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PMID: 21904102 Published · ppublish English Journal Article Research Support, N.I.H., Extramural Review

Key concepts of clinical trials: a narrative review.

Postgraduate medicine ·Vol. 123 ·No. 5 ·2011-09-00 ·Pages 194-204

Umscheid CA, Margolis DJ, Grossman CE

Abstract

The recent focus of federal funding on comparative effectiveness research underscores the importance of clinical trials in the practice of evidence-based medicine and health care reform. The impact of clinical trials not only extends to the individual patient by establishing a broader selection of effective therapies, but also to society as a whole by enhancing the value of health care provided. However, clinical trials also have the potential to pose unknown risks to their participants, and biased knowledge extracted from flawed clinical trials may lead to the inadvertent harm of patients. Although conducting a well-designed clinical trial may appear straightforward, it is founded on rigorous methodology and oversight governed by key ethical principles. In this review, we provide an overview of the ethical foundations of trial design, trial oversight, and the process of obtaining approval of a therapeutic, from its pre-clinical phase to post-marketing surveillance. This narrative review is based on a course in clinical trials developed by one of the authors (DJM), and is supplemented by a PubMed search predating January 2011 using the keywords "randomized controlled trial," "patient/clinical research," "ethics," "phase IV," "data and safety monitoring board," and "surrogate endpoint." With an understanding of the key principles in designing and implementing clinical trials, health care providers can partner with the pharmaceutical industry and regulatory bodies to effectively compare medical therapies and thereby meet one of the essential goals of health care reform.

MeSH Terms
Clinical Trials, Phase I as Topic Clinical Trials, Phase II as Topic Clinical Trials, Phase III as Topic Clinical Trials, Phase IV as Topic Drug Evaluation/methods Humans Randomized Controlled Trials as Topic/ethics,methods,standards United States
Authors & Affiliations
3 authors, click to expand affiliations / ORCID
Umscheid Craig A
Center for Evidence-Based Practice, University of Pennsylvania, Philadelphia, PA, USA.
Margolis David J
Grossman Craig E
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Article Info
Journal
Postgraduate medicine
Abbr.
Postgrad Med
ISSN
1941-9260
DOI
10.3810/pgm.2011.09.2475
Published
2011-09-00
Pages
194-204
Language
English
Region
England
NLM ID
0401147
PMCID
PMC3272827
Subset
IM
Grants
NCI NIH HHS · T32 CA009677 · United States
NCI NIH HHS · T32 CA009677-07 · United States
NCI NIH HHS · T32-CA-009677 · United States
PHS HHS · T32-HP-010026 · United States
External Links
PubMed source DOI: 10.3810/pgm.2011.09.2475 PMC Full Text View journal details
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