World Orphan Drug Congress USA is the leading rare disease event advancing orphan drug development, clinical trials, regulatory strategy, market access, and patient advocacy. Join biotechs, pharma, payers, and regulators to
Programmed at World, in alphabetical order.
Selected from 34 sessions.
This interactive workshop will bring together leaders from research, biotech, pharmaceutical industry, investment and the patient community to explore how AI is already transformin…
People affected by rare liver diseases face recurring challenges across diagnosis, specialist care, evidence generation and access. Yet action is often organised disease by disease…
Artificial intelligence is rapidly becoming an integral part of the rare disease ecosystem, supporting scientific discovery, therapeutic development and strategic decision-making. …
Moving beyond isolated advocacy to accelerate diagnosis, this workshop will holistically explore how early diagnosis fits within the rare disease ecosystem from development of diag…
A medicine may be approved in Europe and still remain out of reach for patients in the Netherlands. Why? Three patient organisations will bring the access challenge to life through…
- The need for a separate approval pathway for Orphan Drugs - How will new Reimbursement models help Pharmaceutical companies navigate Orphan Drug development?
A Rare Networking Opportunity brings together rare disease patient advocates and professionals working towards rare disease therapies - not to pitch or present, but to genuinely co…
An audience of 47 companies, parsed from the program.
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Same brand, other regions