SDS PFDD Meeting
EL-PFDD Meeting for Shwachman-Diamond Syndrome
The raw recording is now available below. Click on the play button on the player (bottom left), not the big button in the middle. The program starts about 26 minutes in. The documentary film starts at about 3:27. A trimmed video will be shared soon. Patients/Caregivers: Please share your experiences by July 4th!
Hosted by:
In Partnership with:
With financial support from:
This project is funded through a Patient-Centered Outcomes Research Institute (PCORI) Eugene Washington PCORI Engagement Award (EASO-42419)
Participate during the live meeting on June 4th!
Live audience polling will be available during the meeting through any mobile device or computer with an internet connection. Easy instructions will be shared during the meeting, and linked here: https://pollev.com/curesds
Live call-in via Zoom (voice only).
Click here to join the Zoom. It's like a green room, where our screeners will ensure you are ready before we proceed.
Meeting ID: 865 7720 8796 Passcode: 090873
Find your local number: https://us02web.zoom.us/u/koiJWmzwE
Additional options open for 30 days until July 4th:
Submit a comment via voice mail or text at +1 617-329-1838. Please include your first name, and country, at a minimum. Also available for 30 days after the meeting.
Submit a comment via email at patientvoice@sdsalliance.org. Please include your first name, and country, at a minimum. Also available for 30 days after the meeting.
Complete the 20-minute Community Survey here.
It focuses on the topics and questions of the SDS PFDD meeting.
What is an EL-PFDD (Externally-Led Patient-Focused Drug Development) meeting?
In short, the SDS PFDD meeting is a very special and unique opportunity for patients and families to come together and share our voices and stories with the purpose of accelerating research and improving care. The meeting is part of our "100 Voices to #CureSDS" campaign and is bound to be the biggest and most impactful gathering of SDS patients and their families, along with the FDA, researchers, and industry. Don't miss it!
The meeting is an Externally-led Patient-Focused Drug Development meeting, which is a special type of meeting developed by the FDA to give FDA and other key stakeholders, including medical product developers (pharma/biotech companies), health care providers (doctors), researchers, and the general public, a unique and important opportunity to hear directly from patients and their families/caregivers, and patient advocates about:
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the symptoms that matter most to them,
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the impact the disease has on patients’ daily lives, and
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patients’ experiences with currently available treatments.
This input can inform FDA’s decisions and oversight both during drug development and the review of a marketing application. It can also help inform medical product developers about the unmet needs and priorities of people living with a particular disorder. The meeting will be live-streamed right here. After the meeting, the recording and a meeting summary in the form of a voice-of-the-patient report will be prepared and published on this page. In these meetings, the patient’s experience is brought to the forefront for the FDA and all other stakeholders to gain a deeper understanding of the condition, in this case, Shwachman-Diamond Syndrome. “Externally-led” refers to PFDD meetings that are led by organizations outside of the FDA. Learn more about PFDD in the video below. The meeting -- as part of the 100 Voices to #CureSDS campaign - is focused on capturing, amplifying, and sharing the Patient Voice to accelerate the development of therapies and cures for people with Shwachman Diamond Syndrome. In addition to the meeting, the campaign includes surveys, interviews, and a resulting publication called the Voice of the Patient Report, which will serve as a resource for regulators and researchers to incorporate the patient perspective into all aspects of research, therapy development, and regulatory decision making.
SDS Community Chat to learn about the SDS PDFF meeting
How can SDS patients, families, and caregivers participate?
The purpose of the EL-PFDD meeting is to amplify and share the "patient voice". Your voice. We want to hear from the entire patient community: patients and families dealing with more severe or milder manifestations of SDS, young and old, from diverse backgrounds and socioeconomic opportunities, before and after transplant, common or rare complications and experiences, and more.
You can share your voice and have an impact on the future of SDS in many ways, including during, before, and after the meeting. Participation is voluntary. You can choose as many ways to participate as you like. The more the better.
Participate LIVE
online or in-person
Register today. Free!
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In-person attendees: Register by May 26th.
You will receive a FREE T-shirt. Travel support is available to qualifying families. Email us to inquire. -
Virtual attendees: Participate via polls, text, chat, voicemail, or live call-in. Details on top of this page and via email to registered participants.
Fill out the community pre-meeting SURVEY
To share your experience with SDS and to get familiar with the topics we will discuss at the meeting, fill out the anonymous PFDD survey. The survey will take 15-30 minutes to complete. The survey is IRB-approved. Results will be shared at the PFDD meeting and become part of the resulting Voice of the Patient report. Complete by May 28th.
Join as a speaker/ panelist (in-person*)
To apply to be considered a speaker/panelist (in person in Cincinnati, OH, on June 4th, 2025), fill out this form by 2/1/25. Selected speakers/panelists will receive coaching and speaker training to develop and deliver their talks effectively. Travel, accommodation, and childcare costs will be covered by the organizer.
Join the SDS Patient LENS Study
To share your experience through an interview-based research study (SDS Patient LENS Study), indicate your by filling our the informed consent form on the SDS-GPS platform. You can then schedule the interview at a time that is convenient for you. The study is IRB-approved and will start enrolling in late January or mid-February. The results of the study will be de-identified before analysis and publication and will inform PFDD planning, documentation, research prioritization, and other opportunities. Apply by 3/3!
* COVID-19 policy for in-person attendance of the PFDD meeting and all meetings hosted by the SDS Alliance:
SDS causes immune deficiency. As such, we take COVID-19 precautions especially seriously. We follow all local guidelines relevant to COVID-19 management. We welcome and encourage facemasks, including N95s, for the protection of yourself, your family, and the community. If you have any sign of a communicable disease, or have been in close contact with someone who has, please change your attendance to online instead of in-person. We are happy to accommodate your needs and enable full participation remotely. Please reach out to our team with any questions.
Meeting Summary / Voice of the Patient Report
Following the EL-PFDD Meeting on June 4th, 2025, we will compile a report in accordance with FDA's guidance. It will be available to download for free, here.
In the meantime, you may find key publications about Shwachman-Diamond Syndrome on the SDS Alliance Publications page.
Contact Us
For any questions, comments, and feedback, please contact the "SDS EL-PFDD" organizing team at patientvoice@sdsalliance.org or (+1) 617-329-1838.
Our programs, including this EL-PFDD meeting, are made possible through support from our donors, partners, and sponsors like you. Thank you. If you or your company would like to support our work please reach out to our PFDD team at patientvoice@sdsalliance.org.
