For companies developing marketing authorization applications (MAAs) for recombinant and human plasma-derived factor VIII and factor IX hemophilia medicines in previously untreated patients (PUPs), the European Medicines Agency (EMA) announced Friday that data should be collected from patient registries rather than from small clinical trials.
Revised guidance from EMA spelling out the changes notes the aim is to optimize the use of these registries and provide parameters for core data sets that should be collected.
“The new approach described in the hemophilia guidelines waiving the requirement for a clinical trial in previously untreated patients was discussed at an EMA workshop on hemophilia registries in July 2015
where the use of registries in hemophilia was explored,” the agency explained.
The revised guideline notes that discussions at the workshop centered around how the number of suitable patients, especially PUPs, to be enrolled in clinical trials is problematic.
“Hence, the conduct of sufficiently informative clinical trials in PUPs to estimate important characteristics of single products is considered difficult. Following a public consultation in 2017, a second workshop on hemophilia 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 hemophilia medicines.”
The workshop discussed recommendations on the appropriate governance of registries, patient consent, data collection, data quality and data sharing, and interoperability between different registries.
“Therefore the obligation to perform clinical trials in PUPs for marketing authorization purposes has been deleted,” the guideline says.
EMA is also offering a parameter set for hemophilia registries in the guideline, seeking information on demographic data, anamnestic information, hemophilia treatment information, inhibitor information and other relevant information on concomitant events.
The revised guideline for hemophilia medicines for factor VIII deficiency
was published in July 2018, and the revised guideline addressing medicines for factor IX deficiency is published today.
The public consultation on the revised factor IX guideline ends 30 June 2019.
Guideline on clinical investigation of recombinant and human plasma-derived factor IX products
Q&A 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)