2026 Confirmed Speakers

Dr Safwaan Adam is Chief Medical and Safety Officer (Fractional) at OpalMedica, providing strategic medical and safety leadership to support the development of innovative products. He combines a strong clinical background and academic record with expertise in clinical and medical strategy, patient...

Dariusz Adamczewski, MD, is Managing Director of Children's Tumor Foundation Europe (CTF Europe), the Brussels-based patient foundation dedicated to neurofibromatosis and schwannomatosis (NF). CTF Europe combines patient advocacy with an active role in drug development and research. He leads...

I'm Hicham, 49, diagnosed with FSHD in 2010. I am a borad member of a french Patient Advocacy Organisation, AMIS FSH, and also member of FSHD Europe.

Omar Ali BSc(Hons)Pharm DipClinPharm MRCPharm ACPPFormer Government Adviser NICEHead of Payers, Verpora  As Head of Payers at Verpora, Omar works with pharmaceutical and life sciences organisations to shape access strategy, value communication and pricing negotiations across global...

Michael Andreou is a market access & pricing leader with over 25 years access and commercial strategy experience in pharma & biotech. He has broad therapeutic area experience, especially in rare diseases, and is currently global Value & Access Lead in Rare Diseases at Biogen focusing on ...

Anjan founded Raya Therapeutic in 2019. Raya is focused on developing multiple drugs to treat ALS and potentially other neurodegenerative diseases. Raya is currently focused on starting a robust P2 trial across multiple clinical centres in the UK in the fall of 2026. In addition, the company plans...

Professor Alexis Arzimanoglou is, since 2019, the elected Coordinator of the European Reference Network for Rare and Complex Epilepsies, ERN EpiCARE. Since October 2023, he is also the Director of the Epilepsy Program at the Barcelona Children's Hospital San Juan de Dios, Universitat de...

Dr. Andrea Ballerini is a physician and clinical pharmacologist whose career spans translational research, healthcare investing, and pharmaceutical business development. After conducting research in oncology, drug delivery, and regenerative medicine in academic and non-profit institutions in Italy...

Fabienne Bartoli – Director General, Snitem Fabienne Bartoli is Director General of Snitem, the French trade association representing the medical technology industry. She has extensive experience in healthcare policy, regulation, and institutional leadership, both in France and internationally. ...

Jana Benesh, PhD, is a rare disease therapeutic expert and neuroscientist within ICON's Centre for Rare Diseases, specializing in the development of therapies for rare and ultra rare neurological and neuromuscular disorders. With more than 15 years of experience spanning neuroscience, clinical...

Mikel Berdud is an economist at the Office of Health Economics (OHE), specialising in pharmaceutical R&D incentives, pricing and reimbursement, and the social value of innovation. He holds a PhD in the economics of organisations and behavioural economics. He is an honorary visiting professor at ...

Tanya Binette is a rare disease strategy expert, bringing patient-centric and rare-specific strategies to study teams and customers, supporting from preclinical through commercialization and across therapeutic areas. She has more than 17 years of strategy development and project management...

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...

Adam Bloomfield MD FAAP is Vice President, Internal Medicine, Rare Disease and Pediatrics, at Premier Research, providing medical monitoring oversight, therapeutic knowledge to Premier Research study programs and supporting business development and consulting activities in the fields of Internal...

Valentina Bottarelli joined EURORDIS – Rare Diseases Europe in 2007. She is now Head of Policy and Public Affairs and leads the organisation’s policy and advocacy strategy, representing it before the EU institutions and stakeholders. She oversees key cross-cutting files, especially the advocacy for ...

Luísa Bouwman is a Regulatory Affairs Professional with more than 10 years of experience in the pharmaceutical industry and a PhD Candidate in Regulatory Science at the University of Lisbon (Portugal). Her research focuses on evidence generation for orphan medicines in the European Union, providing ...

Sara Brambilla is the Global Programme Manager at Rare Diseases International (RDI), where she leads the development of the Global Network for Rare Diseases—an initiative aimed at strengthening international collaboration, connecting specialized expertise, and fostering structured partnerships...

SGS CDMO Solutions is a trusted UK pharmaceutical development and manufacturing partner, helping companies progress products from early formulation and process development through clinical trial supply, scale-up, technology transfer and commercial manufacturing. With experience in small molecule...

