Patient-Reported Outcomes in Clinical Trials of CKD-Related Therapies: Report of a Symposium Sponsored by the National Kidney Foundation and the US Food and Drug Administration

• Published in: American journal of kidney diseases Vol. 62; no. 6; pp. 1046 - 1057
• Main Authors: Perrone, Ronald D., MD, Coons, Stephen Joel, PhD, Cavanaugh, Kerri, MD, Finkelstein, Fred, MD, Meyer, Klemens B., MD
• Format: Journal Article Conference Proceeding
• Language: English
• Published: New York, NY Elsevier Inc 01.12.2013; Elsevier
• Subjects: Activities of Daily Living - classification; anemia; Anemia - therapy; Biological and medical sciences; Chronic kidney disease (CKD); Cooperative Behavior; Endpoint Determination; Humans; Interdisciplinary Communication; Kidney Failure, Chronic - therapy; Kidneys; Malformations of the urinary system; Medical sciences; Nephrology; Nephrology. Urinary tract diseases; nephrotic syndrome; Nephrotic Syndrome - therapy; Patient Outcome Assessment; Patient Satisfaction; patient-reported outcomes; polycystic kidney disease; Polycystic Kidney, Autosomal Dominant - therapy; Quality of Life; Randomized Controlled Trials as Topic; United States; United States Food and Drug Administration; Urinary system involvement in other diseases. Miscellaneous; United States; patient-reported outcomes; polycystic kidney disease; Chronic kidney disease (CKD); anemia; nephrotic syndrome; Glomerulonephritis; Kidney disease; Human; Nephrology; Urinary system disease; Anemia; Report; Polycystic kidney; Hemopathy; Chronic kidney disease; Nephrotic syndrome; Urology; Genetic disease; Treatment; Renal failure; Cyst; Complication; Clinical trial; Benign neoplasm; Food and Drug Administration
• ISSN: 0272-6386; 1523-6838
• DOI: 10.1053/j.ajkd.2013.07.004

The National Kidney Foundation and the US Food and Drug Administration (FDA) convened a symposium in September 2010, bringing together more than 70 experts, including representatives from the FDA, the National Institutes of Health, the Critical Path Institute, nephrologists, patients, and the pharmaceutical industry to discuss the feasibility and process of developing patient-reported outcome (PRO) measures to access how patients feel or function to be used in clinical trials for regulatory review of treatment benefit. Three disease areas were evaluated for development of end point models in which PRO measures may be useful: anemia secondary to chronic kidney disease, autosomal dominant polycystic kidney disease (ADPKD), and nephrotic syndrome. The participants thought it valuable to use observational data to generate hypotheses regarding patient baseline characteristics that are likely to predict clinically important changes in PROs in response to anemia treatment and to design adequately powered blinded randomized controlled trials of anemia treatment using PROs as primary rather than secondary end points. Validated PRO instruments that reflect the patient experience in ADPKD and nephrotic syndrome are essential to incorporate into clinical trials of new therapeutic interventions because glomerular filtration rate decline may occur late in the disease course, at which point therapeutic benefit is less likely. Conference attendees addressed how PRO measures could be used to evaluate, monitor, provide care, and facilitate the introduction of treatments for patients with these challenging conditions.