'Time Saved' As a Demonstration of Clinical Meaningfulness and Illustrated Using the Donanemab TRAILBLAZER-ALZ Study Findings.

In Alzheimer's disease (AD) clinical trials, disease-modifying therapies are expected to slow the rate of disease progression. Treatment effects are evaluated using a validated clinical scale as the difference between treatment and placebo in mean change from baseline to endpoint. Understanding the clinical relevance of this metric is not necessarily intuitive. Expressing active treatment-placebo difference as a time metric (i.e., months saved with treatment) has potential to provide a metric that is more easily and consistently interpreted. Using data from the TRAILBLAZER-ALZ study, time component tests (TCTs) were employed to determine the time saved with donanemab (an amyloid lowering drug) treatment. At study endpoint (Week 76), disease progression was delayed by 5.3 months and 5.2 months as measured by the Integrated Alzheimer's Disease Rating Scale (iADRS) and the Clinical Dementia Rating Sum of Boxes (CDR-SB), respectively.
Competing Interests: Dickson is a full-time employee of Pentara Corporation (Pentara consults with and receives payment from clients working in the neurodegenerative space, particularly in Alzheimer’s disease [including Eli Lilly and Company]; Pentara was contracted to perform the statistical analyses used in this manuscript); Hendrix is the sole owner of Pentara Corporation; Mallinckrodt is a full-time employee of Pentara Corporation, and a minor stockholder in Eli Lilly and other pharma companies through index mutual funds; Wessels, Dowsett, Sparks, Chatterjee are full time employees and minor stockholders at Eli Lilly and Company.