IRDiRC: Towards 1000 new rare diseases treatments by 2027
A new study has been published in the Rare Disease and Orphan Drugs Journal to describe the recent key steps undertaken by the IRDiRC Therapies Scientific Committee (TSC) to support the future approval of 1000 new therapies for rare diseases by 2027. The study highlights that in order to reach this goal, the IRDiRC has developed a new roadmap for the development and registration of drugs specific to rare diseases, leading to the publication of the IRDiRC Orphan Drug Development Guidebook. This guidebook provides tools and recommendations for developers to navigate through the RD development ecosystem. The IRDiRc also aims to foster drug repurposing for the treatment of rare diseases, notably by developing a Drug Repurposing Guidebook and presenting economic models suitable for repurposing approaches. The IRDiRC has also identified data collection in healthcare practice and international research agenda for rare diseases as two important goals to achieve.
