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. 

Does kidney disease put me at a higher risk?

Patients with protein spilling kidney disease are at higher risk for moderate to severe illness from COVID-19, especially when kidney function is below normal. Many patients are on immunosuppressive medications that work by keeping the immune system less active and in turn, makes it harder to fight infections such as COVID-19. Those with advanced kidney weakness, or on dialysis also have weaker immune systems. You may also have high blood pressure and possibly other comorbidities.

Which treatments are considered immunosuppressive medications?

The following medications that are used to treat protein spilling diseases are immunosuppressive medications:

• ACTHAR
• Belatacept
• Cellcept (Mycophenolate mofetil)
• Cyclosporine
• Cytoxan (Cyclophosphamide)
• Imuran (Azathioprine)
• Prednisone, prednisolone (Steroids)
• Rapamune (Sirolimus)
• Rituxan (Rituximab)
• Tacrolimus (Prograf)

I am not on immunosuppressive medications, but I am spilling protein in my urine. Am I still at risk and/or immune compromised?

If you are actively spilling protein in your urine, your immune system is weakened and you are likely at higher risk of developing severe illness from COVID-19. Because the filtering units of your kidneys are not filtering properly, they are spilling all types of protein including immunoglobulins.  Immunoglobulins, also known as antibodies, are proteins  in the blood that help fight bacteria or viruses and their loss makes your immune system weaker.

I am currently relapsing, should I wait to begin taking immunosuppressants until COVID-19 passes?

You should discuss the best plan of action with your nephrologist because each patient is different. However, your priority (even during the COVID-19 outbreak) should always be to stop your protein spilling and protect your kidneys.  If you generally respond to immunosuppressive therapy, it is likely a good idea to begin treatment. Again, please discuss the risks and benefits with your caregiver.

I am currently in remission, should I consider stopping or lowering my immunosuppressive medication?

During the COVID-19 outbreak, it is not recommended to stop or lower your medications. Stopping immunosuppressants can lead to rejection of a transplanted kidney or a relapse of protein spilling. You should never stop your medications without first speaking with your nephrologist first. There may be rare situations where medications can be reduced or stopped, but only under the care of your nephrologist.

Should I stop taking my ACE inhibitor blood pressure medication?

There have been some pre-clinical and clinical studies linking COVID-19 and the renin-angiotensin system. This has led to speculation as to whether use of ACE inhibitors or Angiotensin Receptor Blockers (ARB) increase the risk of moderate to severe illness in  COVID-19; NephJC and other professional societies have found that the evidence is insufficient, and in some cases conflicting—some data even suggesting that ARBs may be protect the lungs in viral pneumonia. Patients who are taking ACE inhibitors or ARBs should not change their medication unless told to do so by their nephrologist.

To read more about ongoing developments on this topic visit http://www.nephjc.com/news/covidace2

Will there be medication shortages?

There will likely be some drugs on backorder due to COVID-19. It’s difficult to know which medications will be impacted. It’s important not to panic or hoard medications. Call your doctor and/or pharmacy to see if you can get medications filled early or in 3-month supply. It may be best to begin using a mail-in pharmacy during the outbreak. You can monitor information regarding specific drug shortages here.

Should I Shelter in Place?

The key point to remember is that you cannot get the infection without exposure to someone who has the infection.  Therefore, you should make every effort to stay at home and limit your exposure. The degree of quarantine may differ depending on where you live. The best plan is to avoid going out for non-essential reasons and when you do to limit exposure to others and practice strict hand hygiene and clean things that you may have touched before washing your hands (such as your phone and your keys)  Getting fresh air and exercise close to your home, with proper social distance from others is certainly safe and may help cope with the stress arising from this situation.

Should I go to my doctors’ appointments?

It is important to first call your doctor to discuss if you need to physically go to your appointment. Many clinics are offering telemedicine, virtual appointments over the phone or on your computer. If your appointment requires testing or some form of physical treatment, you should discuss if it is essential or if you can reschedule for a later date.

I need labs drawn, where is the best place to go?

Many patients require frequent labs to stay on top of their care. You should contact your doctor to determine if your scheduled labs are required now, or if they can wait a month or two. If you must have labs drawn during the COVID-19 outbreak, consider going to a small outpatient lab rather than a lab in the hospital. Always use good hand hygiene and assure the phlebotomist does as well. Schedule an appointment if possible, go first thing when the lab opens and wait in your car for your turn, to limit your exposure.

Is it safe for immune compromised patients to order carry out meals?

Many restaurants are closed to the public to enforce social distancing but are still open for carry out. There is some risk in ordering food from a restaurant because employees may be infected with COVID-19, but there appears to be other a very small risk of food-related transmission. Should you choose to order carry out, be sure it’s from a valued, trusted business, you can transfer the food from the delivered containers to your own plates and you reheat the food prior to eating.  Other precautions include paying over the phone and asking them to put the meal in your trunk instead of passing the food through your window. Once home, repeat hand hygiene after assuring you have cleaned all surfaces.

How can I combat anxiety and depression during this time?

The fears, worry, and stress that come with being at increased risk for moderate to severe COVID-19 illness are real and can lead to even greater amounts of anxiety and depression. Being separated from friends and loved ones while social distancing can also cause feelings of isolation and loneliness. We want you to know that you are not alone in your struggles.

NephCure is offering a 4 week virtual series about “Cultivating Resilience During Anxious Times: Strategies for Staying Steady while Navigating Covid19” Please join us on Thursday, March 26, April 2, 9, & 16 at 7:00 PM EDT via https://zoom.us/j/890400605

We also offer several ways to connect with others going through similar journeys with protein spilling kidney diseases at NephSpace our online support community and direct connections through our Patient to Patient Connections Program. To talk with someone immediately, contact our Patient Advocate, Kelly Helm at khelm@nephcure.org or 610-540-0186 x38.