2025 Speakers

Charlie Aardewijn is a dedicated researcher in (International) Public Health at the Vrije Universiteit Amsterdam. With a background in innovation sciences and a deep commitment to improving the lives of individuals affected by rare diseases, Charlie's work focuses on patient engagement in...

A former government pharmacist payer with over 15 years experience in access and reimbursement for new medicines and advising NHS government guidelines at NICE HTA Omar has been a visiting Lecturer at UCLH Pharmacy Programme and was both Lecturer & Examiner on Independent Prescribing the...

Vytenis Povilas Andriukaitis is a prominent Member of the European Parliament, representing Lithuania for the Lietuvos socialdemokratų partija and sitting with the Progressive Alliance of Socialists and Democrats (S&D) Group. His parliamentary focus is heavily concentrated on...

Alisha Angdembe is a Senior Health Economist who leverages data and analytics to inform high-impact decisions across the healthcare sector. With deep expertise in health economics and a clear understanding of the orphan drug landscape, she brings a unique, evidence-driven perspective. At the World...

Kim Angel is the Executive Director of the International MPS Network (IMPSN) and a committed advocate for individuals and families affected by mucopolysaccharidoses (MPS) and related rare diseases. With a background in organizational leadership and a deep passion for driving meaningful change, Kim...

Derek Ansel, MS, LCGC Derek Ansel is Vice President, Therapeutic Strategy Lead, Rare Disease. Derek has worked in clinical research for more than 13 years with a focus almost exclusively on rare and pediatric diseases, including neurodevelopment disorders, non-malignant hematology, autoimmune...

Anjan founded Raya Therapeutic in 2019. Raya is focused on developing 5 clinical stage NCEs to treat ALS and potentially other neurodegenerative diseases. Prior to Raya, Anjan worked at a variety of biotechs and pharma companies and is a venture partner at CTI Life Sciences Fund 2 (Canada). He has...

I come from a family affected by Huntingtons Disease (HD).  My mother developed the disease throughout my adolescence and her disease had huge impact on the entire family.  My brother passed away with HD 10 years ago and my sister died last year after suffering from various and steadily growing...

Dr Anke Arnold-Tugulu brought with her more than 15 years’ experience in the pharmaceutical industry when she joined Azafaros as Head of Regulatory Affairs in early 2022. An expert in the field, Dr Arnold-Tugulu has worked in various disease areas and at all stages of drug development, playing a...

Dimitrios Athanasiou holds a BA in Business Administration and an MBA in Financial Management. He speaks three European languages and has more than 25 years of experience with international business projects, working in various countries in consulting, developing, and reorganizing companies.When...

Miranda Bailey is the Associate Vice President of HEOR & Pricing at Rocket Pharmaceuticals, with over 15 years of experience driving value, access, and health outcomes in rare diseases and gene therapies. She has created and implemented successful global and local market access, HEOR and RWE...

Andy Barrick- CEO Multiple System Atrophy (MSA) Trust UK Andy Barrick has worked in the UK voluntary sector for over 30 years, the last ten with the MSA Trust. Since 2015 the MSA Trust has doubled the number of people they support and have prioritised engagement with our members. Andy has overseen ...

Stefano Benvenuti is Head of Public Affairs and Market Access at Fondazione Telethon (Italy) since 2021. He is vice-chair of the funders constituent committee of the International Rare Diseases Research Consortium (IRDiRC) and he is member of the Italian National Committee on Rare Disease. Within...

Dr. Juliane Bernholz is an experienced CEO, cell biologist, and molecular geneticist with over 30 years of leadership experience in the pharmaceutical industry having worked and lived in the US and multiple countries in Europe. Currently, she serves as the CEO of AM-Pharma, a private clinical stage ...

Corinne Blanchet, MS, serves as Executive Director of Program Delivery for Rare Disease & Pediatrics at Premier Research, bringing over 26 years of comprehensive experience in clinical research leadership. With a strong foundation in project management, Ms. Blanchet oversees the successful...

Lara Bloom is President and CEO of The Ehlers-Danlos Society, where she leads global efforts to raise awareness of rare, chronic, and invisible conditions—specializing in Ehlers-Danlos syndromes (EDS), hypermobility spectrum disorders (HSD), and related conditions. She spearheads international...

Trained as a Pharmacist. Holds an additional master's degree in health economics. Works within market access for 6 years now. Previously worked for Novartis, covering multiple therapeutic areas: neurological-, dermatologic-, respiratory- and cardiac diseases. Working at Gilead Kite for almost 3 ...

I am the Global Programme Manager at Rare Diseases International, where I oversee the Global Network for Rare Diseases and contribute to projects aimed at improving diagnosis, care, and quality of life for people living with a rare disease and their families. I am a nurse by background, with...

Pascal Brokmeier is Head of Engineering at Every Cure, where he co-leads the development of the organization's MATRIX platform, an AI-powered system that analyzes over 75 million drug-disease combinations to identify promising repurposing opportunities for rare and untreatable diseases. Prior...

Paul Broomhead is a seasoned manufacturing and operations leader with over 30 years of cross-industry experience spanning semiconductors, food, polymers, paper, and pharmaceuticals. A champion of smart operations, lean thinking, and continuous innovation, Paul is guided by the Shingo principles and ...

