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.
