EMA/ACT EU Training: Transitioning trials to the CTR (CTIS) for non-commercial sponsors

ACT EU Training

ACT EU Training for non-commercial sponsors: Transitioning trials to CTIS

9 February 2024 10:00-13:00 CET

Location: Online/ European Medicines Agency, Amsterdam, the Netherlands, Live Broadcast

Event summary

This training event aims to support non-commercial sponsors of clinical trials in transitioning their clinical trials that are expected to continue after 30 January 2025 from the Clinical Trials Directive to the EU Clinical Trials Regulation (Regulation (EU) No 536/2014). Moreover, the event will provide useful training in preparing new clinical trials following the Clinical Trial Regulation (CTR).

From 31 January 2025 onwards only the CTR and its Delegated Acts will [...]


ERICA WP5 Catalogue of Services updated!

To help the ERN community in understanding the process of translation and to support their collective ability to develop knowledge into patient benefit, a catalogue of current translational research services available to the rare diseases research community has been developed.

Now the latest version 23.04.2024 is available: Catalogue of Services


EMA Webinar on Rare World Diseases Day

EMA RD Day webinar

Orphan Medicines Development – ask the European regulator

Thursday, 29 February 2024 Virtual meeting via WebEx / EMA, Amsterdam

The EMA’s Orphan Medicines Office is hosting an interactive webinar on Rare Disease Day on 29 February 2024, to answer questions that developers of products for rare diseases or researchers in this field might have on orphan designations and rare disease development. The webinar will feature:

short presentations on the background of orphan designation and the benefits it has brought to patients; a live question-and-answer session enabling participants to ask their questions on orphan medicines development to a panel of regulatory [...]


Joint HMA/EMA Multi-stakeholder workshop on Patient Registries 12&13 February 2024


The Joint Heads of Medicines Agencies (HMA)/European Medicines Agency (EMA) Multistakeholder workshop on Patient Registries will bring together representatives of registry holders, regulatory agencies, pharmaceutical companies, patients, healthcare professionals, academia, and health technology assessment bodies to address the following objectives:

Day 1 – 12 February afternoon: Discuss the EMA qualification procedure for patient registries with the aim to clarify the benefits, identify current limitations, and propose measures to optimise the process; Day 2 – 13 February all day: Establish the value and enable the use of patient registries for regulatory decision-making by considering contexts of use for which registry [...]


12th edition of the European Conference on Rare Diseases and Orphan Products (ECRD)


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 [...]


1st REMEDi4ALL International Drug Repurposing Conference 6-7 March, 2024 in Barcelona


Join the first international drug repurposing conference “Bridging Boundaries: Innovating, Connecting & Reshaping Drug Repurposing”, #iDR24, co-organised by REMEDi4ALL ,Beacon and MeRIT on 6-7 March 2024 in Barcelona. REMEDi4ALL is Horizon Europe-funded project which aims at building a sustainable drug repurposing platform in Europe with an approach of co-creation amongst researchers, clinicians and patients.

In this global event, key opinion leaders from both the research and patient communities, funders, regulators and representatives from the private sector will participate to collectively advance innovative drug repurposing in Europe and beyond.

Among others rare disease patient organisation EURORDIS is hosting [...]


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 [...]


Presentations ERICA & EJP RD Joint Conference available!

Groepsfoto-Endocrinologie- november 2023 -4-k

The European Rare Disease Research Coordination and Support Action- ERICA and European Joint Programme of Rare Diseases (EJP RD)

Joint Conference took place 21st November 2023 in Amsterdam University Medical Centre.

Main topics:

Examples of Synergies between both projects (ERICA & EJP RD) Collaboration of ERNs and non-ERNs researchers Preparing the path to the European Rare Diseases Research Alliance (ERDERA)

View the full programme (, 172 KB).

Overview of all Presentations ERICA & EJP RD Joint Conference.


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 [...]