You Can Change FSGS Treatment Options: EL-PFDD 2020

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.

This year, NephCure Kidney International and the National Kidney Foundation are coming together to conduct an EL-PFDD meeting, on August 28, 2020, to inform the FDA about the patient’s perspective of living with focal segmental glomerular sclerosis (FSGS). The meeting will be held online. We invite anyone who has FSGS, lives with someone affected by it, or is interested in it to attend this meeting. 

This year’s EL-PFDD meeting on FSGS will be co-chaired by two NephCure Specialists, Drs. Laura Mariani and Suneel Udani. The two of them answer the following questions regarding the importance of patient attendance at the meeting in August.


As a patient with FSGS, why should I consider attending this meeting? 

EL-PFDD co-chair and NephCure Specialist, Dr. Suneel Udani

Because FSGS is rare, most people designing and evaluating the results of clinical trials, that test new medications, have not met anyone with FSGS.  While clinicians taking care of patients with FSGS understand the impact the disease has on your lives, this impact is not something that can always be captured on a blood or urine tests, and therefore regulators who determine which medicine becomes available may not recognize the issues that patients face each day.

This meeting is your chance to explain not only the impact of the disease, but also the limitations of our current therapies. Each patient’s experience is unique, so hearing from as many different people as possible is incredibly helpful.

We encourage you to share not only what you would not only like, but also what you would expect from new treatments in terms of how they are administered, for how long the medication must be taken, the side effects, or anything else you think would be relevant to new treatments.  Each segment of the medical community (clinicians, researchers, industry leaders) benefits from hearing your perspective so that the new treatments available truly meet the needs of the intended population—you.

The EL-PFDD meeting is a unique setting where all these groups—patients, clinicians, scientists, industry, and regulators—are gathered together to listen. Therefore, your attendance and insight are dependent and vital to this meeting’s success.


How does this meeting contribute to putting new medications for FSGS on the pharmacy shelf?

EL-PFDD co-chair and NephCure Specialist, Dr. Laura Mariani

The comments from the meeting can inform the FDA about the urgent need for new, more effective, and less toxic treatment options for FSGS. The FDA tries to bridge communication between patients and the medical community. The FDA aims to develop criteria that not only represents the effectiveness of therapies, but also determines if these therapies truly help people feel better. In gathering patient feedback, this meeting will help companies design better studies to assess the symptoms most relevant to patients and help the FDA interpret the results of those studies with the patient’s needs in mind.

Why does the Food and Drug Administration want to hear from patients?

The FDA recognizes that patients and their family members are the experts on what it is like to live with FSGS and to take the currently approved medications.  This meeting can help the FDA better understand the impact of the disease and its treatments on patients.


As a doctor, why do you believe the EL-PFDD is important?

“As a physician, I have been frustrated with the treatment options for Nephrotic Syndrome since I learned what they were, how effective (or not) they are, and the side effects we ask patients to tolerate. However, for the first time in our history, there are more candidates for treatments and studies to investigate their impact. The potential for multiple new therapies has changed my perspective. Whereas in the past our bar for being excited for a new treatment was simply if it made an impact on abnormal lab tests, now we have to raise the bar to not only look at improving laboratory tests, but also how effective a treatment is at helping someone with the disease live better. We can never have that insight without the patient voice. Rather than hearing about patients’ experience after a new medicine has been approved, the EL-PFDD provides an opportunity for patients’ voices to truly shape the narrative and make sure that the approval process includes the perspective of those living with the disease and what is important to them.” -Dr. Suneel Udani

“I want to be able to offer patients more effective and less toxic therapies for FSGS.  To do that, we need to lower the barriers at every step of the drug development and approval process.  Hearing directly from patients is the best way for our partners in this process to understand the urgency of need in this area and to help all of us improve the lives of patients living with FSGS.” – Dr. Laura Mariani


To register for this year’s EL-PFDD meeting focusing on FSGS on August 28, 2020, please click here

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