Strategy, Advocacy & Partnering For
The Global Orphan Drug Industry

CEO & Partner at Omakase Consulting S.L., with particular interest and experience in evidence generation in rare diseases and achieving Regulatory and Pricing & Market Access for orphan drugs in Spain. Held a number of previous positions as Senior Executive Director and Global Leader in...

José Ángel Aibar is an experienced engineer who holds a senior position in a leading technology company, based at its main global R&D centre in Sophia Antipolis, a technology park on the French Riviera known as Europe's leading science and technology centre. José became involved in the...

Mrs. Bartoli is currently Inspector General at IGAS (Oversight and assessment service for Social affairs Ministry (Health, Labor, Solidarity) She was until the end of 2023, executive general director at the Haute Authority for Health, France, HAS is tasked with the evaluation of health products...

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...

Toon Digneffe Head of Public Affairs & Public Policy - Europe & Canada at Takeda   Toon Digneffe is Head of Public Affairs & Public Policy Europe & Canada at Takeda. He has over 25 years’ experience in government affairs, communications, market access and patient advocacy at...

Josie has worked in rare diseases and innovative therapies for 15 years, and currently works as an independent consultant specialising in strategic market access, patient involvement, policy and stakeholder engagement. She is the Strategic Director for Duchenne UK’s Project HERCULES, an...

Alexandra Heumber Perry is the Chief Executive Officer of Rare Diseases International, the global alliance of Persons Living with a Rare Disease. She has dedicated her entire career to contribute improving healthcare policies to benefit people living with diseases, with a particular focus in...

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...

  At EuropaBio, the European Association for Bioindustries, Adrien leads the development and implementation of the association's policy and advocacy strategy on healthcare biotech issues. Adrien works with a diverse range of stakeholders, including policymakers, regulators, industry...

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

Léon van Wouwe is bringing his clinical development and operational experience to Volv Global SA, to help build bridges between the applied data science solutions the Data Science Team develops and the drug developers, clinical researchers, clinicians, and patients. He has over 20 years of...

Dr Mary Wang is Programme Director at Rare Diseases International (RDI) where she leads RDI’s Global Programme on diagnosis, development and access to essential therapies, and oversees RDI’s technical collaboration with the World Health Organization. Mary has 20 years of experience across sectors...

DURHANE WONG-RIEGER, PHD is Chair of Rare Disease International, President of Asia Pacific Alliance of Rare Disease Organizations, Treasurer of UN NGO for Rare Diseases, Chair of Patient Advocates Constituency Committee of International Rare Disease Research Consortium, and on the Editorial Boards...

Enrico has been the Head of Europe & International Rare Diseases, at the Chiesi Group since November 2019, leading a team fully dedicated to making value-adding treatments available for rare and ultra-rare diseases. He previously had a long tenure in Genzyme and SanofiGenzyme covering different ...

Sheela Upadhyaya is a Life Sciences Consulant and a renowed expert in rare disease with over 25 year experience in the healthcare industry.  She has broad international experience spanning, UK including Scotland and the EU5 with a record of delivering leading edge innovation for rare diseases. ...

Stelios Kympouropoulos  Greek psychiatrist and politician of the New Democracy party who has been serving as a Member of the European Parliament since the 2019 elections. He was born on July 9, 1985 in Athens. At the age of 14 months he was diagnosed with spinal muscular atrophy. At the age of 15...

Virginie returned to EURORDIS-Rare Diseases Europe in March 2024 to take on the role of Chief Executive Officer, after having previously served as the organisation’s Scientific Director. Virginie received her PhD in Neurosciences from King’s College London, UK followed by several postdoctoral...

Kate Holliday is an advanced practice nurse in rare and genetic conditions, patient experience researcher and community engagement expert. She has worked in the health sector for more than 20 years. In 2020, Kate was listed on the 100+ Outstanding Women Nurse and Midwife Leaders by Women in Global...

Frauke Naumann-Winter is a molecular biologist and epidemiologist by training and holds a PhD in Genetics. Frauke Naumann-Winter is currently Head of Unit for Paediatric and Orphan Medicinal Products in the Federal Institute for Drugs and Medical devices and is the German delegate and Vice-Chair at ...

Francis Pang is Senior Vice President, Global Market Access and International Geographic Expansion at Orchard Therapeutics and has more than 20 years of experience in pricing and reimbursement, market access, health economics and corporate development, encompassing leadership roles at Shire Human...

