Selected Bibliography on Small Clinical Trials
This bibliography is a selection of published references dealing with analytical approaches and statistical methods applicable to clinical trials, particularly small clinical trials. The citations span the field broadly and are the result of searches of the Medline database and suggestions from the committee and from the experts in the field who made presentations at the committee's invitational workshop. The references are organized into specific categories to aid the reader. Although comprehensive, the list is selective, and the committee believes that it is an up-to-date bibliography that will assist researchers in learning more about design and analytical methods applicable to small clinical trials.
Bohning, D. 1999. Computer-Assisted Analysis of Mixtures and Applications: MetaAnalysis, Disease Mapping, and Others. Boca Raton, FL: Chapman & Hall/CRC.
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D'Agostino, R. B., and H. Kwan. 1995. Measuring effectiveness. What to expect without a randomized control group. Medical Care 33 (Suppl. 4):AS95–AS105.
Davis, C. E. 1997. Secondary endpoints can be validly analyzed, even if the primary endpoint does not provide clear statistical significance. Controlled Clinical Trials 18:557–560.
Davis, C. E., S. Hunsberger, D. M. Murray, R. R. Fabsitz, J. H. Himes, L. K. Stephenson, B. Caballero, and B. Skipper. 1999. Design and statistical analysis for the Pathways study. American Journal of Clinical Nutrition 69(Suppl. 4):S760–S763.
Dieterich, M., S. N. Goodman, R. R. Rojas-Corona, A. B. Emralino, J, D, Jimenez, and M. E. Sherman. 1994. Multivariate analysis of prognostic features in malignant pleural effusions from breast cancer patients. Acta Cytologica 38:945–952.
Everitt, B. S. 1994. Statistical Methods in Medical Investigations. New York: Halsted Press.
Everitt, B. S. 1995. The Cambridge Dictionary of Statistics in the Medical Sciences. New York: Cambridge University Press.
Gehan, E. A. 1965. A generalized two-sample Wilcoxon test for doubly censored data. Biometrika 52:650–653.
Gehan, E. A., and N. A. Lemak. 1994. Statistics in Medical Research: Developments in Clinical Trials. New York: Plenum Medical Book Co.
Gibbons, J. D., I. Olkin, and M. Sobel. 1999. Selecting and Ordering Populations: A New Statistical Methodology. Philadelphia: SIAM.
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Goodman, S. N. 1992. A comment on replication, p-values and evidence. Statistics in Medicine 11:875–879.
Goodman, S. N. 1993. P values, hypothesis tests, and likelihood: implications for epidemiology of a neglected historical debate. American Journal of Epidemiology 137:485–496; (Discussion, 137:497–501).
Goodman, S. N. 1994. Confidence limits vs power, calculations. Epidemiology 5:266– 268. (Discussion, 5:268–269.
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Goodman, S. N. 1999. Toward evidence-based medical statistics. 2. The Bayes factor. Annals of Internal Medicine 130:1005–1013.
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Levin, B. 1987. Conditional likelihood analysis in stratum-matched retrospective studies with polytomous disease states. Communications in Statistics 16:699–718.
Levin, B. 1990. The saddlepoint correction in conditional logistic likelihood analysis. Biometrika 77:275–285.
Levin, B., and F. Kong. 1990. Barlett's bias correction to the profile score function is a saddlepoint correction. Biometrika 77:219–221.
Levin, B., and H. Robbins. 1981. Selecting the highest probability in binomial or multinomial trials. Proceedings of the National Academy of Sciences USA 78:4663– 4666.
Liang, K. Y., S. G. Self, K. J. Bandeen-Roche, and S. L. Zeger. 1995. Some recent developments for regression analysis of multivariate failure time data. Lifetime Data Analysis 1:403–415.
McPherson, G. 1990. Statistical analysis: the statistician's view. Pp. 154–180 In: Statistics in Scientific Investigation. Its Basis, Application, and Interpretation. New York: Springer-Verlag.