Jonathan D. (Jon ) Campbell, PhD, is the Chief Science Officer (CSO) of the National Pharmacerutical Council (NPC), which sponsors and conducts research on health policy issues related to the development and use of innovative biopharmaceuticals to improve the health of patients. As...

Regulatory affairs professional with 17 years of increasing levels of responsibility with experience spanning multiple components of global regulatory affairs. I have played key roles in the successful registration of innovative and differentiated therapeutic solutions that address unmet medical...

Postdoctoral Researcher at the Health Technology Assessment Area (AETSA) of the Regional Ministry of Health and Families of Andalusia, Seville, Spain. Degree in Biochemistry and a PhD in Pharmacology. Specialization in Evaluation of Medicines and Health Technologies in the Healthcare...

Dr Toby Carter is a Medical Informatics Advisor at Dendrite Clinical Systems, a leading supplier of clinical software for registries across the globe. Dendrite has a considerable and growing portfolio of customers in the Rare Disease sector and we are passionate about improving patient outcomes...

Rosa Castro joined EURORDIS in 2025 as Public Affairs Director. She supports the organisation’s public affairs strategy to advance policies that improve access to treatments for people living with rare diseases across Europe, working to address existing barriers and promote fairer, more coordinated ...

Nathan Chadwick is Senior Director, Therapeutic Strategy Lead, Rare Disease.  With more than a decade of dedicated experience in clinical research, Nathan Chadwick is well-versed in several therapeutic areas, with a specialized focus in neurology and rare disease. At Worldwide, Nathan is at the...

  Anne-Sophie Chalandon works to further enhance Sanofi’s external engagement and policy shaping efforts with rare disease community. She brought a solid experience and demonstrated successes in patient advocacy and policy shaping in the rare disease space. Anne-Sophie is a major in genetic and...

Dr. Yiwei Chen is a pioneering voice in China's rare disease community and the founder of Wonder Sir, the country's leading patient-centered education and innovation hub for rare diseases. For nearly a decade, Dr. Chen has been at the forefront of patient-driven innovation. Through Wonder...

William Cole is a dedicated Life Science Partnership Manager at Kidney Research UK, with a passion for advancing the field of orphan drugs. With a background in life sciences and extensive experience in strategic partnerships, William is committed to driving innovation and collaboration for rare...

Giulia Colombo is a healthcare policy and market access leader with extensive experience at the intersection of payer relations, global advocacy, and public affairs. She currently serves as Global Director of Payer Policy & Partnership at CSL, where she drives strategic initiatives to shape...

Giorgia CRIMI is Patient Advocacy Manager at Fondazione Telethon (Italy). She works on initiatives aimed at strengthening patient advocacy, empowerment and awareness within the rare disease community, fostering collaboration between patients, researchers and institutions. Her work focuses on...

Pam Cusick is an experienced research professional with more than 30 years of expertise in study design, implementation, and analysis. Her background in public health communications and research, coupled with her passion for patient advocacy, dovetail with Rare Patient Voice’s mission and...

Elizabeth de Lange, PhD, is Professor of Predictive Pharmacology at the Division of Systems Pharmacology and Pharmacy of the LACDR (Leiden University). Her group develops predictive (CNS) pharmacokinetic (PK)- pharmacodynamic (PD) relationships in human, in health and disease, mainly using “smart” ...

Business Development Specialist at BRCR Global, a leading network of clinical research sites across the U.S. and Latin America. With a background in business administration and a sharp understanding of industry dynamics, she drives partnerships that bridge scientific innovation with strategic...

Julien Delaye joined EURORDIS in September 2019 as an intern and moved to the position of Public Affairs Assistant on the Rare 2030 project. He then became Public Health Policy Junior Manager and now holds the position of Patient Engagement Manager in HTA. Working closely with the Director of...

Súil is a consultant in in the Life Sciences Practice of CRA, with over seven years of experience supporting global pharmaceutical companies in strategic decision making across all stages of product development. Súil has worked across a range of project types and therapy areas, with emphasis in...

Toon Digneffe is Head of Public Affairs and Partnerships Europe & Canada at Takeda. He has over 20 years’ experience in government affairs, communications, market access and patient advocacy at local, regional and global level.   Mr Digneffe has extensive experience in pharmaceutical...

Danielle is the Global Medical Scientific Advocacy Lead, Rare Disease at Sanofi. She advocates for the understanding and use of rare disease evidence with a range of health care decision makers. She works with multistakeholder consortium across the rare disease ecosystem and leads several patient...

Samuele Doratori, began his career in pharma after a Pharma D, post-doc in Immuno-Oncology, taking on roles of increasing responsibility across Medical Affairs, Marketing, Sales, and Global Product Strategy at Menarini and Servier. With experience spanning cardiometabolic, oncology, respiratory and ...

Jessie Dubief is a dedicated and experienced Social Research Director at EURORDIS-Rare Diseases Europe, a unique non-profit alliance of over 1,000 rare disease patient organisations. With a strong background in social sciences and a passion for patient advocacy, Jessie plays a pivotal role in...

Mr. Elam has been founding, managing and advising healthcare and technology companies for more than 30 years.  Prior to founding Rezolute, Mr. Elam held various senior leadership positions in the industry including Senior Vice President at Nektar Therapeutics (NASDAQ: NKTR) where he ran the...

