ABSTRACT
When one hears the word “orphan,” it may conjure up something out of a Dickens novel-a picture of a homeless, ragged urchin cowering in a doorway in a cold, deserted alley. Unfortunately, relative to orphan diseases and the drugs to treat them, the metaphor has been all too close to the reality of the situation. Many diseases have gone wanting for treatment simply because it is not economically feasible to develop drugs for them. The federal government, however, has the means to make it more attractive to develop drugs for this lonesome group of diseases. In 1983, the Orphan Drug Act created muchneeded changes to provide resources that will develop products to treat diseases and conditions. It has had worldwide impact; orphan drug legislation has been enacted in Japan and is being considered in the European Union [1]. Interestingly, although the FDA has an orphan drugs directorate and advisory body, one will not find the term “orphan drugs” in any Act of the United States, even though, as noted below, these entities are covered.
