November 3, 2020

From Research to Reality

The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada

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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.

The Sponsor:

National Research Council Canada



An international symposium on gene therapy research and practice
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The Question

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?

Key Findings

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.

  • The diversity of gene therapies requires a flexible and tailored approach to addressing access and affordability challenges.
  • Risk-based purchasing agreements and post-market surveillance could mitigate the significant clinical and economic uncertainties associated with approved gene therapies.
  • High prices, complex provision, and the nature of diseases treated by gene therapies exacerbate existing inequities in healthcare access.
  • Different conceptions of value may lead to disagreement over the merits of publicly funding individual gene therapies.
  • Pan-Canadian coordination could control spending and improve access to gene therapies.
  • Stewardship of public investments in gene therapy research could alleviate challenges associated with commercialization and high drug prices.

Expert Panel

The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada