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Original Articles

Advanced Utilization of Intermediate Endpoints for Making Optimized Cost-Effective Decisions in Seamless Phase II/III Oncology Trials

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Pages 224-233 | Received 10 Sep 2018, Accepted 17 Aug 2019, Published online: 11 Oct 2019

References

  • Anderson, K. A. (2006), “Optimal Spending Functions for Asymmetric Group Sequential Designs,” Biometrical Journal, 48, 1–9.
  • Balser, J., Chang, M., and Bliss, R. (2018), “Interpreting the Regulatory Perspective on Adaptive Designs,” Statistics in Biopharmaceutical Research, 10, 123–129. DOI: 10.1080/19466315.2017.1422799.
  • Chakravarty, A., and Sridhara, R. (2008), “Use of Progression-Free Survival as a Surrogate Marker in Oncology Trials: Some Regulatory Issues,” Statistical Methods in Medical Research, 17, 515–518.
  • Chen, C., Anderson, K., Mehrotra, D. V., Rubin, E. H., and Tse, A. (2018), “A 2-in-1 Adaptive Phase 2/3 Design for Expedited Oncology Drug Development,” Contemporary Clinical Trials, 64, 238–242.
  • Chen, C., and Beckman, R. A. (2009a), “Optimal Cost-Effective Go-No Go Decisions in Late Stage Oncology Drug Development,” Statistics in Biopharmaceutical Research, 1, 159–169.
  • ——— (2009b), “Hypothesis Testing in a Confirmatory Phase III Trial With a Possible Subset Effect,” Statistics in Biopharmaceutical Research, 1, 431–440.
  • Chen, C., and Sun, L. (2011), “On Quantification of PFS Effect for Accelerated Approval of Oncology Drugs,” Statistics in Biopharmaceutical Research, 3, 434–444.
  • Chen, C., Sun, L., and Li, C. (2013), “Evaluation of Early Efficacy Endpoints for Proof-of-Concept Trials,” Journal of Biopharmaceutical Statistics, 23, 413–424. DOI: 10.1080/10543406.2011.616969.
  • Deepak, L. B., and Mehta, C. (2016), “Adaptive Designs for Clinical Trials,” The New England Journal of Medicine, 375, 65–74. DOI: 10.1056/NEJMra1510061.
  • Friede, T., Parsons, N., Stallard, N., Todd, S., Valdés-Márquez, E., Chataway, J., and Nicholas, R. (2011), “Designing a Seamless Phase II/III Clinical Trial Using Early Outcomes for Treatment Selection: An Application in Multiple Sclerosis,” Statistics in Medicine, 30, 1528–1540.
  • Gould, A. L., Krishna, R., Khan, A., and Saltzman, J. (2015), “Principled Structured Incorporation of Clinical Knowledge Into Strategic Development Decisions,” Therapeutic Innovation & Regulatory Science, 49, 289–296.
  • Hee, S. W., Hamborg, T., Day, S., Madan, J., Miller, F., Posch, M., Zohar, S., and Stallard, N. (2016), “Decision-Theoretic Designs for Small Trials and Pilot Studies: A Review,” Statistical Methods in Medical Research, 25, 1022–1038. DOI: 10.1177/0962280215588245.
  • Korn, E. L., Freidlin, B., Abrams, J. S., and Halabi, S. (2012), “Design Issues in Randomized Phase II/III Trials,” Journal of Clinical Oncology, 30, 667–671. DOI: 10.1200/JCO.2011.38.5732.
  • Krisam, J., and Kieser, M. (2017), “Optimal Interim Decision Rules Based on a Binary Surrogate Outcome for Adaptive Biomarker-Based Trials in Oncology,” Statistics in Biopharmaceutical Research, 9, 321–332.
  • Kunz, C. U., Friede, T., Parsons, N., Todd, S., and Stallard, N. (2014), “Data-Driven Treatment Selection for Seamless Phase II/III Trials Incorporating Early-Outcome Data,” Pharmaceutical Statistics, 13, 238–246.
  • ——— (2015), “A Comparison of Methods for Treatment Selection in Seamless Phase II/III Clinical Trials Incorporating Information on Short-Term Endpoints,” Journal of Biopharmaceutical Statistics, 25, 170–189.
  • Mushti, S. L., Mulkey, F., and Sridhara, R. (2018), “Evaluation of Overall Response Rate and Progression-Free Survival as Potential Surrogate Endpoints for Overall Survival in Immunotherapy Trials,” Clinical Cancer Research, 24, 2268–2275.
  • Patel, N. R., and Ankolekar, S. (2007), “A Bayesian Approach for Incorporating Economic Factors in Sample Size Design for Clinical Trials of Individual Drugs and Portfolios of Drugs,” Statistics in Medicine, 26, 4976–4988.
  • Patel, N. R., Ankolekar, S. A., Antonijevic, Z., and Rajicic, N. (2013), “A Mathematical Model for Maximizing the Value of Phase 3 Drug Development Portfolios Incorporating Budget Constraints and Risk,” Statistics in Medicine, 32, 1763–1777.
  • Pearce, M., Hee, S. W., Madan, J., Posch, M., Day, S., Miller, F., Zohar, S., and Stallard, N. (2018), “Value of Information Methods to Design a Clinical Trial in a Small Population to Optimise a Health Economic Utility Function,” BMC Medical Research Methodology, 18, 20.
  • Posch, M., Koenig, F., Branson, M., Brannath, W., Dunger-Baldauf, C., and Bauer, P. (2005), “Testing and Estimation in Flexible Group Sequential Designs With Adaptive Treatment Selection,” Statistics in Medicine, 24, 3697–3714. DOI: 10.1002/sim.2389.
  • Schulz, K. F., Bobulsky, S. T., David, F. S., Patel, N. R., and Antonijevic, Z. (2015), “Drug Development and the Cost of Capital,” in Optimization of Pharmaceutical R&D Programs and Portfolios, ed. Z. Antonijevic, New York: Springer, pp. 35–47.
  • Simon, R. (1989), “Optimal Two-Stage Designs for Phase II Clinical Trials,” Controlled Clinical Trials, 10, 1–10.
  • Stallard, N. (1998), “Sample Size Determination for Phase II Clinical Trials Based on Bayesian Decision Theory,” Biometrics, 54, 279–294.
  • ——— (2003), “Decision-Theoretic Designs for Phase II Clinical Trials Allowing for Competing Studies,” Biometrics, 59, 402–409.
  • ——— (2010), “A Confirmatory Seamless Phase II/III Clinical Trial Design Incorporating Short-Term Endpoint Information,” Statistics in Medicine, 29, 959–971.
  • Stallard, N., and Todd, S. (2003), “Sequential Designs for Phase III Clinical Trials Incorporating Treatment Selection,” Statistics in Medicine, 22, 689–703.
  • ——— (2011), “Seamless Phase II/III Design,” Statistical Methods in Medical Research, 20, 623–634.
  • US Food and Drug Administration (2010), “Draft Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics,” available at http://www.fda.gov/downloads/Drugs/guidancecomplianceregulatoryinformation/guidances/ucm201790.pdf.
  • Yin, Y. (2017), “A ‘Backward’ Bayesian Method for Determination of Criteria for Making Go/No-Go Decisions in the Early Phases,” Statistics in Biopharmaceutical Research, 9, 153–159.

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