Canada is great at inventing next-generation therapies. But can we actually get them to patients?

Charmain Brown remembers the excitement of learning a new gene therapy designed to halt the rare condition causing vision loss in her two children had been approved.

But shortly after came a hard lesson: Approved does not mean accessible.

The therapy existed. It demonstrated benefits. Health Canada authorized it. Yet her family still faced years of uncertainty over who could receive it, how it would be delivered and whether the provincial health system would pay for it.

That gap between scientific breakthrough and patient access is becoming one of the defining challenges facing Canadian health care. And we are about to encounter it much more often.

Cell and gene therapies, tissue-engineered treatments and other forms of regenerative medicine are moving from laboratories into clinical care. They offer the possibility of treating and, in some cases, curing diseases that medicine has historically been able only to manage. 

But there’s a problem: 21st-century medicines are being evaluated by a system designed for 20th-century drugs — not for one that can consider the challenges presented by a living therapy. 

This doesn’t work for regenerative medicine where the process is part of the therapy, or for rare diseases where large randomized trials may be impossible because the disease affects only a handful of patients. Some treatments may be manufactured for a single patient.

This discussion matters now because Ottawa has an opportunity to change it. This summer, the federal Pharmaceutical and Life Sciences Sector Task Force called for modernization, including more flexible regulatory reviews, better navigation for innovators and specific measures to support made-in-Canada advanced therapies. The government is now considering those recommendations.

Regenerative medicine should be at the centre of that response. Canada has world-class science, researchers and clinical expertise. What we do not yet have is a system designed to move these novel therapies efficiently from discovery to patient.

Canada has spent billions strengthening domestic biomanufacturing and life sciences capacity.  But health sovereignty is not simply the ability to manufacture medicines here. It’s the ability to discover, develop, evaluate and deliver them to Canadian patients.

Living therapies still move through regulation, health technology assessment, pricing, reimbursement and health system adoption largely as separate steps, and all managed by different entities. Each institution may be doing its job, but patients experience the cumulative result: delay, or even a roadblock. For a newborn with a rapidly progressing genetic disease, such delay can close the treatment window.

These are not conventional pills in unconventional packaging. They require a different model.

A new Stem Cell Network report illustrates the challenge. Based on a workshop involving nearly 50 leaders from across Canada's health and life sciences ecosystem, it examined three therapies connected to Canadian research and expertise: a gene therapy for a rare fatal disease affecting newborns, a tissue-engineered skin replacement for severe burns, and an individualized treatment for a rare pediatric neurodevelopment disorder.

Each exposed a different weakness in the system, and underscored the need for a system that allows for flexibility based on the therapy, its purpose and the size of the population it will serve.

Cate Murray, President and CEO of the Stem Cell Network. 

Now is the time to start planning while promising therapies are still being developed. Hospitals need to prepare staff, facilities, diagnostics and follow-up capacity. Regulators, health technology assessment bodies and payers should work concurrently wherever possible. Funding must recognize the full cost of treatment, including development, manufacturing, specialized care, monitoring and patient travel.

A practical place to start would be to put the existing Advanced Therapeutic Products framework into practice, working with provinces and territories to test coordinated pathways from development through regulation, assessment, reimbursement, clinical adoption and long-term evidence collection.

Canada should also give promising made-in-Canada innovations a clear route through the system. That means early navigation, coordinated advice and prioritization – not a lower bar – so Canadian-developed therapies do not stall in a fragmented system.

There is also a strategic imperative.

Countries are competing for life sciences research, investment, manufacturing and talent. If Canada is unnecessarily difficult to navigate, our best science will flow like water to where it can thrive: where innovation hubs, private capital, adaptive regulation and advanced manufacturing work together symbiotically.

Health sovereignty begins upstream. It means having the scientific, regulatory, manufacturing and clinical capacity to turn discovery into treatment. 

Canada already has much of that capacity — we just need to connect it. And it needs to connect back to therapies being commercialized and deployed in Canadian hospitals.

For Charmain Brown and families like hers, that distinction is anything but theoretical. A medical breakthrough sitting on the far side of a regulatory or reimbursement process may as well not exist for the patient who cannot access it.

Canada invented regenerative medicine. Our next challenge is making sure Canadians benefit from it.

The views expressed are those of the author(s). Canada Healthwatch publishes a range of perspectives and does not necessarily endorse the opinions presented.