November 9th, 2023 - 10:00am - 3:30pm Eastern Time
Please share your perspective on Kidney Xenotransplantation
Patients and Care Partners, please share your perspective on Kidney Xenotransplantation in the comment box below. What do you want the FDA to know? Which risks would you be willing to accept, and why?
The comment period opens on October 20th and will remain open until December 10th.
- any stage of kidney disease (including early & late stages)
- dialysis (in-center hemodialysis, home hemodialysis, and peritoneal dialysis)
- on the kidney transplant waiting list
- not qualified for the kidney transplant waiting list
- currently living with a kidney transplant
- care partner or family member of any of the people listed above
- care partner or family member of someone who has passed away while awaiting a transplant
Prepare for the EL-PFDD Meeting | Important Dates
Important Dates | What | Where |
---|---|---|
Monday, September 11, 2023 | Webinar#1: EL-PFDD Meetings with the FDALed by James Valentine, JD, MHS, Meeting Moderator | Watch the webinar here. (passcode: Xln7fU^a) |
Thursday, October 5, 2023 3:00-4:00pm Eastern | Webinar #2: What is Xenotransplantation?Overview, Risks/Benefits, Clinical TrialsLed by Meeting Chairs: Dr. Vineeta Kumar & Dr. Peter Reese | Watch the recording of the webinar here. (passcode: =^#Eyz8*) |
Wednesday, November 8, 2023 7:00-9:00pm Eastern | Welcome ReceptionFor in-person attendees, register for the welcome reception here. | Bethesda Marriott (5151 Pooks Hill Rd, Bethesda, MD) |
Thursday, November 9, 2023 10:00am-3:30pm Eastern | EL-PFDD MEETINGPre-meeting breakfast & lunch will be included for all registrants. Register for the meeting here. | Bethesda Marriott (5151 Pooks Hill Rd, Bethesda, MD) |
Why Kidney Xenotransplantation? | The Impact of Patient and Care Partner Voices
Xenotransplantation (pronounced zee-no-tran-splan-TAY-shin) is when non-human cells, tissues, or organs are used to treat medical conditions in humans. More people need organs than what is available. On average, seventeen people die each day in the United States waiting for an organ transplant. Finding other sources for organs is critical to save lives.
Xenotransplantation is an experimental treatment. It is only allowed in extremely rare and serious cases. The FDA has not yet allowed clinical trials to begin. These trials will be important to show if this procedure is truly safe and effective.
Patient and care partner experiences are crucial throughout the advancement of this experimental treatment. This EL-PFDD meeting is an opportunity to express your thoughts, experiences, hopes, and concerns about clinical trials on Kidney Xenotransplantation - directly to the FDA and Xenotransplant companies.
We need your help: Please take the Patient & Care Partner Survey and, if interested, register for our meeting to make your voice heard.
Thank you to our meeting sponsors, eGenesis , Makana Therapeutics, and United Therapeutics
Submit Comments After the EL-PFDD Meeting on Fabry Disease
Comments will be accepted up to October 20, 2022 and will be incorporated into the Voice of the Patient report.
- Of all the symptoms of Fabry disease, which 1-3 symptoms have the most significant impact on you or your loved one’s life?
- How does Fabry disease affect you or your loved one on best and on worst days? Describe your best days and your worst days.
- Are there specific activities that are important that you or your loved one cannot do at all or as fully as you would like because of Fabry disease?
- How has your loved one’s ability to cope with the symptoms changed over time?
- What information would you want to know to inform your decision on enrolling in a clinical trial Fabry disease?
- In which type of trial would you be more likely to enroll – a trial for a drug designed to:a. Benefit the kidney alone but not slow or stop the overall disease progressionb. Benefit the heart alone but not slow or stop the overall disease progressionc. Reduce symptoms (e.g., pain, GI involvement, fatigue) alone but not slow or stop the overall disease progression
- For parents of pediatric patients: are you interested in enrolling your child in a clinical trial? If so, what factors would you consider in deciding whether to participate (e.g., age, whether symptoms have begun, whether already on ERT)? If not interested, why not?
- What are you currently doing to help treat your condition or its symptoms?
- How well does your current treatment regimen treat the most significant symptoms of your disease?
- What are the most significant downsides to your current treatments and how do they affect your daily life?
- Assuming there is no complete cure for your condition, what specific things would you look for in an ideal treatment for your condition?
Register for the Meeting
- You'll be able to submit comments on your experience with kidney disease approximately two weeks before the meeting.
- If you are unable to participate in-person and plan to watch the livestream, the link to the livestream will be available on the day of the meeting (Thursday, November 9th, 2023)
Need a hotel room? Book through our National Kidney Foundation room block using the following link: Book your room at the Bethesda Marriott | Book with the National Kidney Foundation (Deadline: October 17th)
Take the Patient & Care Partner Survey
What are EL-PFDD meetings?
