An Orphan Drug Framework (ODF) for Canada

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David K Lee
Barbara Wong


orphan drug, canada, regulation


A rare disease is a life - threatening, seriously debilitating, or serious and chronic condition that affects a relatively small number of patients ( no more than 1 in 2,000) . 1 There have been 6,000 to 7,000 rare diseases identified worldwide . 1 An estimated 80% of rare diseases are genetically - based, while the remainder are a result of viral or bacterial infection or other environment causes. More than half of rare diseases start in early childhood 2 and are degenerative and life - threatening in nature. Although the number of patients affected by each rare disease is small, total number of patients affected by the thousands of rare diseases identified is large. In Canada, one in every 12 Canadians is affected by a rare disease .

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