Are the Hurdles Too High for Access to Gene Therapies in the UK?

OBJECTIVES: Gene therapies have the potential to deliver significant benefit to patients as well as to the NHS in the UK. However, access and reimbursement for gene therapies is inherently challenging. Given their one-off nature and high research & development and manufacturing costs, gene therapies are associated with a complex evidence base, low patient numbers, uncertainty around long-term outcomes (in some cases multiple decades), variability in patient outcomes and issues around affordability and budget impact. In this work we aimed to explore whether access to gene therapies in a cost-effectiveness market like the UK is feasible via the standard health technology assessment (HTA) routes.

METHODS: We undertook a review of the UK reimbursement decisions for gene therapies over the last 5 years (2008-2023). In addition, we performed a review of NHS England ‘smart deals’ since 2018. We sought to identify hurdles in reimbursement.

RESULTS: There are currently five gene therapies reimbursed in the UK, all of which have benefited from the flexibilities of the NICE highly specialized technology (HST) process and the SMC ultra-orphan process. A sixth gene therapy was assessed from NICE through the STA route but was not recommended. Our review of NHS England ‘smart deals’ revealed most of these were interim funding arrangements, population health arrangements and portfolio deals - and not innovative payment mechanisms linked to outcomes - with much of the detail not in the public domain due to confidentiality agreements.

CONCLUSIONS: All gene therapies recommended within the UK required specialized ultra-orphan routes for HTA in combination with tailored commercial agreements. In the absence of a gene therapy specific HTA route, assessing via NICE HST and SMC ultra-orphan processes are paramount to bringing these innovations to the UK population.