Siofradh McMahon is Senior Manager, Clinical Translation and Regulatory Affairs at CCRM. Síofradh works with academic and industry partners to advance promising products and technologies to commercialization. Having worked worldwide, Síofradh brings more than a decade of experience in pharmaceutical and biotech regulatory affairs and policy. She is an active member of the Alliance for Regenerative Medicine’s Regulatory and Value & Access working groups and lends her expertise to groups in Canada too.
Image by Scott Gardner, CCRM
We talk a lot about timely access to medicines in Canada, as it’s true that the time from marketing approval (i.e. Health Canada approval) to reimbursement and adoption (i.e. access for patients) can be long, particularly for speciality or personalized medicines, or drugs to treat rare diseases. There are lots of reasons for this, some of which are specifically Canadian and others that affect all public health-care systems to some extent.
The first thing to understand is that Canada does not have one health-care system. Depending on how you count it, you could say we have about a dozen, or 100+ if you include the private health care insurance providers. The provinces provide health care for their residents, and each province’s health-care system can be structured a little differently, with different decision makers, and differing budgets allocated to their particular health concerns of interest.
This makes sense when we consider the vast geography of Canada and the varied health needs of its people (i.e. the health interventions needed for people in a province like Ontario, with a number of large cities, will be different to what is needed for people living in more rural communities like Newfoundland and Labrador). However, as you can imagine, this can mean a complex mix of stakeholders for therapy developers to negotiate access with, requiring expertise and resources to do it well.
Secondly, as in all countries with public health-care systems, there is a need to be rational with the allocation of public funds. It can therefore be challenging to find the right balance between driving innovation in our health-care systems and maintaining a balanced budget that continues to allow us to provide appropriate health care to all. So it can be difficult to find the right way to incorporate high priced therapies intended to treat small patient populations into a public drug budget without negatively impacting the fundamental mandate of the health-care system, which is to provide the greatest health benefits to the whole population it serves.
Finally, if we broaden our definition of access to include access to experimental therapies in clinical trials, we find that many studies of cell and gene therapies (CGTs) do not include patients in Canada. One of the reasons for this is that the nature of the studies and level of engagement therapy developers require with the study sites can be complex, and therapy developers are often more comfortable and confident with the team at the institution local to them with whom they have worked before. They may also have developed the product in collaboration with that institution, and so they understand the situation better, and can see opportunities to increase efficiency or speed, or reduce risk. Since fewer developers are headquartered in Canada and fewer new companies start up here than in the U.S., we don’t see the same level of activity with new clinical trials in Canada.
You might be thinking that these issues are not specific to CGTs, and you’d be right. However, all of these challenges are magnified when we try to bring highly specialized and complex therapies like CGTs to Canadians in a rational way.
If we take the approved CAR T-cell therapies as an example, these require specialist facilities, resources and expertise at the point of care. It is therefore immediately apparently that not every province in Canada will have access to what is needed to administer these products. We also see this effect in clinical trials, as almost all of the CGT studies that have been run in Canada have been initiated in major urban centres with large hospitals that could be considered centres of excellence in their field. This means that our patient in Newfoundland and Labrador, who is already very unwell, will have to travel to another province for treatment, and likely spend a considerable amount of time away from home. This has a financial cost, as well as a significant psychosocial cost for the patient and can be a considerable barrier for many people.
Speaking of costs, we also need to keep in mind that the cost to the health-care budgets of adopting CGT products is not just about the price of the drug, but also the cost of all those specialist facilities, expertise and resources mentioned above. This is a challenge for all health-care systems, whether publicly funded or paid out of pocket, and continues to be particularly complicated when the products being developed are intended to treat small patient populations, as is the case with CGTs. These patients will also require long-term monitoring for safety and efficacy, which imposes additional burdens on the health-care systems and the patients themselves and, again, additional cost.
So what can we do to solve some of these issues? Well, the good news is that a lot of work is already underway to better understand the “pain points” in the access journey and to find solutions. Recent changes to the health technology assessment process in Canada have already significantly reduced the expected overall time to access, and regulatory modernization efforts at Health Canada will likely have a similar effect in the future; however, there is always more we can do. CCRM is actively involved in helping to build an ecosystem that supports timely and appropriate access to CGTs for patients in Canada, including by increasing the number of Canadian clinical trials, increasing investment in home-grown innovations, and guiding innovators through the development process to help them bring affordable innovations to the patients who are waiting.
My blog is just one of many covering the topic of “What are the hurdles impacting patient access to cell and gene therapies” as part of Signal’s fourth annual blog carnival. Please click here to read what other bloggers think about this.
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