Ria de Haas, PhD, is an experienced patient advocate in the field of neuromuscular diseases and currently serves as CEO of FSHD Europe, a non-profit umbrella organisation representing national facioscapulohumeral muscular dystrophy (FSHD) patient organisations across Europe. As a EUPATI...

Search and evaluation leader with 20+ years in commercial and business development roles across rare disease, specialty pharma and biotech investing decisions. Recent deal experience includes closing four transactions in 2024–25: three European in-licensing deals representing over €1 billion in...

Ben Doak MSc DIC, has built a significant career working in England’s National Health Service (NHS) at a national level for more than 20 years. Most recently, he was the Head of Innovative Treatments, part of NHS England’s National Specialised Commissioning Directorate. In this role, Ben’s primary...

As Director of Strategic Partnerships and Clinical Solutions, Pamela is responsible for shaping the RWD handling strategy for Rivia's clinical trial data platform. Prior to joining Rivia, Pamela was a Director at Biogen where she was responsible for developing and leading Biogen's RWD / RWE ...

Jacob Epstein is an Associate Engagement Manager at Prescient, leading Value, Pricing & Access engagements for global pharmaceutical companies, with a particular focus on rare diseases and oncology. He has extensive experience spanning market access strategy and health policy, helping clients...

Karen Facey worked as a senior statistician in Pharma and medicines regulation before moving to Scotland in 2000 to setup their first national HTA agency. Since 2003 she has been an independent consultant working with all stakeholders on matters relating to HTA in Europe and Canada, with special...

Giada Farinelli is Regulatory Affairs Manager at Fondazione Telethon, a leading Italian biomedical charity dedicated to advancing research and developing treatments for rare genetic diseases. In her role, she supports the regulatory strategy and progression of innovative therapies, helping...

Alessandra Ferlini, born in Bologna, is a medical geneticists and neurologist, associate professor in medical genetics, and head of the Medical Genetics Unit at the University of Ferrara (Italy). She is also Honorary visiting professor at the University College London from 2015. She achieved her...

Dr Sanja Fitzgerald is a market access and HTA advisor supporting biotechnology and pharmaceutical companies on pricing, access, evidence strategy and EU HTA/JCA. Her work focuses on connecting clinical development and evidence-generation decisions with HTA outcomes, patient access and commercial...

Pedro Franco has an extensive educational background, holding a degree in Pharmaceutical Science (PharmD) and a Master's in Organic Chemistry (M.Sc) from the University of Lisbon in collaboration with Imperial College in London, a PhD in Orphan drugs and Rare diseases from the University of...

Nicolas Garcelon has an engineer degree in agronomy (2000) and a PhD in public health medical informatics(2017). He has been working in the medical field for 25 years. From 2001 to 2012, he worked in the medical information department of Rennes University Hospital. Since 2012, he directs the data...

Maja Gasparic is currently Global Medical Director, Rare Hematology at Sanofi, with more than 20 years of experience in the pharmaceutical industry across therapeutic areas including rare diseases and oncology. Throughout her career, she has worked across diverse areas of medical affairs, inclduing ...

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

Josie Godfrey is an independent strategic adviser on access, policy and health technology assessment for rare and complex conditions, with more than 20 years’ experience across the NHS, the Department of Health and Social Care, NICE and the global pharmaceutical industry. She established NICE’s...

Joining PTC in 2014, Adrian was one of the first employees outside the USA. He built and led the commercial organization in Europe, Asia/Pacific, Japan, Middle East & Africa and Latin America Adrian retired from PTC at the end of 2022. After 10 years as a non-Executive Director, in July 2023 he ...

Dr. Julia Hammett-Jamart Ph.D. is the Managing Director of Catalyst and a pioneering impact producer who works with filmmakers to drive real-world policy and social change. Her presence at the World Orphan Drug Congress stems from her role as the Impact Producer for Sky’s the Limit, a forthcoming...

Matthias Heck is Senior Advisor with EUCOPE.

Niklas Hedberg is the Chief Pharmacist at the Dental and Pharmaceutical Benefits Agency (TLV) in Sweden. Niklas is the HTAR Coordination Group co-chair for medicinal products. He was the Chair of the Executive Board for EUnetHTA21 (2021-2023) and for EUnetHTA JA 3 (2018- 2021). Niklas has been...

