Voice of the Patient Report
The Voice of the Patient Report for Polyglutamine Ataxias and DRPLA (Links to PDF) is a comprehensive document created from the feedback gathered at the meeting. It was submitted to the FDA. Future applications for therapy approvals will use the report as a reference when evaluating the effectiveness of the treatment.
EL-PFDD Meeting on Ataxia
View the recording of the Externally-Led Patient Focused Drug Development Meeting for Polyglutamine Ataxias live-stream from September 25, 2020.
What is an Externally-Led Patient-Focused Drug Development Meeting?
An Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting gives the FDA and other key stakeholders, including medical product developers, health care providers, and federal partners an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during drug development and during review of a marketing application. EL-PFDD meetings strengthen understanding of disease and treatment burden. They also raise awareness and channel engagement within the patient community.
Resources
- Externally-Led Patient-Focused Drug Development Meetings – U.S. Food & Drug Administration
- The Voice of the Patient: A Series of Reports from FDA’s Patient-Focused Drug Development Initiative – U.S. Food & Drug Administration
- FDA Credits Recent Drug Approval to Patient Community Engagement; We Applaud the Agency for the Recognition of Its Legacy of PFDD – FDA Law Blog
