INTRODUCTION The term “orphan drug” refers to a drug or biologic that treats a rare disease affecting fewer than 200,000 of the US population. Many diseases that fall into this statistic are not treated simply because it is not economically feasible to develop drugs for them. The Federal government had to find a way to make it more attractive for drug manufacturers to develop drugs for this group of diseases. As a result, former President Ronald Reagan signed into law on January 4, 1983, the Orphan Drug Act (ODA). This Act created much needed changes in order to provide resources that would allow the development of products to treat these rare diseases and conditions. The National Institute of Health (NIH) estimates that there are 6000 rare diseases affecting 25 million Americans. As a result, the ODA has become one of the most important pieces of health care legislation today and has had worldwide impact. In Japan, the term orphan drug describes 2.5 cases or less per 10,000 people. The European Union (EU) criterion for establishing an orphan drug is a condition with a population prevalence of five cases or less per 10,000. Orphan drug legislation has been enacted in Japan and has been in force since April 2000, and in the EU via the Committee on Orphan Medicinal Products of the European Medicine Agency (EMEA), aggressive steps in encouraging the development of orphan drugs have been implemented. However, each country makes its own decisions on the pricing and reimbursement of orphan drug products. In late 2007, the FDA and the EMEA had come to an agreement whereby the same application could be used for both agencies, thereby reducing the time and finances required of companies to apply for orphan drug status.a There are an estimated 7000 so-called “orphan diseases” identified around the world and they occur in more than one out of ten people. These patients faced very limited treatment for these diseases and most had a poor prognosis. With the ODA in the United States and the implementation of the
development of orphan drugs globally, approximately 1800 treatments have entered the research pipeline designated with orphan drug status and more than 300 products have been approved by FDA and other global agencies compared to a mere 10 products developed before 1983 (1).