Optimisation of drug development for the benefit of children

Transcription

1 Joint EFGCP / DIA / EMA Better Medicines for Children Conference 2016 on Optimisation of drug development for the benefit of children 10 & 11 October 2016 European Medicines Agency, London, United Kingdom Organised by conferences@efgcp.eu -

2 Conference Rationale After 10 years of the EU Paediatric Regulation, considerable experience has been gained by all involved in paediatric drug development: companies, regulators, academia, healthcare professionals and, last but not least, patients. It is time to reflect how success has been achieved, what could be challenges and options for the future. Many products have now obtained a paediatric indication and new data are produced on a daily basis. Nevertheless many paediatric trials recruit slowly and drug development plans are not completed. Furthermore, new paths and strategies are becoming available to advance the integration of paediatric drug development with the development of medicines for adults. Paediatric research is high on the global political agenda. The aim of this conference is to discuss how drug development can be further optimised to the benefit of children s health. This will include a discussion on what we have learned during these years of full operation of the EU Paediatric Regulation, new concepts and strategies for an integrated approach at each and every step of paediatric development and sessions dedicated to specific areas of expertise such as neonatology as well as evolving concepts such as the approach to extrapolation from adult data. As always, experts from all involved parties will be present. On day one participants will discuss more specialized and hot topic issues in five breakout sessions. Day two will discuss the holistic integrated approach and the global issues. This will allow participants to discuss face-to-face with all stakeholders, which otherwise usually occurs by or phone. Questions on any topic relating to the Agency s activities can be submitted before the conference to conferences@efgcp.eu, and will be answered by the Paediatric Medicines office at the EMA. Programme Committee Mark Turner Solange Rohou Dimitris Athanassiou Martine Dehlinger-Kremer Roberto de Lisa Mette Due Theilade Thomsen Sabine Fürst- Recktenwald Heidrun Hildebrand Thomas Severin Johannes Van den Anker Faculty Dimitris Athanassiou Daniel Brasseur Joerg Breitkreutz Christina Bucci-Rechtweg Jacqueline Carleer Martine Dehlinger-Kremer Roberto de Lisa Mette Due Theilade Thomsen Sabine Fürst- Recktenwald Heidrun Hildebrand Martin Graham Samuel D. Maldonado University of Liverpool, United Kingdom AstraZeneca, United Kingdom MDA Hellas & United Parent Projects Muscular Distrophy, Greece SynteractHCR, Germany & EUCROF, The Netherlands Shionogi, United Kingdom F. Hoffmann-La Roche, Switzerland Bayer, Germany Novartis, Switzerland Universitäts-Kinderspital beider Basel (UKBB), Switzerland, Children s National Health System, USA & Sophia Children s Hospital, The Netherlands MDA Hellas & United Parent Projects Muscular Distrophy, Greece Heinrich-Heine-University Dusseldorf, Germany Novartis Pharmaceuticals, USA Paediatric Committee (PDCO), & Federal Agency for Medicines and Health Products, Belgium SynteractHCR, Germany & EUCROF, The Netherlands Shionogi, United Kingdom F. Hoffmann-La Roche, Switzerland Bayer, Germany Kinderpharm LLC, United Kingdom Johnson & Johnson, USA Page 2 of 6

3 Dirk Mentzer Koenraad Norga EFGCP/DIA/EMA Annual Conference 2016 Cecile Ollivier Solange Rohou Mark Turner Thomas Severin Johannes Van den Anker Gilles Vassal Lynne Yao Conference Language The language of the conference will be English. Conference Venue European Medicines Agency Churchill Place 30, Canary Wharf London E14 5EU, United Kingdom Website: Registration & Information or visit Paediatric Committee (PDCO), & Paul Ehrlich Institut, Germany Paediatric Committee (PDCO), & Antwerp University Hospital (UZA), Belgium AstraZeneca, United Kingdom University of Liverpool, United Kingdom Novartis, Switzerland Universitäts-Kinderspital beider Basel (UKBB), Switzerland, Children s National Health System, USA & Sophia Children s Hospital, The Netherlands Gustave Roussy, France Food and Drug Administration (FDA), USA Page 3 of 6

