Data from patient registries to replace clinical trials in previously untreated haemophilia patients
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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.
The updated guidelines aim to optimise and facilitate the use of these registries and provide parameters for core data sets that should be collected.
The new approach described in the haemophilia guidelines waiving the requirement for a clinical trial in previously untreated patients was discussed at an EMA workshop on haemophilia registries in July 2015 where the use of registries in haemophilia was explored. Following a public consultation in 2017, a second workshop on haemophilia registries was held on 8 June 2018 which aimed at defining the requirements for practical implementation using existing registries to support post-authorisation observational studies of haemophilia medicines. The workshop discussed Report on Haemophilia Registries Workshopon important aspects such as appropriate governance of registries, patient consent, data collection, data quality and data sharing, and interoperability between different registries.
The revised guideline for haemophilia medicines for factor VIII deficiency was published in July 2018 and the revised guideline addressing medicines for factor IX deficiency is published today.
More information on the practical implementation of the guideline is available in a Questions and answers on the revision of the guidelines on clinical investigation of recombinant and human plasma-derived factor VIII products (EMA/CHMP/BPWP/144533/2009 Rev. 2) and FIX products (EMA/CHMP/BPWP/144552/2009 Rev. 2). EMA’s initiative on patient registries is supported by a task force comprising representatives from its scientific committees and working parties, representatives from the European Commission and experts from national competent authorities.