Canada must move quickly for a successful rare disease strategy

In March 2023, Canada made a funding announcement for its national rare disease drug strategy, including $1.5-billion in new investments over three years. Canada’s commitment to enhancing public coverage for needed medications nationwide was welcomed by the rare disease community. The strategy, however, requires some work as it is missing a few key pieces to ensure the 3.2 million Canadians living with rare diseases get faster access to the treatments they need. Both Europe and the United States’ rare disease strategies include definitions, sometimes called “orphan drug designations.” Canada’s does not, although Health Canada officials have indicated they will discuss what the criteria might look like with each of the provinces to determine a “small set of new and emerging drugs.” They also plan to review the definitions used in other jurisdictions to guide this work. Once there are agreed-upon criteria, the government will work with the provinces to develop a limited list of drugs that will be covered by the federal funding program. That will be an important next step, with governments needing to weigh the considerable risks of developing a national formulary that is too restrictive. While details are being ironed out, the goal should be to ensure this funding leads to more rare disease patients having access to more treatment options than they have today. In the end, it should lead to more patients being treated, full stop. We are hopeful the federal government will move quickly to secure bilateral deals with the provinces. This recent funding announcement and the associated actions to be taken are sure to be on the Council of the Federation’s upcoming agenda this July in Winnipeg. If so, we could see positive movement over the next few months. Moving quickly is essential as two-thirds of Canadians with rare diseases are children. Some of the funding Canada has allocated could lead to improvements in newborn screening and genomic testing. This is incredibly important for early diagnosis and treatment, which significantly improve outcomes for many young patients. Gaby Bourbara is the general manager of Alexion Canada. Photograph courtesy of Gaby Bourbara Even while accelerating efforts, Canada can get this right. A successful rare disease strategy must include five things: enhanced funding for new and existing therapies, the establishment of an orphan drug designation, accelerated reviews and approvals of new drugs, data-sharing across hospitals and research facilities, and enhanced intellectual property protection for rare disease medicines. Canada has become a leader in other therapeutic areas when the government has collaborated with stakeholders to deliver world-leading outcomes, and it should do that again. Even before the federal government announced this new funding for its rare disease strategy, Alexion Canada announced a major expansion of our footprint in Canada, including the establishment of a new research and development hub for rare diseases in Mississauga, Ont. Our new hub will play a critical role in the development of new and innovative medicines. We have been developing innovative therapies for rare diseases for more than 30 years, and we chose to make this investment here because we believe Canada is poised to become a leader in the development and adoption of novel rare disease treatments. The announcement of the strategy reaffirmed to us that Canada is on the right track, but it is imperative that we maintain momentum and get this done right for patients who have few, if any, options. This is an important moment for Canada to maximize this funding announcement and make its national rare disease strategy the gold standard for patient access and outcomes. We have the opportunity to leverage Canadian-led research and development to help find the answers millions of people are looking for. And we can show the world Canada is integral to finding solutions for the future of rare disease research and development. We are almost there. With a few key steps, and by working together with industry, patient advocates, and health-care practitioners, the Government of Canada can get it done. Gaby Bourbara is the general manager of Alexion Canada. The Hill Times