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.

 

2026 Speakers

 

  • Fabienne Bartoli at World Orphan Drug Congress 2026
    Fabienne Bartoli
    Inspector General
    French National Authority for Health
    Stine Bosse at World Orphan Drug Congress 2026
    Stine Bosse
    MEP Renew Group Europe
    European Parliament
  • Karen Facey at World Orphan Drug Congress 2026
    Karen Facey
    Chair HTAi Rare Disease Interest Group
    University of Edinburgh
    Niklas Hedberg at World Orphan Drug Congress 2026
    Niklas Hedberg
    Head Of Pharmacy
    Tandvards och Lakemedelsformansverket
  • Kaja Kantorska at World Orphan Drug Congress 2026
    Kaja Kantorska
    Health and Food Safety Directorate General
    European Commission
    Yann Le Cam at World Orphan Drug Congress 2026
    Yann Le Cam
    Advisor
    VOZ Advisors
  • Alexander Natz at World Orphan Drug Congress 2026
    Alexander Natz
    Secretary General
    EUCOPE - European Confederation of Pharmaceutical Entrepreneurs
    Jonathan Neal at World Orphan Drug Congress 2026
    Jonathan Neal
    Head of Central, South and Eastern Europe Takeda and EFPIA European Markets Committee
    Takeda
  • Francesc Palau at World Orphan Drug Congress 2026
    Francesc Palau
    Distinguished Investigator
    SJD Barcelona Children's Hospital
    Sonia Pulido Sánchez at World Orphan Drug Congress 2026
    Sonia Pulido Sánchez
    Co-Chair of the Subgroup for Joint Scientific Consultations of the HTACG
    AEMPS
  • Violeta Stoyanova-Beninska at World Orphan Drug Congress 2026
    Violeta Stoyanova-Beninska
    Senior Scientific Specialist at Human Division EMA, Chair of Regulatory Science Committee, IRDiRC, former Chair of COMP at EMA
    EMA
    Anne Willemsen at World Orphan Drug Congress 2026
    Anne Willemsen
    Co-chair JCA Subgroup
    Zorginstituut Nederland
  • P.J. Brooks at World Orphan Drug Congress 2026
    P.J. Brooks
    Deputy Director, Division of Rare Diseases Research Innovation, Member of IRDiRC Funders Constituent Committee (FCC)
    NIH/NCATS
    Alexis Arzimanoglou at World Orphan Drug Congress 2026
    Alexis Arzimanoglou
    Coordinator of the European Reference Network for Rare and Complex Epilepsies
    EpiCARE
  • Thomas Butt at World Orphan Drug Congress 2026
    Thomas Butt
    Executive Director, Health Economics & Outcomes Research
    Biomarin
    Matthew Klein at World Orphan Drug Congress 2026
    Matthew Klein
    Chief Executive Officer
    PTC Therapeutics
  • Samantha Parker at World Orphan Drug Congress 2026
    Samantha Parker
    Patient Engagement Lead Rare Diseases and Vice Chair of IRDiRC
    italfarmaco
    Durhane Wong-Rieger at World Orphan Drug Congress 2026
    Durhane Wong-Rieger
    President, Chief Executive Officer
    Canadian Organisation for Rare Disorder & Rare Diseases International
  • OMAR ALI at World Orphan Drug Congress 2026
    OMAR ALI
    HEAD OF PAYERS; FORMER GOVERNMENT PAYER
    VERPORA
    Derek Ansel at World Orphan Drug Congress 2026
    Derek Ansel
    Vice President, Therapeutic Strategy Lead, Rare DiseaseRare Disease
    Worldwide Clinical Trials
  • Lara Bloom at World Orphan Drug Congress 2026
    Lara Bloom
    President and CEO
    The Ehlers-Danlos Society
    Thomas Bols at World Orphan Drug Congress 2026
    Thomas Bols
    Head of Government Affairs and Patient Advocacy, EMEA & APAC
    PTC Therapeutics
  • Sara Brambilla at World Orphan Drug Congress 2026
    Sara Brambilla
