On Enrichment Strategies for Biomarker Stratified Clinical Trials

References

• Baselga, J. (2001). Herceptin® alone or in combination with chemotherapy in the treatment of HER2-positive metastatic breast cancer: pivotal trials. Oncology 61:14–21.
   PubMed Web of Science ®Google Scholar
• Brinkley, J., Tsiatis, A., Anstrom, K. J. (2010). A generalized estimator of the attributable benefit of an optimal treatment regime. Biometrics 66:512–522.
   PubMed Web of Science ®Google Scholar
• Freidlin, B., McShane, L. M., Korn, E. L. (2010). Randomized clinical trials with biomarkers: design issues. Journal of the National Cancer Institute 102:152–160.
   PubMed Web of Science ®Google Scholar
• Horgan, A., Bradbury, P., Amir, E., Ng, R., Douillard, J., Kim, E., Shepherd, F., Leighl, N. (2011). An economic analysis of the INTEREST trial, a randomized trial of docetaxel versus gefitinib as second-/third-line therapy in advanced non-small-cell lung cancer. Annals of Oncology 22:1805–1811.
   PubMed Web of Science ®Google Scholar
• Janes, H., Brown, M. D., Huang, Y., Pepe, M. S. (2014). An approach to evaluating and comparing biomarkers for patient treatment selection. The International Journal of Biostatistics 10:99–121.
   PubMedGoogle Scholar
• Janes, H., Pepe, M. S., Bossuyt, P. M., Barlow, W. E. (2011). Measuring the performance of markers for guiding treatment decisions. Annals of Internal Medicine 154:253–259.
   PubMed Web of Science ®Google Scholar
• Joensuu, H., Kellokumpu-Lehtinen, P.-L., Bono, P., Alanko, T., Kataja, V., Asola, R., Utriainen, T., Kokko, R., Hemminki, A., Tarkkanen, M., et al. (2006). Adjuvant docetaxel or vinorelbine with or without trastuzumab for breast cancer. New England Journal of Medicine 354:809–820.
   PubMed Web of Science ®Google Scholar
• Kerr, K. M. (2013). Clinical relevance of the new IASLC/ERS/ATS adenocarcinoma classification. Journal of Clinical Pathology 66:832–838.
   PubMed Web of Science ®Google Scholar
• Korkaya, H., Wicha, M. S. (2013). HER2 and breast cancer stem cells: more than meets the eye. Cancer Research 73:3489–3493.
   PubMed Web of Science ®Google Scholar
• Korn, E. L., Freidlin, B. (2016). Biomarker-based clinical trials. In S.L. George, X. Wang, H. Pang (Eds), Cancer Clinical Trials: Current and Controversial Issues in Design and Analysis 333–364. Boca Raton, FL: Chapman and Hall/CRC.
   Google Scholar
• Maemondo, M., Inoue, A., Kobayashi, K., Sugawara, S., Oizumi, S., Isobe, H., Gemma, A., Harada, M., Yoshizawa, H., Kinoshita, I., et al. (2010). Gefitinib or chemotherapy for non–small-cell lung cancer with mutated EGFR. New England Journal of Medicine 362:2380–2388.
   PubMed Web of Science ®Google Scholar
• Mandrekar, S.J., Sargent, D.J. (2009). Clinical trial designs for predictive biomarker validation: theoretical considerations and practical challenges. Journal of Clinical Oncology 27:4027–4034.
   PubMed Web of Science ®Google Scholar
• Matsui, S., Choai, Y., Nonaka, T. (2014). Comparison of statistical analysis plans in randomize-all phase III trials with a predictive biomarker. Clinical Cancer Research 20:2820–2830.
   PubMed Web of Science ®Google Scholar
• Mok, T. S., Wu, Y.-L., Thongprasert, S., Yang, C.-H., Chu, D.-T., Saijo, N., Sunpaweravong, P., Han, B., Margono, B., Ichinose, Y., et al. (2009). Gefitinib or carboplatin–paclitaxel in pulmonary adenocarcinoma. New England Journal of Medicine 361:947–957.
