Zhang J, et al. - In the present study, the researchers sought to assess the sensitivity and statistical efficiency of detecting a drug effect in diabetic retinopathy (DR) across different Diabetic Retinopathy Severity Scale (DRSS) endpoints, and present alternative analytical approaches to enable smaller-size DR trials for identifying a reliable efficacy signal before moving into larger confirmatory DR trials. Data from two randomized, double-blinded, controlled Phase III trials, that recruited patients with reduced vision due to center-involved DME and the presence of macular edema documented on optical coherence tomography and simulated data, were used for this investigation. More robust sensitivity and statistical efficiency can be given by DRSS step change. Therefore, the identification of the therapeutic efficacy signal in drug discoveries in DR is a more cost-effective endpoint.