Reflections From Canada's Rare Disease Summit

durhaneToday’s guest blog was written by Durhane Wong-Rieger. Durhane currently serves as the President of the Canadian Organization for Rare Disorders, Chair of the International Alliance of Patients’ Organizations, Chair of the Consumer Advocare Network, and President of the Institute for Optimizing Health Outcomes.

Inspiring discussions grounded in realism. That’s how I would describe last month’s Summit on Access to Drugs for Rare Diseases in Canada that brought together 29 rare disease experts to discuss a path forward for a groundbreaking Access Framework that would dovetail with the pending Canadian Orphan Drug Regulatory Framework. The conversation at the event focused on concrete, realistic actions that our country can take to improve access to treatment for the almost 3 million Canadians who suffer from a rare disorder.

Currently, only 60% of rare disease treatments make it into Canada and many provincial drug plans don’t provide coverage for these new medicines. As I discussed previously, Canada is uniquely positioned to set the global standards for the treatment of rare diseases. By building upon what we already have in place and seeking guidance from other countries providing pathways tor the treatment of rare diseases, Canada can create a system that ensures people with rare diseases have the same opportunities for safe, effective treatments as those affected by more conditions.

Throughout the two-day summit, we deliberately created an environment that sought to recognize the challenges we face while looking for solutions that would actually work. These three components shaped our thoughts on how to find success with this framework:

  • Managed Access Approach – The general consensus was that this method would be our best way forward, building an infrastructure that supports continued access and standardizes existing practices across the country. Canada would be the first country to have a national drug program for rare diseases that is based on managed access, giving our country a chance to truly lead in this arena.
  • Patient Centered – A rare disease, by definition, affects fewer than 1 in 2,000 persons.  However, there are 7,000 rare diseases, with considerable variable across and within diseases, which means we must move away from the “one size fits all” mentality in order to create a patient-centred approach that would allow for individualization of treatment within a structured process.
  • Learn From Existing Experiences – International experts from Italy, the UK, and Spain attended the summit and provided insights from experiences in their home countries. We need to build on what has worked here and around the world to avoid reinventing the wheel.


A managed access plan will not be appropriate for every rare disease drug nor will it ensure that every patient who should get access will. However, properly implemented, managed access will move us much closer to our ultimate goal: to ensure Canadians with rare diseases never experience any unnecessary delay in gaining access to clinical trials or new therapies, relative to the Europeans and Americans. Indeed, if we maintain the course we have started, Canada could become a world leader in developing a state-of-the-art framework for rare diseases.

To provide more information about managed access as a proposed framework, CORD has just hosted two public webinars on the recommendations from the Canmore Summit  You can access them on the CORD website.

We have also set up a survey and ask you to share your personal opinion about the proposed framework.  The compiled results will be included in our submission to the Health Ministers meeting being held at the end of September.  Your input is very important. Please go to: