decision whether to treat or not.  One reviewer.  With no avenue for appeal after any decision is rendered.  He didn’t comment on that in Question Period, but after the decision was delivered I asked the Ministry of Health how we can appeal the ruling.  I was ignored.  I asked the Minister of Health 7 times for a meeting or information about how we can appeal the ruling.  I was ignored.  And I asked the Premier multiple times for a meeting to discuss an appeal.  As you all know, I received a note telling me that he was unable to meet, with apologies “for the inconveniences this may cause.”

To me, this is the most difficult part to accept.  No appeal?  No opportunity to ensure the decision was rendered correctly before we condemn this little boy to a life of pain and suffering…to death?  Prisoners on death row – prisoners there for the heinous crimes they have committed – have multiple opportunities to appeal their decided fate.  Before that, their fates get decided by collections of people, people who have the opportunity to hear all viewpoints, all expert testimony, everything.  They exhaust all avenues in making death sentence rulings, and then we allow those prisoners the opportunity to appeal and fight their fate.  Not so for this little boy, it appears, and that will never be OK with me.

As I was typing this entry, Minister Boudreau dropped over to meet with me to discuss Morgan and his plea for help.  I am thankful that he did, as I was able to present everything I’ve written above.  He made a commitment to take a look at this case but truly believes in the process put in place.  In his words, he will make not promises or commitments, but he will look at things and get back to me.

Knowing this, I implore Minister Boudreau to take a real look at this case and seek a new review of Morgan’s application.  I asked Minister Boudreau to seek outside opinions from a panel of 3 or 5 International and Canadian experts that deal front-line with this disease and this treatment each and every day.  I asked Minister Boudreau to allow a rigorous discussion on all available evidence that exists on this drug and the impact it has on patients, and I asked for expediency because it’s unethical to make a child and his family wait.

I asked this to allow for a transparent and fair process for Morgan.  His life depends on it and it’s the right thing to do.  And in the end, if the Minister doesn’t want to listen to and seek feed back from clinical expert opinion, patient reported outcomes, peer-reviewed published journals, etc., then who will he listen to?  The opinion of 1 person who has never used this treatment and didn’t engage with those experts to help make his decision on Morgan’s fate?

If that’s the case, it’ NOT OK.

I’ll be here for a short while and will be putting out a PR tomorrow morning so that media can cover this story if they choose.  Thank you for all of your support over the past little while – our last entry was seen over 20,000 times in a few short days.  Sharing Morgan’s story is helpful, and staying ready to fight means the world to us.

I’ll update with any news as it develops.

07 Dec 2015
Sticky Post By Posted in Advocacy, Morquio, MPS IVA Permalink

NB Liberals and Rare Diseases

Sticky Post By On 7 December, 2015

Where do the Liberals stand on access to rare disease treatments in New Brunswick?  Just last year, Donald Arsenault  (Liberal health critic and a current Minister in the Gallant Government) said that “that we can – and must – do better.”  

These statements came as he criticized a new rare disease strategy the previous government was about to implement.  At that time, he pushed for more access to treatments for patients, just like the access his government is denying Morgan today.

He’s right – the Liberals must do better.

We’ve had over 15,000 view our blog about Morgan in the past day and a half.  I’ll be traveling to NB this week and hope to meet with decision makers in hopes that they will take steps to review their decision to deny Morgan the care he needs.

For the record, here is the entirety of Mr. Arsenault’s speech.

Statement by Liberal Opposition Health Critic Donald Arseneault

As countries around the world shine a necessary light on rare diseases today, in New Brunswick the discussion is timely.

On a global scale, we know that over 50 per cent of all rare diseases affect children. We also know that many of the people who suffer from rare diseases are never properly treated because symptoms are often misdiagnosed.

And, perhaps of most significance, because so much has yet to be learned about rare diseases in general, people who suffer from them frequently encounter difficulties accessing adequate care and, in many cases, securing financial support for that care.

In New Brunswick, a new prescription drug program will soon be in place. Unfortunately, the new plan will only cover drugs that are currently listed on the provincial formulary. This leaves a lot of people, including those suffering from rare diseases, without the support they so desperately need.

Rare Disease Day is a reminder that we can – and must – do better. We hope that New Brunswickers will join us in drawing further attention to this important cause.

05 Nov 2015
Sticky Post By Posted in Advocacy, Morquio, MPS IVA, Newspaper, Saskatoon Siblings Permalink

'It's a life-changing decision': Saskatoon kids to get enzyme treatment after all

Sticky Post By On 5 November, 2015

BY JONATHAN CHARLTON, THE STARPHOENIX NOVEMBER 3, 2015

When Muhammed Akhter got the phone call saying his three sick children would be getting their critical treatment after all, he had to ask the ministry representative three times be certain.

“It’s a life-changing decision,” he said.

After an external review, the provincial government has decided to cover the cost of Vimizim, an expensive enzyme replacement therapy used to treat the rare Morquio syndrome. This reverses the health ministry’s previous decision not to pay for the drug, which can cost up to $460,000 per year.

The information that went through the initial Common Drug Review process was “fairly limited” in terms of whether Vimizim was effective, Health Minister Dustin Duncan said.

“I’m comfortable with going to essentially a second opinion for myself and having outside experts weigh in and say there is, perhaps, some benefit for these particular patients based on their current conditions.”

Health ministers across the country are facing similar decisions due to different medical opinions, he said.

“In a lot of cases, tretments are changing perhaps more quickly than drug plans and provinces can keep up with them, but as well the medical community.”

The funding is for one year of treatment, after which the children will be examined to see how well it’s working.

