0
Views
0
CrossRef citations to date
0
Altmetric
Research Article

Adaptive seamless phase II/III design with sequential estimation-adjusted urn model

, , , , &
Received 27 Sep 2023, Accepted 05 Jul 2024, Published online: 22 Jul 2024

References

  • Altman, D. G., and J. P. Royston. 1988. The hidden effect of time. Statistics in Medicine 7 (6):629–37. doi: 10.1002/sim.4780070602.
  • Atkinson, A. C., and A. Biswas. 2005. Bayesian adaptive biased-coin designs for clinical trials with normal responses. Biometrics 61 (1):118–25. doi: 10.1111/j.0006-341X.2005.031002.x.
  • Barker, A. D., C. C. Sigman, G. J. Kelloff, N. M. Hylton, D. A. Berry, and L. Esserman. 2009. I‐SPY 2: An adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clinical Pharmacology and Therapeutics 86 (1):97–100. doi: 10.1038/clpt.2009.68.
  • Bai, Z. D., F. Hu, and W. F. Rosenberger. 2002. Asymptotic properties of adaptive designs for clinical trials with delayed response. The Annals of Statistics 30 (1):122–39. doi: 10.1214/aos/1015362187.
  • Berry, D. A. 2004. Bayesian statistics and the efficiency and ethics of clinical trials. Statistical Science 19 (1):175–87. doi: 10.1214/088342304000000044.
  • Berry, D. A. 2012. Adaptive clinical trials in oncology. Nature Reviews. Clinical Oncology 9 (4):199–207. doi: 10.1038/nrclinonc.2011.165.
  • Brannath, W., F. Koenig, and P. Bauer. 2007. Multiplicity and flexibility in clinical trials. Pharmaceutical Statistics 6 (3):205–16. doi: 10.1002/pst.302.
  • Brannath, W., G. Gutjahr, and P. Bauer. 2012. Probabilistic foundation of confirmatory adaptive designs. Journal of the American Statistical Association 107 (498):824–32. doi: 10.1080/01621459.2012.682540.
  • Brannath, W., E. Zuber, M. Branson, F. Bretz, P. Gallo, M. Posch, and A. Racine-Poon. 2009. Confirmatory adaptive designs with Bayesian decision tools for a targeted therapy in oncology. Statistics in Medicine 28 (10):1445–63. doi: 10.1002/sim.3559.
  • Chen, C., K. Anderson, D. V. Mehrotra, E. H. Rubin, and A. Tse. 2018. A 2-in-1 adaptive phase 2/3 design for expedited oncology drug development. Contemporary Clinical Trials 64:238–42. doi: 10.1016/j.cct.2017.09.006.
  • Chen, Y. J., R. Gesser, and A. Luxembourg. 2015. A seamless phase IIB/III adaptive outcome trial: Design rationale and implementation challenges. Clinical Trials (London, England) 12 (1):84–90. doi: 10.1177/1740774514552110.
  • Coad, D. S. 1991. Sequential tests for an unstable response variable. Biometrika 78 (1):113–21. doi: 10.1093/biomet/78.1.113.
  • Hu, F., and W. F. Rosenberger. 2003. Optimality, variability, power: Evaluating response-adaptive randomization procedures for treatment comparisons. Journal of the American Statistical Association 98 (463):671–8. doi: 10.1198/016214503000000576.
  • Hu, F., and L.-X. Zhang. 2004. Asymptotic properties of doubly adaptive biased coin designs for multi-treatment clinical trials. The Annals of Statistics 32 (1):268–301. doi: 10.1214/aos/1079120137.
  • Hu, F., L. X. Zhang, S. H. Cheung, and W. S. Chan. 2008. Doubly adaptive biased coin designs with delayed responses. Canadian Journal of Statistics 36 (4):541–59. doi: 10.1002/cjs.5550360404.
  • Huang, X., J. Ning, Y. Li, E. Estey, J. P. Issa, and D. A. Berry. 2009. Using short-term response information to facilitate adaptive randomization for survival clinical trials. Statistics in Medicine 28 (12):1680–9. doi: 10.1002/sim.3578.
  • Inoue, L. Y., P. F. Thall, and D. A. Berry. 2002. Seamlessly expanding a randomized phase II trial to phase III. Biometrics 58 (4):823–31. doi: 10.1111/j.0006-341x.2002.00823.x.
  • Jin, M., and P. Zhang. 2022. A seamless adaptive 2-in-1 design expanding a phase 2 trial for treatment or dose selection into a phase 3 trial. Statistics in Biopharmaceutical Research 14 (3):334–41. doi: 10.1080/19466315.2021.1914717.
  • Kim, E. S., R. S. Herbst, I. I. Wistuba, J. J. Lee, G. R. Blumenschein, A. Tsao, D. J. Stewart, M. E. Hicks, J. Erasmus, S. Gupta, et al. 2011. The BATTLE trial: Personalizing therapy for lung cancer. Cancer Discovery 1 (1):44–53. doi: 10.1158/2159-8274.CD-10-0010.
  • Kim, E., J. Yang, S. Park, and K. Shin. 2023. Factors affecting success of new drug clinical trials. Therapeutic Innovation & Regulatory Science 57 (4):737–50. doi: 10.1007/s43441-023-00509-1.