4 Programme Monday 10 th October 08:00 Registration & Welcome Coffee 08:30 Welcome Solange Rohou, AstraZeneca, United Kingdom & Mark Turner, University of Liverpool, United Kingdom SESSION 1 LESSONS LEARNT FROM 10 YEARS OF PAEDIATRIC REGULATION Chairpersons: Martine Dehlinger-Kremer, SynteractHCR, Germany & EUCROF, The Netherlands & Speaker Invited 08:45 Key note given by Speaker Invited What have we learnt and how can those learnings inform the debate about the future of the Regulation as legislation and basis for practice 09:00 The Regulatory Perspective Dirk Mentzer, Paediatric Committee (PDCO), & Paul Ehrlich Institut, Germany 09:15 The Industry Perspective Samuel D. Maldonado, Johnson & Johnson, USA 09:30 The Patients Perspective Speaker Invited 09:45 The Academia Perspective Gilles Vassal, Gustave Roussy, France 10:40 Coffee Break 11:00 Parallel breakout sessions (BOS) BOS 1 BOS 2 BOS 3 BOS 4 BOS 5 BREAKOUT SESSIONS Extrapolation: Best practice from EU and US perspectives Chair: Cecile Ollivier, What About Neonates? Chair: Christina Bucci-Rechtweg, Novartis Pharmaceuticals, USA Adolescents and Young Adults How to Provide Best Care? Chair: Sabine Fürst- Recktenwald, F. Hoffmann-La Roche, Switzerland Cardiovascular Drug Development in Children Chair: Thomas Severin, Novartis, Switzerland The EFGCP Children Medicines Working Party and Ethical Issues Chair: Martine Dehlinger-Kremer, SynteractHCR, Germany Page 4 of 6

5 13:00 Lunch Chairpersons: Thomas Severin, Novartis, Switzerland and Mette Due Theilade Thomsen Shionogi, United Kingdom 14:00 Feedback from the 5 parallel breakout sessions Rapporteurs of the Groups 15:15 Update from EMA and selected questions from attendees EMA Representative 15:45 Coffee break SESSION 2 HOW CAN CHILDREN, CARERS AND FAMILIES CONTRIBUTE TO DRUG DEVELOPMENT? Chairpersons: Solange Rohou, AstraZeneca, United Kingdom & Mark Turner, University of Liverpool, United Kingdom 16:00 Panel Discussion 17:30 End of Day 1 Tuesday 11 th October 08:15 Welcome coffee SESSION 3 AN INTEGRATED APPROACH TO PAEDIATRIC DRUG DEVELOPMENT Chairpersons: Johannes Van den Anker, Universitäts-Kinderspital beider Basel (UKBB), Switzerland, Children s National Health System, USA & Sophia Children s Hospital, The Netherlands & Dimitris Athanassiou, MDA Hellas & United Parent Projects Muscular Distrophy, Greece 08:45 Best Practice at all Steps of Paediatric Drug Development Martin Graham, Kinderpharm LLC, United Kingdom 09:05 Pre-Clinical Studies: When and Why? Jacqueline Carleer, Paediatric Committee (PDCO), & Federal Agency for Medicines and Health Products, Belgium 09:25 Formulations are Important at all Stages of Drug Development Joerg Breitkreutz, Heinrich-Heine-University Dusseldorf, Germany 09:45 Exploratory Clinical: Novel Approaches to early Phase Studies Sabine Fürst- Recktenwald,F. Hoffmann-La Roche, Switzerland 10:05 Confirmatory Clinical: when are traditional Phase 3 Studies required and when are other Approaches Suitable? Koenraad Norga, Paediatric Committee (PDCO), & Antwerp University Hospital (UZA), Belgium Page 5 of 6

6 10:25 General Discussion 10:45 Coffee Break SESSION 4 UPDATE ON PROGRESS WITH ICH E11 Chairpersons: Dirk Mentzer, Paediatric Committee (PDCO), & Paul Ehrlich Institut, Germany & Sabine Fürst- Recktenwald, F. Hoffmann-La Roche, Switzerland 11:10 The EU Perspective Daniel Brasseur, 11:30 The US Perspective Lynne Yao, Food and Drug Administration (FDA), USA 11:50 The Industry Perspective Solange Rohou, AstraZeneca, United Kingdom 12:10 The Patient Perspective Speaker invited 12:30 Panel Discussion 13:00 Lunch SESSION 5 CLINICAL TRIAL FEASIBILITY Chairpersons: Roberto de Lisa, & Heidrun Hildebrand, Bayer, Germany 14:00 How can feasibility be enhanced? Roles of networks EnprEMA European Paediatric Clinical Trials Research Infrastructure Paediatric Trials Consortium Methods of Feasibility Assessment Page 6 of 6