Patient-Focused Drug Development Meeting on Fabry Disease

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View the Recorded Meeting

An exciting initiative by the National Kidney Foundation and Fabry Support & Information Group to inform the FDA about the patient perspectives of living with Fabry disease.
Due to the COVID-19 pandemic and to protect the health of our patients with Fabry disease, this will be a virtual meeting.
We want to hear from patients who have all types of symptoms and organ involvement from Fabry disease (kidney, heart, gastrointestinal, etc.).

Register Online

Anyone who lives with or is interested in Fabry disease - with or without kidney involvement - is welcomed to attend. This includes patients of all ages, care partners, families, and friends, as well as physicians, researchers, and other interested individuals.
 
Each person (e.g., child, spouse, etc.) must register separately.
 
Watch this page for updates during week of September 12th:
  • You'll be able to submit comments on your experience with Fabry disease before the meeting
  • The link to join the meeting will be available on the day of the meeting (September 19th)
Register

Take the patient survey

We need your input on how Fabry disease has affected your life. 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.
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 Fabry disease, the goal is to hear from patients what it's like to live with Fabry disease with or without kidney involvement, so the FDA and pharmaceutical companies can understand the patient experience. This patient input can help the FDA make informed decisions on approvals of potential medicines for Fabry disease, and pharmaceutical companies to design medicines and clinical trials that are meaningful for patients.
 

The EL-PFDD meeting on Fabry disease

  • This will be a VIRTUAL MEETING on Monday, September 19, 2022 from 10:00 AM to about 3:15 PM Eastern Time
  • This meeting will be broadcasted with a NOVEL NEWSCAST FORMAT, with patient testimonies and remote audience participation
 
The meeting agenda consists of two brief presentations on Fabry disease by experts in the disease, a presentation by the FDA, four patient panels, and extensive audience discussion sessions. The broad discussion topics will be:
  1. Living With Fabry Disease Nephropathy: Disease Symptoms and Daily Impacts
  2. Clinical Trials in Fabry Disease Nephropathy
  3. Current Challenges to Treating Fabry Disease Nephropathy.
 
The meeting will be co-chaired by two leading nephrologists who are experts in Fabry disease:
Dr. Eric Wallace, University of Alabama at Birmingham
Dr. Anjay Rastogi, David Geffen School of Medicine at UCLA
 
Because the FDA wants to hear from only patients and their care partners, everyone else in the virtual audience will be in listening mode.

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 Pharma industry and tell them:
 
  1. What it’s like to live with Fabry disease on a daily basis
  2. What you need in new medicines to make a meaningful difference in your life
  3. The type of clinical trial in which you might be willing to participate
  4. Many other things the FDA and Pharma want to hear so they understand what matters to Fabry disease patients regarding new treatments for the disease.
 
The FDA can use your input to judge whether new drugs seeking approval meet the needs of Fabry disease patients.
 
Pharmaceutical companies will use your input when they develop new treatments and design clinical trials for Fabry disease.

Event Details

September 19, 2022 | 10:00am – 3:15pm Eastern Time
 
This will be a virtual meeting. The link to join the meeting will be posted on this page on the day of the meeting (September 19).
 
Questions?
 

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 medicines for Fabry disease. The report will be available for the public to read.

Find Out More

Soon after you register, you will receive an invitation to an Informational Webinar on the EL-PFDD Meeting. This webinar will provide the background so you can participate fully in the meeting.

Hear from Previous Patient and FDA Participants

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.

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. The link to join the live stream will be sent before the meeting.
 
EACH PERSON (e.g., CHILD, SPOUSE, ETC.) MUST REGISTER SEPARATELY.

When and for how long is the EL-PFDD Meeting on Fabry disease?

The meeting will be on Monday, September 19, 2022 from 10:00am to about 3:115 Eastern Time

Is this EL-PFDD Meeting on Fabry disease for pediatric patients?

YES! We welcome Fabry disease patients of all ages.

What happens after the meeting?

NKF and FSIG will write the Voice of the Patient Report. This will be sent to the FDA and can be used to help in their decisions to approve potential new treatments for Fabry disease.

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.

The mission of the Fabry Support & Information Group (FSIG) is to raise awareness of Fabry disease and its symptoms, to advocate for community needs and to lead the fight for the most effective treatment possible and eventually a cure. FSIG provides a link for the Fabry community to information, each other, the medical community, researchers, the pharmaceutical industry, and regulatory authorities. FSIG strives to support affected families and individuals lead fuller lives.