Elsie Evans is the Ambassador Programme Manager at the FH Europe Foundation. A EUPATI Fellow with a background in education and lived experience of HoFH, she works to strengthen patient centricity and supports Ambassadors in engaging with stakeholders across healthcare, policy, and research.

Elise has five years of consultancy experience as a health economist and in HTA strategy in various therapeutic areas, including rare pediatric diseases. She has extensive expertise in economic modelling and preparing comprehensive reimbursement  submission dossiers for HTA bodies across the...

Karen Facey PhD CStat worked in senior statistical roles in the pharmaceutical industry and medicines regulation, before becoming CEO of the first national health technology asssessment (HTA) agency in Scotland in 2000. For the past 20 years Karen has had a portfolio career working internationally...

Rita Francisco has a background in research, holding a BSc in Genetics and Biotechnology, an MSc in Molecular Genetics and a PhD in Biology. During her PhD, she strived to develop and implement a people-centric framework to drive biological and clinical research on a family of rare diseases known...

Dr. Lydia Frick is Director Market Access at Kintiga, a leading strategic consultancy with deep local expertise and global reach, combining energy and expertise to create seamless, impactful solutions that transform patient access across Europe. As a psychologist, natural scientist, and market...

Thom Frielink is a seasoned professional in the field of orphan drugs, currently serving as a Senior Associate at Curie Capital BV. With a strong background in healthcare and pharmaceuticals, Thom brings a wealth of experience in strategy development, advocacy, and partnership building within the...

Maciej Gajewski is Vice President and Head of Global Corporate Affairs, Rare Diseases at Ipsen. He is a seasoned corporate affairs leader with over 20 years of experience spanning the global healthcare and pharmaceutical sectors, where he has held executive roles focused public affairs, policy, and ...

PharmD, PhD in cellular biochemistry and pharmacology, post-graduate degree master in Clinical Research of Medicines. My main areas of expertise are: ethics and regulatory, informed consent and assent, health data processing for research purposes, European medicine regulatory procedures, R&D...

Dr. Erwan Gicquel, Government Affairs Lead Europe, Miltenyi Biomedicine GmbH Located in Brussels, Erwan coordinates Miltenyi Biomedicine’s public policy and government affairs activities in Europe. His primary responsibilities consist of monitoring public health, research and market access...

Diego is the Executive Director of the Federación Colombiana de Enfermedades Raras (FECOER), an umbrella organisation bringing together 52 patient groups to represent the voice of the rare disease community in Colombia. Diagnosed with pulmonary hypertension over 30 years ago, Diego used his...

Josie is dedicated to advancing the development and accessibility of treatments for rare diseases. She is a recognised expert in Health Technology Assessment (HTA), market access, and patient advocacy for rare diseases, bringing over 15 years of experience to the field.Josie is known for her...

Wim Goettsch, PhD, is Special Advisor in Health Technology Assessment (HTA) at the Dutch National Health Care Institute (ZIN). In his position at ZIN, he is a Member of the EU HTAR Coordination Group and its subgroup on Joint Scientific Consultation (JSC). Between 2010 and 2013, he was the Deputy...

From 2010 to 2018, served as leader of the Polish Association for Patients with Primary Immunodeficiencies 'Immunoprotect’ and was a member of the executive board of the international umbrella association, the International Patient Organization for Primary Immunodeficiencies (IPOPI). For his...

She leads Global Gov. Affairs & Patient Advocacy at AOP Health, the European pioneer for rare diseases and critical care. She has a background in health economics, corporate development, and EU advocacy.

Alicia Granados is a highly accomplished leader in the field of rare diseases, currently serving as the Head of Global Rare Diseases Medical Scientific Advocacy at Sanofi, where previously she headed its global HTA strategy .With almost 15 years of experience in the pharmaceutical industry, Alicia...

Researcher at the Health Technology Assessment Area (AETSA) of the Regional Ministry of Health and Families of Andalusia, Seville, Spain. Degree in Physiotherapy and master´s degree in Motor Neurocontrol. Currently working a PhD in Clinical Procedures, Interventions and Health Outcomes at the...

Chris has 25+ years of life science experience with executive roles in the pharmaceutical, Biotech, Medtech and Health-tech industries. Chris has led organisations and functions at global, regional and country level and has a wealth of therapeutic experience including working extensively in rare...

Hannah is a Project Manager at Beacon, a UK-based rare disease charity that is building a united rare disease community with patient groups at its heart. She works alongside the Projects Team to manage and deliver patient group training, guided programmes, and community projects. Through this...

Matthias Heck is Senior Advisor at the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE). He focuses on partnership building and provides strategic guidance on legislative and policy matters. A German-qualified lawyer with some 20 years of experience in life sciences, Matthias has...

Niklas Hedberg is the Chief Pharmacist at the Swedish national governmental authority, the Dental and Pharmaceuticals Benefits Agency (TLV). Niklas is the Co-Chair of the HTAR Coordination Group with special expertise in pharmaceutical products. He was the Chair of the Consortium Executive Board...

