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.

本研究旨在评估孤儿药（orphan drugs, ODs）临床试验的方法学特征与欧洲药品管理局（European Medicines Agency, EMA）所授予的注册状态之间的相关性。2019年8月至2020年6月期间，研究团队从欧洲药品管理局的网络注册数据库获取了孤儿药的基础信息。研究人员从欧洲公共评估报告中采集了以下临床数据：受试患者人数、研究设计（随机对照试验（randomized controlled trial）、剂量-反应试验（dose-response trial）、回顾性研究（retrospective study））、试验中心数量、临床研究阶段、研究特征（随机化（randomization）、设盲（blinding）、组对照（group control）、试验臂数量（number of arms））、治疗与研究持续时长、安全性与有效性随访情况、末次随访时间以及生活质量评估结果。本研究对采集的数据开展了描述性分析、比较性分析及单变量分析。