Sample Sizes Based on Exact Unconditional Tests for Phase II Clinical Trials with Historical Controls

by: Myron N Chang, Jonathan J Shuster, James L Kepner

Journal of Biopharmaceutical Statistics, Vol. 14, No. 1. (2004), pp. 189-200.

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Abstract

Investigated in the setting of phase II clinical trials is the two-sample binomial problem of testing H0 : pe = pc H1 : pe > pc, where pe and pc are the unknown target population response rates for the experimental and control groups, respectively, using the usual Z-statistic with pooled variance estimator. The cornerstones that make this paper unique are as follows. First, the emphasis is on determining the sample size given that the control group information has already been collected (historical control). Second, exact unconditional inference, rather than an asymptotic method, is utilized. Sample size tables, contrasting the exact and asymptotic methods, are provided. Although asymptotic results were usually fairly close to the exact results, some important differences were observed.

@article{citeulike:2520689, abstract = {Investigated in the setting of phase II clinical trials is the two-sample binomial problem of testing H0\ :\ pe\ =\ pc H1\ :\ pe\ \>\ pc, where pe and pc are the unknown target population response rates for the experimental and control groups, respectively, using the usual Z-statistic with pooled variance estimator. The cornerstones that make this paper unique are as follows. First, the emphasis is on determining the sample size given that the control group information has already been collected (historical control). Second, exact unconditional inference, rather than an asymptotic method, is utilized. Sample size tables, contrasting the exact and asymptotic methods, are provided. Although asymptotic results were usually fairly close to the exact results, some important differences were observed.}, author = {Chang, Myron N. and Shuster, Jonathan J. and Kepner, James L. }, citeulike-article-id = {2520689}, doi = {http://dx.doi.org/10.1081/BIP-120028514}, journal = {Journal of Biopharmaceutical Statistics}, keywords = {binomial, clinical\_trial, historical\_control, phase\_ii}, number = {1}, pages = {189--200}, posted-at = {2008-03-12 14:25:42}, priority = {0}, publisher = {Taylor \& Francis}, title = {Sample Sizes Based on Exact Unconditional Tests for Phase II Clinical Trials with Historical Controls}, url = {http://dx.doi.org/10.1081/BIP-120028514}, volume = {14}, year = {2004} }

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TY - JOUR ID - citeulike:2520689 L3 - citeulike-article-id:2520689 TI - Sample Sizes Based on Exact Unconditional Tests for Phase II Clinical Trials with Historical Controls JF - Journal of Biopharmaceutical Statistics VL - 14 IS - 1 SP - 189 EP - 200 PB - Taylor & Francis N2 - Investigated in the setting of phase II clinical trials is the two-sample binomial problem of testing H0 : pe = pc H1 : pe > pc, where pe and pc are the unknown target population response rates for the experimental and control groups, respectively, using the usual Z-statistic with pooled variance estimator. The cornerstones that make this paper unique are as follows. First, the emphasis is on determining the sample size given that the control group information has already been collected (historical control). Second, exact unconditional inference, rather than an asymptotic method, is utilized. Sample size tables, contrasting the exact and asymptotic methods, are provided. Although asymptotic results were usually fairly close to the exact results, some important differences were observed. KW - binomial KW - clinical_trial KW - historical_control KW - phase_ii AU - Chang, Myron AU - Shuster, Jonathan AU - Kepner, James PY - 2004/// UR - http://dx.doi.org/10.1081/BIP-120028514 ER -

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