With over 10 years of experience in the fields of health economics and outcomes research (HEOR) and Real-World Evidence (RWE) and 5 years in the clinical field, I am passionate about generating and communicating evidence to support the value proposition of innovative therapies for rare diseases. As ...

Carla Hollak, internist for inherited metabolic disorders at Amsterdam UMC, is UvA professor of 'Medicine and Society'. Carla has been the founder of the department of metabolism in adults for the past 20 years and was at the basis of the expertise center for inherited metabolic disorders...

Sebastian Honoré is the Founder and CEO of Cure Lowe Foundation, a non-profit organization focused on accelerating research and treatment development for rare diseases, with a particular emphasis on Lowe Syndrome. Sebastian founded the organization after his son Walther was diagnosed with Lowe...

Patrick Hopkinson is a Global Market Access/ HEOR expert with over 25 years experience. He is an independent consultant, founder and managing director of PHTA Consulting.  He is a senior leader in the life sciences industry with extensive expertise gained from working across multiple functions in...

Julian is the co-founder and leader of PharmaKrysto, a company focused on developing new treatments for people living with rare kidney disease. He has many years of clinical development, translational science and early stage company formation and funding.

Dr. Raymond A. Huml is the Vice President of Rare Disease Strategy at Sciensus, with over 30 years in the healthcare and biopharmaceutical industries. A leader in rare disease research and global due diligence, he has driven nearly $3 billion in capital commitments. A prolific author and...

Julian Isla is a software engineer by training and works as a Resource Manager for the Artificial Intelligence group at Microsoft. He has 29 years of experience working for international IT companies, helping customers with digital transformation and leveraging the capacity of artificial...

William Jang is a biotechnology executive specializing in the development and global commercialization of microbiome-based therapeutics. He currently serves as Head of Global Business Development at BioMe, Inc., where he drives strategic partnerships, investment initiatives, and regulatory...

UCB New Product Planning Lead for early gene therapy and neurodegeneration portfolio. Past experience in Access/Pricing roles (10 years) from the Cell and Gene Therapy Catapult and ICON Plc. consulting. Author of 10 peer-reviewed articles on access-reated topics in the cell and gene therapy space. 

Dr. Ron Jortner is a scientist and entrepreneur based in Cambridge, UK. He is Founder and CEO of Aspire Biosciences, which finds new treatments for rare diseases using a novel methodology based on engaging patient groups in the R&D process. Ron has been serving the rare disease community for...

Bojan is an innovative, creative and insightful medical expert with a 17+ years of professional experience and results in the fields of Pharma Industry and Clinical Research.   In his current position as Medical Director for Allucent, he is responsible for ensuring the medical/clinical integrity...

Assistant Director, Inserm – Thematic Institute of Genetics, Genomics and Bioinformatics (IT GGB) Coordinator, European Rare Diseases Research Alliance (ERDERA) Dr Daria Julkowska is a leading architect of the European rare diseases research ecosystem. As Coordinator of ERDERA – the European Rare ...

Manohar Katakam, Ph.D. is a biotech and pharmaceutical leader with over two decades of experience building companies, advancing novel therapies, and delivering measurable shareholder value. His entrepreneurial leadership includes founding and scaling multiple life sciences ventures. As President...

Anna Kole is a public health professional with over 20 years of international experience in health education, communication, fundraising, advocacy and policy making for people living with rare disease and other vulnerable populations. Since 2022 she has led Global Patient Engagement for Myasthenia ...

Stelios Kympouropoulos is a psychiatrist, a politician and a social activist. He has been a Member of the European Parliament and a Member of the Group of the European People's Party since 2019. He is a Member of the Committee on Employment and Social Affairs, the Delegation for relations with...

Christina Kyriakopoulou works as a Policy Officer at the Directorate General for Research and Innovation of the European Commission, Directorate People: Health and Society and the unit Health Innovations and Ecosystems. She holds a PhD in biochemistry and she held postdoctoral positions in...

Dr Samantha Lawes is the Director of Research Development & Partnerships at Kidney Research UK, where she provides strategic leadership across the charity’s research growth areas. She oversees scientific portfolio management, research impact, and the development of key fields including...

Jen Lee is a Principal Consultant at HEOR Ltd with over 13 years’ experience managing global and European HEOR and market access programs for rare disease technologies. She has led UK NICE submissions, overseen complex multi-country reimbursement strategies, and brings expertise in evidence...

Dr. Mimi Lee serves as Chief Precision Medicine Officer at GeneDx, where she leads the company’s efforts to make precision medicine the standard of care. A physician–scientist and healthcare innovator, Dr. Lee is driving GeneDx’s vision to harness the power of genomics to predict, prevent, and...

Dr. Fabrice Lejeune is the Scientific Advisor & co-Founder of Genvade Therapeutics. He is the author and inventor of the patents for the lead candidate GV-01 and the drug screening platform. He has been a scientific leader in the search for nonsense mutation correctors for over 20 years. He is...

Vincent Lévêque is a biotech executive with nearly two decades of experience in rare diseases, advanced therapies, and gene therapy commercialization. He has built and led cross-functional organisations across Europe, the Middle East, and Eurasia, with a proven track record of launching...

Dan's eldest daughter Amelie was diagnosed with Tay-Sachs in 2011 at 15 months of age and upon finding that there was no dedicated advocacy group providing support for families affected by this disease in the UK he set set-up the Cure & Action for Tay-Sachs (CATS) Foundation with his wife...

