News

Good off-label use practices: Where are we? What next?

The European Brain Council (EBC) is organizing a meeting at the European Parliament, in Brussels (Belgium), on 14 November 2023, 8:00-9:30 AM CET to discuss “Good off-label use practices: Where are we? What next?“. Marc Dooms, IRDiRC Interdisciplinary Scientific Committee Vice Chair, will offer a presentation about the off-label use of devices. The goals of this event are to reconnect […]

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Co-creating Calls-to-action That Drive Fundamental Change: How revolutionary leadership can inspire, catalyze and sustain long-term impact for hemophilia care

Don’t miss out the last event of the Reuters Hemophilia Changemakers 2023 Series – in partnership with Sanofi – that consists of three virtual events, two that took place in June and September, and the last one planned for November 7, 2023. It will bring together esteemed thought leaders to discuss policies, practices, and outline […]

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IRDiRC Drug Repurposing Guidebook: making better use of existing drugs to tackle rare diseases

The commentary ‘IRDiRC Drug Repurposing Guidebook: making better use of existing drugs to tackle rare diseases’ has been published in Nature Reviews Drug Discovery (https://www.nature.com/articles/d41573-023-00168-9). This guidebook, authored by the IRDiRC Task Force “Drug Repurposing Guidebook”, facilitates drug repurposing for rare diseases by organizing available tools into a standardized framework. The Task Force worked for […]

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Announcement of upcoming FDA Office of Orphan Products Development Natural History Studies grant funding opportunity

The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds for fiscal year (FY) 2024 to support natural history studies for rare diseases and conditions. This funding opportunity is intended to support prospective or retrospective, efficient, and innovative natural history studies with a focus on collaborative […]

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CORD: “Breaking the Last Mile”

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 […]

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KCE – “NEED” for rare diseases

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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