Public labs, SMEs and large companies could propel Canada to the forefront of gene therapy commercialization: CCA report

The Council of Canadian Academies released an expert report November 3 that highlights the challenges and opportunities to the development and commercialization of gene therapies in Canada.

While the challenges are not insignificant, the expert panel found that Canada’s strengths can be translated into sustainable global companies capable of manufacturing affordable therapies in Canada.

The report was sponsored by the National Research Council of Canada which asked the CCA to examine the key legal, regulatory, ethical, social and policy challenges specific to the approval and use of somatic gene and engineered cell therapies in Canada. Somatic gene and engineered cell therapies treat disease by removing or modifying existing genetic material in a patient, or introducing new genetic material, without passing these changes to future generations. Most treat rare genetic disorders and cancers, however they can cost more than $1 million and have limited evidence of long-term safety and durability.

The 122-page report, From Research to Reality, describes the stages involved in the approval and use of gene therapies in Canada, and examines the challenges associated with regulatory oversight, manufacturing, access, and affordability. It also identifies emerging solutions that can capitalize on Canadian strengths in research, manufacturing and health-care accessibility.

Reprinted below are the expert panel’s reflections from the report:

The Panel notes that there are two types of challenges to the deployment of gene therapies: those intrinsic to the Canadian context, and those that arise through the global development of gene therapies.

Many of the intrinsic challenges involved in getting novel drugs to patients — complex, multi-actor decision-making processes, budgetary pressures, commercialization challenges — are exacerbated for gene therapies due to their high cost and complexity, but are not unique.

As such, confronting the access and affordability challenges posed by gene therapies can serve as a valuable test case for other challenges in Canada’s healthcare systems. Extrinsic challenges — which include evolving global research and intellectual property regimes, and the changing regulatory landscape of gene therapies — arise when decisions made outside Canada impact the accessibility and affordability of gene therapies within Canada.

Other countries are grappling with many of the same challenges, and much can be learned from approaches being tested and implemented abroad.

Emerging solutions can draw on existing Canadian strengths. The capacity to manufacture gene therapy components can be built on and improved as production ramps up to support provision across Canada. Additionally, the principle of universal accessibility set out in the Canada Health Act could help motivate the development of novel models that aim to provide these life-changing therapies to patients at reasonable prices.

However, as pan-Canadian principles and approaches are contemplated, jurisdictions should be mindful of the potential for access to be inadvertently constrained through the adoption of lowered common standards. Broad-scale solutions will require participation from all stakeholders, from the bench to the bedside and beyond. This can be supported through the translation of existing strengths in Canadian discovery research into sustainable global companies based in Canada with sufficient access to capital and local manufacturing capabilities.

From an economic perspective, successfully overcoming the challenges outlined in this report could allow Canada to position itself at the forefront of gene therapy commercialization. By building a landscape in which public laboratories, SMEs, and larger commercial players can develop novel and affordable therapies within an effective regulatory framework, Canada will be well positioned to compete globally in this market.

Skills development is an important component of success; in addition to training HQP in the manufacture and provision of gene therapies, complementary skills in the domain of IP law and drug price negotiations could enhance Canada’s position.

Gene therapies are just beginning to be available in Canada, and the evidence on how best to overcome access and affordability challenges is limited. It will be important to document lessons as new models and emerging solutions are applied in various contexts.

The Panel would like to emphasize that discoveries in this field continue to multiply. What constitutes the technological state-of-the-art is in rapid evolution, and may advance through developments in, for example, non-viral vector delivery or improvements in automated manufacturing solutions. This rapidly shifting landscape further justifies the importance of ongoing proactive approaches to tailoring research, oversight, and funding.

From Research to Reality: Main Findings

• 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