Our Patient Advocate Katie Allen has participated in this CORD conference on the 29th and 30st of April 2026 on behalf of our organisation.
Day one of the conference started with a panel addressing questions about where we have come since the announcement of the rare disease drug strategy, and continued to look at the past and present. Whereas day two looked forward to questions about where we go now, and whether we are ready for the changing landscape of not just Canada, but the globe. Overall, it was a great conference with a variety of speakers; it felt like this year, a real effort was made to reach as many provinces as possible, which was great to see, as some conferences have been very Quebec, Alberta, and Ontario-focused.
The first big question the first panel grappled with was how Canada will integrate Real-World Evidence (RWE) into decision-making, and, further, in whose decision-making will it be integrated; will provinces use it them same way as the federal government, or will there be 14+ different systems in place. Health Canada is currently using three case studies attempting to answer the questions of effectiveness and how to utilize that data, versus how to address when the data itself is less reliable (i.e., self-reporting). Further, how do you expand all of these studies into the future and into drugs we don’t know yet for diseases we haven’t even heard of? Overall, Health Canada did reiterate that several drugs on the common set of drugs had RWE, and that was partly why they were chosen. These case studies are great to hear about, but they have yet to show that the system is capable of dealing with ultra-rare or gene-based drugs. Some provinces are looking closely at the Dutch Orphan Drug Access Protocol to see if this can be implemented in Canada.
One major highlight deserving of some recognition is that, through this strategy in the last two years, more than 623 people got new access to drugs and drug treatments. While this is a small percentage of Canada’s population and Canada’s rare disease population, it is potentially over 600 saved lives, and that’s not nothing. Additionally, the Provinces have spent 88% of their agreement funding, meaning they are actively building programs that we can expand and develop. Of the money in question, 87% of it went to access to drugs on the specific list; however, over 41 drugs for rare diseases not on the list also got some coverage utilizing those funds. Critically, though, it was not equal access; some provinces covered some medications, while others refused the same drugs.
We are, however, seeing investments for building system capacity and the beginnings of a system form to address barriers to access across some provinces. This is slow going, though, and in critical need of prioritization if Canada is to compete on the global stage for rare drugs and rare disease research and infrastructure.
One concern that has been raised before and is still not getting addressed adequately is that a significant number of drugs on the list are cancer drugs, not for rare cancers or other rare diseases, but for cancer patients who didn’t respond to traditional cancer treatments. There seems to be no answer for why these drugs were included in the rare disease list, other than it was a way to get them into the Canadian system, and they were easier to deal with than actual rare disease drugs. These are important drugs to bring to Canada and, obviously, are critical for the cancer patients that need them; however, it does seem like this is potentially redirecting the focus from rare disease drugs to just any drug that is expensive and hard to get coverage for, which would be a tragic loss for the community.
The Patented Medicine Prices Review Board (PMPRB) had the opportunity to talk about its role in the Health Canada drug approval process. They were very clear, explaining that they are not another step in an already incredibly long pipeline; it runs parallel to prevent excessively expensive drugs from maintaining an unfavourable price. The systematic problem for rare disease drugs is that there is no path or recognition for rare disease drugs, which tend to be very expensive, with a small population of users. This means there is an inherent disadvantage in the PMPRB’s processes for rare disease drugs.
One concern some physicians raised was that Canada is struggling to keep up with the International Classification of Diseases. The WHA adopted ICD-11 in 2025 to monitor rare diseases better and to aid in diagnosis. Canada has some provinces still using ICD-9, while some have moved to ICD-10. The reality is simply that we are still not ready to widely adopt ICD-11 in Canada, but the federal government is trying to facilitate a move towards this system. An example of how far behind Canada is that British Columbia, my home province, utilizes ICD-10 (adopted in 1992) in emergency medicine; however, for our medical services plan to get continued care and physician billing, we utilize ICD-9 (adopted in 1976). Overall, moving to ICD-11 would be a 50-year step forward for my province, which is greatly needed.
The next presentation discussed the results of the CORD Patient and Caregiver Survey. This survey is a nationwide collection of data from rare disease patients and caregivers. This was the first time economic, as well as social, impacts were addressed by a nationwide survey by CORD. The survey found that over half of rare disease patients report their specialists are over 50km away from their home community. These patients report twice as many out-of-pocket costs for receiving care. It is important to remember that in many provinces, patients travel upwards of 6 hours by car, or fly 2-4 hours, just to receive care within their own province.
The survey showed that this is not a Canadian exclusive issue. Surveys in Japan and the EU show similar levels of travel for care and additional costs for the rare disease communities. One more frustrating statistic is that the levels of delay from the 2014 Canadian survey are nearly the same in the 2025 survey, showing almost no improvement in this area. However, despite this consistent delay in diagnosis and care, especially for less urban residents, there was an increase in access to therapies from 2014 to 2025. So, this could be some evidence that the Rare Disease Drug Strategy is starting to work as designed.
The Canadian system is not evolving at the speed at which medical advancements are moving. Canada has been breaking down drugs into so many groups, then giving priority in different ways to different groups, which is unnecessarily slowing down the medical care system as a whole. In our fragmented system, this will be a major challenge to overcome. The hope is for there to be a unified pathway for rare disease drugs, treatments, and care to move through within all 14+ of the Canadian medical systems working in parallel.
One additional concern that Canada is dealing with beyond our control is the United States’ Most Favoured Nation (MFN) policy. The reality is still not well understood, but it has the potential to really hurt Canada’s ability to bring in new drugs and run drug trials. In short, the US pays the highest drug prices around the world. As of May 2025, an executive order ties the US price of a drug to the lowest price in any OECD country with a GDP per capita of at least 60% of the USA’s GDP per capita; this does include Canada. Now, manufacturers must choose between raising prices abroad and lowering US revenues on their drugs. There are already some real-world consequences of this MFN, even though it’s still in its infancy; new drug launches in the EU have fallen 35% since MFN. There is no data for Canada yet, but it is presumed to be similar.
One of the biggest challenges with current gene treatments readily available in Canada right now is that you may only be able to use them once, as the immune system learns the virus vector used to deliver the treatment. The result of this is that the virus vector cannot be used a second time as the immune system responds and destroys it. This is not something Canada can fix with policy, but it shows the need to expand the study into this area, as it means patients and parents are left trying to decide whether to use current treatments or wait for advancements using the same virus vector, as only one option may exist.
The conference wrapped up with notes to have a follow-up meeting to create a call to action for our provincial and federal governments to continue moving towards expanding care, but to also try to address the continued delays in diagnosis in Canada.
Thanks to Katie Allen (Patient Advocate Canada).