EL-PFDD Meeting

We’re excited to announce that NAF and Cure DRPLA will Host an Externally-Led Patient Focused Drug Development Meeting for Polyglutamine Spinocerebellar Ataxias and DRPLA!

September 25, 2020

10am-3pm EST
Virtual Meeting


Submit Your Comments Early

Please fill out the blue form below to submit your comments to be presented on September 25, 2020.

If you are a person living with SCA 1, 2, 3, 6, 7, 8, 17, DRPLA, or a caregiver, please share your experiences related to the following questions:

  • For the person who is affected with SCA/DRPLA, please identify 1-3 symptoms that have the most significant impact on daily living.
  • How do symptoms impact activities of daily living and quality of life?
  • What are you currently doing to manage your symptoms and how well do these treat your symptoms?
  • What are the most significant downsides to current treatments and how do they affect daily life?

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.

Learn More

Watch our informational webinar to learn more about what this meeting means, how the process will work, and how you can get involved. 

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