title: Pragmatic Trials in Maintenance Dialysis: Perspectives from the Kidney Health Initiative.
abstract: Pragmatic clinical trials are conducted under the real-world conditions of clinical care delivery. As a result, these trials yield findings that are highly generalizable to the nonresearch setting, identify interventions that are readily translatable into clinical practice, and cost less than trials that require extensive research infrastructures. Maintenance dialysis is a setting especially well suited for pragmatic trials because of inherently frequent and predictable patient encounters, highly granular and uniform data collection, use of electronic data systems, and delivery of care by a small number of provider organizations to approximately 90% of patients nationally. Recognizing the potential for pragmatic trials to generate much needed evidence to guide the care of patients receiving maintenance dialysis, the Kidney Health Initiative assembled a group of individuals with relevant expertise from academia, industry, and government to provide the nephrology community with information about the design and conduct of such trials, with a specific focus on the dialysis setting. Here, we review this information, and where applicable, use experience from the ongoing Time to Reduce Mortality in End Stage Renal Disease Trial, a large cluster-randomized, pragmatic trial evaluating hemodialysis session duration, to illustrate challenges and solutions to operational, ethical, and regulatory issues.
authors.affiliation: Renal, Electrolyte and Hypertension Division and Department of Biostatistics and Epidemiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania; ldember@upenn.edu.
authors.forename: Laura M
authors.initials: LM
authors.lastname: Dember
authors.affiliation: Office of Medical Policy and Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland.
authors.forename: Patrick
authors.initials: P
authors.lastname: Archdeacon
authors.affiliation: DaVita Healthcare Partners, Denver, Colorado.
authors.forename: Mahesh
authors.initials: M
authors.lastname: Krishnan
authors.affiliation: Nephrology Division, Tufts Medical Center and Tufts University School of Medicine, Boston, Massachusetts.
authors.forename: Eduardo
authors.initials: E
authors.lastname: Lacson
authors.affiliation: Center for Clinical Standards and Quality, Centers for Medicare and Medicaid Services, Baltimore, Maryland.
authors.forename: Shari M
authors.initials: SM
authors.lastname: Ling
authors.affiliation: Division of Nephrology, The University of Arizona College of Medicine and Southern Arizona Veterans Administration Health Care System, Tucson, Arizona.
authors.forename: Prabir
authors.initials: P
authors.lastname: Roy-Chaudhury
authors.affiliation: Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland.
authors.forename: Kimberly A
authors.initials: KA
authors.lastname: Smith
authors.affiliation: Division of Kidney, Urology, and Hematology, National Institute of Diabetes and Digestive and Kidney Diseases, Bethesda, Maryland.
authors.forename: Michael F
authors.initials: MF
authors.lastname: Flessner
mesh_terms: D035843:Biomedical Research; D006801:Humans; D064792:Pragmatic Clinical Trials as Topic; D006435:Renal Dialysis; D051436:Renal Insufficiency, Chronic / Q000628:therapy*
keywords: cluster randomization; electronic medical record; ethical and regulatory issues; health systems; informed consent