While dialysis is a life-saving therapy for patients with end stage kidney disease (ESKD), a variety of symptoms occur following treatment that significantly diminishes the quality of life of people living with kidney disease. A 2009 study published in the Clinical Journal of the American Society of Nephrology (CJASN) reported that patients on maintenance dialysis experience 11 unique symptoms. Patients have identified the need to alleviate these symptoms as a key research area, prioritizing it above other health outcomes. Alleviation of these symptoms could result in increased quality of life and better patient-reported outcome measures. Despite the patient demand for new therapies to address these symptoms, no drug or device has been approved by the US Food and Drug Administration (FDA) to date.

The Kidney Health Initiative (KHI), a public-private partnership between the American Society of Nephrology and the FDA, responded to this patient care issue by assembling a workgroup to confirm and prioritize symptoms experienced by ESKD patients. After identifying three prioritized symptoms, the group then identified near-term, actionable research goals for these prioritized symptoms. This two-part approach highlighted the innovation gap between the experience of patients and the products available to the physicians to help treat the symptoms. The workgroup was co-chaired by Jennifer E. Flythe, MD, MPH, FASN, from the University of North Carolina Kidney Center, and Rajnish Mehrotra, MD, FASN, from the University of Washington. The group included members from research institutions, pharmaceutical and biotechnology companies, healthcare organizations, government representatives, and patient organizations. They began work in 2015 and published two papers in CJASN in March and November of 2018.

The first paper titled, “Symptom Prioritization among Adults Receiving In-Center Hemodialysis: A Mixed Methods Study”, describes the outcomes of a survey that was sent to patients to prioritize the symptoms they experience after dialysis treatments in order for new products to be developed to address these symptoms. This was the first published study evaluating these issues. The study included focus groups across the United States and an online survey. From these results, three physical symptoms were identified as priorities to patients: insomnia, fatigue, and muscle cramping. In addition, mood symptoms including anxiety, depression, and frustration were a common theme. The group decided to focus the next phase of the project on the three identified physical symptoms. Read more about this paper here.

KHI hosted a workshop of various stakeholders, including patients and care partners, on January 8-10, 2018, in Washington DC to discuss opportunities to address three symptoms identified in the study – fatigue, muscle cramping and insomnia. The goal of the meeting was to:

• Establish collaborative relationships to advance the development of therapies for patient-prioritized symptoms;
• Develop a shared understanding of the symptoms that patients prioritize for intervention and the factors driving those decisions; and
• Identify promising opportunities and pathways for future symptom alleviation research 

Read more about this workshop here .

The second paper published by the workgroup, “Fostering Innovation in Symptom Management among Hemodialysis Patients: Paths Forward for Insomnia, Muscle Cramps, and Fatigue”, summarizes the pathophysiology of the three prioritized symptoms. The paper identifies key knowledge gaps, acknowledges factors that challenge development of new therapies, and offers the nephrology community actionable research goals for insomnia, muscle cramps, and fatigue. The recommendations outlined in this paper provide a call to action for all stakeholders to consider.

This project provides patient organizations, research institutions and industry the tools to collaborate and stimulate innovation in symptom management. Projects like KidneyX could allow a path to innovation in this area by providing the funding to move forward on new therapies. Clinical trials are already being conducted to address insomnia in dialysis patients and a new KHI project will begin to address muscle cramping.

The Kidney Health Initiative's (KHI) mission is to advance scientific understanding of kidney health and patient safety implications of new and existing medical products and to foster development of therapies for diseases that affect the kidney by creating a collaborative environment in which U.S. Food and Drug Administration (FDA) and the greater nephrology community can interact to optimize evaluation of drugs, devices, biologics, and food products.