Kieron joined the Clinigen Group in 2014 and is responsible for delivering consulting services to Biotech and Pharmaceutical companies. Kieron has a vast amount of experience in planning and setting up Pre-Approval/Managed/Expanded Access, International Pharmacy and Named Patient Supply...

Chyrel Lichaa is a dedicated advocate for individuals living with Homozygous Familial Hypercholesterolaemia (HoFH). As a HoFH Patient Ambassador, she actively works to raise awareness about this rare genetic disorder, which causes extremely high levels of LDL cholesterol from birth. Her personal...

Caroline Ling has over 20 years of experience with market access and health economic consultancies and is able to deliver a broad spectrum of strategic and tactical market access projects for both global and local clients, including systematic and pragmatic literature reviews, global value...

Gisela Linthorst joined Azafaros at the start of 2022 and brought with her more than 20 years’ experience in the pharmaceutical industry, with a focus on rare diseases. In 2006, as Manager of Patient Care at Genzyme in the Netherlands, she was responsible for setting up the first homecare projects...

Name: Liu Mi Title and position: Distinguished Professor, Institute: Department of Pharmaceutics, College of Pharmaceutical Sciences, Soochow University CSO,  Beijing Darwin Biotech Co., LTD   Email: mi.liu@suda.edu.cn   Research Areas: (1) New drugs based on proteins derived from stem...

Veronica Lopez Gousset, MPH has over 10 years of experience driving multi-stakeholder initiatives toward patient-centered healthcare change. She works with the patient community to better voice their needs and challenges, with health technology assessment bodies as the gateway to services and...

Dr. Joshua Lorenz-Guertin is Vice President of Research and Development at Sharp Therapeutics. He holds a Ph.D. in Molecular Pharmacology from the University of Pittsburgh, where he specialized in GABAA receptor neuropharmacology. During this time, he developed an innovative dual-fluorescence...

Flaminia currently serves as the Chief Operating Officer of the Orphanet-AISBL, and as Senior Policy Advisor to different organisations on an independent basis. Flaminia has a broad range of experiences from the patient community, the private sector, as well as the EU institutions. Indeed, Flaminia ...

Dr. Maltecca's main research interest is in inherited cerebellar ataxias of primary or secondary mitochondrial origin. She has a background in human genetics with specialized training and expertise in inherited cerebellar ataxias, as well as a solid background in cerebellar physiology and...

Aurora Mateos is Founder and General Director of Menkes International (MIa), a charity focused on fighting the rare disease called Menkes. She is an international lawyer (she has been Consultant to United Nations for more than fiftheen years) on environmental law, specially law of the sea, as well...

Miriam Meeuwissen, MSc is a global biopharmaceutical supply chain leader with more than 20 years of experience in accelerating that breakthrough medicines reach patients around the world. She’s held senior roles at Biogen, MSD/Merck, and Italfarmaco, and leads her company, Launch & Lifecycle...

Michelle L. Mellion, MD earned her undergraduate degree with honors in molecular biology from Colgate University and her medical degree from Wake Forest University School of Medicine. She completed her internship in internal medicine, residency in neurology, and fellowship in clinical...

Dr Michael Merchant is a Scientific Adviser in the Data and Analytics team at NICE. He joined NICE in 2023, to work on Real World Evidence for Clinical Prediction Models and AI predictive technologies for diagnosis and prognosis. This work is in review and will form part of the NICE Real World...

anis Mimouni joined Critical Path Institute (C-Path) on 1 September 2025 as Associate Director for Regulatory Science. In this role, he works closely with the European Office leadership and the global C-Path teams to coordinate EU-funded projects, including, but not limited to, those in the field...

Warnyta Minnaard (1989) is one of the founders of ‘Missie Tumor Onbekend’, the Dutch advocacy and patient organisation for patients and families that deal with cancer of unknown primary (CUP). CUP is the 4th most common metastatic cancer in the Netherlands. After her partner Hederik passed away...

CV: JAN A. MOL, PhD (www.uu.nl/staff/jamol) UNIVERSITY OF UTRECHT, LYMPHOMA COALITION Jan Mol is a retired cell biologist of Utrecht University and patient advocate for people with various forms of blood cancer. Jan Mol graduated from Wageningen University (Molecular Sciences) and did his PhD...

Maria-Cruz Morillo, MS Pharmacy, has a strong background in drug development with 29 years of experience in project management and strategy and IMP supply in both biotech and CROs. Her strategic mindset and demonstrated ability to design and implement effective study protocols has contributed...

“Josephine is the senior policy officer at Cancer Patients Europe (CPE). She joined CPE in 2024 after accumulating more than 6 years of professional experience in various organisations from nonprofits to private sector organisations where she developed expertise in EU health policy, patient...

Sophie Muir is the Founder and Chair of Timothy Syndrome Alliance (TSA), leading efforts to improve understanding, diagnosis and care for individuals with CACNA1C-Related Disorders including Timothy Syndrome: a group of ultra-rare, multisystem conditions that can be life-limiting and are often...

