More than three million Canadians are living with a rare disease. Many have limited or inadequate access to life-saving treatments - this is a gap in our health care system that deserves immediate attention.While there has been much progress in raising the profile of this health challenge, Canada remains the only G7 country without an official dedicated framework or even a standard definition of 'rare disease.'For those vulnerable Canadians impacted by rare disease, a pan-Canadian rare disease strategy is critical. Currently, those with a rare disease must deal with a complex and often difficult journey towards diagnosis. With few support networks, they face great difficulty accessing care and medical expertise.The Government of Canada has earmarked $1 billion over two years, starting in this year to 2023, with up to $500 million per year ongoing for rare disease. It's a positive step in the right direction, but we need a greater sense of urgency to accelerate the development of a formal rare disease strategy and gain clarity on where these funds will go.Thankfully there has been progress - Health Canada has conducted stakeholder consultations. Over 650 clinicians, patients, caregivers, the pharmaceutical industry, and payers have shared their views on how our health systems can better serve Canadians with rare diseases. Amongst them, Pfizer has shared its opinions, including:
- Canada must adopt a definition of rare disease that closely aligns with the international community. Canada should set upon an inclusive process to create a benchmark – one that includes the voices of patients, scientific experts, and health system stakeholders.
- Canada needs a tailored review process for rare diseases. It is critical to have an agile, predictable, and efficient approach to reviewing rare disease drugs in Canada. This approach must engage relevant stakeholders in a structured and inclusive way, considering all relevant factors to rare disease medicines.
- We need open-sourced, dynamic and internationally aligned disease registries. This will ensure all real-world evidence is collected and available for analysis at regular intervals.