Patient-Focused Drug Development Meeting

On November 16, 2015, at the suggestion of the US Food and Drug Administration (FDA), the Amyloidosis Research Consortium (ARC) hosted an externally led Patient-Focused Drug Development meeting to share with the Agency and other stakeholders (e.g., industry) the perspectives of people living with systemic amyloidosis, its impact on their daily lives, and their perspectives on approaches to treating amyloidosis. The meeting was conducted in accordance with the Agency’s Patient-Focused Drug Development (PFDD) initiative, an FDA commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) to more systematically gather patients’ perspectives on their condition and available therapies to treat their condition.

Part 1: Introduction & AL amyloidosis

Patients, caregivers, family members and experts share experiences with AL amyloidosis, including an introduction by Dr. Janet Woodcock, Director, Center for Drug Evaluation and Research, FDA.

Part 2: ATTR amyloidosis

Patients, caregivers, family members and experts share their experience with ATTR amyloidosis.

Voice of the Patient

As a result of the meeting ARC has submitted a comprehensive report entitled “The Voice of the Patient.” This is a detailed summary of the patient testimony presented at the meeting. This report will serve a critical function in communicating to both FDA review staff and Industry, the improvements that patients want and hope to see in their daily lives. Download here.

Cover Letter to FDA

Read the cover letter to Dr. Woodcock and colleagues submitted with the Patient Voice report. Download here.

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