Patient Journey Mapping of the Gene Therapy in Hemophilia B in Spain: The Bhemogen Project

OBJECTIVES: Develop a patient journey for the first gene therapy in haemophilia B in Spain from a multidisciplinary point of view. Generate knowledge on practical and contextual aspects that affect the introduction and practical management of gene therapy by identifying challenges and proposing solutions.

METHODS: Literature review of national sources complemented by grey literature. This information was complemented and validated through interviews to 10 Spanish stakeholders related to haemophilia B, advanced therapies, and healthcare management (haematologists, pharmacists, specialized nurses, specialists in advanced therapies, hospital managers and a patient representative). Validation of results through online group discussions.

RESULTS: The proposed pathway is based on the current care process for haemophilia B patients, incorporating the changes needed for the gene therapy introduction: 1) New relevant roles for the evaluation of patients' eligibility for this treatment option; 2) Need to train healthcare professionals in gene therapy; 3) Development of a specific patient informed consent process; 4) Potential adaptation of hospital areas for safe administration of the therapy and 5) Inclusion of patients in international registries for long-term follow-up. In addition, contextual details related to the structure and functioning of the Spanish National Health System were identified: need to review and adapt reimbursement and acquisition procedures, address cost compensation logistics, and utilise existing patient registries for long term outcomes monitoring.

CONCLUSIONS: This represents the first patient journey of gene therapy for haemophilia B patient in Spain. Its implementation could contribute to guide the effective incorporation of this therapy within the healthcare system and deliver the expected health outcomes. The pathway sets a precedent for future gene therapies, contributing to enhance the processes for the inclusion of this disruptive innovation into the healthcare system.