IRDiRC proudly announces that Dr Petra Kaufmann has been elected as Chair of the Interdisciplinary Scientific Committee (ISC), where she will serve with the newly elected Vice Chair Dr Domenica Taruscio. Dr Kaufmann is the Director of the Office of Rare Diseases Research (ORDR), and Director of the Division of Clinical Innovation (DCI) at the National Center for Advancing Translational Sciences (NCATS). Dr Taruscio is a research director of the National Center for Rare Diseases at Istituto Superiore di Sanità (ISS) and is responsible for the Italian National Registry of Rare Diseases. Dr Kaufmann will follow in the footsteps of Professor Hanns Lochmüller, who is stepping down after having served five years as Chair, but will remain committed to the work of the ISC, and its Task Forces. IRDiRC would like to thank Professor Lochmüller for all his continued efforts as ISC Chair, and is glad that he will remain a valuable member of the Committee.
In mid-February, IRDiRC Scientific Committees and IRDiRC Consortium Assembly meeting was held in Paris, France, prior to the 3rd IRDiRC Conference. These meetings had a dual aim: to discuss the progress that the Consortium Assembly, Scientific Committees, Task Forces and Constituent Committees have made over the past year, and also to work towards shaping and discussing the new set of IRDiRC goals for the next decade. Now that one of IRDiRC’s major objectives, i.e. delivering 200 new therapies for rare diseases, has been reached, and the other objective, the means to diagnose most rare diseases, has made significant progress, the time has come to determine the new vision and objectives for 2017-2027.
The 3rd International Rare Diseases Research Consortium (IRDiRC) Conference took place in Paris, France, on February 8-9, 2017. Around 300 participants from around the globe, including academics, researchers, industry leaders, policy makers and patient advocates, attended the meeting to help shape the future of rare diseases research.
The conference provided a unique opportunity for stakeholders active in the field of rare diseases research to reflect on the progress made in the last decade, particularly since the launch of IRDiRC in 2011, and to look forward to the challenges ahead.
Three plenary sessions opened the conference, presenting the history and achievements of IRDiRC, providing a global view of rare disease scientific achievement, and introducing the state of foundational, diagnostics, and therapeutic rare diseases research. In the first series of parallel sessions, the state of foundational, diagnostics and therapeutics research was investigated further, and included various success stories in each space. This was followed by a parallel session on new approaches to rare diseases, which highlighted the innovative contributions of young investigators towards advancing rare disease research goals. The third parallel session was aimed at exploring trends in the fields of regulatory and access, patient advocacy, and companies.
The conference closed with a forward-looking plenary, following the theme of the conference, transforming rare diseases research. In this session, the concepts for the next set of goals for IRDiRC and the rare diseases research community were outlined. A panel discussion followed, in which the audience was invited to comment, discuss, and ask questions, to further shape the vision and objectives for the next decade. An active and dynamic discussion with the audience and panel followed, which ended the lively and interactive 3rd IRDiRC Conference.
The conference was made possible through the generosity and continuous support of the European Commission, Japan Agency of Medical Research and Development, E-Rare and Orphanet. IRDiRC would like to thank all participants for their active involvement, and their continuous support in helping build and transform rare diseases research.
The 3rd International Rare Diseases Research Consortium (IRDiRC) Conference, will be held on February 8-9, 2016, in Paris, France. The conference gathers stakeholders from around the world to share their experience, knowledge and points of view to shape and advance rare diseases research through foundational, diagnostics, therapeutics and policy development. The full listing of speakers is available online on the IRDiRC Conference website.
Confirmed speakers for the 3rd IRDiRC Conference include:
- Christopher Austin, National Center for Advancing Translational Sciences (NCATS), USA
- Claudio Bordignon, MolMed S.p.A., Italy
- Kym Boycott, Children’s Hospital Eastern Ontario, Canada
- Ruxandra Draghia-Akli, European Commission – DG Research and Innovation (DG RTD), Belgium
- Jonathan Goldsmith, US Food and Drug Administration (FDA), USA
- Melissa Haendel, Monarch Initiative and Oregon Health & Science University, USA
- Edmund Jessop, National Health Service (NHS) England, UK
- Sangeeta Jethwa, Roche Innovation Centre, Switzerland
- Matt Might, University of Utah, USA
- Sophie Nicole, Université Pierre et Marie Curie, France
- Anders Olauson, Ågrenska Foundation, Sweden
- Slavé Petrovski, University of Melbourne, Australia
- Kiran Reddy, Clarus Ventures LLC, USA
- Makoto Suematsu, Japan Agency for Medical Research and Development (AMED), Japan
- Toshiki Takenouchi, Keio University, Japan
- Sonia Vallabh & Eric Minikel, Broad Institute, USA
- Nan Wu, Peking Union Medical College Hospital, China
The strong and exciting program, now online, consists of 4 plenary and 3 parallel sessions. The conference kicks off with an overview of the history and achievements of IRDiRC five year after it was launched. This will be followed by a global view of rare diseases research in 2017, and the state of foundational, diagnostics and therapeutics research.
The first parallel session elaborates further the state of research today, with interesting talks on topics including data standards and exchange, undiagnosed diseases and ex-vivo stem cell therapy. The second parallel session focuses on new approaches to rare diseases and highlights the work of innovative young investigators. The third parallel session provides an overview of trends in the fields of patient advocacy, companies, and regulatory and access.
The conference closes with a forward look plenary to discuss ways to transform rare diseases research over the course of the next 10 years, including a panel discussion on the next set of IRDiRC goals for 2017-2027. Participation and input of the community are vital so we can work together to bring diagnoses and therapies to all rare diseases patients.
Register now and join us in carving out the future of rare diseases research. Abstract submission and early registration deadline is extended to December 7, 2016.
We look forward to welcoming you in Paris, France, in February 2017!