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26 October 2026 | Workshop Day
27-28 October 2026 | Conference & Exhibition
RAI Convention Centre, AmsterdamStrategy, Advocacy & Partnering For
The Global Orphan Drug Industry
The World's Largest Rare Disease Event
The World Orphan Drug Congress is the largest and most established orphan drug & rare disease meeting of its kind across the globe.
From cell and gene therapy, genetic testing, and market access, to real world evidence, this one meeting covers the whole orphan drugs value chain where science, government and manufacturers all come together to create groundbreaking progress.
1,800+
Attendees
250+
Speakers
100+
Exhibitors
3
Days
1,000+
1 to 1 Partnering Meetings
10,000+
Networking Connections
Covering cell & gene therapy, genetic testing, market access, real world evidence, orphan drugs policy and much more
130+ leading solution providers from across the globe, featuring new ways of thinking and novel partnerships to generate solutions.
Networking is at the heart of World Orphan Drug Congress. Our congress allows you to connect directly with thousands of pharma experts across 3 days of built-in 1-2-1 networking
The Start-to-Finish of Orphan Drugs
From regulation and policy to global pricing and gene therapy
Cell & Gene Therapies
Genetic Testing
Gene Editing
Partnering & Investment 
Global Market Access
Expanded Access Programs
Real World Evidence
Global Patient Advocacy
Value Based Pricing & Reimbursement
Epidemiology & Forecasting
Orphan Drugs Policy
Gene Therapy Development
Clinical Trials
Payers, HTA and Pricing
Manufacture2026 Keynote Speakers
Fabienne BartoliInspector GeneralFrench National Authority for Health
Stine BosseMEP Renew Group EuropeEuropean Parliament
Karen FaceyChair HTAi Rare Disease Interest GroupUniversity of Edinburgh
Niklas HedbergHead Of PharmacyTandvards och Lakemedelsformansverket
Kaja KantorskaHealth and Food Safety Directorate GeneralEuropean Commission
Yann Le CamAdvisorVOZ Advisors
Alexander NatzSecretary GeneralEUCOPE - European Confederation of Pharmaceutical Entrepreneurs
Jonathan NealHead of Central, South and Eastern Europe Takeda and EFPIA European Markets CommitteeTakeda
Francesc PalauDistinguished InvestigatorSJD Barcelona Children's Hospital
Sonia Pulido SánchezCo-Chair of the Subgroup for Joint Scientific Consultations of the HTACGAEMPS
Violeta Stoyanova-BeninskaSenior Scientific Specialist at Human Division EMA, Chair of Regulatory Science Committee, IRDiRC, former Chair of COMP at EMAEMA
Anne WillemsenCo-chair JCA SubgroupZorginstituut Nederland
Christopher RudolfFounder & CEOVolv Global
P.J. BrooksDeputy Director, Division of Rare Diseases Research Innovation, Member of IRDiRC Funders Constituent Committee (FCC)NIH/NCATS
Veronica Lopez GoussetDrPH candidateHarvard T.H. Chan School of Public Health
In Partnership With
Sponsor and Exhibitor Hall of Fame
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- Alexion, AstraZeneca Rare Disease
- Metabolic Support UK
- EURORDIS
- Cerner Enviza
- GenCC GmbH
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Working together to build sustainable events