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Defeat Duchenne Canada: National Rare Disease Drug Strategy Update

March 22, 2023

Defeat Duchenne Canada has been fighting for timely access to affordable treatments for Duchenne muscular dystrophy across Canada. Today, the federal government announced measures supporting the first-ever National Strategy for Drugs for Rare Diseases, with an investment of up to $1.5 billion over three years, bringing us one step closer to our goal.

The plan is designed to provide earlier and more equitable access to diagnosis and affordable treatments and will be developed and executed in collaboration with the drug programs in the provinces and territories and the Non-Insured Health Benefits (NIHB) program covering Indigenous Canadians with rare disorders.

Federal Health Minister Jean-Yves Duclos made the announcement of the new funding, accompanied by Durhane Wong-Rieger, President of the Canadian Organization for Rare Disorders (CORD), and patient leaders from the Regroupement québécois des maladies orphelines (RQMO) and Best Medicines Coalition.

“Access and affordability should never be a barrier to care. Through this new Strategy, Canadians living with a rare disease will soon have improved screening and diagnostics, and better access to affordable and effective treatments they need, no matter where they live. We will keep working with provinces and territories and all relevant partners so those living with a rare disease, including children, have the best health outcome possible while their caregivers get the support they need and deserve.”

The Honourable Jean-Yves Duclos, Minister of Health

“CORD is especially gratified that the federal government heard our recommendations for a comprehensive strategy that includes investment in infrastructure, including centres of expertise, that will facilitate faster diagnosis and treatment access with on-going patient monitoring and data collection. This is a major step to value-based pharmacare and healthcare,” said Wong-Rieger.

 Durhane Wong-Rieger, President of CORD

As part of this overall investment, the Government of Canada will make available up to $1.4 billion to provinces and territories through bilateral agreements. This funding will help provinces and territories improve access to new and emerging drugs, as well as support enhanced access to existing drugs, early diagnosis, and screening for rare diseases. This will help patients with rare diseases, including children, have access to treatments as early as possible for a better quality of life.

As a critical next step to advance the development of these bilateral agreements, the Government of Canada will engage with provinces and territories to jointly determine a small set of new and emerging drugs that would be cost-shared and covered in a consistent way across the country for the benefit of patients. Similarly, to further support eligible First Nations and Inuit patients living with rare diseases, $33 million from this funding will go to Indigenous Services Canada’s Non-Insured Health Benefits Program.

In addition, the Government of Canada will also invest $68 million in various initiatives to help improve consistent access to drugs for rare diseases across the country, including:

  • $20 million over three years to the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Canadian Institute for Health Information (CIHI) to improve the collection and use of evidence to support decision-making;
  • $32 million over five years to the Canadian Institutes of Health Research (CIHR) to advance rare disease research with a focus on developing better diagnostic tools and establishing a robust Canadian rare disease clinical trials network; and,
  • $16 million over three years to support the establishment of national governance structures, such as a Health Canada secretariat and a stakeholder Implementation Advisory Group, to support the implementation of the Strategy.

Defeat Duchenne Canada has worked with CORD and Health Canada throughout the last decade, providing extensive feedback from the Canadian Duchenne muscular dystrophy community. Through sharing the lived experiences of our families, healthcare professionals, researchers, and supporters, our voices have been heard and proudly aided in the creation of this strategy.

We look forward to sharing further information on the development and implementation of this strategy, as it brings hope to all Canadians living with rare diseases like Duchenne.

Background: In 2019, the federal government committed $1 billion over two years to set up a national Rare Disease Drug Strategy, with an additional $500 million per year to improve Canadians’ access to drugs treating rare diseases like Duchenne. Three years later, after participating in numerous consultations led by the Canadian Organization for Rare Disorders (CORD) and Health Canada, our community was still waiting – until today.