Targets, trends, excesses, and deficiencies: refocusing clinical investigation to improve patient outcomes

Levin, Adeera, Lancashire, William and Fassett, Robert G. (2013) Targets, trends, excesses, and deficiencies: refocusing clinical investigation to improve patient outcomes. Kidney International, 83 6: 1001-1009. doi:10.1038/ki.2013.91


Author Levin, Adeera
Lancashire, William
Fassett, Robert G.
Title Targets, trends, excesses, and deficiencies: refocusing clinical investigation to improve patient outcomes
Journal name Kidney International   Check publisher's open access policy
ISSN 0085-2538
1523-1755
Publication date 2013-06
Year available 2013
Sub-type Critical review of research, literature review, critical commentary
DOI 10.1038/ki.2013.91
Volume 83
Issue 6
Start page 1001
End page 1009
Total pages 9
Place of publication London, United Kingdom
Publisher Nature Publishing Group
Collection year 2014
Language eng
Abstract Clinical trials in nephrology have focused on achieving targets, supplementing deficiencies, and correcting excesses in order to improve patient outcomes. The majority of interventions have failed to demonstrate benefit and some have caused harm. It may be that therapies aiming to ‘normalize’ parameters may actually disturb evolutionary adaptation, thus causing harm. By refocusing on the physiology of disease, and complexity of adaptation, we may design better trials. We review successful and unsuccessful trials in nephrology and other disciplines and suggest a set of principles by which to design future clinical trials:(1) acknowledge heterogeneity of chronic kidney disease populations and appropriately characterize populations for studies; (2) develop better validated biomarkers (through proteomics, genomics, and metabolomics) to identify responders and nonresponders to interventions; (3) design interventions that mimic physiological processes without collateral detrimental effects; (4) reconsider the status of the randomized-controlled trial as the only ‘gold standard’ and perform large-scale pragmatic trials comparing current care with the intervention(s) of interest, and (5) broaden nephrology research culture so that the majority of patients are enrolled into observational cohorts and intervention studies, which foster greater knowledge acquisition and dissemination. Improved understanding of pathophysiological mechanisms, in conjunction with more innovative but stringent clinical trial design, will ultimately lead to improved patient outcomes.
Keyword Clinical trials
Epidemiology and outcomes
Pathophysiology of renal disease and progression
Q-Index Code C1
Q-Index Status Confirmed Code
Institutional Status UQ

Document type: Journal Article
Sub-type: Critical review of research, literature review, critical commentary
Collections: Official 2014 Collection
School of Human Movement and Nutrition Sciences Publications
School of Medicine Publications
 
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