In the past 9 years, we have witnessed a real transformation in how rare diseases are diagnosed and treated.

The World Orphan Drug Congress USA has always remained up to date with the evolving rare disease landscape to become the largest and most global annual gathering of orphan drug stakeholders.


For, 2019 we have expanded the program to focus on cell and gene therapies for rare diseases as well as rare oncology.

While becoming more comprehensive from a topic area perspective, the World Orphan Drug Congress continues to expand globally, bringing government authorities, payers, industry and patient advocacy groups from over 50 countries.








Together with: