Patient-Focused Drug Development (PFDD) Meeting on FSGS

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

Researchers looking at a computer together

Externally Led Patient-Focused Drug Development (PFDD) Meeting on FSGS

View The Recorded Meeting

An exciting initiative by NKF and NephCure to inform the FDA about the patient perspective of living with focal segmental glomerulosclerosis (FSGS).


Due to the COVID-19 pandemic and to protect the health of our FSGS patients, this will be a virtual meeting.

Register Online

Anyone who lives with or is interested in FSGS - patients of all ages, care-partners, families and friends - are welcomed to attend.


Each person (e.g., child, spouse, etc.) must register separately.


Watch this page for updates during week of August 24:

  • You'll be able to submit comments on your experience with FSGS before the meeting
  • The link to join the meeting will be available August 27th

Take the patient survey

To prepare for the upcoming meeting on FSGS, we need your input on how FSGS affects your life.


Your response will help us make the best meeting possible.


All information you provide in the survey will be kept strictly confidential.


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 who are experts in the particular disease. The goal is to hear from patients who have the disease, in order for the FDA and pharmaceutical companies to understand the patient experience.

The EL-PFDD meeting on FSGS

The NKF and NephCure are conducting an EL-PFDD meeting on FSGS to inform the FDA about the patient perspective of living with this disease. Understanding the patient perspective may help the FDA make informed decisions on approvals of potential medicines for FSGS.
This meeting will be broadcast in a novel newscast format, with patient testimonies, remote audience participation, and Q & A.
The meeting will be co-chaired by two leading nephrologists who specialize in FSGS:
Dr. Suneel Udani Nephrology Associates of Northern Illinois
Dr. Laura Mariani University of Michigan

Event Details

Aug 28, 2020 | 10:00am – 3:15pm Eastern
This will be a virtual meeting. We will email you the link to join the virtual meeting in advance of the meeting date.

Voice of the Patient

After the meeting, a report titled "Voice of the Patient" will be sent to the FDA. This will be a reference for future decisions about potential medicines for FSGS.

Find Out More

Watch a recording of an informational webinar held about the upcoming meeting and view the slides from that meeting.

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.
Inform the FDA 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.
Reveals patients needs regarding new drugs, and what their preferences are for clinical trials for their disease.
Assists the FDA in knowing if a new drug addresses patient needs.


Patients know the FDA and drug sponsors 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 the disease.
In addition, these meetings help patient advocacy groups connect patients with their peers.

Pharmaceutical companies

Drug sponsors gain insights into the major concerns of patients. This helps the companies develop treatments and design clinical trials 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.
With knowledge gained from EL-PFDD Meetings, the FDA can advise pharmaceutical companies on developing potential drugs and therefore help to advance medicines that meet patients' needs.

Hear from Previous Patient and FDA Participants

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.


What do you do with the information from the Patient Survey?

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

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

The meeting will be on Friday, Aug. 28, 2019 from 10:00am to about 3:15pm

Can pediatric patients attend the EL-PFDD Meeting on FSGS?

YES! We welcome FSGS patients of all ages.

What happens after the meeting?

NKF and NephCure will write the Voice of the Patient Report. This will be sent to the FDA and will be used to help in their decisions to approve potential new treatments for FSGS.
The Voice of the Patient Report will be publicly posted on NKF's and NephCure's websites.

Still have questions?

National Kidney Foundation

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.

NephCure Kidney International

The only organization committed exclusively to accelerate research for effective treatments for rare forms of Nephrotic/Nephritic Syndrome, and to provide education and support that will improve the lives of those affected by these protein-spilling kidney diseases.

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.