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EFGCP & DIA Better Medicines for Children Conference
The future of paediatric medicines: putting patients at the heart of innovation and regulatory science
![]() | 12 & 13 October 2020 | Add to calendar | |||||||
![]() | Virtual Conference | See on map |
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Description
This year, in 2020, 13 years after the EU Paediatric Regulation came into force, our aim is to build on achievements and lessons learnt so far and to look at the future of paediatric drug development, with a specific focus on putting patients at the heart of innovation and regulatory science.
The opening panel session moderated by Fabio d'Atri (DG SANTE, European Commission) and involving key speakers representing EMA, FDA, National Authorities, Healthcare Professionals, and Industry, will be the opportunity to share updates in the field and discuss the future of the development of paediatric medicines: what to do more to be better at delivering innovations to children in needs? How the EC/EMA action plan has been progressed?
To give them even more attention, in a separate panel session, the Young Patients, will tell us what the future of paediatric drug development means and looks like for them.
Active engagement of conference participants is expected to discuss challenges and identify solutions on what could be done to optimise children’s access to new medicines:
- What data sources and digital technologies could bring to paediatric development on top of the use of extrapolation?
- How to best define children unmet medical needs?
- How to optimise the PIP process and regulatory approval
A fireside chat with PDCO moderated by Elin Haf Davies (Aparito) and involving the PDCO chair & vice-chair, will give the audience the opportunity not only to understand PDCO's specific activities and priorities but also to further address clarifying questions to PDCO members.
The value of multi-stakeholder collaborations has been shown, since an opportunity to address important scientific gaps, but what is ongoing to foster paediatric development?
The conference will also explore what drug development means for Paediatricians, and in particular during COVID-19 pandemic.
Day 1 - 12 October
Day 2 - 13 October
12:00 | Opening of the Conference |
12:30 | Welcome & introduction to the conference
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12:45 | Plenary Session 1: Optimising the implementation of the Paediatric Regulation The European Commission’s (EC) report released in 2018 identified a number of areas where short-term actions could address the identified shortcomings under the current legal framework. In order to follow up on the report’s conclusions the EC and the European Medicines Agency (EMA) have developed a detailed plan to boost the development of medicines for children in Europe, in consultation with all relevant stakeholders. The action items focused on 5 topic areas:
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▶ | 12:45 | Keynote Where has progress been made and what remains to be discussed?
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▶ | 13:00 | Panel Discussion Panellists will outline their viewpoints on their specific priorities as well as their contribution to address and complete the EC-EMA actions, where appropriate and discuss the future of paediatric drug development.
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▶ | 13:50 | Q&A Session |
14:10 | Plenary Session 2: Voice of Young Patients including Q&A |
▶ | What does the future of paediatric drug development mean and look like for Young patients?
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14:40 | Break Out Sessions 3 breakout sessions to discuss topics in more detail with participants in a workshop fashion.
Breakout session agenda:
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▶ | 14:40 | Introduction to the breakout sessions
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▶ | 14:50 | Break |
▶ | 15:10 | Breakout Session 1: Extrapolation / RWD in drug development A new ICH guideline is currently under development (ICH E11A) to recommend international standards for, and promote harmonisation of, the use of paediatric extrapolation to support the development and authorization of paediatric medicines. Meanwhile, the value and importance of health-related data derived in real life settings or Real-world data are increasing significantly. RWD can be medical health records, registries, biobanks, administrative data, health surveys, observational studies, health insurance data, data generated from mobile applications etc. The EMA Reflection Paper specifies that data sources other than clinical trials can be used in addressing uncertainties in paediatric development. Additionally, the Reflection Paper mentions particularly the use of registries in the context of extrapolation, also important in the context of Real-World Evidence.
This session is the opportunity to update on the ICH activities and will focus on how leveraging the EMA extrapolation framework as a regulatory tool to generate RWD of appropriate quality can be done and give examples on application of RWD in paediatrics.
