IRDiRC meeting in Dublin seeks 200 treatments for rare diseases by 2020


April 30, 2013
This article first appeared in the April 2013 PXE International eNewsletter

Update: July 2013
Click here: IRDiRC 2013 Conference Report
Click here: Policies and Guidelines
Click here: Poster Abstracts
Click here: June 20, 2013 OrphaNews Europe editorial on conference results

The International Rare Diseases Research Consortium (IRDiRC) was launched April 2011 at the initiative of the National Institutes of Health (NIH) and the European Commission to foster international collaboration in rare disease research. From the start Sharon Terry, CEO of Genetic Alliance and PXE International, has been a part of the planning and is now a member of the Executive Committee. The overarching goals of the IRDiRC are to make possible the diagnosis of as many rare diseases as possible and to develop 200 new rare disease treatments by 2020.



The first IRDiRC Conference was held in Dublin April 16 and 17, 2013, gathering more than 400 rare disease stakeholders from around the world. Sharon Terry presented an opening plenary address entitled, The Haystack Is Made of Needles: A Global View of Rare Diseases. The presentation covered the opportunities and challenges for international partnerships in rare disease research.

Ms. Terry introduced Registries for All, which gives individuals the tools to store their health information online and safely participate in clinical trials and registries on their own terms, resulting in expedited research and more treatments for the benefit of all. She also introduced EspeRare, a foundation whose mission, in collaboration with patient groups and other key stakeholders, is to uncover the potential of existing drugs to address severe therapeutic unmet needs in rare disease. Ms. Terry is the first President of EspeRare, which is housed in Geneva, Switzerland.

“This conference is not about talking, it’s about the need to change the existing model. Are you ready to risk?” challenged Ms. Terry. She commented that though she is invited to present at almost all of the rare disease conferences around the world, she chose this one because it is action focused. Setting a common goal, committees and workgroups creates an urgency to actually make a difference sooner than later.