title: A new futility test approach in clinical interim data monitoring.
abstract: Sequential monitoring of efficacy and safety data has become a vital component of modern clinical trials. It affords companies the opportunity to stop studies early in cases when it appears as if the primary objective will not be achieved or when there is clear evidence that the primary objective has already been met. This paper introduces a new concept of the backward conditional hypothesis test (BCHT) to evaluate clinical trial success. Unlike the regular conditional power approach that relies on the probability that the final study result will be statistically significant based on the current interim look, the BCHT was constructed based on the hypothesis test framework. The framework comprises a significant test level as opposed to the arbitrary fixed futility index utilized in the conditional power method. Additionally, the BCHT has proven to be a uniformly most powerful test. Noteworthy features of the BCHT method compared with the conditional power method will be presented.
authors.affiliation: Merck Research Laboratories, Merck & Co., Inc., Rahway, NJ 07065, USA. dixixue@aol.com
authors.forename: Dixi
authors.initials: D
authors.lastname: Xue
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods*; D003627:Data Interpretation, Statistical; D003657:Decision Making; D064420:Drug-Related Side Effects and Adverse Reactions; D057239:Early Termination of Clinical Trials; D023381:Endpoint Determination; D006801:Humans; D004364:Pharmaceutical Preparations / Q000008:administration & dosage; D011336:Probability; D012107:Research Design
chemical_list: D004364:Pharmaceutical Preparations