My name is Cristóbal Muñoz Sánchez, and I hold an MBA in International Management and a Master's degree in International Communication. I am currently working as Project Coordinator at RDIG HTAi and Assistant Project Manager at AETSA-FPS, with a specialization in managing R&D projects in...

Rima Nabbout is Professor of Paediatric Neurology at Université de Paris cité and Director of the French reference centre for rare Epilepsies at Necker Enfants Malades, APHP, Paris, France.She is elected president of the French society for pediatric Neurology.She is coordinator of RHU...

Alexander Natz is the Secretary General of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) in Brussels and advises innovative pharmaceutical and biotech companies, including start-ups, in regulatory and pricing & reimbursement matters from the EU law and German law...

Dan O'Connor is medically (Leicester) and PhD (Imperial) qualified and currently the Director Regulatory and Early Access Policy at The Association of the British Pharmaceutical Industry (ABPI). He joined the ABPI from the Medicines and Healthcare products Regulatory Agency in 2023 where he was ...

Paula had a 26-year career with The Muscular Dystrophy Association (MDA) as Director, Family Support and Clinical Care, supporting patients living with rare neuromuscular disease.   Her clinical experience through  MDA's national multi-disciplinary care center network included  helping patients ...

Monica Otto is an Associate Professor of Practice in the 'Government, Health and Not for Profit' Area at SDA Bocconi School of Management. At SDA Bocconi, she was the Director of the Master in Public Management (2010-2013) and of the Executive Master of Management in International...

Distinguished Investigator and Chair of the Scientific Advisory Committee of the Únicas SJD Center at the Sant Joan de Déu Children's Hospital in Barcelona, ​​where he served as Head of the Genetic Medicine Service and Director of the Pediatric Institute for Rare Diseases. He is a researcher in ...

Clive Phillips is the founder of Making the Most of Now, an advocacy initiative inspired both by his rare condition, Multifocal Motor Neuropathy (MMN), and by his belief that resilience and joy can be found even in the face of uncertainty. With a professional background in management and...

Enrico Piccinini has been Senior Vice President, EU & International, Rare Diseases, Chiesi Group, since November 2019, where he leads a team that works to make treatments available for rare and ultra-rare diseases. He is a member of various biotech, R&D and orphan drug groups and...

Giulia Pierini is a rare disease specialist with deep expertise in patient engagement and advocacy. With over five years dedicated to the rare disease field and more than 50 successful patient-focused projects across multiple rare and complex conditions, she has collaborated with global...

Anke Pisters-van Roy, MD, CZ is a dedicated and passionate healthcare professional with a strong background in orphan drug development and advocacy. With a medical degree and expertise in the field, Anke has made significant contributions to the orphan drug policy through her work at CZ, a leading...

Tom Pulles, MD, is a dedicated and accomplished professional with extensive experience in the pharmaceutical industry. Currently serving as the Vice President and Head of Medical Affairs & Patient Advocacy for Europe at Acadia Pharmaceuticals Inc., Dr. Pulles plays a pivotal role in shaping the ...

Vinciane Quoidbach is a Public Health and Policy Research Project Manager at the European Brain Council, specializing in brain health impact assessments, care and treatment pathway analysis (with a focus on digital health), and evidence-based policymaking. She is currently pursuing an MSc in...

Carelle Reparon-Schuijt is a dedicated and experienced medical advisor for hospital medicines at health insurance company Zilveren Kruis. With a strong background in healthcare and a passion for improving patient outcomes, Carelle brings a unique perspective to the orphan drug industry. Her...

Dr Sumira Riaz (DPsych), Chartered Health Psychologist and Patient Engagement Consultant, Unboxed PsychologyDr Sumira Riaz is a Registered Health Psychologist, Cognitive Behavioural Therapist and Founder of Unboxed Psychology, a behaviour change, patient engagement consultancy. Sumira has 18 years...

Currently the CEO and CSO of Med-Life Discoveries with over 20 years of experience in the biotech sector. My primary objective at the meeting is to seek investment opportunities and/or potential co-development or licensing partners for our rare disease program. 

Kit Roes is Professor of Biostatistics at Radboud University Medical Center Nijmegen (Netherlands). He is chair of the Methodology Working Party of the European Medicines Agency, and senior expert at the Dutch Medicines Evaluation Board. His research focus is design and analysis of clinical trials...

Kent Rogers has been a senior executive across the healthcare industry for over 30 years, focusing on product commercialization, supply chain logistics, pricing strategies, reimbursement, market access, and alliance management. He spent more than two decades in the pharmaceutical industry in...

Stefano Romanelli is the Government Affairs Associate Director of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE). Stefano supports EUCOPE’s policy work on the EU General Pharmaceutical Legislation, the Critical Medicines Act, the Biotech Act and related pharmaceutical...

Kacper is a founder and long-time president of Poland-based Fundacja SMA since 2013. Kacper is currently an Executive Committee Member European Alliance of Neuromuscular Disorders Association (EAMDA) and a founding member of the European Coalition for Newborn Screening (ECNS). He helps to steer an...

Christopher Rudolf is a seasoned technology entrepreneur and business advisor with over 30 years of experience, specialising in data science, AI, and medical informatics. As the founder and CEO of Volv Global SA, he leads the company's mission to transform healthcare through advanced machine...

