In recent years, we have seen Patient-Centered Outcomes, also known as Patient-Reported Outcomes (PROs), be implemented more regularly, specifically in the drug development and approval process. In fact, the Food and Drug Administration (FDA) created a Patient-Focused Drug Development Program specifically aimed at learning about how diseases impact patients’ lives and what an ideal treatment would look like from both patient and caregivers’ point of view. NephCure and our Voice of Patient volunteers continually invest their time to participate in various PRO projects, including Externally-Led Patient-Focused Drug Development Meetings with the FDA, patient and caregiver interviews, panels, and advisory boards with industry partners. These projects have led outside stakeholders towards developing deeper insight into patient priorities and have even played a part in making clinical trial protocols more patient-friendly.
Through daily interactions with glomerular disease patients and caregivers, NephCure consistently sees one very concerning trend. Patients don’t always understand, and many nephrologists aren’t consistently explaining, the correlation between proteinuria and decreased kidney function. The article in the May 2020 edition of the Clinical Journal of the American Society of Nephrology, “Identifying Outcomes Important to Patients with Glomerular Disease and Their Caregivers” clearly confirms this concern. The top 3 most important outcomes among patients include:
- Kidney function
- The need for dialysis or kidney transplant.
However, patients’ consideration of proteinuria ranks twenty-third in priority.
Over time, chronic proteinuria leads to kidney failure.1, 2 Proteinuria results from a dysfunction in the filtering units of the kidney. If a medication can treat that dysfunction, protein spilling into the urine will stop and the filtering units of the kidney (or glomeruli) will remain healthy. However, if protein continues to spill, the glomeruli will begin to scar, causing permanent damage and eventually a reduction in kidney function.
In our professional capacity here at NephCure, the “home” for all individuals who are affected by primary protein-spilling kidney diseases, we have heard too many firsthand accounts of adult patients who have been diagnosed with glomerular disease and whose nephrologist does not treat the proteinuria. Instead, tragically, these patients’ labs are monitored once or twice a year, and they are told to come back when it’s time for dialysis and/or transplant.
NephCure is working to change this practice by educating both the nephrology community and patient families that the number one treatment goal of any glomerular disease should be to stop proteinuria or reduce it as much as possible, which will in turn slow down the progression of the kidney disease and hopefully, in some cases, prevent the need for dialysis and/or transplant.
To that end, we have developed a new patient-focused brochure aimed at educating newly-diagnosed patients and families about the importance of reducing proteinuria in order to maintain their kidney function. We are seeking a collaboration with the broader nephrology community to distribute these at point of care and help NephCure provide support and resources to the primary glomerular disease population.
If you are interested in supporting us in this endeavor, you can request patient materials here. Thank you for your interest in helping us reduce proteinuria, save kidneys, and save lives.
Assistant Director, Patient Advocacy
1 Troost JP, Trachtman H, Spino C, et al. Proteinuria Reduction and Kidney Survival in Focal Segmental Glomerulosclerosis [published online ahead of print, 2020 Jul 23]. Am J Kidney Dis. 2020;S0272-6386(20)30846-5. doi:10.1053/j.ajkd.2020.04.014 https://www.ajkd.org/article/S0272-6386(20)30846-5/fulltext
2 Troost JP, Trachtman H, Nachman PH, et al. An Outcomes-Based Definition of Proteinuria Remission in Focal Segmental Glomerulosclerosis. Clin J Am Soc Nephrol. 2018;13(3):414-421. doi:10.2215/CJN.04780517 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5967666/