Presentation (CTI)

OBJECTIVES: This study evaluates how NICE’s revised Highly Specialised Technologies (HST) routing criteria, effective from April 2025, would apply retrospectively to both Single Technology Appraisals (STAs) for rare diseases that may be considered borderline for HST consideration and completed HST evaluations to date. It aims to assess alignment with the updated framework and examine the consistency of NICE’s interpretation of key eligibility concepts such as “exceptional burden” and “substantial additional benefit” for the appraisals conducted between 2020-2025.­­­
METHODS: A retrospective review was conducted on two cohorts: 1) completed HST appraisals between 2020-2025 and on 2) STAs for rare and ultra-rare diseases flagged through consultation feedback or stakeholder commentary as potential HST candidates during the same period. Each appraisal was reassessed against the revised criteria using the HST routing checklists, ERG reports, and final guidance. Thematic analysis was applied to evaluate how the updated criteria and clinical perspectives from the committee influenced routing and decision-making for these appraisals.
RESULTS: Findings may suggest that several STA technologies, particularly for ultra-rare neurological and metabolic disorders, would likely meet the revised HST criteria today. Conversely, some past HSTs may not meet the clarified thresholds for “substantial additional benefit” or “exceptional burden” under the revised criteria. This has a substantial bearing on whether the technology is recommended or not, given the vastly different willingness-to-pay thresholds for each route.
CONCLUSIONS: While the revised HST criteria aim to bring greater clarity, consistency, and fairness to the appraisal process for ultra-rare conditions, this retrospective review may reveal that their interpretation and application have remained variable. The findings underscore the need for clear operational guidance, especially where criteria are still subjective.