The European Haemophilia Consortium’s (EHC) periodic review of novel treatments in haemophilia and other bleeding disorders is a review entirely developed by the EHC to help educate EHC National Member Organisations (NMOs) about the rapidly changing landscape of medicinal treatment in rare bleeding disorders. The EHC encourages its NMOs to use and adapt this document to their national needs but takes no responsibility for any changes.

The review covers recent major developments and is divided by different types of disorders for which there is an update to report. The information provided in this publication is compiled from multiple sources, including presentations at recent scientific meetings, websites, financial information and writing directly to pharmaceutical companies. It is then redrafted and presented in easy-to-understand language. This review is updated periodically.

The EHC is grateful for the work and support of the Novel Treatment Review Committee, which has overseen the content and production of this document. The EHC also wishes to give Miguel Crato, EHC President, special thanks for his work on these publications.

The EHC greatly welcomes all treatment developments that may benefit patients in the future. The EHC takes no position on any product type or class reported in this review.

We hope that the information provided herein is useful and we are available for any questions.