Agenda
27-29 October 2025
RAI Congress Center, Amsterdam
Amsterdam, 27 - 29 October 2025
Schedule
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Oct 2710:30
Conference pass
Early Access & Patient Advocacy
Clinigen Workshop
Hollistic Approaches to Rare Disease Drug Development: Integrating ESG, Patient Engagement, and Commercial Strategy for Improved EU Access
Session led by: clinigen-group
Oct 2710:30
Conference pass
Working Group 8: Involving Rare Diseases Patients in Clinical Guidelines’ Development
Working Groups
Moderator: Beatriz Carmona-Hidalgo, Postdoctoral Researcher, Junta de Andalucía
Anabel Granja Dominguez, Researcher, Junta de Andalucía
Juan Dario Ortigoza Escobar, Paediatric Neurologist Consultant, Hospital Sant Joan de Déu
Oct 2711:30
Conference pass
Innovation in LMICs: Overcoming Barries to Access and Advancing Global Rare Disease Solutions
Working Groups
Daria Julkowska, Scientific Coordination, European RD Research Alliance, IRDiRC
Debra Bellon, Strategic Engagement Manager, Rare Diseases International
Durhane Wong-Rieger, President, Chief Executive Officer, Canadian Organisation for Rare Disorder & Rare Diseases International
Oct 2714:00
Conference pass
Enabling Digitally-Enhanced Care for Rare Diseases in Europe
Chiesi Workshop
Lysosomal Storage Disorders (LSDs) present significant challenges, including delayed diagnoses, fragmented care pathways, and limited access to specialist care. Digital health technologies offer a transformative opportunity to address unmet needs by enabling earlier intervention, enhancing care coordination, and empowering patients and healthcare providers with innovative tools.
Utilizing interactive tools and featuring multi-stakeholder perspectives from patients to industry and policymakers, this discussion will explore the integration of digital health innovations ranging from AI to telemedicine, digital medical devices, and other connected care technologies into care pathways for rare diseases across Europe
Session led by: 
Enrico Piccinini, Senior Vice President, EU & International, Rare Diseases, Chiesi Group
Maurizio Scarpa, Director, Coordinating Center For Rare Diseases, MetabERN
Pierantonio Russo, FCCP, FAAP, STS, Corporate Chief Medical Officer, EVERSANA
Kim Angel, Executive Director, International MPS Network
Alberta Spreafico, Senior Vice President, Health Innovation, EVERSANA
Oct 2714:00
Conference pass
The Rare Disease Action Plan – Where are we now?
Sanofi Workshop
Rare diseases pose unique challenges to healthcare systems worldwide due to their low prevalence and complex treatment requirements. Traditional financing mechanisms often fail to adequately address the needs of patients with rare diseases, leading to disparities in access to treatment and care. Last June, RDI (Rare Diseases International) launched a campaign for a World Health Assembly (WHA) Resolution on Rare Diseases in 2025. RDI is launching a campaign for a World Health Assembly (WHA) Resolution on Rare Diseases in 2025.
Moderator: Anne-Sophie Chalandon, Head of Global Public Affairs, Rare Diseases and CGT Policies, Sanofi
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Oct 289:10
Conference pass
Keynote panel: Looking ahead, what does the future hold for Europe’s Life Science Strategy and what does it mean for rare diseases?
Keynotes
Fabienne Bartoli, Inspector General, French National Authority for Health
Christina Kyriakopoulou, Senior Expert - Policy Officer Health Research, European commission
Virginie Bros-Facer, CEO, EURORDIS
Oct 2810:00
Conference pass
Keynote panel: How well has your EUHTA implementation gone?
Keynotes
Niklas Hedberg, Head Of Pharmacy, Tandvards och Lakemedelsformansverket
Anne Willemsen, Co-chair JCA Subgroup, Zorginstituut Nederland
Alexander Natz, Secretary General, EUCOPE - European Confederation of Pharmaceutical Entrepreneurs
Jonathan Neal, Head of Central, South and Eastern Europe Takeda and EFPIA European Markets Committee, Takeda
Judith Fernandez, Deputy Director - HTA department, Haute Aurotité de santé (HAS)
Oct 2811:30
Conference pass
Working Group 1: How can we manage the tension between immediate access to innovation and affordability?