Victoria is a Rare Disease Policy Manager at Newcastle University and a co-lead and founder of the Newcastle Centre for Rare Disease. She has been an active participant in the generation of rare disease (RD) policies and policy-related outputs since 2012. For instance, she led the knowledge-base...

Debbie Hellenbrand is an internationally recognized patient advocate, speaker, and founder of Zebralife4us. Since 2015, she has been dedicated to raising awareness, building connections, and improving care for people living with chronic conditions and mental health challenges. Through her...

I’m a health economist and econometrician working at the intersection of health policy, health technology assessment (HTA), and the broader economic value of health.My work focuses on understanding how health interventions translate into real-world outcomes — not only in terms of clinical...

Patrick Hopkinson is a Global Market Access/ HEOR expert with over 30 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...

Mohit Jain is Vice President, Market Access & Pricing, Europe and International at Ascendis Pharma. In this role, he leads market access and pricing strategy across diverse healthcare systems, supporting the development of sustainable pathways for patients to access innovative therapies.With a...

  Daria Julkowska holds a PhD in molecular biology and completed postdoctoral research in cellular biology at Institut Pasteur (Paris). She also holds an MSc in Research Management (Paris Dauphine) and has extensive training in EU research policy and communication. Active in the rare disease field ...

Tim Kanters (PhD) is a senior scientific researcher at the institute for Medical Technology Assessment (iMTA), part of the Erasmus University Rotterdam. In this role, he is involved in cost-effectiveness studies, costing studies and real-world evidence studies in various clinical areas. He was...

Kaja is a biotechnologist from education. She obtained her degree at the International Faculty of Engineering at the Lodz University of Technology in Poland. She has been working since 2009 in the European Commission, DG SANTE. For seven years she has worked in the GMO field where she was...

Ian Katonka - Associate Partner at Kylla Capital Partners and Kylla Corporate Transactions We invest in Seed - Series B stage companies with €250K - 2.5M check sizes for 10-20%of the funding round. Sector and location agnostic 

Batyrkhan is a global Market Access and HEOR leader with 14 years of experience across affiliate, regional, and global roles. His work focuses on shaping evidence, value, and access strategies for innovative therapies, with a particular focus on rare diseases. He brings experience across...

Diana Kwast-Hoekstra, MScN RN, is Founder and President of Stichting (On)beperkt Vooruit! / (Un)limited Forward! Foundation and a PhD Candidate at the University Medical Center Groningen in the Netherlands. She is a patient expert with lived experience, nurse scientist and international patient...

Yann Le Cam is a patient advocate who has dedicated over 30 years of professional and personal commitment to health and medical research NGOs in France, Europe, and the United States in the fields of cancer, HIV/AIDS, and rare diseases. Yann is a strategic advisor at VOZ Advisors, advising the firm ...

Professor James A. Levine, MD, PhD, MBA, is President of Fondation Ipsen, under the aegis of Fondation de France. A physician and scientist, he spent nearly three decades at Mayo Clinic before taking on his current role. His academic appointments have included Professor of Medicine at Mayo...

Veronica Lopez Gousset, MPH, has over 10 years of experience ensuring patients’ voices are represented in healthcare decision-making. She works with the patient community to better voice their needs and challenges, with health technology assessment (HTA) bodies to improve their processes for...

Craig is a highly experienced executive, founder, advisor, and board director with a deep background in biotechnology and life sciences. He’s dedicated his career to advancing new technologies and treatments to improve health, including gene editing, mRNA, microbiome-based treatments, genomic...

Malwina Mejer is a Senior Economist at Copenhagen Economics, where she has worked since 2022 as part of the Healthcare and Life Sciences team. She applies economic analysis to regulatory and policy questions in the life sciences sector, supporting decision-making for policymakers and industry. Her...

Sarah is a healthcare strategy consultant with over 15 years of experience spanning medical affairs strategy, patient engagement and communications. Sarah specializes in patient-centred strategy, with particular interests in behaviour science and embedding the patient voice into clinical...

𝐖𝐡𝐞𝐧 𝐨𝐭𝐡𝐞𝐫𝐬 𝐬𝐞𝐞 𝐡𝐨𝐫𝐬𝐞, 𝐢 𝐚𝐦 𝐥𝐨𝐨𝐤𝐢𝐧𝐠 𝐟𝐨𝐫...

