This paper describes a Bayesian approach to the design and analysis of clinical trials, with special reference to Phase III drug trials. The focus is on two rather general areas that are important in clinical trials: (1) monitoring trials with the possibility of changing the trial's design, and (2) combining various sources of information concerning the effect of the drug. Topics covered include survival analysis, analysis of trials in which the patients have different prognoses, multicenter trials and meta-analysis.
BergerJOBerryDA. Statistical Analysis and the Illusion of Objectivity. Am Scientist.1988;76: 159–165.
2.
BergerJOWolpertRL. The Likelihood Principle. Hayward, California: Institute of Mathematical Statistics; 1984.
3.
BerryDA. Interim analysis in clinical trials: The role of the likelihood principle. Am. Statistician.1987;41: 117–122.
4.
PocockSJ. Group sequential methods in the design and analysis of clinical trial. Biometrika.1977;64: 191–199.
5.
O'BrienPCFlemingTR. A multiple testing procedure for clinical trials. Biometrics.1979;35: 549–556.
6.
McPhersonK.On choosing the number of interim analyses in clinical trials. Stat Med.1982;1: 25–36.
7.
JennisonCTurnbullBW. Repeated confidence intervals for group sequential clinical trials. Cont Clin Trials.1984;5: 33–45.
8.
BerryDA. Inferential aspects of adaptive allocation rules. Proceedings of the Pharmaceutical Section of the American Statistical Association; 1989.
9.
MostellerFGilbertJPMcPeekB.Controversies in design and analysis of clinical trials. In ShapiroSHLouisTA, eds. Clinical Trials.New York: Marcel Dekker; 1983: 13–64.
10.
PetoRPikeMCArmitagePBreslowNECoxDRHowardSVMantelNMcPhersonKPetoJSmithPG. Design and analysis of randomized clinical trials requiring prolonged observation of each patient: 1. Introduction and design. Br J Cancer.1976;34: 585–612.
11.
BerryDALindgrenBW. Statistics: Theory and Methods.Pacific Grove, California: Brooks/Cole. 1990: 282–284.
12.
FreireichEJGehanEFreiESchroederLRWolmanIJAnbariRBurgertEOMillsSDPinkelDSelawryOSMoonJHGendelBRSpurrCLStorrsRHauraniFHoogstratenBLeeS.The effect of 6-mercaptopurine on the duration of steroid-induced remissions in acute leukemia: A model for evaluation of other potentially useful therapy. Blood.1963;21: 699–716.
13.
BergerJOBerryDA. The relevance of stopping rules in statistical inference. In: Statistical Decision Theory and Related Topics IV. 1. New York: Springer-Verlag; 1988: 29–72.
14.
BerryDAFristedtB.Bandit Problems: Sequential Allocation of Experiments. New York: Chapman-Hall; 1985.
15.
BerryDAHoC-H.One-sided sequential stopping boundaries for clinical trials: A decision-theoretic approach. Biometrics.1988;44: 219–227.
16.
BerryDA. Monitoring accumulating data in a clinical trial. Biometrics.1989;45: 1197–1211.
17.
EnasNHBerryDADiesFGonzalesCR. Bayesian methods for monitoring survival in congestive heart failure. In PeaceKE, ed. Biopharmaceutical Sequential Statistical Applications.New York: Marcel Dekker; In press.
18.
LindleyDVSmithAFM. Bayes estimates for the linear model with discussion. J R Stat Assoc [Series B].1972;34: 1–41.
19.
DuMouchelW.Bayesian metaanalysis. In BerryDA, ed. Statistical Methodology in the Pharmaceutical Sciences.New York: Marcel Dekker; 1989: 509–529.
20.
JanicakPGLipinskiJDavisJMComatyJEWaternauxCCohenBAltmanESharmaRP. S-Adenosyl-Methionine (SAMe) in depression: A literature review and preliminary data report. Ala J Med Sci.1988;25: 306–312.
21.
BergerJO. Statistical Decision Theory and Bayesian Analysis. 2nd ed.New York: Springer-Verlag; 1986.
22.
Racine-PoonASmithAFM. Population models. In BerryDA, ed. Statistical Methodology in the Pharmaceutical Sciences.New York: Marcel Dekker; 1989: 139–162.
23.
GrieveAP. Crossover versus parallel designs. In BerryDA, ed. Statistical Methodology in the Pharmaceutical Sciences.New York: Marcel Dekker; 1989: 239–270.
24.
BerryDA. Data dredging, multiple comparisons, and multiple tests: A Bayesian perspective. In BernardoJMDeGrootMHLindleyDVSmithAFM. eds. Bayesian Statistics3. Oxford: Oxford University Press; 1989: 79–94.
25.
PressSJ. Bayesian Statistics. New York: John Wiley & Sons; 1989.
