Accelerating Canada’s Rare Disease Drug Strategy, Post-Election

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

Both the federal and provincial governments should share a common concern that underlies a national rare disease drug strategy, namely, to manage the budgetary impact. Payers express concerns about the “proliferation” of rare disease drugs, as there have been more than 700 orphan drugs — drugs developed for extremely rare or “orphan” diseases — approved in the U.S. and over 200 in the European Union — albeit considerably fewer in Canada, where there has never been an Orphan Drug Act to support the development of rare disease drugs. Most orphan drugs represent the first approved treatment for a specific rare condition, while some are older therapies that have been repurposed for a rare condition or modified formulations of older therapies. Most are novel drugs, many working at cellular, molecular, and genetic levels, like mRNA or CAR T-cell therapies, to treat all types of rare diseases including blood and bleeding disorders, neuromuscular and neurodegenerative conditions, and metabolic conditions.

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.