On Jan. 3, 1983, the Orphan Drug Act was passed by the U.S. Congress with the leadership of Rep. Henry Waxman, to stimulate development of drugs and biological products for the treatment of rare diseases. Today, rare diseases affect over 25 million individuals in the United States that have historically lacked pharmaceutical development attention due to decreased potential for profit. This Act encouraged the research and development of drugs for rare or “orphan” diseases defined as a disease or condition that affects fewer than 200,000 Americans.

In the United States, between 6,000 and 8,000 diseases meet this condition, affecting an estimated 25 million people. Despite the large number of individuals affected by rare diseases, pharmaceutical companies have been reticent to focus their efforts on drug development for these conditions. They believed that they would not feasibly recover the development costs.  Examples range from ALS, Cystic fibrosis. and Duchenne, Fabry, and Gaucher disease to Huntington’s, Rocky Mountain spotted fever and Sickle cell disease to name a handful.

The law provided 7-year marketing exclusivity to sponsors of approved orphan products, a tax credit of 50 percent of the cost of conducting human clinical testing, and research grants for clinical testing of new therapies to treat orphan diseases.