August 4th 2020 Dr. Philip John (P.J.) Brooks has been elected as new Chair of the Interdisciplinary Scientific Committee (ISC). Dr. Brooks is a Program Director in the NIH National Center for Advancing Translational Sciences (NCATS) Office of Rare Diseases Research (ORDR). Dr. Brooks received his Ph.D. in neurobiology from the University of North Carolina at Chapel Hill. […]
Read MoreJune 25, 2020 To further encourage the development of treatments for rare diseases, EMA will waive all fees for scientific advice for academia developing orphan medicines.The academic sector plays an important role in the development of innovative medicines. Their scientific research is often at the source of novel methodologies and innovative medicines with a potential to benefit patients with rare diseases.Early […]
Read MoreJune 25, 2020 To further encourage the development of treatments for rare diseases, EMA will waive all fees for scientific advice for academia developing orphan medicines.The academic sector plays an important role in the development of innovative medicines. Their scientific research is often at the source of novel methodologies and innovative medicines with a potential to benefit patients with rare diseases.Early […]
Read MoreJune 23rd 2020 The Food and Drug Administration
Read MoreJune 23rd 2020 The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds for fiscal years (FY) 2021 – FY 2022 to support innovative and efficient clinical trials for rare diseases and conditions. These studies are intended to provide acceptable data to the FDA that will […]
Read MoreJune 2, 2020 THE CALL IS CLOSED Share your expertise with IRDiRC The Consortium is seeking experts to join its Diagnostics Scientific Committee to work together to the achievement of IRDiRC Goal 1 for 2027: All patients coming to medical attention with a suspected rare disease will be diagnosed within one year if their disorder is known in the medical literature; […]
Read MoreJune 2, 2020 THE CALL IS CLOSED Share your expertise with IRDiRC The Consortium is seeking experts to join its Diagnostics Scientific Committee to work together to the achievement of IRDiRC Goal 1 for 2027: All patients coming to medical attention with a suspected rare disease will be diagnosed within one year if their disorder is known in the medical literature; […]
Read MoreMay 27, 2020 The commentary
Read MoreMay 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 […]
Read MoreEuropean Commission, EMA and FDA agree new priorities to strengthen their collaboration on medicines
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 […]
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