Externally led patient-focused drug development (EL-PFDD) meetings bring together patients and care partners, US Food and Drug Administration (FDA) representatives, pharmaceutical companies, and doctors and researchers who are experts in the particular disease.
- For the meeting on kidney xenotransplantation, the goal is to hear from patients how living with kidney disease (current or past) may influence their perspectives on kidney xenotransplantation.
- This is intended to help the FDA make informed decisions on approvals of potential xenotransplant treatments, and xenotransplantation companies to design clinical trials that are meaningful for patients.
- For more information on kidney xenotransplantation, please visit our Website: Kidney.org - Kidney Xenotransplantation
- For more information on the FDA and previous EL-PFDD meetings, please visit FDA's website: FDA.gov: EL-PFDD Meetings
The EL-PFDD Meeting on Kidney Xenotransplantation
- Expectations/outcomes that are important (benefits of xenotransplantation)
- Burdens/tradeoffs that would be acceptable (risks of xenotransplantation)
- Patient's tolerance for a clinical trial (phase 1)
Do not forget to register for the meeting! Each person needs to register separately, whether you plan to attend in-person or join the live-stream, please register so we can send you the appropriate information!
The EL-PFDD meeting on Kidney Xenotransplantation
- This meeting will be in-person at the Bethesda Marriott (5151 Pooks Hill Road, Bethesda, Maryland, 20814) on Thursday, November 9th, 2023 from 10:00 AM to about 3:15 PM Eastern Time
- There are also many surrounding hotels that may fit your financial, travel, or other needs.
- This in-person meeting will have a live stream and a recording, but only in-person attendees will be able to fully participate. Please consider joining us in-person!
- Because the FDA wants to hear from only patients and their care partners, everyone else in the audience will be in listening mode. Patients and care partners will be invited to talk during discussion sessions.
- We will have a comment box for comments, questions, and input 2 weeks before the meeting, up to 30 days after the meeting.
Do not forget to register for the meeting! Each person needs to register separately, whether you plan to attend in-person or join the live-stream, please register so we can send you the appropriate information!
Why is this meeting important to me?
- How living with kidney disease, a kidney transplant, and/or dialysis may influence how you think about kidney xenotransplantation
- Your perspective on the safety, ethical, personal, societal, and religious considerations of kidney xenotransplantation
- The type of clinical trial for kidney xenotransplantation in which you might be willing to participate (or encourage a loved one to participate)
- Many other things the FDA and Xenotransplant companies want to hear from you so they understand what matters to people with current or past kidney disease and their care partners regarding alternatives to traditional kidney transplantation.
Event Details
Thursday, November 9th, 2023 | 10:00 AM – 3:15 PM Eastern Time
- This meeting will be in-person at the Bethesda Marriott (5151 Pooks Hill Road, Bethesda, Maryland, 20814) on Thursday, November 9th, 2023 from 10:00 AM to about 3:15 PM Eastern Time
- This in-person meeting will have a live stream and will be recorded, but only in-person attendees will have the full experience. Please consider joining us in-person!
- Because the FDA wants to hear from only patients and their care partners, everyone else in the audience will be in listening mode.
- The link to watch the live stream will be posted on this page on the day of the meeting (Thursday, November 9th, 2023).
- Please register so we can send you meeting updates and important details for the webinars, travel, and/or live stream.
Pre-Meeting Activities: Prepare for the EL-PFDD
Informational Webinar #1: EL-PFDD Meetings with the FDA
Led by our meeting moderator, James Valentine, JD
Watch the webinar here.
(passcode: Xln7fU^a)
Informational Webinar #2 on Kidney Xenotransplantation
- Led by Dr. Vineeta Kumar and Dr. Peter Reese, Meeting Chairs
Watch the recording of the webinar here.
(passcode: =^#Eyz8*)
Voice of the Patient Report
After the meeting, a report titled "Voice of the Patient" will be sent to the FDA. This will give the FDA a reference point for their decisions concerning the approval of potential kidney xenotransplantation procedures. The report will be available for the public (including xenotransplantation companies) to read.
Who benefits from EL-PFDD meetings?
Food and Drug Administration (FDA)
Patients
Patient advocacy groups
Xenotransplant Companies
FAQs
National Kidney Foundation
The National Kidney Foundation (NKF) is the oldest and largest non-profit health organization dedicated to preventing kidney diseases and improving the health and well-being of individuals and families affected by kidney disease. Education of patients and healthcare providers is a big part of the NKF’s work, and we support kidney patients by providing all sorts of educational materials, programs, and patient services.