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▶ | 15:10 | Breakout Session 2: Identifying paediatric unmet needs In December 2019, the European Federation of Pharmaceutical Industries and Associations (EFPIA) partnered with EFGCP to organise a multi-stakeholder workshop to explore how to best define and tackle unmet medical needs (UMN) in children, in order to provide a basis for strategic decision making on paediatric medicine development. The workshop objective was to find a broadly accepted approach to defining UMN among paediatric patients, in order to make better and safer medicines for children, and to make sure they are available and affordable. The outcome of the workshop was an outline of potential guiding principles for defining UMN in children, and a programme for testing the principles in selected therapeutic areas over the course of 2020.
This break out session is the opportunity to keep the momentum and gather additional insights that will feed into the generation and subsequent agreement on a more widely acceptable framework by the end of 2021.
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▶ | 15:10 | Breakout Session 3: Regulatory overview: Optimising PIP processes and regulatory approval (PDCO & CHMP) This breakout session will facilitate interactive exchange with the session attendees to generate ideas for better integrating regulatory discussions around PIPs within the overall regulatory context. The session will focus on the regulatory discussions related to paediatrics within a broader life-cycle approach and how PDCO and CHMP are collaborating to improve regulatory outcomes for paediatric medicines.
The session’s goal is to identify what is needed to ensure that the PIP process is efficient and agile, for generating new authorisations for paediatric medicines.
Participants are encouraged to share practical case study experience that will shine a light on best practice examples and hurdles faced when implementing agreed PIP programs and submitting paediatric data for regulatory approval.
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▶ | 17:00 | Break |
17:10 | Plenary Session 3: Fireside Chat |
▶ | Panel Discussion Questions to be submitted ahead of the conference
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18:10 | Plenary Session 4: “What Drug Development means for Paediatricians”
Impact COVID-19 pandemic on paediatrics? This session aims to highlight, following the agreement of a PIP, what drug development means to clinicians and how to balance clinical care with delivering drug research. The session will outline the experience of hospital teams in delivering studies, including those smaller sites that do not have access to dedicated research facilities. Along these lines, the session will also ask whether current PIPs address the day to day needs of children and young people. |
▶ | 18:10 | Discussion & Moderation
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▶ | 18:10 | 20 Minutes session What happens at regional paediatric hospitals when trying to deliver paediatric drug research and top-level clinical care, by presenting the experience of a research operations manager, delivering research during the COVID 19 pandemic as a case study and lessons learned for the future
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▶ | 18:30 | 20 Minutes Session What does drug development research mean to a paediatric clinician? This session will include the experiences of a paediatric specialist who is delivering research alongside a clinical service. The session will include an overview of how they balance the competing priorities of research and clinical commitments and why it is important to consider these parallel activities when designing future research
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▶ | 18:50 | Time for Questions |
▶ | 18:55 | Break |
19:00 | Special Event : with Mark Turner – Senior Lecturer, University of Liverpool Presentation on the last updates on c4c |
▶ | Panel Discussion
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19:30 | Wrap up & day closing
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10:30 | Debrief from Day one and introduction to Day 2
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10:45 | Plenary Session 5: Panel session on Patient Access Despite the clear benefits of the EU Paediatric Regulation towards increased licensed paediatric treatments, even when a new medicinal product is approved for use in children, this does not necessarily result in children access to it. Optimising availability and access to medicines that would greatly benefit children, would improve consistently the tangible effect of the Paediatric Regulation.
This is an important issue which requires consideration from all stakeholders in order to make appropriately studied and authorised medicines available to children. Actual availability and accessibility depend on further arrangements for placing on the market, reimbursement and sufficient pricing, which have to be agreed in each Member State.
