Externally-Led Patient-Focused Drug Development Meeting

In Case You Missed It!

EL-PFDD Meeting for iTTP

The Ree Wynn Foundation hosted the first EL-PFDD Meeting for iTTP on January 26th.

View the video, On Demand, and submit your comments to be included in the Voice of the Patient Report.

The goal of this meeting was to provide the U.S. Food and Drug Administration (FDA), product developers, clinicians, and academic researchers an opportunity to hear perspectives from individuals with iTTP on the health effects and daily impacts of their iTTP, treatment goals, and decision factors considered when seeking out or selecting a treatment.

This meeting was conducted as a parallel effort to FDA’s PFDD initiative to more systematically gather patients’ perspectives on their conditions and available therapies to treat their conditions.

The EL-PFDD was half-day event. It focused on patient voices through panel discussions.

Submit Your Comments

 

Submit a comment on the topics we will be discussing in advance of the EL-PFDD meeting. Comments should be submitted individually.

The comments collected may be shared during the iTTP EL-PFDD and may also be used in the final Voice of the Patient report (with identifying information removed).


  • Which iTTP symptoms have the most significant impact on your life?

  • How does iTTP affect you on best and on worst days? Describe your best and worst days?

  • What specific activities, that are important to you, that you cannot do at all or as fully as you would like because of iTTP?

  • How have your symptoms changed over time? How has your ability to cope with the symptoms changed over time?

  • What do you fear the most as you get older? What worries and frustrates you most about your condition?

  • What are you currently doing to manage your iTTP symptoms?

  • How well do these treatments address the most significant symptoms of your iTTP?

  • What are the most significant downsides to your current treatments and how do they affect your daily life?

  • Short of a complete cure, what specific things would you look for in an ideal treatment for iTTP?

In Case You Missed It!

iTTP Community Webinar

The Ree Wynn Foundation hosted a Community Webinar on January 4, 2024. The event took place over zoom and was attended by 40 people. The audience consisted of patients with TTP, caregivers of patients with TTP, physicians, medical professionals and Pharmaceutical Industry partners. James Wynn of RWF along with James Valentine and Larry Bauer of Hyman, Phelps and McNamara; facilitate a discussion and question and answer period that prepares the community for the upcoming Externally-Led Patient Focused Drug Development Meeting. The EL-PFDD will be held Friday, January 26, 2024.

The Community Webinar answers questions from the community related to the EL-PFDD. Questions such as What is an EL-PFDD? What is TTP? Who should participate in the EL-PFDD? What is the role of FDA? How can I help with iTTP? Should my doctor attend the EL-PFDD? And many more. Watch the video above.

The Ree Wynn Foundation Announced the First EL-PFDD for iTTP

Externally-Led Patient-Focused Drug Development (EL-PFDD) meetings give FDA and other key stakeholders, including medical product developers, health care providers, 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 the FDA’s decisions and oversight during drug development.

On January 26, 2024, The Ree Wynn Foundation will host the Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting.

The goal of this meeting is to provide the U.S. Food and Drug Administration (FDA), product developers, clinicians, and academic researchers an opportunity to hear perspectives from individuals with iTTP on the health effects and daily impacts of their iTTP, treatment goals, and decision factors considered when seeking out or selecting a treatment. This meeting is being conducted as a parallel effort to FDA’s PFDD initiative to more systematically gather patients’ perspectives on their conditions and available therapies to treat their conditions.

The EL-PFDD is a half-day event that spans 4-5 hours. It will focus on patient voices through panel discussions.

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