The Akhter children, who are eight, 10 and 12 years old, all began showing symptoms around age four or five. They all have vision and hearing loss, weak bones and joints, and stunted growth.

Akhter is hopeful – in most cases Vimizim works, with some children even ridding themselves of their wheelchairs, he said.

“The patients who are taking that medicine, they are getting really better.”

Andrew McFadyen, executive director of advocacy group The Isaac Foundation, praised the decision.

“We’re elated, this is incredible news. At the same time, though, it really just brings us in line with what the expert opinion says.”

Difficulties exist not just in Saskatchewan but across the country when it comes to treatments for rare diseases, he said.

In fact, he’s headed to New Brunswick where another patient has been denied coverage for Vimizim.

“I’m hopeful the news out of Saskatchewan will prompt minters and ministries to take a step back and put in (place) a fair review system that gives access to these patients when they need it most.”

jcharlton@thestarphoenix.com

Twitter.com/J_Charlton

© Copyright (c) The StarPhoenix
05 Nov 2015
Sticky Post By Posted in Advocacy, Morquio, MPS IVA, Newspaper, Saskatoon Siblings Permalink

‘It’s a life-changing decision’: Saskatoon kids to get enzyme treatment after all

Sticky Post By On 5 November, 2015

BY JONATHAN CHARLTON, THE STARPHOENIX NOVEMBER 3, 2015

When Muhammed Akhter got the phone call saying his three sick children would be getting their critical treatment after all, he had to ask the ministry representative three times be certain.

“It’s a life-changing decision,” he said.

After an external review, the provincial government has decided to cover the cost of Vimizim, an expensive enzyme replacement therapy used to treat the rare Morquio syndrome. This reverses the health ministry’s previous decision not to pay for the drug, which can cost up to $460,000 per year.

The information that went through the initial Common Drug Review process was “fairly limited” in terms of whether Vimizim was effective, Health Minister Dustin Duncan said.

“I’m comfortable with going to essentially a second opinion for myself and having outside experts weigh in and say there is, perhaps, some benefit for these particular patients based on their current conditions.”

Health ministers across the country are facing similar decisions due to different medical opinions, he said.

“In a lot of cases, tretments are changing perhaps more quickly than drug plans and provinces can keep up with them, but as well the medical community.”

The funding is for one year of treatment, after which the children will be examined to see how well it’s working.

The Akhter children, who are eight, 10 and 12 years old, all began showing symptoms around age four or five. They all have vision and hearing loss, weak bones and joints, and stunted growth.

Akhter is hopeful – in most cases Vimizim works, with some children even ridding themselves of their wheelchairs, he said.

“The patients who are taking that medicine, they are getting really better.”

Andrew McFadyen, executive director of advocacy group The Isaac Foundation, praised the decision.

“We’re elated, this is incredible news. At the same time, though, it really just brings us in line with what the expert opinion says.”

Difficulties exist not just in Saskatchewan but across the country when it comes to treatments for rare diseases, he said.

In fact, he’s headed to New Brunswick where another patient has been denied coverage for Vimizim.

“I’m hopeful the news out of Saskatchewan will prompt minters and ministries to take a step back and put in (place) a fair review system that gives access to these patients when they need it most.”

jcharlton@thestarphoenix.com

Twitter.com/J_Charlton

© Copyright (c) The StarPhoenix
09 Oct 2015
Sticky Post By Posted in Advocacy, Blog Postings, Media, Morquio, MPS IVA, Television Permalink

Medical treatment funding plea for children with rare genetic disease

Sticky Post By On 9 October, 2015

David Giles, Senior Web ProducerBy Senior Web Producer  Global News

WATCH TV NEWS COVERAGE

SASKATOON – Amir Akhter is waiting for a life-changing phone call, a phone call that could determine the future of three of his children. Earlier this week, the Saskatchewan government denied funding an expensive treatment that may help them live longer.

The three children suffer from the same rare genetic disease.

“Skeletal disorder, hearing loss, vision loss, spinal cord problems, their heart, lungs, almost every part of the body,” said Akhter.

READ MORE: Rare genetic disease diagnosed in 3 kids from same Saskatoon family

But a treatment called Vimizim could change things. It costs hundreds of thousands of dollars and the Akhter’s were hoping the province could foot the bill.

That was rejected on Monday by the Saskatchewan government.

“The experts at this point have said there really doesn’t appear to be efficacy when it comes to halting the progression of the disease as they get older, but I want to have a second look at it,” Health Minister Dustin Duncan said at the time.

He added that because the kids are past the age of five, the treatment might not even work.

READ MORE: Treatment funding denied for 3 children with rare genetic disease

The Opposition NDP says that’s not true at all.

“That is the exact opposite of what the evidence says,” said Danielle Chartier, the NDP health critic.

“You can look at Vimizim’s own fact sheet that proves the drug is effective in those older than five.”

Chartier added that another child in the province is getting the same kind of treatment. Her age has not been confirmed.

For Akhter, it’s not about the back and forth disputes, it’s about watching his children deteriorate every single day.

“It’s heartbreaking, but we are not losing our heart, we are still very positive,” said Akhter.

“Hopefully, my kids will be getting that treatment.”

Biomarin Pharmaceuticals, a manufacturer of Vimizim, confirmed that safety and effectiveness have been proven for kids five year or older on their website.

The Health Canada website too says it is the other way around, that efficacy hasn’t been established for those under five.

The family is waiting to hear back about a clarification and a final decision.

Leena Latafat contributed to this story

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)

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.

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

FOR IMMEDIATE RELEASE: MINISTER ORDERS REVIEW OF DECISION TO DENY LIFE-SAVING DRUG.

Sticky Post By On 8 October, 2015

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.”

 # # #

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.

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

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