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Health minister under pressure to approve expensive drug for sick boy

CHRIS MORRIS Legislature Bureau

December 9, 2015

Andrew McFadyen, executive director of The Isaac Foundation, travelled from Ontario to Fredericton on Wednesday to lobby the Liberal government on behalf of Morgan Doucet, a 10-year-old boy from the Miramichi region, who has been denied treatment for a life threatening condition.

Photo: Chris Morris/Legislature Bureau

FREDERICTON • The New Brunswick Health Department is under pressure to reconsider the case of a little boy in northern New Brunswick who has been denied access to an expensive medical treatment that could improve and possibly lengthen his life.

Jake Stewart, opposition Tory MLA for Southwest Miramichi-Bay du Vin, raised the issue in the legislature on Wednesday, appealing to Health Minister Victor Boudreau to reconsider his department’s earlier decision to deny the child, Morgan Doucet of Baie-Sainte-Anne, a promising new treatment for the rare disease, Morquio Syndrome.

The treatment, which would cost about $300,000 a year for Morgan, has been approved by Health Canada but it was not given the go-ahead in New Brunswick following a review. The New Brunswick Health Department formally rejected the Doucet family’s application for help in October and has not provided an avenue for appeal.

Stewart said the family now has decided to allow their situation to become public. He said it is a heartbreaking case, made worse by what he described as a seemingly callous and bureaucratic response to the plight of a child in need.

“It is really simple what they (health officials) have to do: he is a 10-year-old boy; he is the only citizen of New Brunswick who has this disease; it is life-threatening – help him,” Stewart said in an interview.

“That is all we are asking. It’s not about politics – just help this little boy.”

Boudreau told the legislature that he cannot comment on the case specifically because of privacy issues. But he insisted he is not heartless.

“These are very difficult situations,” Boudreau said. “These are not easy files to deal with. I can assure you of that. Everybody in this legislature has a heart and understands that these decisions are difficult to make.

“Without talking about the case in particular, I can tell you that, across the country and in New Brunswick, some cases get approved and some cases do not. We follow a process.”

After question period on Wednesday, Boudreau met privately with Andrew McFadyen of the Ontario-based Isaac Foundation who travelled to Fredericton to press the minister on Morgan’s case.

“He made no commitments or promises,” McFadyen said of Boudreau. “He believes in the process currently in place but he will take a step back and look at this case and be in touch with me.

“I’ve been trying to talk with him about this decision since it was rendered in October and I have been stonewalled. It is encouraging but I had hoped for a bit more, at least a commitment to have a second review of this.”

The Health Department would neither confirm nor deny that the minister has agreed to take another look at the file, citing privacy concerns.

The process followed by the Health Department involved sending the file to an independent reviewer in Ontario who felt the best that could be expected from the costly treatment is “some slowing of the progression” of the disease.

McFadyen’s own son has a different form of Morquio Syndrome. It is believed there are only 33 cases in Canada, and Morgan Doucet is the only known case in New Brunswick.

McFadyen said sufferers of Morquio Syndrome lack an enzyme in their blood that breaks down cellular waste in the body. The waste builds up in the bones, tissues, organs and muscles and leads to such symptoms as heart and airway disease, corneal clouding, stiffening of the joints, shortened stature, and premature death.

He said the treatment being denied to Morgan dramatically slows or halts the disease, improves pulmonary function, walking ability and gives patients the chance at a normal life.

The treatment currently is funded in Saskatchewan, Ontario and Quebec.

Source: https://www.telegraphjournal.com/telegraph-journal/story/45005525/health-minister-under-pressure

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

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.

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

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.

NB Liberals and Rare Diseases

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.

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

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

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

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

LIFE-SAVING TREATMENT APPROVED FOR SASKATOON SIBLINGS

Ministry of Health Reverses Funding Decision; Family Elated That “Hope Has Returned”

FOR IMMEDIATE RELEASE:  The Minister of Health in Saskatchewan has approved funding for a life-saving treatment required by three 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. After initially denying funding for the children, the Minister of Health ordered a review of the files and asked a panel of experts to provide feedback to the Ministry regarding the children and the treatment required.

