{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,3]],"date-time":"2026-10-03T16:21:37Z","timestamp":1791044497293,"version":"4.1.0"},"reference-count":25,"publisher":"American Academy of Pediatrics (AAP)","issue":"4","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2007,4,1]]},"abstract":"<jats:p>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.<\/jats:p>","DOI":"10.1542\/peds.2006-0646","type":"journal-article","created":{"date-parts":[[2007,4,2]],"date-time":"2007-04-02T12:23:29Z","timestamp":1175516609000},"page":"785-789","source":"Crossref","is-referenced-by-count":62,"title":["Creation and Development of the Public Service Orphan Drug Human Botulism Immune Globulin"],"prefix":"10.1542","volume":"119","author":[{"given":"Stephen S.","family":"Arnon","sequence":"first","affiliation":[{"name":"Infant Botulism Treatment and Prevention Program, California Department of Health Services, Richmond, California"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"417","published-online":{"date-parts":[[2007,4,1]]},"reference":[{"key":"2021120823321204100_R1","unstructured":"Orphan Drug Act, US Pub L No. 97-414 (1984, as amended); 21 USC \u00a7360aa et seq"},{"key":"2021120823321204100_R2","doi-asserted-by":"crossref","unstructured":"Haffner ME. Adopting orphan drugs: two dozen years of treating rare diseases. N Engl J Med.\u20082006;354:445\u2013447","DOI":"10.1056\/NEJMp058317"},{"key":"2021120823321204100_R3","unstructured":"National Organization for Rare Disorders. NORD Guide to Rare Disorders. Philadelphia, PA: Lippincott Williams & Wilkins; 2003"},{"key":"2021120823321204100_R4","unstructured":"National Organization for Rare Disorders. Public policy priorities: update from NORD's Washington office. Orphan Dis Update.\u20082005;23:16"},{"key":"2021120823321204100_R5","doi-asserted-by":"crossref","unstructured":"Arnon SS, Schechter R, Maslanka SE, Jewell NP, Hatheway CL. Human Botulism Immune Globulin for the treatment of infant botulism. N Engl J Med.\u20082006;354:462\u2013471","DOI":"10.1056\/NEJMoa051926"},{"key":"2021120823321204100_R6","doi-asserted-by":"crossref","unstructured":"Black RE, Gunn RA. Hypersensitivity reactions associated with botulinal antitoxin. 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