The World Orphan Drug Congress USA 2025 is set to take place from April 22-24, 2025, at the Boston Convention & Exhibition Center in Boston, Massachusetts. This event is expected to attract a significant number of attendees and exhibitors, making it the most comprehensive gathering of stakeholders in the rare disease and orphan drug space. With over 300 speakers, 100 exhibitors, and more than 2,000 attendees, this conference aims to foster collaboration and advance the development of orphan drugs.

The event will feature 14 streams of content, including patient data, next-generation therapies, payers, clinical development and regulatory issues, commercial strategies, rare disease advocacy, global market and patient access, pricing and reimbursement, pitch and partnership opportunities, advanced therapies manufacturing and clinical development challenges, rare oncology advancements, digital health and artificial intelligence applications in diagnosis and discovery. These diverse topics will provide a platform for discussions on the latest developments in the field.

Key speakers at the conference include Peter Marks from the U.S. Food and Drug Administration (FDA), Amy Nicole Nayar from Novartis Gene Therapies, Diego Guarin from Merck's Regional Market Access team, PJ Brooks from the National Institutes of Health (NIH), Durhane Wong-Rieger from Canadian Organisation for Rare Disorders & Rare Diseases International, Melanie Lendnal from the ALS Association's SVP Policy & Advocacy team, and Francis Pang from Orchard Therapeutics.

Sponsors for this event include several platinum sponsors as well as diamond and silver sponsors. The conference offers a range of sponsorship opportunities for companies looking to showcase their products or solutions to industry leaders in the rare disease field.

Attendees can expect to engage in various networking activities such as facilitated meetings, roundtables, and informal hangouts. These interactions are designed to facilitate idea exchange among stakeholders in the rare disease community.

The World Orphan Drug Congress USA is not just a leading event; it has helped shape the future of rare diseases by bringing together pharmaceutical companies, biotech firms, government agencies, patient advocacy groups, payers, investors, and solution providers. It serves as a vital platform for advancing orphan drug development and enhancing access to life-saving therapies for individuals living with rare diseases.