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...

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...

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...

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...

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...

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...

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...

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 ...

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...

Mak Inoue, Ph.D., is a drug discovery executive and scientist with approximately 20 years of experience in pharmaceutical research, innovation, and external collaboration. He is the Founder and CEO of Infirmacea Inc., a preclinical-stage biotechnology company developing safe and accessible DNA...

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...

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...

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...

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...

Frauke is a biologist and regulatory scientist with a background in molecular genetics, epidemiology, evidence-based medicine and oncology. She obtained her PhD in Molecular Biology (Genetics) from the University of Cologne and holds a European MSc in Epidemiology from the University of...

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...

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...

I am a trained physician with an MD degree from 1982 and a PhD in human genetics from the University of Valencia, completed in 1989. From 1985 to 1988, I underwent pediatric training at the Children's Hospital of the La Fe University Hospital in Valencia, Spain, where I also specialized in...

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...

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.

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 ...

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...

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...

  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. ...

Pierre-Alexandre TEULIÉ is a visionary leader and expert in the orphan drug industry, currently serving as the CEO of ODDIFACT. With a proven track record of driving innovation and growth, Pierre-Alexandre is dedicated to advancing the field of orphan drugs through strategic partnerships and...

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 ...

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 ...

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...