Clinical Trial Considerations in Developing Treatments for Early Stages of Common, Chronic Kidney Diseases: A ScientificWorkshop Cosponsored by the National Kidney Foundation and the US Food and Drug Administration

In the past decade, advances in the validation of surrogate end points for chronic kidney disease (CKD) progression have heightened interest in evaluating therapies in early CKD. In December 2020, the National Kidney Foundation sponsored a scientific workshop in collaboration with the US Food and Drug Administration (FDA) to explore patient, provider, and payor perceptions of the value of treating early CKD. The workshop reviewed challenges for trials in early CKD, including trial designs, identification of high-risk populations, and cost-benefit and safety considerations. Over 90 people representing a range of stakeholders including experts in clinical trials, nephrology, cardiology and endocrinology, patient advocacy organizations, patients, payors, health economists, regulators and policy makers attended a virtual meeting. There was consensus among the attendees that there is value to preventing the development and treating the progression of early CKD in people who are at high risk for progression, and that surrogate end points should be used to establish efficacy. Attendees also concluded that cost analyses should be holistic and include aspects beyond direct savings for treatment of kidney failure; and that safety data should be collected outside/beyond the duration of a clinical trial. Successful drug development and implementation of effective therapies will require collaboration across sponsors, patients, patient advocacy organizations, medical community, regulators, and payors. (c) 2022 by the National Kidney Foundation, Inc. Published by Elsevier Inc. All rights reserved.

Automated summary

A scientific workshop sponsored by the National Kidney Foundation and the US Food and Drug Administration (FDA) was held to discuss the value of treating early chronic kidney disease (CKD) and the challenges in conducting trials for early CKD. The attendees reached a consensus on the importance of preventing and treating early CKD progression, using surrogate end points to establish efficacy, and conducting comprehensive cost analyses and safety data collection.