Sensitivity Analysis for Unmeasured Confounding in Medical Product Development and Evaluation Using Real World Evidence

Kavli Affiliate: Xiang Zhang

| First 5 Authors: Peng Ding, Yixin Fang, Doug Faries, Susan Gruber, Hana Lee

| Summary:

The American Statistical Association Biopharmaceutical Section (ASA BIOP)
working group on real-world evidence (RWE) has been making continuous, extended
effort towards a goal of supporting and advancing regulatory science with
respect to non-interventional, clinical studies intended to use real-world data
for evidence generation for the purpose of medical product development and
evaluation (i.e., RWE studies). In 2023, the working group published a
manuscript delineating challenges and opportunities in constructing estimands
for RWE studies following a framework in ICH E9(R1) guidance on estimand and
sensitivity analysis. As a follow-up task, we describe the other issue in RWE
studies, sensitivity analysis. Focusing on the issue of unmeasured confounding,
we review availability and applicability of sensitivity analysis methods for
different types unmeasured confounding. We discuss consideration on the choice
and use of sensitivity analysis for RWE studies. Updated version of this
article will present how findings from sensitivity analysis could support
regulatory decision-making using a real example.

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