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26 October 2026 | Workshop Day
27-28 October 2026 | Conference & Exhibition
RAI Congress 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.
2,000+
Attendees
250+
Speakers
100+
Exhibitors
3
Days
1,000+
1 to 1 Partnering Meetings
10,000+
Networking Connections
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.
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
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
ManufactureIn Partnership With
Past Speakers
OMAR ALIFORMER ADVISER NICE / HEAD OF PAYERSVERPORA
Derek AnselVice President, Therapeutic Strategy Lead, Rare DiseaseRare Diseaseworldwide clinical trials
Fabienne BartoliInspector GeneralFrench National Authority for Health
Stefano BenvenutiHead of Public Affairs and Market Access, Chair of IRDiRC Funders Constituent Committee (CCC)Fondazione Telethon
Corinne BlanchetExecutive Director, Program Delivery, Rare Disease & PediatricsPremier Research
Lara BloomPresident and CEOThe Ehlers-Danlos Society
Thomas BolsHead of Government Affairs and Patient Advocacy, EMEA & APACPTC Therapeutics
Matt Bolz-JohnsonMental Health Lead & Healthcare AdvisorEURORDIS
Anne-Sophie ChalandonHead of Global Public Affairs, Rare Diseases and CGT PoliciesSANOFI
William ColeLife Science Partnership ManagerKidney Research UK
Elizabeth De LangeProfessor in Predictive PharmacologyLeiden Amsterdam Center for Drug Research LACDR
Toon DigneffeHead, Public Affairs & Public Policy, Europe & CanadaTakeda
João DuarteVice President, Head of Global Regulatory Affairs ExcellenceIpsen
Elise EversSenior ConsultantInitiate
Pedro FrancoHead of Europe Global Regulatory and Scientific PolicyMerck
Thom FrielinkSr. AssociateCurie Capital BV
Diego Fernando Gil CardozoPresidentEnfermedades Raras en el Caribe y América Latina (ERCAL)
Sonia GobeilCofounderAtaxia of Charlevoix-Saguenay Foundation
Josie GodfreyDirectorJG Zebra Consulting
Wim GoettschSpecial Advisor HTA & Professor for HTA of PharmaceuticalsZorginstituut
Alicia GranadosGlobal Head Scientific Advocacy and InsightsSanofi
Marcus GuardianCEOInternational Horizon Scanning Initative
Niklas HedbergHead Of PharmacyTandvards och Lakemedelsformansverket
Patrick HopkinsonIndependent HTA ExpertIndependent HTA Expert
Julian IslaDirectorFoundation 29
Anneliene JonkerRare Disease Therapy ResearcherUniversity of Twente
Ron JortnerFounder & CEOAspire Biosciences
Jonathan KornsteinVP, Rare Disease & Pediatrics Clinical DevelopmentCaidya
Stelios Kympouropoulosf.MEPEuropean Parliament
Christina KyriakopoulouPolicy Officer, Health Research ProgrammesEuropean Commission
Yann Le CamFounder & Past-CEO of EURORDIS-Rare Disease EuropeVOZ Advisors
MI LIUProfessorSoochow University
Karen MaloneCEOGeneScape
Aurora MateosFounder & DirectorMenkes International Association
Jan MolPatient AdvocateHematon
Juliet MoritzSenior VP Strategic Solutions and Patient CentricityERGOMED Clinical Research
Alexander NatzSecretary GeneralEUCOPE - European Confederation of Pharmaceutical Entrepreneurs
Jonathan NealHead of Central, South and Eastern Europe Takeda and EFPIA European Markets CommitteeTakeda
Daniel O'ConnorDirector, Regulatory Policy & Early AccessABPI
Francesc PalauDistinguished InvestigatorSJD Barcelona Children's Hospital
Samantha ParkerPatient Engagement Lead Rare Diseases and Vice Chair of IRDiRCItalfarmaco