Working Groups
Bhavesh Patel, Principal, CRA, Charles River Associates
Session led by: cra,-charles-river-associates
Oct 2811:30
Conference pass
Working Group 2: From Insight to Action: Leveraging Patient Experience Data Across the Rare Disease Lifecycle
Working Groups
Understanding patient experiences, preferences, and behaviours is critical to developing treatments that truly meet patient needs. Yet in rare diseases, capturing and applying these insights remains difficult due to small, diverse populations, delayed diagnoses, a lack of standardized methods, and limited regulatory guidance.
This panel will explore how both qualitative, and semi-quantitative patient data can inform more effective, patient-centred decisions across the product lifecycle in the rare disease space. Experts will address key barriers, such as data integration, resource constraints, and methodological gaps, and share practical strategies and real-world examples to overcome them.
Join the panel and gain a clear view of what meaningful patient experience data look like and how they can drive clinical development strategies, as well as pre-launch and market success
Oct 2811:30
Conference pass
Working Group 3: Shifting Strands: Operational Challenges and Ethical Dilemmas of Genetic Testing
Working Groups
Drew Matheson, Executive Director. Franchise Area Lead Respiratory Medicine and Infectious Disease, Worldwide Clinical Trials
Oct 2811:30
Conference pass
Working Group 5: Changes in orphan drug legislation and patient access
Working Groups
Reserved for Clinigen
Oct 2811:30
Conference pass
Working Group 6: Challenge of Demonstrating Clinical Meaningfulness in the Context of Rare Disease
Working Groups
Reserved for Decisive Consulting
Oct 2814:10
Conference pass
Horizon scanning towards better payer decision making
Access & Pricing
Moderator: Marcus Guardian, CEO, International Horizon Scanning Initative
Oct 2814:10
Conference pass
Landscape Analysis from the Bottom Up: Letting the Patients Tell You Which Sites to Use in Your Rare Disease Trial
Clinical Development
Juliet Moritz, Senior VP Strategic Solutions and Patient Centricity, Ergomed
Oct 2814:10
Conference pass
Panel: Platform approach to GLP – moving away from product authorization
Cell & Gene Therapy
To invite:
Constanz Blume, Global Regulatory Affairs, BionTech
Kent Rogers, CEO, Everyone Medicines
Senior EMA Representative
Moderator: Alexander Natz, Secretary General, EUCOPE - European Confederation of Pharmaceutical Entrepreneurs
Oct 2814:10
Conference pass
Regulatory Convergence – innovative approaches for clinical research showcasing ERDERA-INVENTS results
Science & Strategy
Senior Representative, ERDERA
Oct 2814:10
Conference pass
Strategies for securing early stage funding and maximising fundraising opportunities
Investment & Funding
Thom Frielink, Associate, Curie Capital BV
Oct 2814:10
Conference pass
The growing influence of patient voices in Rare
Patient Centricity
Senior Representative, UCB
Oct 2814:10
Conference pass
The need for better diagnosis in rare primary immunodeficiencies
Precision Medicine
Senior Representative, EUCOPE
Oct 2814:10
Conference pass
The utilisation of healthcare resource and economic burden on patients living with rare diseases – Title TBC
Real World Evidence
Reserved for EVERSANA
Session led by: 
Oct 2814:15
Conference pass
Uncovering genetically defined patients for improved diagnosis, drug development and commercial strategy
BioPharma Showcases
Karen Malone, CEO, GeneScape
Oct 2814:25
Conference pass
Natural history studies of how to understand a rare disease
Clinical Development
Christopher Rudolf, Founder and Chief Executive Officer, Volv Global
Leon van Wouwe, Clinical Innovation Director, Volv Global
Oct 2814:30
Conference pass
How to scale individualized therapies – perspectives from the front lines
BioPharma Showcases
Session led by: 
Oct 2814:40
Conference pass
AI & the use of DHT Technologies
Precision Medicine
Violeta Stoyanova-Beninska, Senior Scientific Specialist at Human Division EMA, Chair of Regulatory Science Committee, IRDiRC, EMA
Oct 2814:40
Conference pass
Challenges of Late onset Rare diseases – recommendations from the COLLABORATE Consensus Paper From Complexity to Clarity: Overcoming Reimbursement Challenges for Orphan Drugs
Access & Pricing
Elise Evers, Senior Consultant, Initiate
Georgia Roberts, Senior Consultant HTA, Initiate
Oct 2814:40
Conference pass
CollaboRARE: How to collect patient experience data for regulatory use – Title TBC
Patient Centricity
Moderator: Maria Cavaller Bellaubi, Patient Engagement and Therapeutic Development Director, EURORDIS
Julian Isla, Director, Foundation 29
Oct 2814:40
Conference pass
Optimizing Clinical Trial Design for Orphan Drugs: Addressing Recruitment, Efficacy, and Safety in Rare Diseases
Clinical Development
Carlos Pinoargote, Chief Operating Officer, BRCR Global
Session led by: 
Oct 2814:40
Conference pass
The use of Delphi Panels in RWE
Real World Evidence
Senior Representative, Costello
Session led by: 
Oct 2814:40
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What funding opportunities can patient groups maximise to advocate for new treatments?
