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 significant 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. Hematological conditions are a common target for gene therapies; however the perceptions of those both directly and indirectly impacted by funding decisions have not been investigated. To support HTA of gene therapies, this study explored the perspectives of individuals living with hematological conditions who may become eligible for such treatments, alongside their carers and members of the general population. 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 (GTs); the potential benefits or harms of GTs; any perceived barriers to GT access; and/or uncertainties associated with the respective GT. Here, we compare and contrast health consumer and general population responses, providing valuable insights for HTA decision-making on gene therapy. Additionally, we elucidate barriers to access and associated uncertainties that will require resolution if acceptability and uptake of GT are to be optimised in future.