For the last thirty years, haemophilia A and B patients have been treated using FVIII and FIX replacement concentrates. These therapies are well known to both clinicians and patients. We are now entering a new era in the treatment of haemophilia and rare bleeding disorders. Novel, non-replacement therapies have been approved and are becoming available, which may have a significant impact on the clinical care and quality of life of patients.
The European Medicines Agency (EMA) launched in October 2023 a public consultation on their draft guideline on the clinical requirements for non-replacement therapy in haemophilia A and B. The end of the consultation and deadline for comments was on 30 April 2024.
The European Association for Haemophilia and Allied Disorders (EAHAD), the European Hematology Association (EHA), and the European Haemophilia Consortium (EHC) support EMA’s efforts in drafting the guideline which aims to provide both applicants and regulators with much-needed harmonised marketing authorisation requirements for applications of non-replacement therapies for haemophilia A and B.