NEWS

Congrats to our coordinator Dr. Daria Julkowska for the EURORDIS Black Pearl 2020 – European Rare Disease Leadership Award

Congratulations to our coordinator, Dr. Daria Julkowska, from the EJP RD community, we are very happy for you! Dr. Julkowska won the EURORDIS Black Pearl 2020 – European Rare Disease Leadership Award for her “outstanding leadership and dedication  shown to the rare disease community and the positive impact she has had on rare disease research Read more about Congrats to our coordinator Dr. Daria Julkowska for the EURORDIS Black Pearl 2020 – European Rare Disease Leadership Award[…]

How will pharmacovigilance look in 2030?

A key article from Guido Rasi, EMA’s Executive Director, Sabine Straus, the chair of EMA’s safety committee (PRAC) and Peter Arlett, the Agency’s Head of Pharmacovigilance and Epidemiology, published in Clinical Pharmacology and Therapeutics: Pharmacovigilance 2030. In 2030, regulators will dedicate significantly more time to engaging with patients and healthcare professionals and thus ensuring that the Read more about How will pharmacovigilance look in 2030?[…]

From Lab to Patient

The journey of a medicine assessed by EMA The European Medicines Agency has just published an interactive timeline describing the journey of a medicine for human use authorised through EMA, from initial research to discussions on patient access to medicines across the EU. This tool is based on a booklet published earlier this year, and allows the user to Read more about From Lab to Patient[…]

Paediatric ERNs Crosslinks: The story of Íñigo

This is a video recently published by ERN TransplantChild to promote the collaboration between pediatric European Reference Networks (ERNs). Crosstalk among ERNs is an important task that could help improving healthcare for rare diseases. Therefore, it is necessary that the networks start collaborating and working together in their overlapping areas. We can “work together for Read more about Paediatric ERNs Crosslinks: The story of Íñigo[…]

Real-world evidence to support HTA/payer decisions about highly innovative technologies

RWE multi-stakeholder initiative – Paper for consultation The draft stakeholder guidance of the multi-stakeholder initiative on ‘Real-world evidence (RWE) to support HTA/payer decisions about highly innovative technologies’ is now open for consultation until 6 January 2020. Commissioned by the Belgian healthcare payer INAMI/RIZIV, this work defines how RWE can be best used to close evidentiary gaps for HTA Read more about Real-world evidence to support HTA/payer decisions about highly innovative technologies[…]

Training: Hands-On introduction to NGS variant analysis

This training gives an introduction to the use of several popular NGS analysis software packages.  Target Audience: All scientists When. 20 – 21 January 2020 (10.00 – 17.00) Objectives Using a full publicly available chromosome read-set from one of the 1000 genome samples: ​Perform a complete analysis workflow including QC, read mapping, read coverage analysis, and Read more about Training: Hands-On introduction to NGS variant analysis[…]

European survey on national training activities in clinical research

An interesting publication based on a survey in 11 ECRIN countries, finds significant investment in training in clinical research in individual countries but need for cross-network exchange. Abstract. Investigator-initiated clinical studies (IITs) are crucial to generate reliable evidence that answers questions of day-to-day clinical practice. Many challenges make IITs a complex endeavour, for example, IITs Read more about European survey on national training activities in clinical research[…]

ICPerMed Recognition 2020

Rome (Italy), 6 November 2019 – The International Consortium for Personalised Medicine (ICPerMed) has published the announcement of their third “Best Practice in Personalised Medicine” Recognition 2020. The objective of this recognition is to honour, encourage, promote and disseminate outstanding best practice examples in personalised medicine. Responsible authors (applicants from any country) of candidate proposals Read more about ICPerMed Recognition 2020[…]

“Yakup’s Journey to Hope” video in collaboration with EJP RD

Yakup’s Journey to Hope was created in collaboration with EJP RD and GA4GH Driver Project. Follow the story of Yakup as he embarks on a journey to find a diagnosis for his neurogenetic rare disease condition. At GA4GH Plenary 2019, Dr. Sergi Beltran of EJP RD shares how the driver project has helped one patient Read more about “Yakup’s Journey to Hope” video in collaboration with EJP RD[…]