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 lead to many devastating symptoms including heart and airway disease, corneal clouding, stiffening of the joints, shortened stature, and premature death.

Andrew McFadyen, Executive Director of The Isaac Foundation, an advocacy, research, and family support organization that specializes in MPS related diseases, was thrilled with the news. “I applaud the Minister for his leadership during this review, and thank him for the open and transparent way with which he conducted this review. This is life-saving for these children, and life-changing for their family. This is incredible news.”

Jamie Myrah, Executive Director of The Canadian MPS Society, also thanked the NDP Health Critic, Danielle Chartier, for her work bringing this issue to the legislature and for the advocacy she provided the family during what was a very emotional period.

McFadyen adds, “The Saskatchewan government has taken a leadership role on this and have come in line with the Canadian Expert Opinion and the International Treatment Guidelines for Morquio Syndrome. They have also mirrored the recent recommendations from the UK. Most importantly, they’ve given hope back to this family – hope for a new life and the chance at fighting back against this relentless disease. For that, I am incredibly thankful to Minister Duncan and his team.”

                                                                                                                         #  #  #

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

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 the 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. It was approved by Health Canada in July 2014, and has been 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.

"It's A Sin To Kill A Mockingbird"

Hi Everyone,

While I write this entry, I’m sitting on a plane heading back home.  It’s been a long and stressful week here in Regina, and I’m afraid we’re no closer to getting the help we need for our kids in Saskatchewan.

I started this week with a lot of Hope – we were promised a decision from the Ministry of Health within 7-10 days.  It’s a life or death decision, and one that shouldn’t be taken lightly.  Last Monday, the Minister of Health, Dustin Duncan, ordered a new review of the kids’ applications, and he promised an expedited review of that decision.  Since that time, he’s appeared on television and spoke mis-truths about the drug in question, misleading the public about how well the drug works and who it works best for.

Did the Minister intentionally lie about these things?  Only time will tell but personally, I don’t think he did.  In truth, I think the Minister was given incorrect information from the Ministry of Health – from the very bureaucrats that we’ve been talking to regarding these kids and this drug for over a year.  The information provided to the Minister was that this drug doesn’t work for kids over the age of 5, even though the only data that exists for how well the drug works was collected in children over 5.  In fact, of the 29 patients receiving this drug in the country, 27 of them are over the age of 5.  It was an error, and one that the Minister went in front of the cameras to repeat.

We put out a Press Release on Thursday to refute the claims the Minster made in public.  It was important for us to set the record straight so that the public fully understood the facts that surround this decision.  As an advocacy body, it would have been irresponsible of us to do otherwise.

Since that time, we have been hard-pressed to get any information from the Ministry or the Minister of Health about the status of the review, how long the review will take, or when Amir and his family can expect a decision on the future of their children.  Every time I called the Ministry for an update, I was told there was nothing new to tell me.  The Canadian MPS Society and The Isaac Foundation tried on numerous occasions to get updates and information and each and every time we were presented with the same message “We have nothing new to update.”  It was clear they were directed to keep us in the dark.

In an effort to meet directly with the Minister for an explanation, I flew to Regina.  I sat through question period each day, reached out to the Minister a few times each day, and politely requested an update on the kids’ files.  Our partners at the Canadian MPS Society have been just as ardent in their support of this family, and have also requested updates.

When I arrived at the Legislature on Tuesday, I brought with me a small gift for the Minister – a small card, my blog about Diefenbaker,  and a copy of To Kill A Mockingbird.  The message I inscribed was short.  It read:

Dear Minister Duncan,

I hope we get a chance to connect in person sometime today. In the meantime, I’m leaving you a copy of my favourite book and a blog I wrote a while ago from the grave of John Diefenbacker.

As you know, Mockingbird is a novel about courage, and doing what you know is right, no matter what the cost. It’s how I live my life each and every day, and I hope it gives you a glimpse into why I fight so hard for our kids.

