Kreuth V initiative: European consensus proposals for treatment of haemophilia using standard products, extended half-life coagulation factor concentrates and non-replacement therapies

This report contains the updated consensus recommendations for optimal haemophilia care produced in 2019 by three Working Groups (WG) on behalf of European Directorate for Quality of Medicines & Healthcare in the frame of the Kreuth V Initiative. WG1 recommended the access to prophylaxis for all patients, the attainment of plasma factor trough levels of at least 3-5% when extended half-life FVIII and FIX products are used, treatment regimen personalisation and choice of chromogenic assays for treatment monitoring. It was also emphasized that innovative therapies should be supervised by Haemophilia Comprehensive Care Centres. WG2 recommended mandatory postmarketing data collection to assure the long-term safety and efficacy of new haemophilia therapies, the establishment with adequate support under public control of national patient registries including the core data recommended by EMA and ISTH, and more collaboration to facilitate comprehensive data evaluation in Europe. WG3 discussed methodological aspects of haemophilia care in the context of access decisions particularly for innovative therapies, and recommended that clinical studies should be designed to provide the best possible evidence needed by regulatory authorities, HTA bodies and healthcare providers. The dialogue between all stakeholders in haemophilia care and patient organizations should be fostered to implement these recommendations