Presentation (CTI)

OBJECTIVES: In March 2025, NICE adopted new HST routing criteria for evaluating technologies for ultra-rare diseases, aiming to make the HST programme more consistent and transparent. However, concerns have been raised by industry about the strictness of the new criteria, particularly about the reduced threshold of 300 patients, highlighting possible challenges manufacturers may encounter when qualifying for the HST programme. The purpose of this study is to: 1) analyse whether technologies previously qualifying for the HST programme would still have qualified with the new March 2025 criteria, and 2) analyse whether some products that previously failed to qualify for HST would now have qualified under the new criteria, to explore whether these new criteria can limit access to new rare-disease therapies for patients.
METHODS: All HST appraisals (n = 30) available through the NICE website, as well as failed HST checklists available online, were analysed against the new HST routing criteria. Materials used for this analysis include NICE publications and other scientific publications in the public domain.
RESULTS: Overall, 5 (17%) of the published HST appraisals would no longer have qualified under the updated criteria: 3 due to exceeding the newly established prevalence threshold of 300 patients and 2 due to being an indication expansion. From available data, no previous products that failed to qualify for the HST programme would have now qualified under the new criteria.
CONCLUSIONS: The new HST routing criteria introduce a strict threshold of 300 eligible patients and prevent indication expansions from qualifying for the HST programme. With five previous HSTs not passing these criteria, and no previously failed products qualifying under the new criteria, our analysis suggests that rare-disease assets will be less likely to qualify for HST in future. Securing HST status is crucial to guarantee access for products that otherwise would fail a standard technology assessment.