Regulatory sandboxes: A new frontier for rare disease therapies

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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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