This Rare Disease Day, Canadian families living with rare disease have much to celebrate — and even more to look forward to

Through the Rare Disease Drug Strategy (RDDS), patients with serious and life-threatening rare diseases are gaining faster-than-ever access to innovative therapies.

For those living with rare conditions, time matters. Earlier access can reduce the risk of irreversible organ or tissue damage, preserve function, slow or stabilize disease progression, prevent relapses and, in many cases, prolong survival. The nine therapies currently on the RDDS common list — with more to come — are not incremental advances. They are breakthroughs: safer, more effective treatments and, in some cases, options where none previously existed.

Faster and more equitable access also creates opportunity. As patients across Canada begin treatment earlier, health systems are better positioned to observe, measure and understand the full impact of these innovations. The next phase of RDDS must focus on collecting real-world evidence that documents how these therapies improve outcomes, extend lives, reduce complications and enhance quality of life.

The common thread across RDDS therapies is clear: each addresses serious unmet need and delivers meaningful patient value. Demonstrating that value clearly and credibly will help ensure sustained and equitable access across the country.

Similarly, Ontario’s Funding for Accelerated Supportive Therapies (FAST) program reflects a broader evolution in policy thinking — that timely access and evidence generation can proceed together. While not yet applied to rare-disease drugs, FAST signals how complementary accelerated pathways could further strengthen national progress.

On Rare Disease Day, we celebrate momentum — and commit to sustaining it. RDDS has shown that faster access is possible when patients are placed at the centre of decision-making. The task now is to ensure that this progress endures, so that timely access to life-changing therapies becomes standard practice, not a rare exception.

{{CTA}}