Samantha Parker is Patient Advocacy Lead at Italfarmaco and Vice Chair of the International Rare Disease Research Consortium. She has over two and a half decades of international rare disease experience in the biopharmaceutical industry. She has a proven track record in patient advocacy, small...

Karen is a biomarkers and genetics expert. With years of drug development experience, having worked previously at Janssen Pharmaceuticals and other innovative biotechs, she is dedicated to developing new tools to tackle challenges in the rare disease space.

Rachel has more than 10 years of experience in all development phases of rare disease and cell and gene therapy clinical trials. Rachel is an ultra-rare disease subject matter expert with oversight of 30+ orphan programs in APAC, Europe and North America. She is a specialist in clinical development ...

Stefano Portolano joined Azafaros in 2021, bringing with him 20+ years in orphan disease and speciality drugs and a remarkable track record in building and developing high-performing teams. He successfully led numerous commercial, market access and medical affairs efforts in Europe and the...

Lara Bloom is the President and CEO of The Ehlers-Danlos Society and responsible for globally raising awareness of rare, chronic, and invisible diseases, specializing in the Ehlers-Danlos syndromes (EDS), hypermobility spectrum disorders (HSD), and related conditions. Lara leads coordinated 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 and Physiology with a specialization in Evaluation of Medicines and Health...

Giorgio Iotti, PhD, is Vice President, Global Rare Disease R&D Pipeline and Portfolio of Chiesi Group. He is responsible for pipeline progression of all research and development programs for treatment of rare disorders, evaluation of external opportunities and partnership with academic and...

Jon was appointed in April 2022 as the Head of Portfolio & Pipeline – EUCAN within the Takeda Regional HQ in Zurich.  In this role, Jon leads a team of therapy area aligned Franchise Heads and together they provide strategic direction and support to the Local Operating Companies and to Global...

Dr. Verdun (ver-done) received her undergraduate degree from Duke University and her medical degree from the University of Chicago Pritzker School of Medicine.  She then completed a Pediatrics Residency at Children’s Memorial Hospital-Northwestern University and a Pediatric Hematology-Oncology...

  Lauren Kopsick is the Founder and Executive Director of The Healthcare Navigation Project (THNP) 501c3, ©2024, Patent Pending. THNP is an interactive method to achieve health equity through grassroots health literacy and life skills training in an immersive environment for anyone...

Dr Kirsten Johnson is the President and one of the founders of Fragile X International. She also sits on the boards of EURORDIS - Rare Diseases Europe.Kirsten is a FMR1 premutation carrier and has two adult daughters who live with Fragile X Syndrome. Her husband lives with a rare cancer, hairy cell ...

Alicia Granados, MD & PhD in Public Health from the University of Barcelona. She is a certified specialist in respiratory medicine. Currently serving as Global Head Rare Disorder Medical Scientific Advocacy & Insights at a big biopharma, Sanofi, where she has been heading its global...

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...

Davelyn brings a robust professional background in primary care and the US payer system to industry where she has been pivotal in leading patient-centric initiatives for the past 3.5 years, including spearheading her company's compassionate use program.  As a rare disease parent, Dr. Hood also...

Elise is a health economist with five years of consultancy experience in a range of therapeutic areas, including oncology and rare paediatric liver diseases. She is proficient in the development and adaption of cost-effectiveness and budget impact models, as well as the preparation of submission...

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

my short story published by rare magazine MANZI NDAMUKUNZE, born 31st May 1976, a father of 3 children, one of them is affected by a rare disease. Manzi Ndamukunze is a notable figure in both the microfinance sector and the advocacy for rare diseases. Based in Rwanda, he has extensive experience...

Doctor in Medicine. Paediatric Neurologist in the Hospital Sant Joan de Déu in Barcelona, Spain. Specialist in movement disorders. He is member of the Spanish Society of Pediatric Neurology, the European Pediatric Neurology Society, the Society for the Study of Inborn Errors of Metabolism and the...

Georgia is a HTA submission lead with five years of market access experience. Her area of expertise lies in navigating the landscape of European HTA systems, where she applies her analytical approach and great market access writing abilities to develop and lead various European HTA submissions...