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...

Paula Naughton is a Nurse Practice Development Cordinator at The Health Service Executive (HSE) in Ireland and the Founder of The Join Our Boys Trust. A Registered General and Mental Health Nurse with a MSc in Mental Health. She has practiced extensively as a Specialist Practitione in community...

Marco Nievo serves as Chief Scientific Officer at the Children’s Tumor Foundation Europe , where he leads scientific strategy and advances research initiatives aimed at improving outcomes for people living with neurofibromatosis (NF). His work focuses on drug hunting.  

Dan O'Connor is Executive Director Regulation at the Association of the British Pharmaceutical Industry (ABPI). He is medically (Leicester) and PhD (Imperial) qualified, and on the specialist register for Pharmaceutical Medicine. He joined the ABPI from the Medicines and Healthcare products...

Dr. Wija Oortwijn studied health sciences, holds a PhD in Medicine and has 35 years of relevant professional experience in health technology assessment (HTA) and health policy analysis around the globe. Currently, she is senior lecturer in HTA at Radboud University Medical Center as well as chair...

Sam is an expert in generating evidence to inform decision-making. He has spent the last 15 years leading translational and clinical research programmes across academia, biotech, and healthcare. Sam's research has informed clinical practice and guidelines in oncology, and he has developed a...

Philippe Pakter is an international lawyer in the New York Bar, currently working on a PhD in law at the University of Geneva. His doctoral research focuses on cross-border healthcare law for rare disease patients in Europe. Philippe serves as general counsel for the rare disease patient...

Enrico Piccinini brings a deep sense of purpose and decades of leadership to his role as Senior Vice President, Europe and International at Chiesi Global Rare Diseases. Since 2019, he has led efforts to expand access to therapies across Europe and emerging markets, ensuring that patients with rare...

Vinciane Pirard, MD Vinciane Pirard is a Medical Doctor trained at UCLouvain in Belgium, with an extensive career in the pharmaceutical industry dedicated to rare diseases since joining Genzyme in 2002. She currently serves as Medical Affairs Rare Diseases – Scientific Advocacy and Insights Lead...

Kelly is a pharmacist with a master’s degree in medicines regulatory affairs who joined EMA following several years working in the pharmaceutical industry, primarily in pharmacovigilance. At EMA, Kelly has contributed to the implementation of the 2012 European Union pharmacovigilance legislation...

Manca has over 9 years’ experience in health economic outcomes research and market access consultancy across global and local markets. She specializes in innovative approaches to value demonstration, including how best to use new and emerging technologies to expedite market access.

Oevr 20 years of cross-functional strategic high-performance medical leadership within the Biopharmaceutical environment in Global & Regional Medical Affairs & Strategy, Medical Education, Evidence Generation, Patient Advocacy & Engagement, of which about 15 years in Rare...

Nawab Qizilbash, MD DPhil, is CMO at OXON Epidemiology, a European-based full-service CRO specializing in global data collection and secondary data real-world evidence studies and strategy. He is also Honorary Associate Professor in Epidemiology at the London School of Hygiene and Tropical Medicine ...

Martina Ragazzi is a Translational Project Manager at Fondazione Telethon, where she coordinates the development of advanced therapy medicinal products (ATMPs) for rare genetic diseases, guiding multidisciplinary teams from preclinical research through clinical development.With over ten years of...

Dr. Ravi M. Ram is a public health leader and CEO of Rare Diseases International (RDI). He brings a focus on social participation and bridging conversations among patients and people with lived experience with policymakers and health experts. Based in Nairobi, Kenya, and Toulouse, France, Ravi has...

Clàudia Roca Herms is Corporate Director of Patient Advocacy & Public Affairs at Ferrer, a purpose-driven pharmaceutical company committed to social justice. With nearly two decades of experience in the healthcare industry, she brings together patient advocacy, public affairs, policy, market...

Senior professional with a PhD in biotechnology and over 10 years of experience across public research, pharmaceutical and medical device industries, and public healthcare institutions. Proven expertise in health innovation, clinical research ecosystems, health data governance and institutional and ...

Stephanie Said is a licensed pharmacist with a doctorate in pharmacology and has comprehensive experience in Health Technology Assessment (HTA) of medicinal products. Since 2016, Stephanie Said has been working as a scientific advisor in the GBA's Pharmaceuticals Department focusing on...

