Published online April 2, 2007
PEDIATRICS Vol. 119 No. 4 April 2007, pp. 785-789 (doi:10.1542/peds.2006-0646)
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SPECIAL ARTICLE

Creation and Development of the Public Service Orphan Drug Human Botulism Immune Globulin

Stephen S. Arnon, MD

Infant Botulism Treatment and Prevention Program, California Department of Health Services, Richmond, California

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.

Key Words: infant botulism • botulism immune globulin • BabyBIG • botulinum toxin • botulism • orphan drugs • orphan diseases • drug development

Abbreviations: FDA—US Food and Drug Administration • BIG—Botulism Immune Globulin • BIG-IV—Botulism Immune Globulin Intravenous (Human) • CDHS—California Department of Health Services • IND—investigational new drug • OOPD—Office of Orphan Products Development • IRB—institutional review board • CDC—Centers for Disease Control and Prevention




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R. Koepke, J. Sobel, and S. S. Arnon
Global Occurrence of Infant Botulism, 1976-2006
Pediatrics, July 1, 2008; 122(1): e73 - e82.
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