IRDiRC, officially launched in 2011, was formed to unite public and private sector funders of research, patient advocacy groups, and scientific researchers to advance rare disease research worldwide. At its origin, IRDiRC had two main goals: to contribute to the development of 200 new therapies and the means to diagnose most rare diseases by the year 2020.
At its launch, IRDiRC was composed of 5 public research funders. Today, the Consortium includes nearly 50 members across Europe, North America, Asia, Australia and the Middle East, each – excepting the umbrella patients groups – committed to invest a minimum of USD 10 million in rare diseases research funding over 5 years. Indeed, research in rare disease (RD) is essential as many patients still lack an accurate diagnosis and appropriate treatment.
Information collected on the research project funded by the IRDiRC members show various trends. First the majority of projects correspond to basic or pre-clinical research, while clinical research represents around 8% of the awarded projects. Second basic and pre-clinical research projects mainly focus on rare neurological diseases and developmental disorders while clinical trials are mainly oriented towards rare cancers and rare neurological diseases. Third drug clinical trials constitute the vast majority of trials with more than half of them in earlier phases of development. The Constituent Committees use collected information to evaluate areas of need and addressing those gaps via coordinated implementation activities, such as the development of new funding opportunities to increase the likelihood of success in drug development and clinical translation.
The last six years have seen considerable progress on the IRDiRC’s primary goals. The goal for diagnostics is within reach. IRDiRC Diagnostics and Interdisciplinary committees, in collaboration with Orphanet, analyzed the number of diseases for which clinical test(s) is available: this number increased from 2200 in 2010 to 3600 by 2016. Regarding the goal to deliver 200 new therapies, it was achieved in late 2016 – three years earlier than expected. IRDiRC tracks the number of approvals of medicinal products with a marketing authorization and orphan designation since 2010 in Europe and the US. Between 2010 and 2016, 220 new drugs have reached the market, covering about 170 diseases.
IRDiRC’s Scientific Committees also developed the IRDiRC Policies and Guidelines to serve as recommendations for research funders and researchers to provide guidance on key topics such as ontologies, diagnostics, biomarkers, patient registries, biobanks, natural history studies, therapeutics, models, publications, intellectual property and communication. In addition, the Constituent and Scientific Committees continuously identify barriers that delay therapeutic research and diagnostic for rare disease patients and propose topics to work on for Task Forces (mainly external experts) and issue recommendations to drive policy and systemic changes. To support RD researchers, the Consortium also created a label to highlight key resources (i.e. platforms/tools/standards/guidelines) which, if used more broadly, would accelerate the pace of discoveries and translation into clinical services: the IRDiRC Recognized Resources.
For more details, read the full article