David PearceChair, International Rare Disease Research Consortium (IRDiRC)Sanford Health
Enrico PiccininiSenior Vice President, EU & International, Rare DiseasesChiesi Group
Shawn RitchieChief Executive OfficerMed-Life Discoveries
Georgia RobertsSenior Consultant HTAInitiate
Christopher RudolfFounder & CEOVolv Global
Adrien SamsonHealthcare policy senior managerEuropaBio
Jörg SchüttrumpfChief Scientific Innovation OfficerGrifols
Claire SkentelberyDirector GeneralEuropaBio
Tara SmithExec VP, Innovative TherapiesMed-Life Discoveries
Rachel SmithVice President Therapeutic Area LeadParexel International LLC
Violeta Stoyanova-BeninskaSenior Scientific Specialist at Human Division EMA, Chair of Regulatory Science Committee, IRDiRC, former Chair of COMP at EMAEMA
Kwee Lan TanHead of Team Regional Regulatory LeadBoehringer Ingelheim
Sven TopsChief Market Access OfficerKintiga
Sheela UpadhyayaLife Science Advisor in Rare DiseasesOpenflex Ltd
Anne WillemsenCo-chair JCA SubgroupZorginstituut Nederland
Durhane Wong-RiegerPresident, Chief Executive OfficerCanadian Organisation for Rare Disorder & Rare Diseases International
Martine ZimmermannSenior Vice President, Head of Global Regulatory AffairsIpsen
Giulia PieriniPartner, Strategic Patient Engagement & AdvocacyAlira Health
Sebastian HonoréCEOCure Lowe Foundation
Victor MaertensGovernment Affairs DirectorEuropean Confederation of Pharmaceutical Entrepreneurs (Belgium)
Aisling McMahonKidney Research UK
Warnyta MinnaardPatient advocateMissie Tumor Onbekend
Paolo MorgeseVice President, Public Affairs, EuropeAlliance for Regenerative Medicine
James OakleyCEOKeep Me Breathing
Paula OrandashDirector, Patient EngagementERGOMED Clinical Research
Trevor smithVPInovio
Annette BakkerCEOChildrens Tumor Foundation
Karen FaceyChair HTAi Rare Disease Interest GroupUniversity of Edinburgh
Dieter SchultewolterHead of Global Medical Affairs NeuroscienceTeva Pharmaceuticals
Leon van WouweClinical Innovation DirectorVolv Global
Chris Grimes-CromptonPrincipal ConsultantDECISIVE CONSULTING LTD
Kent RogersCEOEveryONE Medicines
Tom Pulles, MDVP, Head of Medical Affairs & Patient Advocacy, EuropeAcadia Pharmaceuticals Inc.
Diana SinkevichVP Market Access and HEORChiesi
Andy BarrickCEOMultiple System Atrophy Trust UK
Francesca MalteccaGroup LeaderSan Raffaele Scientific Institute
Juliane BernholzCEOA.M. Pharma
Carla HollakProfessor Genetic Metabolic DiseasesAmsterdam umc
Beatriz Carmona-HidalgoPostdoctoral ResearcherJunta de Andalucía
Anabel Granja DominguezResearcherJunta de Andalucía
Alisha AngdembeSenior Health EconomistVisible Analytics
Anjan AralihalliFounderRaya Therapeutic, Inc.
Anke Arnold-TuguluChief Regulatory OfficerAzafaros
Thomas BroekhoffProject Managerhollandbio
Daniel LewiCo-founder & Chief Executive OfficerThe Cure & Action for Tay-Sachs (CATS) Foundation
Gisela LinthorstHead of Patient Engagement and AdvocacyAzafaros
Sara BrambillaGlobal Programme ManagerRare Diseases International
Pascal BrokmeierHead of EngineeringEvery Cure
Henriette BurghoornAssociate Director, Market AccessGilead Sciences
Elly DarkinDirector, Government Affairs & Policy, Europe & Canada, Rare DiseasesAlexion – AstraZeneca Rare Disease
Julien DelayePatient Engagement Manager – HTA, EURORDISEURORDIS
Jessie DubiefSocial Research DirectorEURORDIS
Nevan ElamFounder and Chief Executive OfficerRezolute, Inc.
