A PAG Leader Guide to Presenting the Patient Experience at Your EL-PFDD Session with the FDA.

A Global Genes Quick Guide is a resource for advocates focused on pressing topics causing challenges in their lives and communities. 

Leaders of rare disease advocacy groups are driving change by elevating patient voices to shape the development of new treatments. Externally-Led Patient-Focused Drug Development (EL-PFDD) meetings and FDA listening sessions offer powerful opportunities to spotlight the lived experiences of patients and caregivers, highlight unmet needs, and inform regulatory and research decisions.

These sessions provide a structured yet impactful forum to share what truly matters to those living with rare conditions—insights that often get lost in traditional drug development. Patient advocacy organizations are essential in organizing these efforts, serving as the bridge between communities, clinicians, researchers, and regulators.

This guide is designed to help you create a compelling and effective session that captures the urgency and realities of your disease community. With careful planning and thoughtful speaker selection, your meeting can influence the future of therapy development and ensure patients remain at the center of progress.

What Topics Should Be Covered in an Externally-Led PFDD Meeting?

The FDA recommends that all Externally-Led PFDD meetings center around two core topics:

Topic 1: Living with the Condition/Burden of Disease
This focuses on the daily impact of the disease—how it affects the lives of patients and caregivers physically, emotionally, and socially. This is your opportunity to communicate the real-world burden of the condition.

Topic 2: Current and Future Treatments
This topic explores treatment experiences—what options exist today – If any, what challenges remain, and what patients and families hope future treatments will address.

To make these topics meaningful and relevant, tailor the discussion to the unique aspects of your condition. For example, in Prader-Willi syndrome (PWS), issues like chronic hunger and behavioral challenges may take priority in Topic 1. In the STXBP1-related disorders EL-PFDD, communication and motor issues were highlighted, in addition to seizures.. Personalizing the sub-questions and discussion points ensures your community’s most pressing needs are clearly heard.

How Do You Select Speakers for Each Topic?

Speaker selection is both a strategic and emotional decision. The goal is to showcase the diversity and depth of your community’s lived experience. Here’s a process that works:

• Reflect the full spectrum/Diversity of Experiences: Choose speakers who represent varied experiences—across age, symptom severity, treatment journeys, race/ethnicity, gender, geography, and socioeconomic background.
  
• Match speakers to topics:
  
  • For Topic 1 (burden of disease), prioritize individuals who can clearly describe the daily impact of the condition.
  • For Topic 2 (treatment experiences), try to identify  those who have tried multiple treatments or participated in clinical trials.
    
• Balance perspectives: Include both individuals living with the condition and caregivers, when appropriate.
  
• Clarity and confidence: Choose speakers who are comfortable sharing their story and can stay focused within time limits.
  

How Many Speakers Can You Include?

The number of speakers you feature depends on your session length and how many topics (or sub-topics) you plan to cover. It’s smart to identify more speakers than you plan to use— having backups help ensure you’re always prepared. A typical format for each topic includes:

• 3–5 speakers, each sharing for 5–6 minutes
  
• Total panel time: 30–35 minutes
  
• Followed by a facilitated large-group discussion with audience input (live or virtual)
  

Speaker Format:
Each panelist presents individually and sequentially, not in conversation. This allows each voice to be heard clearly and ensures shared themes can be drawn out in the broader discussion. However, during the facilitated discussion, speakers may comment or expand on each other’s experiences in response to moderated questions or audience prompts.

Preparing Your Speakers

Speaker preparation is essential—not just for staying on-message, but for emotional readiness and confidence. Help your speakers feel supported and empowered with these key steps:

1. Host a Prep Call or Video Meeting
   Review the session format, timing, purpose, and key talking points. Some speakers may be able to discuss multiple points. Share the overall strategy, and clarify what each speaker will cover to ensure they stay focused on delivering their key messages.
2. Share a Written Framework
   Provide prompts to guide story development and keep messages aligned with FDA’s goals, such as:

• “A typical day in our lives looks like…”
  
• “What treatments have helped—or not helped—and why…”
  
• “If I could change one thing about how we are treated, it would be…”
  

3. Offer Supportive Editing
   Help speakers shorten or clarify their remarks while keeping their authentic voice.  Help speakers identify the key takeaways that are critical to share, so they can be clearly stated.

4. Practice and Rehearse
   Hold a virtual rehearsal so speakers can practice delivery, time themselves, and receive constructive feedback.
5. Reassure and Empower
   Remind them: Their lived experience is of prime importance. The FDA values their stories as essential insights for drug development and regulatory decisions.

How to Speak Succinctly

Many families have waited years to be heard—and naturally want to share everything. But for an EL-PFDD meeting, concise, impact-driven storytelling is key.

Why it matters:
FDA reviewers are listening for actionable insights that can inform drug development. Overwhelming detail can dilute your most important messages.