Partner at Invivo Partners, a venture capital firm specializing in early-stage biotech companies. Member of the Board of Directors at OneChain and Telomere Therapeutics and Observer at Arthex, Pulmobiotics, Gyala Therapeutics, Telum, and Esobiotec.  Previously a Project Manager in the Knowledge...

With over 10 years of experience in the pharmaceutical industry, Ana is a recognized expert in the field of Expanded Access Programs (EAP). Her expertise lies in oncology, hematology, Rare Diseases, and she has held various roles in the big Pharma (Roche, Menarini-Stemline), including leading asset ...

Rachel Yang, MD, PhD, a senior advisor at Fudan University, Zhongshan Hospital Rare Disease Center. She’s also served an advisory role at China Alliance for Rare Diseases (CHARD), and Chinese Organization for Rare Disorders (CORD). Rachel entered rare diseases serendipitously in 2015 and was...

Sarka Kubinova, PhD, is the coordinator of the MoCA (Mechanism of Coordinated Access to Orphan Medicinal Products) project. She currently works as an HTA assessor at the State Institute for Drug Control, the national regulatory authority for pharmaceuticals in the Czech Republic. Prior to this, she ...

Nadiah is the President of the Malaysian Rare Disease Society, the Regional Representative for Southeast Asia, Qatar, Oman, UAE, Jordan, Algeria, Iraq & South Korea of the Phelan McDermid Syndrome Foundation and a Council Member of Rare Disease International. She has over 2 decades of...

Iolanda Arbiol, a graduate in International Trade and Protocol and Institutional Relations, with a Master’s degree in Bioethics and Law from the University of Barcelona, is the co-founder and current director of Fundació Dr. Torrent-Farnell, an organization dedicated to improving access to...

Mariam brings over 15 years of experience in the health sector, including extensive experience in evidence generation to support Market Access and healthcare decision-making. She has worked in the public and private sectors, including the Northwest Public Health Observatory and NICE. At previous...

Lindsay has a 20 year career as CEO in the charity sector, developing several small grassroots charities into more developed established organisations. It was her role as CEO of Metabolic Support UK that captured her attention for rare diseases. After serving at MSUK for 6 years she went on to...

Thomas Bols | Head of Government Affairs and Patient Advocacy, EMEA, PTC Therapeutics and board member of Europabio Thomas has been a board member of Europabio since June 2022 and chairs a variety of industry federation working groups.  In the past he held other board positions at several European ...

Nicholas Brooke is Executive Director of PFMD.org and Founder of The Synergist, an incubator which aims to solve some of the most pressing societal problems through building partnerships to solve collective challenges that require collective solutions.  Working with key players from across the...

Darren has >10 years' experience in market access and digital health consultancy; his main role at P4A is identifying and developing innovative solutions for the market access and launch readiness challenges our customers face. Darren has a deep understanding of healthcare markets across...

Founder and President of the Wilhelm Foundation, dedicated to ending the diagnostic odyssey for People Living With Undiagnosed Diseases (PLWUD) worldwide, with a special focus on Low- and Middle-Income Countries.Co-founder and board member of the Undiagnosed Diseases Network International (UDNI)...

Joana Claverol , is the Clinical Research Director at Barcelona Children’s Hospital Sant Joan de Déu, Research Foundation, Spain, where she leads the clinical trials office since 2012 and the Patients engagement in research area since 2015. The Clinical Research Unit is a reference unit for...

Degree in Pharmacy. Postgraduate degree in public health. Postgraduate in Management and Strategic Planning for Non-Profit Organisations. President of ASMD SpainBoard member of FEDER (Spanish Federation of Rare Diseases).Board member of Eurordis (Rare Diseases Europe). After working as a community ...

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. Julien is responsible for organizing the...

João Duarte is currently Vice President and Head of Global Regulatory Affairs Excellence at Ipsen. In this role, he oversees teams responsible for Regulatory Process and Compliance, Transparency, Regulatory Intelligence & Policy, Regional Coordination and Business Operations. He held several...

Adaeze is a Senior Research Consultant in Strategic Market Access Center of Excellence based in the Rotterdam Office. She has worked on several systematic literature reviews to inform network meta-analyses, economic models, global value dossiers and reimbursement dossiers. Adaeze’s experience...

