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...

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...

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...

  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...

Wills Hughes-Wilson is Chief Patient Access & Commercial Planning at Mereo Biopharma and a member of the company’s executive leadership team.  She joined Mereo in March 2018 with responsibility for leading the company’s access and commercialisation strategies across the portfolio.   Prior to...

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...

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 ...

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...

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...

Nathan Chadwick is a Therapeutic Strategy Lead for Rare Disease at Worldwide Clinical Trials. Nathan has over 12 years of industry experience holding progressive roles through project management and operations at multiple CROs. At Worldwide, Nathan is responsible for ensuring the right fit...

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. She has over 2 decades of experience in the field of social impact, disability...

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 North West 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 ...

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...

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 in public health. Postgraduate in management and strategic planning for non-profit organisations.  President ASMD Spain Board of Directors of FEDER (Spanish Federation of Rare Diseases) and Board of Directors of Eurordis (Rare Diseases Europe). After working for ...

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.

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...

Heather is responsible for Global Pricing and Market Access at WEP Clinical, working with our partners to secure optimal pricing, access and reimbursement of licenced portfolios. Heather has been a successful business leader in the Pharmaceutical industry for nearly 20 years, and has extensive...

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...

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...

Nthabeleng Paulette Ramoeli Founder and CEO of RARE DISEASE Lesotho Association. Nathabeleng is a Professional Holisic Therapist and Speaker and a patient advocate living with EDS and multiple rare diseases.

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...

Valérie is the Head of Value & Access and Government Affairs for Rare Diseases, at the Italfarmaco Group since March 2023. She is leading the Italfamarco Rare Diseases Value & Access organization, bringing together years of experience, dedication, innovation and curiosity, striving to...

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...

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...

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 ...

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...

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 ...