Boston Convention and Exhibition Center, Boston, MA
April 23-25, 2024
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TIMES & LOCATION
Tuesday, April 23 – Pre-Conference Day
12:00pm - 1:00pm – Badge Pickup
1:00pm - 5:00pm – Seminars & Workshops
Wednesday, April 24 – Day 1
7:45am - Badge Pickup & Expo Opens
8:35am - Opening Keynotes
1:35pm- Afternoon Keynotes
4:45pm - Closing Keynotes
5:45pm - co*cktail Reception
6:40pm- Expo Hall Closes
Thursday, April 25 – Day 2
8:00am - Badge Pickup & Expo Opens
8:50am - Opening Keynotes
2:35pm- Afternoon Keynotes
4:35pm - Expo Hall Closes
Venue and badge pickup location
Boston Convention & Exhibition Center
415 Summer St, Boston, MA 02210
Badge Pickup – In Front of Hall C
Breaking down silos and fostering collaboration to help rare disease patients
The World Orphan Drug Congress brings together leading pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors and solution providers. The conference is a place to meet and brainstorm ways to advance orphan drug development and improve access to life-saving therapies.
Join us for three days in Boston for the most comprehensive program and inclusive gathering of rare disease stakeholders.
300+
Speakers
2,000+
Attendees
100+
Exhibitors
16
Streams of Content
2024Speakers
Peter Marks Director, Center For Biologics Evaluation And Research (CBER) U.S. Food and Drug Administration
Sarah Glass Chief Operating Officer n-Lorem Foundation
Paul Aliu Head Global Governance Office, Novartis Chief Medical Office (CMO) Novartis
PJ Brooks Acting Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS) National Institutes of Health
Alexander Natz Director General EUCOPE - European Confederation of Pharmaceutical Entrepreneurs
Mary Wang Programme Director Rare Diseases International
Emil Kakkis Chief Executive Officer Ultragenyx Pharmaceutical
Peter Saltonstall Chief Executive Officer National Organization for Rare Disorders (NORD)
VIEW ALL 2024 SPEAKERS
Our World-Class Conference Features The Following Themes
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 reimbursem*nt 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 & REIMBURsem*nT
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 reimbursem*nt?
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?
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.
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2024Sponsors & Exhibitors
Title Sponsor
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Exhibiting Partner
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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.
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Companies that attend World Orphan Drug Congress USA
"If there is one event you want to go to as a stakeholder in rare diseases, it is the World Orphan Drug Congress USA. This conference has helped to define the world of rare diseases, it’s not just a leading event, it has actually helped create the future for rare diseases" President And Chief Executive Officer, Canadian Organization For Rare Disorders
"The support, kindness, courtesy and interest shown by WODC Team in our company’s drug development program for orphan disease glioblastoma, to aid us to secure funding to run registration trials" Chief Executive Officer, Tactical Therapeutics Inc
New Location:
Boston Convention & Exhibition Center
Boston, MA
April 23-25, 2024
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