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HomeOur VoicePfizer Canada’s Recommendations Regarding The Development Of A National Strategy For Drugs For Rare

Tuesday, June 7, 2022

While many countries have moved towards a Rare Disease framework to facilitate access to innovative medicine for patients with rare diseases, there is an opportunity for Pfizer Canada to play a role in defining a sustainable Rare Disease framework for Canada, working collaboratively with Health Canada, pharmaceutical industry partners, patient groups and clinicians. The following are the key questions related to our work in Canada.

What’s the situation?

Rare disease therapies face unique regulatory, health technology assessment (HTA) and reimbursement challenges. Many aspects of the current Canadian system are designed with other purposes and medicines in mind, and not taking into the unique nature of rare disease innovative medicines. Consequently, the availability of rare disease treatments is often delayed and inconsistent across the country.

Canadians with rare diseases encounter a wide range of challenges in managing their health. The screening, detection and diagnosis of many rare conditions may not always be timely.

Canada continues to face inherent challenges to rare disease research. Concerted work to nurture and sustain recognized expertise, including centers of clinical excellence for rare diseases, is a key component of managing these issues.

Pfizer’s leadership in Rare Disease is grounded in our commitment to scientific innovation as an enabler to improve patients’ lives. In addition to several currently marketed therapies, our rare disease pipeline includes clinical programs for hemophilia A and B, Duchenne muscular dystrophy (DMD), dilated cardiomyopathy due to lamin A/C gene mutation as well as many other rare disease research programs.

Why is Pfizer Canada engaged in this issue?

There is an urgent need to lead the conversation towards a sustainable rare disease strategy in Canada. Optimizing patient access and transforming the Canadian treatment landscape in Rare Disease are priorities. This is a key opportunity to define the required policy elements to improve the ability of all rare disease stakeholders to collaborate and build a better health system for Canadians with rare diseases.

This current consultation is an important recognition from the Government of Canada that continuing to engage an extensive stakeholder cohort is required to build and implement a sustainable rare disease framework. 

As we’ve seen with the COVID-19 vaccines and therapies, there are many benefits of this level of collaboration for Canadians. First and foremost, having access to the therapies they need can help Canadians live healthier and more productive lives. Caregiver and healthcare system burdens can be reduced. According to an October 2021 IQVIA study of 11 developed countries, Canada already has the second lowest expenditure on pharmaceuticals as a percentage of total health spending.[1] With spending well below average, Canada has room to grow its investments in the most value-added therapies to improve health and budgetary outcomes across the health system. A healthy population will contribute to the country’s economic prosperity and the future performance of our health system.

We can position our sector as a central driver of improved health outcomes and greater economic security for all Canadians by prioritizing regular interactions with each other, putting in place policies that enable innovation adoption, and establishing timely value-based procurement practices.

Meaningful collaboration is required. The importance of a modern and resilient healthcare system must be a priority.

What are the top areas of focus for Pfizer Canada?

Patient care at the centre of any policy – Aiming at greater leadership and alignment throughout the development, review, and commercialization process of new products for rare diseases with a patient-centric mindset. The focus should start from a holistic and inclusive consideration that is based on the patient perspective and need.

Embed and utilize the best available expertise - There are multiple opportunities for Canada to learn from global best practices to build a more inclusive and transparent healthcare system for rare disease patients. Patients and patient groups are key in raising awareness and advocating for pan-canadian supportive policies. Stakeholders are critical partners and should be viewed as mandatory participants in supporting better decisions and a better policy.

Dedicated and tailored review process - Canada has been resistant to setting out distinct, separately calibrated methods for the evaluation and reimbursement of rare disease innovations. This must change given the unique aspects of rare diseases.

Best available evidence - A more dynamic assessment model is warranted. Budget and value considerations need to be more flexible and tied to evolving information, rather than allowing perceived limitations stand as a barrier to evaluation, reimbursement and improved patient care.

Novel reimbursement models to advance timely access - A Canadian rare disease policy should incorporate a commitment to collecting and assessing the best available global evidence for rare diseases at all stages over the product lifecycle, including real-world data collected in the post-market space.

Why is a Canadian Rare Disease Framework so important?

Science is now offering the possibility of new treatments for many medical conditions with unmet needs, including rare diseases. In the absence of a dedicated policy framework, the complex Canadian regulatory and reimbursement system has struggled to balance the dual imperatives of providing timely patient access in an equitable and sustainable manner.

[1] IQVIA, « Drug Expenditure Dynamics 1995-2020 : Understanding medicine spending in context,, Accessed April 2022

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