After leaving the Mayo Clinic, from 1988 to 2007, he held academic, clinical and administrative leadership positions as Chief of Cardiac Surgery, Pediatric Cardiac Surgery and Heart Transplantation  at several Academic Hospitals in the USA, including Temple University, St Christopher’s...

Maurizio Scarpa, MD PhD, paediatrician, is the Director of the Regional Coordinating Centre for Rare Diseases at the University Hospital of Udine, Italy. He is Professor of Paediatrics at the Dept. for the Woman and Child Health, University of Padova, Italy, and the Co-Founder of the Brains For...

Dr Joerg Schuettrumpf is the Chief Scientific Innovation Officer of Grifols, where he drives the company’s strategic focus a provider of innovative plasma medicines and other biopharmaceuticals. Dr Schuettrumpf is a physician-scientist specialised in Transfusion Medicine and Hemostaseology with...

Ruchi Sharma is the CEO of Stemnovate Limited, a biotechnology company focusing on stem cell research and drug discovery. With a PhD from the University of Edinburgh and research experience at the University of Cambridge, she founded Stemnovate to develop solutions for rare diseases and...

Lydia Shotton is an accomplished professional with extensive experience in EU Government Affairs within the healthcare and life sciences sector. Currently serving as the Associate Director at the Alliance for Regenerative Medicine, Lydia plays a pivotal role in shaping policy and advocacy...

Currently serving as the Vice President of Market Access EU & International and Global HEOR in the Global Rare Disease Unit of Chiesi Group, Diana Sinkevich oversees market access and value strategies, health technology assessment, and evidence generation across Europe and international markets ...

Tara Smith is the Executive Vice President, Innovative Therapies at Med-Life Discoveries LP, a North American company developing a first-in-class plasmalogen metabolic replacement treatment. Their current focus is the ultra-rare disease Rhizomelic chondrodysplasia punctata (RCDP). Tara has over 15...

Rachel Smith is an ultra-rare disease subject matter expert with over 10 years of experience in clinical research, sponsor, and CRO roles across APAC, Europe, and North America. She oversees 30+ multimillion-dollar orphan disease programs funded by both sponsors and academic institutions. Rachel...

Dr. Trevor Smith is a molecular and cellular immunologist, with over 20 years of research experience in the fields of infectious disease, allergy, autoimmunity and oncology. Trevor has published over 40 articles across these disciplines, with 1000+ citations. Currently, Dr. Smith heads a...

Adlane Soudani is Global Asset Lead at IPSEN. Adlane has 20+ years of international leadership in the pharmaceutical industry with extensive experience in the rare disease space. Over the years, Adlane has led strategic initiatives across multiple therapeutic areas, global launches, asset...

Alberta Spreafico is a global health innovation pioneer with 15 years of experience advancing digital health policy and systemic innovation strategies. She has co-founded and led international development programmes integrating digital technologies across health systems in both high- and low-income ...

Lotte Steuten is Currently the Deputy Chief Executive at the Office of Health Economics, and an Honorary Professor of Health Economics at City St George's, University of London, UK. Lotte brings a wealth of experience in driving strategic initiatives and building the evidence base for...

CEO of Trialize. Chairman of HL7 UA. 

Pernille Storm is a leading expert in global pricing and patient access, specializing in rare and neuroscience-driven innovation. With a background as Vice President for Global Pricing & Patient Access at Lundbeck, she has guided commercial strategy across the full lifecycle. from early R&D ...

Dr. Violeta Stoyanova-Beninska is a medical doctor with a PhD in molecular genetics and a Master of public health degree. As a regulator and scientist Violeta has dedicated her career to clinical work, research in genetics and regulation of medicines and in particular to therapies for rare and...

Aleš Štrancar has been the Managing Director of Sartorius BIA Separations since its founding in 1998. He is a key inventor of the CIM Convective Interaction Media® monolithic columns technology and has co-invented numerous analytical methods for pDNA, mRNA, AAV, Adeno, and other viruses. Aleš has...

Gerdi Strydom is the Managing Director at Valid Insight, a consultancy that specialises in global market access strategy, payer evidence, and value communication. With over 20 years of leadership experience in the biopharmaceutical industry, consulting, and working as a payer, she offers a...

Karina is a freelance journalist from Germany who has lived in San Francisco and later Copenhagen for a total of nine years. She gained scientific knowledge through studying laboratory technology and working in research for several years. Due to a chronic illness and invisible disability, she found ...

Kwee Lan Tan is the Head of the Regional Regulatory Lead Emerging Markets team at Boehringer Ingelheim International GmbH. In this role, she leads a team of Regional Regulatory Leads to accelerate and ensure access to life-changing medicines for patients. Before her international role in...

Jose Terencio is Senior Vice President of Discovery at Grifols, leading the internal research teams as well as the external Innovation arm of Grifols named GIANT (Grifols Innovation And New Technologies) and also CEO of Araclon Biotech. With more than 25yrs+ of experience in the Pharma and Biotech...

Nitza Thomasson: recognized neuroscience expert (PhD in Neuroscience, Sorbonne University, Paris and Post doc , Northwestern University, Chigago) with leadership experience across pharma and biotech (Sanofi, Fovea, Tissium). An entrepreneur at heart, she co-founded pioneering companies in the field ...

