INCORPORATING HISTORICAL CONTROL DATA IN PLANNING PHASE-II CLINICAL-TRIALS

被引：49

作者：

THALL, PF

SIMON, R

机构：

[1] Statistics, Computer & Information Systems Department, George Washington University, Washington, District of Columbia

[2] Biometric Research Branch, National Cancer Institute, Rockville, Maryland, 20892

来源：

STATISTICS IN MEDICINE | 1990年 / 9卷 / 03期

关键词：

D O I：

10.1002/sim.4780090304

中图分类号：

Q [生物科学];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

Phase II studies of new medical treatments often use historical data on the standard treatment for comparative evaluation. Incorrectly disregarding inherent variability in the historical data may lead to erroneous conclusions regarding the efficacy of the experimental treatment. We propose an approach to phase II trial design which accounts for both inter‐study and intra‐study variation. Our results indicate that it is sometimes best to randomize a proportion of the patients to a control arm. We choose this proportion to maximize the precision of the estimated experimental treatment effect. We evaluate operating characteristics of the design numerically, and provide illustrations based on historical data from cancer chemotherapy trials. Copyright © 1990 John Wiley & Sons, Ltd.

引用

页码：215 / 228

页数：14

共 19 条

[1] Simon R., How large should a phase II trial of a new drug be?, Cancer Treatment Reports, 71, pp. 1079-1085, (1987)
[2] Pocock S.J., The combination of randomized and historical controls in clinical trials, Journal of Chronic Diseases, 29, pp. 175-188, (1976)
[3] Machin D., On the possibility of incorporating patients from non‐randomising centres into a randomised clinical trial, Journal of Chronic Diseases, 32, pp. 347-353, (1979)
[4] Makuch R.W., Simon R., Sample size considerations for non‐randomized comparative studies, Journal of Chronic Diseases, 33, pp. 175-181, (1980)
[5] Dixon D.O., Simon R., Sample size considerations for studies comparing survival curves using historical controls, Journal of Clinical Epidemiology, 41, pp. 1209-1214, (1988)
[6] Tarone R.E., The use of historical control information in testing for a trend in proportions, Biometrics, 38, pp. 215-220, (1982)
[7] Krewski D., Smythe R.T., Dewanji A., Colin D., Statistical tests with historical controls, Carcinogenicity, pp. 23-38, (1988)
[8] Gail M., Williams R., Byar D., Brown C., How many controls?, Journal of Chronic Diseases, 29, pp. 723-731, (1976)
[9] Brittain E., Schlesselman J.J., Optimal allocation for the comparison of proportions, Biometrics, 38, pp. 1003-1009, (1982)
[10] Brooks R.J., Optimal allocation for Bayesian inference about an odds ratio, Biometrika, 74, pp. 196-199, (1987)

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