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The Power And Influence Of Patient Advocacy Groups And Venture Philanthropy On Orphan Drug Development

November 3, 2012

      The power and influence of patient advocacy groups and venture philanthropy on orphan drug development is tremendous. 

       In a September 17, 2012, article, the author Tim Freeman identifies and discusses several features of orphan drug development that is attractive for pharmaceutical companies.     

       One feature, is patient advocacy groups and venture philanthropy. Per Freeman,”…. these groups have an instrumental role in developing orphan drugs. Because rare diseases affect small numbers of patients, finding patients to enroll in studies can be difficult. However, advocacy groups can use established lines of communication to generate excitement about a novel therapy and educate potential patients about the importance of participating in clinical research .….”.   

        Some patient advocacy groups provide large amounts of money in both preclinical and clinical research programs studying new orphan drugs for rare diseases : 

–     The Cystic Fibrosis (CF) Foundation gave $75 million to Vertex Pharmaceuticals for the “collaboration of scientific, clinical, and financial support for the development of orphan drug Kalydeco (Ivacaftor)”  

–     Orphan Druganaut September 10, 2012 Blog Post titled,”The Human Side of an Orphan Drug” discusses Sarepta Therapeutics’ orphan drug Eteplirsen for Duchenne Muscular Dystrophy (DMD) based on an article in the Seattle Times.  This is an example where advocacy groups are looking and finding venues to speed the drug’s progress. The Duchenne Alliance, an advocacy group for DMD, “is talking to Sarepta about ways to support additional testing that would bring the drug to more advanced cases”. 

       Other recent actions of patient advocacy groups helping orphan drug development : 

–     The use of social media tools by patient advocacy groups to help get Vertex Pharmaceuticals’ orphan drug Kalydeco available to all who need it 

–     The Cystic Fibrosis (CF) Canada organization asking for patient input for Vertex Pharmaceuticals’ orphan drug Kalydeco. 


Orphan Druganaut October 5, 2012 Blog Post titled, ”Orphan Drug Company Genzyme’s 2012 Patient Advocacy Leadership (PAL) Awards” 

Orphan Druganaut October 3, 2012 Blog Post titled,”Patient Families, Advocacy Groups, And Celebrities Campaigning To Bring Kalydeco To The UK” 

Copyright  © 2012-2013, Orphan Druganaut Blog.  All rights reserved.

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