The year 2017 was a very rich and productive one for IRDiRC: 9 new members, 8 scientific publications, 6 new IRDiRC Recognized Resources, the official start of 3 Constituent Committees, and most significantly, the unveiling of 3 new goals supporting IRDiRC’s new vision for the next decade.
In February, IRDiRC organized its 3rd conference in Paris, France, to celebrate the considerable progress on its original goals in the past 6 years: the goal to deliver 200 new therapies was achieved in early 2017 – three years earlier than expected – and the goal for diagnostics is within reach. It signaled the time to look ahead to new challenges and shape IRDiRC’s vision for 2017-2027.
At the same time, the Constituent Committees started their activities with the mission to better coordinate, enhance information exchange and identify actions towards IRDiRC objectives. These committees are currently under the leaderships of:
• Daria Julkowska, representing the Agence Nationale pour la Recherche (ANR), for the Funders Constituent Committee,
• Sharon Terry, representing Genetic Alliance, for the Patients Advocates Constituent Committee, and
• Mathew Pletcher, representing Roche, for the Companies Constituent Committee.
In April, IRDiRC updated its governance to reflect the evolution of the Consortium and additionally empowered patient advocate groups to apply for IRDiRC membership. This call was heard as IRDiRC counts currently 6 additional patient advocacy organizations as members, in comparison to 3 in 2016.
In August, concomitantly with the publications of 3 vision and goal articles (1, 2, 3), IRDiRC announced its new vision and goals for the next decade via a press release that was widely disseminated to the members’ network. Additionally, IRDiRC also published articles on its Policies and Guidelines, A Global Approach to Rare Diseases Research and Orphan Products Development, International Cooperation to Enable the Diagnosis of All Rare Genetic Diseases, and the position statement of the Patient-Centered Outcome Measures Task Force, while contributed to some others, as on the importance of international collaboration for rare diseases research. More publications are currently in preparation to further disseminate the activities of the Consortium.
With the new goals in place, the Constituent and Scientific Committees set out to conceive and propose actions to support and advance these objectives. The first steps in the creation of the new roadmap for IRDiRC were presented at the Consortium Assembly meeting that took place last November in Tokyo, Japan. The Consortium is currently in the process of analyzing and prioritizing the proposed actions, and aims to deliver an aspirational and pragmatic roadmap in early 2018.
Through 2017, IRDiRC welcomed a total 9 new members. Among the 54 members, 31 participate as funders, 14 for companies, and 9 patient advocacy organizations. Importantly, the global coverage of the Consortium also expanded as IRDiRC welcomed its first members in India and South Africa while strengthening its presence in Australia, Canada, China, Japan, UK and USA. IRDiRC looks forward to welcoming more members, in particular from regions that are currently under-represented, ensuring their vital voice, experience and input are integrated into global rare diseases research agenda, bringing diagnostics and therapies to patients globally.