Linear-rank testing of a non-binary, responder-analysis, efficacy score to evaluate pharmacotherapies for substance use disorders.

ABSTRACT: The design of pharmacological trials for management of substance use disorders is shifting toward outcomes of successful individual-level behavior (abstinence or no heavy use). While binary success/failure analyses are common, McCann and Li (CNS Neurosci Ther 2012; 18: 414-418) introduced "number of beyond-threshold weeks of success" (NOBWOS) scores to avoid dichotomized outcomes. NOBWOS scoring employs an efficacy "hurdle" with values reflecting duration of success. Here, we evaluate NOBWOS scores rigorously. Formal analysis of mathematical structure of NOBWOS scores is followed by simulation studies spanning diverse conditions to assess operating characteristics of five linear-rank tests on NOBWOS scores. Simulations include assessment of Fisher's exact test applied to hurdle component.

SUBMITTER: Holmes TH 

PROVIDER: S-EPMC6690342 | biostudies-literature | 2018 Jul

REPOSITORIES: biostudies-literature