Panel Abstract:
Imagine a future where Canada has developed a cure for diabetes, or treatments that stop or delay the progression of muscular dystrophy and Alzheimer’s. And now imagine that, as a country, we cannot get that treatment to market – to patients – because of red tape. This imagined future is not far off – and Canada needs to be prepared. The rapid pace of medical innovation, especially in cell and gene therapy and regenerative medicine, will bring groundbreaking treatments to some of the most devastating diseases. This, in turn, demands regulatory innovation to improve public access to new therapies while also addressing high development costs, inaccessible prices, and lagging reimbursement policies. Join Canada’s Stem Cell Network and ThéCell as they host an interactive discussion to explore the topics of regulation, patient access, and affordability, with the goal of informing a strong, inclusive, and flexible regulatory framework for Canada’s future.

Summary of Conversations

The panel explored access, affordability, and adoption of regenerative medicines in Canada, emphasizing the need to translate scientific advancements into clinical applications. Discussions highlighted challenges such as regulatory pathways, pricing, and equitable access across the country. The importance of a community approach involving government, industry, researchers, and patients was underscored. Speakers shared insights on regulatory flexibility, quality and manufacturing complexities, and clinical trial designs for rare diseases. The necessity of collaborative and trusted partnerships and the need for transparent, open conversations and flexibility were emphasized as key to advancing innovative treatments. Examples like cell therapies for Type 1 diabetes showcased the potential impact and the need for political will to prioritize health research and clear regulatory pathways.

Take Away Messages/ Current Status of Challenges

• Need for strong, transparent regulatory pathways for hospital-based cell therapies and small biotechs.
• Pricing of therapies and risk-sharing strategies with payers remains a significant hurdle.
• Accessibility disparities exist based on location, hindering equitable access to advanced therapies.
• Patient perspectives and expectations must be central to policy and regulatory decisions.
• Canada’s reimbursement landscape is complex, varying by province and funding source.
• The healthcare system is currently facing a crisis, limiting resources and focus on future innovations.
• Clinical trial design for rare diseases presents unique challenges.
• Data sharing must be efficient, equitable, and ethical, with good governance at its heart.

Recommendations/Next Steps

• Foster collaborative, trusted partnerships to facilitate transparency and flexibility in information sharing.
• Engage decision-makers early to determine funding pathways and evidence requirements.
• Promote early engagement with decision makers at all levels to figure out the funding pathway and evidence requirements.
• Develop innovative risk-sharing models, such as outcomes-based agreements, to address the high cost of therapies.
• Conduct tabletop exercises to simulate decision-making processes and identify potential challenges.
• Improve infrastructure to support value assessment and outcome tracking for innovative treatments.
• Streamline communication and interpretation of policies for effective implementation across agencies.
• Build patient confidence by ensuring innovations are easily understood.