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In 1983 the U.S. enacted a law giving special protection from competition for drugs for rare diseases.
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The drug industry is currently trying out 300-plus drugs for rare diseases, more than double the number in 1989.
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The Orphan Drug Act, enacted in 1982, provides incentives--including tax credits and exemption from user fees--for the development of drugs for rare diseases.
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To address this shortfall, the National Institutes of Health announced last year that the new Therapeutics for Rare and Neglected Diseases Program would develop drugs for rare diseases.
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But in some cases, as with drugs for rare diseases or studies done in children, the FDA will lengthen this exclusivity, or grant it where no patent exists.
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The industry fees will also be used to advance the development of drugs for rare diseases, increase communications with small or new companies, and boost standardized electronic data usage to improve quality and effectiveness.
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As Genzyme did in its early days, BioMarin focuses on drugs for rare genetic diseases, usually those in which one enzyme is defective.
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Investors hoping Genzyme, the largest maker of drugs for rare genetic diseases, is the acquisition target purchased out-of-the-money call options on the stock.
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As Genzyme (nasdaq: GENZ - news - people ) did in its early days, BioMarin (nasdaq: BMRN - news - people ) focuses on drugs for rare genetic diseases, usually those in which one enzyme is defective.
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Such super-high-priced drugs for ultra-rare diseases are the biggest success story in pharma right now (see BioMarin and even Genzyme for other examples).
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First, drugs are increasingly a specialty business, with companies focusing on treatments for genetic diseases, uncommon cancers, and other rare ailments.
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