On June 25, FDA issued a notice in the Federal Register requesting public comments on their Draft Guidance for Industry on Expedited Programs for Serious Conditions, which was issued to fulfill the Food & Drug Administration Safety & Innovation Act (FDASIA) (Public Law 112-144) mandate. Unfortunately, the draft guidance does not address the issues specific to rare disease drug development as required by FDASIA.
The EveryLife Foundation and its Working Group of patient organizations and industry stakeholders have collaborated on the attached comments on the draft guidance. We urge your organization to submit comments to the FDA requesting them to revise the guidance to ensure rare diseases have access to Accelerated Approval. If your organization is not able to submit comments, we encourage you to add your organization’s name in support of the attached comments which will be sent to the FDA. It is vital for the FDA to hear from the patient community on this issue. FDASIA requires the FDA to issue the final guidance by July 2014.
Public Comments are due to the FDA by Monday, August 26th. If you would like to sign on to the EveryLife Foundation comments, please email LWhite@EveryLifeFoundation.org by 3pm Eastern, Friday August 23rd.
For more information on this issue, please go to: EveryLifeFoundation.org.
Thank you for taking action to help spur the development of safe and effective treatments for rare disease patients.
Emil Kakkis, MD, Ph.D, President
EveryLife Foundation for Rare Diseases