Pragmatic Clinical Trials in CKD: Opportunities and Challenges.
Randomized controlled trials in CKD lag in number behind those of other chronic diseases, despite the high morbidity and mortality experienced by patients with kidney disease and the exorbitant costs of their health care. Observational studies of CKD frequently yield seemingly paradoxic associations of traditional risk factors with outcomes, making it difficult to extrapolate the results of trials conducted in people with normal kidney function to patients with CKD. However, many completed trials in CKD have been limited by intermediate outcomes of unclear clinical significance or narrow eligibility criteria that limit external validity, and implementation of proven therapies remains a challenge. It is therefore imperative that the nephrology community capitalize on recent interest in novel approaches to trial design, such as pragmatic clinical trials. These trials are meant to promote research within real world settings to yield clinically relevant results with greater applicability than those of traditional trials, while maintaining many advantages, such as controlling for potential sources of bias. We provide a description of pragmatic clinical trials and a discussion of advantages, disadvantages, and practical challenges inherent to this study design, in the context of specific scientific questions relevant to patients with CKD.
Division of Nephrology and Department of Medicine, Kidney Research Institute, University of Washington, Seattle, WA; deboer@u.washington.edu.
Ian H
IH
de Boer
Division of Nephrology, University of Tennessee Health Science Center, Memphis, TN; Nephrology Section, Memphis VA Medical center, Memphis, TN.
Csaba P
CP
Kovesdy
Section of Nephrology, Department of Medicine, Selzman Institute for Kidney Health, Baylor College of Medicine; Section of Nephrology, Michael E. DeBakey Veterans Affairs Medical Center, Houston, TX.
Sankar D
SD
Navaneethan
Kidney Health Research Collaborative and Division of Nephrology, University of California San Francisco, San Francisco, CA.
Carmen A
CA
Peralta
Division of Nephrology, University of California San Francisco, San Francisco, CA.
Delphine S
DS
Tuot
Division of Nephrology, Department of Internal Medicine, UT Southwestern Medical Center, Dallas, TX.
Miguel A
MA
Vazquez
Division of Nephrology, Department of Medicine, Johns Hopkins University School of Medicine; and Welch Center for Prevention, Epidemiology and Clinical Research, Johns Hopkins Medical Institutions, Baltimore, MD.
Deidra C
DC
Crews
D006801:Humans; D064792:Pragmatic Clinical Trials as Topic / Q000592:standards; D051436:Renal Insufficiency, Chronic / Q000628:therapy*
chronic kidney disease; clinical trial; outcomes