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Externally-Led Patient-Focused Drug Development Meeting on Ataxia

This is a recording of the meeting that was streamed live on September 25, 2020. 

Voice of the Patient Report

The Voice of the Patient Report for Polyglutamine Ataxias and DRPLA is now available to download. This report summarizes information from the Externally Led Patient-Focused Drug Development (EL-PFDD) meeting. It 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. 

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!

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. This engagement can help lead to approval of new treatments in the future.

"Benefit-risk assessment is the foundation for FDA’s regulatory review of human drugs and biologics; input from PFDD meetings can provide important data obtained directly from patients and caregivers."

FDA Law Blog

What Happens Next?

The Voice of the Patient Report is a comprehensive document created from the feedback gathered at the meeting. It will be submitted to the FDA. Future applications for therapy approvals will use the report as a reference when evaluating the effectiveness of the treatment. The Voice of the Patient Report for Polyglutamine Ataxias and DRPLA will be published on this page as soon as it is available.

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