Art contest – Skin to skin

From February 28th 2023 to May 31st 2023

The René Touraine Foundation and its international network on Rare Skin Diseases have been organizing an international art contest since 2015: The Story of my Skin / Ma peau raconte. This year’s theme is Skin to Skin.

The aim of this event is to raise public awareness of rare skin diseases, to allow patients to express themselves and to encourage discussion. Please find more information here

Rare Diseases Day

February 28th

World Diseases Day is a globally-coordinated movement on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.

• It was set up and is coordinated by EURORDIS and 65+ national alliance patient organization partners.
• It provides an energy and focal point that enables rare diseases advocacy work to progress on the local, national and international levels.

Please find more information here: https://www.rarediseaseday.org/what-is-rare-disease-day/

NEW ERICA WP4 Webinar: Essential requirements before thinking about a clinical trial

Tuesday 28 February 2023 12:00 – 13:00 CET

In this webinar, Viviana Giannuzzi, Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus will overview what should be done at the very least before starting a clinical trial in a study site. All the requirements will be discussed according to the role that an investigator may have in the trial. Some guidance and sources will be also provided.

More information about the Webinar, here.

Organised by ERICA in collaboration with EJP RD and ERN BOND

Additional information, registration and updates at ERICA website

Please submit any questions to ERICA WP4 team francesca.gurioli@ior.it or ERICA coordinating office

An European Medicines Agency (EMA) survey about the development of a multi-stakeholder platform to promote collaboration for improving clinical trials in the EU!

EUSurvey – Survey (europa.eu) – open until 03 March 2023 at midnight CET!

We have been asked by European Medicines Agency to inform you of the launch of a public consultation concerning the development of a multi-stakeholder platform to promote collaboration for improving clinical trials in the EU as foreseen by Accelerating Clinical Trials in the EU (ACT EU) priority action 3.

This platform will enable regular dialogue between all EU stakeholders on clinical trials, and facilitate the evolution of the clinical trials environment by helping to identify key advances in clinical trial methodologies, technology and science. The platform will serve as a neutral space for the discussion of challenges and the development of practical solutions to enable and drive change. It is envisaged that there will be several phases of development before reaching its final design.

A concept paper outlining the proposal for the creation of the platform has been published together with the public consultation. The objective of which is to gauge interest in the platform, get feedback on priority topics for discussion, and provide comments on the proposal. The multi-stakeholder platform will have its kick-off meeting in Q2 2023.

The public consultation can be accessed at the following survey EUSurvey – Survey (europa.eu) and will remain open until 03 March 2023 at midnight CET.

Those interested can find more information on the web page Accelerating Clinical Trials in the EU (ACT EU).

For any questions, stakeholders can contact ACTEU@ema.europa.eu

EJP-RD Advanced Course “Statistical and Operational Challenges with Master Protocols”

March 24th, at 4-6:30 pm CET

Join the upcoming fourth EJP-RD advanced course titled “Statistical and Operational Challenges with Master Protocols,” held by Professor Franz König. The course will take place on March 24th, 2023 at 4-6:30 pm CET.

Please note that preregistration is mandatory to secure your spot. Additional details, including how to register, can be found on the EJP RD website.

ERN-Skin Workshop: Epidermolysis bullosa: from genes to translation into therapies

The workshop will deliver expert knowledge on the following aspects of EB: clinical and genetic basis, wound and interdisciplinary management, how to design and conduct clinical trials, gene therapy, and models used in translational research.

End of March 2023 (Preliminary date)

Freiburg, Germany

EURORDIS Open Academy Schools 2024 – Applications are open!

June 3rd-7th, 2024 in Barcelona, Spain

Eurordis organizes its 2024 edition of the Open Academy Schools in-person on 3-7 June in Barcelona.

The programme will include off-site visits to research facilities/hospitals, lots of contact time with faculty and EURORDIS staff and the chance to network with fellow patient advocates and researchers, supporting each other in learning and practice during the training and beyond.

Apply before October 27th for an immersive week of learning.

More information here

GRIDD Study – Global Research on the Impact of Dermatological Diseases

From June 5th until September 28th

GlobalSkin in collaboration with Cardiff University (UK) and University Medical Centre Hamburg-Eppendorf (Germany) launches the Global Research on the Impact of Dermatological Diseases (GRIDD) Study.

The mission is to generate comprehensive global data about the impact of dermatological conditions affecting skin, hair, nails, and mucosa.

Take the survey by clicking here

For more information click here

Take part in the Patient Partnership Framework Survey

Deadline: 25th June

EURORDIS invites you to take part in a survey to help develop a Patient Partnership Framework for the ERNs. The survey will take approximately 10 minutes to complete.

You may access the online survey here.

The aim of the Patient Partnership Framework is to formalise a guiding structure for all patient-clinician cooperation. For more information, please click here.

EJP-RD advanced course: “Replicated N-of-1 Randomized Controlled Trials for Rare Diseases”

June 30th, at 4-6:30 pm CET

Fifth EJP-RD advanced course titled “Replicated N-of-1 Randomized Controlled Trials for Rare Diseases” held by Professor Patrick Onghena.

