Rare Diseases



Helping Canadians with Rare Diseases

 

GOVERNMENT OF CANADA RARE DISEASE STRATEGY OFFERS HOPE AND SOLUTIONS FOR PATIENTS


With the creation of a regulatory pathway for rare disease medicines, the Government will be providing patients with greater access to innovative treatments addressing their unique medical needs.

BIOTECanada strongly supports the Government’s initiative to make available to Canadian patients the best in rare disease patient care. Our member companies are primary innovators and developers of the biologic medicines used in treating rare diseases.

Orphan drugs are used in the treatment of rare disorders. These rare diseases affect less than 1 in 2000 people in Canada and are often severe, life threatening, and progressively debilitating genetic diseases. Biotechnology companies across Canada are developing medicines with the ability to offer hope to Canadian patients living with rare disorders. Our members are encouraged by today’s announcement to pursue developments of these rare medicines under favorable operating environment conditions here in Canada. 
 
Patients living with a rare disease overcome hurdles everyday. Important research in developing treatments for rare diseases is being done here in Canada in company laboratories and throughout the research community. The ability to bring those treatments to patients in Canada is an objective shared by government and industry.

With the intent to create a pathway for orphan medicines Canadian patients will now be on equal footing with other G20 countries. The initiative announced today will help Canada to compete in attracting investment to nurture those products into the marketplace, and see new Canadian solutions for unmet medical needs developed in Canada. The industry is encouraged by this initiative and looks forward to working with the Government to develop the policy framework.

Currently, 21 Canadian companies have received U.S. Food and Drug Administration Orphan Product designations for the products they are developing; four have received FDA product approvals. Canada has been alone among developed countries without a policy for rare disorders. The U.S. Congress passed the Orphan Drug Act of 1983 to stimulate the development of medicines for rare diseases. Japan introduced similar legislation in 1993, Australia in 1998 and the EU in 2000.




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