Presentation (CTI)

OBJECTIVES: In health technology assessment (HTA), NICE generally focuses on direct costs and health effects for the scoped population, which risks undervaluing wider NHS patient population treatment effects. We examined precedent for NICE considering factors beyond the reference case, and the value of accounting for these broader effects.
METHODS: We undertook a targeted review and methodological analysis of appraisals where the committee extended consideration beyond the reference case to align with NICE’s stated principles. We then drew on the health economics literature to identify other effects that fit those principles but fall outside the reference case, along with methods to quantify them.
RESULTS: In two appraisals of gene therapy for inherited blood disorders, the committee concluded that the eligible population experiences health inequalities that the therapy could mitigate. Although equity is routinely considered qualitatively, here the committee accepted a quantitative supplementary analysis, citing NICE's principle of reducing health inequalities. This set an example: when justified by NICE principles, the committee may consider value elements outside the reference case through supplementary analyses to inform judgment. The health economics literature describes capacity and displacement effects, whereby a treatment frees up scarce NHS capacity that could benefit other patients. Transplant waiting lists are one example: modelling the list as a queue shows measurable effects on others in the healthcare system through altered wait times and, in turn, survival, quality of life, and resource use.
CONCLUSIONS: These broader effects are not explicitly excluded by the reference case but typically fall outside the appraisal-defined population scope. Considering such effects aligns with NICE's principle seven of basing recommendations on population benefits and value for money. NICE should consider quantified benefits beyond the immediate patient population, to ensure continued alignment with its principle of providing full population benefits and value for money.