Presentation (CTI)

OBJECTIVES: This analysis aimed to retrospectively evaluate whether selected drugs previously appraised by NICE would have met the updated 2025 eligibility criteria for the Highly Specialised Technologies (HST) programme, which targets ultra-rare, debilitating conditions.
METHODS: A sample of 30 drugs appraised by NICE between 2015 and 2024 was selected based on their indication for rare or ultra-rare diseases and use of the previous HST route. Each drug was assessed against the four refined HST routing criteria implemented in April 2025. Data sources included NICE appraisal documents and published literature. The drugs were deemed to have hypothetically eligible for the new HST route only if they met all four criteria.
RESULTS: In total, 4 HST appraisals were excluded from analysis due to being superseded by more recent appraisals. The most common HST disease area was endocrinal, nutritional, and metabolic conditions. Of the 25 drugs reviewed, 21 (90%) met all four of the new HST criteria. 2 drugs were deemed to definitely not meet the new criteria, and the outcome for the remaining 3 was uncertain. The most common exclusion reason was failure to meet the ultra-rare prevalence threshold (Criterion 1A). Notably, 3 drugs that were previously rejected under the TA pathway might have had a more favourable outcome under HST routing.
CONCLUSIONS: Overall, it seems as though the new HST routing criteria will lead to mostly the same outcomes for drugs for ultra-rare diseases. However, the extent to which this conclusion holds will depend on how NICE interprets its own guidelines, the wording of which is often subjective (leading to the 3 uncertain results in our analysis).