"Time Saved" Calculations to Improve Decision-Making in Progressive Disease Studies.

ABSTRACT:

Background

Disease modifying therapies (DMTs) may be most beneficial in early disease, when progression is slow and changes small, with clinical relevance difficult to interpret.

Objectives

Time component tests (TCTs) translate differences between treatments from mean change, vertical distance between longitudinal trajectories, into intuitively understood time saved, horizontal distance between trajectories, which can be readily combined across endpoints in a global TCT (gTCT).

Design

The value of composites, time savings estimates, and combination scores to optimize measurement and interpretation of DMTs are demonstrated, along with construction details and simulation studies.

Setting

TCT methods were applied to a randomized phase II clinical trial.

Partici

SUBMITTER: Dickson SP 

PROVIDER: S-EPMC11060991 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature