Posted by Michael Wonder on 30 Nov 2018
Data from patient registries to replace clinical trials in previously untreated haemophilia patients
30 November 2018 - EMA has published revised guidelines on the tests and studies needed to support marketing authorisation applications for certain haemophilia medicines.
The revision introduces an important change in relation to the investigation of recombinant and human plasma-derived factor VIII and factor IX haemophilia medicines in previously untreated patients: for this very small subset of haemophilia patients data should be collected from patient registries rather than from small clinical trials, that may not be fully representative of how the medicine is used day-to-day once it’s on the market.
This new approach relies on data from registries as a source of high-quality, real-world data to support regulatory decision-making. It reflects that in this subset of patients, clinical data are difficult to obtain as the subset is highly-selected and the numbers available for trials are very small.
