A recent report of the joint action Towards the European Health Data Space (TEHDAS) examines EU-level and national funding for health data collection, access and use for secondary purposes, such as research. To ensure the economic sustainability of the forthcoming European health data space (EHDS), the EU and the member states will need to solve how…
A recent report of the joint action Towards the European Health Data Space (TEHDAS) identifies a minimum set of technical services – basic software items – required for the cross-border secondary use of health data in Europe. The secondary use of health data involves using health data, such as patient records, for research, decision-making, and…
The National Institutes of Health (NIH), National Center for Advancing Translational Sciences (NCATS) and the U.S. Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER) are hosting a jointly sponsored virtual workshop to explore Regulatory Fitness in Rare Disease Clinical Trials. The virtual workshop will take place over two days on May 16th – 17th. Academic investigators, patient…
The European Medicines Agency (EMA) is organising a joint EMA/HMA/TEHDAS Data Quality Framework for Medicines workshop as a virtual meeting on April 7th from 13.00 – 17.15 CET. This workshop seeks to engage stakeholders early in the drafting process of an EU Network data quality framework, to ensure a robust starting point, in line with their…
As part of the EJP RD Training series, an advanced-level training webinar on the topic « Composite endpoints including patient relevant endpoints (Quality of Life) » aimed at introducing the generalized pairwise comparison ideas and concepts as well as demonstrating its benefit in small sample trials, more particularly in including patient relevant endpoints, such as quality of…
A funded networking event “European Forum on Visceral Myopathy 2022” is being organised in Camogli, Italy. This event has received funding support from the EJP RD’s Networking Support Scheme (NSS) funding opportunity. The in-person event will take place over three days from April 27th – 29th. Special interest will be devoted to clinical diagnosis and patient management, genetics and onset molecular…
In the context of EJP RD’s ERN Workshops, a face-to-face workshop entitled « Modelling & Simulation: Research Methodologies for Small Populations in Rare Diseases » aimed at facilitating discussion and exchange of knowledge on the M&S methodologies and strategies as innovative and promising enough for facing complex multifactorial or rare diseases and conditions that require highly specialised treatments and resources is being organised…
In the context of EJP RD’s ERN Workshops, a face-to-face workshop entitled “Functional exploration of genetic variants in cardiac diseases” aimed at presentation of functional explorations of variants of interest identified using Drosophila, C. elegans, Zebrafish and iPSc models to understand the molecular bases of genetic diseases, in particular heart and muscle diseases, is being organised by Philippe Chevalier of the Hospices Civils de…
A funded networking event « International Vasculitis Patient Conference » is being organised as a satellite hybrid event to the 20th International ANCA Vasculitis Workshop 2022. This event has received funding support from the EJP RD’s Networking Support Scheme (NSS) funding opportunity as part of the sixth round of funding in September 2021. The hybrid event will take place over two days from…
The Global Alliance for Patient Access has made available a policy paper on unmet needs in Neuromyelitis Optica Spectrum Disorders (NMOSD) in Europe, published in multiple languages. A rare autoimmune condition of the central nervous system, NMOSD causes deterioration to a person’s optic nerve and spinal cord. It affects over 10,000 people in Europe but…