The Saskatchewan Ministry of Health has denied 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. The Isaac Foundation and The Canadian Society for Mucopolysaccharide & Related Diseases (Canadian MPS Society) are calling on Health Minister Dustin Duncan and Premier Brad Wall to take action, review and reverse the decision, and ensure that treatment begins immediately. Without access to therapy, Sara, Khadija, and Muhammad face a long and painful decline and death at a young age. The Isaac Foundation and the Canadian MPS Society will meet directly with Minister Duncan at 11:00 am on Monday, October 05th to urge the Minister to intervene and save the children.
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.
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 lives of the children. “The decision to deny this life-saving treatment for these children 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 these kids from receiving the care they require is needless and callous, and shows a reckless disregard for what our Canadian Health Care System purports to stand for –a health care system that was ironically born in this very Province.” He adds, “The Ministry is telling this family to enter a long-term palliative approach to care for their children instead of ensuring that disease progression is halted now. They are allowing them to die, instead of taking leadership and doing the responsible and ethical thing by providing them with the help they need. It’s shamefully disgraceful.”
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.
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, couldn’t agree more. “We know this treatment works and the Province knows that ERT’s for MPS diseases are currently the only chance patients have of living long, healthy 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 Saskatchewan – 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.”
Naturally, the Akhter family has been devastated by the diagnosis of their kids. “Since receiving these diagnoses, we’ve been sick with guilt, grief, and worry. Our world will never be the same,”said the siblings’ father, Amir. “The only comforting factor since receiving this news has been the knowledge that a treatment is available to help my kids. We want to be able to do everything we can for our children to give them everything they deserve in this world, including a fair shot at a healthy life. We’ve now lost hope –our kids have been handed a death sentence by this government. This disease is not their fault.”
McFadyen questions the process used by the Saskatchewan Ministry of Health in making their decision, noting the family first requested access in March of 2014. He notes that in August 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 these children from beginning their treatments 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 the medication. One individual is making life-altering decisions on behalf of this Ministry and the people of Saskatchewan, and it’s led to these kids lives being left in peril.”
He is hopeful that a review and a reversal of this decision can take place immediately, and both he and Myrah are urging the Health Minister and the Premier to take action now. Says McFadyen, “Brad Wall’s government has the opportunity to do the right thing and ensure that Sara, Khadija, and Muhammad receive the treatment they so desperately need. It’s the role of government to protect and ensure fair and equitable access to Health Care for all people in Saskatchewan, 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 these children now. They can’t afford to wait.”
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For more information about this topic, or to schedule an interview with Andrew McFadyen, please call Andrew at 613-328-9136 or email Andrew at mcfadyena@me.com. The Isaac Foundation can also arrange interviews with parents of patients currently receiving this treatment in Canada.
