March 2021

One of the ways we can propel progress is through advocacy. It seems impossible, to be able to sit with legislatures and policy makers, and to drive forward new processes and projects that can positively impact all rare diseases.  The EveryLife Foundation turns this into reality for us, and you can get involved, too!  

In the beginning of 2021, I joined three Community Congress Working Groups: Public Policy, Regulatory, and COVID-19. All three are uniquely positioned to bring together rare disease patients and organizations, to discuss and push creative solutions to major challenges.  

This month, the STAT Act (Speeding Therapy Access Today Act of 2021), is in final review. This 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 improve rare disease coordination, stakeholder engagement, and policy development within FDA by expanding existing authority to create a Rare Disease Center of Excellence; inform rare disease policies and actions by creating 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.  

 You can Act and Share Your Story as well to help demonstrate the power of this Act and to drive home its importance with our state and national government officials.