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Research Articles

Using Surrogate Endpoints in Adaptive Designs with Delayed Treatment Effect

ORCID Icon, ORCID Icon, , &
Pages 661-670 | Received 21 Jul 2020, Accepted 24 May 2021, Published online: 26 Jul 2021

References

  • Benboubker, L., Dimopoulos, M. A., Dispenzieri, A., Catalano, J., Belch, A. R., Cavo, M., Pinto, A., Weisel, K., Ludwig, H., Bahlis, N., and Banos, A. (2014), “Lenalidomide and Dexamethasone in Transplant-Ineligible Patients With Myeloma,” New England Journal of Medicine, 371, 906–917. DOI: 10.1056/NEJMoa1402551.
  • Berry, D. A., Zhou, S., Higley, H., Mukundan, L., Fu, S., Reaman, G. H., Wood, B. L., Kelloff, G. J., Jessup, J. M., and Radich, J. P. (2017), “Association of Minimal Residual Disease With Clinical Outcome in Pediatric and Adult Acute Lymphoblastic Leukemia: A Meta-Analysis,” JAMA Oncology, 3, e170580. DOI: 10.1001/jamaoncol.2017.0580.
  • Blumenthal, G. M., Karuri, S. W., Zhang, H., Zhang, L., Khozin, S., Kazandjian, D., Tang, S., Sridhara, R., Keegan, P., and Pazdur, R. (2015), “Overall Response Rate, Progression-Free Survival, and Overall Survival With Targeted and Standard Therapies in Advanced Non–Small-Cell Lung Cancer: US Food and Drug Administration Trial-Level and Patient-Level Analyses,” Journal of Clinical Oncology, 33, 1008. DOI: 10.1200/JCO.2014.59.0489.
  • Borghaei, H., Paz-Ares, L., Horn, L., Spigel, D. R., Steins, M., Ready, N. E., Chow, L. Q., Vokes, E. E., Felip, E., Holgado, E., and Barlesi, F. (2015), “Nivolumab Versus Docetaxel in Advanced Nonsquamous Non–Small-Cell Lung Cancer,” New England Journal of Medicine, 373, 1627–1639. DOI: 10.1056/NEJMoa1507643.
  • Cancer.Net (2019), “Lung Cancer: Non-Small Cell: Statistics,” available at https://www.cancer.net/cancer-types/lung-cancer-non-small-cell/statistics.
  • Cui, L., Hung, H. J., and Wang, S. J. (1999), “Modification of Sample Size in Group Sequential Clinical Trials,” Biometrics, 55, 853–857. DOI: 10.1111/j.0006-341x.1999.00853.x.
  • Fine, G. D. (2007), “Consequences of Delayed Treatment Effects on Analysis of Time-to-Event Endpoints,” Drug Information Journal: DIJ/Drug Information Association, 41, 535–539.
  • Food and Drug Administration (FDA). (1998), “Guidance for Industry: E9 Statistical Principles for Clinical Trials,” FDA, Washington DC, USA, available at https://www.fda.gov/media/71336/download
  • Food and Drug Administration (FDA). (2019), Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics, Washington, DC: FDA.
  • Friede, T., Stallard, N., and Parsons, N. (2020), “Adaptive Seamless Clinical Trials Using Early Outcomes for Treatment or Subgroup Selection: Methods, Simulation Model and Their Implementation in R,” Biometrical Journal, 62, 1264–1283. DOI: 10.1002/bimj.201900020.
  • Harousseau, J. L., Palumbo, A., Richardson, P. G., Schlag, R., Dimopoulos, M. A., Shpilberg, O., Shpilberg, O., Kropff, M., Kentos, A., Cavo, M., Golenkov, A., and Komarnicki, M. (2010), “Superior Outcomes Associated With Complete Response in Newly Diagnosed Multiple Myeloma Patients Treated With Nonintensive Therapy: Analysis of the Phase 3 VISTA Study of Bortezomib Plus Melphalan-Prednisone Versus Melphalan-Prednisone,” Blood, The Journal of the American Society of Hematology, 116, 3743–3750.
  • Harrington, D. P., and Fleming, T. R. (1982), “A Class of Rank Test Procedures for Censored Survival Data,” Biometrika, 69, 553–566.
  • Hartford, A., Thomann, M., Chen, X., Miller, E., Bedding, A., Jorgens, S., Liu, L., Chen, L., and Morgan, C. (2020), “Adaptive Designs: Results of 2016 Survey on Perception and Use,” Therapeutic Innovation & Regulatory Science, 54, 42–54. DOI: 10.1007/s43441-019-00028-y.
  • Li, Q., Lin, J., and Lin, Y. (2020), “Adaptive Design Implementation in Confirmatory Trials: Methods, Practical Considerations and Case Studies,” Contemporary Clinical Trials, 98, 106096. DOI: 10.1016/j.cct.2020.106096.
  • Lin, J., and Bunn, V. (2017), “Comparison of Multi-Arm Multi-Stage Design and Adaptive Randomization in Platform Clinical Trials,” Contemporary Clinical Trials, 54, 48–59 DOI: 10.1016/j.cct.2017.01.003.
  • Lin, J., Bunn, V., and Liu, R. (2019), “Practical Considerations for Subgroups Quantification, Selection and Adaptive Enrichment in Confirmatory Trials,” Statistics in Biopharmaceutical Research, 11, 407–418.
  • Liu, M., Li, Q., Lin, J., Lin, Y., and Hoffman, E. (2020), “Innovative Trial Designs and Analyses for Vaccine Clinical Development,” Contemporary Clinical Trials, 100, 106225. DOI: 10.1016/j.cct.2020.106225.