McPherson, G. 1990. Studying association and correlation. Pp. 475–508 In: Statistics in Scientific Investigation. Its Basis, Application, and Interpretation. New York: Springer-Verlag.
Meinert, C. L., 1986. Clinical Trials: Design, Conduct, and Analysis. New York: Oxford University Press.
Murray, D. M. 1998. Design and Analysis of Group-Randomized Trials. Monographs in Epidemiology and Biostatistics, Vol. 29. New York: Oxford University Press.
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Racine-Poon, A., and J. Wakefield. 1998. Statistical methods for population pharmacokinetic modelling. Statistical Methods in Medical Research 7:63–84.
Sevin, S. 1996. Statistical Analysis of Epidemiologic Data. Monographs in Epidemiology and Biostatistics, Vol. 25. New York: Oxford University Press.
Sevin, S. 1998. Modern Applied Biostatistical Methods: Using S-Plus. Monographs in Epidemiology and Biostatistics, Vol. 28. New York: Oxford University Press.
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Brunier, H. C., and J. Whitehead. 1994. Sample sizes for phase II clinical trials derived from Bayesian decision theory. Statistics in Medicine 13:2493–2502.
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Gould, A. L. 1995. Planning and revising the sample size for a trial. Statistics in Medicine 14:1039–1051.
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McMahon, R. P., M. Proschan, N. L. Geller, P. H. Stone, and G. Sopko. 1994. Sample size calculation for clinical trials in which entry criteria and outcomes are counts of events. Statistics in Medicine 13:859–870.
Mehta, C. R., N. R. Patel, and P. Senchaudhuri. 1998. Exact power and sample-size computations for the Cochran-Armitage trend test. Biometrics 54:1615–1621.
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Simon, R. M., and R. W. Makuch. 1982. Sample size requirements for comparing time-to-failure among k treatment groups. Journal of Chronic Disease 35:861–867.
Stallard, N. 1998. Sample size determination for phase II clinical trials based on Bayesian decision theory. Biometrics 54:279–294.
Wels, G., A. Cranney, B. Shea, and P. Tugwell. 1997. Responsiveness of endpoints in osteoporosis clinical trials. Journal of Rheumatology 24:1230–1233.
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Whitehead, J. 1986. Sample sizes for phase II and phase III clinical trials: an integrated approach. Statistics in Medicine 5(5):459-464.
Whitehead, J. 1996. Sample sizes calculations for ordered categorical data. Statistics in Medicine 15:1065–1066.
Whitehead, J., K. Bolland, and M.R. Sooriyarachchi. 1998. Sample size review in a head injury trial with ordered categorical responses. Statistics in Medicine 17:2835–2847.
Wolfe, R., and J. B. Carlin. 1999. Sample-size calculation for log-transformed outcome measure. Controlled Clinical Trials 20:547–554.
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Benson, K., and A. J. Hartz. 2000. A comparison of observational studies and randomized, controlled trials. New England Journal of Medicine 342:1878–1886.
Black, H. R., W. J. Elliott, J. D. Neaton, G. Grandits, P. Grambsch, R. H. Grimm, Jr., L. Hansson, Y. Lacouciere, J. Muller, P. Sleight, M. A. Weber, W. B. White, G. Williams, J. Wittes, A. Zanchetti, and T. D. Fakouhi. 1998. Rationale and design for the Controlled Onset Verapamil Investigation of Cardiovascular Endpoints (CONVINCE) trial. Controlled Clinical Trials 19:370–390.
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Buyse, M., S. L. George, S. Evans, N. L. Geller, J. Ranstam, B. Scherrer, E. Lesaffre, G. Murray, L. Edler, J. Hutton, T. Colton, P. Lachenbruch, and B. L. Verma. 1999. The role of biostatistics in the prevention, detection and treatment of fraud in clinical trials. Statistics in Medicine 18:3435–3451.
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