“Success” in the world of orphan drug development: it’s a matter of perspective
According to data from a variety of sources, there are between 6,500 and 7,000 disorders that meet the criterion for definition as an “orphan disease” here in the USA: a prevalence of < 200,000 patients living with the particular disorder at any specific point in time. To date, the U.S. Food & Drug Administration (FDA) […]