treatment denied

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

HEALTH MINISTER REFUSES TO HEAR EXPERT OPINION REGARDING DENIAL OF LIFE-SAVING MEDICATION FOR MORGAN DOUCET

Sticky Post By On 17 December, 2015

Minister Refuses To Allow Renowned MPS Expert To Join Family Meeting; Doucet’s Family In Fredericton to Meet with Child And Youth Advocate Thursday

The family of ailing 10-year-old Morgan Doucet has cancelled a scheduled meeting with the Minister of Health in New Brunswick after the Minister refused to allow an internationally renowned expert to participate in the discussions. Doucet’s case has made headlines throughout the Province after the Minister of Health denied access to the life-saving drug that he requires. Morgan suffers from MPS IVA (Morquio Syndrome) and requires the life-saving treatment immediately in order to halt further progression of his devastating disease. Without access to therapy, Morgan faces a long and painful decline and death at a young age.

Andrew McFadyen, Executive Director of The Isaac Foundation, an advocacy, research, and family support organization that specializes in MPS related diseases, has slammed the Minister for his refusal to meet with one of the world’s foremost experts in MPS and related diseases. “It’s unconscionable to me that Minister Boudreau would refuse to allow this expert to participate in the meeting he had scheduled with Morgan’s family. Unbelievable, really. He has denied Morgan access to the life-saving medication he needs, hasn’t allowed his family an avenue to appeal his ruling, and then refuses to hear from a true expert that deals with this disease on a daily basis – an expert who is internationally recognized as the best in his field. It leads me to believe that he’s afraid to be proven wrong on such an important issue, which is deplorable because a life hangs in the balance. Pride and politics should never play a role in the health and well being of a child, and it appears that’s exactly what is happening with Morgan Doucet with respect to the Minister’s steadfast refusal to hear more evidence on this matter.”

Doucet’s parents will be joined McFadyen at the provincial legislature in Fredericton on Thursday, December 17 to watch the morning legislative session and to meet with the province’s Child and Youth Advocate, Norman Bosse. PC Jake Stewart, the Doucets’ MLA, will join the family during that meeting.

Bosse has agreed to look into the process used to deny access to Morgan’s medication, a process that McFadyen says was destined to return a negative decision all along. He notes that after an initial application for treatment in October of 2014, the file was finally sent to Ontario for review by one individual alone, with no opportunity allowed for a rigorous discussion with the rest of the MPS Experts throughout Canada and Internationally –experts that deal with the disease on a daily basis and some of whom have seen first-hand how well other children receiving the treatment are doing.

“The Minister and the Ministry’s only comment on this case is that the drug didn’t get a positive review from the Common Drug Review (CDR). What he doesn’t say is that all drugs used to treat other MPS diseases have received negative CDR reviews and the Province pays for access to all. Right now, Morgan is the only MPS patient in the province who has been denied this coverage, and that’s disgraceful.”

Health Canada approved the treatment Morgan requires in July of 2014 and it has been recommended for use in patients by the Canadian Expert Opinion on Morquio Syndrome. In addition, the International Expert Opinion on Morquio Syndrome considers the treatment their gold standard of care for patients throughout the world. It is reimbursed for patients in Saskatchewan, Ontario, and Quebec.

In Canada, there are 33 patients receiving the treatment. According to McFadyen, all of them are doing exceptionally well. “These patients have seen dramatic improvements and have had their disease progression either halted or dramatically slowed down. Morgan deserves the same chance at life that those other patients have. This is Canada – where equal access to our health care system isn’t a privilege, it’s a right.It’s callous and cruel that the Minister of Health is forcing this child and this family to enter a palliative approach to his disease when we know he can be helped. We know he can be saved.”

Jamie Myrah, Executive Director of the Canadian MPS Society, a national patient association that serves those affected by MPS and related lysosomal diseases, says Doucet’s case has angered and galvanized the people of New Brunswick. Doucet was front-page news on Monday, and has been featured by numerous provincial and national media outlets since his treatment was denied. The Isaac Foundation’s online posts about Morgan have received over 100,000 visitors during that time, and hundreds of emails have been sent to the Premier and the Minister of Health supporting access for Morgan. McFadyen has posted an online form that the people can fill out and all messages get delivered instantly to the Minister’s and the Premier’s email inbox.

“People in New Brunswick and across Canada are understandably upset about this decision and they want their voices heard,” says Myrah.

McFadyen finishes, “We won’t accept the Minister making medical decisions and overruling and contradicting medical experts, clinicians, and researchers regarding best practice and treatment for Morgan Doucet. If he won’t look at the clinical expert opinion, the patient reported outcomes or the peer-reviewed published journals, then who will he listen to?”

Both The Isaac Foundation and The MPS Society are renewing their call for Health Minister Victor Boudreau and Premier Brian Gallant to take action, review and reverse their initial decision, and to ensure that treatment begins immediately for Morgan Doucet.

# # #

The Doucet Family and The Isaac Foundation will be available for interested media at the Legislature on Thursday, December 17. 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.

