The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds for fiscal years (FY) 2023 – FY 2025 to support clinical trials for rare diseases and conditions. The purpose of this funding opportunity announcement is to fund clinical trials of products evaluating efficacy and/or safety in support of a […]
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In the context of the Belgian Presidency of the Council of the European Union, the Belgian minister of health wants to make a focal point of identifying and using evidence on unmet patient and societal needs, with the objective of developing a healthcare system and policy that is more needs- than supply-driven. KCE and Sciensano […]
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The 2024 edition of the Open Academy Schools will take place in-person on 3-7 June in Barcelona. The face-to-face intensive week of the Open Academy School on Medicines Research & Development (formerly EURORDIS Summer School) and the Open Academy School on Scientific Innovation & Translational Research (formerly EURORDIS Winter School) take place during the same week, incorporating parallel sessions, focusing on School-specific topics, and joint […]
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Europe’s Most Exciting Orphan Drug Event The World Orphan Drug Congress is the largest and most established orphan drugs & rare diseases meeting globally. Now in its 14th annual edition, the World Orphan Drug Congress Europe will continue to bring experts from the whole value chain under one roof, where you get to meet decision […]
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Following a positive opinion from the European Medicines Agency (EMA), the transfer of marketing authorization of Strimvelis from Orchard Therapeutics has been approved by the European Commission. Fondazione Telethon (Italy) will now be responsible for providing the gene therapy to eligible patients in the European Union. Fondazione Telethon, one of the main Italian biomedical charities, […]
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Don’t miss out the new IRDiRC publication developed by Therapies Scientific Committee (TSC) members Daniel O’Connor, Annemieke Aartsma-Rus, Anneliene Jonker, and Regulatory Scientific Committee (RSC) member, Michela Galbado – “Defining rare conditions in the era of personalized medicine“. “Despite these consensus features, there is currently no common global agreement on the impact and widespread application […]
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New Publication: Making Rare Disease Research Attractive to Companies by IRDiRC Chrysalis Task Force
Accessible at https://doi.org/10.1177/26330040231188979, the Chrysalis Task Force identified key financial and non-financial factors that make rare disease research and development attractive to companies. More information on the Chrysalis Task Force here: https://irdirc.org/chrysalis-project/ « It was a real pleasure working with such a talented IRDiRC Task Force. We also truly appreciate the critical input provided by the […]
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Under the Horizon Europe Framework Programme, a new two-stage call for proposals for “Establishing novel approaches to improve clinical trials for rare and ultra-rare diseases” was published on 27 July, 2023. The project generated from the topic should not only develop capacities and capabilities to execute innovative trial designs, but also plan to identify solutions […]
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We are delighted to announce that IRDiRC Therapies Scientific Committee (TSC) Vice Chair, Anneliene Jonker (University of Twente, The Netherlands), will give a talk on the drug repurposing guidebook at the RExPO23 Conference, that will take place on October 25-26, 2023 in Stockholm, Sweden. Anneliene will participate in Session 4 – Regulatory Perspectives, on the […]
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Taking place in February to mark the occasion of Rare Disease Day, the EURORDIS Black Pearl Awards celebrate the inspirational qualities of people living with a rare disease along with those who go that extra mile to make a difference to their lives. The deadline for nominations for the EURORDIS Awards 2024 has been extended […]
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