An Inside Look at Precision and Regenerative Medicine in Canada
July 21, 2026
The medications of the future are here; the access pathways, not quite. We examine the treatments, the system, and the cracks to fill in
Not even 50 years old when stage-4 colorectal cancer crashed into her life in 2010, Robyn McGee got the best treatment available to her at the time. But the Nova Scotia resident didn’t stop there. When the Canadian healthcare system ran out of funded treatment options for her, she used her own money to pay for a genomic analysis of her tumour, hoping its double-stranded secrets would open the door to new treatment options. In fact, her tumour’s genetic profile suggested that a drug normally used to treat other forms of cancer might work.13
The gamble paid off. Her cancer blood markers plummeted, indicating a response to the other drug. McGee had successfully orchestrated a treatment strategy known as precision medicine. She lived until 2024, about 12 years beyond doctors’ expectations.14
Like McGee, Brian Armit of Dryden, Ontario resolved to turn every stone after his young-adult son, Evan, developed melanoma. By 2025, Evan had only one recourse left: tumour-infiltrating lymphocyte (TIL) therapy, a type of immunotherapy that harnesses a patient's own immune cells to attack solid tumors. Because the therapy was still undergoing regulatory review in Canada, Evan would have to travel to Boston for treatment, at an estimated cost of $750,000 to $1 million.15 After Ontario’s out-of-country coverage program denied his request – twice – Brian took his case to Ontario’s premier, Doug Ford, who suggested he file an appeal.
Evan died at the age of 23, nine days after Health Canada approved TIL therapy and four days after the appeal’s final verdict: denied.
As these stories make clear, treatment for complex diseases such as cancer has become extremely sophisticated – and, for one reason or another, beyond reach for many Canadians. The science has galloped ahead, with access scrambling to catch up. To close the gap, we need to understand how these high-precision treatments work, what they can and cannot deliver, and what it would take to make them accessible them at scale.
TERMS OF THE TRADE
The treatment categories we’ll be talking about here – personalized, precision, and regenerative medicine – overlap considerably. Personalized medicine describes a medical intervention that conforms to a specific patient profile,1 while precision medicine uses biological information about a specific patient to determine the type of therapy that has the best chance of working.2 For example, patients whose breast tumours overexpress a protein called HER2 can be treated with medications that block HER2 receptors. With oncology leading the way, precision medicine now reaches into numerous other therapeutic areas, including rheumatology, cardiology, and even psychiatry.
Regenerative medicine, which encompasses cell therapy and gene therapy, seeks to restore cells and tissues that have died or become diseased.3 Cell therapy took a big leap forward in 2019 with Kymriah, a class of treatment known as CAR-T, which was the first of its kind approved in Canada. Indicated for certain types of lymphoma and leukemia, Kymriah reprograms a patient’s T-cells to recognize and attack cancer cells.16 As an example of gene therapy, the ophthalmology drug Luxturna, indicated for vision loss due to a gene mutation, supplies a working gene to replace the mutated one.17
Here’s one way to look at the categories: all precision medicine is personalized, and many regenerative treatments double as precision treatments. For example, regenerative cell therapies yield the best results if they’re tailored to each patient’s profile.
This raises the question: how do clinicians obtain the “biological intel” that enables them to customize treatment in the first place? The job often falls to comprehensive genomic profiling (CGP), an in-depth DNA analysis that identifies hereditary vulnerabilities and tumour mutations.5 In a similar vein, biomarker testing measures a biological substance or process to find out how a disease behaves in a particular patient.6 These two categories also intersect: a patient’s genomic profile can help clinicians track down actionable biomarkers.
PRECISION MEDICINE IN CANADA
On a health systems level, precision medicine depends on having a robust genomic data ecosystem. Enter the Canadian Genomics Strategy, a long-range federal government plan to accelerate the use of genomics in medicine and other areas such as agriculture and renewable energy.18 The government has committed more than $1.6 billion over 20 years for large-scale genomics projects, placing Canada among the world leaders in basic and applied genomic research.
Genome Canada will be leading the charge through its Canadian Precision Health Initiative (CPHI). Announced in March 2025, the program plans to collect data from more than 100,000 human genomes that reflect the diversity of Canada’s population.19 That’s a lot of DNA. Individual projects within CPHI include Care4Rare EXPAND, a four-year program to gather genomic data specific to rare diseases, and a gene-sequencing effort to enable earlier detection of rare diseases in newborns.
If all goes as envisioned, this genomic juggernaut should help clinicians unearth the strengths and vulnerabilities in their patients’ genetic profiles, culminating in “more precise, personalized, predictive, preventative, and cost-effective healthcare options” for Canadians.19
PANCREATIC PRECISION
About 90 percent of pancreatic cancer cases are driven by genetic mutations that lock a protein called RAS in the “on” position, leading cells to divide and grow uncontrollably. Because of the RAS protein’s smooth surface – picture a golf ball – scientists have struggled to develop drugs that can attach to it and inhibit its function.20 Until daraxonracib, that is.
