e-2444 (Health)
- Keywords
- Infants
- Pharmaceuticals
- Special Access to Drugs and Health Products Program
- Spinal muscular atrophy
Original language of petition: English
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.
- Open for signature
- February 24, 2020, at 3:40 p.m. (EDT)
- Closed for signature
- March 25, 2020, at 3:40 p.m. (EDT)