Two-stage optimal designs based on exact variance for a single-arm trial with survival endpoints.

Journal of Biopharmaceutical Statistics
Guogen Shan

Abstract

Sample size calculation based on normal approximations is often associated with the loss of statistical power for a single-arm trial with a time-to-event endpoint. Recently, Wu (2015) derived the exact variance for the one-sample log-rank test under the alternative and showed that a single-arm one-stage study based on exact variance often has power above the nominal level while the type I error rate is controlled. We extend this approach to a single-arm two-stage design by using exact variances of the one-sample log-rank test for the first stage and the two stages combined. The empirical power of the proposed two-stage optimal designs is often not guaranteed under a two-stage design setting, which could be due to the asymptotic bi-variate normal distribution used to estimate the joint distribution of the test statistics. We adjust the nominal power level in the design search to guarantee the simulated power of the identified optimal design being above the nominal level. The sample size and the study time savings of the proposed two-stage designs are substantial as compared to the one-stage design.

References

Jun 5, 2003·American Journal of Transplantation : Official Journal of the American Society of Transplantation and the American Society of Transplant Surgeons·Allan D Kirk
Oct 2, 2003·Journal of the National Cancer Institute·Dianne M FinkelsteinDavid A Schoenfeld
Mar 13, 2012·Pharmaceutical Statistics·Guogen ShanGregory E Wilding
Aug 21, 2013·Journal of Biopharmaceutical Statistics·Guogen ShanGregory E Wilding
Oct 9, 2013·Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology·David R SpigelAmy C Peterson
Oct 24, 2014·Pharmaceutical Statistics·Jianrong Wu
Oct 27, 2015·Journal of Statistical Planning and Inference·Andrew WatersMarina Vannucci
Nov 4, 2015·Statistics in Medicine·Guogen ShanShawn Gerstenberger
Aug 4, 2016·BMC Medical Research Methodology·Guogen ShanTao Jiang
Apr 5, 2019·BMC Medical Research Methodology·Guogen Shan, Hua Zhang
Apr 30, 2019·Therapeutic Innovation & Regulatory Science·Guogen ShanGregory E Wilding

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Citations

Feb 9, 2021·Contemporary Clinical Trials Communications·Guogen Shan
Jun 5, 2021·Pharmaceutical Statistics·Jianrong WuChia-Wei Hsu

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