Optimal adaptive single-arm phase II trials under quantified uncertainty

References

• Aylin, S., A. Birkenbach, A. Berlind, and J. Eyraud. Examination of clinical trial costs and barriers for drug development. Technical report, Eastern Research Group, Inc., Lexington, 2014.
   Google Scholar
• Banerjee, A., and A. A. Tsiatis. 2006. Adaptive two-stage designs in phase II clinical trials. Statistics in Medicine 25 (19):3382–3395. doi:10.1002/sim.2501.
   PubMed Web of Science ®Google Scholar
• Battelle Technology Partnership Practice. Biopharmaceutical industry-sponsored clinical trials: Impact on state economies. Technical report, 2015.
   Google Scholar
• Berger, J. O. 1990. Robust Bayesian analysis: Sensitivity to the prior. Journal of Statistical Planning and Inference 25 (3):303–328. doi:10.1016/0378-3758(90)90079-A.
   Web of Science ®Google Scholar
• Bezanson, J., A. Edelman, S. Karpinski, and V. B. Shah. 2017. Julia: A fresh approach to numerical computing. SIAM Review 59 (1):65–98. doi:10.1137/141000671.
   Web of Science ®Google Scholar
• Denne, J. S. 2001. Sample size recalculation using conditional power. Statistics in Medicine 20 (17–18):2645–2660.
   PubMed Web of Science ®Google Scholar
• Dunning, I., J. Huchette, and M. Lubin. 2017. JuMP: A modeling language for mathematical optimization. SIAM Review 59 (2):295–320. doi:10.1137/15M1020575.
   Web of Science ®Google Scholar
• Englert, S., and M. Kieser. 2013. Optimal adaptive two-stage designs for phase II cancer clinical trials. Biometrical Journal 55 (6):955–968. doi:10.1002/bimj.v55.6.
   PubMed Web of Science ®Google Scholar
• Frank Liu, G., G. Ray Zhu, and L. Cui. 2008. Evaluating the adaptive performance of flexible sample size designs with treatment difference in an interval. Statistics in Medicine 27 (4):584–596. doi:10.1002/sim.3348.
   PubMed Web of Science ®Google Scholar
• Fúquene, J. A., J. D. Cook, and L. R. Pericchi. 2009. A case for robust bayesian priors with applications to clinical trials. Bayesian Analysis 4 (4):817–846. doi:10.1214/09-BA431.
   Web of Science ®Google Scholar
• Ivanova, A., B. Paul, O. Marchenko, G. Song, N. Patel, and S. J. Moschos. 2016. Nine-year change in statistical design, profile, and success rates of phase II oncology trials. Journal of Biopharmaceutical Statistics 26 (1):141–149. doi:10.1080/10543406.2015.1092030.
   PubMed Web of Science ®Google Scholar
• Jung, S. H., T. Lee, K. M. Kim, and S. L. George. 2004. Admissible two-stage designs for phase II cancer clinical trials. Statistics in Medicine 23 (4):561–569. doi:10.1002/sim.1600.
   PubMed Web of Science ®Google Scholar
• Kunzmann, K., and M. Kieser. 2016. Optimal adaptive two-stage designs for single-arm trial with binary endpoint. arXiv preprint arXiv:1605.00249.
   Google Scholar
• Kunzmann, K., and M. Kieser. 2017. Point estimation and p-values in phase ii adaptive two-stage designs with a binary endpoint. Statistics in Medicine 36 (6):971–984. doi:10.1002/sim.7200.
   PubMed Web of Science ®Google Scholar
• Kunzmann, K., and M. Kieser. 2018. Test-compatible confidence intervals for adaptive two-stage single-arm designs with binary endpoint. Biometrical Journal 60 (1):196–206. doi:10.1002/bimj.v60.1.
   PubMed Web of Science ®Google Scholar
• Mander, A. P., and S. G. Thompson. 2010. Two-stwo-stage designs optimal under the alternative hypothesis for phase ii cancer clinical trials. Contemporary Clinical Trials 31 (6):572–578. doi:10.1016/j.cct.2010.07.008.
   PubMed Web of Science ®Google Scholar
• Simon, R. 1989. Optimal two-stage designs for phase II clinical trials. Controlled Clinical Trials 10 (1):1–10. doi:10.1016/0197-2456(89)90015-9.
   PubMedGoogle Scholar
• Spiegelhalter, D. J., and L. S. Freedman. 1986. A predictive approach to selecting the size of a clinical trial, based on subjective clinical opinion. Statistics in Medicine 5 (1):1–13. doi:10.1002/sim.4780050103.
   PubMed Web of Science ®Google Scholar
• Thall, P. F., and R. Simon. 1994. A bayesian approach to establishang sample size and monitoring criteria for phase ii clinical trials. Contemporary Clinical Trials 15 (6):463–481. doi:10.1016/0197-2456(94)90004-3.
   Google Scholar
• Thomas, D. W., J. Burns, J. Audette, A. Carroll, C. Dow-Hygelund, and M. Hay. Clinical Development Success Rates 2006-2015. Technical report, BIO, 2016.
   Google Scholar