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Cost-effective designs for trials with discrete-time survival endpoints

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  • Jóźwiak, Katarzyna
  • Moerbeek, Mirjam

Abstract

In studies on event occurrence, the timing of events may be measured continuously using thin precise units or discretely using time periods. The design of trials with continuous-time survival endpoints has been studied for years, but very little is known about the design of trials with discrete-time survival endpoints. The optimal designs for trials where observations are recorded at discrete points in time is calculated using the generalized linear model and Weibull distribution. Applying a cost function, the optimal number of subjects and time periods are found in such a way that a sufficient power level is achieved at a minimal cost or the power level is maximized for a fixed budget. Taking the budget for a trial and the cost ratio between recruiting a new subject and obtaining a measurement per subject into account, it is observed that the cost ratio and the shape of the survival function have the greatest influence on the optimal design.

Suggested Citation

  • Jóźwiak, Katarzyna & Moerbeek, Mirjam, 2012. "Cost-effective designs for trials with discrete-time survival endpoints," Computational Statistics & Data Analysis, Elsevier, vol. 56(6), pages 2086-2096.
    • Handle: RePEc:eee:csdana:v:56:y:2012:i:6:p:2086-2096
    DOI: 10.1016/j.csda.2011.12.018
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    References listed on IDEAS

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    1. Lima Passos, Valéria & Tan, Frans E.S. & Berger, Martijn P.F., 2011. "Cost-efficiency considerations in the choice of a microarray platform for time course experimental designs," Computational Statistics & Data Analysis, Elsevier, vol. 55(1), pages 944-954, January.
    2. Hashimoto, Elizabeth M. & Ortega, Edwin M.M. & Paula, Gilberto A. & Barreto, Mauricio L., 2011. "Regression models for grouped survival data: Estimation and sensitivity analysis," Computational Statistics & Data Analysis, Elsevier, vol. 55(2), pages 993-1007, February.
    3. Tekle, Fetene B. & Tan, Frans E.S. & Berger, Martijn P.F., 2008. "Maximin D-optimal designs for binary longitudinal responses," Computational Statistics & Data Analysis, Elsevier, vol. 52(12), pages 5253-5262, August.
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    Cited by:

    1. Xiao-Dong Zhou & Yun-Juan Wang & Rong-Xian Yue, 2021. "Optimal designs for discrete-time survival models with random effects," Lifetime Data Analysis: An International Journal Devoted to Statistical Methods and Applications for Time-to-Event Data, Springer, vol. 27(2), pages 300-332, April.
    2. Safarkhani, Maryam & Moerbeek, Mirjam, 2014. "The influence of a covariate on optimal designs in longitudinal studies with discrete-time survival endpoints," Computational Statistics & Data Analysis, Elsevier, vol. 75(C), pages 217-226.

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