The Rare Disease Clinical Outcome Assessment (COA) is an initiative of the Critical Path Institute‘s Consortium with the same name, a public-private partnership that is focused on optimizing COA selection during the drug development for rare diseases. The COAs included in the the Rare Disease COA Resource represent a series of tools that are most […]
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The US National Institutes of Health (NIH) published a new Notice of Funding Opportunity (NOFO) with the aim to provide support for applications for IND-enabling studies for the development of a novel in vivo genome editing therapeutic platform (genome editor plus delivery system) for two or more disease indications, using the same genome editor, route of administration, and […]
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The Changing Focus of Regulatory Frameworks Around the Globe and the Opportunities for Harmonization
IRDiRC is proud to share that Regulatory Scientific Committee Vice Chair, Violeta Stoyanova-Beninska, is one of the guest editors of the research topic “The Changing Focus of Regulatory Frameworks Around the Globe and the Opportunities for Harmonization” in Frontiers in Medicine. As regulation related to the development, registration and monitoring of medicinal products is at […]
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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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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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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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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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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 – 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 new […]
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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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