Type I error probabilities based on design-stage strategies with applications to noninferiority trials

被引:6
作者
Rothmann, M [1 ]
机构
[1] FDA, WOCII, Ctr Drug Evaluat & Res, Div Biometr 1, Rockville, MD 20857 USA
关键词
noninferiority trials; active controlled trials;
D O I
10.1081/BIP-200040847
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
When testing the equality of means from two different populations, a t-test or large sample normal test tend to be performed. For these tests, when the sample size or design for the second sample is dependent on the results of the first sample, the type I error probability is altered for each specific possibility in the null hypothesis. We will examine the impact on the type I error probabilities for two confidence interval procedures and procedures using test statistics when the design for the second sample or experiment is dependent on the results from the first sample or experiment (or series of experiments). Ways for controlling a desired maximum type I error probability or a desired type I error rate will be discussed. Results are applied to the setting of noninferiority comparisons in active controlled trials where the use of a placebo is unethical.
引用
收藏
页码:109 / 127
页数:19
相关论文
共 10 条
[1]   Placebo-controlled trials and active-control trials in the evaluation of new treatments - Part 2: Practical issues and specific cases [J].
Ellenberg, SS ;
Temple, R .
ANNALS OF INTERNAL MEDICINE, 2000, 133 (06) :464-470
[2]  
*FDA, 2001, MED STAT REV XEL
[3]  
Fleming T. R., 1991, COUNTING PROCESSES S
[4]  
FLEMING TR, 1990, J ACQ IMMUN DEF SYND, V3, pS82
[5]   Valid inference in random effects meta-analysis [J].
Follmann, DA ;
Proschan, MA .
BIOMETRICS, 1999, 55 (03) :732-737
[6]   Design and analysis of non-inferiority mortality trials in oncology [J].
Rothmann, M ;
Li, N ;
Chen, G ;
Chi, GYH ;
Temple, R ;
Tsou, HH .
STATISTICS IN MEDICINE, 2003, 22 (02) :239-264
[7]  
ROTHMANN M, 2001, P AM STAT ASS BIOPH, P1
[8]   AN APPROXIMATE DISTRIBUTION OF ESTIMATES OF VARIANCE COMPONENTS [J].
SATTERTHWAITE, FE .
BIOMETRICS BULLETIN, 1946, 2 (06) :110-114
[9]   Placebo-controlled trials and active-control trials in the evaluation of new treatments - Part 1: Ethical and scientific issues [J].
Temple, R ;
Ellenberg, SS .
ANNALS OF INTERNAL MEDICINE, 2000, 133 (06) :455-463
[10]   NONPARAMETRIC-ESTIMATION FOR A SCALE-CHANGE WITH CENSORED OBSERVATIONS [J].
WEI, LJ ;
GAIL, MH .
JOURNAL OF THE AMERICAN STATISTICAL ASSOCIATION, 1983, 78 (382) :382-388