On permutation tests for comparing restricted mean survival time with small sample from randomized trials

2020 ◽  
Vol 39 (20) ◽  
pp. 2655-2670 ◽  
Author(s):  
Miki Horiguchi ◽  
Hajime Uno
Keyword(s):  
Survival Time ◽  
Randomized Trials ◽  
Permutation Tests ◽  
Small Sample ◽  
Mean Survival Time ◽  
Restricted Mean Survival Time

scholarly journals Are restricted mean survival time methods especially useful for noninferiority trials?

2021 ◽  
pp. 174077452097657
Author(s):  
Boris Freidlin ◽  
Chen Hu ◽  
Edward L Korn
Keyword(s):  
Survival Time ◽  
Proportional Hazards ◽  
Small Sample ◽  
Operating Characteristics ◽  
Mean Survival Time ◽  
Kaplan Meier ◽  
Noninferiority Trials ◽  
Restricted Mean Survival Time ◽  
Experimental Treatments ◽  
Event Rates

Background: Restricted mean survival time methods compare the areas under the Kaplan–Meier curves up to a time [Formula: see text] for the control and experimental treatments. Extraordinary claims have been made about the benefits (in terms of dramatically smaller required sample sizes) when using restricted mean survival time methods as compared to proportional hazards methods for analyzing noninferiority trials, even when the true survival distributions satisfy proportional hazardss. Methods: Through some limited simulations and asymptotic power calculations, the authors compare the operating characteristics of restricted mean survival time and proportional hazards methods for analyzing both noninferiority and superiority trials under proportional hazardss to understand what relative power benefits there are when using restricted mean survival time methods for noninferiority testing. Results: In the setting of low-event rates, very large targeted noninferiority margins, and limited follow-up past [Formula: see text], restricted mean survival time methods have more power than proportional hazards methods. For superiority testing, proportional hazards methods have more power. This is not a small-sample phenomenon but requires a low-event rate and a large noninferiority margin. Conclusion: Although there are special settings where restricted mean survival time methods have a power advantage over proportional hazards methods for testing noninferiority, the larger issue in these settings is defining appropriate noninferiority margins. We find the restricted mean survival time methods lacking in these regards.

Estimation on conditional restricted mean survival time with counting process

2020 ◽  
pp. 1-15
Author(s):  
Junshan Qiu ◽  
Dali Zhou ◽  
H.M. Jim Hung ◽  
John Lawrence ◽  
Steven Bai
Keyword(s):  
Survival Time ◽  
Counting Process ◽  
Mean Survival Time ◽  
Restricted Mean Survival Time

A Model-Independent Method to Determine Restricted Mean Survival Time in the Analysis of Survival Curves

2019 ◽  
Vol 2 (1) ◽  
pp. 66-68 ◽  
Author(s):  
Andrea Messori ◽  
Vera Damuzzo ◽  
Laura Agnoletto ◽  
Luca Leonardi ◽  
Marco Chiumente ◽  
...  
Keyword(s):  
Survival Time ◽  
Independent Method ◽  
Survival Curves ◽  
Mean Survival Time ◽  
Restricted Mean Survival Time ◽  
Model Independent

scholarly journals Extrapolating Parametric Survival Models in Health Technology Assessment Using Model Averaging: A Simulation Study

2021 ◽  
Vol 41 (4) ◽  
pp. 476-484
Author(s):  
Daniel Gallacher ◽  
Peter Kimani ◽  
Nigel Stallard
Keyword(s):  
Survival Time ◽  
Survival Data ◽  
Mean Squared Error ◽  
Model Averaging ◽  
Information Criterion ◽  
Health Technology ◽  
Weighted Averaging ◽  
Health Technologies ◽  
Mean Survival Time ◽  
Restricted Mean Survival Time

Previous work examined the suitability of relying on routine methods of model selection when extrapolating survival data in a health technology appraisal setting. Here we explore solutions to improve reliability of restricted mean survival time (RMST) estimates from trial data by assessing model plausibility and implementing model averaging. We compare our previous methods of selecting a model for extrapolation using the Akaike information criterion (AIC) and Bayesian information criterion (BIC). Our methods of model averaging include using equal weighting across models falling within established threshold ranges for AIC and BIC and using BIC-based weighted averages. We apply our plausibility assessment and implement model averaging to the output of our previous simulations, where 10,000 runs of 12 trial-based scenarios were examined. We demonstrate that removing implausible models from consideration reduces the mean squared error associated with the restricted mean survival time (RMST) estimate from each selection method and increases the percentage of RMST estimates that were within 10% of the RMST from the parameters of the sampling distribution. The methods of averaging were superior to selecting a single optimal extrapolation, aside from some of the exponential scenarios where BIC already selected the exponential model. The averaging methods with wide criterion-based thresholds outperformed BIC-weighted averaging in the majority of scenarios. We conclude that model averaging approaches should feature more widely in the appraisal of health technologies where extrapolation is influential and considerable uncertainty is present. Where data demonstrate complicated underlying hazard rates, funders should account for the additional uncertainty associated with these extrapolations in their decision making. Extended follow-up from trials should be encouraged and used to review prices of therapies to ensure a fair price is paid.

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