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Ema: patient records and real world data for clinical studies on hemophilia

January 10, 2019

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A new step on haemophilia clinical studies arrived from the European Medicines Agency. In fact, the EMA guideline dedicated to clinical studies on haemophilia has been revised, in particular for recombinant products based on factor IX derived from human plasma. The new guideline synergically complements the provisions contained in the guideline dedicated to coagulation factor VIII deficiencies (July 2018).

The new guideline on factor IX refers to:

  • Products for which a new application for marketing authorization is submitted
  • Products already authorized that are subject to significant changes in the production process (i.e. introduction of additional stages of inactivation / virus removal or new purification procedures).

Real world data for patients with new treatment

Patients never treated before are subjects to novelties. The data for the clinical studies, both for the factor VIII and for the factor IX, should be obtained from the registers of patients rather than small-scale clinical trials targeting only these sub-populations.

The more traditional approach, in fact, according to Ema could be not fully representative of the real use of the drug, once on the market, as these are very small groups of highly selected patients. The new haemophilia drug development guidelines have fully embraced the new approach to pharmaceutical development based on the use of high quality real world evidence, the goal of which is facilitated by the suggestion of a set of key data that should always be collected within patient records. The new consolidated approach in the new guidelines started in 2015, with a first workshop on the topic organized by Ema, followed by a public consultation in 2017 and a second workshop on hemophilia registers in June 2018.

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