President & CEO, Canadian Organization for Rare Disorders
What is the definition of a rare disease and which drugs should be included under a Canadian Rare Drug Agency? CORD shares their vision for a pathway forward.
As a family at risk for hemophilia, you will receive genetic counselling, prenatal testing, and newborn screening. Upon diagnosis, you will be referred to a specialist with a team that will develop an individualized comprehensive care treatment plan for your infant that includes the right drug therapy. Importantly, he will be enrolled in a patient registry, his outcomes monitored, and alternative therapies considered when appropriate. As importantly, you will have the same access to all the same drugs with no co-pay regardless of where you live or your drug plan. Most importantly, you will have access to improved therapies, as they become available, such as slow-release drugs that you infuse twice a month instead of daily.
How did hemophilia, and almost no other rare disease, achieve this status? A major contributing factor was the tragedy in the 1980s when global contamination of the blood supply infected thousands with HIV and hepatitis C, including hundreds of Canadians living with hemophilia. A royal commission, lawsuits, and patient advocacy led to the establishment of publicly accountable, independent blood agencies, Canadian Blood Services (CBS) and Hema-Québec. (HQ) They oversee the purchase and distribution of therapies for hemophilia and some but not all rare blood disorders. It’s a model that has worked for two decades.
Most importantly, you will have access to improved therapies, as they become available, such as slow-release drugs that you infuse twice a month instead of daily.
As president of the Canadian Hemophilia Society during four commission years and a patient representative on the inaugural CBS Board of Directors, now as president of the Canadian Organization for Rare Disorders (CORD), I fashioned our vision of the National Rare Disease Drug Strategy on the CBS model. In February 2019, the federal government announced $1 billion for a rare disease drug strategy, and CORD began collecting opinion and expertise. In March 2021, in response to Health Canada’s consultation, CORD submitted a 12-step proposal, namely:
- patient engagement.
- Canadian Rare Drug Agency (CDRA).
- investment in research and development.
- accelerated access environment.
- Removal of pricing regulations that create barriers to innovative products.
- Regulatory process to encourage rare drug trials and submissions.
- Access pathways for urgent needs.
- Multiple funding models.
- Managed access programs to address data uncertainty.
- Concurrent Health Canada and HTA reviews.
- Real-world evidence.
- Networked centres of expertise.
What is the definition of a rare disease and which drugs should be included under a Canadian Rare Drug Agency? Currently, not every therapy for hemophilia is under CBS/HQ mandate nor is every CBS/HQ therapy used solely for “blood” conditions. Similarly, not every therapy for a designated rare disease should be managed by CRDA, nor should all products for all “rare” diseases be procured, reviewed, and managed through CDRA procedures. A primary consideration is the urgency of need and uncertainty in the evidence to assess benefits and risks, Finally, what are the potential relationships among the Canadian Rare Drug Agency and Canadian Blood Services/Hema-Québec? Broadly speaking, if It ain’t broke, don’t fix it. If CBS/HQ are managing a condition or a therapy, it should continue to do so. The best-case scenario is the capacity for mutual learning and support among all agencies.