Standing Committee on Health

Comité permanent de la santé

For immediate release

Ottawa, February 28, 2019 -

Bill Casey, Chair of the House of Commons Standing Committee on Health, presented the Committee’s twenty-second report today entitled, Canadians Affected by Rare Diseases and Disorders: Improving Access to Treatment.

In presenting the report in the House, Mr. Casey emphasized that “This report is an important step forward in supporting access to treatments for rare diseases in Canada. I want to thank my colleagues on the Health Committee for their work in making this enlightening study possible. Our hope as a Committee is that Canadians battling rare diseases have access to more affordable treatments going forward, and we are happy to work with partners to make this possible.”

Drawing on witness testimony and briefs, the Committee’s report examines the key challenges Canadians with rare diseases and disorders face in accessing treatment. These challenges include barriers in the process to approve drugs for rare diseases for sale in Canada; the high prices of these drugs; reimbursement for the cost of these drugs through provincial and territorial drug coverage plans; and early access to diagnostic tests.

Witnesses explained to the Committee that addressing these barriers is an urgent matter. Rare diseases are life-threatening and debilitating conditions. They most often affect children and account for one-third of deaths in the first year of life. For 94% of these conditions, there is currently no cure.

The Committee believes that there is no time to waste in addressing the critical issues that Canadians with rare diseases and disorders face in accessing treatment. Its report therefore outlines 19 recommendations that focus on:

• better coordination of regulatory approval and reimbursement processes for drugs for rare diseases;

• reducing the prices of drugs for rare diseases;

• covering the costs of drugs for rare diseases; and

• collecting real world evidence on the safety, efficacy and cost-effectiveness of drugs for rare diseases.

The Committee believes that the federal government needs to play a leadership role in working together with provincial and territorial governments, pharmaceutical manufacturers, patients and health care providers to implement these recommendations.