Questionnaire for HCPs regarding Haemophilia Gene Therapy: Knowledge, Information Sources, and Treatment Considerations

As innovative gene therapy treatments like Roctavian® (approved in Europe in August 2022 and in the US in June 2023) and HEMGENIX® (approved in the US in November 2022 and in Europe in February 2023) become more accessible in clinical practice across Europe, we seek to understand the perspectives of all stakeholders involved in haemophilia care.

Importantly, this survey aims to investigate how perceptions of gene therapy have evolved over the past 1-3 years, following the approval and introduction of these treatments.

The survey covers various aspects, including demographic information, knowledge assessment, experience with gene therapy in clinical trials, information sources, treatment considerations, and educational need and explores topics like understanding of AAV gene therapy mechanisms, awareness of approved therapies, factors influencing treatment decisions, and preferred information sources.

 Recent research highlights significant knowledge and practice gaps in the field that affect both healthcare providers and patients. An international survey revealed that 35% of physicians involved in haemophilia care reported difficulty explaining adeno-associated viral (AAV) gene therapy, while 40% expressed limited comfort answering patient questions about clinical trial results. Though 75% of healthcare providers answered knowledge questions correctly, 13% provided incorrect answers and 12% were uncertain of proper responses.

Patient awareness also shows considerable limitations. Among French patients surveyed, 39.5% estimated their knowledge of gene therapy to be limited, and more than 60% reported never or rarely discussing gene therapy with their haemophilia care team. Notably, 53.7% expressed desire for better information from their healthcare providers. Similarly, studies with UK parents of children with haemophilia indicated insufficient knowledge for informed decision-making.

This survey builds upon these findings by specifically addressing the European context and incorporating questions about the impact of recent gene therapy approvals on perceptions and decision-making. We aim to provide a comprehensive view of the current knowledge of gene therapy, identify areas for improvement in education and communication, and understand how perceptions have shifted since the approval of gene therapies.

The findings from this study will directly impact clinical practice by identifying specific knowledge gaps, informing targeted educational initiatives, improving communication between HCPs and PwH, and enhancing shared decision-making processes. By addressing these requirements, we can ensure both HCPs and PwH are adequately prepared to navigate treatment options in this new era of haemophilia care, ultimately improving patient outcomes and quality of life.