BACKGROUND: NICE introduced the highly specialised technology (HST) programme in 2015 to consider drugs for ultra-rare diseases. Discrete Willingness to Pay (WTP) thresholds were introduced in April 2017, starting at £100,000/QALY up to a maximum of £300,000/QALY, on a sliding scale for undiscounted QALY gains above 10.

OBJECTIVES: This study examined whether introduction of discrete WTP thresholds has impacted the publication of positive HST guidance.

METHODS: HSTs that were in development or had published guidance were identified on the NICE website and divided into those with submission dates before and after the introduction of WTP thresholds. HSTs that had not been through at least one committee meeting by December 2019 were excluded. Time from first committee to posting of final evaluation determination (FED) or March 1^st, 2020, whichever was earlier, was recorded as well as whether the FED was positive.

RESULTS: 18 HSTs were included. 7 HSTs were pre-threshold, of which 6 (86%) had published guidance, all positive, and one had a negative FED but no guidance. 11 HSTs were post-threshold, of which 6 (55%) had published guidance, all positive. Of the 5 without published guidance, 2 had received FEDs (both negative) and 3 still had no FED. For those HSTs with a FED, average time from first committee to FED was 11 months pre-threshold vs. 9 months post-threshold. For the 3 HSTs still awaiting a FED, 15 months (3 – 22 months) had elapsed.

CONCLUSION: Compared with pre-threshold HSTs, a smaller proportion of post-threshold HSTs appear to be reaching FED within the same timeframe. Furthermore, in contrast with NICE single technology appraisals (STAs), only positive FEDs appear to be converted into HST guidance. With the caveat of small numbers, publication of NICE HST guidance appears to be reduced and positive guidance more challenging to achieve in the post-threshold era.