Sensitivity Analyses of Clinical Trial Designs: Selecting Scenarios and Summarizing Operating Characteristics

References

• Bélisle, C. J. (1992), “Convergence Theorems for a Class of Simulated Annealing Algorithms on Rd,” Journal of Applied Probability, 885–895. DOI: 10.2307/3214721.
   Web of Science ®Google Scholar
• Berry, S. M., Carlin, B. P., Lee, J. J., and Muller, P. (2010), Bayesian Adaptive Methods for Clinical Trials, Boca Raton, FL: CRC Press.
   Google Scholar
• Bookstein, F. L. (1989), “Principal Warps: Thin-Plate Splines and the Decomposition of Deformations,” IEEE Transactions on Pattern Analysis and Machine Intelligence, 11, 567–585. DOI: 10.1109/34.24792.
   Web of Science ®Google Scholar
• Goodfellow, I., Bengio, Y., and Courville, A. (2016), Deep Learning, Cambridge, MA: MIT Press.
   Google Scholar
• Green, S., Liu, P.-Y., and O’Sullivan, J. (2002), “Factorial Design Considerations,” Journal of Clinical Oncology, 20, 3424–3430. DOI: 10.1200/JCO.2002.03.003.
   PubMed Web of Science ®Google Scholar
• Han, K., Ren, M., Wick, W., Abrey, L., Das, A., Jin, J., and Reardon, D. A. (2014), “Progression-Free Survival as a Surrogate Endpoint for Overall Survival in Glioblastoma: A Literature-based Meta-Analysis from 91 Trials,” Neuro-Oncology, 16, 696–706. DOI: 10.1093/neuonc/not236.
   PubMed Web of Science ®Google Scholar
• Hobbs, B. P., Barata, P. C., Kanjanapan, Y., Paller, C. J., Perlmutter, J., Pond, G. R., Prowell, T. M., Rubin, E. H., Seymour, L. K., Wages, N. A., et al. (2019), “Seamless Designs: Current Practice and Considerations for Early-Phase Drug Development in Oncology,” JNCI: Journal of the National Cancer Institute, 111, 118–128. DOI: 10.1093/jnci/djy196.
   PubMedGoogle Scholar
• Hornik, K. (1991), “Approximation Capabilities of Multilayer Feedforward Networks,” Neural Networks, 4, 251–257. DOI: 10.1016/0893-6080(91)90009-T.
   Web of Science ®Google Scholar
• Husmann, K., Lange, A., and Spiegel, E. (2017), “The r Package Optimization: Flexible Global Optimization with Simulated-Annealing.”
   Google Scholar
• Iasonos, A., Gönen, M., and Bosl, G. J. (2015), “Scientific Review of Phase I Protocols with Novel Dose-Escalation Designs: How Much Information is Needed?” Journal of Clinical Oncology, 33, 2221–2225. DOI: 10.1200/JCO.2014.59.8466.
   PubMed Web of Science ®Google Scholar
• Jenkins, M., Stone, A., and Jennison, C. (2011), “An Adaptive Seamless Phase II/III Design for Oncology Trials with Subpopulation Selection Using Correlated Survival Endpoints,” Pharmaceutical Statistics, 10, 347–356. DOI: 10.1002/pst.472.
   PubMed Web of Science ®Google Scholar
• Jones, R. L., Attia, S., Mehta, C. R., Liu, L., Sankhala, K. K., Robinson, S. I., Ravi, V., Penel, N., Stacchiotti, S., Tap, W. D., et al. (2017), “Tappas: An Adaptive Enrichment Phase 3 Trial of TRC105 and Pazopanib Versus Pazopanib Alone in Patients with Advanced Angiosarcoma (AAS),” Journal of Clinical Oncology, 35, TPS11081. DOI: 10.1200/JCO.2017.35.15_suppl.TPS11081.
   Web of Science ®Google Scholar
• Kirkpatrick, S., Gelatt, C. D., and Vecchi, M. P. (1983), “Optimization by Simulated Annealing,” Science, 220, 671–680. DOI: 10.1126/science.220.4598.671.
   PubMed Web of Science ®Google Scholar
• Leshno, M., Lin, V. Y., Pinkus, A., and Schocken, S. (1993), “Multilayer Feedforward Networks with a Nonpolynomial Activation Function Can Approximate Any Function,” Neural Networks, 6, 861–867. DOI: 10.1016/S0893-6080(05)80131-5.
   Web of Science ®Google Scholar
• Mayer, C., Perevozskaya, I., Leonov, S., Dragalin, V., Pritchett, Y., Bedding, A., Hartford, A., Fardipour, P., and Cicconetti, G. (2019), Simulation Practices for Adaptive Trial Designs in Drug and Device Development,” Statistics in Biopharmaceutical Research, 11, 325–335. DOI: 10.1080/19466315.2018.1560359.
   Web of Science ®Google Scholar
• McKay, M. D., Beckman, R. J., and Conover, W. J. (2000), “A Comparison of Three Methods for Selecting Values of Input Variables in the Analysis of Output from a Computer Code,” Technometrics, 42, 55–61. DOI: 10.1080/00401706.2000.10485979.
   Web of Science ®Google Scholar
• Mehta, C., Liu, L., and Theuer, C. (2019), “An Adaptive Population Enrichment Phase III Trial of TRC105 and Pazopanib Versus Pazopanib Alone in Patients with Advanced Angiosarcoma (Tappas Trial),” Annals of Oncology, 30, 103–108. DOI: 10.1093/annonc/mdy464.
   PubMed Web of Science ®Google Scholar
• Michael, J., and Schucany, W. (2002), “The Mixture Approach for Simulating New Families of Bivariate Distributions with Specified Correlations,” The American Statistician, 56, 48–54. DOI: 10.1198/000313002753631367.
   Web of Science ®Google Scholar
• Niewczas, J., Kunz, C. U., and König, F. (2019), “Interim Analysis Incorporating Short-and Long-Term Binary Endpoints,” Biometrical Journal, 61, 665–687. DOI: 10.1002/bimj.201700281.
   PubMed Web of Science ®Google Scholar
• Pallmann, P., Bedding, A. W., Choodari-Oskooei, B., Dimairo, M., Flight, L., Hampson, L. V., Holmes, J., Mander, A. P., Odondi, L., Sydes, M. R., et al. (2018), “Adaptive Designs in Clinical Trials: Why Use Them, and How to Run and Report Them,” BMC Medicine, 16, 1–15. DOI: 10.1186/s12916-018-1017-7.
   PubMedGoogle Scholar
• Rasmussen, C. E. (2003), “Gaussian Processes in Machine Learning,” in Summer School on Machine Learning, eds. O. Bousquet, U. Luxburg, and G. Rätsch, pp. 63–71, Berlin: Springer.
   Google Scholar
• Razavi, S., Jakeman, A., Saltelli, A., Prieur, C., Iooss, B., Borgonovo, E., Plischke, E., Piano, S. L., Iwanaga, T., Becker, W., et al. (2021), “The Future of Sensitivity Analysis: An Essential Discipline for Systems Modeling and Policy Support,” Environmental Modelling & Software, 137, 104954. DOI: 10.1016/j.envsoft.2020.104954.
   Web of Science ®Google Scholar
• Spall, J. C. (2005), Introduction to Stochastic Search and Optimization: Estimation, Simulation, and Control, Hoboken, NJ: Wiley.
   Google Scholar
• Thorlund, K., Haggstrom, J., Park, J. J., and Mills, E. J. (2018), “Key Design Considerations for Adaptive Clinical Trials: A Primer for Clinicians,” BMJ, 360, k698. DOI: 10.1136/bmj.k698.
   PubMedGoogle Scholar
• US Food and Drug Administration (2020), “Interacting with the FDA on Complex Innovative Trial Designs for Drugs and Biological Products,” updated December.
   Google Scholar