For this session we will bring together HTAs, payers, patients and industry representatives for an open discussion of the actions needed. |
▶ | Panel Discussion
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12:15 | Plenary Session 6: Session Enpr-EMA: CT preparedness / International collaboration WG |
▶ | Panel Discussion
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12:35 | Plenary Session 7: Debrief, Conclusions and Discussions - Breakout Sessions |
▶ | 12:35 | Debrief & Conclusions from the Breakout sessions |
▶ | 12:55 | Discussion & Questions |
13:05 | Plenary Session 8: The Go- FAIR Initiative / Use Data |
▶ | Panel Discussion
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13:35 | Plenary Session 9: The FAIR (Fostering Age Inclusive Research) Trials Initiative – Inclusion of Adolescent in Adult trials |
▶ | Panel Discussion
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14:20 | Conclusions & Farewell
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▶ | All the Faculty |
14:25 | End of the Conference |
Committee | |
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Angelika Joos | Merck Sharp & Dohme (Europe) Inc. |
Angeliki Siapkara | Medicines and Healthcare products Regulatory Agency (MHRA) |
Begonya Nafria Escalera | Sant Joan de Déu Children's Hospital |
Dimitrios Athanasiou | EMA PDCO |
Elin Haf Davies | Aparito |
Katie Rizvi | Youth Cancer Europe Fundation |
Koenraad Norga | EMA PDCO, Universitair Ziekenhuis Antwerpen |
Martine Dehlinger-Kremer | EUCROF, PRA Health Sciences, PRA Health Sciences – Mannheim |
Rhian Thomas-Turner | NHS United Kingdom |
Sabine Fürst-Recktenwald | F.Hoffmann-La Roche AG, Roche |
Solange Corriol-Rohou | AstraZeneca |
Thomas Severin | Novartis |
Faculty | |
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Andrew Thompson | EMA |
Angelika Joos | Merck Sharp & Dohme (Europe) Inc. |
Angeliki Siapkara | Medicines and Healthcare products Regulatory Agency (MHRA) |
Anja Schiel | Norwegian Medicines Agency (NOMA) & EMA SAWP Chair) |
Ann Marie Janson Lang | CFTG, Swedish MPA |
Anna Sherriffs | ScotCRN |
Authority Representative | |
Begonya Nafria Escalera | Sant Joan de Déu Children's Hospital |
Cecile Ollivier | Aparito |
Christina Bucci-Rechtweg | Novartis |
Dehlinger-Kremer Martine | EFGCP, EUCROF, PRA Health Sciences |
Dimitrios Athanasiou | EMA PDCO |
Elin Haf Davies | Aparito |
Elke Stahl | CTFG, BfArM |
Evert Jan van Lente | Payer Representative - AOK-Bundesverband |
Fabio d'Atri | European Commission, DG SANTE |
Femida Gwadry-Sridhar | Pulse Infoframe |
Gesine Bejeuhr | Bayer AG |
Gilles Vassal | Institut Gustave Roussy |
Ivett Jakab | EPF's Youth Representative |
James Ryan | Industry Representative - AstraZeneca |
Karl-Heinz Huemer | EMA PDCO |
Katie Rizvi | Youth Cancer Europe Fundation |
Koenraad Norga | EMA PDCO |
Lynne Yao | FDA |
Marie-Yvonne Douste-Blazy | Paediatric Expert |
Marin Daniel | Young Advocate of Kids Barcelona, Sant Joan de Déu Children's Hospital |
Mark Turner | University of Liverpool |
Martine Dehlinger-Kremer | EUCROF, PRA Health Sciences, PRA Health Sciences – Mannheim |
National Cancer Research Institute, Barcelona | Max Williamson |
Nawel van Lin | Duchenne Data Foundation |
Niklas Hedberg | HTA Representative - EunetHTA |
Peter-Bram 't Hoen | Radboud UMC |
Pirkko Lepola | Enpr-EMA & FinPedMed |
Rhian Thomas-Turner | NHS United Kingdom |
Rime Allouche | Kids France |
Rob Hemmings | Concilium |
Robert Shaddy | Pediatrician-in-Chief, Children's Hospital Los Angeles |
Sabine Fürst-Recktenwald | Roche |
Sabine Scherer | EMA PDCO |
Solange Corriol-Rohou | AstraZeneca |
Thomas Severin | Novartis |
Zinedine Metenani | UCB |
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European Forum for Good Clinical Practice
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