The Orphan Drug Act of 1983 defines a rare or orphan disease as one diagnosed in fewer than 200,000 people in the United States. This may seem like a small number relative to the overall U.S. population, but with more than 7,000 different rare diseases, the numbers add up.
In all, 25 million Americans-nearly 1 out of every 10 people in the country-suffer from a rare disease. Their conditions range from the more familiar to those largely unknown, including:
Bringing a new drug or therapy to market requires a significant investment of time and money. In exchange, companies expect to make a profit. But when no more than 200,000 people need a product, companies lack a financial incentive to pursue drug discovery, development and approval. As a result:
KGI’s Center for Rare Disease Therapies brings attention to the impact and severity of rare diseases in the United States. The center elevates the need for treatment to a problem of national importance that calls for an effective response.
Celebrating 25 years of innovation! 25.kgi.edu.