Investment & Funding
Stefano Benvenuti, Head of Public Affairs and Market Access, Fondazione Telethon
Federica Miotto, head of medical affairs, Fondazione Telethon
Oct 2814:45
Conference pass
ALEETO™️, protein complex derived from stimulated stem cells, a novel neural repair reagent from bench to clinic
BioPharma Showcases
Oct 2814:50
Conference pass
IRDiRC Taskforce on prevention: Opportunities and Challenges in the shift from treatment to prevention
Science & Strategy
More speakers to be confirmed..
Moderator: Daniel O'Connor, Director, Regulatory Policy & Early Access, ABPI
Oct 2814:55
Conference pass
Panel: How will be the JCA for ATMPs? Questions and answers from the ATMP ecosystem
Cell & Gene Therapy
More speakers to be confirmed..
Moderator: Mark Battaglini, Chief Strategy Officer, Alliance for Regenerative Medicine
Paolo Morgese, Vice President, Public Affairs, Europe, Alliance for Regenerative Medicine
Oct 2815:00
Conference pass
Oct 2815:10
Conference pass
Aspire4Rare: Acting Now for People Living with Rare Diseases
Access & Pricing
Senior Representative, UCB
Oct 2815:10
Conference pass
Beyond Recruitment: The Value of Patient and Care Network Education in Rare Disease Clinical Research
Patient Centricity
Paula Orandash, Director, Patient Engagement, Ergomed
Oct 2815:10
Conference pass
Bridging the Gap: Clinical Trials as a Standard of Care in Rare Diseases
Clinical Development
Derek Ansel, Vice President, Therapeutic Strategy Lead, Rare Disease, Worldwide Clinical Trials
Oct 2815:10
Conference pass
Leveraging Compassionate Use Programs to Accelerate Investigational Drug Development in the United States
Real World Evidence
Ana Tediosi, Head of Expanded Access Program Strategy, WEP Clinical
Session led by: 
Oct 2815:10
Conference pass
The Role of Foundations in Advancing Rare Disease Research: Funding Models and Success Stories
Investment & Funding
Sebastian Honoré, Cure Lowe Foundation
Oct 2815:10
Conference pass
The role of patient advocacy in access to whole genome sequencing for cancer of unknown primary
Precision Medicine
Oct 2815:30
Conference pass
Clinical stage program on rare genetic disease Hypophosphatasia
BioPharma Showcases
Juliane Bernholz, Chief Operating Officer, A.M. Pharma
Oct 2815:45
Conference pass
What kind of data/assets can patient groups bring into pre-clinical or clinical development?
BioPharma Showcases
Ron Jortner, Founder & CEO, Aspire Biosciences
Oct 2816:00
Conference pass
Metabolic plasmalogen replacement therapy for a rare peroxisomal disorder – Title tbc
BioPharma Showcases
Oct 2816:10
Conference pass
AI and it’s use in access and pricing
Access & Pricing
Sven Tops, Chief Market Access Officer, Kintiga
Oct 2816:10
Conference pass
An integrated approach to gathering the voice of the patient in an ultra-rare pediatric disease
Patient Centricity
Oct 2816:10
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Panel: Innovative funding mechanisms and business models to de-risk Orphan Drug investment
Investment & Funding
Invited:
Francis Pang, SVP, Global Market Access & International Geographic Expansion, Orchard Therapeutics
Liz Fowler, Former Director, CMS Innovation Center; Former VP, Global Health Policy, J&J
Rosana Sovani, Partner, Head of Legal Public Affairs, LS CUBE Studio Legale
Moderator: Victor Maertens, Government Affairs Director, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
Oct 2816:10
Conference pass
Panel: Issues on how to characterise rare diseases
Science & Strategy
Invited:
Senior HTA Representative
Senior Clinician
Senior Geneticist
Moderator: Sheela Upadhyaya, Life Science Advisor in Rare Diseases & Special Advisor, Openflex Ltd
Oct 2816:10
Conference pass
Panel: The use of rare disease registries – bridging the gap between regulatory and patients
Real World Evidence
Invited speakers:
