FREE NEJM E-TOC    HOME   |   SUBSCRIBE   |   CURRENT ISSUE   |   PAST ISSUES   |   COLLECTIONS   |   Search Term  Advanced Search

Sign in | Get NEJM's E-Mail Table of Contents — Free | Subscribe

FOCUS ON RESEARCH Previous Volume 354:445-447 February 2, 2006 Number 5 Next

	Since this article has no abstract, we have provided an extract of the first 100 words of the full text and any section headings.

	Full Text PDF PDA Full Text Purchase this article Add to Personal Archive Add to Citation Manager Notify a Friend E-mail When Cited E-mail When Letters Appear Related Article by Arnon, S. S. PubMed Citation

In 1982, when the Orphan Drug Act was passed as an amendment to the Federal Food, Drug, and Cosmetic Act,¹ few suspected the extent to which this law would alleviate the plight of patients with rare diseases. The law defines an orphan drug as one with efficacy against a disease affecting fewer than 200,000 people in the United States or one that scientists and economists at the Food and Drug Administration (FDA) determine will not be profitable for seven years after FDA approval.² In the 24 years since this law was passed, 282 such drugs and biologic products, providing treatment . . . [Full Text of this Article]

Source Information

Dr. Haffner is the director of the Office of Orphan Products Development at the Food and Drug Administration, Rockville, Md.

This article has been cited by other articles:

• Anderson, G. F. (2009). Spurring New Research For Neglected Diseases. Health Aff (Millwood) 28: 1750-1759 [Abstract] [Full Text]  
• Dresser, R., Frader, J. (2009). Off-label prescribing: a call for heightened professional and government oversight.. J Law Med Ethics 37: 476-486  
• Zurynski, Y, Frith, K, Leonard, H, Elliott, E (2008). Rare childhood diseases: how should we respond?. Arch. Dis. Child. 93: 1071-1074 [Abstract] [Full Text]  
• Braiteh, F., Kurzrock, R. (2007). Uncommon tumors and exceptional therapies: paradox or paradigm?. Molecular Cancer Therapeutics 6: 1175-1179 [Abstract] [Full Text]  
• Arnon, S. S. (2007). Creation and Development of the Public Service Orphan Drug Human Botulism Immune Globulin. Pediatrics 119: 785-789 [Abstract] [Full Text]  
• Scheindlin, S. (2006). Rare Diseases, Orphan Drugs, and Orphaned Patients. Mol. Interv. 6: 186-191 [Full Text]