OBJECTIVES: Since February 2022, eligibility for the National Institute of Health and Care Excellence (NICE) Highly Specialized Technologies (HST) program has been based on four revised criteria. Criteria 1 and 2 are based on the rarity of the treated condition and are quantitatively defined, whereas Criterion 3 (significantly shortens life/severely impairs quality of life [QoL]) and Criterion 4 (no satisfactory treatments/significant benefit over existing treatments) require judgement. We investigated how NICE judge Criteria 3 and 4 to be met.

METHODS: A targeted literature review was conducted to assess previous research into this topic, finding no published research that analyzed the outcome of applications using the new HST criteria. NICE ‘guidance in development’ and HST committee meeting minutes published from 01/02/2022 to 03/03/2023 (date of search) were systematically reviewed to identify all published HST checklists; these summarize whether a technology meets each criterion and why.

RESULTS: Ten checklists were identified; of these, six and three met Criteria 3 and 4, respectively. A median life expectancy of 66.5 years and a 20-year survival rate of 75% were both sufficient to meet Criterion 3 based on life expectancy and cholestatic pruritus (itching) was sufficient to demonstrate a severe impairment on QoL. Criterion 4 was only met based on lack of treatment options, rather than significant benefit over existing therapies. If alternative treatments exist, evidence of a statistically significant efficacy benefit over standard of care seems to be required (improved convenience/safety is insufficient). Lack of adequate treatments must also apply to a significant proportion of the patient population.

CONCLUSIONS: Criterion 3 can be met with a median life expectancy of 66.5 years or 20-year survival rate of 75%. Criterion 4 is the most difficult to meet, with high thresholds for evidence given the rarity of the conditions involved.