Category: IRDiRC

IRDiRC Newsletter has been just released

May – June 2020 Edition has been released today! This released edition has a flash news style (read 5-7 min) with info on nomination call for a Committee, leadership changes, a new company member, the IRDiRC Orphan Drug Development Guidebook paper, the recently launched IRDiRC official LinkedIn page, and a short summary of the success […]

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IRDiRC Companies Constituent Committee (CCC): NEW Leadership

June 3, 2020 Dr. Katherine Beaverson has been elected  as new Chair of the IRDiRC Company Constituent Committee (CCC), she is the Patient Advocacy Director for the Rare Disease Research Unit at Pfizer.  The IRDiRC CCC brings together representatives from the for-profit pharmaceutical and biotech that work together to identify common bottlenecks to expedite the execution of rare disease research in the company […]

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IRDiRC Companies Constituent Committee (CCC): NEW Member

June 3, 2020 IRDiRC welcomes Illumina Inc. as new member to its Company Constituent Committee, which  brings together representatives from the for-profit pharmaceutical and biotech members of IRDiRC. Illumina is a global leader in genomics and strives to enable clinical laboratories worldwide the ability to offer best-in-class solutions needed to achieve a timely diagnosis and […]

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FDA In Brief: FDA Requests Input on Rare Disease Clinical Trial Networks

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 […]

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IRDiRC at the ECRD

IRDiRC 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” […]

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IRDiRC Funders Committee: New Leadership

IRDiRC 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 […]

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New IRDiRC Publication

A 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 […]

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Webinar. Rare disease community response to COVID-19: Patient group and industry perspectives​

WORLD 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 […]

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Dr Lucia Monaco interview on Rare Diseases

The Chair  of the Consortium Assembly, Dr Lucia Monaco, has been recently interviewed by Raconteur for its Rare Diseases Report published by The Times – The Sunday Times- on its July edition.  Dr Monaco interview was one of the nine articles chosen for this focus and she  well highlighted the relevance of “the fight for equal access […]

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