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

This multi-stakeholder workshop will be the opportunity to define mutually acceptable criteria for joint and informed decision making on paediatric unmet medical needs.

Description

Event Workshop
Type Face To Face
Language English
Date(s) 12 Dec. 2019
Day(s) 1 Day(s)
Duration 8 hours/ day

As a result of the European Commission’s assessment of 10 years of experience with the Paediatric Regulation Paediatric Unmet Medical Needs have been recognized by all concerned including the paediatric community, as a major concern in the management of diseases in children. This is why the topic was included by the European Commission and the European Medicines Agency in an action plan released in October 2019.

Unmet medical needs are perceived differently by diverse stakeholders. Therefore, to better define them a common understanding is needed with the input of all stakeholders including academics, industry, parents/patients’ associations, HTA bodies, regulators, and the EU Commission, to agree on a definition and on perceived areas of needs, and to identify areas with or without medical need.

To initiate a process of solution finding, a roadmap has been identified with as a first step, a Workshop on PAEDIATRIC UNMET MEDICAL NEEDS. The workshop is jointly organised by the European Forum for Good Clinical Practice’s (EFGCP) Children’s Medicines Working Party and the European Federation of Pharmaceutical Industry and Associations (EFPIA) in Amsterdam on December 12, 2019.

The workshop will be the opportunity to exchange opinions and develop mutually acceptable criteria for joint and informed decision making on paediatric medical needs by all stakeholders including academics, industry, parents/patients’ associations, HTA bodies, regulators, and the EU Commission.

Prof. Gilles Vassal (Institut Gustave-Roussy, France) and Prof. Carlo Giaquinto (University of Padova, Italy) will co-chair this workshop that will be attended by academic experts, patient representatives, industry sponsors, EU regulators (EMA & PDCO), as well as Fabio d'Atri (EU Commission) and an FDA representative.

Ahead of the workshop a questionnaire will be sent to academia experts. Answers to the questionnaire will help identifying therapeutic areas to be discussed at the workshop as well as acceptable unmet medical criteria/algorithm.

This workshop will be the first step of this roadmap initiative, followed in 2020 by disease-specific meetings, similar to those set up by Prof. Vassal through the ACCELERATE platform. These disease-specific meetings will help drawing final conclusions and recommendations to be presented and discussed at a follow-up workshop at the end of 2020.

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

Thursday 12 December 2019
09:00 Welcome coffee
Opening remarks and objectives of the meeting
09:15 Introduction
Gilles Vassal, Institut Gustave Roussy, France
Stakeholders’ perception of Paediatric UMN and expectations Chrissi Pallidis, EMA, The Netherlands Gilles Vassal, Institut Gustave Roussy, France
09:25 Academia point of view
Carlo Giaquinto, University of Padova, Italy
Regulators from Europe and US
Koenraad Norga, EMA PDCO, Universitair Ziekenhuis Antwerpen, Belgium
Lynne Yao, FDA, USA
EU Commission
Fabio d’Atri, European Commission, DG Santé, Belgium
Parents/Patients representatives point of view
Dimitrios Athanasiou, United Parent Projects Muscular Dystrophy (UPPMD) & MDA HELLAS, Greece
Industry point of view
Solange Corriol-Rohou, AstraZeneca, France
10:45 Coffee break
How to identify paediatric UMN? Gilles Vassal, Institut Gustave Roussy, France Koenraad Norga, EMA PDCO, Universitair Ziekenhuis Antwerpen, Belgium
11.00 Setting the scene & proposals
Karl-Heinz Huemer, EMA PDCO, Austrian Medicines and Medical Devices Agency, Austria
Lucia d’Apote, Amgen, Italy
Marie-Yvonne Douste-Blazy, Servier, France
Proposed methodology - A multi-stakeholder panel discussion
Q&A on methodology & Conclusions of the morning sessions
Fabio d’Atri, European Commission, DG Santé, Belgium
12:20 Conclusions from the Morning Sessions
Carlo Giaquinto, University of Padova, Italy
Gilles Vassal, Institut Gustave Roussy, France
12:30 Lunch
Paediatric Unmet Medical Needs in Selected Therapeutic Areas
14:00 Introduction
Carlo Giaquinto, University of Padova, Italy
Gilles Vassal, Institut Gustave Roussy, France
14:15 Neuropsychiatry in paediatrics
Alessandro Zuddas, Università degli Studi de Cagliari, Italy
15:00 Respiratory diseases in paediatrics
Jonathan Grigg, Queen Mary University of London, United Kingdom
15:45 Oncology in paediatrics
Asimo Koukougianni, Karkinaki NGO, Greece
Gilles Vassal, Institut Gustave Roussy, France
16:25 Future therapeutic areas: Neonatology
Jun Oh, University Medical Center Hamburg, Germany
Mark Turner, University of Liverpool, United Kingdom
17:00 Conclusions of the afternoon session & wrap-up