In Mockingbird, Atticus says “Courage is not a man with a gun in his hand. It’s knowing you’re licked before you begin but you begin anyway and you see it through no matter what. You rarely win, but sometimes you do.”Our kids have been fighting battles – large and small – every single day of their lives. This fight for their only Hope shouldn’t be one of them.Warm Regards,

A.

image1-8It was a small gesture, but one filled with a message I felt was important to send.  It’s a message that hangs on my wall in my office, with the photo you see above.  In the end, it was an explanation on WHY we do what we do.  Since that time,  I’ve not been able to get any news on our kids, and the lack of information for us to share with Amir and his family is disheartening.  For the family, it’s cruel and heartless.  They go to sleep every night wondering if the next day is the day they will hear news about the future for their kids.  As Amir said, “It’s like Night and Night.  There is no shining anymore.”

On the way to the airport tonight, I started thinking about Mockingbird, and Atticus, and the message I was sending.  As many of you know, it’s my favourite story, and Atticus is someone I try to emulate as I work my own way through fatherhood, adulthood, and through this tough world of rare diseases.  I started thinking about what that book means to me, and the beautiful morals the story tries to bestow on its readers.

In reflecting on Mockingbird, I think I may have inscribed my message wrong for the Minister.  Instead of Atticus’ wisdom on what true courage is, I should have left him with another quote – the one where the novel takes it’s title from.  I should have left him with this:

“Shoot all the blue jays you want, if you can hit ’em, but remember it’s a sin to kill a mockingbird.”

His sister follows by saying “Mockingbirds don’t do one thing but make music for us to enjoy . . . but sing their hearts out for us. That’s why it’s a sin to kill a mockingbird.

It’s the key quote in the novel and for me it rings true each and every time I have to fight for what is right for our kids.

You see, our beautiful children are just like those Mockingbirds – they don’t do anything except bring us joy.  We put everything we have into our children – we see our Hopes in them, our dreams in them.  We’d do anything for them, even sacrifice ourselves for their survival.  They are the first thing we think of when we wake up, the last thing we think about before we go to bed.  For those of us up at night dealing with the rare disease our children are battling, it is them that keep us awake.  We live through them, laugh with them, and cry when they are hurting.  They are our everything – our joy and our lives.  They are, quite simply, our Mockingbirds.

When our kids have to fight for treatment – especially treatments that we KNOW work – it’s like the governments are out hunting Mockingbirds.  Once – just once I want these people to step back and hear their song.  I want Minister Duncan to truly hear them.  And when he does, I want him to take an objective look at the files that are before him – a look free of bias, of dollar signs, free of anger.  Because if he does, I know he’ll have the courage to save these kids.

I’ll keep you all up to date as things progress.  For now, I’m going to open up MY copy of Mockingbird, and try to enjoy the flight home.

With Love,

A.

“It’s A Sin To Kill A Mockingbird”

Hi Everyone,

While I write this entry, I’m sitting on a plane heading back home.  It’s been a long and stressful week here in Regina, and I’m afraid we’re no closer to getting the help we need for our kids in Saskatchewan.

I started this week with a lot of Hope – we were promised a decision from the Ministry of Health within 7-10 days.  It’s a life or death decision, and one that shouldn’t be taken lightly.  Last Monday, the Minister of Health, Dustin Duncan, ordered a new review of the kids’ applications, and he promised an expedited review of that decision.  Since that time, he’s appeared on television and spoke mis-truths about the drug in question, misleading the public about how well the drug works and who it works best for.

Did the Minister intentionally lie about these things?  Only time will tell but personally, I don’t think he did.  In truth, I think the Minister was given incorrect information from the Ministry of Health – from the very bureaucrats that we’ve been talking to regarding these kids and this drug for over a year.  The information provided to the Minister was that this drug doesn’t work for kids over the age of 5, even though the only data that exists for how well the drug works was collected in children over 5.  In fact, of the 29 patients receiving this drug in the country, 27 of them are over the age of 5.  It was an error, and one that the Minister went in front of the cameras to repeat.

We put out a Press Release on Thursday to refute the claims the Minster made in public.  It was important for us to set the record straight so that the public fully understood the facts that surround this decision.  As an advocacy body, it would have been irresponsible of us to do otherwise.

