OBJECTIVES: To investigate the time it takes the National Institute for Health and Care Excellence (NICE) to conduct a Highly Special Technology (HST) appraisal following a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Product for Human Use (CHMP).

METHODS: All products (N=26) with publicly available NICE HST appraisal timelines were reviewed alongside the corresponding EMA marketing authorisation (MA) dates. In the base case scenario (n=19), the appraisal timelines of 6 HSTs were excluded. Reasons included: the draft scope being available only after the CHMP opinion (n=2), the original appraisal timelines no longer being publicly available (n=1) and the HST being a partial/full update, and replacements of other HSTs (n=4)

RESULTS: From the NICE HST programme’s launch in 2013 to June 2023, 26 products have been appraised. The mean duration from NICE's invitation to submit to the Final Evaluation Determination (FED) publication is 16.1 months (median: 12.7; range: 7.7–38.4). In the base case scenario, the mean duration from CHMP opinion to the first Committee Meeting (CM) is 8.3 months (6.4; 0.9–26.9), and the mean duration from CHMP opinion to FED publication is 17.2 months (14.3; 7.9–31.7). For 88% of the appraisals (n=22/26), ≥2 CM were required; 32% (n=8) required ≥3 CMs.

CONCLUSIONS: This analysis reveals that the mean delay from CHMP opinion to the first CM is 8.3 months and 17.2 months until FED publication. Overall, HST appraisals average 16.1 months, significantly longer than NICE's target of 5.6 months. Delays stem from factors inherent to ultra-orphan disease treatments, including sparse data, development of de novo health economic models, expected high costs of medicines, and MA and appraisal scheduling delays. By increasing transparency and taking an evidence-based and pragmatic approach to the process, industry and NICE could do more to tackle these issues.