Prof. Peter Mol, Professor, Drug Regulatory Science, University Medical Center Groningen, CHMP Member, CBG-MEB
Senior ERN Representative
Senior Patient Representative
Moderator: Karen Facey, Senior Research Fellow, University of Edinburgh
Oct 2816:10
Conference pass
Reimbursement timelines and key access challenges for Cell & Gene therapies in the Netherlands vs other EU countries
Cell & Gene Therapy
Angela Borghouts - de Ruijter, Associate Director Market Access, Gilead Sciences
Oct 2816:10
Conference pass
Sustainability of lifetime dosing for antisense oligonucleotides in rare diseases: The need for scalable national models
Clinical Development
Rachel Smith, Executive Director, Global Head of Rare Disease, CoE, Parexel
Oct 2816:10
Conference pass
The First Closed-Loop Breathing Pacemaker: Toward an Artificial Pancreas Respiratory Drive
Precision Medicine
James Oakley, CEO, Keep Me Breathing
Oct 2816:25
Conference pass
Integrating market access into regulatory strategies
Access & Pricing
Oct 2816:30
Conference pass
A new definition of Unmet Medical Need in EU pharma legislation; impact on innovation and access for patients
Cell & Gene Therapy
Thomas Bols, Head of Government Affairs and Patient Advocacy, EMEA & APAC, PTC Therapeutics
Oct 2816:40
Conference pass
Panel: EU’s Rare Opportunity: Navigating the new Joint Clinical Assessment route in Europe
Access & Pricing
Senior Representative, EVIDERA
more speakers to be confirmed
Julien Delaye, Patient Engagement Manager – HTA, EURORDIS, EURORDIS
Oct 2816:40
Conference pass
Patient centricity – from ambition to implementation
Patient Centricity
Dieter Schultewolter, Head of Global Medical Affairs Neuroscience, Teva Pharmaceuticals
Andy Barrick, CEO, Multiple System Atrophy Trust UK
Oct 2816:40
Conference pass
Rare Kidney conditions HE Report
Clinical Development
Aisling McMahon, Kidney Research UK
Oct 2816:50
Conference pass
A gene therapy approach for ARSACS: insights from preclinical models
Cell & Gene Therapy
Daniele De Ritis, Researcher, San Raffaele Scientific Institute
Francesca Maltecca, Group Leader, San Raffaele Scientific Institute
Oct 2816:55
Conference pass
Considerations when conducting your rare pediatric programmes
Clinical Development
Jonathan Kornstein, VP, Rare Disease & Pediatrics Clinical Development, Caidya
Oct 2817:10
Conference pass
Diversity and Equity in Clinical Studies: Impact on Rare Disease Studies
Clinical Development
Corinne Blanchet, Executive Director, Program Delivery, Premier Research
Session led by: 
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Oct 299:00
Conference pass
Keynote Panel: Leveraging new technology for smarter decision-making in Rare Disease drug development and access
Keynotes
Moderator: Claire Skentelbery, Director General, EuropaBio
Christopher Rudolf, Founder and Chief Executive Officer, Volv Global
Julian Isla, Director, Foundation 29
Stelios Kympouropoulos, MEP, European Parliament
Mohit Jain, Vice President, Market Access, International Operations, IntraBio
Wim Goettsch, Special Advisor HTA & Professor for HTA of Pharmaceuticals, Zorginstituut
Michael Merchant, National Institute for Health and Care Excellence
Oct 299:45
Conference pass
Keynote panel: New treatments on the block – 2025’s rare disease pipeline
Keynotes
Moderator: Paolo Morgese, Vice President, Public Affairs, Europe, Alliance for Regenerative Medicine
P.J. Brooks, Deputy Director, Division of Rare Diseases Research Innovation, NIH/NCATS
Violeta Stoyanova-Beninska, Senior Scientific Specialist at Human Division EMA, Chair of Regulatory Science Committee, IRDiRC, EMA
Gabriella Almberg, Head of Health System Policy & Public Affairs, UCB
Oct 2911:15
Conference pass
Accelerating rare disease diagnosis beyond birth
Precision Medicine
Stefano Portolano, CEO, Azafaros
Oct 2911:15
Conference pass
Access and HTA pathways for OMPs – do they improve access and drive incentive for innovation?
