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Statistical Methods in Medical Research 4ed

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Unequal-sized groups<br />

It is usually optimal when compar<strong>in</strong>g two groups to have equal numbers <strong>in</strong> each<br />

group. But sometimes the number available <strong>in</strong> one group may be restricted, e.g.<br />

for a rare disease <strong>in</strong> a case±control study (§19.4). In this case the power can be<br />

<strong>in</strong>creased to a limited extent by hav<strong>in</strong>g more <strong>in</strong> the other group. If one group<br />

conta<strong>in</strong>s m subjects and the other rm, then the study is approximately equivalent<br />

to a study with n <strong>in</strong> each group where<br />

That is,<br />

2 1 1<br />

ˆ ‡<br />

n m rm :<br />

…r ‡ 1†n<br />

m ˆ : …4:43†<br />

2r<br />

This expression is derived by equat<strong>in</strong>g the expressions for the standard error of<br />

the difference between two means used <strong>in</strong> a two-sample t test, and is exact for a<br />

cont<strong>in</strong>uous variable and approximate for the comparison of two proportions.<br />

Fleiss et al. (1980) give a formula for the general case of compar<strong>in</strong>g two proportions<br />

where the two samples are not of equal size, and their formula is suitable<br />

for the <strong>in</strong>verse problem of estimat<strong>in</strong>g power from known sample sizes.<br />

The total number of subjects <strong>in</strong> the study is<br />

which is a m<strong>in</strong>imum for r ˆ 1.<br />

Other considerations<br />

…r ‡ 1† 2<br />

4r<br />

4.6 Sample-size determ<strong>in</strong>ation 145<br />

The situations considered <strong>in</strong> this section are relatively simpleÐthose of compar<strong>in</strong>g<br />

two groups without any complicat<strong>in</strong>g features. The determ<strong>in</strong>ation of sample<br />

size is often facilitated by the use of tables and, <strong>in</strong> addition to those mentioned<br />

earlier, the book by Lemeshow et al. (1990) conta<strong>in</strong>s a number of sets of tables.<br />

Even <strong>in</strong> these simple situations it is necessary to have a reasonable idea of the<br />

likely form of the data to be collected before sample size can be estimated. For<br />

example, when compar<strong>in</strong>g means it is necessary to have an estimate of the<br />

standard deviation, or <strong>in</strong> compar<strong>in</strong>g proportions the approximate size of one<br />

of the proportions is required at the plann<strong>in</strong>g stage. Such <strong>in</strong>formation may be<br />

available from earlier studies us<strong>in</strong>g the same variables or may be obta<strong>in</strong>ed from a<br />

pilot study. In more complicated situations more <strong>in</strong>formation is required but<br />

(4.41) can be used <strong>in</strong> pr<strong>in</strong>ciple, provided that it is possible to f<strong>in</strong>d an expression<br />

for s, the standard deviation relevant to the comparison of <strong>in</strong>terest.<br />

2n,

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