IRDiRC is delighted to share the manuscript “How to START? Four pillars to optimally begin your orphan drug development“, developed by the members of the IRDiRC Task Force “Orphan Drug Development Guidebook (ODDG)” together with the members of the Therapies Scientific Committee (TSC). The paper proposes a checklist for orphan drugs named START – a tool that provides an overview of the key pillars to be considered at the starting point of orphan drug development: STakeholder mapping, Available information on the disease, Resources, and Target patient value profile.
Published in the Orphanet Journal of Rare Diseases: https://link.springer.com/article/10.1186/s13023-023-02845-9.