Rare Drug Development Symposium

Starts:  Jun 8, 2022 09:00 (ET)
Ends:  Jun 10, 2022 17:00 (ET)

Join us June 8-10, 2022 for the 2022 RARE Drug Development Symposium in Philadelphia, PA!

Rare disease research is rapidly evolving.  But new models come with new challenges that must be addressed in order to bring treatments to those who need them as quickly and effectively as possible. 

You can help shape the future of rare disease research and treatment development.

The Rare Drug Development Symposium, in partnership with the Orphan Disease Center of the University of Pennsylvania, brings together rare disease advocacy leaders and researchers to identify barriers to research, envision solutions, and accelerate progress for our communities.

This two-day, in-person-only summit offers a unique opportunity to engage in interactive small-group workshops with experts and peers, followed by a “speed greeting” networking event designed to foster productive connections between advocates, researchers, and industry leaders. Topics include:

  • Disruptive Innovations in Trials: What are the new models? Who is doing it well, and how can other communities participate? What do we need to address from a regulatory perspective?
  • AI-Driven Screening Platforms and New Approaches to Therapeutics: How can we pull meaningful results from massive data sets to find drug targets, whether repurposed or original.
  • Emerging Commercial Models & Partnerships: How do we address small populations which aren’t commercially viable? What is the role of advocates in holding INDs, leveraging limited resources to fund research, and ensuring that drugs they support are accessible?
  • Fostering Successful Connections Between Stakeholders to Accelerate Progress:  What can we do to help investigators find mentors and connect to researchers and advocates? How do internship programs build bridges?

A Year-Long Initiative in Rare Drug Development: In addition to activities surrounding the RARE Drug Development Symposium in June, Global Genes will provide educational resources to help advocates and organizations become better equipped to work with researchers and industry partners throughout the drug development process. This will include:

  • Three 60-90 minute webinars 
    • Webinar 1: What Does It Take to Become a Research-Ready Organization?
    • Webinar 2: Getting Your Community Ready for Clinical Trials
    • Webinar 3: Financing Research
  • A series of short educational video vignettes discussing topics covered at the RARE Drug Development Symposium
  • A Drug Development Toolkit, including information, infographics, checklists, and videos

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