Laura I. Furlong is Co-founder and Scientific Director of MedBioinformatics. She holds a PhD in Biology from the University of Buenos Aires and an MSc in Bioinformatics from Pompeu Fabra University. With more than 18 years of experience in translational bioinformatics, one of her main interests is...

Félix Hugo Galarza Delgado is a prominent leader and activist in the field of rare diseases in Ecuador and Latin America. As President of the Ecuadorian Federation for Rare Diseases (FERPOF) and of the Ecuadorian Foundation for Muscular Dystrophy and Rare Diseases (FEDIMURA), Félix has dedicated...

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...

David is a Senior Life Science Professional and Serial Entrepreneur with 18 years of Chair, CEO and CBO leadership experience, coupled to a near 30-year track successful track record of global commercialisation of novel pharmaceutical and biotechnology platforms. 

Ritu is president of DEBRA International (and co-founder and head of the global organisation’s Singapore chapter), supporting people living with the rare genetic disorder, Epidermolysis Bullosa (EB). She is co-chair Singapore’s HTA consumer panel (ACE), serves as Vice-President of Globalskin, as...

Dr. Ron Jortner is a scientist and entrepreneur based in Cambridge, UK. He is Founder and CEO of Aspire Biosciences, a company seeking and developing new treatments for rare diseases using a novel, patient-focused methodology. Ron has been serving the rare disease community for years, working...

Tomasz Kluszczynski is a healthcare, life sciences and HEOR consultant and a visiting professor at Warsaw and Frankfurt Universities. He is the author of White Papers and articles on HTA Development in LMICs,  Equity in Patient Access, Patient Empowerment and Value Based Healthcare. Tomasz works...

Hasan is a pharmacist with a profound expertise in clinical oncology. Armed with a PhD in this specialized field, he has devoted years to conducting real-world data studies that delve into the effectiveness and safety of cancer drugs in practical, everyday healthcare scenarios. He has experience in ...

Jonathan Kornstein is Vice President, Rare Disease and Pediatrics at Caidya and has over 29 years of expereince in the Pharmaceutical and CRO industry. He has demonstrated a depth of project management and clinical operations experience in a multitude of therapeutic areas with a focus on rare and...

Evert Küppers is an all-rounded executive with over 25 years of operational experience in pharma & biotech with focus on Business & Corporate Development, Fundraising, and R&D Operations. As CEO, he led successfully cross-functional, international Management and R&D teams and closed ...

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...

Strategy Consultant specializing in pharmaceutical and biotech industries.Manage and lead project engagements focusing on determining the commercial potential of new therapies and helping clients develop strategies for drug development, pricing, market access and reimbursement, and...

Sicily Mburu, MBcHB  is a health professional with over 10 years of global health experience, holding dual MSc in Epidemiology and Health Economics & Policy. As a Scientific Officer at IFPA, she has published case on rare psoriatic disease and orphan drug designation. Sicily also serves as...

15 years in strategic consulting, pricing, market access planning; former VP Market Access Europe at Evidera; Partner at PricewaterhouseCoopers Strategic Consulting. Prior: Department of Health Berlin and Department of Health England

15+ years of Rare Disease Patient Advocacy Strategy experience focused on the clinical development process​Chemist and embryologist specialized in pre-implantation genetic testing PGT ​Founder and President of the ZIVOT (LIFE) Patient Organization  (Serbia & Balkan Region)​Experience in...

Cristina Montané, linked to patient associations in which she has worked as a volunteer for more than twenty years. She completed the Postgraduate Course in Patient Advocacy at the UIC and the UCM. She is a board member of the Catalan Patient Advisory Council, Citizen Participation Director of the...

A molecular neurobiologist with over 16 years of experience in academic institutions and CROs. Director in ICON’s Centre for Rare Disease with broad expertise in rare diseases and precision medicine trials​​​​​​. 7 years of experience in clinical program project management covering phase I-III...

Emilie has six years of experience in rare disease market access. Areas of expertise include strategy development, policy (including revision of the General Pharmaceutical Legislation), access in low- and middle-income countries and value communication. Prior to joining Dolon, Emilie obtained a BSc ...

Sue is no stranger to the challenges of market access! Having spent the first few years of her career as a UK hospital pharmacist, managing formularies; she understands only too well the need to target limited resource and optimise new drug use.  She has never lost this grounding in the realities...

