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Appendix C

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.

GENERAL

Bohning, D. 1999. Computer-Assisted Analysis of Mixtures and Applications: MetaAnalysis, Disease Mapping, and Others. Boca Raton, FL: Chapman & Hall/CRC.

Cai, J., H. Zhou, and C. E. Davis. 1997. Estimating the mean hazard ratio parameters for clustered survival data with random clusters. Statistics in Medicine 16:2009–2020.

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.



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Page 177 Appendix C 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. GENERAL Bohning, D. 1999. Computer-Assisted Analysis of Mixtures and Applications: MetaAnalysis, Disease Mapping, and Others. Boca Raton, FL: Chapman & Hall/CRC. Cai, J., H. Zhou, and C. E. Davis. 1997. Estimating the mean hazard ratio parameters for clustered survival data with random clusters. Statistics in Medicine 16:2009–2020. 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.

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Page 178 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. Gilbert, P. B., S. G. Self, and M. A. Ashby. 1998. Statistical methods for assessing differential vaccine protection against human immunodeficiency virus types. Biometrics 54:799–814. 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. Goodman, S. N. 1999. Toward evidence-based medical statistics. 1. The P value fallacy. Annals of Internal Medicine 130:995–1004. Goodman, S. N. 1999. Toward evidence-based medical statistics. 2. The Bayes factor. Annals of Internal Medicine 130:1005–1013. Goodman, S. N., and J. A. Berlin. 1994. The use of predicted confidence intervals when planning experiments and the misuse of power when interpreting results. Annals of Internal Medicine 121:200–206. Goodman, S. N., D. G. Altman, and S. L. George. 1998. Statistical reviewing policies of medical journals: caveat lector? Journal of General Internal Medicine 13:753–756. Halloran, M. E., and D. Berry, eds. 2000. Statistical Models in Epidemiology, the Environment, and Clinical Trials. The Ima Volumes in Mathematics and Its Applications, Vol. 116. New York: Springer-Verlag. Hoppensteadt, F. C., and C. S. Peskin. 1992. Mathematics in Medicine and the Life Sciences. New York: Springer-Verlag. Ildstad, S. T., D. J. Tollerud, M. E. Bigelow, and J. P. Remensnyder. 1989. A multivariate analysis of determinants of survival for patients with squamous cell carcinoma of the head and neck. Annals of Surgery 209:237–41. Jennison, C., and B. W. Turnbull. 1983. Confidence intervals for a binomial parameter following a multistage test with application to MIL-STD 105D and medical trials. Technometrics 25:49–58. Khoury, M. J., T. H. Beaty, and B. H. Cohen. 1993. Fundamentals of Genetic

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