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Challenging Conventional Patient Recruitment And Care Strategies For Rare Disease Studies

Read this article to find out more about orphan diseases.

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The number of orphan drug designations doubled between 2007 and 2014; and drugs for rare diseases accounted for 41% of new FDA-approved drugs in 2016. Aided by a variety of supportive FDA policies, orphan drugs have had a higher overall success rate than their counterparts from 2006-2015: 25% of orphan drugs advanced successfully from Phase I to FDA approval versus only 9.6% of nonorphan drugs, spurring investment from companies and giving new hope to patients suffering from heretofore untreatable diseases. Read this article to find out more.