Rare Disease Day 2014: Orphan Drug Trial Design and Analysis

Posted by Marc Gastonguay on May 31, 2017 11:45:27 AM

In recognition and support of Rare Disease Day, 2014, the scientists and staff at Metrum Research Group have focused the weekly journal club on articles related to this topic, for the month of February…

Though deemed “rare”, approximately 25—30 million Americans, and 300 million people worldwide, have been diagnosed with one of >6,800 known rare diseases. An estimated 50% of those affected are children. The prevalence of individual rare diseases can range from dozens in the US (progeria, hypophosphatasia, Niemann-Pick, for example), to thousands globally (such as, Crohn’s disease and cystic fibrosis). Although traditionally considered impractical for big pharma development, innovation and development of therapeutics for rare diseases have led to promising treatments for some diseases in recent decades. In the period of time from the passage of the Orphan Drug Act of 1983 until May 2010, the FDA approved 353 orphan drugs and granted orphan designations to 2,116 compounds. As of 2010, at 200 orphan diseases have become treatable (Armstrong). Still, rare diseases pose a critical unmet medical need.

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Topics: Rare Disorders

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