Amrita is a Senior Director in OPEN Health’s Strategic Market Access Team and India Office Lead. Amrita joined OPEN Health in 2015 and has worked on a number of UK HTA submissions, systematic literature reviews, global value story and dossier development, landscape assessments and multi-HTA...

Michele Pistollato, PhD is a Principal at Charles River Associates. He specialises on policy topics in Life Sciences, ranging from value assessment and pricing dynamics for pharmaceuticals to public policy issues regarding specific therapy areas. For over 10 years he has worked extensively on the...

After graduating from ESCP Europe, a Paris Business School, Xénia Proton de la Chapelle worked in leading positions in various big international companies before running her own textile company. After the birth of her 4th child suffering from Central Congenital Hypoventilation Syndrome (CCHS), a...

Lauren has an MA in Anthropology and Development Studies from the London School of Economics and has worked in the disability sector for over 20 years. She has worked with the rare disease community in the UK and Europe since 2011 and joined the Rareminds team in 2022. Her previous experience...

Having been with Clinigen for over 12 years now, I am currently responsible for providing Regulatory advice both internally at Clinigen Group, but also working with our clients to compliantly manage their regulatory strategy for managed / early access programmes. Liaising with experts and Competent ...

Luca Trentin is a dedicated healthcare professional specializing in patient engagement and advocacy. For the last five years, Luca has focused on driving patient-focused innovation to improve healthcare outcomes. His multifaceted expertise enables him to bridge the gap between patients and...

Sonia Valero, Head of Clinical Operations and Patient Advocacy representative, Vivet Therapeutics Pharmacist by training with a specialization in neurosciences, Sonia Valero has over 25 years of experience in drug development across early to late phases in CRO, pharma, and biotech. She has...

Gabriella Almberg is a Director, Global Policy Lead at UCB. She has worked at UCB since January 2021. She is a member of the multi-stakeholder Together4RD Steering Group. Previous roles include working at Novo Nordisk as Associate Policy Director, and almost 7 years at the trade...

Alison Cave is Chief Safety Officer at the Medicines and Healthcare products Regulatory Agency. She holds a BSc Honours degree and PhD and has significant academic research and regulatory experience with roles at Kings College London, the Wellcome Trust, UK Research and Innovation and the European...

Peter Fish is a medical doctor with a degree in Human Genetics and an MBA. He has previously founded and managed a number of tech, healthtech and biotech companies and organisations, many of which focused on scalable mechanisms for delivering stratified and personalised medicine. He joined...

Ella joined NICE in 2019.  She has been a Public Involvement Adviser since 2022 and is currently the Senior Public Involvement Advisor for medicines evaluations. She is responsible for patient and public involvement in Health Technology Assessments work, Specifically medicines (Technology...

Daniel-Aníbal García Diego holds a Law degree from the University of Cantabria and is a practicing lawyer with the Illustrious Bar Association of Madrid (Ilustre Colegio de Abogados de Madrid - ICAM). He has a Master’s in Economic Analysis of Law and Public Policy from the Ortega y Gasset...

Nitin Mahadev has over 25 years of experience and expertise in pharmaceuticals. He currently serves as the Global Gene Therapy and Hemophilia Lead at Pfizer and is responsible for managing a portfolio of in-line hemophilia assets and leading the launch and commercialization of Pfizer’s pipeline in...

Dr. Shawn Ritchie is currently the CEO and Chief Scientific Officer at Med-Life Discoveries (MLD). Dr. Ritchie obtained his PhD in Biochemistry and has over 20 years of experience identifying biomarkers of disease and translating biomarker discoveries into commercial diagnostic and therapeutic...

Gloria M. Palomo holds a BsC in Biochemistry and is an enthusiast of Neuroscience and Molecular Biology, which were the topics followed in her PhD studies. During her postgraduate studies at Universidad Autónoma de Madrid, Gloria focused her work on a rare neurodegenerative disease, Friedreich´s...

Monica has worked across the community, health, education, business and government sectors. Monica is a Council Member of Rare Disease International and currently serving the genetic, undiagnosed and rare disease communities as the Chief Executive, Genetic Support Network of Victoria (GSNV). She is ...

Juan Manel Fontanet  Sacristan  currently serves as a specialist in the Medication Area of the Catalan Health Service (CatSalut) and as an associate professor in the Department of Pharmacology at the Faculty of Medicine, UAB (Parc Taulí Hospital Teaching Unit). He is a pharmacist specialized in...

