Evolution of haemophilia integrated care in the era of gene therapy: Treatment centre's readiness in United States and EU.

Comprehensive, integrated care provided by a multi-disciplinary team of experts improves outcomes and is widely recommended for people with haemophilia (PWH).1-6 Although care models vary, the principles of management are consistent between countries. The likely approval of gene therapy for PWH in the next few years has the potential to alter the course of disease and drive an evolution in haemophilia care.7, 8 A group of haemophilia experts including physicians, a haemophilia nurse, a physiotherapist and PWH met in September 2020 to discuss strategies for safely introducing gene therapy into clinical practice and to identify its potential long-term effects on haemophilia care models in the USA and Europe. Experts agreed on four universal principles for the introduction of gene therapy (Table 1) and furthermore identified a range of topics that need to be discussed and resolved locally in order to implement these principles. Going forward, the national/regional haemophilia networks should lead the debate on the implications of gene therapy for haemophilia treatment centres (HTCs) and develop workable solutions to the challenges and opportunities that gene therapy presents. [...