e-2444 (Health)

Petition to the Minister of Health

• Spinal muscular atrophy (SMA) is a rare disease that attacks the body’s muscles and affects one out of every 8,000 to 10,000 people worldwide;
• Type 1 specifically is the most severe, diagnosed prior to age one;
• There is a drug, Spinraza, that alleviates symptoms of SMA to an extent but is not a cure and requires ongoing and expensive medication for life;
• A possible cure, a gene therapy called Zolgensma, produced by the pharmaceutical company Novartis, is approved for sale in the United States;
• The cost of a one-time dose is a prohibitive $2.8 million and therefore completely out of reach for a middle class family;
• The federal government committed in 2019 to work with provinces and territories to develop a national strategy for high-cost drugs for rare diseases;
• Health Canada's Special Access Program “may provide access to a drugs (for serious or life-threatening conditions) that cannot otherwise be sold or distributed in Canada”; and
• The program’s guidelines require that “conventional therapies have been considered and ruled out, have failed, are unsuitable, and/or unavailable” in order to be eligible.