Better Medicines for Children, A DIA/EFGCP Conference
Martine Dehlinger-Kremer, PhD
- Vice President, Pediatric Development
- Synteract, United States
Dr. Martine Dehlinger-Kremer has more than 30 years industry experience including 26 years of progressively higher levels of Regulatory and Medical Affairs leadership responsibility. She has contributed to the global development of numerous products, including orphan drugs and biosimilars, from early development through clinical studies and registration. She has participated in more than 100 NDAs and MAAs (local format, eCTD, NeeS). She has served as Chair of EUCROF Paediatric Working Group since 2008. She holds a doctorate in sciences from University J.W. Goethe, Frankfurt, and academic studies degree in neurophysiology from Louis Pasteur University, Strasbourg. Award:PharmaVOICE 100’s Most Inspiring People in Life Sciences, August 2015
Mette Due Theilade Thomsen, PhD
- Managing Director
- PIP Adviser, Denmark
Mette is Managing Director of "PIP Adviser" which provides expert regulatory advice on PIPs and PSPs. She has a background in authorities & industry. In the Danish Medicines Agency (2000-2006), she was nonclinical assessor of MAAs and EU Scientific Advice. She was member of the SWP, Gene Therapy WP, Biosimilar WP and PGWP. Also a rapporteur for CHMP and ICH guidelines. As Scientific Officer in EMA, Mette handled PIP procedures with PDCO, was in the FDA-EMA paediatric cluster and established the PDCO Nonclinical Expert Group. In Novo Nordisk (2009-2017), Mette lead all PIP/PSP procedures, was paediatric strategy advisor across the company and was in the EFPIA paed group. Mette also spent a year as Senior Director in Shionogi, London.
- Global Program Head
- Bayer AG, Germany
Heidrun Hildebrand has 20 plus years’ experience in drug development. Heidrun started her carrier in the Pharmaceutical industry as Quality Manager within Clinical Development and held different positions with increasing responsibility. Since 2017 Heidrun is part of the new Therapeutic Area Pediatric Development at Bayer. Heidrun is one of he co-leads of the EFPIA Consortium driving the IMI2 initiative to create a pan-European Paediatric Clinical Trials. Network.
Geneviève Le Visage, PharmD, MSc
- Regional Head RA Europe Policy & Liaison
- Novartis Pharma AG, Switzerland
Pharmacist by training, with a masters in Science Communication Currently heads the team responsible for regulatory intelligence and policy for the EU for Novartis in Basel, Switzerland, supporting project teams in establishing their strategy for filing in the EU, including for paediatrics, and bringing this experience to ongoing discussions on the Paediatric Regulation. Prior to this, she worked for ten years at the European Medicines Agency in London, mainly in the field of information for the public. Co-chair of the EFPIA priority working group for Paediatrics.
Cécile Ollivier, MS
- Chief Innovation and Regulatory Science Officer
- Aparito , Netherlands
A senior health engineer with over 10 years of global drug development experience in children & rare diseases. Previously, a scientific officer in the Paediatric division of Science & Innovation at EMA, providing technical & regulatory guidance on the design, conduct & interpretation of paediatric developments across multiple therapeutic areas. Reviewed over 200 PIPs and SA across all phases of development. Recognised for leading the EMA extrapolation strategy and activities including the EMA/FDA harmonisation for Gaucher disease & global harmonisation of criteria for development in paediatric PAH with patients, healthcare professionals, FDA & Health Canada. An expert in the E11 R(1) working group & paediatric standing group for the ICH.
- Founder, Little People Association & Temerarii Club
- The Romanian Community of Cancer Survivor Adolescents and Young Adults, Romania
Katie is the founder and acting CEO of the Little People children’s cancer charity in Romania & Republic of Moldova providing psychosocial support services in 10 hospitals with nearly 100% patient reach. She has set up the Romanian Community of Childhood Cancer Survivor Teenagers and Young Adults. Katie was a co-chair of the FP 7 project PanCareSurFup WP6/7 Implementation/Feasibility sub-group and has been responsible for patient accrual for WP 17.6 survey for ENCCA. She’s a steering group member for the European Network for Teenagers and Young Adults with Cancer. Since May 2015 she is one of the Directors of PanCare. In 2016 she became a member of the EFGCP Children’s Medicines Working Party. She is the co-founder of Youth Cancer Europe.
Siri Wang, PhD
- Scientific Director; PDCO Member
- Norwegian Medicines Agency, Norway
Siri Wang is pharmacist by education and holds a PhD in pharmacology from the University of Oslo. She has been working for 15 years in hospital pharmacy; as head of the production unit, as assistant chief pharmacist, and for 10 years as counseling pharmacist and clinical pharmacist, specifically involved in paediatric and geriatric medicine. In 2005 Siri was appointed to EMA’s Paediatric Working Group (PEG), and has been delegate to the Paediatric Committee (PDCO) since 2007. She was chair of PDCO’s Formulation Working Group from 2008 until 2013. Siri has been at the Norwegian Medicines Agency (NOMA) since 2007 and is currently holding a position as Scientific Director at NOMA, with particular focus on paediatric medicine.