    Global Programme Manager
    Rare Diseases International
    Paul Broomhead at World Orphan Drug Congress 2026
    Paul Broomhead
    Head of Site
    SGS Integrated CDMO Solutions
  • Anna Bucsics at World Orphan Drug Congress 2026
    Anna Bucsics
    Project Advisor
    MoCA
    Ria de Haas at World Orphan Drug Congress 2026
    Ria de Haas
    CEO
    FSHD Europe
  • Peter Fassler at World Orphan Drug Congress 2026
    Peter Fassler
    Director Market Access
    Gilead Sciences
    Josie Godfrey at World Orphan Drug Congress 2026
    Josie Godfrey
    Independent Market Access and Rare Disease Adviser
    Independent Rare Disease Expert
  • Alicia Granados at World Orphan Drug Congress 2026
    Alicia Granados
    Global Head Scientific Advocacy and Insights
    Sanofi
    Patrick Hopkinson at World Orphan Drug Congress 2026
    Patrick Hopkinson
    Independent HTA Expert
    Independent HTA Expert
  • Mohit Jain at World Orphan Drug Congress 2026
    Mohit Jain
    Vice President, Market Access & Pricing, Europe and International
    Ascendis Pharma
    Ron Jortner at World Orphan Drug Congress 2026
    Ron Jortner
    Founder & CEO
    Aspire Biosciences
  • Daria Julkowska at World Orphan Drug Congress 2026
    Daria Julkowska
    Scientific Coordinator
    ERDERA - INSERM
    Paolo Morgese at World Orphan Drug Congress 2026
    Paolo Morgese
    Vice President, Public Affairs, Europe
    Alliance for Regenerative Medicine
  • Josephine Mosset at World Orphan Drug Congress 2026
    Josephine Mosset
    Senior Policy Officer
    Cancer Patients Europe
    Frauke Naumann-Winter at World Orphan Drug Congress 2026
    Frauke Naumann-Winter
    Head of Unit
    BfArM
  • Tom Pulles, MD at World Orphan Drug Congress 2026
    Tom Pulles, MD
    VP, Head of Medical Affairs & Patient Advocacy, Europe
    Acadia Pharmaceuticals Inc.
    Asli Raman at World Orphan Drug Congress 2026
    Asli Raman
    Senior Technology Transfer and Business Development Manager
    Institute for BioEngineering of Catalonia (IBEC)
  • Mariem Sarghini at World Orphan Drug Congress 2026
    Mariem Sarghini
    Founder
    MSAQS
    Maurizio Scarpa at World Orphan Drug Congress 2026
    Maurizio Scarpa
    Director, Coordinating Center For Rare Diseases
    MetabERN
  • Lydia Shotton at World Orphan Drug Congress 2026
    Lydia Shotton
    Associate Director EU Government Affairs
    Alliance for Regenerative Medicine
    Evert Jan van Lente at World Orphan Drug Congress 2026
    Evert Jan van Lente
    management4health AG
  • Wieteke Wouters at World Orphan Drug Congress 2026
    Wieteke Wouters
    Programme Director
    hollandbio
    Kwee Lan TAN at World Orphan Drug Congress 2026
    Kwee Lan TAN
    Head of team
    Boehringer Ingelheim
  • Veronica Lopez Gousset at World Orphan Drug Congress 2026
    Veronica Lopez Gousset
    DrPH candidate
    Harvard T.H. Chan School of Public Health
    Viviana Giannuzzi at World Orphan Drug Congress 2026
    Viviana Giannuzzi
    Head of Research and Innovation Department
    Fondazione per la ricerca farmacologica Gianni Benzi onlus
  • Adrian Goretzki at World Orphan Drug Congress 2026
    Adrian Goretzki
    President
    Healthcare Education Institute
    Kristina Larsson at World Orphan Drug Congress 2026
    Kristina Larsson
    Head of Orphan Medicines, Division for Human Medicines Evidence Generation
    EMA
  • Kit Roes at World Orphan Drug Congress 2026
    Kit Roes
