Accelerating Access to Therapies for Rare Diseases
This act aims to speed up the development and access to treatments for rare diseases. It establishes a new office within the FDA to coordinate efforts, support research, and facilitate communication between drug manufacturers and insurers, potentially leading to faster access to innovative therapies for patients.
Key points
Creation of a new Intercenter Institute on Rare Diseases and Conditions within the FDA to serve as a central coordination and knowledge hub.
Establishment of the ALTITUDE Program to address challenges in developing therapies for very small patient populations.
Introduction of an early feedback program for third-party payors to inform coverage decisions for new rare disease therapies.
Formation of a Rare Disease and Condition Drug Advisory Committee, including experts and patient representatives, to advise on therapy development.
Expired
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