Dirk Mentzer, DrMed, MD
- PDCO Chair 2013-2019; Head of Pharmacovigilance Unit
- Paul-Ehrlich-Institut, Germany
Paediatric training in Frankfurt (DE) and Belfast/UK with special interest in Neonatology, Haematology, infectious disease. Paediatric Consultant in Frankfurt and Uelzen. Since 2004 working as Head of PhV at Paul-Ehrlich-Institut (NCA), Coopted member for Paediatric PhV from 2006-2012 and member of PDCO at EMA from 2007-2013. Chair of PDCO since 2013.
Ralph Bax, MD, PhD
- Head of the Paediatric Medicines Office
- European Medicines Agency, Netherlands
After studying medicine and health care management, Ralph Bax completed his training as a paediatrician at the Children’s University Hospital in Munich, Germany. He specialised in neonatology and neonatal neurology and led the department of developmental neurology. His main research interests were in the field of brain electrophysiology and haemodynamics in preterm neonates. In 2007 he joined the paediatric team at the EMA, where he mainly worked in the field of assessing PIPs in neonatology, neuropaediatrics and on public health aspects such as unmet paediatric medical needs. Ralph is the neonatal contact point at EMA and is the Agency’s liaison to the International Neonatal Consortium. Since January 2016 he has taken on his current role.
- Professor of Pharmaceutical Technology
- Heinrich-Heine-University Düsseldorf, Institute of Pharmaceutics and Biopharmace, Germany
- Policy Officer, Deputy Head of Unit
- Unit B5 Directorate General for Health and Food Safey, European Commission , Belgium
Fabio has a PhD in biochemistry and a master’s in management of biotech companies. After working several years as a university researcher, both in the public & private sector, he joined the EU Commission in 2004 where he has worked in several areas of the food legislation, from nutrition to food contact materials. In 2011 he joined the units dealing with pharmaceutical products in DG SANTE. He has worked on quality of medicines, falsified medicines and clinical trials. During 2016 & 2017 Fabio was seconded to the European Centre for Disease Prevention and Control (ECDC) in Stockholm and worked in particular on vaccination & antimicrobial resistance related issues. He has been in charge of the paediatric medicines file since late 2018.
- Co-founder and Joint CEO
- Duchenne UK, United Kingdom
David Jones, MS
- Expert Pharmacotoxicologist, Clinical Trials Unit
- Medicines and Healthcare products Regulatory Agency (MHRA), United Kingdom
David R Jones Expert Scientist (Pharmaco-Toxicologist). I have been a Toxicologist for over 30 years. I am responsible for the Safety Assessment of Clinical Trials Applications within the UK. I am the UK's representative on the EU's Safety Working Party (SWP). I also represent the SWP at ICH.
Anja Schiel, PhD
- Senior Adviser/Statistician, Unit for HTA and Reimbursement
- Norwegian Medicines Agency (NoMA), Norway
Anja Schiel has studied Biology at the Johannes Gutenberg-University, Mainz, Germany. She received her PhD from the Free University in Amsterdam in 2006 and worked several years as Post-Doc on a range of subjects focusing on oncology, immunology and molecular biology, first at the University of Leiden and later at the University of Oslo, before starting at the Norwegian Medicines Agency (NoMA) in 2013. At NoMA she is working as special adviser/Statistician/Methodologist both on regulatory (EMA) and HTA projects. She has been Chair of the Biostatistics Working Party for the last 3 years and currently is the Chair of the Scientific Advice Working Party at EMA.
Mark Turner, MD, PhD, MRCP, FFPM
- Co-Director International Neonatal Consortium
- NIHR Clinical Research Network Children's, Theme Liverpool Women's Hospital, United Kingdom
Neonatologist with a special interest in early phase drug development and improving the availability of high quality medicines to children and babies. Has studied 20 medicines in neonates and pregnant women including antibiotics, excipients, treatments for in utero growth restriction, inotropes, postnatal steroids and surfactant. Current work is focused on developing research infrastructure for pediatric medicines research in Europe and beyond.
Thomas Kühler, PhD, MSc
- Head GRSP EU/AMEE
- Sanofi R&D, France
A PhD chemist by training with late Nobel Prize Laureate Donald J. Cram. In recognition of his contributions to the field of Medicinal Chemistry and longstanding experience in drug discovery he was appointed Associate Professor in Medicinal Chemistry at Uppsala University in Sweden. He has garnered his work experience from Hässle AB in Sweden (now AstraZeneca), the Medical Products Agency, Novo Nordisk A/S in Denmark, and most recently Sanofi in France. Thomas has served on the Board of the Drug Information Association in the US. He also was a member and chair of the Board of The Organisation for Professionals in Regulatory Affairs in the UK. Currently, he’s serving on the Board of the MedTech & Pharma Platform headquartered in Switzerland.
