EFPIA, EATRIS, ELIXIR, BBMRI, ECRIN statement on the role of research infrastructures to boost patient-centred research and innovation in Europe

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The European Union has invested heavily in health research. Since the early 2000’s it supported the creation of European Research Infrastructures (pan-European organisations devoted to health research) like BBMRI, EATRIS, ECRIN, ELIXIR, and funded the Innovative Medicines Initiative (IMI), the world’s largest public private partnership (PPP) in health. The combination of PPPs such as the IMI and European infrastructures…

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UNO SGUARDO RARO (A Rare Glance)

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The rare disease international film festival is the first and only film festival on rare diseases in Europe. It collects and promotes the best video works on rare diseases and social inclusion through an international competition and works with public and private partners, both national and international, the rare diseases community, disability advocates, entertainment and…

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Rare disease perspectives in Central – Eastern Europe

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On September 16th 2019, a workshop “Rare disease perspectives in Central – Eastern Europe”, twinned to the General Assembly of EJP RD, will be organized in Gdansk, Poland. The main aims of this conference are discuss challenges and opportunities of rare disease research in Central – Eastern Europe countries and areas of involvement within EJP…

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EMA’s human medicines committee (CHMP) recommends authorisation of new treatment for phenylketonuria, a rare inherited metabolic disease

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EMA’s human medicines committee (CHMP) has recommended the authorisation of Palynziq (pegvaliase), a new medicine for patients aged 16 and older with phenylketonuria, a rare but potentially serious inherited metabolic disease. Patients suffering from this disorder do not have the enzyme that breaks down phenylalanine, an amino acid which is found in most foods containing…

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New results of #RareBarometer survey on patient’s preferences on rare disease data sharing & protection

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A new EURORDIS-led article, « Share and protect our health data: an evidence based approach to rare disease patients’ perspectives on data sharing and data protection – quantitative survey and recommendations » has just been published in the Orphanet Journal of Rare Diseases. The peer-reviewed article is freely accessed, it was written in collaboration with Rebecca Dimond,…

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FROM HORIZON 2020 TO HORIZON EUROPE: MISSION-ORIENTED APPROACH TO MAXIMIZE RESEARCH AND INNOVATION PROGRAMS IN EUROPE.

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The success of European research missions, which aim to stimulate innovation to solve major societal problems, will require a complete change in the way governments think about business and risk, according to Professor Mariana Mazzucato, special advisor to the European Commissioner for Research, Science and Innovation, Carlos Moedas. On 4 July 2019, Prof. Mazzucato published a report…

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D1.2 Criteria to assess how patient engagement expectations are met

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Patients Active in Research and Dialogues for an Improved Generation of Medicines WP1 – Defining stakeholders’ preferences, needs and expectations This report describes the work that was done as part of PARADIGM’s work package 1 (WP1), which aimed to identify and define stakeholders’ preferences, needs and expectations in patient engagement (PE) through an online survey…

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Survey on online training needs in the context of rare diseases research

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EJP-RD is a multifaceted program aimed at supporting Rare Disease (RD) research in every possible way. This  include funding of primary research, networking and training capacities as well as the development and application of tools and methodologies facilitating the generation, analysis, and dissemination of data all the way from genetic/translational research to clinical trials. Dr. Roseline Favresse &…

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