From Research to Reality

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.