by Makeuswell

The “Rare” Or “Orphan” Disease Market Opportunity

Rare diseases affect 30 million people in the USA. And more than 300–400 million worldwide. They often cause chronic illness, disability, and premature death… A large number of rare disease patients [estimated 65-75%] remain undiagnosed for years and many even die without an accurate diagnosis. 

— A guide for the diagnosis of rare and undiagnosed disease

“Orphan Drugs” are defined as drugs with a target patient population of less than 200,000 people or more than 200,000 people, but with a finding that research and development costs are unlikely to be recovered. They are usually developed to treat rare diseases.

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