ARC Creates Pathway for Patient Engagement with FDA
Much has been said about the need to put patients at the center of drug development. The Amyloidosis Research Consortium (ARC), is doing just that. The FDA’s Patient-Focused Drug Development initiative is a commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) that aims to more systematically gather patient perspectives on their condition and available therapies to treat their condition. This initiative gives patients with a particular disease the opportunity to share their personal experiences and to highlight the unmet needs of patients affected by the disease.
ARC is concerned, with drugs in development for amyloidosis, that the FDA should have full knowledge of the impact of these diseases. To meet the unmet needs of amyloidosis patients it is imperative for the FDA to have a better understanding of amyloidosis patients and ARC was not prepared or willing to wait years for the opportunity to have the voices of amyloidosis patients heard. ARC is the first organization to work independently with the support of the FDA to plan and deliver a Patient-Focused drug development meeting, and report. The meeting was held last November and written up in a previous post entitled: Patient Forum with FDA
As a result of the meeting ARC has submitted a comprehensive report entitled “The Voice of the Patient”. This is a detailed summary that includes the patient testimony presented at the meeting and their personal perspectives shared in written submissions from patients.
This report will serve a critical function in communicating, to both FDA review staff and industry, what improvements patients want and hope to see in their daily lives. The FDA believes that the long-term impact of this program will be a better, more informed understanding of how we might find ways to develop new treatments for these diseases.
The end of the Patient Voice document incorporates the patient’s input into a benefit-risk assessment and provides important insight that will aid in the FDA’s understanding of what patients truly value in a treatment and inform the agency’s evaluation of the benefits and risk of future treatments for amyloidosis patients.