From Research to Reality
The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada
The potential for gene therapies to treat serious diseases was first recognized in the 1960s. After decades of discovery and clinical research, that potential is advancing to practice in Canada.
A handful of somatic gene therapies have received market authorization and hundreds more are being tested in clinical trials worldwide. In Canada, four gene therapies have been approved for use, but there are significant challenges that come with introducing these therapies into healthcare systems while ensuring safety, access, and affordability
Rapid scientific advances mean potentially life-changing treatments are approaching the clinic at an accelerated pace. Choices faced by decision-makers about authorizing and funding gene therapies are complicated by uncertain long-term outcomes, high prices, and patient demand.
From Research to Reality describes the stages involved in the approval and use of gene therapies in Canada, and examines challenges associated with regulatory oversight, manufacturing, access, and affordability, and identifies promising approaches to address them.
National Research Council Canada
What are the key legal/regulatory, ethical, social, and policy challenges specific to the approval and use of somatic gene and engineered cell therapies in Canada?
Few gene therapies have been approved for use worldwide and there is consequently limited evidence on their implementation in healthcare systems. But examples are emerging as various jurisdictions explore ways to ensure timely market authorization decisions and fair and affordable access. There is an opportunity to learn from approaches being tested and implemented domestically and abroad, and apply emerging solutions in Canada.