The Promise of Gene Therapy

Canada’s Path to Embracing the Future
 

February 7, 2024

For gene therapies in Canada, the future is now.

Gene therapy has the potential to offer eligible patients with rare genetic diseases a transformational clinical benefit and improved quality of life.

This ground-breaking field of medical science holds immense promise for transforming and advancing healthcare, especially in areas of high unmet need.

Understanding Gene Therapy
Gene therapy is a new generation of medicine, where a functional gene is delivered to a targeted tissue in the body to produce a missing or non-functional protein.^[1] By using genes as medicine, the underlying cause of a disease can now be targeted at the cellular level. Unlike traditional medications, which often require frequent administration and often focus on managing symptoms and disease progression, gene therapy aims to provide a long-term treatment benefit with potentially just one administration.^[2]
Pfizer is advancing research on gene therapy to deliver a range of potentially transformational medicines to patients living with genetic diseases.

This innovative approach to treatment holds enormous potential to address a wide range of diseases, including rare genetic disorders like hemophilia and Duchenne Muscular Dystrophy, and is offering new hope for patients and their families. Gene therapy could enable patients to live without the need for ongoing treatments or the burden of daily disease management.^[3] 

Navigating the Challenges
The promise of gene therapy is vast though there are challenges to address as Canada embraces this new approach. While gene therapy holds promise for people with genetic diseases, it will not be the appropriate solution for every patient. Through ongoing research and clinical experience, we will continue to learn about the long-term potential risks and benefits of these one-time gene therapy treatments.^[4]

Importantly, Canada’s regulatory and reimbursement frameworks need to be prepared and adapted to accommodate gene therapy's unique characteristics. As with all new innovations, striking a balance between long-term patient benefit and cost is crucial. As a reflection of the value of treatment benefits to patients, health systems and society, as well as the substantial investments in science and manufacturing, the upfront current cost of gene therapy appears to be generally high when spread across limited patient populations.

Canadian patients and healthcare providers expect and deserve the opportunity to receive these treatments as they are approved. Collaborative efforts and dialogue between health technology assessment bodies, payers, researchers, the patient community, and industry leaders, such as Pfizer Canada, are essential in navigating these challenges and realizing the full value of gene therapy innovation.

Building Canada's Gene Therapy Ecosystem
Canada must take strategic steps to build a thriving gene therapy treatment ecosystem. While many gene therapies remain in the research stage, companies are increasingly investing in these technologies for different uses and submitting applications for market authorization. Several products have already been approved in and outside of Canada or are in the advanced stage of clinical research.^[5]

The landscape of gene therapy in Canada must adapt to facilitate patient access to these potentially transformative treatments. Providing access to gene therapy should be based on a holistic look at the value of a therapy that is guided by the potential benefits and risks generated for patients, health systems and societies, as well as the cost saving for a potential long-term benefit. There can be multiple novel reimbursement approaches that take these factors into account. When used appropriately, novel financing models allow payers to manage clinical uncertainty, budget impact, and sustainability of the health care system, while providing recognition of the innovation being delivered for many rare diseases.

Pfizer’s Commitment
Cutting edge cell and gene therapies have the unprecedented opportunity of transforming healthcare. Canada has the potential to be at the forefront of this transformation through leveraging its research strengths, fostering stakeholder collaboration, and adopting supportive and proactive public policies.

Gene therapy development is complex, requiring specialized and novel methodologies, as well as a high level of expertise. Pfizer embraces this challenge as we embrace science and strive to cure and prevent human disease.

We’re going beyond helping people manage their diseases. We want them to thrive in every stage of life.


[1] NIH Genetics Home Reference. What is Gene Therapy? https://ghr.nlm.nih.gov/primer/therapy/genetherapy. Accessed February 10, 2020.
[2] NIH Genetics Home Reference. How does Gene Therapy Work? https://ghr.nlm.nih.gov/primer/therapy/procedures. Accessed February 10, 2020.
[3] NIH Genetics Home Reference (GHR). What is Gene Therapy? https://ghr.nlm.nih.gov/primer/therapy/genetherapy. Accessed February 10, 2020.
[4]  Your Genome. Facts: What is Gene Therapy? http://www.yourgenome.org/facts/what-isgene-therapy. Accessed February 10, 2020.
[5] Gene Therapy: An Overview of Approved and Pipeline Technologies, CADTH https://www.cadth.ca/gene-therapy-overview-approved-and-pipeline-technologies. Accessed July 18, 2023