National Kidney Foundation's Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting on Alport Syndrome (AS)

Thank you for visiting the National Kidney Foundation's website for the Externally-led Patient-Focused Drug Development (EL-PFDD) Meeting on Alport syndrome (AS).
From here you can learn about:

About Us

The National Kidney Foundation, a major voluntary nonprofit health organization, is dedicated to preventing kidney disease, improving the health and well-being of individuals and families affected by kidney disease and increasing the availability of kidneys for transplantation. 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. Visit our website.
The Alport Syndrome Foundation's (ASF) mission is to improve the lives of those affected by Alport syndrome through education, empowerment, advocacy and the funding of research. The ASF is the leading independent resource for patients and families worldwide, collaborating with medical professionals and researchers across the globe to serve patients by centralizing medical information, hosting patient meetings and conferences, facilitating advocacy and education, and providing a network of support. Visit our website.

About EL-PFDD Meetings

EL-PFDD meetings bring together patients and their care-partners, representatives from the U.S. Food and Drug Administration (FDA), pharmaceutical companies interested in developing drugs for the disease, and doctors who are experts in the particular disease- all to hear from patients about the disease in question. In these meetings, the patient's experience is brought to the forefront for the FDA and pharmaceutical companies to understand. "Externally-led" refers to PFDD meetings that are led by organizations outside of the FDA.

EL-PFDD meetings benefit:

  • FDA in its role to approve new medicines
    • EL-PFDD meetings help the FDA to understand what it's like to live with a particular disease, and therefore, what symptoms and burdens matter most to patients.
    • These meetings inform the FDA on what side effects patients may be willing to accept to gain a certain level of symptom relief or slowing of their disease progression.
    • Such knowledge helps the FDA determine whether a potential drug's risks or deficits will be worth it's benefits to patients, and how closely it will meet patients' needs.
    • EL-PFDD meetings can support making medicines available to patients by helping to identify areas of unmet need in the patient population.
    • These meetings inform the FDA on patients' preferences regarding participation in clinical trials. This enables the Agency to advise pharmaceutical companies on their trials.
    • Ultimately, EL-PFDD meetings assist the FDA in knowing if a new drug will address the needs of patients.
  • Pharmaceutical companies
    • Drug sponsors obtain insights into the major concerns of patients, thus helping them develop treatments that coincide with patients' needs and preferences.
    • Drug sponsors learn certain disease symptoms or treatment side effects that are, or are not, tolerable by the patients and can develop drugs accordingly.
    • Advice to pharmaceutical companies from FDA- while a potential drug is being developed- can help to advance medicines that meet patients' needs.
  • Patients live with their disease
    • Patients know the FDA and drug sponsors have heard their voices (in person).
    • Patients' experiences are validated, they feel less isolated.
    • Hearing other patients voice their experiences and needs helps to refine patients' advocacy skills.
    • More effective advocacy heightens public awareness and knowledge of the disease.
  • Patient advocacy groups
    • Identify needs for patient education and advocacy
Through all the above, EL-PFDD meetings help the FDA to bring new, relevant medicines to patients.
More information on EL-PFDD meetings can be found at:

About the Alport Syndrome EL-PFDD Meeting

The NKF and ASF are conducting an EL-PFDD meeting for AS to learn the patient perspective on living with this disease. Understanding the patient perspective will help the FDA when it decides whether to approve potential medicines for AS.
The upcoming EL-PFDD meeting for AS will convene patients, care-partners, individuals from the FDA and pharmaceutical companies, and doctors who specialize in AS. These participants will assemble to hear from patients, in person, what it's like to live with AS.
The meeting will be on Friday, Aug. 3, 2018 from 8:30 AM – 1:30 PM at the College Park Marriott Hotel & Conference Center in Hyattsville, MD.
If you cannot attend the EL-PFDD meeting in person, you can join the meeting by webcast.
The agenda consists mainly of two patient panels and discussion sessions on living with AS and treatments for the disease.
Dr. Clifford Kashtan, an international authority on AS, will be the honorary chairman of the meeting.
Dr. Clifford Kashtan
Dr. Clifford Kashtan
University of Minnesota
The meeting will be co-chaired by two leading nephrologists who specialize in AS:
Dr. James Simon
Dr. James Simon
Cleveland Clinic Foundation
Dr. Michelle Rheault
Dr. Michelle Rheault
University of Minnesota
The output of the meeting will be a report entitled "Voice of the Patient", which will be sent to the FDA, who will use it as a reference for future decisions on potential medicines for AS.

If you are an AS patient, please take the Patient Survey.

