Research output: Contribution to Journal/Magazine › Journal article › peer-review
Research output: Contribution to Journal/Magazine › Journal article › peer-review
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TY - JOUR
T1 - Active-control trials with binary data
T2 - a comparison of methods for testing superiority or non-inferiority using the odds ratio.
AU - Siqueira, Arminda
AU - Whitehead, Anne
AU - Todd, Susan
PY - 2008/2/10
Y1 - 2008/2/10
N2 - This paper considers methods for testing for superiority or non-inferiority in active-control trials with binary data, when the relative treatment effect is expressed as an odds ratio. Three asymptotic tests for the log-odds ratio based on the unconditional binary likelihood are presented, namely the likelihood ratio, Wald and score tests. All three tests can be implemented straightforwardly in standard statistical software packages, as can the corresponding confidence intervals. Simulations indicate that the three alternatives are similar in terms of the Type I error, with values close to the nominal level. However, when the non-inferiority margin becomes large, the score test slightly exceeds the nominal level. In general, the highest power is obtained from the score test, although all three tests are similar and the observed differences in power are not of practical importance.
AB - This paper considers methods for testing for superiority or non-inferiority in active-control trials with binary data, when the relative treatment effect is expressed as an odds ratio. Three asymptotic tests for the log-odds ratio based on the unconditional binary likelihood are presented, namely the likelihood ratio, Wald and score tests. All three tests can be implemented straightforwardly in standard statistical software packages, as can the corresponding confidence intervals. Simulations indicate that the three alternatives are similar in terms of the Type I error, with values close to the nominal level. However, when the non-inferiority margin becomes large, the score test slightly exceeds the nominal level. In general, the highest power is obtained from the score test, although all three tests are similar and the observed differences in power are not of practical importance.
KW - binary data
KW - likelihood ratio test
KW - non-inferiority
KW - odds ratio
KW - score test
KW - Wald test
U2 - 10.1002/sim.2975
DO - 10.1002/sim.2975
M3 - Journal article
VL - 27
SP - 353
EP - 370
JO - Statistics in Medicine
JF - Statistics in Medicine
SN - 1097-0258
IS - 3
ER -