Presentation (CTI)

OBJECTIVES: Introduced by NICE in 2013, the Highly Specialised Technologies (HST) route appraises treatments for ultra-rare diseases, where evidence generation is constrained by small patient populations. In April 2025, the NICE board approved revisions to the HST routing criteria intended to improve consistency and transparency in their application. This research identifies the factors driving routing decisions between HST and the single technology appraisal (STA) pathway, and offers practical recommendations for manufacturers of orphan medicines seeking HST consideration.
METHODS: A targeted search of the NICE website was conducted in May 2026 to identify HST topic selection decisions issued by the NICE Prioritisation Board against the four HST qualifying criteria since April 2025. Decisions and accompanying rationales were manually extracted from published HST checklists.
RESULTS: Nine appraisals have been considered using the refined HST criteria, only one of which was routed to HST. Across the nine appraisals, three (33.3%) satisfied the criterion that the disease be ultra rare and debilitating, six (66.7%) satisfied the criterion that the technology should be an innovation for the ultra-rare disease, four (44.4%) satisfied the criterion that the technology be limited to the population in its licensed indication, and only one (11.1%) satisfied the criterion that there should be no existing effective treatment options for the same ultra-rare disease. Disagreements between NICE and manufacturers centred on point prevalence estimates for the disease and treatable population, and the adequacy of existing treatment options. Further divergence was noted regarding the extent of disease burden on patients and whether a technology could be considered to provide substantial additional benefit to patients/carers.
CONCLUSIONS: Despite efforts from NICE to make HST routing more predictable, meaningful differences in interpretation persist between NICE and manufacturers. Early alignment on prevalence estimates, the comparator treatment landscape, and the framing of disease burden will be critical for manufacturers pursuing HST routing.