A standardized metric to enhance clinical trial design and outcome interpretation in type 1 diabetes.

ABSTRACT: The use of a standardized outcome metric enhances clinical trial interpretation and cross-trial comparison. If a disease course is predictable, comparing modeled predictions with outcome data affords the precision and confidence needed to accelerate precision medicine. We demonstrate this approach in type 1 diabetes (T1D) trials aiming to preserve endogenous insulin secretion measured by C-peptide. C-peptide is predictable given an individual's age and baseline value; quantitative response (QR) adjusts for these variables and represents the difference between the observed and predicted outcome. Validated across 13 trials, the QR metric reduces each trial's variance and increases statistical power. As smaller studies are especially subject to random sampling variability, using QR as the out

SUBMITTER: Ylescupidez A 

PROVIDER: S-EPMC10632453 | biostudies-literature | 2023 Nov

REPOSITORIES: biostudies-literature