Category: IRDiRC

May 27, 2020 The commentary ‘Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook’ has been published in Nature Reviews – Drug Discovery. This guidebook, which has been authored by the IRDiRC Task Force “Orphan Drug Development Guidebook (ODDG)”, is aimed at facilitating drug development for rare diseases by organizing available tools […]

June 26, 2020 Senior officials from the European Commission (EC – DG SANTE), EMA and the United States Food and Drug Administration (FDA) held their 2020 bilateral regulatory dialogue meeting. The collaboration between EMA and FDA formally started in 2003 and the two agencies have now almost daily interactions, most of them structured around working […]

Survey assessing the Member States’ rules on health data in the light of GDPR The European Commission has initiated a study that aims to examine in which manners the processing of personal health date is governed across the EU and how this might affect the cross-border exchange of health data in the EU.The present survey […]

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 […]

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 […]

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 […]

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 […]

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” […]

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 […]

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 […]