Access & Pricing
Senior Representative, Ipsen
Session led by: 
Oct 2911:15
Conference pass
Advancement of analytical tools for testing and characterization of AAV gene therapy products
Manufacturing
Bart van Montfort, Scientific Director Biologics & Scientific Leader Gene Therapy, Johnson and Johnson
Oct 2911:15
Conference pass
Diversifying Rare Disease Research: Strategies for Expanding Participation
Clinical Development
Senior Representative, Allucent
Session led by: 
Oct 2911:15
Conference pass
DMAb Technology: The Transformational Potential of Next Gen DNA Medicine in Rare Disease
Cell & Gene Therapy
Trevor Smith, VP Preclinical R&D, Inovio Pharmaceuticals
Oct 2911:15
Conference pass
Panel: Defining the building blocks to enhance the research and therapeutic development pathway to be more psychologically informed
Patient Centricity
Matt Bolz-Johnson, Mental Health Lead & Healthcare Advisor, EURORDIS
Oct 2911:15
Conference pass
Panel: Launch to Global – Best practices to successfully launch Orphan Drugs in Europe
Science & Strategy
Senior Representative, Mereo Pharma
Oct 2911:15
Conference pass
Strategic use of RWD for the use towards HTA submissions
Real World Evidence
Invited:
Frauke Naumann-Winter, Committee for Orphan Medicinal Products, Institute for Drugs and Medical Devices – TBC
Oct 2911:45
Conference pass
Maximizing Value & Positioning in the Emerging Competitive Rare Disease Arena
Access & Pricing
Bhavesh Patel, Principal, CRA, Charles River Associates
Oct 2911:45
Conference pass
Navigating the Uncharted: Patient Experience Mapping in Rare Disease
Real World Evidence
Senior Representative, Ipsen
Session led by:
Oct 2911:45
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Panel: Competition of regulatory ecosystems in approving medicines: policy implications in the case of Europe
Science & Strategy
Moderator: Pedro Franco, Head of Europe Global Regulatory and Scientific Policy, Merck
Oct 2911:45
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Panel: Ensuring ATMP Manufacturing Excellence in EU – the need for a policy framework and opportunities in the Biotech Act
Manufacturing
Invited speakers:
Senior Representative, Miltenyi Biotech
Senior Representative, European Commission
More to be confirmed..
Moderator: Victor Maertens, Government Affairs Director, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
Oct 2911:45
Conference pass
Panel: The Impact of Biotechnology: Evolution and Revolution in Rare Diseases
Cell & Gene Therapy
Senior Representative, HollandBio
Moderator: Claire Skentelbery, Director General, EuropaBio
Adrien Samson, Healthcare policy senior manager, EuropaBio
Oct 2911:45
Conference pass
Personalised medicine dilemma – how do we generate data?
Precision Medicine
Senior ERN Representative
Oct 2911:45
Conference pass
Rare Kidney conditions HE Report
Clinical Development
Aisling McMahon, Kidney Research UK
Oct 2912:00
Conference pass
Panel: Implications of EU HTA Joint Clinical Assessments to Patient Engagement Practices in Rare Diseases
Patient Centricity
This panel will explore thepractical implications that industry and patient organizations need to consider when engaging given the evolving landscape of HTA in Europe
Moderator: Veronica Lopez Gousset, Independent Consultant, VLG Consulting
Alexander Natz, Secretary General, EUCOPE - European Confederation of Pharmaceutical Entrepreneurs
Sheela Upadhyaya, Life Science Advisor in Rare Diseases & Special Advisor, Openflex Ltd
Oct 2912:15
Conference pass
Convergence on rare disease and precision medicine – a real drive on drug development and patient advocacy
Precision Medicine
Durhane Wong-Rieger, President, Chief Executive Officer, Canadian Organisation for Rare Disorder & Rare Diseases International
Oct 2912:15
Conference pass
How do you assess evidence in Rare? The work of HTAi RDIG
Access & Pricing
Alicia Granados, Head, Global Rare Diseases Medical Scientific Advocacy, Sanofi
Oct 2912:15
Conference pass
Unique Challenges in Recruiting Patients for Rare Indication Paediatric Studies: Overcoming Difficulties through a Site and CRO Perspective
Clinical Development
Rachel Paiva Petean Abu Taleb, Medical Monitor, Veristat
Oct 2912:15
Conference pass
Volume and willingness to pay – strengthening access to medicines for rare diseases to sustainable costs
Real World Evidence
Invited:
Sofie Alverlind,Coordinator and Project Leader,TLV
Oct 2913:45
Conference pass
MoCA and JSC in practice - a multistakeholder perspective
