ERICA 2nd General Assembly 20-22 June 2022, Bologna, Italy

The ERICA 2nd General Assembly took place in Bologna as a hybrid meeting.

Hosted by Istituto Ortopedico Rizzoli (IOR) and ERN BOND Coordinator Luca Sangiorgi.

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.

This was a great opportunity to discuss the progress and future of the ERN related Research activities and to participate in the WP-Specific Expert Working Group sessions to exchange the success stories and to brainstorm with the [...]

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Rare Disease Clinical Trial Webinar: Composite endpoints including patient relevant endpoints (Quality of Life)

When assessing the efficacy of a treatment in any Clinical Trial, it is recommended by the International Council for Harmonisation to select a single meaningful endpoint. However, a single endpoint is often not sufficient to reflect the full clinical benefit of a treatment in multifaceted diseases, which is often the case in rare diseases. Therefore, the use of a combination of several clinical meaningful endpoints is preferred. Combining endpoints on a test statistics level or on the level of p-values, in general, ignores the correlation between the endpoints, while combining information on the subject level in composite endpoints does [...]

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ERN Workshop Modelling & Simulation: Research Methodologies for Small Populations in Rare Diseases

In the context of EJP RD’s ERN Workshops, a face-to-face workshop entitled “Modelling & Simulation: Research Methodologies for Small Populations in Rare Diseases” aimed at facilitating discussion and exchange of knowledge on the M&S methodologies and strategies as innovative and promising enough for facing complex multifactorial or rare diseases and conditions that require highly specialised treatments and resources is being organised by Donato Bonifazi of the Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF).

The in-person event will take place over two days on July 4th – 5th at the Hotel Excelsior in Bari, Italy.

The workshop is open by [...]

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ERN Workshop Functional exploration of genetic variants in cardiac diseases

In the context of EJP RD’s ERN Workshops, a face-to-face workshop entitled “Functional exploration of genetic variants in cardiac diseases” aimed at presentation of functional explorations of variants of interest identified using Drosophila, C. elegans, Zebrafish and iPSc models to understand the molecular bases of genetic diseases, in particular heart and muscle diseases, is being organised by Philippe Chevalier of the Hospices Civils de Lyon.

The in-person event will take place over two days on June 14th – 15th at the Hospices Civils de Lyon in Lyon, France.

The workshop is open by prior registration and selection to cardiologists, [...]

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Explore the Rare Diseases Clinical Trials Toolbox

Drug development programmes in rare diseases have many challenges, some of which differ from those facing researchers working on common diseases, like the lack of clinical research experts and the scarcity of patients. Moreover, academic sponsors and investigators end up getting involved not only in the scientific aspects of the research but also having to navigate the operational coordination and management themselves. The Rare Diseases Clinical Trials Toolbox has been developed as a practical aid for developers of clinical trials on medicinal products for human use regardless of therapeutic area.

The toolbox aims to collect the accumulated knowledge, experience, and [...]

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EJPRD- ERICA 3rd Joint Workshop “Finding RD registry data in the Virtual Platform”

This workshop is the third of the ERN data strategy workshop series. It is dedicated to the various stakeholders expected to interact with the EJP RD Virtual Platform.

In this workshop, the feedback received from the ERN registries on the level of discovery that should be authorised for either anonymous or authenticated users of the Virtual Platform will be discussed first. The practical use of the VP will be presented in a Demo session. Subsequently, a hands-on session will allow participants to test the Virtual Platform and provide their feedback. Researchers as well as stakeholders from industry, the member states [...]

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ERICA WP3 ‘Patient centred research’ Expert Working Group Kick-off Meeting 8-10 March 2-5 pm

We are glad to start the new ‘ERICA year’ with the Kick-off meeting of WP3 Patient Centred Expert Working Group (PCEWG) that will be held in two consecutive sessions on Tuesday 8th and Thursday 10th March 2022 from 2 to 5 pm (CET).

Session I will be dedicated to present the work done so far for the identification and codification of instruments for the assessment of Patient Reported Outcomes (PRO) in rare diseases (RD). Session I will give the frame for an open discussion in Session II to better understand and define the RDs community needs in terms of building [...]

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EMA Virtual meeting Repurposing ‘WALK-IN’ CLINIC

The pilot project to support the repurposing of medicines was launched at the end of October 2021 by EMA and the Heads of Medicines Agencies (HMA)

To support not-for-profit organisations and academia ahead of the upcoming deadline for submission (28th February 2022), EMA is hosting a ‘walk-in’ clinic webinar on 17 February 17h-18h30. Representatives of the Repurposing Observatory Group leading the pilot will also participate to the webinar.

EMA together with representatives of the Repurposing Observatory Group (RepOG) is organising this open event to:

provide a brief reminder of the Repurposing pilot provide information on how EMA real-world evidence [...]

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EJPRD- ERICA Joint Workshop “Finding RD registry data in the Virtual Platform”

A hands-on session will be held for you (as Virtual Platform (VP) end-users) to test the first version of the VP, provide feedback and address any further open questions.

This workshop is the second of the ERN data strategy workshop series. In this workshop, different approaches to making data and cases safely findable via the EJP RD Virtual Platform (VP) will be described, along with a short technical overview of how this is achieved.

This includes an elaboration of the difference between Resource Discovery (incorporating completely safe querying of very limited/obfuscated data fields) and far deeper Record Interrogation queries by [...]

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EMA Webinar for SMEs and Academia on the Clinical Trials Regulation and the Clinical Trials Information System

The way clinical trials are conducted in the EU will change when the Clinical Trials Regulation (Regulation (EU) No 536/2014) becomes applicable and the Clinical Trials Information System (CTIS) goes live on 31 January 2022.
The EMA is organising a webinar to remind SMEs and academic sponsors of clinical trials of the main changes brought by the Regulation and its impact on their trials-related activities.
The aim of the training is to give an overview of the Regulation and introduce the new process for submitting clinical trials information in the EU/EEA. In addition, it covers the functionalities of [...]

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