About the Patient Survey

To prepare for the upcoming AS EL-PFDD meeting, the NKF and ASF need your input on how living with AS has affected your life. This topic will be discussed in depth at the meeting, but your responses beforehand will help us to plan the most successful meeting possible.
The Survey should take only about 10-15 minutes.
At the end of the Survey you will land on the page to register for the meeting in person or by webcast. You can also register now, but please take the Survey!
All information you provide in the Survey will be kept strictly confidential. If any information is reported, only aggregate information (grouped, no identification shown) will be used.

How to Register for the EL-PFDD Meeting on AS

To attend the EL-PFDD meeting in person or by webcast, click here.


  1. If I take the Survey, what will you do with the information I give you?
    • The information you provide will remain strictly confidential and will be used for planning the meeting.
    • Some information that you provide may be presented at the EL_PFDD meeting, in the Voice of the Patient report, or maybe in other documents. However, patient identification will always be removed before using, and only information that has been grouped from many patients will be reported.
  2. Is there a charge to attend the meeting?
    • No, this meeting is free and open to the public.
  3. How do I register to attend the EL-PFDD meeting- in person or by webcast?
    • At the end of the Survey, you will land on a registration page, or you can register now.
  4. Do I have to take the Survey to register for the EL-PFDD meeting?
    • No, but we really need your input from the Survey. By taking the survey, you will make an important contribution to the meeting- your responses will help us to plan a meeting that will have an impact, and by doing so, you will help the AS community.
  5. What if I want to participate in the meeting but can't attend in person?
    • The meeting will be broadcast by webcast, but you must register for it.
  6. Can patients who are kids come to the EL-PFDD meeting on AS?
    • YES! We need patients of all ages.
  7. Where, when, and for how long is the EL-PFDD meeting on AS?
    • The meeting will be on Friday, Aug. 3, 2018 from about 8:30 AM to 1:30 PM at the College Park Marriott Hotel and Conference Center in Hyattsville, MD.
  8. If I'm not from the Maryland area, where should I stay the night before the meeting?
    • We have arranged for a discounted rate ($155 per night) at the College Park Marriott Hotel and Conference Center, so you can stay in the same hotel as the conference site. In order to get this discounted room rate, you must first register here. After you complete the registration form you will be prompted through to our hotel request form. Please do not contact the hotel directly.
  9. What about food during the meeting?
    • A pre-meeting dinner the night before the meeting, and breakfast and a box lunch on the meeting day will be provided. All meals will be kidney-friendly.
  10. Can I bring my care-partner (parent, guardian, spouse) to the EL-PFDD meeting on AS?
    • YES, but he/she must register
  11. If I come to the EL-PFDD meeting on AS, do I have to do anything?
    • Some patients will be asked to serve on a panel to discuss what it's like to live with AS and to take the treatments for the disease.
    • If you are asked to be a panelist, you will be coached on this aspect.
    • If you are not on the panel, we hope you will actively participate in the discussion from the audience.
  12. What happens after the meeting?
    • The NKF and ASF will write a meeting report, the Voice of the Patient report. This will be sent to the FDA and will be used to help in decisions on whether to approve potential new treatments for AS.
    • The Voice of the Patient report will be posted on our organizations' websites; you will be able to read it.
  13. What did patients who attended a previous EL-PFDD meeting for kidney disease say about that meeting?

    Here are samples of patients' comments from the recent NKF EL-PFDD meeting on complement-3 glomerulopathy (C3G), when they were asked what they got out of the meeting:
    • "It made me feel part of a community and…made me feel not alone with my disease."
    • "It helped me understand my disease more."
    • "Great meeting with both professionalism and down to earth reality of situations. That the FDA and NKF [were]…willing to go to these lengths to learn of patients' issues and needs. That there is hope for better treatments."
    • "It was most beneficial learning that a lot of the symptoms I've experienced are the same as others, so it made me feel less lonely and like I can relate more to others surrounding me."
    • "It helped to grow my support system!"
    • "I liked that everyone was invited to speak freely and participate. It was an excellent opportunity to tell our stories."
    • "I made contact with a patient experiencing a similar disease complication that I have been struggling with, and was able to get a referral for a knowledgeable doctor. I learned some new info on the difficulties of drug trials for such a small patient population."
    • "I learned that I have the ability to be a voice and a leader for my [C3G] community."
    • "Lots of very good information about why treatments are so difficult to develop and approve for rare diseases…"
    • "Meeting other C3GN families [was very helpful because having] a rare and life-changing disease…can feel scary and overwhelming…because you feel incredibly alone."
    • "That there are many people out there that are working very hard to help us."
    • "Being able to tell my story, be heard, and people empathizing and understanding the struggles this disease faces you with is indescribable."
    • "This has truly changed my whole view on C3G."

Contact Us

If you have any questions, please email us: NKF or ASF.