 

BACKROUNDER  

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

 

Photo Source:  CBC.ca

10 Dec 2015
Sticky Post By Posted in Advocacy, Blog Postings, Morquio, MPS IVA Permalink

LIFE-SAVING TREATMENT DENIED FOR AILING NEW BRUNSWICK CHILD

Sticky Post By On 10 December, 2015

FOR IMMEDIATE RELEASE

PDF Version – www.theisaacfoundation.com/nbtreatment.pdf

Ministry of Health Denies Funding; Premier and Minister of Health Refuse to Allow Appeal; Treatment Already Being Funded in Saskatchewan, Ontario and Quebec

The New Brunswick Ministry of Health has denied funding for a life-saving treatment required by an ailing 10-year old Baie-Sainte-Anne child. Morgan Doucet suffers from MPS IVA (Morquio Syndrome) and requires the life-saving treatment immediately in order to halt further progression of his devastating disease. The Isaac Foundation and The Canadian Society for Mucopolysaccharide & Related Diseases (Canadian MPS Society) are calling on Health Minister Victor Boudreau and Premier Brian Gallant to take action, review and reverse the decision, and ensure that treatment begins immediately.

Without access to therapy, Morgan faces a long and painful decline and death at a young age. The Isaac Foundation met with Minister Boudreau on December 9, 2015 but the Minister declined to make any commitments regarding an appeal for Morgan. The family has publicly given permission for the Minister to comment on Morgan’s application and condition so that the Minister can be forthcoming with the public about why he isn’t allowing an appeal.

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. 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. It is currently being funded in Saskatchewan and for patients in Ontario and Quebec.

Andrew McFadyen, Executive Director of The Isaac Foundation, an advocacy, research, and family support organization that specializes in MPS related diseases, is disappointed by the decision and is urging an immediate reversal to save the Morgan’s life. “The decision to deny this life-saving treatment for Morgan is outrageous, heartless, and cruel. This 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. To prevent this child from receiving the care he requires is needless and callous, and shows a reckless disregard for what our Canadian Health Care System purports to stand for.” He adds, “The Ministry is telling this family to enter a long-term palliative approach to care for their child instead of ensuring that disease progression is halted now. They are allowing him to die, instead of taking leadership and doing the responsible and ethical thing by providing him with the help he needs. It’s shamefully disgraceful.”

McFadyen is puzzled as to why access for treatment was denied, especially considering the weight of available evidence and Canadian and International expert guidelines urging it’s approval. In addition, the Province already provides access to patients suffering from every other form of MPS requiring the same kind of treatment. He expects it comes down to financial considerations due to the high cost of the drug, coupled with a recent negative recommendation from the Common Drug Review (CDR), that has clouded the judgment of decision makers at the Ministry.

Jamie Myrah, Executive Director of the Canadian MPS Society, a national patient association that serves those affected by MPS and related lysosomal diseases, notes that there were serious issues with the initial review that denied Morgan the treatment he needs.   “The initial review was done by a reviewer who has never used this treatment, using the flawed CDR report as the basis for his decision. This recently happened with three children in Saskatchewan. However, when the Minister realized the review for those files was flawed, he ordered a new review using real experts dealing with this disease and this treatment. He chose International experts to take a second look at the files and those experts came back with a decision to treat. We feel the Premier should also take a second look at Morgan’s file. We’re not asking for much – just a second look at this decision. Morgan’s life depends on it and I would hope the Premier and the Minister of Health would want to ensure they get things right.”

McFadyen also questions the process used by the New Brunswick Ministry of Health in making their decision, noting the family first requested access in October of 2014. He notes that in March of 2015, the file was finally sent to Ontario for review by one individual alone, with no opportunity allowed for a rigorous discussion with the rest of the MPS Experts throughout Canada –experts that deal with the disease on a daily basis and some of whom have seen first-hand how well other children receiving the treatment are doing. “The only thing stopping this child from beginning his treatment is the lack of funding by the Province. It’s incredulous to me that one individual gets to pick and choose who does or doesn’t get access to the life-saving treatment they need, especially considering the fact that a precedent has been set with other children in this province and in other provinces across the country receiving access to similar medications. One individual is making life-altering decisions on behalf of this Ministry and the people of New Brunswick, and it’s led to Morgan’s life being left in peril.”

Myrah adds, “We know this treatment works and the Province knows that ERT’s for MPS diseases are currently the only chance patients have of living longer, healthier lives. We recognize that the CDR gave this treatment a negative recommendation, but it did so for all the other MPS ERT’s as well; yet every province in the country – including New Brunswick – still ensures access for those patients. While ERT treatment for MPS is expensive, it is often just as expensive – if not more expensive – to not treat these kids. It simply doesn’t make sense to make these children suffer a slow and painful decline when we could stop or dramatically slow the progression of the disease with the treatment being requested.”

Both Myrah and McFadyen are hopeful that a review and a reversal of this decision can take place immediately, and they are urging the Health Minister and the Premier to take action now. Says McFadyen, “Brian Gallant’s government has the opportunity to do the right thing and ensure that Morgan receives the treatment he so desperately needs. It’s the role of government to protect and ensure fair and equitable access to Health Care for all people in New Brunswick, regardless of whether they are impacted by a rare disease or not. This family needs to concentrate on treating their children’s disease, not wading through a bureaucratic maze of paperwork and pinning their hopes on the appeals process or the recommendation of one person alone –a person who has never used this therapy or seen it’s effectiveness – at the Ministry of Health. We strongly call on this government to take action and save the life of this little boy now. He can’t afford to wait.”

# # #

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.

 BACKROUNDER  

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.

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.