EFPIA & EFGCP Multi-Stakeholder Workshop on Paediatric Unmet Medical Needs

Virtual Workshop

Description

Event Workshop
Type Virtual Event
Language English
Date(s) 28 & 30 Jun. 2021
Day(s) 2 Day(s)
Duration 6 hours/ day
    Objectives

  • Understand stakeholders’ perspectives and needs.
  • Identify criteria to help define paediatric unmet medical needs using four conditions as case examples, which represent all possible paediatric development options: Asthma, NASH (Non-Alcoholic Steato-Hepatitis), Duchenne Muscular Dystrophy and Haemophilia.
  • Inform on revision of the EU Paediatric and Orphan Regulations through workshop outputs.

    • Background Information

      This workshop is organised as a follow-up to the EFGCP-EFPIA Multi-Stakeholder workshop on Paediatric Unmet Medical Needs, co-chaired by Prof. Vassal and Prof. Giaquinto and held in Amsterdam in December 2019. See the PUMN WS 2019 Report

      The 2019 workshop which gathered participants from Academia, EMA (European Medicines Agency), PDCO (Paediatric Committee), European Commission (EC), US FDA, patients’ organisations and industry concluded that for the definition of UMNs, no one-size-fits-all approach can work and that approaches might need to be different in different areas of indications. Follow-up activities that were then planned, were put on hold until end 2020, mainly because of the COVID-19 pandemic.

      Paediatric Unmet Medical Needs (UMNs) is recognised a major concern in the management of diseases in children not only by the paediatric community but also by both the EC and the EMA which released a joint action plan in November 2018, subsequently updated in 2020.

      In 2020, important changes for the regulatory framework occurred, with the EC publishing major documents relating to the revision of the Paediatric and of the Orphan Regulation: the Staff Working Document on the Joint Evaluation of these two regulations and the Inception Impact Assessment. The revision of these two legislations is also one of the actions of the Pharmaceutical Strategy for Europe released in November 2020. As a result, legislative and/or non-legislative proposals to amend these two regulations are expected by the end of 2021/early 2022.

      In this context, and as a follow-up to the 2019 EFGCP-EFPIA workshop, a second workshop is now proposed with the objective through stakeholders’ perspectives and needs, to identify criteria to help define paediatric UMNs using four conditions as case examples: Asthma, NASH, Duchenne Muscular Dystrophy and Haemophilia. These conditions were selected as representing all possible paediatric development options. Moreover, workshop outputs are expected to also inform the revision of the European Paediatric and Orphan Regulations.


      Join the discussion and support us with your insight, experience and connections in this Initiative which is crucial for the future of our paediatric patients.

Programme

Monday 28 June 2021
12:45 Connection to virtual room
13:00 Opening remarks & Objectives of the workshop
Fabio d’Atri, European Commission, DG Santé, Belgium
Fawzi Benzaghou, Ipsen, EFPIA, USA
Martine Dehlinger-Kremer, EFGCP CMWP Chair, ICON Plc, Germany
13:30 Session 1 - Paediatric Unmet Medical Needs in Haemophilia Anja Schiel, EMA SAWP, NoMA, Norway
Amanda Bok, European Haemophilia Consortium, Belgium
Christoph Male, Medical University of Vienna, Austria
Declan Noone, European Haemophilia Consortium, Ireland
Günter Auerswald, Zentrum für Kinderheilkunde und Jugendmedizin, Germany
Janina Karres, EMA, The Netherlands
Karen Buckland, Norway Institute of Child Health, United Kingdom
Lynne Yao, FDA, USA
Niklas Hedberg, EunetHTA, TLV, Sweden
Rocio Fernandez Fresquet, EMA, The Netherlands
15:30 Break
15:45 Session 2 - Paediatric Unmet Medical Needs in Asthma Karl-Heinz Huemer, PDCO, Austria
Claudia Scharl, European Representation Austrian Federation of Social Assurance; MEDEV, Austria
Dobromir Penkov, EMA, The Netherlands
Louise Fleming, Royal Brompton Hospital, United Kingdom
Lynne Yao, FDA, USA
Marielle Pijnenburg, Erasmus University, The Netherlands
Mikaela Odemyr, EFA (EU Federation of Allergy and Airways Diseases Patients’ Associations) and ELF (EU Lung Foundation), Sweden
Niklas Hedberg, EunetHTA, TLV, Sweden
17:45 Day 1 Overall conclusion
Anja Schiel, EMA SAWP, NoMA, Norway
Karl-Heinz Huemer, PDCO, Austria
18:15 End of Day 1
Wednesday 30 June 2021
12:45 Connection to virtual room
13:00 Opening remarks
Jun Oh, University Medical Centre Hamburg-Eppendorf, Germany
13:15 Session 3 - Paediatric Unmet Medical Needs in NASH Gilles Vassal, Institut Gustave Roussy, France
Anja Schiel, EMA SAWP, NoMA, Norway
Chrissi Pallidis, EMA, The Netherlands
Franck Anania, FDA, USA
Henkjan Verkade, Univ. of Groningen, The Netherlands
Jun Oh, University Medical Centre Hamburg-Eppendorf, Germany
Marko Korenjak, President, European Liver Patients Association, Slovenia
Ulrich Baumann, Univ. Medical School Hannover, Germany
15:15 Break
15:30 Session 4 - Paediatric Unmet Medical Needs in Duchenne Muscular Dystrophy Carlo Giaquinto, University of Padova, Italy
Anja Schiel, EMA SAWP, NoMA, Norway
Dimitrios Athanasiou, World Duchenne Organisation, Greece
Michaela Guglieri, Newcastle University, United Kingdom
Nathalie Goemans, Leuven University, Belgium
Roberto de Lisa, EMA, The Netherlands
Sally Hofmeister, World Duchenne Organisation, Germany
Sylvie Benchetrit, ANSM, France
17:30 Break
17:40 Day 2 Overall conclusions
Carlo Giaquinto, University of Padova, Italy
Gilles Vassal, Institut Gustave Roussy, France
18:00 Panel session - Overall conclusion & Key messages to inform the revision of the regulation
18:45 End of the Workshop