From The Industry: Building a national strategy to support Canadians with rare diseases

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.GovCanHealth has promised $500 million to help Canadians with rare diseases - via healthing_ca .RareDiseases raredisorders rarediseaseday MD_Canada healthing canada

Health Canada has promised $500 million to help Canadians with rare diseases, but success will depend on understanding the patient journey, innovative coverage processes and real-world data.To optimize patient access to these therapies, establishing an effective process to collect and analyze real-world data and evidence is key. GETTYIn some cases, it means a patient’s ability to receive primary care or a consultation from a specialist.

As Health Canada builds out the initial framework in collaboration with stakeholders across the industry, it is critical it considers the factors throughout the entire patient journey and implement an innovative approval and coverage process, with payment ideally tied to long-term, real-world studies on effectiveness and safety.Due to lack of disease understanding and awareness, it can take a patient, on average,to receive an accurate rare disease diagnosis.

Those barriers can be compounded by affordability-related challenges. As of 2019, there were 93 drugs for rare diseases approved in Canada that cost more than $100,000 per patient per year, according to the. The costs of these drugs can also pose a challenge to the long-term sustainability of government and employer-sponsored drug plans.

 

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