General Director, Directorate-General for the Common Catalogue of Services of the National Health System and Pharmacy, Spanish Ministry of Health . From 2009-2022, Head of Medicines for Human Use Department- Spanish Medicines Agency . PhD in Medicine (Complutense University of Madrid) specialised...

Gloria M. Palomo studied  Biochemistry at Universidad Autónoma de Madrid. She is an enthusiast of Neuroscience, Biotechnology and Molecular Biology, which were the topics followed in her postgraduate studies. During her PhD studies at Universidad Autónoma de Madrid, Gloria focused her work on a...

Prof. Dr. Silvia Martín Lluesma, MD-PhD, is Head of Advanced Therapies Program at VHIO (Barcelona, Spain). Simultaneously, she serves as Professor of Cell and Gene Therapy (CGT) at the University CEU San Pablo – Montepríncipe (Madrid, Spain). She has a B.Sc. in Biological Sciences by the UCM...

Ian Rentsch has over 25 years’ experience in Multinational Corporate Affairs, Outsourcing Management, Clinical Research Development , Real World Data generation & Healthcare Technology Solutions working in large multinational companies as well as in Start Ups both Globally out of Europe as well ...

A dedicated advocate for rare disease patients, Toni co-founded DEBRA South Africa after years of connecting and supporting individuals affected by Epidermolysis Bullosa (EB) around the country. Armed with her degrees in Psychology and 10-year experience as a qualified Life Line lay counsellor, she ...

Dr. Arantxa Sancho is the Director of the Medical-Scientific Affairs Department at Farmaindustria. She holds a degree in Medicine and Surgery from the Autonomous University of Madrid and a PhD in Pharmacology. Her extensive experience encompasses both research and regulatory affairs. Before joining ...

Jordi Carreras-Puigvert obtained his MSc in Biotechnology by the Universitat Autònoma de Barcelona, and holds a PhD in Life Sciences from the university of Leiden, the Netherlands. After a postdoc funded by a Marie Curie fellowship, and co-leading the Cancer Therapy group, focused on phenotypic...

Robin Marcus is Marken’s Head of Global Decentralized Trials Market Development and has over 30 years of experience driving business optimization, championing innovation and generating aggressive revenue growth. She has successfully launched new products, divisions and companies that have exceeded...

Dr. Ina Sarel is a biotechnology executive with over 20 years of experience in product development from discovery and Proof of Concept through pre-clinical and clinical stages. Ina has broad expertise in stem/progenitor cell therapy, CMC, and regulatory requirements and previously developed a stem...

Medical Monitor at Veristat Rachel Abu Taleb, M.D, is a Medical Monitor at Veristat and a pediatrician by training and education, bringing significant expertise in many different therapeutic areas, responsible for all areas of medical monitoring of studies including patient safety, data...

After a Phd in Philosophy and a first experience in a competitiveness-focus Brussels think tank, Matteo Scarabelli joined Eurordis - the European Organisation of rare disease patients - to be responsible for the involvement of patients in research and decision making for orphan drugs. For two years ...

Kyle has nearly 20 years’ experience in the life sciences industry with expertise in sales, commercial and market access strategies, access planning, and value communication. At Red Nucleus, he has also been busy assessing value, setting price, and developing innovative contracts, with specific...

Islam El-Tantawy is the General Manager of the Insights Research Organization & Solutions (IROS), an M42 company and the homegrown Contract Research Organization in the UAE. At the helm of IROS, he is responsible for overseeing daily business operations, developing and implementing growth...

Michael Epstein begun his career in US healthcare policy analysis and then moved into life sciences commercialization consultancy, where he has worked for over 20 years. He is former senior Principal, Global Market Access Consulting, Evidera / PPD, and former Director, Commercial Strategy &...

PhD qualified with 20+ years of experience in Basic Research, Clinical Trial Monitoring, Patient Registry design and monitoring, Healthcare Policies design and implementation, Multidisciplinary Reference Units accreditation and monitoring, Patient Advocacy and Bioethics, within an academic Health...

With over 25 years of experience in clinical research, Almenia has been involved in numerous projects that integrate healthcare and technology, such as EHR2EDC. Her expertise lies in site selection strategy, investigator identification, and investigator relationship management. Beyond her...

