Haemophilia Gene Therapy: Patient Selection and Experience in the Era of Precision Medicine

Abstract

The emergence of gene therapy as a therapeutic modality for haemophilia has generated a spectrum of responses within the patient population, ranging from enthusiastic endorsement to measured scepticism. Following the European Medicines Agency's approval of two novel gene therapies for haemophilia B, the imperative for sophisticated patient selection protocols has become increasingly apparent to ensure both the safe administration of therapy and optimisation of clinical outcomes. Haemophilia gene therapy trials to date have demonstrated that psychosocial factors are critical components of the gene therapy pathway alongside clinical eligibility to facilitate rigorous follow-up protocols and maximize the probability of sustained transgene expression. Experiences of participants in haemophilia gene therapy clinical trials are valuable for informing prospective recipients during pre-consent counselling to appropriately calibrate treatment expectations and thoroughly comprehend the multifaceted risk-benefit profile, including the inherent uncertainties associated with long-term outcomes.