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

Katarzyna Jóźwiak and Mirjam Moerbeek

Computational Statistics & Data Analysis, 2012, vol. 56, issue 6, 2086-2096

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.

Keywords: Discrete-time longitudinal data; Survival analysis; Optimal design; Cost function (search for similar items in EconPapers)
Date: 2012
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Citations: View citations in EconPapers (2)

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Persistent link: https://EconPapers.repec.org/RePEc:eee:csdana:v:56:y:2012:i:6:p:2086-2096

DOI: 10.1016/j.csda.2011.12.018

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