External Newsletter n° 9. May 2020
In this edition of May we would like to share with you the latest EJP RD initiatives such as open calls, training workshop, and the funded ERN workshops. Also, the EJP RD has been recently featured on the EU Health Newsletter (April edition).
EJP RD together with IRDiRC had a co-joint virtual stand (Hall B) at the ECRD congressRead about the success of this event!
We would like to address a special thanks to the 476 persons that have contributed with their time to the EJP RD Research Community Survey!
In addition, you will find info on virtual meetings as well as relevant documents (guidelines and guidebook) on Horizon Strategy, drug development, and patient healthcare, and latest Covid-19 related actions taken by several EU organizations and institutions.
ECRD Conference
Patients, scientists, researchers, policy makers from Europe and beyond gathered together to discuss the future of the rare disease field. Importantly, the scenarios defined by the Rare 2030 foresight study were discussed and the entire rare disease community will work on the actions to make the most desired option a reality.
Please, note that all participants of the congress will have access to the presentations and posters for 1 year.
Congrats to Eurordis for having rearranged this conference into virtual event in such a successful way and short time!
Don’t forget the EJP RD Helpdesk is available for any inquires and services provided by the EJP RD including Clinical Trials Support Office and Innovation Management Advice.
Quality assurance, variant interpretation and data management
in the NGS diagnostic era
Registration for this course is now open, deadline is 20 July.
The course will take place in Istanbul, Turkey on 12-14 October. A mitigation plan due to Covid-19 is in place. The aim is to teach participants about the evaluation of the pathogenic nature and clinical significance of genetic variants, on the criteria that have to be set to the NGS analysis pipelines and on the use of international databases.
The targeted audience is: Laboratory scientists (European Board Medical Genetics registered), junior scientists, clinical geneticists, other medical doctors in training, policy makers and assessors for laboratory accreditation, and patient representatives, with a basic knowledge in biology or medicine.
To ensure active participation and exchange with teaching staff and fellow participants, a maximum of 35 participants will be admitted to the training course. There are #2 travel fellowships available, participants from EU-13 countries and Turkey are encouraged to apply.
The workshop and registration are free of charge.
In February 2020 the EJP RD launched its first “European Reference Networks (ERNs) Training and support program” as part of the ‘Training and Empowerment’ activities that aim to fill the gap in education on rare diseases research.
The “Research Training Workshop” call is aimed at identifying workshop topics to train ERN researchers and clinicians in relevant innovative themes with a cross-ERN added value. Selected applicants receive financial support for the organization of a 2-days workshop for 20 participants. Training themes include innovative and/or diagnostic research methodologies, interdisciplinary treatment approaches such as gene therapy and transplantation, etc. Topics can be proposed by the ERNs or by investigators belonging to EJP RD beneficiary institutions.
The first round of submission and evaluation have been finalized in March. As a result, three workshops were recommended for funding. The workshops will take place in the upcoming months. The information on their launch and more details will be provided and updated on the EJP RD website. Here is the list of the funded workshops:
  • Launching Advisory Committees for Therapeutics (ACT) in rare diseases. Joanne Lee, Newcastle University, UK
  • Intranodal Magnetic Resonance Lymphangiography Studies Workshop. Willemijn M. Klein, Radboud University Medical Center Nijmegen, The Netherlands. VASCERN
  • Methods to study rare diseases, their causes and treatments: The Basics & Beyond.
    Olaf Dekkers. Leiden University Medical Center (LUMC), The Netherlands. Endo-ERN
EJP RD Rare Diseases Research (RDR) Challenges Call is open
This innovative call aims at solving specific research challenges in the field of therapeutic development for rare diseases as well as at facilitate and fund collaborative projects between industry, academia, SMEs, and patient organizations. The total budget is 1.5 Mio €. The call is open to academia, clinical/public health sector, SMEs, and rare disease patient representatives from eligible countries involved in the EJP RD; deadline is 30 June at 2pm CEST. Industry partners have been involved in the identification of four challenges that are the topics of the call and that were validated by the EJP RD partners including patients. It is still possible to establish consortia via a B2B tool.
EJP RD Networking Support Scheme (NSS) Call is open
Next applications’ collection date is 1 September at 2 pm CEST. The NSS call aims to encourage sharing of knowledge on rare diseases and rare cancers of health care professionals, researchers and patients.
The NSS also aims to enable or increase the participation of underrepresented countries in Europe in new and in existing research networks on rare disease(s) or rare cancer(s).
Eligible applicants are health care professionals, researchers and patient advocacy organizations from countries involved in the EJP RD.
