EJP RD: Online Training Statistical and Operational Challenges with Master Protocols

Statistical and Operational Challenges with Master Protocols (1)

During this EJPRD webinar, distinguished lecturer Franz Köning will explain what is meant by master protocols comparing basket, umbrella and platform trials. Great opportunity to gain valuable insights into cutting-edge statistical methods for Rare Diseases Clinical Trials. Don’t miss this incredible opportunity to expand your knowledge and enhance your research skills.

📚Title: Statistical and Operational Challenges with Master Protocols

🇬🇧Language: English

🚨Registration deadline: 23 March 2023

ℹ️: https://www.ejprarediseases.org/event/statistical-and-operational-challenges-with-master-protocols/



Congratulations to Alberto Pereira

Albertos oratie ERICA

ERICA and Endo-ERN Network Coordinator Professor Alberto Pereira held his inspiring Inaugural Lecture in January 26th on the topic 2034 , where he considered the possibilities for rare disease care during the next decade at Amsterdam UMC and beyond. As the coordinator of two important projects that will change the rare disease landscape in Europe – Endo-ERN & ERICA – the lecture was a welcome opportunity for all his colleagues to glimpse the future that will be the work of the next decade.

Congratulations Alberto on behalf of ERICA Consortium and thank you for being a great ambassador for [...]



IRDiRC: Opening for a new member for Interdisciplinary Scientific Committee


The Interdisciplinary Scientific Committee (ISC) is a multi-stakeholder, multi-disciplinary group of experts in rare diseases medical research with a strong expertise in data sharing, registries, biobanks and natural history studies.
ISC has one opening for a new member with experience in rare diseases data sharing, ontologies, natural history, biobanking and registries. Interested candidates are invited to send their resume, biosketch and letter of motivation to  scisec-irdirc@ejprarediseases.org before the 2nd of April, 2023.
More information about the Interdisciplinary Scientific Committee (ISC): https://irdirc.org/isc/



EJP RD Joint Transnational Call now open!

JTC 2023 (2)

The European Joint Programme on Rare Diseases launches today the Joint Transnational Call 2023, a funding opportunity for research projects on the development of new analytic tools and pathways to accelerate diagnosis and facilitate diagnostic monitoring of rare diseases.

This year’s topic is: “Natural History Studies addressing unmet needs in Rare Diseases” The aim of the funding opportunity is to enable scientists in different countries to build an effective collaboration on a common interdisciplinary research project based on complementarities and sharing of expertise, with expected impact to use the results in the future for benefit of patients.

Don’t miss [...]



”MOOC on Diagnosing Rare Diseases: from the Clinic to Research and back

ERN Workshops (30)

In the context of EJP RD’s ERN Workshops, a face-to-face workshop entitled “Epidermolysis bullosa: from genes to translation into therapies” is being organized by Prof. Dr. Cristina Has of the University Hospital Freiburg.

The in-person event will take place on 23-24 March 2023 in CIBSS – Centre for Integrative Biological Signalling Studies in Freiburg, Germany.

Registration is open until December 15th.

Registration and more



IRDiRC Announces The Creation of Regulatory Science Committee to Tackle Regulatory Challenges in Rare Disease Research

IRDiRC Designs (7)

IRDiRC, a global collaborative initiative with the vision to enable all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of coming to medical attention, today announced the formation of the Regulatory Science Committee (RSC). The new RSC “brings broad expertise across different stakeholders and geographies allowing for a balanced synergy that will provide insights and guidance into the advancement of IRDiRCs mission” said David A. Pearce, Chair of IRDiRC.

Read the full press release here.



ERN Research Mobility Fellowship funding opportunity!


The European Joint Programme on Rare Diseases (EJP RD) is glad to announce that the last round for the Research Mobility Fellowships funding opportunity is now open until November, 13th, 2022. The call aims to support PhD students, postdocs, and medical doctors in training to undertake scientific visits fostering specialist research training outside their countries of residence.

The exchange can be carried out (1) within the same ERN (Full Members and Affiliated Partners), (2) between different ERNs (Full Members and Affiliated Partners), or (3) between ERN Full Members / Affiliated Partners and non-ERN institutions.

Either home or host (secondment) institution [...]



The Smart Guidance tool for the FAIRification of rare disease registries


The Smart Guidance tool for the FAIRification of rare disease registries was released on the 18th of July. This is a questionnaire-based tool built on the Data Stewardship Wizard that will guide users (e.g., data stewards) through the process of making their registry more FAIR. It covers various aspects like the composition of the FAIRification team, best practices for data representation, and giving access to your data.
The tool is accessible via: https://smartguidance-rd.ds-wizard.org/ (after signing up)
The User Guide is available here which gives you guidance on how to get started on a Smart Guidance project [...]