Creation and development of the public service orphan drug human botulism immune globulin

The public service orphan drug Human Botulism Immune Globulin for the treatment of infant botulism would not have come into existence without the federal Orphan Drug Act and the funding mechanism that it provided to conduct pivotal clinical trials. Nonetheless, creating, developing, and achieving licensure of Human Botulism Immune Globulin took approximately 15 years and approximately $10.6 million (2005 dollars) to accomplish. Use of Human Botulism Immune Globulin to treat patients with infant botulism has resulted thus far in more than 30 years of avoided hospital stay and more than $50 million (2005 dollars) of avoided hospital costs. To provide a possible paradigm for others, the circumstances that enabled a state public health department to create, test, license, and distribute an orphan drug are described here.