RARE Drug Development Symposium 2023

At the RARE Drug Development Symposium, rare disease advocates can acquire the knowledge, skills and connections they need to advance therapy development for their communities. You will be equipped to identify inflection points when the involvement of rare disease patients and advocates is crucial: who to work with, what strategies to use, and how to ensure the patient voice is heard at each step of the road.

Hosted in partnership with the Orphan Disease Center of the University of Pennsylvania, RDDS 2023 focuses on collaboration – the foundation of success in rare disease research. You will meet individuals, advocacy leaders, industry, and research experts who have been there and done it, over two days of in-person only panel discussions, hands-on workshops, and expert office hours that will help you:

Leverage the power of data to drive basic research
What makes data valid and reliable? How do registries differ? Once you have invested in a registry, what partnership models can help you move the needle toward translational research?

How new technologies can expand research opportunities
Find out how artificial intelligence is being used for drug discovery and repurposing, transforming 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.

Refine 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.