Patient, consumer and societal values, perceptions and preferences on high-cost gene therapies

Novel cell and gene therapies hold great potential to alter the clinical course of a range of chronic diseases, yet uncertainties remain regarding their long-term efficacy and impact. These uncertainties pose challenges for Health Technology Assessment (HTA) committees, which systematically assess health interventions seeking public subsidy. Haematological conditions are a common target for gene therapies; however, the perspectives of those both directly and indirectly impacted by funding decisions for therapies in this disease area have not been investigated. To support future HTA considerations of gene therapies, we explored the perspectives of individuals living with haematological conditions who may become eligible for such treatments, alongside their caregivers and members of the general population in Australia. We undertook interviews and focus groups with 55 participants with lived experience and 32 general population members to elicit responses on what might influence decision making on gene therapies; the potential benefits or harms of gene therapies; any perceived barriers to gene therapy access; and/or uncertainties associated with the respective gene therapy. Here, we describe the parallel perspectives of health consumers and the general population, providing five key areas of consideration for HTA decision-making: individual patient and disease factors; geographical, financial and clinical access; balancing disease and quality of life benefits with adverse effects; individual and health system costs; and remaining treatment uncertainties. These will require resolution before widespread acceptability and uptake of gene therapy.