Healthcare & Medical

World Orphan Drug Congress 2026

The World Orphan Drug Congress 2026 brings together leaders in strategy, advocacy, and partnering from the orphan drug industry. It serves as a forum for industry professionals, regulators, patients, and other stakeholders to discuss the latest trends, innovations, and challenges in the orphan drug field.

Visit the official siteRegisterSponsorship open
61Speakers
34Sponsors
1Exhibitors
14On the bill · sessions
87Companies · in total
§I

Speakers

Programmed at World, in alphabetical order.
Adrian Haigh
Senior Vice President and General Manager Emea and Asia Pacific Region · PTC Therapeutics
Alessandra Ferlini
Director of the Medical Genetics Section & Unit · University of Ferrara
Alexion Pharma Netherlands B.V.
Alexion Pharma Netherlands B.V.
Alexis Arzimanoglou
Coordinator of the European Reference Network for Rare and Complex Epilepsies · EpiCARE
Alicia Be
Senior Account Manager · FIPRA International
Alicia Granados
Global Head Scientific Advocacy and Insights · Sanofi
Amanda Bok
Chief Partnerships Officer · The Synergist
Anjan Aralihalli
President & Founder · Raya Therapeutic, Inc.
Anna Bucsics
Project Advisor · MoCA
Annemieke Aartsma Rus
Professor Of Translational Genetics, Department Of Human Genetics · Leiden University Medical Center (Netherlands)
Annette Bakker
CEO · Childrens Tumor Foundation
Bos
Board Member · SMA Europe
Daria Julkowska
Head of Strategy & Partnerships in Health · ERDERA - INSERM
Dariusz Adamczewski
Managing Director · Children’s Tumor Foundation Europe
David Allison
Chief Executive Officer · TREAT-NMD
Debra Bellon
Strategic Engagement Manager · Rare Diseases International
Derek Ansel
Vice President, Therapeutic Strategy Lead, Rare DiseaseRare Disease · Worldwide Clinical Trials
Dorota Zgodka
Steering Committee member · HTAi Rare Disease Interest Group
Enrico Costa
Head of International Affairs Office & Italian Delegate at the COMP · Italian Medicines Agency (AIFA)
Fabienne Bartoli
Inspector General · French National Authority for Health
Gulcin Gumus
Research and Policy Senior Manager · EURORDIS
Hicham ALAOUI
Patient · AMIS FSH / FSHD EUROPE
§II

Sponsors

The houses behind the program. Tiers as disclosed.
Alliance for Regenerative MedicineSupporting Partner
Americas Health FoundationSupporting Partner
AsebioSupporting Partner
Becaris PublishingSupporting Partner
BIOCATSupporting Partner
BioInsightsSupporting Partner
Biopharma AsiaSupporting Partner
Check RareSupporting Partner
EATRISSupporting Partner
Endo ERNSupporting Partner
EpiCARESupporting Partner
ERDERASupporting Partner & Exhibitor
ERN BONDSupporting Partner
ERN eUROGENSupporting Partner
EuropaBioSupporting Partner
FH Europe Foundation NGOSupporting Partner & Exhibitor
Gene Therapy NetSupporting Partner
HealthEconomics.comSupporting Partner
Health-HollandSupporting Partner
hollandbioSupporting Partner
IRDiRCSupporting Partner
LMNA CardiacSupporting Partner
Market Access TodaySupporting Partner
MarketsandMarketsSupporting Partner
National Pharmaceutical CouncilSupporting Partner
NAVLIN Daily by EversanaSupporting Partner
Pathways To TrustSupporting Partner & Exhibitor
Patient WorthySupporting Partner
Rare Disease 360Supporting Partner
RARE RevolutionSupporting Partner & Exhibitor
SCREEN4CARESupporting Partner
touchENDOCRINOLOGYSupporting Partner
touchHAEMATOLOGYSupporting Partner
touchOPHTHAMOLOGYSupporting Partner
§III

Agenda

Selected from 14 sessions on the bill.
  • Oct 26 10:00

    ERDERA: AI for Rare Diseases: From Promise to Practice

    Artificial intelligence is rapidly becoming an integral part of the rare disease ecosystem, supporting scientific discovery, therapeutic development and strategic decision-making. …

    Workshops
  • Oct 26 14:00

    Early Diagnosis – What’s Needed to Make Change Happen?

