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Background to EDRD Supplemental Process

Why Now? Canadians have the Opportunity of a Lifetime to design the world’s best Rare Drug Program directed to assure patients have access to today’s and tomorrow’s therapies. The Canadian government has committed $1 billion to set up Canada’s Rare Disease Drug Strategy, with on-going invest of $500 million each year. The Rare Drug Program will be grounded in Canada’s Rare Disease Strategy.

What’s the Vision? The Pan-Canadian Rare Drug Program will assure innovative and essential rare therapies are delivered to patients as soon as possible and consistent with Canadian principles of accessibility, comprehensiveness, universality, portability, and publicly accountable administration.

Evidence:

  • The lack of robust evidence to support efficacy, safety, and cost-effectiveness (due to small patient numbers and difficulties conducting well-designed clinical trials). 
  • No best practices for generating or evaluating real-world evidence, which could help inform appropriate funding decisions  where there is high uncertainty.

Pricing:
  • High drug costs threaten drug program sustainability and access for patients.
  • The high price[s] of EDRDs often does not appear to be justified.
  • Growing number of drugs for rare diseases [is] posing an increasing challenge for payers.

Access:
  • Complex/specialized drugs ... address unmet needs ... leading to greater urgency from clinicians and patients to obtain timely access.
  • There is variability between jurisdictions both in terms of funding availability and criteria for treatment.

Communications:
  • Perceived lack of transparency and communication between the national drug review processes and patients and clinicians.

Question Title

* 1. As a member of the Canadian Organization for Rare Disorders, you are invited to provide your opinion on a proposed process for providing access to rare disease drugs.  CORD has been asked to coordinate the feedback from member organizations.  We are reaching out to all members to inform you about the process and to solicit your feedback.  We will summarize all of the input and submit to the Provincial/Territorial EDRD Working Group.

To help us better interpret the responses, please indicate your PRIMARY affiliation with your patient organization or your role within the rare disease community.

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