The European Conference on Rare Diseases & Orphan Products (ECRD) is the event that allows the dialogue and collaboration of over 1500 stakeholders in the rare disease community. The 12th edition coordinated by EURORDIS and co-organized by Orphanet will be celebrated next summer, from 15th to 16th May 2024.
The ECRD is the largest, patient-led, rare disease policy-shaping event held in Europe.
By bringing together people with rare diseases and patient advocates, policy makers, healthcare industry representatives, clinicians, regulators and Member State representatives, EURORDIS harnesses [...]
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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 [...]
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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 [...]
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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 [...]
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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.
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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 [...]
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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) [...]
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ERICA WP4 Clinical Trial Support webinar
Wednesday May 24th
From 15:00 to 16:00 CEST
Speakers
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’.
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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 [...]
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