Access & Pricing
Sarka Kubinova, Medical evaluator, MoCA coordinator, Czech Republic State Institute for Drug Control
Oct 2913:45
Conference pass
Navigating FDA Processes, including overcoming a partial clinical hold
Clinical Development
Senior Representative, Rezolute Bio
Oct 2913:45
Conference pass
New platforms for RNA therapy development and regulatory considerations – ERDERA
Cell & Gene Therapy
Daria Julkowska, Scientific Coordination, European RD Research Alliance, IRDiRC
Oct 2913:45
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Panel: Drug Repositioning – How to get shelved assets that have value for the Orphan drug value chain
Science & Strategy
Moderator: Annette Bakker, CEO, Childrens Tumor Foundation
Oct 2913:45
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Panel: Exceptional Use of an Orphan Drug for an Ultra Rare Disease
Real World Evidence
Denis Broun, Director Givopax, Former Executive Director of UNTAID, World Health Organization, & Medical Director, Menkes International Association
Elena Godoy, Attending Pediatrician, Department of Pediatrics, Málaga Regional University Hospital
Oct 2913:45
Conference pass
The EU Biotechnology and Biomanufacturing Initiative Opportunities for Investment into Rare Disease Innovation and delivery
Manufacturing
Adrien Samson, Healthcare policy senior manager, EuropaBio
Claire Skentelbery, Director General, EuropaBio
Oct 2913:45
Conference pass
The story of Marco: Ultra rare case study towards the treatment of Menkes Disease
Patient Centricity
Oct 2914:00
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ARSACS (Ataxia Charlevoix-Saguenay): Finding Solutions for Rare Disease Patients – title TBC
Patient Centricity
Invited speaker:
Dr. Bart Van de Warrenburg, Professor of Neurology & Medical Lead, Center for Rare and Genetic movement disorders, Radboudumc
Sonia Gobeil, Cofounder, Ataxia of Charlevoix-Saguenay Foundation
Oct 2914:05
Conference pass
Transitioning from US coordinated clinical trials to commercialization in Europe
Clinical Development
Tom Pulles, MD, VP, Head of Medical Affairs & Patient Advocacy, Europe, Acadia Pharmaceuticals Inc.
Oct 2914:05
Conference pass
Will the bubble burst on rare disease and orphan drugs? Paying for innovation and access.
Access & Pricing
Oct 2914:10
Conference pass
Strategies for securing early stage funding and maximising fundraising opportunities
Investment & Funding
Thom Frielink, Associate, Curie Capital BV
Oct 2914:25
Conference pass
Strategic Evidence Generation – Exploring Multiple Stakeholder Needs
Access & Pricing
Josie Godfrey, Director, JG Zebra Consulting
Oct 2914:25
Conference pass
The Role of Basket & Umbrella Clinical Trials in Tackling Rare Diseases
Clinical Development
Senior Representative, C-Path
Oct 2914:30
Conference pass
ATMPs: Essential Tools or Essential Medicines?
Cell & Gene Therapy
Senior Representative, Miltenyi Biotec
Oct 2914:30
Conference pass
Oct 2914:30
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Pharmacometrics and manufacturing for exceptional use of an orphan drug
Manufacturing
Denis Broun, Director Givopax, Former Executive Director of UNTAID, World Health Organization, & Medical Director, Menkes International Association
Oct 2914:30
Conference pass
Unmet Medical Need in Haemophilia
Patient Centricity
Invited:
Senior Representative, Novo Nordisk
European Hemophilia Consortium
ERN Blood
Oct 2914:45
Conference pass
Patients voice in clinical trials design and outcomes
Clinical Development
Senior Representative, FH Europe
Oct 2915:40
Conference pass
Panel: Driving Patient-Centric Innovation: Pharma’s Involvement and Stakeholder Collaboration
Patient Centricity
More speakers to be confirmed..
Oct 2916:30
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Keynote Panel: In what way can we mobilise, enable and leverage international policies around the world for PLWRDs?
Keynotes
Invited: Dr Mohamed Hassany, Assistant Minister for Public Health Initiative, Ministry of Health Egypt
Moderator: Yann Le Cam, Founder & Past-CEO of EURORDIS-Rare Disease Europe, EURORDIS-Rare Diseases Europe
Anne-Sophie Chalandon, Head of Global Public Affairs, Rare Diseases and CGT Policies, Sanofi
Alexandra Heumber Perry, Chief Executive Officer, Rare Diseases International
Nadiah Hanim Abdul Latif, President, Malaysian Rare Disorders Society
last published: 19/Jun/25 12:15 GMT
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