Areas of expertise: Internal Medicine. Rare disease. Clinical Epidemiology. HTA. Rational Use of Medicines. Pharmacovigilance. She is a member of the ISPOR Rare Diseases special interest group. Dr. Vinhas de Souza is a MD, with Master of Science in Clinical Epidemiology at NIHES-Erasmus University (NL) and PhD in Medicine-area: HTA and rare diseases- UFRGS (BR). She acts as Consultant to the Brazilian National Commission for Incorporation of Health
Technologies (CONITEC): medicines, rare diseases and HTA.
She is a Researcher at the Clinical Genetics HTA Group and Collaborates with the Exceptional Medicines Commission (COMEX), both at the Hospital de Clínicas de Porto Alegre/HCPA/Brazil.
Also, at HCPA, she works at the 'Drugs and Therapeutics Committee' and at the 'HCPA Research Committee and Ethics in Research Committee'.
Formerly she was a Scientific Administrator at European Medicines Agency/EMA in London, UK (risk management and safety: areas oncology, haematology and autoimmune diseases).
Steve is a Senior Advisor to the Director, NCATS at NIH. He helped establish the Office of Orphan Products Development at FDA in 1982, the rare diseases initiative at NIH in 1989, Director of the Office of Rare Diseases Research from 1993-2014. His major focus has been on stimulating research with rare diseases and orphan products and developing information for patients, families, health care providers, research investigators, the biopharmaceutical industry, and the public. The emphasis included information about rare diseases, active and completed clinical trials, ongoing research, and patient advocacy groups. He has co-authored numerous journal articles, book chapters on Rare Diseases Epidemiology.
As driving passion of his career in biotech and pharma, Sebastian has set out to maximize his expertise in the commercialization of innovative therapies for (ultra-)rare diseases. As exciting part of this journey, Sebastian is currently supporting Novartis’ ocular gene therapy launch in Europe as a Brand Director, driving therapeutic transformation for one specific inherited retinal disorder. Prior to that, he spent 6 years at Alexion Pharmaceuticals in various regional leadership roles across Europe, Latin America, and in local functions in Germany, leading successful launches for different innovative therapeutic agents across diverse therapy areas, such as Neurology, Metabolic disorders or nephrology. Prior to focusing his career on rare diseases, he spent 9 years with MSD Switzerland, building his commercial acumen through local marketing and sales functions of increasing responsibilities. Sebastian holds a Master’s Degree in Political Sciences of the University of Lausanne and is married with two small children.
Kimon Doukoumetzidis is the Brand Director for Ocular Genetic Diagnosis at Novartis, where he leads the design and implementation of innovative diagnosis and patient identification strategies across multiple European markets, for a first-in-class ocular gene therapy. A molecular geneticist with a passion for rare disease therapy commercialization, Kimon spent many years in academic research before taking the leap of faith into the pharmaceutical industry. He started his career at Novartis 8 years ago, where he held positions of increasing responsibility across different functions (medical affairs and commercial) and multiple European countries. During this time, Kimon contributed to the successful launches of a handful of specialty medicines, including one blockbuster and two innovative treatments in rare diseases, in different therapeutic areas encompassing all together no less than 12 different indications. Kimon studied Molecular and Cellular Biochemistry at the University of Paris and received his doctorate in Molecular Genetics from the University of Zurich. He lives in central Switzerland with his wife.
Dairine is a pharmaceutical industry executive with 20 years experience in the pharmaceutical, biologic and device industries, including 9 years in senior management positions in the Irish Health Products Regulatory Authority (HPRA) working with both the European Medicines Agency (EMA) and the European Commission. She led the establishment of the National Health Regulatory Agency for Pharmaceutical Products in Bahrain and has been involved in the successful global clinical development and post-marketing management of many pharmaceutical products across multiple therapeutic areas, many for rare diseases. She was global Vice President, Strategic Regulatory Affairs at ICON for 2 years before co-founding and successfully leading the start-up phase of Open Orphan Plc, an Irish CRO for orphan drugs.
She is currently a NED for Soleno Therapeutics Europe Ltd., Omnispirant Ltd. and Reneurx Ltd. and serves on the board of Fighting Blindness as well as providing strategic support to pharma companies focussed on development of a novel therapies for rare diseases.
Dairine is a pharmacist and holds a PhD in pharmacy and a certificate in international business.
Alex is a pioneer in the use of technology and digital communication with extensive experience both in-house in pharma and on the agency side.
During his 30+ years of experience he has designed and implemented Global education, communication & PR programmes working with healthcare professionals of all types.
Alex has worked with major pharma companies and patient associations across several rare disease areas including multiple sclerosis, Fabry disease, cystic fibrosis and several others.
Many of the programmes Alex worked on were designed to build disease specific rare disease communities, to raise awareness and speed diagnosis. All with the aim of hopefully improving outcomes for the people and families affected by them.
Rüdiger is a scientist by training with a Ph.D. in pharmacology who has been working in the pharmaceutical industry for 25 years. He held various roles in Sales and Marketing before serving as Business Unit Director in Germany for large pharmaceutical corporations (J&J, BMS). He worked as General Manager for Germany and Central / Eastern Europe for Shire (today part of Takeda) before founding the first foreign affiliate for Ultragenyx, a company exclusively focused on Rare Diseases with a high unmet medical need, as General Manager in 2016. Rüdiger has launched multiple Orphan Drugs in Germany, Poland and other CEE countries, represented his company in national and international industry associations working groups on Orphan Drugs and has ample experience in pricing and commercializing Orphan Drugs in different health care systems.
Christopher is the CEO and chairman at Volv Global, an applied data science company. Data Science, AI and data are the lifeblood of Volv and Christopher is excited to be bringing their cutting-edge offerings to market. Volv’s mission is to speed science, reduce the cost of healthcare and so enhance the economic output and well-being for all.
Christopher has over 20 years of experience as a technology entrepreneur and business advisor, having worked for many Blue-Chip organisations helping solve their critical global scale data problems.
Christopher is also a visiting professor at 2 leading universities in Switzerland, EPFL and UNIL, teaching executive management on the current cutting-edge approaches to create value from data and dealing with current issues in data management.
Volv’s product inTrigue works for Rare Disease companies and applies Volv’s approaches to shortening time to diagnosis as well as finding hidden undiagnosed patient cohorts better than any other.