The members of the International Rare Disease Research Consortium’s (IRDiRC) Therapies Scientific Committee (TSC) have discussed and agreed on a specific set of recommendations to guide policies and funding strategies so as to reach its goal of 200 new therapies by 2020, based on IRDiRC Polices & Guidelines which were adopted in April 2013. They focus on the improvement of guidelines for the clinical development of orphan drugs; the alignment of scientific and regulatory guidance and the enhancement of the continuous data collection and assessment all along the life cycle of therapy.
It is expected that the implementation of all IRDiRC’s policy and funding recommendations as a whole will significantly increase their overall impact. These recommendations address a specific number of bottlenecks associated with biomedical research which will further foster the collaboration of rare disease therapy development. While respecting the individuality of each regulatory and health technology assessment agency, the recommendations encourages collaboration among the different agencies on a number of fields.
The recommendations of the TSC are structured around three axes; recommendations for IRDiRC Funding Organizations, which are subdivided in strategic recommendations, criteria for research funding, priorities for research funding and priorities for gap analysis funding. A second axis of recommendations is related to regulatory processes and the final one towards the metrics of progress.