How to START? Four pillars to optimally begin your orphan drug development

How to START? Four pillars to optimally begin your orphan drug development

Abstract Drug development is a complex, resource intensive and long process in any disease area, and developing medicines to treat rare diseases presents even more challenges due to the small patient populations, often limited disease knowledge, heterogeneous clinical manifestations, and disease pro...

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Bibliographic Details
Main Authors: Anneliene Hechtelt Jonker, Liliana Batista, Michela Gabaldo, Virginie Hivert, Diego Ardigo, on behalf of the IRDiRC ODDG TF and IRDiRC TSC
Format: Article
Language:English
Published: BMC 2023-08-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Orphan drugs
Drug development
Stakeholder analysis and engagement
Data gathering
Rare diseases
Patients’ needs
Online Access:https://doi.org/10.1186/s13023-023-02845-9

Internet

https://doi.org/10.1186/s13023-023-02845-9

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