    Professor
    Radboudumc
    Christopher Rudolf at World Orphan Drug Congress 2026
    Christopher Rudolf
    Founder & CEO
    Volv Global
  • Thomas Broekhoff at World Orphan Drug Congress 2026
    Thomas Broekhoff
    Program Manager Medicines
    hollandbio
    Pedro Franco at World Orphan Drug Congress 2026
    Pedro Franco
    Senior Director, Head of Europe Global Regulatory & Scientific
    Merck
  • Dariusz Adamczewski at World Orphan Drug Congress 2026
    Dariusz Adamczewski
    Managing Director
    Children’s Tumor Foundation Europe
    Anjan Aralihalli at World Orphan Drug Congress 2026
    Anjan Aralihalli
    President & Founder
    Raya Therapeutic, Inc.
  • Annette Bakker at World Orphan Drug Congress 2026
    Annette Bakker
    CEO
    Childrens Tumor Foundation
    Stefano Benvenuti at World Orphan Drug Congress 2026
    Stefano Benvenuti
    Head of Public Affairs and Market Access
    Fondazione Telethon
  • Giorgia Crimi at World Orphan Drug Congress 2026
    Giorgia Crimi
    Patient Adovacacy and engagement manager
    Fondazione Telethon
    Magdalena Daccord at World Orphan Drug Congress 2026
    Magdalena Daccord
    Chief Executive Officer
    FH Europe Foundation
  • Toon Digneffe at World Orphan Drug Congress 2026
    Toon Digneffe
    Head Public Affairs & Public Policy
    Takeda
    Elsie Evans at World Orphan Drug Congress 2026
    Elsie Evans
    Ambassador Programme Project Manager
    FH Europe Foundation NGO
  • Marcus Guardian at World Orphan Drug Congress 2026
    Marcus Guardian
    CEO
    International Horizon Scanning Initative
    Debbie Hellenbrand at World Orphan Drug Congress 2026
    Debbie Hellenbrand
    Patient advocate and Professional Speaker
    Pathways To Trust
  • Anneliene Jonker at World Orphan Drug Congress 2026
    Anneliene Jonker
    Rare Disease Therapy Researcher
    University of Twente
    Manohar Katakam at World Orphan Drug Congress 2026
    Manohar Katakam
    Chief Executive Officer
    SteroTherapeutics
  • James Levine at World Orphan Drug Congress 2026
    James Levine
    President, Foundation Ipsen
    James Levine - Ipsen
    Daniel O'Connor at World Orphan Drug Congress 2026
    Daniel O'Connor
    Director, Regulatory Policy & Early Access
    ABPI
  • Francis Pang at World Orphan Drug Congress 2026
    Francis Pang
    SVP Global Market Access and International Geographic Expansion
    Orchard Therapeutics
    Solange Roumengous at World Orphan Drug Congress 2026
    Solange Roumengous
    Head of partnerships
    Banque Nationale de Données Maladies Rares (BNDMR) - Assistance Publique - Hôpitaux de Paris (APHP)
  • Ruchi Sharma at World Orphan Drug Congress 2026
    Ruchi Sharma
    CEO
    Stemnovate Limited
    Claire Skentelbery at World Orphan Drug Congress 2026
    Claire Skentelbery
    Director General
    EuropaBio
  • Rachel Smith at World Orphan Drug Congress 2026
    Rachel Smith
    Vice President, Rare and Genetic Diseases
    Parexel International LLC
    Ishaan Tewarie at World Orphan Drug Congress 2026
    Ishaan Tewarie
    EQT Life Sciences
  • Sheela Upadhyaya at World Orphan Drug Congress 2026
    Sheela Upadhyaya
    Life Science Advisor in Rare Diseases
    Openflex Ltd
    Leon van Wouwe at World Orphan Drug Congress 2026
    Leon van Wouwe
    Clinical Innovation Director
    Volv Global
  • Miriam Wagner Long at World Orphan Drug Congress 2026
    Miriam Wagner Long
    Program lead
    FSHD World Alliance

