Statistical Test/Estimation Methods Used in Contemporary Phase III Cancer Randomized Controlled Trials with Time-to-Event Outcomes
- PMID: 32043795
- PMCID: PMC7011662
- DOI: 10.1634/theoncologist.2019-0464
Statistical Test/Estimation Methods Used in Contemporary Phase III Cancer Randomized Controlled Trials with Time-to-Event Outcomes
Abstract
There exist a wide variety of statistical methods for clinical trials. Despite decades of clinical trial research, little is known about which methods are used in data analysis practice of the real world. This review describes the evidence of existing practices on survival data analysis and identifies potential opportunities to improve them.
Keywords: Interpretability; Robustness; Survival data analysis; Test/estimation coherency; Treatment decision making.
Conflict of interest statement
Figures

Similar articles
-
Empirical power comparison of statistical tests in contemporary phase III randomized controlled trials with time-to-event outcomes in oncology.Clin Trials. 2020 Dec;17(6):597-606. doi: 10.1177/1740774520940256. Epub 2020 Sep 15. Clin Trials. 2020. PMID: 32933339
-
Quantitative decision-making in randomized Phase II studies with a time-to-event endpoint.J Biopharm Stat. 2019;29(1):189-202. doi: 10.1080/10543406.2018.1489400. Epub 2018 Jul 3. J Biopharm Stat. 2019. PMID: 29969380
-
Interval and point estimation in adaptive Phase II trials with binary endpoint.Stat Methods Med Res. 2019 Sep;28(9):2635-2648. doi: 10.1177/0962280218781411. Epub 2018 Jun 19. Stat Methods Med Res. 2019. PMID: 29921157
-
Design of cancer screening trials/randomized trials for evaluation of cancer screening.World J Surg. 2006 Jul;30(7):1152-62. doi: 10.1007/s00268-006-0074-9. World J Surg. 2006. PMID: 16794902 Review. No abstract available.
-
Guidelines for time-to-event end point definitions in sarcomas and gastrointestinal stromal tumors (GIST) trials: results of the DATECAN initiative (Definition for the Assessment of Time-to-event Endpoints in CANcer trials)†.Ann Oncol. 2015 May;26(5):865-872. doi: 10.1093/annonc/mdu360. Epub 2014 Jul 28. Ann Oncol. 2015. PMID: 25070543 Review.
Cited by
-
Biomarkers in Oncology: Complexities in Biomarker-Driven Studies and Statistical Analysis.JCO Precis Oncol. 2024 Jul;8:e2400358. doi: 10.1200/PO.24.00358. JCO Precis Oncol. 2024. PMID: 39038245 Free PMC article.
-
Optimal two-stage designs based on restricted mean survival time for a single-arm study.Contemp Clin Trials Commun. 2021 Jan 23;21:100732. doi: 10.1016/j.conctc.2021.100732. eCollection 2021 Mar. Contemp Clin Trials Commun. 2021. PMID: 33553801 Free PMC article.
-
Regression models for average hazard.Biometrics. 2024 Mar 27;80(2):ujae037. doi: 10.1093/biomtc/ujae037. Biometrics. 2024. PMID: 38771658 Free PMC article.
-
Sensitivity Analysis for Restricted Mean Survival Time When Survival Curves Have Divergent Tails.Ther Innov Regul Sci. 2023 May;57(3):467-471. doi: 10.1007/s43441-022-00484-z. Epub 2023 Jan 3. Ther Innov Regul Sci. 2023. PMID: 36596962
-
On Sample Size Determination for Augmented Tests Based on Restricted Mean Survival Time in Randomized Clinical Trials.Biom J. 2025 Apr;67(2):e70046. doi: 10.1002/bimj.70046. Biom J. 2025. PMID: 40123326 Free PMC article.
References
-
- Cox DR. Regression models and life‐tables. J Royal Stat Soc Ser B Stat Methodol 1972;34:187–220.
-
- Fleming TR, Harrington DP. Counting Processes and Survival Analysis. New York, NY: John Wiley & Sons, 1991.
Publication types
MeSH terms
LinkOut - more resources
Full Text Sources
Medical