• Lani Knutson

STAT Act Aims to Speed Treatments for Rare Disease

Use the template here to urge your Congressperson to support it!

In 1983, the U.S. Congress passed The Orphan Drug Act, a law that facilitates and accelerates the development of orphan drugs. These drugs were previously undeveloped or neglected because of their limited potential for commercial gain (i.e. drugs for rare diseases with small populations)—in fact, prior to 1983, only 38 orphan drugs were approved for use. Yet since this law was passed in 1983, 770 orphan drugs have been approved by the FDA. In 2016, The 21st Century Cures Act provided funding to the National Institutes of Health (NIH) and the FDA to encourage development of medical products and devices, and to streamline the process of getting them into the market.

Both laws and the efforts of advocates have resulted in new treatments and therapies for rare diseases. However, less than 10% of rare diseases have a FDA-approved treatment. If we continue to develop and approve treatments at our current pace, it will take over 200 years to have treatments for all rare diseases. Yikes!

Thanks to the work of the Rare Disease Legislative Caucus and rare disease advocates from all over the US, the Speeding Therapy Access Today (STAT) Act of 2021 (H.R. 1730/S. 670) was introduced in both the Senate and House last month to address the speed in which treatments are being developed. 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:

  • Accelerate rare disease therapy development by expanding existing authority within the FDA to create a Rare Disease Center of Excellence

  • Optimize interagency coordination by creating a Rare Disease and Condition Drug Advisory Committee

  • Advance science-based regulatory policies to support development of therapies to treat very small rare disease populations

  • Facilitate access to therapies in both public and commercial plans

Like the rest of the rare disease community, we want treatments for all subtypes of CMD, and this piece of legislation will help us get there faster. We are asking members of the CMD community in the United States to contact their federal Senators and Representative and ask them to cosponsor this bill. It only takes a few minutes using this template provided by the EveryLife Foundation.

If you are interested in learning more about Cure CMD’s advocacy efforts, please contact Lani Knutson, Advocacy Team Lead, at lani.knutson@curecmd.org.


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