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Harper Government Takes Action to Help Canadians with Rare Diseases - Launch of First Ever Canadian Framework to Increase Access to New Treatments and Information and Orphanet-Canada Online Portal

October 3, 2012
For immediate release

Factsheet: An Orphan Drug Framework for Canada

Factsheet: Orphanet-Canada

OTTAWA - The Honourable Leona Aglukkaq, Minister of Health, today announced two initiatives aimed at bringing new hope to Canadians with rare diseases: the Harper Government will create a new approach for the authorization of "orphan drugs" and announced the Canadian launch of Orphanet - a comprehensive database of information and services for rare diseases.

"Too often, Canadians dealing with rare diseases are faced with difficulties in accessing the information and medication they need," said Minister Aglukkaq.  "I am pleased to announce that our Government will introduce a new approach that will better support the development and authorization of drugs for rare diseases and launch of a new Web portal to assist patients in finding the information and services they need." 

A rare disease is a life-threatening, seriously debilitating, or serious chronic condition that only affects a very small number of patients.  It's estimated that there over 7,000 unique rare disorders, many of which are genetic. While some of these rare disorders may affect only a handful of Canadians, in all, hundreds of thousands of Canadians are dealing with these conditions and need effective treatments.  Drugs used to treat rare diseases are often referred to as orphan drugs.

Today, when an orphan drug is not available in Canada, the patient's doctor can apply individually for each patient through Health Canada's Special Access Programme. While facilitating access - the current approach also represents a significant burden to the healthcare system. A regulatory framework designed and used specifically to approve drugs to treat small, vulnerable populations will more effectively address this need. A key focus of this new approach will be on international information-sharing and collaboration for the development and regulation of orphan drugs.

The new framework will be designed to spur research and innovation in Canada. Building on existing international experience and knowledge, it will provide Health Canada with new tools to gather and share information, including the registration of clinical trials.  It will also facilitate patient participation in this highly specialized area.

"On behalf of the 2.8 million Canadians with rare disorders, the Canadian Organization for Rare Disorders is extremely grateful to Minister Aglukkaq, to the Members of Parliament, and to Health Canada and CIHR for sponsorship of Orphanet Canada and the proposed regulatory framework to support innovative research and approval of drugs for small patient populations.  We are especially pleased to have participated in the consultations toward the framework and look forward to the regulations being enacted as soon as possible."

Orphanet is the world's online reference portal for information on rare diseases.  Its comprehensive database of information contributes to improvements in the diagnosis, care, and treatment of patients with rare diseases.  With support from the Canadian Institutes of Health Research (CIHR), the portal is now available in Canada.  It will provide Canadians with expert, peer-reviewed information on rare diseases, as well as a specialized services directory.  

"With this launch, Canada becomes the first country from North- or South America to participate in this exciting initiative," said Dr. Paul Lasko, Scientific Director of the CIHR Institute of Genetics.  "There are many Canadian families which are affected by one of the several thousand different rare diseases.  Through this one user-friendly site, these families will now benefit from services such as specialized clinics, medical laboratories, clinical trials, and registries."

For more information, visit the Next link will take you to another Web site Orphanet-Canada website.

Lab technician Megan Vanstone, Minister Aglukkaq, and Sean Grover, a young patient with a rare disease, conduct a lab test.

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Media Enquiries:
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Canadian Institutes of Health Research
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mediarelations@cihr-irsc.gc.ca

Health Canada Media Relations
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Cailin Rodgers
Office of the Honourable Leona Aglukkaq
Federal Minister of Health
(613) 957-0200

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