Member Login About NKF Recursos en Español

Externally Led Patient-Focused Drug Development (EL-PFDD) Meeting on Kidney Xenotransplantation

Patient-Focused Drug Development Meeting on Kidney Xenotransplantation

View the Recorded Meeting

November 9th, 2023 - 10:00am - 3:30pm Eastern Time

LINK TO MEETING AGENDA

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.

You will not receive a read receipt. Your comments or questions may be read during the meeting and/or published in the Voice of the Patient Report.
This meeting, hosted by the National Kidney Foundation, is an important and exciting opportunity to inform the U.S. Food and Drug Administration (FDA) about the patient and care partner perspectives of kidney disease and kidney xenotransplantation.
We want to hear from people with the following experiences:
  • 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 FDA

Led 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 Trials

Led 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 Reception

For 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 MEETING

Pre-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

 
If you attended the EL-PFDD meeting on Fabry disease on September 19 and did not enter the discussion, or if you did not attend, but viewed the live stream of the meeting, you can enter the discussion by sending a comment using the form (link, or whatever the proper term is) below. Your comments should address the topics that were discussed in the meeting (outlined below).

Comments will be accepted up to October 20, 2022 and will be incorporated into the Voice of the Patient report.
 
Section 1: "Disease Symptoms and Daily Impacts"
  1. Of all the symptoms of Fabry disease, which 1-3 symptoms have the most significant impact on you or your loved one’s life?
  2. How does Fabry disease affect you or your loved one on best and on worst days? Describe your best days and your worst days.
  3. 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?
  4. How has your loved one’s ability to cope with the symptoms changed over time?
 
Section 2: "Clinical Trials for Fabry disease"
  1. What information would you want to know to inform your decision on enrolling in a clinical trial Fabry disease?
  2. 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 progression
    b. Benefit the heart alone but not slow or stop the overall disease progression
    c. Reduce symptoms (e.g., pain, GI involvement, fatigue) alone but not slow or stop the overall disease progression
  3. 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?
 
Section 3: "Current Challenges to Treating Fabry Disease"
  1. What are you currently doing to help treat your condition or its symptoms?
  2. How well does your current treatment regimen treat the most significant symptoms of your disease?
  3. What are the most significant downsides to your current treatments and how do they affect your daily life?
  4. 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

Anyone who has experienced kidney disease (current or past) OR anyone who is interested in kidney xenotransplantation is welcomed and encouraged to attend in person. This includes patients of all ages, care partners, families, and friends, as well as physicians, researchers, and other interested individuals.
 
Each person (e.g., patient, care partner, parent, spouse, etc.) must register separately.
 
Watch this page for updates before, during, and after 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)

Register Here

Take the Patient & Care Partner Survey

We need your input on how living with kidney disease (current or past) may affect your perspectives on kidney xenotransplantation. We need your input even if you can’t attend the meeting.
 
Your response will help us create the best meeting possible.
 
All information you provide in the survey will be kept strictly confidential.
 
For more information, please visit kidney.org or view our webpage on Kidney Xenotransplantation.
Take the 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

 
The meeting agenda consists of two brief presentations on kidney xenotransplantation and clinical trials by experts, a presentation by the FDA, patient panels, and extensive audience discussion sessions.
 
The broad discussion topics will be:
  1. Expectations/outcomes that are important (benefits of xenotransplantation)
  2. Burdens/tradeoffs that would be acceptable (risks of xenotransplantation)
  3. 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!

Register Here

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!

Register Here

EL-PFDD meeting Co-Chairs

The meeting will be co-chaired by two leading transplant nephrologists who are experts in Kidney Disease and Kidney Xenotransplantation:
Dr. Vineeta Kumar, University of Alabama at Birmingham
Dr. Peter Reese, University of Pennsylvania

Why is this meeting important to me?

This EL-PFDD Meeting is a unique opportunity for you and your care partners to speak directly to the FDA and the Xenotransplant companies and tell them:
 
  1. How living with kidney disease, a kidney transplant, and/or dialysis may influence how you think about kidney xenotransplantation
  2. Your perspective on the safety, ethical, personal, societal, and religious considerations of kidney xenotransplantation
  3. The type of clinical trial for kidney xenotransplantation in which you might be willing to participate (or encourage a loved one to participate)
  4. 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.
 
The FDA can use your input to judge whether new kidney xenotransplant trials seeking approval meet the needs of those needing a new kidney (and their care partners).
 
Xenotransplant companies will use your input when they design clinical trials for kidney xenotransplantation.

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.
 
Questions?
 

Pre-Meeting Activities: Prepare for the EL-PFDD

After you register, you will be added to our list and will receive updates as they become available.
 
