title: Creation and development of the public service orphan drug Human Botulism Immune Globulin.
abstract: 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.
authors.affiliation: Infant Botulism Treatment and Prevention Program, California Department of Health Services, 850 Marina Bay Pkwy, Room E-361, Richmond, CA 94804, USA. sarnon@dhs.ca.gov
authors.forename: Stephen S
authors.initials: SS
authors.lastname: Arnon
mesh_terms: D001906:Botulism / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality; D002986:Clinical Trials as Topic / Q000191:economics; D017046:Cost Savings; D005260:Female; D005380:Financing, Government / Q000331:legislation & jurisprudence*; D006078:Government Programs / Q000458:organization & administration; D006801:Humans; D007136:Immunoglobulins / Q000191:economics* / Q000494:pharmacology / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D020380:Needs Assessment; D009965:Orphan Drug Production / Q000191:economics / Q000331:legislation & jurisprudence*; D015397:Program Evaluation; D011635:Public Health Administration; D012109:Research Support as Topic; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D007136:Immunoglobulins; C068584:botulism immune globulin