Bayesian trial designs

L. M. Haines, 1. Perevozskaya, and W. F. Rosenberger, Bayesian optimal designs for Phase 1 clinical trials. Biometrics 59 591-600 (2003). 

Cheng Y, Shen Y (2005) Bayesian adaptive designs for clinical trials. Biometrlka 92 633-646. 

Kadane J (1996) Bayesian Methods and Ethics in Clinical Trials Design. John Wiley Sons, Inc., New York... 

D. A. Berry, P. Muller, and A. P. Grieve, Adaptive Bayesian Designs for Dose-Ranging Trials. Case Studies in Bayesian Statistics, Vol. V. Springer Verlag, New York, 2002, pp. 99-181. 

Both major schools of statistical inference, the Bayesian and the frequentist, require prior consideration of possible analyses at the time trials are run. The Bayesian school requires specification of prior probabilities. To make these genuine priors they have to be truly prior and hence determined before the data are obtained. The frequentist school requires specification of the tests that will be performed and the decisions that will be made depending on various outcomes. For both schools, in order to design salient experiments, it is necessary to pay attention to very many aspects of design. 

Simon R, Freedman LS (1997) Bayesian design and analysis of two x two factorial clinical trials. 

For this and other reasons, I am not particularly keen on the use of assurance as the primary criterion for designing a clinical trial, although it may well be useful to calculate it in addition. My own view is that if I am going to be Bayesian about sample size calculation I would rather be hanged for a sheep than a lamb and use the methods of section 13.2.15. 

The general pharmaceutical physician cannot be expected to be able to generate Bayesian statistical plans for him/herself. These require an experienced statistician and, it may be added, a statistician who is not, him/herself, philosophically opposed to Bayesian rather than frequentist thinking. The decision to employ a Bayesian design for a clinical trial will be viewed as courageous in most companies, and there will be many clinical trials for which an orthodox, frequentist approach will be selected, for several good reasons. Overall, the gen-... 

Bayesian designed trials provide probabilities of treatment effects that apply directly to the next patient who is similar to those treated in any completed or ongoing trial. This approach provides probabilities that can be used in formal decision analysis. 

Chen, M.-H., Ibrahim, J. G., Lam, R, Yu, A., and Zhang, Y. (2011) Bayesian design of non-inferiority trials for medical devices using historical data, Biometrics 67,1163-1170. 

In general, the use of Bayesian network meta-analysis has broad applicability to evaluate AEs between related drugs. These methods can provide insight to prescribers and also assess cost-effectiveness. The direct probability statements that result from the Bayesian approach are helpful to decision makers evaluating a variety of medical products for a given therapeutic area/indication. Furthermore, the information obtained from the network meta-analysis can be utilized throughout the medical product development life cycle in simulations to design future clinical trials. 

Covers several Bayesian approaches, including a meta-experimental design for evaluating cardiovascular risk, adaptive trials for drug safety, and network meta-analysis for safety evaluation... 

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