A key requirement is education about gene therapy for haemophilia, for PWH and their families, and for the multidisciplinary team (MDT) of HCPs. Patient education by HCPs has been predominantly verbal or within clinical trials thus far. Materials to help guide decisions on gene therapy have been produced by patient organisations such as the European Haemophilia Consortium (EHC) [9]. However, lack of understanding of available resources and their reliability may deter PWH from seeking gene therapy education. Guidance for PWH may be valuable, such as a centrally compiled list of easily accessible educational materials. National patient advocacy groups will be crucial in delivering patient education.

PWH usually seek information from HCPs at their current treatment centre. Materials for all MDT members will be necessary to ensure sufficient understanding to explain this complex topic to PWH, or signpost resources, the goal of which is to allow true shared decision-making. Materials exist that could facilitate HCP education, produced by groups such as those listed in Fig. 1A [10,11,12,13,14,15,16]. The World Federation of Hemophilia Shared Decision Making Tool is one such readily available resource [14]. Furthermore, the European Association for Haemophilia and Allied Disorders established a working group in 2019 to provide information to PWH and HCPs on practical and safety-related aspects of gene therapy [17]. Although special interest groups share such materials with members, widespread knowledge of resources is limited. An inventory of appropriate available resources could provide initial direction to HCPs.

Unmet needs throughout the patient journey, potential next steps, and educational requirements for PWH. A Current unmet needs throughout the gene therapy for haemophilia journey and potential starting points regarding education, psychological support, and guidance. Time periods are indicated by a light grey background; unmet needs are applicable to all time periods across which they span. B Summary of key educational topics required for PWH. Education for PWH on these topics should begin as soon as possible pre-infusion, ideally in childhood

HCP education should include a diverse set of relevant topics. Some of the essential topics requiring education, however, may not be apparent until gene therapy implementation, as topics and extent of education may depend on currently unknown factors, e.g. the responsibilities of different centres and long-term monitoring requirements. National specialist support platforms could help identify and meet specialists’ educational needs. The UK Haemophilia Centre Doctors’ Organisation (UKHCDO) and The Haemophilia Society could be instrumental in achieving this.

Consistent messaging across educational materials for PWH, their families, and HCPs is lacking but it will be important to manage expectations and avoid confusing misinformation. As echoed in the 2022 EHC conference, this could be achieved through collaborative working groups with input from academia, clinicians, PWH and their families, and industry. For example, in the gene therapy for spinal muscular atrophy (SMA) setting, the SMA UK network has been valuable in delivering education and facilitating consistency of resources. The Advanced Therapy Treatment Centres network [13] and Specialist Pharmacy Service (SPS) [18] resources have been achieved through such working groups. Due to the dynamic nature of the field, it will be important for educational materials to be continuously updated with further advances, as is the case with SPS resources.