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Generate evidence for medical breakthroughs and unmet treatment needs in rare disease research

58 minutes

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May 14, 2015

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In this webinar, learn about new opportunities for rare diseases and how generating the right evidence can help you meet increasing multi-stakeholder demands. Incorporate the patient voice, deliver value at every step of the research process.

Key discussion topics:

  • Rare disease research landscape: An overview Adaptive pathways and the unmet need in rare disease research
  • Patient engagement and retention in long-term observational rare disease studies
  • Role of stakeholders in rare disease registries: Collaborating on study design to plan earlier in product development and meet multi-stakeholder needs
  • New trends in rare disease research: Repurposing drugs under the US OPEN Act


Eric Faulkner, MPH
Practice Leader, Emerging Technology Value Demonstration, Access & Commercialization

Kathryn Starzyk
Senior Director of Epidemiology