Communaute Morquio Quebec

November 24, 2015 16:12 ET

Unequal Access to Medication Denounced at National Assembly

Patient group calls for universal access to treatment for Morquio A syndrome while second Patient d'exception request is approved by RAMQ

QUEBEC CITY, QUEBEC--(Marketwired - Nov. 24, 2015) - Today, representatives of the Communauté Morquio du Québec delivered a message to Members of the National Assembly (MNA) on their concerns about the serious disparities in access to medication for the treatment of a rare and devastating disease. Individuals affected by Morquio A syndrome and their family members were received by MNA Sylvain Pagé (Parti québecois, Labelle) at the National Assembly, and delivered a petition signed by over 2,500 people that calls for immediate and universal access to VIMIZIM™ (elosulfase alfa) to treat those diagnosed by this condition. In welcoming the group, MNA Sylvain Pagé acknowledged the petition and reinforced its message with a Statement to the Assembly.

Access to treatment is a patchwork approach

Morquio A syndrome is an ultra-rare, severely debilitating and progressive disease that substantially limits both the length and quality of life of those affected by it. The condition impacts major organ systems in the body, and can lead to heart disease, skeletal abnormalities, breathing difficulties, and vision and hearing loss. Unfortunately, many Quebeckers impacted by this disease are not guaranteed access to the first and only available treatment that targets the underlying cause of Morquio A. Only patients who have private healthcare insurance, were enrolled in the manufacturer's clinical trials for the drug, or qualify under the Patient d'exception procedure of the Régie de l'assurance maladie du Québec (RAMQ) can benefit from this life-altering medication.

This patchwork approach to treatment coverage not only deprives some Morquio A patients from accessing a drug proven to be safe and effective, but also introduces uncertainty for those who are currently on treatment. For example, individuals are put at risk of having their VIMIZIM access terminated if they lose their private insurance coverage, or transition out of the access program linked to participation in clinical trials. Likewise, patients must work with their physicians to apply for coverage under the Patient d'exception procedure, and approvals are neither guaranteed nor long-term in nature. The only safeguard against this uncertainty would be for the Quebec government to list VIMIZIM on the public formulary, and provide publicly-funded access to all patients who may benefit from this treatment.

"Due to the severity and time-sensitive nature of this situation, our patient group came to the National Assembly today to implore provincial authorities to address the growing disparities in access to treatment for Morquio A syndrome," said Lucie Boudreau, President of the Communauté Morquio du Québec. "The clinical effectiveness of VIMIZIM has been recognized by Health Canada, and the drug is accessible to patients in a number of countries worldwide. Just yesterday, authorities in the United Kingdom reached an agreement to provide access to VIMIZIM treatment for the next five years. Quebec needs to follow this example and list the drug on the public formulary without delay. Only this will eliminate the patchwork approach we have now, and extend coverage to all our loved ones who so desperately need this treatment."

Lucie also stressed that though this message is best delivered in person, the Communauté cannot keep coming back to the National Assembly to emphasize the same points, year after year.

Hope exists for those with no other options

Ms. Laurie Patry-Pelletier, a young woman living with Morquio A syndrome, was also in attendance today at the National Assembly. Her story is one of hope and perseverance. Laurie was diagnosed with Morquio A at the age of six, and she struggled for years with debilitating pain that left her confined to a wheelchair for hours each day. Laurie enrolled in VIMIZIM clinical trials in 2012, and has since benefited greatly from the drug, becoming more independent and mobile as a result of her treatment.

Laurie's temporary access to VIMIZIM was set to end in early 2016, which was extremely concerning to her and her family. She was particularly worried that her condition would deteriorate without treatment, forcing her to again rely on her wheelchair to get around. Just when Laurie thought that all hope was lost, she found out earlier in November that her Patient d'exception application was approved for public reimbursement of VIMIZIM for a six-month period. This news came as a relief as Laurie had waited months for a decision.

"I can't begin to express how relieved I am to know that I will have access to VIMIZIM well into 2016. This drug helps me stay mobile, active and prevents my condition from worsening," said Laurie. "Though it's encouraging that I and others living with Morquio A can temporarily access VIMIZIM under the Patient d'exception procedure, it is not enough. It is important that the province provides all members of our community with long-term access to VIMIZIM by listing the drug on the public formulary."

About Morquio A Syndrome

Mucopolysaccharidosis IVA (MPS IVA), also known as Morquio A syndrome, is an ultra-rare genetic disease. People with this disease have a deficient enzyme (N-acetylgalactosamine-6-sulfate, or GALNS) that is essential to the breakdown and removal of chains of sugar molecules called glycosaminoglycans, or GAGs. The excessive storage of these molecules causes skeletal dysplasia, short stature and joint abnormalities which limit mobility and endurance. Malformation of the chest impairs respiratory function, and looseness of joints in the neck cause spinal instability and potentially spinal cord compression. Other symptoms may include hearing loss, corneal clouding and heart disease.

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