EJP RD Rare Disease Training:“Training on strategies to foster solutions of undiagnosed rare disease cases”

EJPRD training

REGISTRATION IS NOW OPEN here ONLINE REGISTRATION until 14 January 2024. Registration will remain open for the reserve list only until 4 February 2024.

The International Course “Training on strategies to foster solutions of undiagnosed rare disease cases” 13-15 March 2024 is part of a series of training activities proposed by the EJP RD. The Course is made up of 3 days training organized by ISS, Istituto Superiore di Sanità, Rome, Italy. The course will be only in person.

Several initiatives have been undertaken at national and international level for undiagnosed rare diseases aimed at identifying clinical pathways [...]



XVI Foresight Training Course “Repurposing to cover unmet needs: the current scenario in Europe and the proposed changes to the Pharmaceutical Legislation”


Fondazione Gianni Benzi, that is organising the XVI Foresight Training Course Repurposing to cover unmet needs: the current scenario in Europe and the proposed changes to the Pharmaceutical Legislation, that will be held on 18 December 2023 in Bari, Italy and online.

The Course will deal with:

Current and proposed regulatory provisions and decision-making to be undertaken in the EU for Research & Development (R&D) and marketing authorisation; Industry and academic approaches and collaboration to repurposing as a R&D opportunity; Commercial perspectives and strategies to repositioning drugs; The meaning of repurposing in the paediatric context; Need for specific [...]



WP5 Educational Webinar 3:“Bridging the gap between promising preclinical data and a successful clinical trial”


In this Webinar we are focusing on the importance of translational research referred to “bench-to-bedside” by showcasing two successful models for bridging the gap between preclinical and clinical research.

Paediatric Oncologist Professor Gilles Vassal from Institute Gustave Roussy, distinguished member of the ERNPaedCan, will present the ACCELERATE, an international multistakeholder organization to improve and accelerate new drug development for children and adolescent with cancer.

Joanne Lee from Newcastle University will talk about ACT concept (Advisory Committees for Therapeutics) and introduce the ACT toolkit. She will also provide background about the TACT Neuromuscular model and talk about [...]



ERICA Webinar: REMEDI4ALL / drug repurposing and clinical trial readiness by Anton Ussi


ERICA WP4 Clinical Trial Support webinar

Monday October 2nd
From 13:00 to 14:00 CEST

Speaker: Anton Ussi

This webinar will discuss challenges associated with getting ready for and conducting clinical trials in drug repurposing, with a focus on differences between trial requirements for future on-label or off-label use. The discussion will also highlight key services and tools available to the community, as well as the REMEDi4ALL projects support offering.




WP5 Innovation workshop on drug repurposing in rare diseases


WP5 Translation and Innovation is organizing Innovation workshop focused on drug repurposing in rare diseases.

In the first part of the workshop, we will showcase success stories from two different ERNs as an inspiration for the community. Eduard J van Beers from ERN EuroBloodNet will give talk about mitapivat story and building relations with biomedical industry into investigator-initiated research programmes, and Nicola Specchio from ERN EpiCare will talk about drug repurposing in epilepsy.

In the second part of the workshop, expert Donald Lo from EATRIS (REMEDI4ALL project) will lead interactive session on [...]



ERICA WP3 ‘Patient centred research’ Expert Working Group

WP3 PCEWG 31.05.23

ERICA WP3Patient-Centred Research Patient Centered Expert Working Group (PCEWG) was held on the 31st of May between 3 pm and 6 pm (CET time).

The workshop’s objective was to present the updates on patient-centered work leads by WP3 During the workshop, WP3 presented the version 2.0 of the PROMs Repository, the approach to identify suitable PROMs for rare diseases with no specific PROMs (diseases functional impact clustering and concepts coding works) [...]



ERICA WP4 Webinar: Framework for Patient Engagement in Clinical Trials


ERICA WP4 Clinical Trial Support webinar

Wednesday May 24th
From 15:00 to 16:00 CEST


Virginie Hivert, EURORDIS- Rare Diseases Europe María Cavaller Bellaubi, EURORDIS- Rare Diseases Europe

During the webinar ‘Framework for Patient Engagement in Clinical Trials’ they will talk about ‘what is patient engagement’ and its good practices and patient engagement in the ERNs’.




ERICA WP4 Webinar: Introduction on Patient-Reported Outcomes and considerations before including them in a clinical trial. Study case from the ERN-EuroBloodNet


ERICA WP4 Clinical Trial Support webinar

Wednesday May 10th 2023

From 12:30 to 13:30 CEST

The main objective of the webinar will be to give an overview of Patient-Reported Outcomes Measures (PROMs) and Patient-Reported Experience Measures (PREMs) in clinical research.

Céline Desvignes-Gleizes from Mapi Research Trust will provide an introduction on PROMs and PREMs and accordingly, will address the following questions:

What are PROMs & PREMs? What do I need to know about PROMs & PREMs before including them in a clinical trial? At what point of the preparation of a clinical [...]



3rd ERICA General Assembly 6-7th July 2023 in Madrid, Spain

GA groupfoto

The ERICA 3rd General Assembly took place 6-7 July in Madrid as a face-to-face meeting.

Hosted by Servicio Madrileno de Salud- Hospital Universitario La Paz (SERMAS-HULP). Coordination office of ERN-TransplantChild.

The aim of the ERICA consortium, in which all 24 European Reference Networks (ERNs) take part, is to build on the strength of the individual ERNs and create a platform that integrates all ERNs research and innovation capacity.

The aim of the General Assembly (GA) is to inform all the beneficiaries about the progress of the planned and completed ERICA project activities in more detail and to raise more awareness [...]