Strategy, Advocacy & Partnering For
The Global Orphan Drug Industry

A former government pharmacist payer with over 15 years experience in access and reimbursement for new medicines and advising NHS government guidelines at NICE HTA Omar has been a visiting Lecturer at UCLH Pharmacy Programme and was both Lecturer & Examiner on Independent Prescribing the...

William Cole is a dedicated Life Science Partnership Manager at Kidney Research UK, with a passion for advancing the field of orphan drugs. With a background in life sciences and extensive experience in strategic partnerships, William is committed to driving innovation and collaboration for rare...

Elizabeth de Lange, PhD, is Professor of Predictive Pharmacology at the Division of Systems Pharmacology and Pharmacy of the LACDR (Leiden University). Her group develops predictive (CNS) pharmacokinetic (PK)- pharmacodynamic (PD) relationships in human, in health and disease, mainly using “smart” ...

Toon Digneffe is Head of Public Affairs and Partnerships Europe & Canada at Takeda. He has over 20 years’ experience in government affairs, communications, market access and patient advocacy at local, regional and global level.   Mr Digneffe has extensive experience in pharmaceutical...

Diego is the Executive Director of the Federación Colombiana de Enfermedades Raras (FECOER), an umbrella organisation bringing together 52 patient groups to represent the voice of the rare disease community in Colombia. Diagnosed with pulmonary hypertension over 30 years ago, Diego used his...

Alicia Granados is a highly accomplished leader in the field of rare diseases, currently serving as the Head of Global Rare Diseases Medical Scientific Advocacy at Sanofi, where previously she headed its global HTA strategy .With almost 15 years of experience in the pharmaceutical industry, Alicia...

Patrick Hopkinson is a Global Market Access/ HEOR expert with over 25 years experience. He is an independent consultant, founder and managing director of PHTA Consulting.  He is a senior leader in the life sciences industry with extensive expertise gained from working across multiple functions in...

Name: Liu Mi Title and position: Distinguished Professor, Institute: Department of Pharmaceutics, College of Pharmaceutical Sciences, Soochow University CSO,  Beijing Darwin Biotech Co., LTD   Email: mi.liu@suda.edu.cn   Research Areas: (1) New drugs based on proteins derived from stem...

Aurora Mateos is Founder and General Director of Menkes International (MIa), a charity focused on fighting the rare disease called Menkes. She is an international lawyer (she has been Consultant to United Nations for more than fiftheen years) on environmental law, specially law of the sea, as well...

CV: JAN A. MOL, PhD (www.uu.nl/staff/jamol) UNIVERSITY OF UTRECHT, LYMPHOMA COALITION Jan Mol is a retired cell biologist of Utrecht University and patient advocate for people with various forms of blood cancer. Jan Mol graduated from Wageningen University (Molecular Sciences) and did his PhD...

Distinguished Investigator and Chair of the Scientific Advisory Committee of the Únicas SJD Center at the Sant Joan de Déu Children's Hospital in Barcelona, ​​where he served as Head of the Genetic Medicine Service and Director of the Pediatric Institute for Rare Diseases. He is a researcher in ...

Currently the CEO and CSO of Med-Life Discoveries with over 20 years of experience in the biotech sector. My primary objective at the meeting is to seek investment opportunities and/or potential co-development or licensing partners for our rare disease program. 

Dr Joerg Schuettrumpf is the Chief Scientific Innovation Officer of Grifols, where he drives the company’s strategic focus a provider of innovative plasma medicines and other biopharmaceuticals. Dr Schuettrumpf is a physician-scientist specialised in Transfusion Medicine and Hemostaseology with...

Tara Smith is the Executive Vice President, Innovative Therapies at Med-Life Discoveries LP, a North American company developing a first-in-class plasmalogen metabolic replacement treatment. Their current focus is the ultra-rare disease Rhizomelic chondrodysplasia punctata (RCDP). Tara has over 15...

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 accelerate and ensure access to life-changing medicines for patients. Before her international role in...

  Martine Zimmermann joined Ipsen in January 2023 as Senior Vice President and Head of Regulatory & Quality Affairs, based in Boulogne (France) and since January 2024, SVP, Head of Global Regulatory Affairs. Previously she was Senior Vice President and Head of Regulatory & Quality...

Giulia Pierini is a rare disease specialist with deep expertise in patient engagement and advocacy. With over five years dedicated to the rare disease field and more than 50 successful patient-focused projects across multiple rare and complex conditions, she has collaborated with global...

Warnyta Minnaard (1989) is one of the founders of ‘Missie Tumor Onbekend’, the Dutch advocacy and patient organisation for patients and families that deal with cancer of unknown primary (CUP). CUP is the 4th most common metastatic cancer in the Netherlands. After her partner Hederik passed away...

Chris has 25+ years of life science experience with executive roles in the pharmaceutical, Biotech, Medtech and Health-tech industries. Chris has led organisations and functions at global, regional and country level and has a wealth of therapeutic experience including working extensively in rare...

Kent Rogers has been a senior executive across the healthcare industry for over 30 years, focusing on product commercialization, supply chain logistics, pricing strategies, reimbursement, market access, and alliance management. He spent more than two decades in the pharmaceutical industry in...

Tom Pulles, MD, is a dedicated and accomplished professional with extensive experience in the pharmaceutical industry. Currently serving as the Vice President and Head of Medical Affairs & Patient Advocacy for Europe at Acadia Pharmaceuticals Inc., Dr. Pulles plays a pivotal role in shaping the ...

Currently serving as the Vice President of Market Access EU & International and Global HEOR in the Global Rare Disease Unit of Chiesi Group, Diana Sinkevich oversees market access and value strategies, health technology assessment, and evidence generation across Europe and international markets ...

Andy Barrick- CEO Multiple System Atrophy (MSA) Trust UK Andy Barrick has worked in the UK voluntary sector for over 30 years, the last ten with the MSA Trust. Since 2015 the MSA Trust has doubled the number of people they support and have prioritised engagement with our members. Andy has overseen ...

Dr. Juliane Bernholz is an experienced CEO, cell biologist, and molecular geneticist with over 30 years of leadership experience in the pharmaceutical industry having worked and lived in the US and multiple countries in Europe. Currently, she serves as the CEO of AM-Pharma, a private clinical stage ...

Carla Hollak, internist for inherited metabolic disorders at Amsterdam UMC, is UvA professor of 'Medicine and Society'. Carla has been the founder of the department of metabolism in adults for the past 20 years and was at the basis of the expertise center for inherited metabolic disorders...

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. Specialization in Evaluation of Medicines and Health Technologies in the Healthcare...

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