Strategy, Advocacy & Partnering For
The Global Orphan Drug Industry

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...

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...

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...

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...

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...

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...

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...

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...

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 ...

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...

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...

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...

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...

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 ...

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...

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...

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 ...

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...

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...

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 ...

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 ...