title: Statistical Perspectives on Subgroup Analysis: Testing for Heterogeneity and Evaluating Error Rate for the Complementary Subgroup.
abstract: Substantial heterogeneity in treatment effects across subgroups can cause significant findings in the overall population to be driven predominantly by those of a certain subgroup, thus raising concern on whether the treatment should be prescribed for the least benefitted subgroup. Because of its low power, a nonsignificant interaction test can lead to incorrectly prescribing treatment for the overall population. This article investigates the power of the interaction test and its implications. Also, it investigates the probability of prescribing the treatment to a nonbenefitted subgroup on the basis of a nonsignificant interaction test and other recently proposed criteria.
authors.affiliation: a Division of Biometrics III , Office of Biostatistics, OTS, CDER, FDA , Silver Spring , Maryland , USA.
authors.forename: Mohamed
authors.initials: M
authors.lastname: Alosh
authors.affiliation: b Office of Biostatistics, OTS, CDER, FDA , Silver Spring , Maryland , USA.
authors.forename: Mohammad F
authors.initials: MF
authors.lastname: Huque
authors.affiliation: c Department of Biostatistics , University of North Carolina at Chapel Hill , Chapel Hill , North Carolina , USA.
authors.forename: Gary G
authors.initials: GG
authors.lastname: Koch
mesh_terms: D000465:Algorithms; D000903:Antibiotics, Antineoplastic / Q000627:therapeutic use; D000970:Antineoplastic Agents / Q000627:therapeutic use; D001241:Aspirin / Q000627:therapeutic use; D002986:Clinical Trials as Topic / Q000706:statistics & numerical data*; D017326:Clinical Trials, Phase III as Topic / Q000706:statistics & numerical data; D000077144:Clopidogrel; D004317:Doxorubicin / Q000627:therapeutic use; D005260:Female; D006801:Humans; D007511:Ischemia / Q000188:drug therapy; D010051:Ovarian Neoplasms / Q000188:drug therapy; D010975:Platelet Aggregation Inhibitors / Q000627:therapeutic use; D015203:Reproducibility of Results; D012107:Research Design / Q000706:statistics & numerical data; D018401:Sample Size; D013988:Ticlopidine / Q000031:analogs & derivatives / Q000627:therapeutic use; D019772:Topotecan / Q000627:therapeutic use
chemical_list: D000903:Antibiotics, Antineoplastic; D000970:Antineoplastic Agents; D010975:Platelet Aggregation Inhibitors; D019772:Topotecan; D004317:Doxorubicin; D000077144:Clopidogrel; D013988:Ticlopidine; D001241:Aspirin
keywords: Complementary subgroup error rate; Supportive assessment; Targeted subgroup, Testing for interaction