Orphan Diseases, Drugs for

In the United States, diseases that affect fewer than 200,000 people are referred to as rare or orphan diseases. This definition of rare diseases varies in Europe and other countries, including Japan and Australia. There are more than 7,000 such diseases, and they are usually complex, often have a genetic origin, and are life threatening. More rare disorders being discovered every day.

A drug or a biological agent that is intended for the safe and effective treatment or in the diagnosis or prevention of such rare diseases is termed as an orphan drug. The development of orphan drugs began in 1983 with the creation of the Orphan Drug Act. Before the Orphan Drug Act was passed, only 10 new drugs were developed for rare diseases. Currently, ...