Anna has worked with FSHD families since 2018. With the FSHD Society, she has helped launch multiple volunteer-driven initiatives, recently including the FSHD Navigator program, which serves families worldwide. In 2024, she organized the inaugural FSHD Day on Capitol Hill, to deepen the...

Chris Hendriksz qualified as a medical doctor in South Africa in 1985 from the University of Pretoria. He completed his Master’s degree in Sports Medicine at the same university. Following a rare disease diagnosis of his own child, he moved to the United Kingdom, where he continued his...

Julian Isla is the founder of the European Dravet Syndrome Federation, an European organization of patients with Dravet Syndrome. Julian Isla founded Dravet Syndrome Foundation twelve years ago. Dravet Syndrome Foundation is committed to find new treatment for Dravet Syndrome, an epileptic...

Maciek Klein is the Global Chief Business Officer at Saventic Health, a MedTech start-up leveraging Artificial Intelligence to screen electronic health records and identify patients with rare and hidden diseases. With a background in Economics and Business, Maciek leads customer acquisition and...

Dr. Levine, President Fondation Ipsen, has three decades of experiencein the healthcare sector principally at Mayo Clinic. For the last 6 years hehas been President of Fondation Ipsen, an international science foundationfocused on biotech innovation in Rare Diseases.A physician and scientist, James ...

Milan is an Executive Medical Director at Allucent with over 15 years of experience in the fields of clinical research and drug development.   Throughout his career, he has made significant contributions to bringing several drugs to the market. Milan's professional background encompasses...

Stuart Mealing is an Associate Director of York Health Economics Consortium and leads the Consortium’s health economic modelling and patient reported outcomes workstream.  He is a member of NICE's Highly Specialised Technologies Evaluation Committee and the NIHR Invention for Innovation (I4I)...

Dr David Morrow is the ATMP and Vaccine Senior Scientific Program Manager at EATRIS which is the European Research Infratstructure for translational medicine. David received a BSc (Hons) in Molecular Biology from University College Dublin in 2001 and a PhD in Vascular Biology from Dublin City...

Begonya Nafria has long experience in the field of patient and family participation in research initiatives. Her personal history as a caregiver for an adult with cerebral palsy has linked her to various projects and initiatives in the field of defending patients' rights. Her areas of expertise ...

I have a background in biochemistry and have furthered my studies in informatics, specifically focusing on databases. My professional experience began as a sales representative, which provided me with valuable knowledge of clinical practice and the limitations faced by physicians and patients. I...

Louise is a skilled, proficient leader and market access professional. Highly regarded in the international pharmaceutical community, Louise is customer-focused, building solid and long-term relationships with pragmatism and vision. With more than 30 years of experience in the pharmaceutical and...

Jennifer has over 24 years of experience in clinical development and medical affairs across small (Vicuron, Viropharma, Nabriva), mid-sized (Ipsen) and large pharmaceutical companies (Wyeth, Merck, GSK). Prior to this, Jennifer trained as a physician in internal medicine and infectious...

Simon is a pharmaceuticals and biotechnology executive, with over 15 years’ experience leading on biologic and small molecule pricing, reimbursement and launch across global markets.He is currently Vice President Market Access and HEOR and member of the International Leadership Team of Amicus...

Oskar Smrzka brings a rich background across fundamental science and biotech industry, from rare genetic diseases to cancer. Leveraging his multidisciplinary scientific and technological expertise, he founded Ablevia, a Vienna/Austria-based startup, aimed at targeting anti-drug and anti-vector...

Bob Stevens is the Group CEO for the MPS Society and Rare Disease Research Partners. Bob also has various other roles both in the UK and globally including LSD Global Chair, LSD Collaborative Vice Chair, Fabry International Network and International MPS Network Board Member. He also is a...

Daniel Moreira is the co-founder of Cellbyte, where he developed an AI-powered copilot designed to support Pricing & Market Access (P&MA) teams in the pharmaceutical industry. His focus is on creating intuitive solutions that simplify data research, enabling teams to perform advanced...

Genevieve has more than 11 years’ professional experience in clinical trial research. As Associate Director, Statistical Consulting at Allucent, she is responsible for statistical consulting activities for Phases I-IV studies globally. This involves assisting sponsors to support clinical program...

