Exploring patient and clinician perspectives on the benefits and risks of emerging therapies for the treatment of haemophilia: a qualitative study

Abstract

Abstract Background Enhanced horizon scanning for emerging treatments has identified that both haemophilia A and haemophilia B pathways will be enriched with a range of new medicines with varying benefit-risk profiles. Patient and clinician views on the balance of the benefits and risks associated with emerging therapies will affect their use for the treatment of haemophilia, while also introducing the need for effective communication strategies to enable informed patient-clinician decision-making. Aim This study aimed to explore patient and clinician views on the perceived benefits and risks of emerging therapies for the treatment of haemophilia. The study also aimed to gain insight into clinician-patient communication on benefit and risk and how this shapes decision-making on new therapeutic options. Methods Qualitative methods, using online focus groups and one-to-one interviews, were guided by a defined set of questions. Data were collected in 2022 and 2023 from a sample of adult patients identified through the national patient group (The Haemophilia Society) and clinicians identified through the NHS England Clinical Reference Group (CRG) and HAEM-NET. Data were analysed thematically. Findings Seven patients and ten clinicians (seven consultant haematologists; three advanced nurse practitioners) participated. Five summary themes were identified, three of which related to clinician-patient communication: (i) active vs. passive patients; (ii) health literacy; (iii) external factors. One theme related to gene therapy perspectives on benefit-risk, and one theme concerned the legacy of the infected blood scandal. Conclusion There remains scepticism about gene therapy across all research participant groups, which suggests that uptake is likely to be relatively slow, with divergence anticipated between haemophilia A and B. Treatment decision-making and benefit-risk discussions are complex and multifaceted issues which in haemophilia are heavily influenced by the infected blood scandal. Clinicians frame treatment decision-making which necessitates the requirement for benefit-risk training and high-quality tailored patient gene therapy information materials.