Innovative cell and gene therapies are often single-administration treatments with high-upfront costs but the potential for long-term health gains and possibly cure. Given the uncertainty in outcomes at launch and the unrecoverable high costs, the US Institute for Clinical and Economic Review (ICER) recently developed novel methods for assessing these treatments.

We reviewed these methods and recent health technology assessments (HTAs) by the UK National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) of tisagenlecleucel and voretigene neparvovec, to examine whether ICER’s new methods are already considered in HTAs in the UK.

The adapted ICER methods require cure-fraction modeling, discounting of outcomes and costs at the standard 3%, and a threshold analysis of the duration of the beneficial effect. A cost-effectiveness threshold of $150,000 per quality-adjusted life-year (QALY) gained is used to guide evaluation of long-term value for money. ICER will also consider benefits beyond QALYs, including the value of treatment choice and potential benefit or disadvantage of option value. In the HTAs of tisagenlecleucel and voretigene neparvovec, NICE considered cure-fraction models and reduction of the discount rate to 1.5%, and acknowledged value beyond health benefits, such as patients retaining independence and research advancing the broader field of gene therapy. SMC accepted additional value in the single administration, and reduced patient and carer burden. Both NICE and SMC applied higher cost-effectiveness thresholds for ultra-rare diseases.

While higher cost-effectiveness thresholds and cure-fraction models are already considered in HTAs for costly, single, or short-term therapies in the UK, ICER’s consideration of the value of treatment choice and option value is a novel approach. ICER plans to implement the adapted methods in January 2020. A review of future ICER assessments will indicate whether the new methodology provides a better way to demonstrate the value of potentially curative treatments.