Mila is a Senior Manager in Clinical Operations at Allucent, with over 15 years of experience in clinical research and operations. Throughout her career, she has worked in Immunology, Microbiology, and Molecular Biology, providing her with extensive knowledge in the development of clinical trials...

Consultant in rare diseases.Founder and president of ANDO Portugal, the Skeletal Dysplasia National Association. Member of the Committee for Orphan Medicinal Products (COMP) at the European Medicine Agency as patient representativeEuropean Patient Advocacy groups (ePAG) representative at the...

Wendy Erler is the Vice President of Patient Experience, Patient Advocacy and Patient Insights at Astra Zeneca, Alexion Rare Disease where she is responsible for leading the enterprise innovation model to gather and incorporate patient insights throughout the drug development and commercialization...

Mónica Inês is currently the HTA Value & Evidence Team Lead for GTx Hemophilia at Pfizer. Mónica has over 25 years of experience in the pharmaceutical industry, HEOR consulting companies and health care national payers, across several therapeutic areas and geographies. She is a health economist ...

Stefan Neudoerfer is an experienced pharmaceutical market access leader with a strong commercial focus, results-driven with proven strategic and operational experience in roles spanning across European regional and global Market Access, global and regional finance & controlling, general...

Stefano Romanelli is the Senior Government Affairs Manager of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE). Stefano supports EUCOPE’s policy work on the EU Pharmaceutical Strategy, the EU general pharmaceutical legislation and related dossiers. Prior to joining...

Ross’s drive is to enable patient access to medicine – a prime professional responsibility as a clinical pharmacist. An early career in the NHS specialising in renal, haematology and neonatal medicine enabled him to understand complex medicine and therapeutics and bridge individualised patient care ...

Manel Cascalló, PhD, has more than 12 years of experience as the CEO of various biotechnology companies. Dr. Cascalló is an expert in the clinical development of oncolytic viruses and the inventor of several patents for the use of adenoviruses as antitumoral agents. Author of many original...

Colin has lead EUCAN & International Government Affairs for Alexion, AstraZeneca’s Rare Disease Unit, since May 2023, responsible for creating international government affairs strategy and executing global policy strategy in rare diseases, while creating opportunities for prioritization of rare ...

Mum to David age 27 with Ring Chromosome 20 Syndrome, I am co-founder/CEO for Ring20 Research and Support UK CIO a patient group setup to address the needs of those living with r(20) syndrome – a difficult to diagnose ultra-rare epilepsy with no recommended treatment, no patient registry, no...

SPEAKER’S BIO Elizabeth EstesOSIC Executive Director With her hands-on knowledge and passion for what she does, Elizabeth is a highly sought after healthcare strategist, speaker, teacher and mentor. Part futurist and part idea machine, Elizabeth’s passion has culminated in her latest role as...

Leire Solís is Senior Manager for Health Policy and Advocacy at the International Patient Organisation for Primary Immunodeficiencies (IPOPI) since 2014. In this capacity, she is involved in advocacy programmes at international, European and national level to ensure that the patient’s voice is...

Sofie Alverlind is a project manager and coordinator at the Swedish Dental and Pharmaceuticals Benefit agency (TLV), a government agency responsible for determining which pharmaceuticals to be subsidised by the state. She is currently leading a Government assigned project on analysing and...

Doctor in Medicine and Health Economist, he worked in various positions in the public sector (CatSalut) before founding the first European company in Health Economics and Outcomes Research, which was later acquired by IMS Health. He then joined IMS’s Senior Leadership Team in Europe. With over 35...

Maria Judit Molnar, MD, PhD, is a professor of neurology, psychiatry, clinical genetics, and clinical pharmacology, and a doctor of the Hungarian Academy of Sciences. She serves as the director of the Institute of Genomic Medicine and Rare Disorders at Semmelweis University and is a member of the...

Parvathy is a Rare Disease Specialist and healthcare professional with a Masters in Clinical Nutrition. Parvathy is an expert in Patient Advocacy and Patient caregiver education. Parvathy has dedicated her life to empowering people worldwide to advocate for themselves no matter their location or...

David Pearce is the chair of the consortium assembly for the International Rare Diseases Research Consortium (IRDiRC) that represents funding agencies, companies, and patient advocate groups that from approximately 80 countries.  He completed his undergraduate Bachelor of Science Degree with honors ...