EMA’s Public stakeholder meeting: development and authorisation of safe and effective COVID-19 vaccines in the EU

The European Medicine Agency (EMA) will organise a public meeting on 11 December 2020 to inform European citizens about the EU regulatory processes for the approval of COVID-19 vaccines and the Agency’s role in their development, evaluation, approval and safety monitoring. The COVID-19 pandemic is an unprecedented global crisis, with devastating health, social and economic Read more about EMA’s Public stakeholder meeting: development and authorisation of safe and effective COVID-19 vaccines in the EU[…]

2nd TransplantChild workshop : Novel strategies in paediatric transplantation

The European Research Network TRANSPLANT-CHILD is organising the  2nd TransplantChild workshop on translational research: “Novel strategies in paediatric transplantation” which will take place on November 30th, 2020 online. The main aim of the course is addressing training in paediatric transplantation according to the holistic approach of the network, providing skills to perform a common and Read more about 2nd TransplantChild workshop : Novel strategies in paediatric transplantation[…]

Survey on administration devices for children’s care

We are pleased to share with you the initiatives dedicated to paediatric patients and families that the European Paediatric Translational Research Infrastructure (EPTRI) has launched in collaboration with European Paediatric Formulation Initiative (EuPFI) to help finding ways of making children’s care devices more user-friendly. The survey questions have been reviewed by the young members of the Italian, Albanian and Spanish Read more about Survey on administration devices for children’s care[…]

The Economist Intelligence Unit report on rare neurological diseases challenges for policy makers, patients and healthcare professionals

EJP RD member and Coordinator of the European Reference Network for Rare Neurological Diseases (ERN-RND) Holm Graessner , alongside with Mary Kearney from the Irish College of General Practitioners and Primary Care Physician and  Antonio Federico, Professor of Neurology and Director Unit Clinical Neurology and Neurometabolic Diseases in the University of Siena were involved in the production of the Economist Intelligence Unit’s report entitled “Navigating rare neurological diseases: meeting the Read more about The Economist Intelligence Unit report on rare neurological diseases challenges for policy makers, patients and healthcare professionals[…]

Remembering Prof. Dr. Gert Jan van Ommen

It is with great sadness that we’ve learned Prof. Dr. Gert Jan van Ommen passed away. EJP RD honors his creative scientific mind as well as his huge contribution and leadership of the Human Genetics department at Leiden University Medical Centre. We send our deepest condolences to his family, friends and collaborators in this difficult Read more about Remembering Prof. Dr. Gert Jan van Ommen[…]

RE(ACT) Congress and IRDiRC Conference joint online event

EJP RD is glad to be a part of the organisation of the RE(ACT) Congress and IRDiRC Conference joint online event that will take place on 13-15 January 2021. It aims to bring together scientific leaders, patients, and policy makers to advance research on rare diseases. Patients and patient organisations will share their perspectives and experiences.  To obtain your discount, register here before 31st of December.   Read more about RE(ACT) Congress and IRDiRC Conference joint online event[…]

The European Commission jointly evaluates the Orphan and Paediatric Regulations

The European Pharmaceutical Strategy was recently released and it includes strengths and weaknesses of the legal instruments and the European Commission is preparing the review of the legislation on medicines for rare diseases and children: the Orphan and Paediatric Regulations. The EFFECTIVENESS of both regulation on the R&D of new medicines for these patients and their timely availability and coverage of unmet needs, the EFFICIENCY in improving their Read more about The European Commission jointly evaluates the Orphan and Paediatric Regulations[…]

Rare Diseases Cures Accelerator – Data and Analytics Platform (RDCA-DAP) 2020 Virtual Workshop

To showcase the importance of data sharing in rare disease drug development and to highlight the first year milestones, the Critical Path Institute (C-Path) and the National Organization for Rare Disorders (NORD®) hosted the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) 2020 Virtual Workshop on Monday, October 19th. RDCA-DAP is a platform that serves Read more about Rare Diseases Cures Accelerator – Data and Analytics Platform (RDCA-DAP) 2020 Virtual Workshop[…]

EMA’s Workshop on Support for Orphan Medicines Development

EJP RD will be presenting at The European Medicines Agency’s Workshop on Support for Orphan Medicines Development which will take place via broadcast on 30 November 2020. The goal of this event is to emphasise on an early medicine development and regulatory interactions.  Existing tools such as orphan designation, protocol assistance and PRIME scheme will be Read more about EMA’s Workshop on Support for Orphan Medicines Development[…]