Design of Major Randomized Trials: Part 3 of a 4-Part Series on Statistics for Clinical Trials

Stuart J Pocock; Tim C Clayton; Gregg W Stone

doi:10.1016/j.jacc.2015.10.036

Design of Major Randomized Trials: Part 3 of a 4-Part Series on Statistics for Clinical Trials

J Am Coll Cardiol. 2015 Dec 22;66(24):2757-2766. doi: 10.1016/j.jacc.2015.10.036.

Authors

Stuart J Pocock¹, Tim C Clayton², Gregg W Stone³

Affiliations

¹ Department of Medical Statistics, London School of Hygiene and Tropical Medicine, London, United Kingdom. Electronic address: stuart.pocock@lshtm.ac.uk.
² Department of Medical Statistics, London School of Hygiene and Tropical Medicine, London, United Kingdom.
³ Columbia University Medical Center, New York-Presbyterian Hospital, and the Cardiovascular Research Foundation, New York, New York.

PMID: 26700838
DOI: 10.1016/j.jacc.2015.10.036

Abstract

This paper provides practical guidance on the fundamentals of design for major randomized controlled trials. Topics covered include the choice of patients, choice of treatment and control groups, choice of primary and secondary endpoints, methods of randomization, appropriate use of blinding, and determination of trial size. Insights are made with reference to contemporary major trials in cardiology.

Keywords: avoidance of bias; randomized controlled trial; sample size; selection of patients; study design; treatments and endpoints.

Publication types

Review

MeSH terms

Cardiovascular Diseases / therapy*
Data Interpretation, Statistical
Humans
Patient Selection*
Randomized Controlled Trials as Topic / methods*
Randomized Controlled Trials as Topic / statistics & numerical data*