Health Canada releases What We Heard Report from the Public Engagement on the National Strategy for Drugs for Rare Diseases

Too many Canadians are struggling to access the drugs they need, especially if they have a rare condition. Work continues on the national strategy to support Canadians who rely on these drugs. Today, we are releasing the What We Heard Reportfrom the public online engagement for theNational Strategy for Drugs for Rare Diseases, which showcases Canadians’ feedback on how we can ensure rare disease patients get better access to the effective drugs they need.

From January 27 to March 26, 2021, Health Canada undertook public and stakeholder engagement to seek ideas and views on what a national strategy could look like. Canadians were invited to participate by completing an online questionnaire, sending a written submission by email or mail, or attending one of five virtual public town halls. Virtual meetings were also organized with stakeholders.

The engagement garnered diverse perspectives from over 650 individuals and organizations, including patients with lived experience, family members, and caregivers. Throughout the engagement, participants overwhelmingly felt a national approach was the most important element for a strategy and should be guided by key principles and values such as transparency, accountability, flexibility and being patient-centred. The report, which is available on our website, reflects these key themes and feedback.

Health Canada will use the What We Heard Report to closely review the ideas and suggestions gathered from the consultations to inform the development of the national strategy. The federal government will continue working closely with provinces and territories, health system partners, Indigenous partners and other stakeholders to build a strategy that works for all Canadians. Health Canada will conduct a series of in-depth follow-up discussions with partners and stakeholders on various elements of the What We Heard Report over the summer, and a public consultation will take place this fall to inform a comprehensive model for the national strategy. Our website will be updated with more information when available.

With the support of all those involved, the Government of Canada will work towards the launch of a national strategy in 2022.

Quotes

“Too many Canadians struggle to access the drugs they need, especially if they have a rare condition. I would like to thank the many Canadians that provided their valuable insights through these consultations and engagements. Your views and feedback will guide us as we develop a national strategy that provides support for Canadians to get better access to rare-disease treatments.”

The Honourable Patty Hajdu
Minister of Health

Quick facts

• In January 2021, Health Canada launched a national online engagement to let Canadians—especially patients with rare diseases, their families and other interested stakeholders—share their views and ideas for a national strategy. 
• Over 650 individuals and organizations participated in 21 virtual public town halls and stakeholder meetings. Health Canada received nearly 140 online questionnaire responses, and 100 emailed submissions.
• Participants voiced that a single national framework for rare disease treatments would make access fairer and more consistent across the country. 
• Participants were supportive of building a better evidence-base for decision-making and taking an innovative approach to approval and coverage for drugs for rare diseases. 
• Budget 2019 announced up to $1 billion over two years, starting in 2022-23, with up to $500 million per year ongoing, to help Canadians with rare diseases access the drugs they need. This includes working with willing provinces, territories and stakeholders to establish a national strategy for drugs for rare diseases.
• Rare diseases are life-threatening, seriously debilitating and sometimes chronic in nature. They are often genetic conditions, with onset either at birth or early childhood.
• It is estimated that there are between 6,000 and 8,000 different rare diseases that have been identified to date. They are considered rare because they each affect small numbers of people, ranging from a handful of individuals to a few thousand.
• The prices of drugs for rare diseases can range from $100,000 to more than $2 million yearly, per patient.