Research Paper

Successfully Commercializing Orphan and Ultra-Orphan Drugs

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Orphan and ultra-orphan diseases are the less traveled but increasingly promising path for the biopharmaceutical industry. Although small in patient population compared to more recognized specialty diseases, the 7,000 rare diseases identified today affect up to 30 million patients in the US, almost 10% of its population. A startling 95% of these diseases still lack a medication or therapy, and diagnosis can take five to seven years on average, as they are not top-of-mind for physicians.

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