Dr. Durhane Wong-Rieger
President & CEO, Canadian Organization for Rare Disorders
For the past year, the Canadian Organization for Rare Disorders (CORD) has committed itself nonstop to the mission of forging a rare disease drug strategy that works for all Canadians and that simultaneously addresses the unique and sometimes disparate priorities of various stakeholders.
CORD is determined to continue its trajectory toward implementing Canada’s Rare Drug Strategy in 2022, as was promised in 2019 and reiterated in 2020 and 2021 by the federal government. Over the past year, there has been a roadmap developed to optimize access to therapies for Canadians with rare diseases, and while we’re not yet there, the progress, cooperation with stakeholders, and momentum seen gives us confidence that this goal is well within reach.
Commitment to progress
Most impactful are the “durable” gene replacement, modification, or editing therapies. These highly-advanced therapeutic products also tend to cost more. In reality, the number of eligible patients is still very small, and the impact on overall drug expenditure remains very small (two percent of drug budgets in Canada) but nevertheless, the “sticker shock” of an individual therapy has been enough to send payers into apoplexies and predictions of health budget bankruptcy.
Beginning last September, CORD hosted over 25 multi-stakeholder consultations to solicit ideas and to engender consensus toward an optimal strategy for optimizing access and cost-effectiveness of rare drug treatments. In January to March, the federal government hosted a series of stakeholder meetings and in its report, arrived at just about the same position as did CORD. Agile and adaptable regulatory pathways should be coordinated with early and appropriate assessment for reimbursement that includes “managed access” to allow for forms of “coverage with evidence developing” and “outcome-based agreements” to collect patient evidence in “real-world” use to inform appropriate access and value of therapies.
CORD is continuing its series of consultations immediately post-election this fall, so by the time the new government gets sorted out, we’ll be ready with our plan for collaborative action toward the spring 2020 initiation of Canada’s rare disease drug strategy.