Superiority of combining two independent trials in interim futility analysis.

TitleSuperiority of combining two independent trials in interim futility analysis.
Publication TypeJournal Article
Year of Publication2020
AuthorsDeng, Qiqi, Ying-Ying Zhang, Dooti Roy, and Ming-Hui Chen
JournalStat Methods Med Res
Volume29
Issue2
Pagination522-540
Date Published2020 02
ISSN1477-0334
KeywordsClinical Trials, Phase III as Topic, Data Analysis, Data Interpretation, Statistical, Dose-Response Relationship, Drug, Humans, Medical Futility, Models, Statistical, Research Design
Abstract

Traditionally, statistical methods for futility analysis are developed based on a single study. To establish a drug's effectiveness, usually at least two adequate and well-controlled studies need to demonstrate convincing evidence on its own. Therefore, in a standard clinical development program in chronic diseases, two independent studies are generally conducted for drug registration. This paper proposes a statistical method to combine interim data from two independent and similar studies for interim futility analysis and shows that the conditional power approach based on combined interim data has better operating characteristics compared to the approach based on single-trial interim data, even with small to moderate heterogeneity on the treatment effects between the two studies.

DOI10.1177/0962280219840383
Alternate JournalStat Methods Med Res
Original PublicationSuperiority of combining two independent trials in interim futility analysis.
PubMed ID30957713
PubMed Central IDPMC6783334
Grant ListP01 CA142538 / CA / NCI NIH HHS / United States
R01 GM070335 / GM / NIGMS NIH HHS / United States
Project: