ABSTRACT
Millions of people in the United States and many more throughout the world are afflicted by one of the estimated 5000 to 8000 rare diseases or conditions that have been identified to date.1 While some diseases such as cystic fibrosis or hemophilia are well characterized, little is known about the vast majority of rare diseases. Despite the great need for novel safe and effective treatments for these diseases or conditions, the small patient populations may not be perceived by sponsors as representing viable markets to make a profit or even recoup the costs of research and development. Sponsors may also be wary in instances where the drug may not be patentable. Thus, even with the existence of drugs with potential uses for rare diseases or conditions, there may be reluctance to “adopt” them for commercial pursuit. As such, these drugs have come to be known as “orphan”
Introduction ....................................................................................................... 189 The Orphan Drug Act of 1983 and Its Amendments ........................................ 190 Incentives for Orphan Drug Development .........................................................191
