OBJECTIVES: Cell and gene therapies have characteristics that pose additional challenges for HTA. The objectives of this research were to assess how HTA bodies are responding to these challenges and to identify where further initiatives are required.

METHODS: HTA reports and supporting documents were analysed for 9 cell and gene therapies in 10 indications from the US (ICER), Canada (CADTH), England (NICE), Scotland (SMC), France (HAS) and Italy (AIFA). Data were extracted using a recently published checklist for assessing gene therapies that specifies 19 key characteristics.¹

RESULTS: In total 31 HTA reports were analysed. The key clinical characteristics of cell and gene therapies were widely considered in the reports: surrogate endpoint (84% of reports), rare disease (71%), serious condition (94%), single arm trial (100%), pediatric population (100%), adverse consequences (100%), size of clinical trial (100%), length of clinical trial (97%), extrapolation to long-term outcomes (65%). The consideration of key economic characteristics and other key aspects of the economic evaluation was more variable: serious disease (94%), value to caregivers (45%), insurance value (0%), scientific spillovers (10%), lack of alternatives (71%), substantial improvement in life expectancy (65%), discounting (74%), different discount rates (55%), uncertainty (100%), alternative payment models (29%).

CONCLUSIONS: HTA bodies are considering many of the key characteristics of cell and gene therapies, although this response is variable. The main areas where further initiatives are required include the validation of surrogate endpoints, the criteria for accepting single arm clinical studies, the assessment of family and scientific spillovers, estimating insurance value, exploring scenarios using different discount rates and the use of alternative payment models to reduce uncertainty.