Enlarge this image The Food and Drug Administration has seen a sharp increase in applications for drug to treat rare diseases. An…
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Enlarge this image Dr. Scott Gottlieb, Food and Drug Administration commissioner, told Kaiser Health News the incentives intended to spur development of…
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Enlarge this image The Government Accountability Office will investigate potential abuses of the Orphan Drug Act. Ron Cogswell/Flickr hide caption toggle caption…
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Enlarge this image The list price for a year’s treatment with Emflaza is $89,000. The drug is available outside the U.S. for…
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Enlarge this image Senate Judiciary Committee Chairman Chuck Grassley, R-Iowa, is looking into how the Orphan Drug Act may be affecting a…
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Luke Whitbeck, 2, has Gaucher disease, a rare genetic disorder. Cerezyme, an orphan drug, has been a lifesaver for him. YouTube Before…
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More than 30 years ago, Congress overwhelmingly passed a landmark health bill aimed at motivating pharmaceutical companies to develop new drugs for…
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Enlarge this image Sarepta Therapeutics was awarded a voucher for a fast-track drug review by the Food and Drug Administration when the…
