Shared decision-making for gene therapy in haemophilia care

Abstract

Shared decision-making (SDM) is an important part of patient-centred care in which healthcare professionals (HCPs) and patients/caregivers jointly reach care decisions through a two-way exchange and synthesis of information based on clinical evidence and patient preference. SDM was described in haemophilia care in 2014 as two-sided intervention to aid patient decision-making. However, as the range of haemophilia treatments has expanded, identifying the optimal haemophilia treatment for an individual has become more complex. This is particularly so in the case of gene therapy, a onetime-only, irreversible treatment. In this context, it is vital that people with haemophilia (PwH) and their families continue to be involved in care decisions in an informed and interactive way. For gene therapy, this must include being well informed about the gene therapy process, enabling the patient to engage in fully informed SDM and consent, and ensuring that issues around long-term durability, potential side effects, the need for long-term follow-up are understood with a recognition that the ‘unknown unknowns’ are also unknown to HCPs. Both HCPs and patient organisations have a key role to play in providing PwH with access to relevant information and education, tailored to individual needs and free of jargon. Considerable education and support are required before PwH can make a truly informed decision about having gene therapy. Use of structured SDM tools such as the SHARE approach can help to support this. There is a need for SDM educational tools that include written/visual information and the use of standardised checklists may be helpful for both PwH and HCPs. The most important part of this process is that PwH want to undergo gene therapy – and this is only an option if they are fully educated and informed by fully educated and informed healthcare teams.

Keywords

• shared decision-making
• haemophilia
• gene therapy
• patient education
• informed consent