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Original Articles

Adaptive Informational Design of Confirmatory Phase III Trials With an Uncertain Biomarker Effect to Improve the Probability of Success

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Pages 237-247 | Received 08 Jul 2015, Published online: 16 Sep 2016

References

  • Beckman, R. A, Clark, J., Chen, and C. (2011), “Integrating Predictive Biomarkers and Classifiers into Oncology Clinical Development Programs: An Adaptive, Evidence-Based Approach,” Nature Review Drug Discovery, 10, 735–749.
  • Bretz, F., Maurer, W., Brannath, W., and Posch, M. (2009), “A Graphical Approach to Sequentially Rejective Multiple Test Procedures,” Statistics in Medicine, 28, 86–604.
  • Bretz, F., Schmidli, H., König, F., Racine, A., and Maurer, W. (2006), “Confirmatory Seamless Phase II/III Clinical Trials with Hypotheses Selection at Interim: General Concepts,” Biometrical Journal, 48, 623–634.
  • Chen, C., and Beckman, R. A. (2009), “Hypothesis Testing in a Confirmatory Phase III Trial with a Possible Subset Effect,” Statistics in Biopharmaceutical Research, 1, 431–440.
  • Freidlin, B., Korn, E. L., and Gray, R. (2014), “Marker Sequential Test (MaST) Design,” Clinical Trials, 11, 19–27.
  • Freidlin, B., and Simon, R. (2005), “Adaptive Signature Design: An Adaptive Clinical Trial Design For Generating and Prospectively Testing a Gene Expression Signature for Sensitive Patients,” Clinical Cancer Research, 11, 7872–7878.
  • He, W., Pinheiro, J., and Kuznetsova, O. M. (eds). (2014), Practical Considerations for Adaptive Trial Design and Implementation, New York: Springer.
  • Jiang, W., Freidlin, B., and Simon, R. (2007), “Biomarker-Adaptive Threshold Design: A Procedure for Evaluating Treatment with Possible Biomarker-Defined Subset Effect,” Journal of National Cancer Institute, 99, 1036–1043.
  • Kelly, K., Altorki, N. K., Eberhardt, W. E. E., O'Brien, M. E. R., Spigel, D. R., Crino, L., Tsai, C.-M., Kim, J.-H., Cho, E. K., Szczesna, A., Burghuber, O., Hoffman, P. C., Keshavjee, S., Orlov, S., Serwatowski, P., Wang, J., Foley, M. A., Horan, J. D., Park, J. W., Shepherd, F. A. et al. (2014), “A Randomized, Double-Blind Phase 3 Trial of Adjuvant Erlotinib (E) Versus Placebo (P) Following Complete Tumor Resection with or without Adjuvant Chemotherapy in Patients (pts) with Stage IB-IIIA EGFR Positive (IHC/FISH) Non-Small Cell Lung Cancer (NSCLC): RADIANT Results,” Journal of Clinical Oncology, 32.
  • Magnusson, B., and Turnbull, B. W. (2013), “Group Sequential Enrichment Design Incorporating Subgroup Selection,” Statistics in Medicine, 32, 2695–2714.
  • Mehta, C., Schäfer, H., Daniel, H., and Irle S. (2014), “Biomarker Driven Population Enrichment for Adaptive Oncology Trials with Time to Event Endpoints,” Statistics in Medicine, 33, 4515–4531.
  • Muller, H. H., and Schafer, H. (2004), “A General Statistical Principle for Changing a Design Any Time During the Course of a Trial,” Statistics in Medicine, 23, 2497–2508.
  • Shentu, Y., Chen, C., Pang, L., and Beckman, R. A (Under review), “Adaptive Alpha Allocation in a Confirmatory Phase III Trial with Possible Subset Effect,” in ASA Proceedings of the Joint Statistical Meetings 2015, Biopharmaceutical Section.
  • Spiessens, B., and Debois, M. (2010), “Adjusted Significance Levels for Subgroup Analyses in Clinical Trials,” Contemporary Clinical Trials, 31, 647–656.
  • Stallard, N., and Todd, S. (2011), “Seamless Phase II/III Designs,” Statistical Methods in Medical Research, 20, 623–634.
  • Uno, H., Claggett, B., Tian, T., Inoue, E., Gallo, P., Miyata, T., Schrag, D., Takeuchi, M., Uyama, Y., Zhao, L., Skali, H., Solomon, S., Jacobus, S., Hughes, M., Packer, M., and Wei, L. J. (2014), “Moving Beyond the Hazard Ratio in Quantifying the Between-Group Difference in Survival Analysis,” Journal of Clinical Oncology, 55, 2208.
  • Von Pawel, J., Tseng, J., Dediu, M, Schumann, C., Moritz, B., Mendell-Harary, J., Jin, X., Feng, W., Copigneaux, C., Beckman, R. A., et al. (2014), “Phase 2 HERALD Study of Patritumab (P) with Erlotinib (E) in Advanced NSCLC Subjects,” Journal of Clinical Oncology, 32, 5s.
  • Wang, S. J., O'Neill, R. T., and Hung, H. M. J. (2007), “Approaches to Evaluation of Treatment Effect in Randomized Clinical Trials with Genomic Subset,” Pharmaceutical Statistics, 6, 227–244.
  • Wiens, B. L., and Dmitrienko, A. (2005), “The Fallback Procedure for Evaluating a Single Family of Hypotheses,” Journal of Biopharmaceutical Statistics, 15, 929–942.

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