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April 22-24, 2025
Boston Convention & Exhibition Center | Boston, MA
The World’s Most Important Orphan Drug & Rare Disease Event
300+
Speakers
2,000+
Attendees
100+
Exhibitors
14
Streams of Content
Breaking down silos and fostering collaboration to help rare disease patients
The World Orphan Drug Congress convenes top pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors and solution providers. This conference provides a forum to discuss and advance orphan drug development and enhance access to life-saving therapies for individuals living with a rare disease.
Join us in Boston for three days of the most comprehensive program and inclusive gathering of rare disease stakeholders.
The World Orphan Drug Congress USA is the defining event for the rare disease and orphan drug space globally.
Your ticket provides access to the premium conference, exhibition and networking opportunities with thousands of industry leaders.
Hear about the latest developments in the rare disease and orphan drug space from top thought leaders in the industry.
Discover innovative products and solutions revolutionizing the future of rare disease.
Connect with fellow rare disease and orphan drug advocates from around the world – all with a shared passion to improve the lives of rare disease patients.
Sponsor the World Orphan Drug Congress USA
Get your product or solution in front of the leaders in the rare disease industry — from pharma, biotechs, governments, payers, investors and patient/patient advocates.
The World Orphan Drug Congress USA is a place where innovation and expertise is showcased, solutions are found and learning is done.
2025 Sponsors & Exhibitors
Platinum Sponsor
Gold Sponsor
Gold Sponsor
Gold Sponsor
Silver Sponsor
Silver Sponsor
Silver Sponsor
Silver Sponsor
Silver Sponsor
Silver Sponsor
Silver Sponsor
Silver Sponsor
Exhibitor
Exhibitor
Exhibitor
Exhibitor
Exhibitor
Networking Is Our Sweet Spot
By App. By Facilitated Meetings. By RoundTables. By Hangin’ Out.
So you can: Exchange ideas, Build brand, Form friendships and partnerships, Grow your professional network, Explore the future, Uncover new opportunities.
Highlighted Speakers
Highlighted Speakers
The Start-to-Finish of Orphan Drugs
From regulation and policy to global pricing and gene therapy
PATIENT DATA
With so few patients, patient data/real-world-data (RWD/RWE) is the key element to advancing the rare disease space. What are best practices right now for data collection, ownership and sharing to benefit rare disease diagnosis, drug development and access. 
NEXT GENERATION THERAPIES
The rare disease space is a breeding ground for innovative approaches in drug development and platform technologies. What are the products and approaches that will be making headlines in 5-10 years from now? 
PAYERS
US-based insurers and single payer agencies around the world have a lot of impact of access and reimbursement for orphan products. Hear from top US and international payers on their strategies for orphan drugs. 
CLINICAL DEVELOPMENT & REGULATORY
Understanding the regulatory and clinical landscape for rare disease drug development. How can companies, regulators and patient advocates work together to further patient voice, real world evidence, and decentralized clinical trials. 
COMMERCIAL
What are the best commercial strategies for orphan products? How can companies bring products to new markets, build more efficient commercial operations, and develop effective launch strategies? 
RARE DISEASE ADVOCACY WORLD
It all starts and ends with patients. So what are the challenges facing rare disease patients and patient organizations today? How can rare disease patient groups rethink funding strategies, partnerships with industry and advance policies around access? 
GLOBAL MARKET & PATIENT ACCESS
Access is often the crucial and challenges last step for rare disease patients and developers. How can all stakeholders work together to ensure rare disease patients have access to appropriate treatments globally. 
PRICING & REIMBURSEMENT
Pricing orphan products, especially one-time treatments, is complex and a key element to ensuring timely access for rare disease patients. What are the current best practices for pricing orphan products, negotiating innovative contracts and working with payers around the world? 
PITCH & PARTNER
The orphan drug space is booming with innovation, M&A activity and partnerships. We play match maker for up and coming biotechs in the rare space and investors & pharma partners looking for their next blockbuster. 
ADVANCED THERAPIES - MANUFACTURING
What are the current roadblocks in manufacturing cell and gene therapies and how can companies efficiently scale-up for commercial production? 
ADVANCED THERAPIES - CLINICAL DEVELOPMENT
The regulatory and clinical development landscape is ever evolving for cell and gene therapies. How are companies ensuring continued development of these potentially transformative products and what clinical hurdles remain? 
RARE ONCOLOGY
As the rare cancer field evolves and diagnosis becomes more precise, how can all stakeholders work together to advance drug development for rare cancer patients? 
ADVANCED THERAPIES - COMMERCIALIZATION
Rare disease patients are uniquely positioned to benefit from the advancements of cell and gene therapies. What are the best strategies for commercializing these innovative products and how do health systems need to evolve to accommodate patient access and reimbursement? 
DIGITAL HEALTH & ARTIFICIAL INTELLIGENCE
Approaches in AI and digital health span the full development life cycle of drug development and commercialization. What are the best approaches to implementing these technologies in patient finding, clinical development and commercialization for rare diseases? 
DIAGNOSIS
It takes an average of seven years for an accurate diagnosis. How can all stakeholders broaden access to Newborn Screening, Whole Genome Sequencing and Genetic testing to shorten the diagnostic odyssey for rare disease patients? 
DISCOVERY
Technologies and processes are ever. How is the growing access to data, and use of AI and machine learning impacting drug discovery in the rare disease space?
President And Chief Executive Officer, Canadian Organization For Rare Disorders
Chief Executive Officer, Tactical Therapeutics Inc
Join us in Boston
April 22-24, 2025
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