Related Experiment Video
Updated: Feb 20, 2026

Radiosynthesis, Quality Control, and Small Animal Positron Emission Tomography Imaging of 68Ga-Labelled Nano Molecules
Published on: October 4, 2024
Marketing authorisation of orphan medicines in Europe from 2000 to 2013
Matthias P Hofer1, Hanna Hedman1, Maria Mavris1
1European Medicines Agency, 30 Churchill Place, Canary Wharf, London E14 5 EU, UK.
Abstract:
An analysis was performed on a data set of 157 orphan designated medicines with an outcome for marketing authorisation application (MAA) between 2000 and 2013. The intention was to understand the factors associated with marketing authorisation success, the challenges developers face regarding orphan medicine development, and how scientific advice (SA) is used during development. The results demonstrated that orphan medicines have a lower success rate compared with non-orphan medicines and that determinants for marketing authorisation success were company size and compliance with SA. Compliance with SA could help orphan medicine developers overcome clinical development challenges.
Related Concept Videos
Prescription, Nonprescription and Orphan Drugs
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
Drug Regulation
FDA Approved Drugs: Changes to Approved Drugs
Drug Products: Biologics, Biosimilars and Interchangeables
Clinical Trials: Overview
Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions

