Join us for this 2-day multi-stakeholder event on 29-30 January covering the day’s most pressing ethical issues in non-trial preapproval access at CUPA Con 2024! In-person attendees will be able to take advantage of interactive workshops, various networking opportunities, a cocktail reception, and other perks. For those unable to attend in-person, tickets providing access to […]
Read More
IRDiRC is delighted to announce that multiple representatives of the Task Force “Drug Repurposing Guidebook”, including Therapies Scientific Committee (TSC) vice chair Anneliene Jonker (Duchenne Parent Project, The Netherlands) and TSC member Marjon Pasmooij (Medicines Evaluation Board, The Netherlands), will present at the International Drug Repurposing Conference, #iDR24, co-organised by REMEDi4ALL, Beacon for Rare Diseases […]
Read More
On December 14, CDER and Duke-Margolis will host a jointly sponsored virtual public meeting on advancing the development of therapeutics through rare disease patient community engagement. The purpose of this public meeting is to highlight and build upon existing actionable approaches for engaging patients, patient groups, and related experts when developing necessary evidence to support […]
Read More
The Chan Zuckerberg Initiative (CZI) is expanding the Rare As One Network and a new cycle will be open next year (including for organisations outside the USA). A call for applications is now in effect. CZI has funded 2 Cycles so far, and learned how successfully patient organisations are engaging in research collaborations, generating significant […]
Read More
AFM-Téléthon opens the call for proposals for 2024, open to both French and foreign teams, that aims to support research along the following thematics: A. Fundamental research and physiopathology of diseases of the neuromuscular system B. Development of innovative therapeutic approaches for rare genetic diseases The selected projects will be subject to an agreement with […]
Read More
The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) announces the 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 and standardized approaches to […]
Read More
The Rare Care Centre provides globally connected holistic cross-sector care coordination, together with other streams of activities to build capability and capacity to improve the lives of children and families living with rare diseases. The Centre provides a state-wide service based at Perth Children’s Hospital. For families living remotely, having a child with a rare […]
Read More
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 […]
Read More
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 […]
Read More
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 […]
Read More