National Kidney Foundation's and IgA Nephropathy Foundation's Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting on IgA Nephropathy
Thank you for visiting our website for the Externally-led Patient-focused Drug Development (EL-PFDD) Meeting on IgA nephropathy (IgAN).
From here you can learn about:
- What are EL-PFDD meetings and why you should care
- The upcoming EL-PFDD meeting for IgAN
- How to take our IgAN Patient Survey
- How to register for the EL-PFDD meeting on IgAN
- How to reserve a room at the conference hotel
- How to contact the National Kidney Foundation or IgA Nephropathy Foundation with questions
- FAQs, including how patients felt about a previous EL-PFDD meeting on kidney disease
- The National Kidney Foundation and IgA Nephropathy Foundation
Externally-led Patient-focused Drug Development (EL-PFDD) meetings bring together patients and their care-partners, representatives from the US 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 four groups of stakeholders:
- 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 also 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.
- EL-PFDD meetings reveal to the FDA what patients need regarding new drugs, and what their preferences are for clinical trials for their disease.
- Such knowledge helps the FDA determine whether a potential drug's risks or limitations 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.
- Overall, EL-PFDD meetings assist the FDA in knowing if a new drug will address the needs of patients.
- Pharmaceutical companies
- Drug sponsors get insights into the major concerns of patients. This helps the companies develop treatments that match patients' needs and preferences.
- Drug sponsors learn which disease symptoms or treatment side effects are, or are not, tolerable by the patients. This helps the companies develop drugs that matter to patients.
- Advice to pharmaceutical companies from FDA- while a potential drug is being developed- can help to advance medicines that meet patients' needs.
- 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 increases public awareness and knowledge of the disease.
- Patient advocacy groups
- EL-PFDD meetings help these groups identify what needs exist 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 here.
The NKF and IGANF are conducting an EL-PFDD meeting on IgAN to inform the FDA on 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 IgAN.
The meeting will convene patients, care-partners, individuals from the FDA and pharmaceutical companies, and doctors who specialize in IgAN. These participants will assemble to hear from patients, in person, what it's like to live with IgAN.
- The meeting will be on Monday, Aug. 19, 2019 from 9:00 AM to about 3:30 PM at the College Park Marriott Hotel & Conference Center in Hyattsville, MD.
- There will be an informal pre-meeting buffet dinner on Sunday, Aug. 18, 7:00 PM at the hotel.
- If you cannot attend the EL-PFDD meeting in person, you can join by webcast, but you will not be able to participate in the discussion by webcast.
- During EL-PFDD meetings, only the moderator, patients, and caregivers talk; everyone else listens.
- The agenda consists mainly of two patient panels and audience discussion sessions on living with IgAN, treatments for the disease, and clinical trials for IgAN.
The meeting will be co-chaired by two leading nephrologists who specialize in IgAN:
Dr. Bruce Julian
University of Alabama at Birmingham
Dr. Pietro Canetta
Dr. Bruce Julian
University of Alabama at Birmingham
Dr. Pietro Canetta
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 IgAN.
If you are an IgAN patient, please take the Patient Survey.
To prepare for the upcoming EL-PFDD meeting on IgAN, the NKF and IGANF need your input on how living with IgAN 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 about 15 – 20 minutes.
ALL INFORMATION YOU PROVIDE IN THE SURVEY WILL BE KEPT STRICTLY CONFIDENTIAL. If information is reported, only aggregate information (grouped, no identification shown) will be used.
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 for the meeting, but please take the survey!
IMPORTANT: If you will attend the meeting with someone else, each person must register separately.
If you attend the meeting by webcast, you will not be able to participate in the audience discussion.
If you are an IgAN patient or a care-partner you can reserve a room for the night before the meeting (Aug. 18) at the College Park Marriott Hotel and Conference Center (site of the EL-PFDD meeting) at the discount rate of $159.
For attendees who are not patients or care-partners, please contact the hotel directly to reserve a room at the customary rate.
- 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 appear at the meeting, in the Voice of the Patient Report, or maybe in other documents. However, patient identification will always be removed before presenting information, and information will always be presented as grouped data from many patients.
- 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- in person? or by webcast?
- At the end of the Survey, you will land on a registration page, or you can register now.
- If you will attend the meeting with someone else, each person must register separately.
- Do I have to take the Survey to register for the EL-PFDD meeting?
- No, but we really need your input for the Survey to help us plan a meeting that will have an impact.
- Where, when, and for how long is the EL-PFDD meeting on IgAN?
- The meeting will be on Monday, Aug. 19, 2019 from 9:00 AM to about 3:30 PM at the College Park Marriott Hotel and Conference Center in Hyattsville, MD.
- Where should I stay the night before the meeting?
- We have arranged for a discounted rate ($159 per night) at the College Park Marriott Hotel and Conference Center, so you can stay in the hotel where the conference is.
- To get this rate, you must reserve your room and be an IgAN patient or care-partner.
- For attendees who are not patients or care-partners, please contact the hotel directly to reserve a room at the customary rate.
- If you prefer to stay elsewhere in the area, you may arrange to do so.
- What if I can't afford the travel and lodging expenses to come to the meeting?
- The NKF and IgAN have a limited scholarship fund to help patients who demonstrate a need for assistance. Please contact us if you need assistance.
- Can patients who are kids come to the EL-PFDD meeting on IgAN?
- YES! We need IgAN patients of all ages.
- 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 as kidney-friendly as possible.
- Can I bring my care-partner (parent, guardian, adult aid) to the EL-PFDD meeting on IgAN?
- YES, we want to hear from care-partners as well as patients.
- If I come to the EL-PFDD meeting on IgAN, 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 IgAN and what it's like to take the treatments for the disease.
- If you are asked to be a panelist, you will be coached on this part.
- If you are not on the panel, we hope you will participate from the audience in the general discussion.
- What happens after the meeting?
- The NKF and IGANF 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 their decisions on whether to approve potential new treatments for IgAN.
- The Voice of the Patient report will be posted on NKF's and IGANF's websites; you will be able to read it.
- What did patients who attended a previous EL-PFDD meeting for kidney disease say about that meeting?
- "This has truly changed my whole view on C3G."
- "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."
Here are samples of patients' comments from NKF's previous EL-PFDD meeting on complement-3 glomerulopathy (C3G), a rare kidney disease, when they were asked what they got out of the meeting:
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. Visit our website.
The IGA Nephropathy Foundation's (IGANF) mission is to eradicate IgAN, while also addressing the many problems faced by the patients and their families who live with this disease. As a recognized 501(C) (3) non-profit, public health organization, we are committed to research for a cure, public education, and patient service. Visit our website.