The H-CARE Pilot Survey, the new Rare Barometer survey designed to provide a detailed understanding of patients’ experiences of healthcare, is now live! The long term goal of the H-CARE Survey is to develop a centralised patient feedback mechanism across the 8000+ rare and complex diseases, and across the 24 European Reference Networks (ERNs). In…
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On January 7, 2020, the EU Joint Programme – Neurodegenerative Disease Research (JPND) initiative launched a transnational call for novel imaging and brain stimulation methods and technologies related to neurodegenerative diseases. Neurodegenerative diseases are debilitating and largely untreatable conditions that are strongly linked with age. Worldwide, there are estimated to be 47 million people suffering from…
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EPTRI (European Paediatric Translational Research Infrastructure) is an EU funded project aimed to design the framework for a new infrastructure dedicated to paediatric research that will work to accelerate the paediatric drug development processes from medicines discovery, biomarkers identification and preclinical research to developmental pharmacology, age tailored formulations and medical devices. The final goal is…
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The aim of this survey is to gain the views and experiences of people living with rare endocrine disorders about the research they think should be prioritized related to their disease within European countries. This initiative is taken in order to provide a better understanding of patient needs and challenges in this area. This questionnaire…
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Congratulations to our coordinator, Dr. Daria Julkowska, from the EJP RD community, we are very happy for you! Dr. Julkowska won the EURORDIS Black Pearl 2020 – European Rare Disease Leadership Award for her « outstanding leadership and dedication shown to the rare disease community and the positive impact she has had on rare disease research…
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A key article from Guido Rasi, EMA’s Executive Director, Sabine Straus, the chair of EMA’s safety committee (PRAC) and Peter Arlett, the Agency’s Head of Pharmacovigilance and Epidemiology, published in Clinical Pharmacology and Therapeutics: Pharmacovigilance 2030. In 2030, regulators will dedicate significantly more time to engaging with patients and healthcare professionals and thus ensuring that the…
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The journey of a medicine assessed by EMA The European Medicines Agency has just published an interactive timeline describing the journey of a medicine for human use authorised through EMA, from initial research to discussions on patient access to medicines across the EU. This tool is based on a booklet published earlier this year, and allows the user to…
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This is a video recently published by ERN TransplantChild to promote the collaboration between pediatric European Reference Networks (ERNs). Crosstalk among ERNs is an important task that could help improving healthcare for rare diseases. Therefore, it is necessary that the networks start collaborating and working together in their overlapping areas. We can « work together for…
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Photo submissions for the Photo Award 2020 are now open, until 16th January 2020. The EURORDIS Photo Award is an opportunity to visually express what it means to live with a rare disease and to share your story with the rare disease community and beyond. The contest is open to all nationalities, ages and diseases.…
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RWE multi-stakeholder initiative – Paper for consultation The draft stakeholder guidance of the multi-stakeholder initiative on ‘Real-world evidence (RWE) to support HTA/payer decisions about highly innovative technologies’ is now open for consultation until 6 January 2020. Commissioned by the Belgian healthcare payer INAMI/RIZIV, this work defines how RWE can be best used to close evidentiary gaps for HTA…
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