New Canadian rare disease strategy welcomed as offering hope and solutions for patients

The Canadian government this week announced a new national framework to improve access to “orphan drugs” for people who suffer from rare disorders. Orphan drugs are typically expensive and difficult to research and bring to market, given the small patient population in which they can be studied.

The new framework, announced by federal Health Minister Leona Aglukkaq, will provide a national strategy for authorizing and monitoring the use of orphan drugs as well as helping to spur R&D of new drugs to treat rare diseases. Ms Aglukkaq also announced that Canada will launch Orphanet, a site dedicated to improving diagnosis, care and treatment of rare diseases.

BIOTECanada, the national industry association representing biotechnology companies, welcomed the announcement, saying: “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,” commented Andrew Casey, president and chief executive of BIOTECanada, whose member companies are primary innovators and developers of the biologic medicines used in treating rare diseases.