Prioritizing Symptoms of ESRD Patients for Developing Therapeutic Interventions

Abstract

Patients with kidney failure have a high symptom burden; yet, there are no drugs that have been approved by the US Food and Drug Administration (FDA) for improvement in patient-reported outcomes for this population of patients. In order to catalyze drug development and/or testing currently approved drugs for indications for patients with advanced kidney disease, this project convened a multi-disciplinary group of stakeholders to prioritize symptoms to be targeted for alleviation taking into account the importance placed by patients, ability to identify therapeutic targets, engagement with industry and dialysis provider partners, and solicit input from the FDA to facilitate regulatory approval.

Patient Care Issue

Although patients are the final consumer, they rarely have an opportunity to influence products that come to market. Patients with kidney failure undergoing maintenance dialysis report a wide range of persistent or recurrent symptoms. Surveys of patients undergoing maintenance dialysis indicate that they have, on an average, 11 symptoms, with the most common being feeling tired, difficulty falling or staying asleep, itching, muscle cramps, pain, and sexual dysfunction.¹

Challenge

Not surprisingly, there is a significant association between symptom burden and impairment in quality of life. It is, thus, reasonable to suggest that improvement in some of these symptoms may lead to a meaningful improvement in patient-reported outcomes and/or quality of life. Despite the high burden of symptoms in patients undergoing maintenance dialysis, there is no drug or device that has been approved by the FDA that results in improvement in any patient-reported outcomes.

Solution

KHI has formed a workgroup that will confirm and prioritize the symptoms experienced by patients with kidney failure with additional input from clinicians, researchers, dialysis providers, and industry. The workgroup will then facilitate the engagement of manufacturers and patent-holders with drugs or devices not previously tested in the nephrology space to determine their application and indication for patients with kidney failure.

Selected References:

1. Abdel-Kader K, Unruh ML, Weisbord SD. Symptom burden, depression, and quality of life in chronic and end-stage kidney disease. Clinical journal of the American Society of Nephrology : CJASN 2009;4:1057-64.