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National Ataxia Foundation


EL-PFDD Meeting

The Voice of the Patient Report (PDF) for Polyglutamine Ataxias and DRPLA is now available. This report summarizes information from the Externally-Led Patient Focused Drug Development (EL-PFDD) meeting we hosted on September 25, 2020. 
We would like to offer our thanks to the patient panelists, phone callers, and individuals who submitted email comments. You bravely shared your experiences with symptoms in your daily lives. With your input, we were able to document the significant physical and emotional impact for people living with Ataxia and hopes for future treatments. We heard directly from people living with Ataxia about their struggles with coordination, walking, speech, swallowing, and tremors. This information was included in the Voice of the Patient report. It will help key stakeholders understand what people living with Ataxia want from future treatments and clinical trials. It will ensure that the concerns of patients are taken into account.
We appreciate the efforts of the Ataxia community in making this a successful meeting! 

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 Patient Report Cover

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.

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