Sensitive Detection of Therapeutic Efficacy with the ETDRS Diabetic Retinopathy Severity Scale.

ABSTRACT:

Background

The Early Treatment Diabetic Retinopathy Study Diabetic Retinopathy Severity Scale (DRSS) is a standard approach to measure diabetic retinopathy (DR) severity. Many clinical trials evaluating drug intervention for DR rely upon demonstration of a therapeutic effect through measurement of a 2- or 3-step improvement or progression on the DRSS; however, these binary endpoints require a relatively large sample size for a reliable estimate of therapeutic efficacy, especially when the SOC (eg, anti-VEGF) is used as a control. This study was designed to evaluate the sensitivity and statistical efficiency of detecting a drug effect in DR across different DRSS endpoints, and present alternative analytical approaches to enable smaller-size DR trials for detecting a reliable efficac

SUBMITTER: Zhang J 

PROVIDER: S-EPMC7751690 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature