References
- Andrillon, A., S. Chevret, S. M. Lee, and L. Biard. 2020. Dose-finding design and benchmark for a right censored endpoint. Journal of Biopharmaceutical Statistics 1–16. doi:10.1080/10543406.2020.1821702.
- Bai, X., Q. Deng, and D. Liu. 2020. Multiplicity issues for platform trials with a shared control arm. Journal of Biopharmaceutical Statistics 1–13. doi:10.1080/10543406.2020.1821703.
- European Medicines Agency (EMA). ICH E9 (R1) addendum on estimands and sensitivity analysis in clinical trials to the guideline on statistical principles for clinical trials. Step 5. 17 February 2020. Accessed December 20, 2020. https://www.ema.europa.eu/en/documents/scientific-guideline/ich-e9-r1-addendum-estimands-sensitivity-analysis-clinical-trials-guideline-statistical-principles_en.pdf.
- Food and Drug Administration (FDA). Statistical reviews for application number 761052Orig1s000 (Brineura). 2017.Accessed December 20, 2020. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/761052Orig1s000StatR.pdf.
- Food and Drug Administration (FDA). Approval package for application number 207103Orig1s008 (IBRANCE). 2019a. Accessed December 20, 2020. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/207103Orig1s008.pdf.
- Food and Drug Administration (FDA). Multi-discipline review for application number 212306Orig1s000 (XPOVIO). 2019b. Accessed December 20, 2020. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/212306Orig1s000MultidisciplineR.pdf.
- Hong, H., C. Wang, and G. L. Rosner. 2020. Meta-analysis of rare adverse events in randomized clinical trials: Bayesian and frequentist methods. Clinical Trials 18:3–16. doi:10.1177/1740774520969136.
- Ivanova, A., E. Israel, L. M. LaVange, M. C. Peters, L. C. Denlinger, W. C. Moore, L.B. Bacharier, M.A. Marquis, N.M. Gotman, M.R. Kosorok, et al. 2020. The precision interventions for severe and/or exacerbation-prone asthma (PrecISE) adaptive platform trial: statistical considerations. Journal of Biopharmaceutical Statistics. doi:10.1080/10543406.2020.1821705.
- Ivanova, A., and B. Qaqish. 2020. Power calculations for the sequential parallel comparison design with continuous outcomes. Journal of Biopharmaceutical Statistics 1–9. doi:10.1080/10543406.2020.1818252.
- Joshi, N., C. Nguyen, and A. Ivanova. 2020. Multi-stage adaptive enrichment trial design with subgroup estimation. Journal of Biopharmaceutical Statistics 1–12. doi:10.1080/10543406.2020.1832109.
- Li, P., R. Liu, J. Lin, and Y. Ji. 2020. TEPI-2 and UBI: Designs for optimal immuno-oncology and cell therapy dose finding with toxicity and efficacy. Journal of Biopharmaceutical Statistics Epub ahead of print. PMID: 32951518: 1–14. doi:10.1080/10543406.2020.1814802.
- Lin, X., and Y. Ji. 2020. The Joint i3+3 (Ji3+3) design for phase I/II adoptive cell therapy clinical trials. Journal of Biopharmaceutical Statistics 1–13. doi:10.1080/10543406.2020.1818250.
- Mt-Isa, S., N. Wang, C. E. Hallgreen, T. Callreus, G. Genov, I. Hirsch, S. Hobbiger, K.S. Hockley, D. Luciani, L.D. Phillips, et al. Pharmacoepidemiological Research on Outcomes of Therapeutics by a European ConsorTium (PROTECT). Review of methodologies for benefit and risk assessment of medication. April 10, 2013. Accessed October 21. 2020. http://protectbenefitrisk.eu/documents/ShahruletalReviewofmethodologiesforbenefitandriskassessmentofmedicationMay2013.pdf.
- Psioda, M. A., and X. Xue. 2020. A Bayesian adaptive two-stage design for pediatric clinical trials. Journal of Biopharmaceutical Statistics 1–18. doi:10.1080/10543406.2020.1821704.
- Public Law 114-255. December 13, 2016. Accessed December 27, 2020.https://www.congress.gov/bill/114th-congress/house-bill/34/.
- U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER). Guidance for Industry, E2C (R2) Periodic Benefit-Risk Evaluation Report (PBRER).July 2016. Accessed January 28, 2021. https://www.fda.gov/media/83371/download.
- U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER). Guidance for Industry, M4E: The CTD-Efficacy. July 2017. Accessed January 28, 2021. https://www.fda.gov/media/93569/download.
- Wang, T., X. Wang, S. L. George, and H. Zhou. 2020. Design and analysis of biomarker-integrated clinical trials with adaptive threshold detection and flexible patient enrichment. Journal of Biopharmaceutical Statistics 1–17. doi:10.1080/10543406.2020.1832110.
- Wei, B., T. M. Braun, R. N. Tamura, and K. Kidwell. 2020. Sample size determination for Bayesian analysis of small n Sequential, Multiple Assignment, Randomized Trials (snSMARTs) with three agents. Journal of Biopharmaceutical Statistics 1–12. doi:10.1080/10543406.2020.1815032.
- Yin, J., and Y. Yuan. 2020. Checkerboard: A Bayesian efficacy and toxicity interval design for phase I/II dose-finding trials. Journal of Biopharmaceutical Statistics 1–20. doi:10.1080/10543406.2020.1815033.