Andrew is the owner and lead consultant of Regnitio, a company he founded in October 2025 after a long regulatory career. He was  a statistician at the EMA for 11 years, and previously spent 7 years at MHRA, initially as a statistical assessor and subsequently Head of Epidemiology. He has a strong...

Costello Medical is an independent consultancy that provides scientific support in the analysis, interpretation and communication of clinical and health economic data. As a Principal Consultant in the Rare Diseases Team, Eleanor works with a patient-focused, multidisciplinary team that leverages...

Giuseppe Turchetti (PhD, Fulbright Scholar) is Full Professor of Economics and Management of Innovation and Healthcare the Scuola Superiore Sant’Anna in Pisa (ITALY), where is also Vice-Rector for Relationships with Companies and Institutions. His main research interests are in the fields of the:...

Sophie H. Turner is the founder of Empowered By Us, a patient-led rare disease inclusion initiative working across Europe to embed lived experience, equity, and co-creation into access, policy, and system design. Based in the Netherlands, she collaborates with advocacy networks, research...

Janneke van der Kamp is Chief Executive Officer of Norgine, a mid-sized EU-based pharmaceutical company with 1,500 employees and approximately $600 million in annual sales. Innovation drives Norgine’s mission to deliver medicines that change lives. The company targets unmet medical needs across a...

Prof. Bart van de Warrenburg is a neurologist, focusing on rare movement disorders, which are often genetic. He is founder and lead of the Radboudumc expert center for rare and genetic movement disorders, which is also part of the European Reference Network for Rare Neurological...

Silvia van der Flier is a dedicated and experienced Medical Advisor at VGZ, a leading healthcare insurance provider. With a background in medicine and a passion for improving patient outcomes, Silvia plays a crucial role in shaping healthcare policies and strategies within the orphan drug...

I have 20+ years’ global experience in clinical development and operations, uniting data science with pharma and research. I drive cross-functional collaboration to advance innovative treatments. Working for Volv Global I connect real world health challenges with AI/data science...

Benjamen Varsano is a seasoned professional in the pharmaceutical industry with a strong focus on operational strategy. Currently serving as the Director of Operational Strategy at Ergomed, Benjamen brings a wealth of experience and expertise to the field of orphan drugs. With a proven track record ...

Dr Vikkula, MD PhD (1992-93 Helsinki Finland), a professor of genetics, became interested in vascular anomalies at Harvard Medical School 1993-97, and unravelled key concepts in their pathophysiology: genes, second-hits and decisively somatic mutations (2009). He is at the forefront of developing...

Delphine has over 15 years of experience working in Global Regulatory Affairs. At Clinigen, she provides regulatory expertise and leadership to clients across a range of projects, including regulatory applications in Europe, the US and at EU National Competent Authorities and advises clients on...

Miriam Wagner Long has been a dedicated fundraising professional for nonprofit organizations since 2005. She has worked extensively in the U.S., focusing on sustainable funding strategies especially for healthcare related organizations. Since returning to Germany in 2011, Miriam has continued to...

Katie Waller is Head of Patient Programmes at The Lily Foundation, the UK's leading mitochondrial disease patient organisation. Before joining the Lily team, she trained as a paediatric nurse and then worked as a research nurse, working on early phase clinical trials with patients with...

Dr Mary Wang is Patient Engagement Director at Genespire, ensuring that the perspectives of people living with rare diseases inform the development of advanced therapies. She previously served as Global Programme Director at Rare Diseases International (RDI), leading initiatives on...

Michael Wilbur is an independent consultant in patient advocacy and corporate affairs. Former roles include: •Chief Operating Officer at EURORDIS-Rare Diseases Europe (6 years) •Head of Planning and Delivery –NHS England / NHS Improvement  (10 years) •Associate Director of Research and Online...

Kate Witkowska is a Strategic Partnerships Director with a passion for translating genomic science into real-world impact. She began her journey in Canada, earning a PhD in Molecular Physiology from the University of Alberta, where she uncovered novel pathways regulating urate balance—work powered...

Sorrel Wolowacz has more than 2 decades of experience in health economics consulting and 3 decades of experience in the health care industry, including health outcomes, clinical research, and product development. Her research experience is in the areas of economic modelling, real-world...

Wieteke Wouters is program director and deputy director at hollandbio, the Dutch biotechnology industry association. Hollandbio aims to make life better with biotech - for people, animals and our planet. Under the motto “challenge the status quo”, hollandbio concentrates its efforts on removing the ...

Kaja, a dedicated MPharm professional (PharmD/US) with a solid background in genomics and entrepreneurship, is passionate about advancing healthcare through innovative approaches. Specializing in genomic medicine, rare genetic disorders, and AI in healthcare, she focuses on early diagnosis of...

Chris Zhou is Vice President of Partnerships at ACA Pharma, where he has led strategic alliances since joining in 2024. He previously founded and managed Hauseit, a tech-enabled real estate brokerage specializing in commission rebates and reduced commission listings. Earlier in his career, he...

  Martine Zimmermann joined Ipsen in January 2023 as Senior Vice President and Head of Regulatory & Quality Affairs, based in Boulogne (France) and since January 2024, SVP, Head of Global Regulatory Affairs. Previously she was Senior Vice President and Head of Regulatory & Quality...