ERN Workshop on contemporary outcome measures in neuromuscular diseases

In the context of EJP RD’s ERN Workshops, a hybrid workshop on « contemporary outcome measures in neuromuscular diseases » aimed at understanding the advancement of technologies with digital outcome measures in neuromuscular diseases is being organised by Prof. Sabrina Sacconi of University Hospital Nice. The hybrid event (in-person on site and online by videoconference) will take place over two days on March 4th – 5th,…

ERN Workshop on endocrine cancer: A challenge in adults and children

In the context of EJP RD’s ERN Workshops, a face-to-face workshop on « Endocrine cancer: A challenge in adults and children » aimed at giving an update on the molecular background and clinical management of rare endocrine malignancies is being organised by Prof. Barbara Jarzab of the M. Sklodowska-Curie National Research Institute. The in-person event will take place on May 4th, 2022 at the M. Sklodowska-Curie National…

ERN Workshop on anonymisation and pseudonymisation of rare disease patients⁠: Legal and technical aspects

Topic: Anonymisation and pseudonymisation of rare disease patients: legal and technical aspects Date and Time: December 15th, 2021 from 15.00 – 16.00 CET. Organisers/Moderators : Franz Schaefer and Clémence Le Cornec Speakers: Spencer Gibson (University of Leicester), Guillaume Byk (DG-Santé – European Commission) and Antonio Cutillo (Joint Research Center) Description: This workshop will be the first of the…

EJP RD co-organises meeting on Domain specific Common Data Elements (DCDEs) Curation

EJP RD is co-organising a meeting, along with the European Rare Disease Research Coordination and Support Action consortium (ERICA) and the Joint Research Centre (JRC), on Domain specific Common Data Elements (DCDEs) Curation targeted towards clinicians and people with a medical background to join. The online meeting will take place on December 9th, 2021 from 16.00 – 17.00 CET. In…

XIV Foresight Training Course (FTC): « The health emergency: Regulatory crash and future perspectives »

The Fondazione Gianni Benzi is organizing the XIV Foresight Training Course (FTC) on the theme of « The health emergency: Regulatory crash and future perspectives » as a virtual meeting that will take place on December 10th, 2021 from 11.20 – 18.30 CET. The agenda features the following sessions: Session 1 – How the European regulatory framework reacted to the COVID-19 emergency: Extraordinary…

1st International Conference on Rare Diseases and Paediatric Research

The 1st International Conference on Rare Diseases and Paediatric Research is being organized by 95, Rare Alliance Greece together with Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF) and « Athena » Research Center in Information, Communication and Knowledge Technologies. The Conference is aimed at opening the discussion with all relevant stakeholders on the challenging topic of research…

European Medicines Agency (EMA) publishes guideline on registry-based studies

The European Medicines Agency (EMA) has announced the publication of a guideline on registry-based studies following its adoption by the EMA’s cross-Committees Task Force on registries and the Committee for Medicinal Products for Human Use (CHMP). The guideline provides pharmaceutical organisations with key methods and good regulatory practices on the planning and conduct of studies…