Panelists for the subsequent one-hour panel discussion:

• Dr. Alex Sverdlov, Novartis
• Prof. Dr. Matthieu Roustit, Université Grenoble Alpes, France
• Prof. Dr. Stephen Senn, retired
• Dr. Virginie Hivert, EURORDIS

Please note that preregistration is mandatory to secure your spot. Additional details, including how to register: EJP RD website.

Recordings of the previous EJP-RD webinars:

• Webinar #1 “Does Randomization matter in clinical trials?” – presented by Prof. Dr. Ralf-Dieter Hilgers
• Webinar #2 “Composite endpoints including patient relevant endpoints (Quality of Life)” – presented by Dr. Johan Verbeeck
• Webinar #3 “The Statistical Evaluation of Surrogate Endpoints in Clinical Trials” – presented by Prof. Dr. Geert Molenberghs
• Webinar #4 “Statistical and operational challenges with master protocols” – presented by Prof. Dr. Franz König

EJP-RD Variant Prioritization and Enrichment for Rare Diseases Workshop

September 20th-22nd, in Amsterdam UMC, Istanbul, Turkey

EJP-RD will organize a workshop on Variant Prioritization and Enrichment for Rare Diseases from September, 20th to 22nd 2023.

A three-day immersive event in which participants will learn the fundamentals of Whole Exome Sequencing (WES) including terminology, pre and post-alignment processing, variant calling using Genome Analysis Toolkit (GATK) pipeline and annotation/filtration/interpretation of the variants, and more!

Registration is open here until September 9th, 2023

More information, here

ERICA Webinar: REMEDI4ALL / drug repurposing and clinical trial readiness by Anton Ussi

October 2nd, from 13:00 to 14:00 CEST

This ERICA webinar will discuss challenges associated with getting ready for and conducting clinical trials in drug repurposing, with a focus on differences between trial requirements for future on-label or off-label use.

The discussion will also highlight key services and tools available to the community, as well as the REMEDi4ALL projects support offering.

Registration and more information, here

EJP RD – Free online course: MOOC – “Diagnosing Rare Diseases: From The Clinic to Research and Back”

Launched on October 16th, 2023 and available until December 8th, 2023 – Online

The new facilitation window for the MOOC Diagnosing Rare Diseases: From The Clinic to Research and Back has been launched on October 16th, 2023. This update will be available until December 8th, 2023.

For more information click here

EJP RD – Free online course: MOOC From Lab to Clinic: Translational Research for Rare Diseases

Launched on October 16th, 2023 and available until December 2nd, 2023 – Online

The French Foundation for Rare Diseases is delighted to announce the upcoming opening of a new facilitation window of the MOOC From Lab to Clinic: Translational Research for Rare Diseases on October 9th. This update will be available until December 2nd 2023.

With expert guidance, explore drug discovery, clinical trials, regulatory approval, and challenges in this unique field. Gain insights from patients and experts, understand trial planning, design, and data sharing. By the end, you’ll have a comprehensive understanding of how evidence is built for safe and effective treatments.

More information, here

EJP RD Virtual Platform: unleashing the Power of FAIR Resources for Rare Disease Research

EJP RD has produced their new EJP RD Virtual Platform (VP) for the research community. The go-to hub is dedicated for Findable, Accessible, Interoperable, and Reusable (FAIR) resources.

It includes catalogues of resources, registries, biobanks, knowledge bases and tools compliant with agreed standards.

For more information explore the VP Portal now by, clicking here

ERICA – WP5 Educational Webinar 3: “Bridging the gap between promising preclinical data and a successful clinical trial”

October 19th, 2023 – From 16:00 to 17:00 CET

ERICA is organizing a 3rd education webinar on “Bridging the gap between promising preclinical data and a successful clinical trial” on October 19th, 2023 at 16:00 CET.

This webinar focuses 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.

For more information and for registration, click here

CMTC-OVM – 2023 Members conference

October 28th-29th, 2023 in the Netherlands and online

CMTC-OVM will organize their annual worldwide member’s conference in the Netherlands, on October 28th-29th, 2023. This year they will organize a ‘Hybrid’ conference again. This means that members can choose to participate Online/Virtual or be present In-Person at their location in the Netherlands.

For more information about the program and for registration, click here

ERICA & EJP-RD Joint Conference

November 21st, in Amsterdam UMC, Amsterdam, NL

ERICA and EJP-RD will organize their joint conference on November, 21st 2023. There will also be a pre-conference dinner on Monday 20th November.

During this meeting the will provide a close overview of all the relevant activities and joint efforts in the field of Rare Disease Research during the lifetime of EJP RD and ERICA with a further focus on the future RD Partnership & ERNs Research activities.

Registration and more information, here

EJP RD workshop: Managing RD biological sample data in Biobanks

December 13th-14th Milan, Italy

EJP RD is organizing a 2 day immersive workshop on Managing RD biological sample data in Biobanks. This training workshop is the conclusive event of a series of capacity building programs on biobanking put forth by the European Joint Programme on Rare Diseases.

It is aimed at biomedical researchers, medical professionals, and biobank managers who want to learn about the path to follow for exploiting the high value of the biological sample repositories in the RD research. The role of ERNs samples and how to optimise them for networking/ research networks is a basis for the development of the present course.

Registration and more information, here