EPAR's of orphan drugs from EMA 2019-2020

The aim of this study was to assess the correlation between the methodological characteristics of clinical trials on orphan drugs (ODs) and the registration status granted by the European Medicines Agency (EMA). From August 2019 to June 2020, general information on ODs was obtained from the EMA’s web-based registry. The following clinical data were collected from European public assessment reports: the number of patients, study design (randomized controlled trial, dose-response trial, retrospective study), number of sites, clinical phase, study characteristics (randomization, blinding, group control, number of arms), treatment and study duration, safety and efficacy follow-up, time of the last follow-up visit, and quality-of-life assessment. Descriptive, comparative, and univariate analyses of data were conducted.