American Journal of Epidemiology Vol. 137, No. 7: 787-796
Copyright © 1993 by The Johns Hopkins University School of Hygiene and Public Health
research-article |
Sample Sizes for Prevention Trials Have Been Too Small
1School of Public Health, University of Minnesota Minneapolis, MN.
2The EMMES Corporation Potomac, MD
Reprint requests to Fred Ederer, The EMMES Corporation, 11325 Seven Locks Road, Suite 214, Potomac, MD 20854.
Planners of several large prevention trials have overestimated the expected incidence of events in the control group, largely because they failed either to recognize or to adequately correct for various effects of population selection. Consequently, the studies have been too small in size or too short in duration to achieve their stated objectives. The selection effects include those engendered by the choice of the target population, the self-selection of volunteers, and protocol exclusions. This paper presents a taxonomy of these effects and the likely direction of their influence on the incidence of events and on mortality rates from other causes. Little information is available to help samplesize planners in adjusting for these effects. A few studies have provided information on the extent to which control group incidence rates have fallen short of expectations. In particular, researchers from the University of Minnesota's Colon Cancer Control Study have provided a detailed comparison of event incidence and all-cause mortality rates with general population rates (Am J Epidemiol 1993; 137: 797–810). Other studies should publish similarly detailed information to assist sample-size planners of prevention trials. Until more information is published, this paper provides preliminary guidelines for prevention trial sample-size planning.
epidemiologic methods; mortality; randomized controlled trials; selection bias; study design
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