 

Conference

Covering cell & gene therapy, genetic testing, market access, real world evidence, orphan drugs policy and much more
 

Exhibition

130+ leading solution providers from across the globe, featuring new ways of thinking and novel partnerships to generate solutions.
 

APPLY TO SPONSOR/EXHIBIT

Networking

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  

GET YOUR TICKET NOW

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
Manufacture

In Partnership With

Beacon World Orphan Drug Congress 2025 Supporting PartnerERDERA World Orphan Drug Congress 2025 Supporting PartnerEURORDIS World Orphan Drug Congress 2025 Supporting PartnerHollandBIO World Orphan Drug Congress 2025 Supporting PartnerRDI World Orphan Drug Congress Europe 2025 Supporting PartnerEuropaBio World Orphan Drug Congress 2025 Supporting PartnerBeacon World Orphan Drug Congress 2025 Supporting PartnerERDERA World Orphan Drug Congress 2025 Supporting PartnerEURORDIS World Orphan Drug Congress 2025 Supporting PartnerHollandBIO World Orphan Drug Congress 2025 Supporting PartnerRDI World Orphan Congress Europe 2025 Supporting PartnerEuropaBio World Orphan Drug Congress 2025 Supporting Partner

Sponsor and Exhibitor Hall of Fame

 

  • Platinum Sponsor

    Sciensus at World Orphan Drug Congress 2025

    Platinum Sponsor

    UCB at World Orphan Drug Congress 2025
  • Gold Sponsor

    Chiesi Global Rare Diseases at World Orphan Drug Congress 2025

    Gold Sponsor

    Clinigen Group at World Orphan Drug Congress 2025
  • Gold Sponsor

    CRA, Charles River Associates at World Orphan Drug Congress 2025

    Gold Sponsor

    Eversana at World Orphan Drug Congress 2025
  • Gold Sponsor

    Ipsen at World Orphan Drug Congress 2025

    Gold Sponsor

    Norgine at World Orphan Drug Congress 2025
  • Gold Sponsor

    Parexel International LLC at World Orphan Drug Congress 2025

    Gold Sponsor

    PrimeVigilance at World Orphan Drug Congress 2025
  • Gold Sponsor

    RTI Health Solutions at World Orphan Drug Congress 2025

    Gold Sponsor

    SANOFI at World Orphan Drug Congress 2025
  • Gold Sponsor

    Sartorius BIA Seperations at World Orphan Drug Congress 2025

    Gold Sponsor

    Takeda at World Orphan Drug Congress 2025
  • Gold Sponsor

    Volv Global at World Orphan Drug Congress 2025

    Gold Sponsor

    Wemedoo AG at World Orphan Drug Congress 2025
  • Gold Sponsor

    worldwide clinical trials at World Orphan Drug Congress 2025

    Silver Sponsor

    ACA Pharma at World Orphan Drug Congress 2025
  • Silver Sponsor

    Allucent at World Orphan Drug Congress 2025

    Silver Sponsor

    HEOR at World Orphan Drug Congress 2025
  • Silver Sponsor

    Initiate Consultancy at World Orphan Drug Congress 2025

    Silver Sponsor

    Premier Research at World Orphan Drug Congress 2025
  • Silver Sponsor

    WEP Clinical at World Orphan Drug Congress 2025

    Bronze Sponsor

    Alchemist Consulting Ltd at World Orphan Drug Congress 2025
  • Bronze Sponsor

    Alira Health at World Orphan Drug Congress 2025

    Bronze Sponsor

    BRCR Global at World Orphan Drug Congress 2025
  • Bronze Sponsor

    Clinipace Inc. d/b/a Caidya at World Orphan Drug Congress 2025

    Bronze Sponsor

    Costello Medical at World Orphan Drug Congress 2025
  • Bronze Sponsor

    Dendrite Clinical Systems at World Orphan Drug Congress 2025

    Bronze Sponsor

    GeneScape at World Orphan Drug Congress 2025
  • Bronze Sponsor

    Kintiga at World Orphan Drug Congress 2025

    Bronze Sponsor

    Rare Patient Voice, LLC at World Orphan Drug Congress 2025
  • Bronze Sponsor

    SGS Integrated CDMO Solutions at World Orphan Drug Congress 2025

    Bronze Sponsor

    Valid Insight at World Orphan Drug Congress 2025
  • Associate Sponsor

    Avanzanite Bioscience at World Orphan Drug Congress 2025

    Associate Sponsor

    Azafaros at World Orphan Drug Congress 2025
  • Associate Sponsor

    Everyone medicines at World Orphan Drug Congress 2025

    Associate Sponsor

    IQVIA at World Orphan Drug Congress 2025
  • Associate Sponsor

    Sharp Therapeutics at World Orphan Drug Congress 2025

    Session Facilitator

    Servier at World Orphan Drug Congress 2025
  • Exhibitor

    Aixial inc at World Orphan Drug Congress 2025

    Exhibitor

    Ataxia of Charlevoix-Saguenay Foundation at World Orphan Drug Congress 2025
  • Exhibitor

    BAP Pharma at World Orphan Drug Congress 2025

    Exhibitor

    Decisive Consulting Ltd. at World Orphan Drug Congress 2025
  • Exhibitor & Supporting Partner

    Dravet Syndrome Foundation Spain at World Orphan Drug Congress 2025

    Exhibitor

    Envoya Inc. at World Orphan Drug Congress 2025
  • Exhibitor & Supporting Partner

