RARE DISEASE DEFINITIONS
The European Commission defines a rare disease as one that affects no more than 1 individual in 2000.3 In the European Union, rare diseases affect 6‒8% of the total population (around 27‒36 million people). Most rare diseases (80%) are of genetic origin; others may be degenerative or proliferative.4
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The United States Orphan Drug Act defines a rare disease or orphan disease as a condition that affects fewer than 200,000 individuals in the United States. It is estimated that between 25‒30 million Americans live with a rare disease.5
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Rare diseases in Japan, known as “rare and intractable diseases”, are defined as diseases with unknown aetiology with no effective treatment that present a major financial and psychological burden. Rare diseases in Japan are defined as affecting fewer than 50,000 patients in total in the country.6
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In Australia, orphan drugs are indicated as treatments for the prevention or diagnosis of a life-threatening or seriously debilitating condition. The prevalence threshold for orphan drug eligibility criteria is treatment of a condition that affects fewer than 5 in 10,000 (or 1 in 2000) individuals.7
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