  • Mangal, N., Salem, A. H., Li, M., Menon, R., and Freise, K. J. (2018), “Relationship Between Response Rates and Median Progression-Free Survival in Non-Hodgkin’s Lymphoma: A Meta-Analysis of Published Clinical Trials,” Hematological Oncology, 36, 37–43. DOI: 10.1002/hon.2463.
  • Mehta, C. R., and Pocock, S. J. (2011), “Adaptive Increase in Sample Size When Interim Results Are Promising: A Practical Guide With Examples,” Statistics in Medicine, 30, 3267–3284. DOI: 10.1002/sim.4102.
  • Munshi, N. C., Avet-Loiseau, H., Rawstron, A. C., Owen, R. G., Child, J. A., Thakurta, A., Sherrington, P., Samur, M. K., Georgieva, A., Anderson, K. C., and Gregory, W. M. (2017), “Association of Minimal Residual Disease With Superior Survival Outcomes in Patients With Multiple Myeloma: A Meta-Analysis,” JAMA Oncology, 3, 28–35. DOI: 10.1001/jamaoncol.2016.3160.
  • Nakashima, K., Horita, N., Nagai, K., Manabe, S., Murakami, S., Ota, E., and Kaneko, T. (2016), “Progression-Free Survival, Response Rate, and Disease Control Rate as Predictors of Overall Survival in Phase III Randomized Controlled Trials Evaluating the First-Line Chemotherapy for Advanced, Locally Advanced, and Recurrent Non–Small Cell Lung Carcinoma,” Journal of Thoracic Oncology, 11, 1574–1585. DOI: 10.1016/j.jtho.2016.04.025.
  • Navada, S., Lai, P., Schwartz, A. G., and Kalemkerian, G. P. (2006), “Temporal Trends in Small Cell Lung Cancer: Analysis of the National Surveillance, Epidemiology, and End-Results (SEER) Database,” Journal of Clinical Oncology, 24, 7082–7082.
  • O’Brien, P. C., and Fleming, T. R. (1979), “A Multiple Testing Procedure for Clinical Trials,” Biometrics, 549–556. DOI: 10.2307/2530245.
  • Pocock, S. J. (1977), “Group Sequential Methods in the Design and Analysis of Clinical Trials,” Biometrika, 64, 191–199.
  • Proschan, M. A., Lan, K. G., and Wittes, J. T. (2006), Statistical Monitoring of Clinical Trials: A Unified Approach, Springer Science & Business Media.
  • Reck, M., Rodríguez-Abreu, D., Robinson, A. G., Hui, R., Csőszi, T., Fülöp, A., Gottfried, M., Peled, N., Tafreshi, A., Cuffe, S., and O’Brien, M. (2016), “Pembrolizumab Versus Chemotherapy for PD-L1–Positive Non–Small-Cell Lung Cancer,” New England Journal of Medicine, 375, 1823–1833. DOI: 10.1056/NEJMoa1606774.
  • Royston, P., and Parmar, M. K. (2011), “The Use of Restricted Mean Survival Time to Estimate the Treatment Effect in Randomized Clinical Trials When the Proportional Hazards Assumption Is in Doubt,” Statistics in Medicine, 30, 2409–2421. DOI: 10.1002/sim.4274.
  • Sinha, A. K., Moye, L., III, Piller, L. B., Yamal, J. M., Barcenas, C. H., Lin, J., and Davis, B. R. (2019), “Adaptive Group-Sequential Design With Population Enrichment in Phase 3 Randomized Controlled Trials With Two Binary Co-Primary Endpoints,” Statistics in Medicine, 38, 3985–3996. DOI: 10.1002/sim.8216.
  • Sun, L., and Wan, Y. (2018), “Conditional Power and Predictive Power Based on Right Censored Data With Supplementary Auxiliary Information,” Statistics in Medicine, 37, 2690–2699. DOI: 10.1002/sim.7673.
  • Sun, L. Z., Li, W., Chen, C., and Zhao, J. (2020), “Advanced Utilization of Intermediate Endpoints for Making Optimized Cost-Effective Decisions in Seamless Phase II/III Oncology Trials,” Statistics in Biopharmaceutical Research, 12, 224–233.
  • Uno, H., Claggett, B., Tian, L., Inoue, E., Gallo, P., Miyata, T., Schrag, D., Takeuchi, M., Uyama, Y., Zhao, L., and Skali, H. (2014), “Moving Beyond the Hazard Ratio in Quantifying the Between-Group Difference in Survival Analysis,” Journal of clinical Oncology, 32, 2380. DOI: 10.1200/JCO.2014.55.2208.
  • Xu, J., Lam, K. F., Chen, F., Milligan, P., and Cheung, Y. B. (2017), “Semiparametric Estimation of Time-Varying Intervention Effects Using Recurrent Event Data,” Statistics in medicine, 36, 2682–2696. DOI: 10.1002/sim.7319.
  • Yang, S., and Prentice, R. (2005), “Semiparametric Analysis of Short-Term and Long-Term Hazard Ratios With Two-Sample Survival Data,” Biometrika, 92, 1–17. DOI: 10.1093/biomet/92.1.1.
  • Ye, J., Ji, X., Dennis, P. A., Abdullah, H., and Mukhopadhyay, P. (2020), “Relationship Between Progression-Free Survival, Objective Response Rate, and Overall Survival in Clinical Trials of PD-1/PD-L1 Immune Checkpoint Blockade: A Meta-Analysis,” Clinical Pharmacology & Therapeutics, 108, 1274–1288. DOI: 10.1002/cpt.1956.
  • Zucker, D. M., and Lakatos, E. (1990), “Weighted Log Rank Type Statistics for Comparing Survival Curves When There Is a Time Lag in the Effectiveness of Treatment,” Biometrika, 77, 853–864.

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