Yann Le Cam is a patient advocate who has dedicated 25 years of professional and personal commitment to health and medical research non-governmental organisations in France, Europe and the United States in the fields of cancer, HIV/AIDS and rare diseases.He has three daughters, the eldest of whom...

Ségolène Aymé is Emeritus Director of Research at the French Institute of Health and Medical Research (INSERM). She was the founder of Orphanet in 1997 and Executive Manager up to 2011. She chaired the EU Committee of Experts on rare Diseases from 2010 to 2013 and is now a member of the Commission...

Kristina Larsson joined the orphan team of the EMA as the Head of Office in July 2014. Before that she spent 8 years as a scientific officer in the scientific advice team of the EMA, mostly focusing on oncology, inborn errors of metabolism and biosimilar monoclonal antibodies. Before joining the...

In July 2013, Dr. Klaus-Dieter Langner was appointed Chief Scientific Officer, responsible for all tasks within Research and Development and is a member of the Corporate Executive Board.From January 2011 he was Executive Vice President and Chief Operating Officer of Grünenthal Innovation. In...

Sheela Upadhyaya is currently the Associate Director of the Highly Specialised Technology program at NICE and is responsible for running the program to evaluate medicines and technologies for rare and ultra-rare conditions for commissioning in the NHS.Prior to joining NICE, she was responsible for...

Paul has over 20 years of clinical development experience having worked at GlaxoSmithKline, Johnson & Johnson and Novartis. In his current role as Global Head Rare Disease Clinical Development at Roche Pharma Research and Early Development (pRED), he leads a group which provides dedicated and...

Dr Draghia-Akli is Deputy Director-General for Research & Innovation at the European Commission, responsible for the Research Programmes. She serves as the Commission representative in the Governing Boards of most Joint Technology Initiatives of the EU with different industrial sectors. Dr...

Dr. François Meyer is currently Advisor to the President of the French National Authority for Health (HAS, Haute Autorité de Santé), with a particular focus on International Affairs. He joined HAS in 2005 with the responsibility of setting up the Health Technology Assessment (HTA) Division. In this ...

A physician by training, Dr Pirard has 25 years of experience in pharmaceutical industry, mostly in medical affairs at Solvay, Genzyme, Wyeth and now Sanofi -Genzyme. She has been active in the field of rare genetic disorders as global medical director for over 10 years.  Since 2012 she is involved ...

Michael’s responsibilities encompass medical information, scientific communication and education, collaborative data generation and Celgene-sponsored studies operated by Medical Affairs across Celgene’s Hematology and Oncology franchises in the EMEA region.Michael joined Celgene from GSK, where he...

Kristin Svanqvist is Head of Unit for HTA and Reimbursement at the Norwegian Medicines Agency (Noma) . She is a pharmacist by training and has been working with health economic evaluations of pharmaceuticals at Noma since 2004.The agency is responsible for the STA and HTA  for all pharmaceuticals...

Kateřina Kopečková, M.D. serves as a  senior medical oncologist at the Comprehensive Cancer Centre at the University Hospital of Motol, Prague, Czech Republic. Kateřina Kubáčková graduated with an M.D. from the Charles University Prague. She was  trained in internal medicine and clinical...

Stephanie S. Okey, M.S. has 28 years of bio-pharma experience, 26 of those years in biotechnology.  She was most recently  Head of North America and SVP, Rare Diseases business  and General Manager US, for Genzyme, a Sanofi Company, through July, 2015.  Stephanie spent  7+ years at...