Adrian GoretzkiPresidentHealthcare Education Institute
Jaap GroothoffMedical SpecialistAmsterdam umc
Daria JulkowskaScientific CoordinatorERDERA - INSERM
Maria-Cruz MorilloGlobal Therapeutic Operations Lead, Rare DiseasesAllucent
Alberta SpreaficoSenior Vice President, Health InnovationEversana
Lonneke Timmersstrategic advisorZorginstituut Nederland / National Health Care Institute
Sophie H. TurnerFounder & Strategic Patient AdvocateEmpowered By Us
Silvia van der FlierMedical AdvisorVgz
Wieteke WoutersProgramme Directorhollandbio
Kim AngelExecutive DirectorInternational MPS Network
Daniele De RitisResearcherSan Raffaele Scientific Institute
Pierantonio Russo, FCCP, FAAP, STSCorporate Chief Medical OfficerEversana
Angela Borghouts - de RuijterAssociate Director Market AccessGilead Sciences
Peter MolUMCG - University Medical Center Groningen
Michael MerchantScientific AdviserNational Institute for Health and Care Excellence
Maurizio ScarpaDirector, Coordinating Center For Rare DiseasesMetabERN
Kieron LewisDirector of Strategic ConsultingClinigen Group
Joe StandingProfessor of PharmacometricsUniversity College London
Francis PangSVP Global Market Access and International Geographic ExpansionOrchard Therapeutics
Nathan ChadwickSenior Director, Therapeutic Strategy Lead - Rare Diseaseworldwide clinical trials
Raymond HumlVice President, Rare Disease StrategySciensus
William JangTeam MemberBioMe, Inc.
Manohar KatakamChief Executive OfficerSteroTherapeutics
Sumira RiazPsychologist and founderUnboxed Psychology
Eleanor ThurtlePrincipal ConsultantCostello Medical
Miikka VikkulaProfessor of Human Geneticsde Duve Institute
Dimitrios AthanasiouBoDWORLD DUCHENNE ORGANIZATION / UPPMD
Viviana GiannuzziHead of Research and Innovation DepartmentFondazione per la ricerca farmacologica Gianni Benzi onlus
Rachel HarrisonDirector, Managed Access Programargenx
clive phillipsCEOMaking the Most of Now
Anke Pisters-van RoyMDCZ
Carelle Reparon-SchuijtMedical Advisor IMFZilveren Kruis
Michael WilburFounderMW Advocacy Solutions
Sorrel WolowaczHead European Health EconomicsR.T.I. Health Solutions
Danielle DongGlobal Medical Scientific Advocacy Lead, Rare DiseaseSanofi
Rita FranciscoSurvey Project ManagerEURORDIS
Hannah HarveyProjects ManagerBeacon for rare diseases
Anna KoleGlobal Patient Engagement LeadUCB Pharma
Ruchi SharmaCEOStemnovate Limited
Bart van de WarrenburgProfessor of NeurologyRadboudUMC
Silvy MardiguianHead of HEOR, Europe & New MarketsBeOne Medicines
Susanne MichelHead of Market Access and HEOR EuropeClinigen
Charlie AardewijnResearcher and patient-voice advocateVU Amsterdam
Pedro CampinoSr. Dir., Regulatory TA Lead – ImmunoglobulinsCSL Behring
Giulia ColomboGlobal Director Payer Partnershipcsl
Joslyn CrowePatient Engagement & AdvocacyLexeo Therapeutics
Pam CusickSenior Vice PresidentRare Patient Voice, LLC
Jesper JorgensenNew Product Planning LeadUCB
Bojan JovicMedical DirectorAllucent
Jen LeePrincipal ConsultantHEOR Ltd
Vincent LévêqueCo-founder & CEOMirava Bio
Joshua Lorenz-GuertinVice President, Research & DevelopmentSharp Therapeutics
Flaminia MacchiaChief Operating OfficerORPHANET-AISBL
Monica OttoAssociate Professor of PracticeSDA Bocconi School of Management
Vinciane QuoidbachManager, Public Health And Policy, Research ProjectEuropean Brain Council
Adlane SoudaniGlobal Asset Lead Rare DiseaseIpsen
Ales StrancarMDSartorius BIA Seperations
Gerdi StrydomManaging DirectorValid Insight
Jose Terencio AlemanySenior Vice President of DiscoveryGRIFOLS SA
Nitza ThomassonExecutive Director, Global Head of R&D NeurologyServier
Tracey TingleDirector, Commercial Strategy, Market AccessWEP Clinical
Miriam Wagner LongProject Mercury // World FSHD AllianceFSHD World Alliance // FSHD Society
Katie WallerHead of Patient ProgrammesThe Lily Foundation
Kate WitkowskaStrategic Partnerships DirectorGenomics England
Kaja ZarakowskaHead of Medical Diagnosis InnovationUCB
Julian HowellCEOPharmaKrysto Ltd
Fabrice LejeuneCSOGenvade Therapeutics
Kit RoesProfessorRadboudUMC
Janneke van de KampCEONorgine
Monique van ScherpenzeelCEO & FounderGlycoMScan B.V.
