Despite surge in orphan drug designations, approvals still lag

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Access through your institution Buy or subscribe In 1983, US lawmakers passed the Orphan Drug Act to encourage pharmaceutical companies to pursue treatments for largely ignored diseases


affecting small populations. And for the next 15 years or so, the number of rare diseases given orphan drug status hovered between about 40 and 80 per year. But over the last decade, that


number began steadily increasing, and last year the US Food and Drug Administration (FDA) granted a record 192 designations. Developing orphan drugs “makes a lot of business sense if you


don't have a blockbuster,” says Syamala Ariyanchira, an independent Malaysia-based pharmaceutical consultant who estimates that the orphan drug market will exceed $80 billion this year.


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calculated during checkout ADDITIONAL ACCESS OPTIONS: * Log in * Learn about institutional subscriptions * Read our FAQs * Contact customer support Authors * Monica Heger View author


publications You can also search for this author inPubMed Google Scholar RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE Heger, M. Despite surge in


orphan drug designations, approvals still lag. _Nat Med_ 17, 236 (2011). https://doi.org/10.1038/nm0311-236b Download citation * Published: 07 March 2011 * Issue Date: March 2011 * DOI:


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