Proposed Legislation Highlight
H.R. 1730/S. 670
What it would do: The STAT Act seeks to enact targeted, impactful, and attainable policy reforms at the Food and Drug Administration (FDA) to accelerate development of therapies across the spectrum of rare diseases and disorders and facilitate patient access to such therapies.
The STAT Act will expand existing authority within the FDA to create a Rare Disease Center of Excellence; create a Rare Disease and Condition Drug Advisory Committee; fund regulatory science and related activities to support development of therapies to treat very small rare disease populations; and strengthen rare disease patient access to FDA-approved therapies in both public and commercial plans.
Why it is important: The FDA plays a significant role in providing access to treatments and cures for the rare disease community. The actions in this bill would encourage, accelerate, and provide more access to treatments. Collaboration is also key to this legislation, providing all stakeholders input into the development of treatments and therapies.
Why it is important to the Cure CMD community: Like the rest of the Rare Disease community, we want treatments and cures! This legislation will help us get there.