IRDiRC is pleased to announce that the IRDiRC‘s Rare Diseases Research Initiatives State of Play 2019-2021 Report will be published on Tuesday, 05 July 2022.
This report is based on scientific articles and press releases published between 2019 and 2021; it seeks to inform stakeholders and the rare diseases community about the developments and observed trends in the field of rare diseases research. A chapter is dedicated to an update on the global rare diseases research landscape for the past ten years to celebrate IRDiRC’s 10th anniversary.
More information here: https://irdirc.org/resources-2/state-of-play/
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The ERICA 2nd General Assembly took place in Bologna, Italy as a hybrid meeting.
We had three productive days full of interactive sessions and we are thankful for everyone’s active contributions both in Bologna and remotely.
All the presentations are now available at the ERICA Website
Download ERICA 2nd GA Summary Report (
, 666 KB)
Presentations 2GA Bologna
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On June 30th, 2022 EJP RD has launch the Innovation Management Toolbox (IMT), a free-to-use and curated reference library of resources in rare disease translational medicine that will provide investigators with self-help resources specific to their needs. The database will be maintained actively!
Learn about the IMT: https://www.ejprarediseases.org/innovation-management-toolbox/ Access the IMT: https://imt.ejprarediseases.org/
Video tutorial for users: https://youtu.be/H92b6WEWFhw
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The webinar on “Composite endpoints including patient relevant endpoints (Quality of Life)” is now available here
In this webinar you will learn about:
The procedures to combine multiple endpoints and its limitations The properties and the flexibility of the class of generalized pairwise comparison tests The potential advantages and disadvantages of designing a clinical trial in rare disease with generalized pairwise comparisons primary analysis
You can watch also webinar on Randomization procedures in Rare Disease Clinical Trials here.
All the available ERN Research related webinars: https://erica-rd.eu/events/webinars/
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Joint ERICA and EJP RD Workshop “Ethics and regulatory considerations for ERN Data Access Committee members” is now reviewable!
This workshop is the fourth of the ERN data strategy workshop series. It primarily aims to train the members of the ERNs Data Access Committee on the legal and ethical aspects to consider when examining an incoming data access request.
The recording and all the presentations are now available!
View this workshop
Speakers Clémence Le Cornec (ERKNet/EJPRD/ERICA – Heidelberg Universitätsklinikum) Franz Schaefer (ERKNet/EJPRD/ERICA – Heidelberg Universitätsklinikum) Faisal Ahmed (Endo-ERN – University of Glasgow) Jelena Malinina (EURORDIS) Leonardo Cervera [...]
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The Blackswan Foundation , IRDiRC and EJPRD are hosting the 7th RE(ACT) Congress and 5th IRDiRC Conference that will be held in person in Berlin, Germany from March 15th – 18th, 2023.
The joint event “RE(ACT) Congress and IRDiRC Conference 2023” aims to bring together scientific leaders and experts and young scientists from various breakthrough scientific fields to present cutting-edge research, exchange ideas, and discuss rare diseases research policies. Patients and patient organizations committed to research will also be in attendance to share their experiences and perspectives.
The RE(ACT) Congress and IRDiRC Conference 2023 represents an exciting program with [...]
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New: continuous enrollment
The third run of the MOOC (Massive Open Online Course) “Diagnosing Rare Diseases: from the Clinic to Research and back” co-developed by EJP RD, ERN Ithaca, ERN GENTURIS and the French Foundation for Rare Diseases has opened on April 18th.
Registration is free and open at this link.
We specifically encourage medical and biomedical science students to register and follow the MOOC. The topics covered include:
The diagnostic process and the types of genetic tests available for rare diseases The differences in rare genetic diseases patient pathways Technological advances for diagnostic research The [...]
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Happy to share: the birth of the Journal of Rare Diseases (JORD) published by Springer Journal of Rare Diseases is an open access, peer-reviewed journal with clinical and basic research interests in rare and genetic diseases. The journal welcomes submissions in all fields of rare diseases. In addition to original research articles, the journal publishes reviews, letters to the editor, case reports, and short communication, thereby providing a forum for discussions on cutting edge perspectives in the field of rare diseases.
Uniquely dedicated journal to rare diseases in the region. Growing interest and steadily increasing publications in the field [...]
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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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