Michelle MellionChief Medical OfficerEveryONE Medicines
Boris SevarikaCo-Founder and CEONanoZymeX
Leonid StoianovCEOTrialize
Delphine WagnerSenior Director, Regulatory AffairsKinesys Consulting
Cristobal Muñoz-SánchezProject management/RDIG projects coordinatorJunta de Andalucía
Yiwei ChenFounderWonder Sir
Giorgia CrimiPatient Advocacy ManagerFondazione Telethon
Elizabeth DeFendisGovernment Business Development SpecialistBRCR Global
Yannick LampoBDSherwood Pharmaceuticals
Samantha LawesDirector of Research Development & PartnershipsKidney Research UK
Sophie MuirChair of TrusteesTimothy Syndrome Alliance (TSA)
Chris ZhouVice PresidentACA Pharma
Vytenis AndriukaitisMEPEuropean Parliament
Rosa CastroPublic Affairs DirectorEURORDIS
Andrew CumminsVice President of Business DevelopmentSciensus
Erwan GicquelGovernment Affairs HeadMiltenyi Biotec
Mathieu LoiseauDirector of Rare Clinical ServicesSciensus
Douglas NivenStrategy Leader in Market AccessSciensus
Andrew ThomsonConsultantRegnitio
Hanka Dekkerexec directorVKS
Mimi LeeCheif of Precision MedicineGeneDx
Veronica Lopez GoussetDrPH candidateHarvard T.H. Chan School of Public Health
Josephine MossetSenior Policy OfficerCancer Patients Europe
Lydia ShottonAssociate Director EU Government AffairsAlliance for Regenerative Medicine
Anton UssiCEOEATRIS ERIC
Mary WangDirector, Patient EngagementGenespire
Astri ArnesenPresidentEuropean Federation of Neurological Associations
Anissa BounabiAssistant scientific coordinatorERDERA - INSERM
Paul BroomheadHead of SiteSGS Integrated CDMO Solutions
Vincent DamotteScientific Project Manager - INVENTSINSERM
Maciej GajewskiVice President, Head Global Corporate Affairs, Rare DiseasesIpsen
Remon HelmyAssociate Director HEORAlexion
Rima NabboutMD/PhDHopital Necker- Enfants Malades
Mark SheehanProfessorUniversity of Oxford
Laurens SluijtermanPostdoctoral ResearcherRadboudUMC
Samuele DoratoriHead of NeurologyServier
Jennifer SchranzSVP, Global Head Rare DiseasesIpsen
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“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
- 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
- Metabolic Support UK
“Great networking opportunities, great panels and discussions!”
- EURORDIS
- EURORDIS
“Good mix of plenaries and workshops, lots of meeting opportunities, relevant topic selection, very friendly and supportive organizer staff”
- Cerner Enviza
- Cerner Enviza
“Excellent organization, very interesting sessions and great networking opportunities.”
- GenCC GmbH
- GenCC GmbH
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