Ultra-rare disease community hopeful after first patient receives public funding for life-changing medication
Quebecers with Morquio A urge province to provide immediate, long-term coverage of VIMIZIM for all patients
MONTREAL, May 15, 2015 /CNW Telbec/ - Today, patients and families affected by Morquio A syndrome across Québec are recognizing International MPS (Mucopolysaccharidosis) Awareness Day with a glimmer of hope: the Régie de l'assurance maladie Québec (RAMQ) recently approved funding of VIMIZIM™ (elosulfase alfa) – the first and only available treatment that addresses the underlying cause of the disease – for one patient in the province. Although this funding decision represents a positive outcome for one individual, the Morquio A community in Québec is now intensifying its efforts to ensure that all people touched by this disease receive publicly-funded access to the life-changing treatment they so desperately need.
"I am thrilled that the RAMQ approved my daughter Maude's application for access to VIMIZIM. I am sure it will have a deep and lasting impact on the treatment of her condition and her quality of life," says Dominique Auclair, mother of Maude, a ten year old girl living with Morquio A syndrome. "I am grateful to provincial authorities for approving my daughter's access to this life-changing drug, and we both urge the government of Québec to mark MPS Awareness Day by facilitating immediate access to VIMIZIM for all Morquio A patients who could benefit from this treatment."
A devastating disease with a revolutionary treatment
Morquio A is an ultra-rare, severely debilitating and progressive disease that substantially limits both the quality and length of life of those affected by it. There is no cure. Every day, Quebecers living with Morquio A face seemingly insurmountable health challenges that impact their mobility, lung and heart function, daily living activities and life-expectancy. Until recently, only supportive care was available to Morquio A patients to treat the symptoms of their disease, including: medication, multiple surgeries, and ongoing occupational physical therapy.
Approved by Health Canada in July 2014, VIMIZIM is an enzyme replacement therapy that is designed to target the underlying cause of Morquio A and slow its progression. Deemed to provide clinical benefit to patients, VIMIZIM was considered for approval under Health Canada's Priority Review process for drugs that offer major advances in treatment, or provide treatment where no adequate therapy exists.
First Morquio A patient in Québec receives funding for life-altering treatment…
Last month's RAMQ decision to fund VIMIZIM through the Patient d'exception procedure for Maude Leclerc is a positive development for those living with this debilitating and progressive disease.
Patient d'exception recognizes the exceptional circumstances under which individual patients benefit from a particular drug that is not listed on Québec's approved Liste de medicaments, or where a drug on this list has not been approved to treat specific ailments. If the conditions for Patient d'exception are met, a patient can access treatment under provincial health insurance. This is significant as most patients and their families are unable to carry the costs of treatment by themselves. The cost of treatment with VIMIZIM is comparable to other life-changing therapies for ultra-rare diseases.
…while others are left to deteriorate without publicly-funded access
Unfortunately for members of the broader Québec Morquio A community, public funding for VIMIZIM has not yet been put in place. Those who rely on public drug plans were disappointed with the February 2015 evaluation by the provincial Institut national d'excellence en santé et en services sociaux (INESSS). In its recommendation to the Québec Minister of Health and Social Services, INESSS did not recommend placing VIMIZIM on the provincial Liste de medicaments.
The negative INESSS recommendation means that Morquio A patients in Québec will not be able to access VIMIZIM unless they have private insurance that will cover the treatment. Though patients may work with their doctors to submit applications to RAMQ for funding under the Patient d'exception program, this can create a patchwork of inconsistent coverage where funding decisions are individualized and open to arbitrary assessment.
Long-term funding for life-changing medication urgently needed
Now that the Québec government has set a precedent and made VIMIZIM accessible to one Morquio A patient through public funding, it is vital for the province to provide widespread and long-term coverage of this important treatment to all those who may benefit.
"While we are encouraged to see that one patient is receiving publicly-funded access to VIMIZIM, it is critical that the provincial government ensures there is immediate access to this life-changing treatment for all Quebecers living with this devastating disease," says Gail Ouellette, President and Director General, Regroupement Québécois des Maladies Orphelines. "The longer this decision is stalled, the more patients' disease will progress and their health will decline. Immediate action is required to improve the lives of Morquio A patients in Québec – their lives can't wait."
For further information on Morquio A syndrome and what you can do to help, please visit www.mylifecantwait.ca.
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. Initial symptoms often become evident in the first five years of life. The disease substantially limits both the quality and length of life of those affected.
About RQMO
The Regroupement Québécois des Maladies Orphelines (RQMO) regroups 26 rare disease associations and individuals living with rare diseases, as well as family members and caregivers. RQMO's mission is to inform and educate while providing support to patients suffering from rare and orphan diseases, as well as their families and health professionals, through a Information and Resource Centre for Rare Diseases (www.rqmo.org). RQMO also works to advance knowledge and research by facilitating collaboration between patients and researchers.
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References: |
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1: Medline Plus. U.S. National Library of Medicine – National Institutes of Health. Morquio syndrome. Available at: www.nlm.nih.gov/medlineplus/ency/article/001206.htm. Accessed on May 5, 2015. |
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2: The Canadian Society for Mucoploysaccaride & Related Diseases Inc. A Guide to Understanding Mucopolysaccharidosis (MPS) IV. |
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3: Borlot et al. Mucopolysaccharidosis type IVA: Evidence of primary and secondary central nervous system involvement. American Journal of Medical Genetics Part A. Available at: http://onlinelibrary.wiley.com/doi/10.1002/ajmg.a.36424/abstract. Accessed on May 5, 2015. |
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4. Regier, Debra S et al. Mucopolysaccharidosis Type IVA. Available at: www.ncbi.nlm.nih.gov/books/NBK148668/. Accessed on May 5, 2015. |
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5. Health Canada. VIMIZIM (Drugs and Health Products). Available at: http://www.hc-sc.gc.ca/dhp-mps/prodpharma/sbd-smd/drug-med/sbd_smd_2014_vimizim_170340-eng.php#sbd Accessed on May 5, 2015. |
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7. VIMIZIMMC – Maladie de Morquio de type A. INESSS. February 2015. Available at: http://www.inesss.qc.ca/fileadmin/doc/INESSS/Inscription_medicaments/Avis_au_ministre/Fevrier_2015/Vimizim_2015_02_CAV.pdf Accessed on May 5, 2015. |
SOURCE Regroupement québécois des maladies orphelines
please contact: Olivier Gagnon, Cohn & Wolfe, (514) 845-2257, ext. 7087, [email protected]
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