title: A note on penalty-free interim analyses of clinical studies with binary responses.
abstract: In oncology studies with binary responses, it is often desirable to demonstrate the anti-tumor activity of a test drug before the planned study completes. We propose penalty-free interim analysis strategies that do not change the rejection criterion for the test of null hypothesis at the end. The strategies are applied to standard one-arm and two-arm studies. Although the motivating studies are from oncology, the findings in this research note are equally applicable to similar settings. A broad list of references is provide to stimulate further research on the topic.
authors.affiliation: Merck Research Laboratories, BL 3-2, PO Box 4, West Point, PA 19486, USA. Cong_chen@merck.com
authors.forename: Cong
authors.initials: C
authors.lastname: Chen
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use; D001699:Biometry / Q000379:methods*; D002986:Clinical Trials as Topic / Q000706:statistics & numerical data*; D003627:Data Interpretation, Statistical; D006801:Humans; D015233:Models, Statistical; D009369:Neoplasms / Q000188:drug therapy; D016032:Randomized Controlled Trials as Topic / Q000706:statistics & numerical data; D013997:Time Factors
chemical_list: D000970:Antineoplastic Agents