Summary
In the field of haemophilia, gene therapy is an innovation with the potential to bring about a significant improvement in patients’ quality of life. The aim is to examine the organisational impact of introducing this innovation. We are examining the impact on hospital department activities, on the patient’s care pathway, on cooperation between healthcare professionals, and so on, in order to identify the barriers and drivers of the uptake of this innovation.
In France, more than 12,000 people are affected by haemophilia A (HA) and B (HB), rare bleeding disorders, of which at least 2,500 cases are severe. In severe forms, recurrent bleeding leads to major complications (life-threatening bleeding, disabling joint damage, chronic pain and significant psychological and social consequences), making this a major public health issue.
Background and issues
The standard treatment is prophylaxis, based either on FVIII or FIX concentrates, or on mimetic drugs such as emicizumab. Despite these advances, joint protection remains inadequate and costs are very high (€150,000 to €300,000 per patient per year).
Recent advances in gene therapy, particularly through phase 3 clinical trials involving a single injection of an AAV-vectorised transgene, offer significant prospects. However, they raise major organisational challenges relating to their roll-out and the adaptation of care pathways.
Objectives
To analyse the organisational impact of the introduction of gene therapy and identify the barriers and drivers to its uptake, in particular with regard to:
- hospital organisations
- care pathways
- cooperation between healthcare professionals
Methodology
Conducting semi-structured interviews with key stakeholders in the healthcare sector at three university hospitals (Marseille, Brest and Lille): clinicians, dispensing and compounding pharmacists, coordinating registered nurses, a clinical biologist and patients. Interviews were also carried out with experts: the head of the haemophilia reference centre, a pharmacist from the MHEMO network, the DGOS, and innovation advisors from the French Haemophilia Association.
