Enrichment design with patient population augmentation
lu cui, ABBVIE *Bo Yang, ABBVIE Lanju Zhang, Abbvie Inc Yijie Zhou, ABBVIE Keywords: Biomarker, Enrichment Design Guidance, Screening, Weighted Test Statistic The advancement in science (e.g., biomarkers from genomics, proteomics) has provided opportunities to identify patient sub-populations that may be more responsive to a treatment. Clinical trials can be enriched on such sub-populations to improve the probability of success in demonstrating the benefit of new treatments. In 2012 FDA issued a draft guidance document to facilitate trial design with an enrichment strategy. In this talk, we consider an enrichment trial design where efficacy in the enriched population and further in general patient population can be evaluated. Specifically, a new weighted test statistic is derived to assess the treatment effect in a general patient population under an enriched trial setting, coupled with novel design based on screening information for weight determination. The proposed design and analysis method enhance the probability of success compared with a traditional all-comer trial design. It allows a generalization of the enrichment trial result to an all-comer population despite the fact the enriched population is disproportionally distributed in the study. Sample size determination and data collection are discussed along with examples and simulations.
|
Key Dates
-
November 1 - December 17, 2013
Online proposal submission for a session, short course and Town Hall Open -
January 6 - March 11, 2014
Online proposal submission for Roundtables Open -
April 30 - May 28, 2014
Abstract Submission Open -
June 4, 2014
Online Registration Opens -
August 8 - August 22, 2014
Invited Abstract Editing -
August 11, 2014
Short Course materials due from Instructors -
September 1, 2014
Housing Deadline -
September 15, 2014
Cancellation Deadline and Registration Closes @ 11:59 pm EDT -
September 22 - September 24, 2014
Marriott Wardman Park, Washington, DC