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Rare Drug Development Symposium 2023: Collaborate: Go Farther, Together

May 1, 2023 - May 3, 2023

global advocacy alliance

Join us May 1-3, 2023 in Philadelphia, Pennsylvania for the RARE Drug Development Symposium 2023

Collaboration is the foundation of success in rare disease research.  Knowing who to work with, what strategies to use, and how to prepare for discussions can help you overcome barriers posed by small populations and limited funding.
At the Rare Drug Development Symposium (RDDS) 2023, a partnership between Global Genes and the Orphan Disease Center of the University of Pennsylvania, you’ll connect to  rare disease advocacy leaders, researchers, clinicians, industry professionals and policy developers who will share their experience and provide you with models to help your community push forward. This 2-day, in-person only event includes dynamic presentations, followed by workshops and expert office hours that will enable you to:

Create A Comprehensive Research Strategy
Understand the end-to-end process of drug discovery and development for rare disease and participate in a workshop where you’ll analyze your community’s potential and needs.

Leverage the Power of Data
What makes data valid and reliable? Once you have invested in a registry, what partnership models can  help you move the needle toward translational research?

Use AI for Drug Discovery and Repurposing
Find out how artificial intelligence is being used to transform an often complex, decades-long-mission into a more efficient process, reducing the timeline and cost to bring therapies to patients.

Understand Regulatory Considerations
Whether your organization is local or global, regulatory guidelines must be taken into account at each step. Learn how to navigate the requirements through case studies and expert advice.

Who should attend:

  • Patient Advocacy Group leaders
  • Patients
  • Members of the rare disease community who want to learn more about the drug development process, the unique ways this applies to the development of therapeutics for rare diseases, and how they can engage with the process
  • Clinicians and researchers focused on rare disease therapeutic targets

View 2022 Highlights

To learn more about the 2022 Symposium, read the summary report hereWe look forward to seeing you at the 2023 Symposium.

A Year-Long Initiative in Rare Drug Development

In addition to activities surrounding the RARE Drug Development Symposium in June, Global Genes is providing educational resources to help advocates and organizations become better equipped to work with researchers and industry partners throughout the drug development process. This includes:

Rare Research Roadmap

Rare Research Roadmap: This toolkit was designed to help advocates, individuals, families, and organizations better understand some of the potential routes to treatment and some of the key concepts that are part of these processes to work with researchers and industry partners throughout the drug development process.

Watch webinars and videos of experts that happened before and after the 2022 RARE Drug Development Symposium.


May 1, 2023
May 3, 2023
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Global Genes
(+1) 949-248-RARE
View Organizer Website


Sheraton Downtown
201 N 17th St
Philadelphia, PA 19103 United States
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