Tips for Staying Focused:

• Lead with Impact:
  Focus on what best illustrates your experience. Instead of telling the whole story, answer:
  “What’s one moment or symptom that most clearly shows how this disease affects your life?”
• Stick to 1–2 Core Messages:
  Think about what you want the FDA or researchers to remember most. Build your comments around those points.
  
• Use Guided Prompts:
  Try questions like:
  
  • “What’s the hardest part of a typical day?”
    
  • “What’s one thing you wish doctors or scientists understood better?”
    
  • “What would a meaningful improvement in your life look like?”
    
• Reassure and Redirect:
  Let speakers know: They don’t have to say everything at once. More details can be shared in written comments.

Sample Speaker Prep Guide for Externally-Led PFDD Meetings

Purpose: Share the guide below with your selected patient and caregiver speakers to help them prepare for a panel presentation during an FDA Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting.

General Format

• Each speaker will present solo for 5–6 minutes.
• You will be part of a 30–35 minute panel under one of two core topics:

o Topic 1: Health Effects and Daily Impacts

o Topic 2: Current Approaches to Treatment

• After the panel, there will be a moderated discussion with audience participation.

What to Talk About

Focus on specific, impactful details. Your story is powerful when it illustrates the real-world burden of the condition.

If you are speaking on Topic 1: Health Effects and Daily Impacts

• When was your (or your loved one’s) diagnosis?
• What are the most challenging symptoms or aspects of daily life?
• Describe a “day in the life.” What is a typical day like for you/your loved one?
• How does this condition affect:

o Daily routines?

o Emotional well-being?

o Family dynamics?

o Social or educational opportunities?

• What symptoms are most difficult to manage or most feared?

If you are speaking on Topic 2: Current Approaches to Treatment

• What treatments, therapies, or services have you used (including clinical trials)?
• What has helped, and what hasn’t worked?
• How do you weigh the benefits and downsides (side effects, costs, burden)?
• What are the biggest unmet needs?
• If you could create the ideal treatment, what would it do?

Tips for Writing and Speaking

• Stick to the time limit (5–6 minutes).
• Focus on 1–3 powerful examples instead of trying to tell everything.
• Speak from the heart but stay focused on what FDA needs to hear: real-world impact, unmet needs, and treatment gaps.
• Write it out in advance.
• Practice out loud or with a timer.
• You can read your remarks or speak freely—whatever feels right for you.
  
  

Thank you to Dorothea Lantz. This guide is based on materials prepared by Dorothea for use by the Prader-Willi Syndrome Association | USA in their EL PFDD sessions. 

Members of the Global Genes Global Advocacy Alliance can request mentorship pairings on topics like EL PFDDs – reach out to our team to join, or for assistance at [email protected] 

Take some time to read and watch these additional resources to help you plan your Externally-Led PFDD. 

Additional Resources:

• 7 Ways to Engage the FDA:  Watch the 2023 RARE Advocacy Summit session, discussing how to bring the patient voice into the discussion when communicating with regulatory authorities, and how to avoid missteps in the research process that are costly in terms of both time and money
• Planning an Externally-Led PFDD – an NIH Toolkit: Review a resource from the NIH to help your group plan a successful EL PFDD
• Externally-led Patient-Focused Drug Development Meetings,   Information directly from the FDA, including the goal and overview of the PFDD program, dates, list of upcoming EL-PFDD meetings, and past meeting reports by condition.
• Guide to Patient Involvement in Rare Disease Therapy Development. This guide from the EveryLife Foundation’s Rare Disease PFDD Workshop Series gives Patient Advocacy Leaders a set of actions to consider as they prepare for a PFDD session.
• Mapping Patient Inclusion in Drug Development: Watch the video recording from this Global Genes session to get a strong understanding of the drug discovery and preclinical development process
• From Molecules To Medicine  – A Global Genes Toolkit, providing an overview of how new drugs are developed and approved in the U.S., to help you participate more effectively in the development of treatments for rare diseases.
• Prader-Willi Syndrome EL PFDD:Download the Voice of the Patient Report and watch their EL PFDD recording
• STXBP1-related disorder EL PFDD -: Download the Voice of the Patient Report, watch their EL PFDD recording, and review their meeting agenda and transcript
• Canary Advisors partners and consults with advocacy organizations to prepare EL-PFDDs and patient listening sessions.  Examples they have worked on include:
  • Foundation for Sarcoidosis Research EL-PFDD recording
  • DEE-P Connections/Rare Epilepsy Network Listening session summary

Do you have a story of your success in participating in an EL-PFDD ?  Submit your story for us to consider publishing it here.

Global Genes 2025 Quick Guide Series is our way to respond to a common need we have heard for more information in the rare community. Think of them as the answer to one frequently asked question or pain point we have heard from our Rare Concierge, our online and in person communities and our Global Advocacy Alliance.  Each one is complete with links to additional related resources across various media types, both those that are authored by Global Genes and from outside sources as well.

This series is supported in part by: Amgen, Amicus, Beam Therapeutics, Biocryst,  BridgeBio, Chiesi, Mallinckrodt Pharmaceuticals, Merck, and Parexel