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The Role of Patient Advocacy Groups in Rare Disease Drug Development

Partnering with patient advocacy groups can lead to significant improvements in rare disease drug development.

Patient advocacy groups are an essential yet often underutilized partner in accelerating drug development in the rare disease space.  In this webinar, Raymond Huml, Vice President of Medical and Scientific Strategy and Head of the Rare Disease Consortium, shares his experience in working with specifically with muscular dystrophy patient advocacy groups at the 2020 World Orphan Drug Congress (WODC) USA Conference to better develop rare disease treatments. This presentation addresses: 

  • Why rare disease patients are hard to find and enroll into clinical trials 
  • How patient advocacy groups can partner with regulators and the industry 
  • Why thinking ahead to marketing access, price, and development is critical in early clinical development 
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