The clinical assessment of studies on orphan drugs in relation to the EMA's authorization marketing decisions in Europe.

ABSTRACT:

Introduction

The main objective of this study was to assess the correlation between the methodological characteristics of clinical trials on orphan drugs and the special statuses granted by the European Medicines Agency (EMA).

Material and methods

Data were collected for all medicines with orphan designation assigned by 2020. From August 2019 to June 2020, special statuses (authorization statuses and registration requirements) and general information on orphan drugs were obtained from the EMA's web-based registry. The following clinical data were collected: number of patients, clinical phase, randomization, masking, control group, treatment durations, and safety and efficacy follow-ups. Descriptive, comparative, multivariate, and univariate analyses of data were conducted.

SUBMITTER: Jakubowski S 

PROVIDER: S-EPMC11994444 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature