https://scholars.lib.ntu.edu.tw/handle/123456789/348091
標題: | Inference on treatment effects for targeted clinical trials under enrichment design | 作者: | JEN-PEI LIU Lin Jr. R. Chow, S.-C. |
關鍵字: | Bootstrap method; EM algorithm; Positive predictive value; Targeted clinical trials | 公開日期: | 2009 | 卷: | 8 | 期: | 4 | 起(迄)頁: | 356-370 | 來源出版物: | Pharmaceutical Statistics | 摘要: | After completion of a human genome project, the disease targets at molecular level can be identified. As a result, treatment modality for molecular targets can be developed. In practice, targeted clinical trials are usually conducted for evaluation of the possibility and feasibility of the individualized treatment of patients. However, the accuracy of diagnostic devices for identification of such molecular targets is usually not perfect. Therefore, some of the patients enrolled in targeted clinical trials with a positive result by the diagnostic device might not have the specific molecular targets and hence the treatment effects of the targeted drugs estimated from targeted clinical trials could be biased for the patient population truly with the molecular targets. Under an enrichment design for targeted clinical trials, we propose to use the EM algorithm and bootstrap method for obtaining the inference of the treatment effects of the targeted drugs in the patient population truly with molecular targets. A simulation study was conducted to empirically investigate the bias and variability of the proposed estimator and the size and power of the proposed testing method. Simulation results demonstrate that the proposed estimator is unbiased with adequate precision and the confidence interval can provide satisfactory coverage probability. In addition, the proposed testing procedure can adequately control the size with sufficient power. A practical example illustrates the utility of the proposed method. Copyright ? 2009 John Wiley & Sons, Ltd. |
URI: | http://www.scopus.com/inward/record.url?eid=2-s2.0-73949142341&partnerID=MN8TOARS http://scholars.lib.ntu.edu.tw/handle/123456789/348091 |
DOI: | 10.1002/pst.364 | SDG/關鍵字: | accuracy; algorithm; article; bootstrapping; confidence interval; drug targeting; human; probability; simulation; statistical analysis; therapy effect; Algorithms; Biostatistics; Clinical Trials as Topic; Computer Simulation; Confidence Intervals; Drug Delivery Systems; Humans; Individualized Medicine; Models, Statistical; Treatment Outcome |
顯示於: | 農藝學系 |
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