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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.
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.
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.
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.
Fill out the PFDD survey
To share your experience with SDS via an online survey, please fill out the PFDD survey on the SDS-GPS platform. The survey consists of about 15 questions and takes about 30 minutes to complete. The survey and program are IRB-approved and will be launched in early February. (If you already have an SDS-GPS account, you will be able to find the PFDD survey on your dashboard. If you don't have an SDS-GPS account yet, join SDS-GPS now).
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.
* 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.
Agenda (Draft!)
Tentative, subject to change!
All times in ET (New York Time)
10:00 - 10:05 AM
Welcome and Logistics
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Welcome and meeting/session overview by the Meeting Host
Dr. Eszter Hars, Mother of an SDS Patient, President & CEO, SDS Alliance
10:05 - 10:15 AM
Opening Remarks by a Representative of the FDA
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FDA's persective on PFDD meeting in general, and this meeting in particular
[Speaker TBD], Food and Drug Administration
10:15 - 10:30 AM
Clinical Overview of Shwachman-Diamond Syndrome
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Clinical Overview of Shwachman-Diamond Syndrome
Clinical SDS Expert [Speaker TBD]
10:30 - 10:35 AM
Introduction and Meeting Overview
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Introduction, Meeting Overview, Logistics, Orientation by the Meeting Host
Dr. Eszter Hars, Mother of an SDS Patient, President & CEO, SDS Alliance
10:35 - 10:45 AM
Demographic Polling
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Live polling of the audience in the room AND remote
Moderator
10:45 AM - 12:15 PM
Patient & Caregiver Panel 1: Living with SDS – Symptoms and Daily Impacts
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Panel discussion
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Audience polling
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Moderated audience discussion
12:15 - 1:30 PM
Lunch
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Lunch is provided to the in-person attendees. Please use the break to re-charge.
1:30 - 1:40 PM
Overview of Current Treatment and Management Approaches
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Clinical Overview of Current Treatment and Management Approaches
Clinical SDS Expert [Speaker TBD]
1:40 - 3:15 PM
Patient & Caregiver Panel 2: Current and future treatments for SDS
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Panel discussion
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Audience polling
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Moderated audience discussion
3:15 - 3:25 PM
Meeting Summary
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A summary of the meeting with a focus on the patient voice
[Speaker TBD]
3:25 - 3:30 PM
Closing Remarks
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Opportunities for further feedback and Voice-of-the-patient report; Patient focused activities moving forward.
Dr. Eszter Hars, Mother of an SDS Patient, President & CEO, SDS Alliance
Speakers (coming soon)
In alphabetical order
Patient/Caregiver Panelist/Speaker
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FDA Representative
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Dr. Eszter Hars
Dr. Eszter Hars, Mother of an SDS patient, and Founder/President/CEO/Science Director of the Shwachman-Diamond Syndrome Alliance. Dr. Hars holds a Ph.D. in Molecular Biology from the University of Medicine and Dentistry of New Jersey, where she studied cancer and leukemia. She has over 20 years of experience in scientific research and the biotech industry. As VP of Regulatory Affairs at CytoVera Inc., a lab equipment developer for hematopoeitic stem cell banking, Dr. Hars was in charge of regulatory approval of medical devices by the U.S. Food and Drug Administration. Dr. Hars has also managed business development as well as customer relationships at Quosa Inc., an information technology company, which was acquired in 2012 by Elsevier, the largest scientific publisher in the world. Currently, Dr. Hars is President of a biotech startup that specializes in developing new tools for various new therapies, including CAR-T cell cancer therapy and beta cell replacement therapy for diabetes. Dr. Hars has been engaged in SDS community building and volunteering wherever possible, since her daughter was diagnosed with Shwachman-Diamond Syndrome (SDS) in 2015. In 2020, Dr. Hars founded the SDS Alliance, a 501(c)(3) nonprofit organization. Through the SDS Alliance, Dr. Hars is dedicated to accelerating the development of new therapies for SDS.
Clinical Expert
Add...
Patient/Caregiver
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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.