   PubMed Web of Science ®Google Scholar
• Morara, M., Ryan, L., Houseman, A., Strauss, W. (2007). Optimal design for epidemiological studies subject to designed missingness. Lifetime Data Analysis 13:583–605.
   PubMed Web of Science ®Google Scholar
• Paik, S., Kim, C., Wolmark, N. (2008). HER2 status and benefit from adjuvant trastuzumab in breast cancer. New England Journal of Medicine 358:1409–1411.
   PubMed Web of Science ®Google Scholar
• Polley, M.-Y. C., Freidlin, B., Korn, E. L., Conley, B. A., Abrams, J. S., McShane, L. M. (2013). Statistical and practical considerations for clinical evaluation of predictive biomarkers. Journal of the National Cancer Institute 105: 1677–1683.
   PubMedGoogle Scholar
• Sargent, D. J., Conley, B. A., Allegra, C., Collette, L. (2005). Clinical trial designs for predictive marker validation in cancer treatment trials. Journal of Clinical Oncology 23:2020–2027.
   PubMed Web of Science ®Google Scholar
• Sauter, J., Butnor, K. (2016). Clinical and cost implications of universal versus locally advanced-stage and advanced-stage-only molecular testing for epidermal growth factor receptor mutations and anaplastic lymphoma kinase rearrangements in non–small cell lung carcinoma: A tertiary academic institution experience. Archives of Pathology and Laboratory Medicine 140:358–361.
   PubMed Web of Science ®Google Scholar
• Schmidt, C. (2011). How do you tell whether a breast cancer is HER2 positive? Ongoing studies keep debate in high gear. Journal of the National Cancer Institute 103:87–89.
   PubMedGoogle Scholar
• Shi, Y., Li, J., Zhang, S., Wang, M., Yang, S., Li, N., Wu, G., Liu, W., Liao, G., Cai, K., et al. (2015). Molecular epidemiology of EGFR mutations in asian patients with advanced non-small-cell lung cancer of adenocarcinoma histology–mainland china subset analysis of the PIONEER study. PLoS one 10:e0143515.
   PubMed Web of Science ®Google Scholar
• Simon, R., Maitournam, A. (2004). Evaluating the efficiency of targeted designs for randomized clinical trials. Clinical Cancer Research 10:6759–6763.
   PubMed Web of Science ®Google Scholar
• Strauss, W. J., Ryan, L., Morara, M., Iroz-Elardo, N., Davis, M., Cupp, M., Nishioka, M. G., Quackenboss, J., Galke, W., Özkaynak, H., etal. (2010). Improving cost-effectiveness of epidemiological studies via designed missingness strategies. Statistics in Medicine 29:1377–1387.
   PubMed Web of Science ®Google Scholar
• Tajik, P., Zwinderman, A. H., Mol, B. W., Bossuyt, P. M. (2013). Trial designs for personalizing cancer care: a systematic review and classification. Clinical Cancer Research 19:4578–4588.
   PubMed Web of Science ®Google Scholar
• Wang, X., Ma, J., George, S., Zhou, H. (2012). Estimation of AUC or partial AUC under test-result-dependent sampling. Statistics in Biopharmaceutical Research 4:313–323.
   PubMed Web of Science ®Google Scholar
• Wang, X., Ma, J., George, S. L. (2013). ROC curve estimation under test-result-dependent sampling. Biostatistics 14:160–172.
   PubMed Web of Science ®Google Scholar
• Wang, X., Zhou, H. (2010). Design and inference for cancer biomarker study with an outcome and auxiliary-dependent subsampling. Biometrics 66:502–511.
   PubMed Web of Science ®Google Scholar
• Yang, B., Zhou, Y., Zhang, L., Cui, L. (2015). Enrichment design with patient population augmentation. Contemporary Clinical Trials 42:60–67.
   PubMed Web of Science ®Google Scholar