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...

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...

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...

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 ...

Tom is a strategic, high-performance medical leader within the Biopharmaceutical environment with experience in Global & Regional Medical Affairs & Strategy, Medical Education, Evidence Generation, Patient Advocacy & Engagement, of which more than a decade in Rare Diseases. He is a...

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 ...