In a US-led study of daraxonracib, those who took the daily pill survived for over a year, about six months longer than those who received chemotherapy alone.20 For pancreatic cancer, that’s a big deal. Dr. Jennifer Knox, head of pancreatic cancer at the Princess Margaret Hospital in Toronto, explained that “so many things just don’t work [for pancreatic cancer]”, and a doubling of survival time has “never been seen” before.21 While Health Canada hasn’t received a submission for daraxonracib yet, Dr. Knox plans to run clinical trials to make the drug available to as many patients as possible.
On the regenerative medicine front, an April 2026 Watch List report prepared by Canada’s Drug Agency (CDA-AMC) flagged five areas to keep an eye on: gene therapies, stem cell therapies, adoptive cell therapies (like CAR-T), bioengineered skin, and structural scaffolds and biomaterials.22
COMPANION CHALLENGE
If you want to treat people with specific genetic profiles, you need first to identify your target population. That’s why precision therapies rely on diagnostic tests to identify the right patients for the treatment. Not surprisingly, these “companion diagnostics” can add detours to the access journey. Eon Ting, Director, Payer Evidence, Methods, & Access Affairs at AstraZeneca, noted this challenge at a CDA-AMC symposium session in April 2026.23 “It’s a real-world problem,” he said.” Before a patient can get one of these precision drugs, they have to get a biopsy, or blood test, or something similar to find out if the drug is right for them. Do we have the right equipment and staff to offer these tests at scale, when patients need them?”
“Before a patient can get a precision treatment, they have to get a test to find out if the right is drug for them. Do we have the right equipment and staff to offer these tests at scale?”
Eon Ting
Director, Payer Evidence, Methods & Access Affairs
AstraZeneca
A 2026 progress report on the use of genomic testing in medicine has pronounced Canada “partially ready,” with Ontario and Nova Scotia making the biggest strides since 2023.24 The report included a list of action items to help move things along, from using electronic technology to integrate laboratory information systems to publishing a transparent process for adding new genomic tests to laboratories.
Ting, for his part, applauds the CDA-AMC’s foresight in updating its companion diagnostics process to prepare for the abundance of precision treatments in the pipeline. Specific changes announced by the agency include greater prominence to companion diagnostic tests in drug reviews, an improved presubmission phase that alerts the CDA-AMC to diagnostic testing requirements that may impact health system resources, and a dedicated team to conduct reviews of companion diagnostics.25
STARTING EARLY23
The estimated 60 new cell and gene therapies expected in the next decade will require a matching increase in screening capacity. Newborn screening can help identify some serious conditions right after birth, enabling the infants to receive life-changing treatments at the starting gate.
In 2025, CDA-AMC published guidance to support newborn screening programs in Canada. The guidance included a recommended list of 25 conditions to screen for in newborns across Canada, and a list of 29 additional conditions to monitor and possibly add to the list.
Can a public health system realistically support these next-gen tests? A cost-benefit analysis called Life Years Gained and Healthcare Dollars Saved, hot off the press in Current Oncology journal, concludes that it can.11 The investigators go further still, arguing that the tests can save big dollars over the long term – along with saving lives.
Here’s how their math nets out: over a 6-year period, universal CGP across the five tumour types with the highest mortality – lung, colorectal, pancreas, breast, prostate – would yield an additional 3,440 life years along with a healthcare system savings of $87-134 million, a win-win by any measure.11
The paper reporting on the analysis predicted that the value of CGP will compound over time, as researchers identify patient groups most likely to benefit from precision treatments and indications for existing treatments expand to include new tumour types. But the first priority, the paper argues, is to establish CGP as the standard of care for the deadliest cancers: “Funding should be allocated so that CGP is first publicly covered for the five tumour types that contribute to the highest cancer-related mortality.”11 Ideally, this investment will “serve as the impetus for program extension to other tumour types as emerging biomarkers are realized.”
The Colorectal Cancer Resource and Action Network (CCRAN) is on the case, having just wrapped up a conference focused on establishing a national CGP strategy for metastatic cancer care in Canada.27 CGP can “help to improve both longevity and quality of life for metastatic cancer patients,” says CCRAN president and CEO Filomena Servidio-Italiano, “but only if a patient and their treating oncologist are able to avail themselves of this wonderful technology, which sadly is currently inequitably available across tumour types and across the country.”27 Indeed, two-thirds of surveyed Canadian oncologists cannot access CGP for over 80% of their metastatic patients.11
“Comprehensive genomic profiling can help improve both longevity and quality of life for metastatic cancer patients – but only if a patient can access this wonderful technology, which sadly is currently not equitably available.”
