Orphan drugs in the United States of America

As early as 1983, the Public Health authorities realised that legislation on orphan drugs was needed signing of the ‘Orphan Drug Act’. This law defines the ‘orphan drug’ with regard to prevalence (frequency) of the disease for which it is indicated in the American population. In the US, the concept of ‘orphan drug’ does not simply cover pharmaceutical or biological products. It also covers medical devices and dietary or diet products. The OOPD (Office of Orphan Products Development) was created within the FDA (Food and Drug Administration). It is in charge of promoting the availability of safe and efficacious products for the treatment of rare diseases. The ‘orphan’ status allows the drug sponsor to benefit from incentives for the development of these products until the marketing approval.