OBJECTIVES: NICE’s highly specialised technology (HST) evaluations are highly restrictive in terms of entry criteria. Consequently, rare disease medicines are often assessed through NICE’s standard, single technology appraisal (STA) route. This study analysed publicly available HST checklists to assess the extent to which checklist criteria were met, identify if any themes emerged and how HST/STA routing decisions were impacted.

METHODS: Available HST checklists, downloaded from the NICE website, were reviewed quantitatively, and assessed against final routing outcomes/status. Researchers evaluated the findings to interpret whether patterns exist in relation to decision-making against the four HST criteria.

RESULTS: As of February 2023, 9 HST checklists were available on the NICE website. Of these 9 appraisals, only 2 were deemed eligible for HST; 5 proceeded as STA and 2 were suspended (one due to manufacturer decision following STA-routing decision; one due to EMA-license issues). Checklist evaluation showed that the first 3 HST criteria were ‘met’ at least 5/9 (56%) times. However, the last criterion (regarding benefit over existing treatment options) was ‘not met’ 6/9 times (67%). Criteria stratification, based on HST/STA outcome, showed that HST appraisals met all 4 criteria. However, for the seven STA-routed appraisals, criteria decisions were deemed ‘met’ or (at the other extreme) ‘unmet’ in equal measure (n = 11 times), with only 2 ‘partially met’ or 4 ‘unclear’ decisions made.

CONCLUSIONS: This evaluation suggests that rare disease products can only achieve HST routing when all checklist criteria are met. Interestingly, the last criterion (benefit over existing treatment options) was mostly unmet, highlighting product innovation within a sparce competitor landscape as the biggest determination for HST routing success or failure. Criteria assessment decisions are polar extreme (with highest frequency of decisions either deemed ‘met’ or ‘unmet’), suggesting firm decision-making by TSOP against the checklist criteria.