New IRDiRC Roadmap 2020

IRDiRC Roadmap 2020 has been presented by Dr. Lucia Monaco, chair of the consortium, in the Jan – Feb 2020 newsletter recently released. IRDiRC has created #4 new actions to tackle relevant and urgent matters that are impacting directly the lives of people with rare diseases:

  • Chrysalis Project: Identify key criteria that would make rare diseases research more attractive to industry for research and development
  • Integrating New Technologies for the Diagnosis of Rare Disease: Identify new 1) technologies in development or in experimental use which are likely to increase the diagnostic rate for patients with rare diseases, 2) opportunities to enable the safe, widespread clinical adoption of the most elective technologies in a meaningful timeline
  • Shared Molecular Etiologies: Expand patient access to rare disease clinical trials by focusing on shared molecular etiologies underlying multiple rare diseases
  • Rare Disease Treatment Access Working Group: Address the goal of leaving no one behind, requiring that access to treatments be available for all rare disease patients, especially in low-and-middle-income countries