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Canadian Rare Disease Patients Are Getting Impatient — Here’s Why

Durhane Wong-Rieger

President & CEO,  Canadian Organization for Rare Disorders

The Canadian Organization for Rare Disorders is urging the Canadian government to accelerate its National Strategy for Drugs for Rare Diseases implementation.

In 2005, Ontario ombudsman Andre Marin issued a clarion call on behalf of a rare disease community that was struggling to make its voice heard. The issue was the Ontario government’s delay in implementing newborn screening. Ombudsman Marin legitimized our patient “right to be impatient.” He unflinchingly denounced the “consequence of politicians and bureaucrats failing to sense the urgency and […] to remedy the situation peremptorily.” 

Fast forward to 2023, days after Rare Disease Day. The federal government announced $1.5 billion to make good on a 2019 promise to implement a national rare disease drug strategy to “increase access to promising and effective drugs for rare diseases.” The Canadian Organization for Rare Disorders (CORD) and the entire rare disease community cheered. We proposed an initial list of drugs that save or extend patient lives. They reduce the risk of disability, catastrophic harm, or life-threatening surgery. Already approved and recommended, these drugs are stalled by bureaucratic inertia: in price negotiations with the pan-Canadian Pharmaceutical Alliance, in budget impact assessments with provincial Health Ministries, or with drug plan managers who claim no more monies to allocate. Meanwhile, the $1.4 billion designated for drug funding appears to be hopelessly stalled in federal-provincial negotiations. 

Urgent action is needed

Patients whose lives and well-being are at risk have the right to be impatient. The governments can and must act to make urgently needed drugs immediately available. The CORD has proposed an implementation plan in which rare disease therapies that are approved and recommended can be started immediately with a defined cohort of patients along with a plan for ongoing monitoring, data collection, and assessment of benefit and potential harm.

Ombudsman Marin’s opinion is worth repeating: “Government responsibilities to citizens are owed at large, not in isolation by divisions of government. The right thing to do […] is to discharge those responsibilities faithfully and with commitment, while trying to achieve reorganization. It is never appropriate to abdicate those responsibilities while waiting for reorganization to occur.” This was true in 2005, and it’s true in 2024. 

To learn more, visit raredisorders.ca.

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