EMA Consults on Revised Factor VIII Product Guidelines

Regulatory NewsRegulatory News | 30 October 2017 |  By 

The European Medicines Agency (EMA) on Monday issued two draft revised guidelines on plasma-derived and recombinant coagulation factor VIII products that remove requirements for conducting clinical studies in previously untreated patients (PUPs).


Factor VIII products are used to treat or prevent bleeding in patients with hemophilia A (congenital factor VIII deficiency). The development of inhibitor antibodies is one of the greatest potential complications involved with treating patients with hemophilia A. When these antibodies appear, they can make factor VIII medicines less effective at controlling bleeding.

According to EMA, inhibitors occur in about 30% of PUPs with severe hemophilia A, typically within 100 days of treatment.

Revised Guidelines

EMA says the two guidelines, one detailing the clinical requirements for supporting the marketing authorization of factor VIII products and the other describing the information that should be included in the summary of product characteristics (SmPC) for those products, are being revised to reflect the difficulty of conducting formal clinical studies in PUPs.

Under the previous versions of the guidelines, EMA required clinical studies in PUPs for all novel recombinant factor VIII products and for factor VIII products made using new production methods. EMA also required PUP studies on a case-by-case basis for plasma-derived factor VIII products.

"In light of increasing scientific knowledge, the number of suitable patients especially previously untreated patients (PUPs) to be enrolled in clinical trials is problematic," EMA writes.

As such, EMA says that sponsors will no longer be required to conduct formal clinical trials in previously untreated patients, and instead will be required to monitor previously untreated patients "through a well-defined and well-managed disease registry."

Guideline on the clinical investigation of recombinant and human plasma-derived factor VIII products

Guideline on core SmPC for human plasma derived and recombinant coagulation factor VIII products


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