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Activities  >  Events and Activities  >   EFPIA & EFGCP Multi-Stakeholder Workshop on Paediatric Unmet Medical Needs
Workshop
EFPIA & EFGCP Multi-Stakeholder Workshop on Paediatric Unmet Medical Needs
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.
28 & 30 JUNE 2021 Add to calendar
Virtual Conference See on map
DescriptionProgrammeFacultyPractical Information
Description

    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.

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