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

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.