Accelerating Access to Therapies for Rare Diseases
This act aims to speed up the development and availability of drugs for rare diseases. Citizens will gain faster access to new therapies, along with support in understanding treatment options and their financing. A dedicated institute and advisory committee will be established to coordinate efforts and provide guidance on rare disease matters.
Key points
Establishment of an Intercenter Institute on Rare Diseases and Conditions within the FDA to coordinate knowledge and efforts.
Creation of a Rare Disease and Condition Drug Advisory Committee, comprising experts and patient representatives, to advise on therapy development.
Introduction of the ALTITUDE Program to identify and address challenges in developing therapies for very small patient populations.
Implementation of an early third-party payor feedback program to inform coverage policies and issuing bulletins for Medicaid programs.
Increased financial support for research and development of drugs for rare diseases, including individualized therapies.
Expired
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