Jean Saslawsky is Chief Executive Officer of EURORDIS-Rare Diseases Europe, the independentvoice representing people living with rare diseases across Europe. He joined EURORDIS in June2026, bringing more than 25 years of leadership experience across the health, humanitarian andsocial impact...

Maurizio Scarpa, MD, PhD, pediatrician, is the Director of the Rare Disease Program at the   Regional IRCCS San Gerardo dei Tintori Research Hospital in Monza, Italy. He is Professor of Pediatrics at the Dept. for the Woman and Child Health, University of Padova, Italy, and the Co-Founder of the...

László Schiller-Tamás is a digital health strategist and delivery professional with over 15 years of experience in digital transformation, including seven years dedicated to healthcare technology solutions. As Solution Delivery Lead at Genontech Digital Health Solutions Ltd., he leads the design...

Claire holds a PhD in biochemistry and has led organisations that advocate for commercial delivery of science for 25 years, starting within the Cambridge UK biotechnology cluster. She has worked to champion biotechnology across sectors with leadership roles in the Council of European...

Dr. Violeta Stoyanova-Beninska, MD, PhD, MPH has combined in her professional career clinical work, research in genetics, and regulation of medicines, particularly of therapies for rare diseases. Violeta has joined the European Medicines Agency in 2024 as senior scientific specialist at the Human...

Jasiek Tabeau is a glioblastoma patient, patient advocate and founder of Glioblastoma.care, a foundation dedicated to helping patients explore treatment opportunities and improving access to innovative care. Following his diagnosis in October 2025, Jasiek applied his professional experience in...

  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 drive regulatory strategies and accelerate patient access to innovative therapies across Emerging Markets. ...

I am Chief Therapeutics & Clinical Strategy Officer and President of Europe at Worldwide Clinical Trials, where I lead enterprise therapeutic and clinical strategy, strengthen the company’s scientific positioning, and support the development and delivery of complex, globally integrated clinical ...

Sheela Upadhyaya Founder, Rarely Ordinary | Rare Disease, Health Policy and Life Sciences Expert Sheela Upadhyaya is the founder of Rarely Ordinary and a highly respected rare disease, health policy and life sciences expert. With more than 20 years’ experience across the NHS, NICE, government and ...

Başak Uysal (Dr, PhD) is Project Manager of the Data Service Hub within the European Rare Diseases Research Alliance (ERDERA) coordination team, where she works at the intersection of rare disease research, data infrastructures and collaborative innovation. With a background in molecular...

Professor Clara van Karnebeek is a world-renowned pediatrician and geneticist fighting for the future of children with rare diseases. As Director of the Emma Personalized Medicine Center and United for Metabolic Diseases, she integrates genomics and metabolomics to discover novel treatments for...

Mr van Lente is a health economist and an independent international consultant specialising in health financing. He has over 30 years' experience working in the German healthcare sector, including as Director of the AOK Federal Association, Germany's largest health insurance group. He spent ...

Sanne van Rijn is a patient advocate, EUPATI fellow and psychologist with Vereniging D-CAA, bringing both lived-experience insight and professional expertise to rare-disease advocacy. She is committed to ensuring that the needs, priorities and voices of people living with rare conditions and their...

Lidewij Vat, PhD, works at Synergist, where she works for the Patient-Focused Medicines Development (PFMD) initiative and the From Testing to Targeted Treatments (FT3) consortium, leading the Patient Engagement and Patient Experience Data (PE&PED) workstream. With over 15 years of experience in ...

I am Director of the HTA Programme and Strategic Partnerships at the Centre for Innovation in Regulatory Science (CIRS), an independent, not-for-profit organisation, where I lead global HTA strategy, policy research and international engagement.My work focuses on the intersection of regulatory...

Jay Weaver, PharmD, MPH, is Vice President of Payer Relations at PANTHERx Rare Pharmacy, where he leads strategies to strengthen partnerships with PBMs and health plans to ensure access to innovative therapies for rare disease patients. With over two decades of experience spanning pharmacy...

Director Development at DEBRA Research since February 2025 Programme Leader, AIC468 at AiCuris from February 2022 to January 2025 Clinical and Research Manager, University Hospital Cologne, November 2015 to January 2022, leading multi-stakeholder research networks and international clinical...