Non-oncologic orphan drug approvals across the world: Types of evidence required and time to approval

https://www.sciencedirect.com/science/article/pii/S1359644625002429 Drug Discovery Today Non-oncologic orphan drug approvals across the world: Types of evidence required and time to approval A new analysis from the International Rare Diseases Research Consortium (IRDiRC) Regulatory Scientific Committee (RSC) sheds light on the persistent global delays patients face in accessing innovative therapies for rare diseases. The study reviews 53 orphan medicines approved between 2021–2022 across six major regulatory regions.