June 2, 2020 The U.S. Food and Drug Administration (FDA) announced a Request for Information and Comment on Rare Disease Clinical Trial Networks. In particular, the FDA is requesting public input on practical steps and successful approaches relating to the start-up, implementation and sustainment of global clinical trials networks, including specific considerations for establishing such networks […]
Read MoreIRDiRC has been actively involved in the ECRD online conference that will take place this week: May 14 – 15, 2020.In particular, IRDiRC members joined several events planned during this 2-days event. On Thursday 14th, Dr. Durhane Wong‐Rieger was one of the speakers of the “Getting our rights ‘right’: An international framework for rare diseases” […]
Read MoreIRDiRC is delighted to announce the new leadership on the Funders Constituent Committee (FCC), Dr Adam Hartman, NIH, as Chair and Dr Catherine Nguyen, INSERM, as Vice-Chair. Dr Hartman and Dr Nguyen will be in charge for the next three years. The Committee mission is to facilitate high-level coordination of funding initiatives to maximize the impact […]
Read MoreA new IRDiRC publication is out! The Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook has been just published by Nature Reviews – Drug Discovery. The paper has been authored by the IRDiRC Task Force “Orphan Drug Development Guidebook (ODDG)”. This IRDiRC Guidebook is aimed at facilitating drug development for rare […]
Read MoreChrysalis Project The Funders Constituent Committee (FCC) has set up a jointly Task Force with the Companies Constituent Committee (CCC) and the Patient Advocates Constituent Committee (PACC). The overarching goal of the Chrysalis Project is to identify key criteria (in terms of investments of time and resources) that would make rare diseases research more attractive […]
Read MoreConsideration of legislative framework to support the diagnostic odyssey commonly encountered in the instance of rare disease. New paper on the undiagnostic odyssey, an important topic faced by many rare diseases patients and families – you can find it under “Diagnositc” -. Western Australia has led the way in Australia in being the first state […]
Read MoreWORLD ORPHAN DRUG CONGRESS USA WEBINARS organizes this event scheduled on Wednesday April 22, 12 – 1pm EDT. There are many unprecedented challenges facing the rare disease community due to COVID-19 — maintaining patient access to rare disease therapies, managing delays in clinical trials, and ensuring patient support are all top of mind. Join this webinar […]
Read MoreThe Rare Diseases Are Not Rare! Challenge is a competition sponsored by the National Center for Advancing Translational Sciences (NCATS), which is part of the National Institutes of Health. We are asking entrants to use their creativity to raise awareness.This is an opportunity that combines art, science and medicine with the hope that it will bring enjoyment […]
Read MoreECRD will take place ONLINE on 14-15 May (more information) The ECRD is recognised globally as the largest, patient-led rare disease event in which collaborative dialogue, learning and conversation takes place, forming the groundwork to shape future rare disease policies.Leading, inspiring and engaging all stakeholders to take action, the Conference is an unrivalled opportunity to network and exchange invaluable knowledge with all stakeholders in the […]
Read MoreIRDiRC has created a new section on its Resources and Recommendations in order to provide additional knowledge on rare diseases from a different perspective, the Social and Humanities Sciences. The first two articles have been recently published on undiagnosed and team work: Capturing Team Dynamics in the Wild: The Communication Analysis Tool by Klonek et al., […]
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