Since that time, we have been hard-pressed to get any information from the Ministry or the Minister of Health about the status of the review, how long the review will take, or when Amir and his family can expect a decision on the future of their children.  Every time I called the Ministry for an update, I was told there was nothing new to tell me.  The Canadian MPS Society and The Isaac Foundation tried on numerous occasions to get updates and information and each and every time we were presented with the same message “We have nothing new to update.”  It was clear they were directed to keep us in the dark.

In an effort to meet directly with the Minister for an explanation, I flew to Regina.  I sat through question period each day, reached out to the Minister a few times each day, and politely requested an update on the kids’ files.  Our partners at the Canadian MPS Society have been just as ardent in their support of this family, and have also requested updates.

When I arrived at the Legislature on Tuesday, I brought with me a small gift for the Minister – a small card, my blog about Diefenbaker,  and a copy of To Kill A Mockingbird.  The message I inscribed was short.  It read:

Dear Minister Duncan,

I hope we get a chance to connect in person sometime today. In the meantime, I’m leaving you a copy of my favourite book and a blog I wrote a while ago from the grave of John Diefenbacker.

As you know, Mockingbird is a novel about courage, and doing what you know is right, no matter what the cost. It’s how I live my life each and every day, and I hope it gives you a glimpse into why I fight so hard for our kids.

In Mockingbird, Atticus says “Courage is not a man with a gun in his hand. It’s knowing you’re licked before you begin but you begin anyway and you see it through no matter what. You rarely win, but sometimes you do.”Our kids have been fighting battles – large and small – every single day of their lives. This fight for their only Hope shouldn’t be one of them.Warm Regards,

A.

image1-8It was a small gesture, but one filled with a message I felt was important to send.  It’s a message that hangs on my wall in my office, with the photo you see above.  In the end, it was an explanation on WHY we do what we do.  Since that time,  I’ve not been able to get any news on our kids, and the lack of information for us to share with Amir and his family is disheartening.  For the family, it’s cruel and heartless.  They go to sleep every night wondering if the next day is the day they will hear news about the future for their kids.  As Amir said, “It’s like Night and Night.  There is no shining anymore.”

On the way to the airport tonight, I started thinking about Mockingbird, and Atticus, and the message I was sending.  As many of you know, it’s my favourite story, and Atticus is someone I try to emulate as I work my own way through fatherhood, adulthood, and through this tough world of rare diseases.  I started thinking about what that book means to me, and the beautiful morals the story tries to bestow on its readers.

In reflecting on Mockingbird, I think I may have inscribed my message wrong for the Minister.  Instead of Atticus’ wisdom on what true courage is, I should have left him with another quote – the one where the novel takes it’s title from.  I should have left him with this:

“Shoot all the blue jays you want, if you can hit ’em, but remember it’s a sin to kill a mockingbird.”

His sister follows by saying “Mockingbirds don’t do one thing but make music for us to enjoy . . . but sing their hearts out for us. That’s why it’s a sin to kill a mockingbird.

It’s the key quote in the novel and for me it rings true each and every time I have to fight for what is right for our kids.

You see, our beautiful children are just like those Mockingbirds – they don’t do anything except bring us joy.  We put everything we have into our children – we see our Hopes in them, our dreams in them.  We’d do anything for them, even sacrifice ourselves for their survival.  They are the first thing we think of when we wake up, the last thing we think about before we go to bed.  For those of us up at night dealing with the rare disease our children are battling, it is them that keep us awake.  We live through them, laugh with them, and cry when they are hurting.  They are our everything – our joy and our lives.  They are, quite simply, our Mockingbirds.

When our kids have to fight for treatment – especially treatments that we KNOW work – it’s like the governments are out hunting Mockingbirds.  Once – just once I want these people to step back and hear their song.  I want Minister Duncan to truly hear them.  And when he does, I want him to take an objective look at the files that are before him – a look free of bias, of dollar signs, free of anger.  Because if he does, I know he’ll have the courage to save these kids.

I’ll keep you all up to date as things progress.  For now, I’m going to open up MY copy of Mockingbird, and try to enjoy the flight home.

With Love,

A.

Medical treatment funding plea for children with rare genetic disease

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

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.

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