Patient-Focused Drug Development Meeting on Kidney Xenotransplantation
This meeting, hosted by the National Kidney Foundation, was 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.
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, fourteen 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 was an opportunity to express your thoughts, experiences, hopes, and concerns about clinical trials on Kidney Xenotransplantation - directly to the FDA and Xenotransplant companies.
Thank you to our meeting sponsors, eGenesis , Makana Therapeutics, and United Therapeutics
Read the Voice of the Patient Report Here!
The Voice of the Patient Report is a Meeting Summary from the EL-PFDD Meeting on Kidney Xenotransplantation, held on November 9th, 2023 in Bethesda, MD. This report reflects the National Kidney Foundation’s accounts of the perspectives of patients and care partners who participated in an Externally Led Patient-Focused Drug Development Meeting, an effort to support the FDA’s Patient-Focused Drug Development (PFDD) Initiative.
Meeting Transcript
Meeting Resources
Read the Voice of the Patient Report Here!
The Voice of the Patient Report is a Meeting Summary from the EL-PFDD Meeting on Kidney Xenotransplantation, held on November 9th, 2023 in Bethesda, MD. This report reflects the National Kidney Foundation’s accounts of the perspectives of patients and care partners who participated in an Externally Led Patient-Focused Drug Development Meeting, an effort to support the FDA’s Patient-Focused Drug Development (PFDD) Initiative.
Meeting Transcript
Other Meeting & Xenotransplantation Resources:
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: Overview of Kidney Xenotransplantation
- Led by Dr. Vineeta Kumar and Dr. Peter Reese, Meeting Chairs
- Watch the recording of the webinar here. (passcode: =^#Eyz8*)
Watch the Update Webinar | Xenotransplantation: Updates on Animal-to-Human Transplants
- Webinar took place on June 25, 2024
- Led by Dr. Tatsuo Kawai, Dr. Vineeta Kumar, and Dr. Peter Reese, Moderated by Heather Murphy
- Watch the recording here.
For more information on kidney xenotransplantation
- Visit our A to Z page: Kidney.org - Kidney Xenotransplantation
For more information on the FDA and previous EL-PFDD meetings
- Visit FDA's website: FDA.gov: EL-PFDD Meetings
Questions? Email NKF
- Xenotransplantation Initiatives | Medical Project Director: Heather Murphy heather.murphy@kidney.org
- Transplant Initiatives Manager: Ellen Florek ellen.florek@kidney.org
FDA PFDD Program
“Externally-led Patient-Focused Drug Development meetings give FDA and other key stakeholders, including medical product developers, health care providers, federal partners, an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during drug development and during our review of a marketing application.
Click here to learn more about Externally-led Patient-Focused Drug Development meetings.”
How do EL-PFDD Meetings Amplify the Patient Voice?
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 was 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.
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)
Voice of the Patient Report
The Voice of the Patient Report is an executive summary of the EL-PFDD meeting, held in Bethesda, Maryland on November 9th, 2023. This will give the FDA a reference point for their decisions concerning the approval of potential kidney xenotransplantation procedures. The report is available for the public (including xenotransplantation companies) to read.
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.