Online Program Home
My Program

Abstract Details

Activity Number: 307 - Clinical Trial Design-3
Type: Contributed
Date/Time: Tuesday, July 31, 2018 : 8:30 AM to 10:20 AM
Sponsor: Biopharmaceutical Section
Abstract #329467 Presentation
Title: Statistical Leadership in Clinical Trials: Opportunities from the Draft Estimand Guidance
Author(s): Jonathan Siegel*
Companies: Bayer HealthCare Pharmaceuticals Inc.
Keywords: Estimand; missing data; informative; intercurrent events; statistical leadership; counterfactual

The R1 estimand E9 addendum addresses a gap between standard assumptions and clinical experience. Missing values, currently modeled as noise, are often strongly correlated with treatment outcomes. The estimand concept reconceptualizes patient dropout etc. as intercurrent events, informative qualitative data descriptive of or counterfactual to the treatment outcomes of interest. As current methods, surveyed, depend on strong assumptions, the guidance emphasizes appropriate visit schedules and monitoring practices, and sensitivity analyses to detect departures from assumptions. The recognition of gaps between traditional assumptions and clinical reality provides new opportunities for statistical leadership, from selecting endpoints and making scheduling and monitoring choices in trial designs to developing new, more robust methods embracing the paradigm of informative intercurrent events. It increases the value of conceptual understanding, interfunctional communications, clear goals, proactive planning and design, prevention, and statistics conceived as an engineering discipline, vs. focus on post-hoc analysis/adjustment. Key opportunities for leadership with examples are outlined.

Authors who are presenting talks have a * after their name.

Back to the full JSM 2018 program