    ERDERA at World Orphan Drug Congress 2025

    Exhibitor & Supporting Partner

    ERN SKIN at World Orphan Drug Congress 2025
  • Exhibitor & Supporting Partner

    EURORDIS at World Orphan Drug Congress 2025

    Exhibitor & Supporting Partner

    FH Europe Foundation at World Orphan Drug Congress 2025
  • Exhibitor

    Genvade Therapeutics at World Orphan Drug Congress 2025

    Exhibitor

    GNH Pharmaceuticals USA at World Orphan Drug Congress 2025
  • Exhibitor

    IDEA Regulatory at World Orphan Drug Congress 2025

    Exhibitor

    Ikris Pharma Network at World Orphan Drug Congress 2025
  • Exhibitor & Supporting Partner

    IRDiRC at World Orphan Drug Congress 2025

    Exhibitor & Supporting Partner

    Kidney Research UK at World Orphan Drug Congress 2025
  • Exhibitor

    Medscape Education Global at World Orphan Drug Congress 2025

    Exhibitor

    Onyx Scientific at World Orphan Drug Congress 2025
  • Exhibitor

    OPEN Health at World Orphan Drug Congress 2025

    Exhibitor

    Proteintech at World Orphan Drug Congress 2025
  • Exhibitor

    QuickSTAT A Kuehne + Nagel Company at World Orphan Drug Congress 2025

    Exhibitor & Supporting Partner

    RARE Revolution at World Orphan Drug Congress 2025
  • Exhibitor

    Rivia AG at World Orphan Drug Congress 2025

    Exhibitor

    Sherwood Pharmaceuticals at World Orphan Drug Congress 2025
  • Exhibitor

    Siron B.V. at World Orphan Drug Congress 2025

    Startup

    Annogen at World Orphan Drug Congress 2025
  • Startup

    Azur Health Science at World Orphan Drug Congress 2025

    Startup

    BioMe, Inc. at World Orphan Drug Congress 2025
  • Startup

    Cellbyte at World Orphan Drug Congress 2025

    Startup

    CureAge Therapeutics at World Orphan Drug Congress 2025
  • Startup

    Genvade Therapeutics at World Orphan Drug Congress 2025

    Startup

    GlycoMScan B.V. at World Orphan Drug Congress 2025
  • Startup

    iollo - The AI Scientist at World Orphan Drug Congress 2025

    Startup

    Ksilink at World Orphan Drug Congress 2025
  • Startup

    LatM - Life Science Consultants, Lda at World Orphan Drug Congress 2025

    Startup

    Mirava Bio at World Orphan Drug Congress 2025
  • Startup

    NanoZymeX at World Orphan Drug Congress 2025

    Startup

    Netabolics at World Orphan Drug Congress 2025
  • Startup

    NeuroCores, Inc. at World Orphan Drug Congress 2025

    Startup

    Olvia Biopharma at World Orphan Drug Congress 2025
  • Startup

    ORPHANET-AISBL at World Orphan Drug Congress 2025

    Startup

    PharmaKrysto Ltd at World Orphan Drug Congress 2025
  • Startup

    Scarlet Therapeutics at World Orphan Drug Congress 2025

    Startup

    Senovia Biosciences at World Orphan Drug Congress 2025
  • Startup

    Trialize at World Orphan Drug Congress 2025

    Startup

    Unboxed Psychology at World Orphan Drug Congress 2025

 

“Always a great conference, where key players in the rare disease field come together and share ideas, and more importantly, agree on some concrete actions“ – IPSEN
“The leading congress that addresses the subjects most important to patients with rare diseases and treatments by orphan drugs.”
- Alexion, AstraZeneca Rare Disease
“Great access to important colleagues, ability to connect or book time slots on Terrapinn Events App was great, varied sessions and good opportunities to network outside of the sessions”
- Metabolic Support UK
“Great networking opportunities, great panels and discussions!”
- EURORDIS
“Good mix of plenaries and workshops, lots of meeting opportunities, relevant topic selection, very friendly and supportive organizer staff”
- Cerner Enviza
“Excellent organization, very interesting sessions and great networking opportunities.”
- GenCC GmbH

 

Join the global rare disease community
in Amsterdam

26 - 28 October 2026
 

Get involved at World Orphan Drug Congress Europe 2026

 

 

TO SPONSOR


Steph Scanlon

Steph.Scanlon@terrapinn.com

 

TO SPEAK


Abdu Kauroo
Abdu.Kauroo@terrapinn.com

 

MARKETING & PRESS


Jasper Cameron

Jasper.Cameron@terrapinn.com

 

Stay Up-to-Date

Join our mailing list to receive exclusive content and offers.

By submitting, you agree to receive email communications from Terrapinn, including upcoming promotions and discounted tickets and news.