OBJECTIVES: NICE and SMC differentially assess medicines for rare and ultra-rare diseases. The SMC ultra-orphan and NICE’s HST programs were introduced 2018 and 2013, respectively. Our objective was to compare the process and outcomes for appraisals undertaken via the SMC ultra-orphan pathway (UOP) against their corresponding NICE appraisals.

METHODS: Submissions to SMC via the UOP between 01/10/2018 and 31/05/2023 were analyzed, alongside their reciprocal NICE appraisals. For each technology, relevant dates (marketing authorization, submission, decision), indications and outcomes (decisions, number of consultations, managed access) were extracted from the appraisal and EMA website.

RESULTS: Overall, 19 medicines (20 indications) had been granted interim access in Scotland via UOP. Of these, 11 (11 indications) were assessed via NICE HST, and 8 (9 indications) via STA. Among the 20 indications accessible in Scotland, 17 were recommended by NICE (5 subject to a managed access agreement [MAA]). Advice on 3 was under development.

The overall average submission-to-decision time (SDT) was 2.4 (SMC) and 10.6 (NICE) months. Among the 9 HSTs with a recommendation, the average SDT was 2.5 (SMC) and 13.59 (NICE) months. The average SDT for the 5 technologies requiring MAA was 2.69 (SMC) and 10.42 (NICE) months.

CONCLUSIONS: Assessment of ultra-rare diseases by SMC and NICE are divergent. Only 55% of medicines assessed through the UOP were considered eligible for HST. Although further data collection and reassessment is key to the UOP, only 25% were subject to a MAA by NICE, and this did not meaningfully improve the SDT.

Whilst NICE and SMC tended to make similar decisions, the time-to-decision was approximately 4x longer with NICE than SMC. Therefore, the different approaches have the potential for significant inequity of access to rare disease medicines. However, as manufacturers generally submitted to NICE ahead of SMC, faster SMC time-to-decision does not currently equate to earlier access in Scotland.