Rare Drug Development Symposium 2022 Highlights
Thank you to all who made the 2022 Rare Drug Development Symposium 2022, presented with Global Genes, a success. Please check back for updates to this page as new content related to the 2022 Symposium will be added.
Participants had a unique opportunity to engage in a series of interactive small-group workshops with experts and peers as we discuss disruptive innovations in rare disease research. This was followed by a “speed greeting” networking event designed to foster productive connections between advocates, researchers, and industry leaders.
During the event there were sessions covering the following topics:
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 the new 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?
Location: Sheraton Philadelphia Downtown
201 North 17th Street
Philadelphia, PA 19103
The Orphan Disease Center co-hosts the Rare Drug Development Symposium in partnership with Global Genes.