The Food & Drug Administration (FDA) recently issued a draft guidance on Expedited Approval Programs which they say fulfills the FDASIA mandate on Accelerated Approval for Rare Diseases, however the guidance does not address specific issues related to rare disease drug development as required by FDASIA. We need to do better!
Representative Bilirakis (R-FL) is circulating a letter in Congress calling on the FDA to fully implement FDASIA and give rare disease access to Accelerated Approval. URGENT ACTION is needed from the patient community to ask Members of Congress to sign onto the letter.
Please CLICK HERE to call your Member of Congress. It’s easy & only takes 5 minutes. Just enter your address & your Congress Member’s phone number will be displayed with a sample script you can use to ask your Member to sign onto the letter.
Access to Accelerated Approval brings down the time and cost to development treatments leading to a surge in investment and development activity for even the most rare disorders, giving more patients with rare diseases earlier access to safe and effective treatments.
To see the Congressional Letter to FDA & to learn more about the issue, click here.
Thank you for taking action!
Emil Kakkis, MD PhD
President & Founder
EveryLife Foundation for Rare Disease