Regulatory sandboxes: A new frontier for rare disease therapies

Regulatory sandboxes have emerged as an innovative mechanism to facilitate the development and approval of new technologies, including pharmaceuticals. Their application in the context of rare disease therapies presents a promising avenue to accelerate the development, approval, and access to disease-modifying and life-saving therapies. Given the complexities of rare diseases and the regulatory hurdles faced by orphan medicinal products, regulatory sandboxes offer a structured yet flexible environment where new regulatory approaches can be tested and refined.
Acces this publication

publication

X29500087

Year of publication

2025

Author

Galliano Zanello , Violeta Stoyanova-Beninska , Oxana Iliach , Daniel Scherman , Samantha Parker , David A. Pearce

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