BACKGROUND: NICE updated its methods and processes guide in February 2022 with the aim of enabling earlier access to new, innovative medicines. Prior to the methods review, rare disease technologies routed via the STA pathway faced barriers to timely access.

OBJECTIVES: This study explored the impact of the NICE methods and processes update on access timelines for rare disease STAs.

METHODS: Non-oncology STAs published from January 1^st 2023 onwards were extracted from the NICE website. STAs were differentiated by rarity (prevalence of <1/2,000 people), appraisal committee (A, B, C or D) and year of final appraisal committee (ACM) meeting. Six rare disease STAs were randomly selected, and each was matched to a randomly selected non-rare disease STA according to NICE committee and year of final ACM (if applicable). For each appraisal, time to access, time in NICE, and number of ACMs were analysed. Results were compared with a similar analysis done by Hale et al. (2023) preceding the NICE methods and processes update.

RESULTS: Despite updates to the methods and processes manual, rare disease STAs are still subjected to a greater mean time to access (514 days; range: 46 to 1,265 days) versus non-rare disease STAs (280 days; range: 54 to 406 days). The mean time to access for rare disease STAs remained similar to timelines observed prior to the introduction of the methods and processes update (504 days), while the average number of ACMs also remained constant (2 ACMs). The time in NICE for rare disease STAs proceeding the methods and processes update was, on average, 178 days shorter compared to the sample assessed by Hale et al. (2023) prior to its introduction.

CONCLUSIONS: Despite updates to the NICE methods and processes manual, rare disease technologies are still subjected to similar delays in access timelines.