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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 to discuss the path toward new treatment options. These meetings serve as a platform for patients who have the disease to make their voices heard and provide the FDA and pharmaceutical companies insight into the patient experience.
This year, NephCure Kidney International and the National Kidney Foundation are coming together to conduct an EL-PFDD meeting on August 27, 2021, to inform these key groups about the patient perspective of living with membranous nephropathy (MN). The meeting will be held online. We invite anyone who has MN, lives with someone affected by it, or is interested in it to attend this critical meeting.
This year’s EL-PFDD meeting on MN will be co-chaired by Drs. Laurence Beck and J. Ashley Jefferson. Read on to hear from them about the importance of patient attendance at this EL-PFDD meeting.
As a patient with membranous nephropathy, why should I consider attending this meeting?
Dr. Jefferson: MN is a rare disease, but one that has a major impact on those who suffer from it. Although nephrologists taking care of MN patients understand the effects of this disease, many of the people designing and evaluating the results of clinical trials and testing new medications haven’t met anyone with MN and 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 what you would like to see from new treatments for MN in terms of how they are administered, how long the medication must be taken, the side effects, or anything else you think would be relevant to the development of new treatments.
The EL-PFDD meeting is a unique setting where patients, clinicians, scientists, industry leaders, and regulators are all gathered together to listen. Therefore, your attendance and insight vital is vital.
How does this meeting contribute to putting new medications for membranous nephropathy on the pharmacy shelf?
Dr. Beck: All new medications must go through a rigorous process to make sure they work effectively to treat the disease in the safest manner possible before these new therapies can be used by your doctors. This process involves clinical trials, often designed by the pharmaceutical company, and ultimate approval by the FDA if the medication shows success in the clinical trial.
By sharing your views on which factors are most important to you as a patient, both the pharmaceutical companies and the FDA can make sure to focus on these factors when designing and running the clinical trial and when evaluating the results of the trial for the medication’s final approval for use in treating MN.
Why does the FDA want to hear from patients?
Dr. Beck: It’s important for both the FDA and the companies making these medications to understand from you, the patient, what it’s like to live with MN, what the most troubling symptoms are, what side effects or inconveniences you’ve had from prior therapies, and what you would like to see in terms of future treatments for this condition. There are many factors that the FDA considers when deciding whether or not to approve a medication for clinical use, and patient-reported outcomes are becoming increasingly important in this decision-making process.
As a doctor, why do you believe the EL-PFDD is important?
Dr. Jefferson: This is an exciting time in the management of MN. We’ve made major advances in the science underlying this disease in the last 10-15 years, and we now have a much better understanding of what causes MN in many patients. The key now is to translate this knowledge into the most effective treatments with the fewest side effects.
To do this, we need the help of patients at an early stage in the design of clinical studies to help us understand what is and isn’t acceptable in a study and study medication, and make sure the approval process for new treatments includes the perspective of people living with the disease. In my clinic, I want to see new, evidence-based, effective, and safe treatments become available to treat my patients and alleviate the burden that this disease places on patients and their families.
Dr. Beck: As a physician who treats this rare disease, the EL-PFDD gives me the opportunity to hear directly from you, the patient, about your experiences with the disease and its therapy, as well as your goals and hopes for different, better, and safer therapies.
I’ve been interested in MN since I became a kidney specialist and have learned a lot from patients like you. There have been a number of exciting advances in the past few years, but it’s also clear we need to keep thinking hard and creatively about this disease and how to develop novel and more effective ways to safely treat it, keep it from causing further kidney damage, and prevent it from coming back once treated. Doctors, researchers, pharmaceutical companies, and the FDA highly value your input about these matters and your willingness to help us move forward together to meet these common goals.