    Moving beyond isolated advocacy to accelerate diagnosis, this workshop will holistically explore how early diagnosis fits within the rare disease ecosystem from development of diag…

    Sanofi Workshop
  • Oct 26 14:00

    HTAi: Implementing HTA recommendations, Evidence assessment, economic modelling, and how legislation drives HTA change

    HTAi Workshop
  • Oct 26 15:30

    How can we improve access to orphan drugs in the Netherlands? - Title TBC

    - The need for a separate approval pathway for Orphan Drugs - How will new Reimbursement models help Pharmaceutical companies navigate Orphan Drug development?

    Alexion Workshop
  • Oct 26 17:00

    A Rare Networking Opportunity - Offsite Networking Drinks

    A Rare Networking Opportunity brings together rare disease patient advocates and professionals working towards rare disease therapies - not to pitch or present, but to genuinely co…

    Alexion Workshop
  • Oct 27 10:00

    Keynote Panel: The next frontier in rare disease, where will the breakthroughs come from?

    - What’s in the pipeline and what rare diseases have been targeted? - How have global launch strategies been impacted by MFN? - Emerging platforms (gene, cell, RNA etc) and novel t…

    Keynotes
  • Oct 27 11:30

    Invisible patients or visible impact? Examining patient involvement and influence in Europe’s Joint Clinical Assessments (JCAs) of new medicines

    Europe’s first JCAs have now been published, providing initial opportunity to examine how rare disease patient and caregiver perspectives are incorporated into assessment and wheth…

    Working Groups
  • Oct 27 11:30

    Who Makes the cut? Defining a minimum evidence standard for demographic diversity

    Working Groups
  • Oct 27 12:55

    Start-up Pitch 1: Infirmacea Inc.

    EXPO Stage - Biopharma Showcases
Intermission
Part the Second · For the Buyer

What does it mean when 87 companies show up, and 2 bet on three roles at once?

87 companies. two are betting more than once.

The numbers below are derived from the speakers, sponsors, and exhibitors on this page, cross-referenced into one ledger. They are the only thing here that 10times.com cannot tell you.

Who's in the room
2All threespeak · spons · exh
32Sponsoringsponsor only
1Exhibitingexhibitor only
52Companies Speakingspeaker only

The Triple-Threat

EpiCARE·hollandbio

Speakers by seniority

48% of the speakers carry senior titles: C-suite, Founder, VP, or Director-level.

  • C-suite915%
  • Founder / Owner23%
  • VP-level47%
  • Director / Head of1423%
  • Manager / Lead610%
  • Engineer · IC35%
  • Research / Science12%
  • Advisor / Investor58%
  • Other roles1728%

Of 61 on the bill · classified by free-text title

Most-represented companies
  1. 01EpiCARESp·S1V
  2. 02hollandbioSp·S1V
  3. 03Alliance for Regenerative MedicineSp·S
  4. 04Americas Health FoundationSp·S
  5. 05AsebioSp·S
  6. 06Becaris PublishingSp·S
  7. 07BIOCATSp·S
  8. 08BioInsightsSp·S
  9. 09Biopharma AsiaSp·S
  10. 10Check RareSp·S
  11. 11EATRISSp·S
  12. 12Endo ERNSp·S
  13. 13ERDERASp·S
  14. 14ERN BONDSp·S
Sponsors by tier
supporting partner30
88% of 34
supporting partner & exhibitor4
12% of 34
At peer events but not here

These companies sponsored two or more peer events recently, but aren't on this program. For an organizer, that's a list of warm prospects.

Related events · by shared sponsors

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