“Ethics and regulatory considerations for ERN Data Access Committee members”

We are happy to invite you to take part in a workshop addressing “Ethics and regulatory considerations for ERN Data Access Committee members”.

This workshop is jointly organised by ERICA and EJP RD and will take place online on 30th June from 10 am to 12:30 pm.

It primarily aims to train the members of the ERNs Data Access Committee members on the legal and ethical aspects to consider when examining an incoming data access request. All other interested stakeholders are welcome to join.

See the detailed agenda (, 213 KB).

Please [...]

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Training Webinar: Does Randomization matter in RD clinical trials?

As part of the EJP RD Training series, an advanced-level training webinar on the topic “Does Randomization matter in RD clinical trials?” (held on 14.12.2021)

The training consists of two parts: a 1.5-hour training in less frequently applied randomization procedures and their performance on the level of evidence, followed by 1 hour of panel discussion with experts from regulatory, pharma-industrial, academic, and clinical fields.

Rare Diseases (RD) is an area where national and international multidisciplinary collaborations are valuable to overcome some of the present challenges in research. Comparative trials serve as a major basis for evaluation of new therapies in [...]

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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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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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Pseudonymisation and Anonymisation

This workshop will be the first of the ERN data strategy workshop series. During this first workshop, we will discuss the techniques and algorithms to anonymise and pseudonymise healthcare data from patients with rare diseases. A special focus will be given to the challenges associated with preserving the privacy of patients with rare diseases. The advantages and drawbacks of pseudonymised and anonymised data both in terms of research outcomes and privacy protection level will be discussed. Finally, in relation with the GDPR regulation, the legal implications of sharing anonymised and pseudonymised data with external stakeholders will be examined. Following these [...]

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