Filomena Servidio-Italiano
President and CEO
Colorectal Cancer Resource and Action Network
READY… OR NOT?
All told, precision and regenerative treatments aren’t consistently reaching the patients who need them – a gap expected to persist until implementation barriers from across the entire product life cycle are addressed.22 Hurdles to overcome include scalability, health system readiness, risk of harms, affordability, and access disparities – the usual suspects.
The drug Carvykti exemplifies how cost considerations weigh on decisions about public coverage and patient access. After Health Canada approved the CAR T-cell immunotherapy for multiple myeloma in early 2023, CDA-AMC recommended that provincial governments pay for the drug if they could get a significant reduction on the $632,455 sticker price for the one-time infusion.28 The pCPA and the drug’s manufacturer, Johnson & Johnson Innovative Medicine, began closed-door negotiations on the price, only to reach an impasse in the fall of 2025. At the time, 13 other countries were already reimbursing the drug, which has the potential to significantly increase survival. Myeloma Canada issued a call to action, mobilizing stakeholders to send close to 6,000 emails to elected representatives throughout Canada.29 As of February 2026, the negotiations are back on.30 While the news gives hope to myeloma patients, they’re still waiting.
Economics aside, the new tests and treatments depend on a complex and dynamic infrastructure. Consider the leukapheresis, cell processing, and bridging and lymphodepleting chemotherapy built into the Carvykti protocol, all requiring specialized centres and specially trained staff.31 As noted in CDA-AMC’s reimbursement review of Carvykti, “managing various protocols for preparation and delivering each product type poses an administrative burden.”31
Is our health system ready for this challenge? Not quite, according to a recent analysis of inequities in cancer care delays across Canada.32 The paper identified several significant roadblocks – from failure of clinicians to discuss genomic medicine with patients to lack of laboratories to provide testing and delays in reporting results – and concluded that Canada must “prioritize access to advanced molecular testing to ensure systems are in place to quickly bring innovation and evidence-based treatments to Canadian cancer patients, regardless of their place of residence or socioeconomic status.”
To rein in costs, the system may also need to consider alternative service delivery models, such as greater centralization.33 At the same time, the need to share information across databases – a crucial aspect of genomic testing – will call for creative problem-solving to break down current silos.33 Above all, readiness depends on harmonization across jurisdictions. The updated State of Readiness Progress Report puts it succinctly: “What does ‘ready’ look like? Service models that are highly standardized are required. Larger health systems may require more care coordination to accomplish this.”24
TOWARD A TIPPING POINT
So what comes next? One thing we can say for sure: the changes ahead will impact all stakeholders, from patients and doctors to pharmaceutical manufacturers and payers. We suggest keeping an eye on these developing areas:
For patients: Biomarker testing is poised to scale up. With the deployment of provincial genetics programs across the country, patients can expect genomic testing to become the norm. In a country as spread out as Canada, equity will undoubtedly pose challenges: how can we ensure that patients in rural and remote communities have access to these tests?
For health providers: As genomic profiling becomes the standard of care for metastatic cancer, specialists such as oncologists, rheumatologists, gastroenterologists, and pathologists will need training in genomics. Experts also predict a shortfall in genetic counsellors. The growing need for specialized medical lab technologists will likewise require training. Expect post-secondary education programs in advanced laboratory procedures to proliferate.
For pharmaceutical companies: With the unstoppable advance of genomic science, one-time curative gene therapies will flourish. Demand for these therapies will fuel the growth of home-grown manufacturing capabilities. The BioCanRx immunotherapy network has already crossed this Rubicon: using automated equipment, members of the network have begun producing a CAR-T cell therapy called CLIC-19 in Canada.34 The high cost of these drugs speaks to the need for innovative market access agreements to spread the risk and knock down access hurdles.
For payers: As one-time, high-cost therapies replace their chronic care equivalents, payers and price negotiators will need to conduct even more complex budget impact analyses to help them plan ahead. Like pharmaceutical companies, payers stand to gain from accelerated access pathways with risk-sharing options.
So here we are: well into the preparation phase, if a little sluggish on implementation. Fortunately, our leaders are taking steps to meet the moment. As a case in point, Quebec’s recently unveiled Policy in Genomic Medicine will invest $2.5 million to support genomic profiling in oncology, funding for companion tests, and a fellowship program to train laboratory geneticists, among other initiatives.9 We also have a solid legacy of innovation to build on: It was in Toronto, after all, that a pair of researchers discovered hematopoietic stem cells in 1961.22
In brief, we have the science and the vision to forge ahead. What remains to be seen is how quickly the access infrastructure can catch up. Tick tock.
