Dr. Durhane Wong-Rieger
President & CEO, Canadian Organization for Rare Disorders
What could the promised Pan-Canadian Rare Disease Drug Program mean for families living with rare diseases? It could mean that three-year-old Jason, diagnosed with neuroblastoma and whose cancer hasn’t responded after 18 months of chemotherapy, surgery, and radiation, could get access to a “rescue” drug combination. While it’s already reimbursed in the U.S. and Europe, it isn’t yet in Canada — making Jason’s only chance of acquiring it to be through donated product from the company.
A Pan-Canadian Rare Drug Program could mean that Zane, identified at birth with spinal muscular atrophy (SMA), a disease that destroys the motor neurons, could get immediate access to a therapy that replaces the faulty SMA gene. Given at birth, Zane wouldn’t develop irreversible muscle damage and would develop normal capacity to breathe, swallow, and move.
As importantly, a Pan-Canadian Rare Drug Program would encourage companies to launch innovative therapies and to place clinical trials in Canada because there’d be a supportive and timely pathway from research and development to access.
A plan for the future
After decades of advocacy, Canada’s rare disease community is being offered the opportunity of a lifetime — but time is short. In February 2019, the Canadian government committed $1 billion to a national Rare Disease Drug Strategy to be put in place in 2022. The Canadian Organization for Rare Disorders is pushing forward an ambitious multi-stakeholder consultation plan, established in October 2020 and continuing through the first half of 2021. The ultimate goal is to achieve consensus on a Pan-Canadian Rare Drug Program that is:
- A comprehensive national framework
- Integrated with a rare disease strategy including diagnosis, expert centres,community support, research, and drug access
- Realistic, feasible, sustainable, and beneficial for all Canadians
- Supported by all stakeholders
- Ready to go on Jan. 1, 2022