GA4GH 8th Plenary is going virtual, save the dates: September 29 – 30 2020. The event will bring together organizations and stakeholders from the genomics and health community for two days of keynotes, talks, updates, and workshops that will focus on advancing development work for the immediate data sharing needs of the community. More details, including registration, will be shared in the coming weeks.
ELIXIR All Hands 2020 meeting will take place virtually 8 - 10 June 2020 and will bring together members of the ELIXIR community from across the ELIXIR Nodes and collaborators from partner organizations. ELIXIR’s achievements and activities will be reviewed and plans for the future will be discussed. Participants from ELIXIR Nodes and invited guests are welcome. Program is available. Registration and posters submission are open.
Latest progress on Horizon Europe’s Implementation Strategy provides the framework for carefully designed rules and efficient processes across the entire program and project life cycle. This is a living document that allows the program to deliver its impacts as efficient and user-friendly as possible and will receive further updates. The rules and simplification measures put in place for Horizon 2020 were widely acknowledged by stakeholders as a big step towards a more attractive and easier to use program, based on this experience, work has been ongoing on this document to set out how the program will be managed in practice to achieve its ambitious objectives.
Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook has been published in Nature Reviews Drug Discovery. The paper is authored by the Orphan Drug Development Guidebook (ODDG) Task Force that is helping to reach the IRDiRC goal 21000 new therapies for rare diseases will be approved’ by 2027.
The aim is to support drug development for rare diseases by organizing available tools in the USA, Europe and Japan into a standardized framework and make them ready for optimal uses. The ODDG materials are now available on a user-friendly website, to guide those who are less familiar with this complex enterprise. By enhancing the use of available tools, delays in development timelines can be avoided, risks and costs reduced, patient and regulatory acceptability improved.
ERN TRANSPLANT-CHILD PATIENT’S GUIDE gives some important recommendations for transplanted patients and their families to take care of their health after this complex procedure. The guide is common for all types of transplants and includes different aspects of the post-transplantation process such as medication, rejection, infections, and emotions
The Patient’s Guide currently is available in English, Spanish, and French.
Covid-19 Global Response – Communication Toolkit developed by the European Union (EU) is now available. Take advantage of this resource to join the social media campaign and help to raise awareness on the important initiatives led by the EU on the fight against Covid-19. The opening event of Coronavirus Global Response, a worldwide pledging effort to raise €7.5 billion of funding to rapidly develop universally available and affordable diagnostics, treatments, and vaccines against the virus, has already raised €7.4 billion, including a significant contribution of €1 billion mobilized by Horizon 2020.
The communication toolkit will allow us to easily join the social media campaign and support such global effort.
Covid-19: Commission issues guidance to mitigate clinical trial disruption in the EU in order to ensure their continuation in the EU during the Covid-19 pandemic. The aim is to mitigate the disruption of clinical research and therefore the negative effects of the pandemic, without compromising on quality and safety. The guidance offers recommendations for simple and flexible measures to respond to the current situation, and to ensure that patients participating in clinical trials across the EU can continue receiving their medicines.
ECRIN COVID-19 TASKFORCE has been established with its national partners to confront the pandemic. ECRIN is working together with the healthcare partners to take care of patients as first priority. At the same time, ECRIN is also supporting the researchers by joining forces worldwide to find a cure and a vaccine.
Sharing research data is a critical step in this global cooperation effort and the ECRIN Taskforce, organized in #8 different initiatives, is focused on high relevant topics around research, clinical trials, policy, and networking.
ELIXIR COVID-19 SLACK WORKSPACE has been launched to discuss and exchange ideas on Covid-19 research initiatives, consider collaboration opportunities across ELIXIR Nodes, and share results of work on the virus with other ELIXIR members. Please, get in touch with ELIXIR If you are interested in joining this initiative.
Learn more about upcoming webinars organized by the ERNs (ERN-BOND, ERN-RND – ERN-EuroNMD - European Academy of Neurology (EAN), ERN-EpiCARE, ERN-ENDO, eUROGEN, ERN – Guard Heart, ERKNet, EuroBloodNet, Transplant Child) and by Fondazione Giovanni Bensi & ARISE
Information regarding updated ERNs annual meetings scheduled in 2020-2021 can be found here.
Three opening positions are available at EJP RD member institutions:
  • SCIENTIFIC PROJECT MANAGER, ERN-EYE, Hôpitaux Universitaires de Strasbourg (HUS). France 
  • HELPDESK ASSISTANT, ERN-NMD, Institution of Myology. France
  • TECHNOLOGY TRANSFER ASSOCIATE, Fondazione Telethon. Italy
This initiative has received funding from the European Union's Horizon 2020
research and innovation programme under GA N°825575
EJP RD is coordinated by the National Institute for Health and  Medical Research (INSERM)
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