The International Rare Disease Research Consortium (IRDiRC)
Integrating national funding for rare disease research within common international goals and policies
For rare disease research, coordination of efforts is key to success, in order to maximise scarce resources. Worldwide sharing of information, data and samples, as well as harmonised regulatory requirements, are needed to boost research. Duplication of research efforts needs to be avoided, and links between teams working on similar issues must be created.
This is the premise behind the creation of the International Rare Disease Research Consortium (IRDiRC), an ambitious international initiative to pool resources and harmonise policy in order to accelerate research.
IRDiRC was launched in April 2011 following a European Commission and US National Institute of Health initiative. It brings together regulatory agencies, researchers, patient group representatives, members of the biopharmaceutical industry, and health professionals.
The two main objectives of the Consortium are to deliver 200 new therapies for rare diseases and diagnostic tools for most rare diseases by 2020.
“Patients, expertise and knowledge of rare diseases are indeed rare, and European and international cross-country collaboration has an extremely high added value,” says Valentina Botarelli, EURORDIS European Affairs Senior Advisor. “IRDiRC is the first truly global initiative to coordinate efforts across the world in the field of rare disease research. It holds promise for a leapfrog in rare disease research in the near future.”
IRDiRC will provide a scientific and policy framework to “guide research activities and foster collaboration among stakeholders to systematically explore all the opportunities to accelerate the development of diagnostics and therapies for rare diseases”. The Consortium will ensure fair sharing of the workload amongst countries and avoid funding overlap but will not fund research as such. Research funding will remain the prerogative of public (or private) funding agencies, but it will handle some “glue money” to fund coordination tasks. Each research funder has the freedom to join the Consortium provided that their research programme adopts the general objectives and policies of the Consortium, and contributes at least 10 million euros over 5 years.
At present, the following governmental funding agencies have officially joined the Consortium: the European Commission, the National Institute for Health (US), the Canadian Institute of Health Research (Canada), the National Research Agency INSERM (France), the Federal Ministry of Education and Research (Germany), the National Institute of Health Carlos III (Spain), the Organisation for Health Research and Development (Netherlands), the National Institute for Health Research (UK). Also the French Muscular Dystrophy Association (AFM) and Telethon Italia, non-governmental funding agencies, joined as full members.
So far there have been three workshops: a preparatory meeting in Reykjavík, Iceland in October 2010, an official kick-off meeting in Bethesda, USA in April 2011 and a third conference in Montreal, Canada in October 2011. Each of these milestones has reinforced the commitment by all stakeholders to the stated goals of the Consortium. They have all agreed on the need to establish and provide access to harmonised data and samples, perform the molecular and clinical characterisation of rare diseases, boost translational, preclinical and clinical research, and streamline ethical and regulatory procedures.
EURORDIS has played a frontline role throughout. Together with the AFM, our US strategic partner NORD, the US Genetic Alliance and other patient-led organisations we have been present at all three meetings to represent patients’ interests. This was also an opportunity to circulate EURORDIS’ most recent Position Paper on patients needs and priorities in rare disease research.
“Rare disease patients have always been the strongest voice advocating for international cooperation, notably in the field of research. The presence of rare disease patients guarantees the democratic involvement of civil society in policy topics that affect them directly,” says EURORDIS Chief Executive Officer, Yann Le Cam. “It gives greater credibility to the whole Consortium undertaking and chance to keep sight of the ultimate goal”.
EURORDIS has been particularly active in feeding the discussions on funding preclinical and clinical investigation. At the Montreal meeting, we worked closely with the EMA and FDA Regulators Working Group, to propose concrete measures to get closer to the goal of 200 new therapies by 2020. The main suggestion is to target those products which have the highest chances of success - orphan drugs having received designation in the EU and in the US. Pre-clinical and clinical projects developing appropriate models to generate suitable human data should also be encouraged in order to develop compounds, without orphan drug designation, that have a high potential for rare diseases. In all cases, scientific advice (Protocol Assistance in the EU) should be made compulsory and a condition for funding to maximise the chances of success.
The Consortium will be governed by an Executive Committee and three Scientific Committees, encompassing Diagnostics, Therapies and Horizontal aspects (including national history, biobanking, registries etc). The Scientific Committees will advise the Executive Committee on research priorities for future R&D investments and on progress in funded research.
Dr Maria Mavris, EURORDIS’ Therapeutic Development Director, was nominated to represent patients on the Therapies Scientific Committee. EURORDIS is also calling for patient representatives to be part of IRDIRC’s governance structure on the Executive Committee.
“In under two years IRDiRC has ensured the commitment of the funding agencies and the research community and agreed on its governance. If this dynamic is sustained, we can believe that more therapies will be available for rare disease patients in the next decade and beyond,” argues Yann Le Cam. “Let’s go for it!”
Find more information on the European Commission’s DG Research and Innovation website.
This article was first published in the February 2012 issue of the EURORDIS newsletter
Author: Paloma Tejada
Photo credits: © Inserm/Patrice Latron & EURORDIS
The International Rare Disease Research Consortium (IRDiRC) www.eurordis.org