A general approach for sample size calculation for the three-arm 'gold standard' non-inferiority design

被引:26
作者
Stucke, Kathrin [1 ]
Kieser, Meinhard [1 ]
机构
[1] Heidelberg Univ, Inst Med Biometry & Informat, D-69120 Heidelberg, Germany
关键词
non-inferiority; three-arm trial; sample size calculation; optimal allocation; multiple comparisons; intersection-union test; PLACEBO-CONTROLLED TRIAL; BINARY END-POINTS; ESTABLISHING EFFICACY; MULTIPLE-SCLEROSIS; STATISTICS; DIFFERENCE; ISSUES;
D O I
10.1002/sim.5461
中图分类号
Q [生物科学];
学科分类号
07 ; 0710 ; 09 ;
摘要
In the three-arm gold standard non-inferiority design, an experimental treatment, an active reference, and a placebo are compared. This design is becoming increasingly popular, and it is, whenever feasible, recommended for use by regulatory guidelines. We provide a general method to calculate the required sample size for clinical trials performed in this design. As special cases, the situations of continuous, binary, and Poisson distributed outcomes are explored. Taking into account the correlation structure of the involved test statistics, the proposed approach leads to considerable savings in sample size as compared with application of ad hoc methods for all three scale levels. Furthermore, optimal sample size allocation ratios are determined that result in markedly smaller total sample sizes as compared with equal assignment. As optimal allocation makes the active treatment groups larger than the placebo group, implementation of the proposed approach is also desirable from an ethical viewpoint. Copyright (C) 2012 John Wiley & Sons, Ltd.
引用
收藏
页码:3579 / 3596
页数:18
相关论文
共 33 条
[1]  
[Anonymous], ICH TOPIC E 10
[2]  
[Anonymous], MVTNORM MULTIVARIATE
[3]  
Burchardi N, 2010, THESIS
[4]  
Committee for Medicinal Products for Human Use (CHMP), REFLECTION PAPER ON
[5]  
Committee for Medicinal Products for Human Use (CHMP), GUIDELINE ON CLINICA
[6]  
Committee for Proprietary Medicinal Products (CPMP), NOTE FOR GUIDANCE ON
[7]   Non-inferiority trials: design concepts and issues - the encounters of academic consultants in statistics [J].
D'Agostino, RB ;
Massaro, JM ;
Sullivan, LM .
STATISTICS IN MEDICINE, 2003, 22 (02) :169-186
[8]   TEST STATISTICS AND SAMPLE-SIZE FORMULAS FOR COMPARATIVE BINOMIAL TRIALS WITH NULL HYPOTHESIS OF NONZERO RISK DIFFERENCE OR NON-UNITY RELATIVE RISK [J].
FARRINGTON, CP ;
MANNING, G .
STATISTICS IN MEDICINE, 1990, 9 (12) :1447-1454
[9]   Blinded sample size reestimation with count data: Methods and applications in multiple sclerosis [J].
Friede, Tim ;
Schmidli, Heinz .
STATISTICS IN MEDICINE, 2010, 29 (10) :1145-1156
[10]   CLINICAL AND STATISTICAL ISSUES IN THERAPEUTIC EQUIVALENCE TRIALS [J].
GARBE, E ;
ROHMEL, J ;
GUNDERTREMY, U .
EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY, 1993, 45 (01) :1-7