President & CEO, Canadian Organization for Rare Disorders
An effective rare disease drug strategy for Canada will require infrastructure and research investments, alternative managed access pathways, and more.
In 2019, the federal government committed $1 billion to invest in a rare disease drug strategy with an ongoing investment of $500 million annually. Following three years of nationwide multi-stakeholder consultations, the Canadian Organization for Rare Disorders (CORD) is proposing a three-pillar rare disease strategy to deliver optimal value to patients, the health care system, and society.
Recommendation 1 is to invest in infrastructure for early and accurate diagnosis, access to specialists, and patient-centered management.
Recommendation 2 is to invest in alternative managed access pathways to deliver drug therapies as soon as possible in order to achieve optimal patient outcomes.
Recommendation 3 is to invest in Canadian research on rare drug development, clinical trials, and outcomes monitoring.
Pushing for change
Orphan drugs are, by definition, either the first or a significantly improved therapy for a rare disease that is also severe, progressive, or life-threatening. About 80 per cent of rare diseases have a genetic origin and many transformative therapies are based on understanding the gene and reducing its impact. But to achieve maximum benefits, we need to diagnose and treat patients as early as possible, preferably before they’re symptomatic. Therefore, to optimize value from a rare disease drug strategy, we need to invest in infrastructure, namely, a Canadian rare disease expert network that includes centres of expertise, clinicians and researchers, and community providers. The goals of this network are to provide rare disease patients with access to early, accurate diagnosis and referrals to specialists, regardless of where they live in Canada.
In parallel, we must invest in an accelerated drug access process from early clinical trials to regulatory approval to real-world access with real-world patient monitoring. As the federal government moves to announce the launch of the rare disease drug strategy, potentially by the end of March 2023, CORD looks forward to working with all the stakeholders on implementation.