On October 14, 2023, the press conference of “Breaking the Last Mile – Research Report and Policy Recommendations on the Implementation of Rare Disease National Drugs (2023)” was successfully held by Chinese Organization for Rare Disorders (CORD) in Shanghai! Since the Chinese National Medical Insurance Administration – China Healthcare Security (CHS) launched the National Medical […]
Read MoreThe IQSEC2 Research and Advocacy Foundation will provide a 1-year grant, with a no cost extension of up to one year granted with approval, to support research related to IQSEC2-related disorder. The foundation will award either one grant of $60,000 or two grants for $30,000. The Foundation, in collaboration with the Orphan Disease Center, is […]
Read MoreThe 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 […]
Read MoreIn 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 […]
Read MoreThe 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 […]
Read MoreEurope’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 […]
Read MoreFollowing 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, […]
Read MoreDon’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 […]
Read MoreNew 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 […]
Read MoreUnder 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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