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Report On Orphan Drugs In Development For Rare Diseases

October 5, 2012

      PhRMA wrote a thorough and fascinating January 2011 Report titled, “Orphan Drugs in Development For Rare Diseases”. Interesting statistics from this report :

–    460 drugs in development to treat or prevent rare diseases

–    25 – 30 million Americans have 1 of the nearly 7,000 rare diseases

–    Since 1983, > 350 drugs have been approved to treat rare diseases

–    In the 1970’s, < 10 drugs were approved to treat rare diseases

–    Under the US Orphan Drug of 1983, 2,313 drugs have received FDA Orphan Drug Designation (ODD) as of January 2011.

       The Report breaks down by therapeutic category (i.e. Gastrointestinal Disorders, Growth Disorders, etc.) each of the 460 orphan drugs in development.  For each orphan drug the following information is provided :

–    Product Name

–    Sponsor or Developer

–    FDA Official Treatment Designation

–    Development Status.     

     On page 44 of the Report, there is a chart and description of the drug discovery, development, and approval process. 

Copyright  © 2012-2013, Ann-Teresa Cusenza and Orphan Druganaut Blog.  All rights reserved.

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