References
Personalized Medicine Coalition. The Age of Personalized Medicine. https://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/pmc_age_of_pmc_factsheet.pdf
Medline Plus. What is precision medicine? https://medlineplus.gov/genetics/understanding/precisionmedicine/definition/
US FDA. Focus Area: Regenerative Medicine. https://www.fda.gov/science-research/focus-areas-regulatory-science-report/focus-area-regenerative-medicine
US FDA. What is gene therapy? https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy
National Human Genome Research Institute. A Brief Guide to Genomics. https://www.genome.gov/about-genomics/fact-sheets/A-Brief-Guide-to-Genomics
US FDA. About Biomarkers and Qualification. https://www.fda.gov/drugs/biomarker-qualification-program/about-biomarkers-and-qualification
National Cancer Institute. BRCA Gene Changes: Cancer Risk and Genetic Testing. https://www.cancer.gov/about-cancer/causes-prevention/genetics/brca-fact-sheet
Swain SM, et al. Targeting HER2-positive breast cancer: advances and future directions. Nat Rev Drug Discov 2022 Nov 7;22(2):101–126.
Lancement de la Politique québécoise en médecine génomique. PR Newswire. June 22, 2026. https://www.newswire.ca/fr/news-releases/lancement-de-la-politique-quebecoise-en-medecine-genomique-845313566.html
Canadian Organization for Rare Diseases (CORD). About CORD. https://www.raredisorders.ca/about/about-cord
Snow S et al. Life years gained and healthcare dollars saved. Current Oncology 2026;33:191.
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McDonald M. Cancer patients, survivors gather to talk about the future of 'precision medicine'. CBC News, Nov. 16, 2023. https://www.cbc.ca/news/canada/nova-scotia/cancer-patients-survivors-precision-medicine-1.7030000
White Family funeral home announcement, 2024. https://www.whitefamilyfuneralhome.com/obituaries/178299
Grant K. Ontario parents hope son’s death can change Canada’s approach to novel cancer treatments. The Globe and Mail, June 1, 2026. https://www.theglobeandmail.com/canada/article-an-ontario-family-hopes-their-sons-death-can-change-canadas-cancer/
Kymriah product monograph. https://pdf.hres.ca/dpd_pm/00077617.PDF
Luxturna product monograph. https://pdf.hres.ca/dpd_pm/00083578.PDF
Canadian Genomics Strategy. Government of Canada. 2025. https://ised-isde.canada.ca/site/ised/en/genomics/canadian-genomics-strategy#s2
Canadian Precision Health Initiative. Genome Canada. https://genomecanada.ca/challenge-areas/canadian-precision-health-initiative/
Azmi A. Targeting KRAS in Pancreatic Cancer: What is daraxonrasib’s mechanism of action? Cancer Network. April 30, 2026. https://www.cancernetwork.com/view/targeting-kras-in-pancreatic-cancer-what-is-daraxonrasib-s-mechanism-of-action-
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2026 watch list: regenerative medicine. Canadian Journal of Health Economics. April 2026, Volume 6, Issue 4. https://www.canjhealthtechnol.ca/index.php/cjht/article/view/ER0016/ER0016
Accelerated Access Pathways Plenary Session, CDA-AMC Symposium, April 24, 2026.
Towards the routine use of genome-based testing in Canada’s largest regions: An update to the State of Readiness Progress Report. 2026. https://cdn.prod.website-files.com/62874663569d676deb3ae6c8/698b5ffc8fed6075c2de3db0_An%20update%20to%20the%20State%20of%20Readiness%20Progress%20Report.pdf
Improving Our Assessments of Drugs With Companion Diagnostic Tests. CDA-AMC. Last updated June 9, 2026. https://www.cda-amc.ca/news/improving-our-assessments-drugs-companion-diagnostic-tests
New pan-Canadian recommendations for newborn screening in Canada. CDA-AMC. March 31, 2025. https://www.cda-amc.ca/news/new-pan-canadian-recommendations-newborn-screening-canada
CCRAN 4th annual pan-tumour biomarkers conference announcement. https://www.ccran.org/ccran-biomarkers-conference
Lima E. Canadians can wait years to get drugs regulators have already deemed safe. Why? The Globe and Mail. October 31, 2025.
Myeloma Canada Welcomes Restart of pCPA Negotiations for Carvykti. Myeloma Canada. February 26, 2026. https://myeloma.ca/2026/02/26/myeloma-canada-welcomes-restart-of-pcpa-negotiations-for-carvykti
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Snow S et al. Barriers and unequal access to timely molecular testing results: addressing the inequities in cancer care delays across Canada. Curr. Oncol 2024;31:1359.
Husereau D et al. Effective and efficient delivery of genome-based testing – What conditions are necessary for health system readiness? Healthcare 2022;10:2086.
Made-in-Canada CAR-T platform. BioCanRX. https://biocanrx.com/researchers/made-canada-car-t-platform/