Multiple-arm superiority and non-inferiority designs with various endpoints.
Multiple-arm dose-response superiority trials are widely studied for continuous and binary endpoints, while non-inferiority designs have been studied recently in two-arm trials. In this paper, a unified asymptotic formulation of a sample size calculation for k-arm (k>0) trials with different endpoints (continuous, binary and survival endpoints) is derived for both superiority and non-inferiority designs. The proposed method covers the sample size calculation for single-arm and k-arm (k> or =2) designs with survival endpoints, which has not been covered in the statistic literature. A simple, closed form for power and sample size calculations is derived from a contrast test. Application examples are provided. The effect of the contrasts on the power is discussed, and a SAS program for sample size calculation is provided and ready to use.
Chang
Mark
M
Millennium Pharmaceuticals Inc., Cambridge, MA 02139, USA. mark.chang@statisticians.org
D018849:Controlled Clinical Trials as Topic / Q000379:methods* / Q000706:statistics & numerical data; D003627:Data Interpretation, Statistical; D004305:Dose-Response Relationship, Drug; D023381:Endpoint Determination; D006801:Humans; D015233:Models, Statistical; D012107:Research Design; D018401:Sample Size; D016019:Survival Analysis; D016896:Treatment Outcome