Gene therapy is permitted, but must be approved by Health Canada’s Biologics and Genetic Therapies Directorate (BGTD), which is responsible for overseeing all clinical trials. The BGTD applies the Food and Drugs Act, which includes the Food and Drug Regulations and the Medical Devices Regulations. The BGTD requires more detailed chemistry and manufacturing information for biological drugs to be approved than other drugs for human use.
The BGTD differentiates between high risk, moderate risk and low risk biological drugs. Every lot of a high-risk drug is tested before being sold. Moderate risk biological drugs are periodically tested. Manufacturers of low risk biological drugs are only required to contact Health Canada to certify that safety testing has been completed.
The reason for the tight oversight is because under Canada’s national health program, the cost of expensive gene therapy will almost certainly be borne by the public health-care system and not private drive insurance, and will be delivered in select hospitals.
The Canadian Agency for Drugs and Technologies in Health (CADTH), the independent organization that advises the provinces and territories on whether they should cover new drugs, puts new oncology drugs, including those developed through gene therapy, through an evaluation called the pan-Canadian Oncology Drug Review, which ends with a clear recommendation for or against public funding.
There are multiple gene therapy research programs being developed, including:
Products/Research
- Urea cycle disorder: The University of Calgary Cumming School of Medicine (CSM) and Alberta Health Services (AHS) gave the first Canadian a gene therapy treatment for urea cycle disorder, a rare genetic disease that causes ammonia to build up in the body and can lead to brain damage or death.
- Lipoprotein lipase deficiency: The ECOGENE-21 Clinical Research Center completed a clinical trial to treat lipoprotein lipase deficiency, a genetic disease that leads to problems with the pancreas and liver and can lead to diabetes.
- Choroideremia: The University of Alberta conducted a clinical trial for ocular gene therapy for choroideremia, which causes progressive vision loss in males.
- Cancer: Clinical trials have been completed and the CAR T-cell therapies for patients with certain types of leukemia and non-Hodgkin’s lymphoma have been approved.
Regulatory Timeline
2011: Food and Drug Regulations amended.
1985: Canada passes the Food and Drugs Act, which includes the Food and Drug Regulations.
NGO Reaction
None
Additional Resources
- Genetic Literacy Project’s FAQ on gene editing
- Library of Congress summary of Canada gene regulations includes detailed analysis of the country’s evolving biosafety laws and liabilities
- Current state of Health Canada regulation for cellular and gene therapy products: potential cures on the horizon
- Gene Therapy: An Overview of Approved and Pipeline Technologies