We have two webinars to help you prepare for the EL-PFDD Meeting! We hope you can attend these in real-time, but we will also record the webinars so you can watch them if you are unable to attend!
 
 

  • 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)

The FDA gains understanding of what it's like to live with a particular disease. For this unique meeting, we will also explore how these experiences may shape your opinion of Kidney Xenotransplantation.
 
The FDA becomes informed of side effects and risks patients may be willing to accept to gain a certain level of symptom relief or slowing of their disease progression.
 
The FDA learns about patients' needs regarding new drugs, and what their preferences are for clinical trials for their disease.
 
EL-PFDD meetings assist the FDA in knowing if a new drug addresses patient needs.

Patients

Patients know the FDA and drug (or Xenotransplant) companies have heard their voices. 
 
Patients' experiences are validated, reducing feelings of isolation. 
 
Hearing other patients voice their experiences and needs helps patients to better self-advocate.
 
EL-PFDD meetings can help to bring new treatments to the market, which benefits patients.

Patient advocacy groups

EL-PFDD Meetings help these groups identify what needs exist for patient education and advocacy.
 
More effective advocacy increases public awareness and knowledge of a disease or an experimental treatment method like Kidney Xenotransplantation.
 
In addition, these meetings help patient advocacy groups connect patients with their peers.

Xenotransplant Companies

Xenotransplant companies gain insights into the major concerns of patients. This helps the companies develop treatments and design clinical trials that match patients' needs and preferences.
 
Xenotransplant companies learn which disease symptoms or treatment side effects are, or are not, tolerable by the patients. In this meeting, they can also learn about which benefits and risks lead to acceptance (or lack of acceptance) of Kidney Xenotransplantation. This helps the companies develop treatments that matter to patients.
 
With knowledge gained from EL-PFDD Meetings, xenotransplant companies receive advice from the FDA to develop trials and products to advance experimental treatments that meet patients' needs.

Hear from Previous Patient and FDA Participants

Patient Participant

I liked that everyone was invited to speak freely and participate. It was an excellent opportunity to tell our stories.

FDA Participant

I can say with certainty…that the stories that you shared with us today, your experiences with the disease will certainly impact how we at FDA will think about this disease, will approach our discussions with sponsors moving forward, and will really shape the development in the future of therapies for this disease.

Patient Participant

Being able to tell my story, be heard, and people empathizing and understanding the struggles this disease faces you with is indescribable.

FDA Participant

I think that your stories really highlight the urgency with which we need to find effective treatments for this disease, and also treatments that have better safety profiles than those that are currently out there.

Patient Participant

I liked that everyone was invited to speak freely and participate. It was an excellent opportunity to tell our stories.

FDA Participant

I can say with certainty…that the stories that you shared with us today, your experiences with the disease will certainly impact how we at FDA will think about this disease, will approach our discussions with sponsors moving forward, and will really shape the development in the future of therapies for this disease.

Patient Participant

Being able to tell my story, be heard, and people empathizing and understanding the struggles this disease faces you with is indescribable.

FDA Participant

I think that your stories really highlight the urgency with which we need to find effective treatments for this disease, and also treatments that have better safety profiles than those that are currently out there.

Patient Participant

I liked that everyone was invited to speak freely and participate. It was an excellent opportunity to tell our stories.

FAQs

If I take the Patient Survey, what will you do with the information I provide?
The information you provide will remain strictly confidential and will be used for planning the meeting.
 
Some of your responses may be included in the meeting, in the Voice of the Patient Report, or in other associated documents. Patient identification will always be removed and patient information will be presented as grouped data from many patients. If you have questions about how your responses will be used, please Email NKF

Is there a charge to attend the meeting?
No, this meeting is free and open to the public.

How do I register to attend the EL-PFDD meeting?
Click here to register for the meeting. We are encouraging everyone to join us in person, if possible! If you are unable to travel, the link to join the livestream will be available before the meeting.
 
EACH PERSON (e.g., PATIENT, CARE PARTNER, CHILD, SPOUSE, ETC.) MUST REGISTER SEPARATELY.

When and for how long is the EL-PFDD Meeting on Kidney Xenotransplantation?
The meeting will take place on Thursday, November 9th, 2023 from 10:00am to about 3:15 Eastern Time

Is this EL-PFDD Meeting on Kidney Xenotransplantation for pediatric patients?
YES! We welcome kidney patients of all ages and their care partners.

What happens after the meeting?
The NKF will write the Voice of the Patient Report. This will be posted on our website and sent to the FDA and can be used to help in their decisions to approve potential treatments involving kidney xenotransplantation.

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.

Pre EL-PFDD Webinar: Overview of Kidney Xenotransplantation

30 East 33rd Street, New York, NY 10016

© 2024 National Kidney Foundation Inc.