Nathalie Seigneuret, PharmD
- Senior Scientific Project Manager
- Innovative Medicines Initiative (IMI), Belgium
Nathalie Seigneuret is a Senior Scientific Project Manager at Innovative Medicines Initiative (IMI), responsible for the coordination and management of projects within the scientific team. Having a regulatory background, she also serves as a liaison for regulatory matters. Before joining IMI in 2011, she was a scientific administrator at the European Medicines Agency (EMA) where she gained experience in the centralised procedure and other activities notably in pediatrics with the implementation of the EU Pediatric Regulation at the Agency level. Nathalie Seigneuret is a Doctor in Pharmacy with specialisation in drug development and regulatory affairs.
Karen Van Malderen
- Federaal Agentschap voor Geneesmiddelen en Gezondheidsproducten
- Agence Fédérale des Médicaments et des Produits de Santé, Belgium
Pharmacist by training, with an additional Master’s Degree in Applied Toxicology. Karen has joined the Belgian Medicines Agency in 2011 where she works as a non-clinical assessor of marketing and clinical trial applications and scientific advice, with particular focus on paediatric medicines. She is a member of the PDCO’s Non-Clinical Working Group (NcWG) since 2012 and has been appointed as chair of this group in 2017. She is also the Belgian delegate to the PDCO. Since recently, she also represents the EU at ICH S11.
Dimitrios Athanasiou, MBA
- Patient Advocate
- WORLD DUCHENNE ORGANIZATION (WDO) , MDA HELLAS, Greece
Dimitrios Athanasiou is a PDCO member representing EURORDIS. He holds a BA and an MBA in Financial Management He attended Eurordis Summer School and European Patient Academy (EUPATI) acquiring basic biotech and regulatory knowledge. As a full time patient advocate in DMD, he is a board member of World Duchenne Organization (WDO) and a board member of EFGCP, Co-Chairing the Children Medicines Working Party (CMWP).
Elin Haf Davies, BSN, PhD
- aparito, United Kingdom
Elin Haf Davies PhD began her career as a Children’s Nurse, before going on to gain her PhD at University College London. She was part of the paediatric team at the European Medicine Agency responsible for implementing the Paediatric Regulation in Europe. After 6 years as a regulator Elin left to start aparito, a digital health company providing remote patient monitoring specifically for clinical trials. Elin is also part of the IMI consortium ADAPTSMART, focusing on adaptive pathways.
- Healthcare Program Director
- SYSNAV, France
Samuel D. Maldonado, MD, MPH
- Vice President, Head of Child Health Innovation Leadership Department
- Janssen Research & Development, LLC , United States
Dr. Maldonado is Vice President of Pediatric Drug Development at J&J. Previously; he was Medical Officer at the FDA for 8 yrs, where he participated in scientific and policy processes leading to the Pediatric Exclusivity Initiative. His experience in pediatric drug development expands over 25 years
- Duchenne Parent Project, Netherlands
Founder and President of the Duchenne Parent Project Netherlands since 1995, Chair and co-founder of the World Duchenne Organization and board member of Eurordis. She is the mother of an adult son with Duchenne MD & orthodontist by training; chair of the Ethics Board & member of the Executive Committee of TREAT-NMD; volunteer for Eurordis; a patient expert for the European Medicines Agency. Involved in several publications regarding Standards of Care and drug development for Duchenne Muscular Dystrophy, concerning development of outcome measures & role of Biomarkers. She is co-author of a handbook on psychosocial aspects of DMD. She participates in several EU funded projects, such as Asterix, Adapt Smart, RD-Connect Vision-DMD & Share4Rare.
- F. Hoffmann-La Roche AG, Switzerland
Aude Clement is a Global Regulatory Leader at Roche. For the past 6 years, she has been working on drug development programs in rare paediatric conditions: Duchenne muscular dystrophy and spinal muscular atrophy. She also leads an internal rare diseases regulatory experts network and contributes to Roche’s Digital Health activities. Prior to her current role, Aude held positions of increasing responsibility in EU and international regulatory affairs, across therapeutic areas, for 17 years at Roche, Sanofi-Aventis and Pfizer. Aude holds a French Doctorate in Pharmacy and a Master's Degree in International Drug Development and Registration.
Andreas Hartmann, PhD
- Executive Director
- Novartis Pharma, Switzerland
22 years of experience in early and late stage pre-clinical and clinical drug development of small molecules, biologics and gene therapy products. Current position: Executive Director, PreClinical Safety, Novartis Pharma, Switzerland. Role: Global PCS Therapeutic Area Head Neuroscience and Marketed Products. Previous positions at Roche and University of California, San Francisco.