Abstract
Drug development is a continuum. Information from completed trials and other sources may be available when we design a new trial. In this chapter we consider the Bayesian approach to incorporate existing information into future trial planning. Under the Bayesian approach, prior distributions are used to describe the accumulated knowledge about unknown parameters such as those representing treatment effects. The prior distributions are then used to calculate the probability of a successful trial or assurance probability. In this chapter, we look at closed-form expressions for assurance probabilities under some definitions of success. We can use simulations to estimate these probabilities when close-form expressions do not exist. With the use of prior distributions for the parameters of interest, we show how to assess the positive and negative predictive values of a design with its companion decision rule.
Data! Data! Data! I can’t make bricks without clay.
Sir Arthur Conan Doyle
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References
Choy, S. L., O’Leary, R., & Mengersen, K. (2009). Elicitation by design in ecology: Using expert opinion to inform priors for Bayesian statistical models. Ecology, 90(1), 265–277.
Cornfield, J. (1969). The Bayesian outlook and its application. Biometrics, 25(4), 617–657.
Gore, S. M. (1987). Biostatistics and the medical research council. Medical Research Council News, 35, 19–21.
Hobbs, B. P., & Carlin, B. P. (2008). Practical Bayesian design and analysis for drug and device clinical trials. Journal of Biopharmaceutical Statistics, 18(1), 54–80.
Kinnersley, N., & Day, S. (2013). Structured approach to the elicitation of expert beliefs for a Bayesian-designed clinical trial: A case study. Pharmaceutical Statistics, 12, 104–113.
Kowalski, K. G., French, J. L., Smith, M. K., Hutmacher, M. M. (2008a). A model-based framework for quantitative decision-making in drug development. Presented at the American Conference on Pharmacometrics, Tucson AZ, March 12. Retrieved December 7, 2016, from http://www.acop7.org/assets/Legacy_ACOPs/2008ACOP/Main_Program_Presentations/8-kowalski_final.pdf
Kowalski, K. G., Olson, S., Remmers, A. E., & Hutmacher, M. M. (2008b). Modeling and simulation to support dose selection and clinical development of SC-75416, a selective COX-2 inhibitor for the treatment of acute and chronic pain. Clinical Pharmacology & Therapeutics, 83(6), 857–866.
Kynn, M. (2008). The ‘heuristics and biases’ bias in expert elicitation. Journal of the Royal Statistical Society, Series A, 171(1), 239–264.
Lalonde, R. L., Kowalski, K. G., Hutmacher, M. M., et al. (2007). Model-based drug development. Clinical Pharmacology & Therapeutics, 82(1), 21–32.
Lunn, D., Jackson, C., Best, N., et al. (2013). The BUGS book: A practical introduction to Bayesian analysis. Boca Raton: Chapman Hall/CRC Press.
Milligan, P. A., Brown, M. J., Marchant, B., et al. (2013). Model-based drug development: A rational approach to efficiently accelerate drug development. Clinical Pharmacology & Therapeutics, 93(6), 502–514.
O’Hagan, A., Buck, C. E., Daneshkhah, A., et al. (2006). Uncertain judgements: Eliciting experts’ probabilities. Hoboken, NJ: Wiley.
O’Hagan, A., Stevens, J. W., & Campbell, M. J. (2005). Assurance in clinical trial design. Pharmaceutical Statistics, 4(3), 187–201.
OpenBUGS. http://www.openbugs.net/w/FrontPage
R Core Team. (2014). R: A language and environment for statistical computing. Vienna: R Foundation for Statistical Computing. isbn:3-900051-07-0. http://www.R-project.org/
SAS. (2013). SAS/stat ® user’s guide, version 13.1. Cary, NC: SAS Institute Inc.
Schmidli, H., Gsteiger, S., Roychoudhury, S., et al. (2014). Robust meta-analytic-predictive priors in clinical trials with historical control information. Biometrics, 70(4), 1023–1032.
Spiegelhalter, D. J., Abrams, K. R., & Myles, J. P. (2004). Bayesian approaches to clinical trials and health-care evaluation. Hoboken, NJ: Wiley.
Walley, R. J., Smith, C. L., Gale, J. D., & Woodward, P. (2015). Advantages of a wholly Bayesian approach to assessing efficacy in early drug development: A case study. Pharmaceutical Statistics, 14, 205–215.
WinBUGS. http://www.mrc-bsu.cam.ac.uk/software/bugs/the-bugs-project-winbugs/
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Chuang-Stein, C., Kirby, S. (2017). Incorporating Information from Completed Trials in Future Trial Planning. In: Quantitative Decisions in Drug Development. Springer Series in Pharmaceutical Statistics. Springer, Cham. https://doi.org/10.1007/978-3-319-46076-5_5
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DOI: https://doi.org/10.1007/978-3-319-46076-5_5
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