  • Koenig, F., W. Brannath, F. Bretz, and M. Posch. 2008. Adaptive Dunnett tests for treatment selection. Statistics in Medicine 27 (10):1612–25. doi: 10.1002/sim.3048.
  • Liu, Q., M. A. Proschan, and G. W. Pledger. 2002. A unified theory of two-stage adaptive designs. Journal of the American Statistical Association 97 (460):1034–41. doi: 10.1198/016214502388618852.
  • Marcus, R., P. Eric, and K. R. Gabriel. 1976. On closed testing procedures with special reference to ordered analysis of variance. Biometrika 63 (3):655–60. doi: 10.1093/biomet/63.3.655.
  • Müller, H. H., and H. Schäfer. 2001. Adaptive group sequential designs for clinical trials: Combining the advantages of adaptive and of classical group sequential approaches. Biometrics 57 (3):886–91. doi: 10.1111/j.0006-341x.2001.00886.x.
  • Neyman, J. 1934. On the two different aspects of the representative method: The method of stratified sampling and the method of purposive selection. Journal of the Royal Statistical Society 97 (4):558–606. doi: 10.2307/2342192.
  • Rosenberger, W. F., N. Stallard, A. Ivanova, C. N. Harper, and M. L. Ricks. 2001. Optimal adaptive designs for binary response trials. Biometrics 57 (3):909–13. doi: 10.1111/j.0006-341x.2001.00909.x.
  • Schaid, D. J., S. A. M. Wieand, and T. M. Therneau. 1990. Optimal two-stage screening designs for survival comparisons. Biometrika 77 (3):507–13. doi: 10.1093/biomet/77.3.507.
  • Stallard, N., and S. Todd. 2003. Sequential designs for phase III clinical trials incorporating treatment selection. Statistics in Medicine 22 (5):689–703. doi: 10.1002/sim.1362.
  • Thall, P. F., R. Simon, and S. S. Ellenberg. 1988. Two-stage selection and testing designs for comparative clinical trials. Biometrika 75 (2):303–10. doi: 10.1093/biomet/75.2.303.
  • Thall, P. F., R. Simon, and S. S. Ellenberg. 1989. A two-stage design for choosing among several experimental treatments and a control in clinical trials. Biometrics 45 (2):537–47. doi: 10.2307/2531495.
  • Thall, P. F., and J. K. Wathen. 2007. Practical Bayesian adaptive randomisation in clinical trials. European Journal of Cancer (Oxford, England: 1990) 43 (5):859–66. doi: 10.1016/j.ejca.2007.01.006.
  • Thompson, W. R. 1933. On the likelihood that one unknown probability exceeds another in view of the evidence of two samples. Biometrika 25 (3-4):285–94. doi: 10.1093/biomet/25.3-4.285.
  • US Food and Drug Administration. 2006. Innovation or stagnation: Critical path opportunities report and list.
  • US Food and Drug Administration. 2018. Expansion cohorts: Use in first-in-human clinical trials to expedite development of oncology drugs and biologics guidance for industry.
  • Villar, S. S., J. Wason, and J. Bowden. 2015. Response-adaptive randomization for multi-arm clinical trials using the forward looking Gittins index rule. Biometrics 71 (4):969–78. doi: 10.1111/biom.12337.
  • Wang, S.-J., H. M. J. Hung, and R. T. O'Neill. 2006. Adapting the sample size planning of a phase III trial based on phase II data. Pharmaceutical Statistics 5 (2):85–97. doi: 10.1002/pst.217.
  • Wason, J. M., and T. Jaki. 2012. Optimal design of multi‐arm multi‐stage trials. Statistics in Medicine 31 (30):4269–79. doi: 10.1002/sim.5513.
  • Wei, L. J., and S. Durham. 1978. The randomized play-the-winner rule in medical trials. Journal of the American Statistical Association 73 (364):840–3. doi: 10.1080/01621459.1978.10480109.
  • Yin, G., and Y. Shen. 2005. Adaptive design and estimation in randomized clinical trials with correlated observations. Biometrics 61 (2):362–9. doi: 10.1111/j.1541-0420.2005.00333.x.
  • Yuan, Y., X. Huang, and S. Liu. 2011. A Bayesian response‐adaptive covariate‐balanced randomization design with application to a leukemia clinical trial. Statistics in Medicine 30 (11):1218–29. doi: 10.1002/sim.4218.
  • Zang, Y., and J. J. Lee. 2014. Adaptive clinical trial designs in oncology. Chinese Clinical Oncology 3 (4):49. doi: 10.3978/j.issn.2304-3865.2014.06.04.
  • Zhang, L. X., F. Hu, and S. H. Cheung. 2006. Asymptotic theorems of sequential estimation-adjusted urn models. The Annals of Applied Probability 16 (1):340–69. doi: 10.1214/105051605000000746.
  • Zhu, H., and F. Hu. 2012. Interim analysis of clinical trials based on urn models. Canadian Journal of Statistics 40 (3):550–68. doi: 10.1002/cjs.11140.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.