OBJECTIVES: Highly specialised technology (HST) evaluations in England are recommendations by the National Institute for Health and Care Excellence (NICE) on the use of new and existing highly specialised medicines for very rare conditions within the National Health Service (NHS). In Scotland, treatments considered eligible within the ultra-orphan pathway after initial assessment are available on the NHS; and the Scottish Medicines Consortium (SMC) will reassess the medicine and decide on routine availability after 3 years following full assessment. We aimed to compare the SMC decisions for the treatments identified for NICE HST evaluation.

METHODS: All published HST guidance were identified from the NICE guidance database. Corresponding treatments were identified in the SMC medicines advice with a comparison of assessment status and decisions.

RESULTS: In England, 14 HST published reports by NICE were identified. Corresponding searches for Scotland of these treatments found that: 3 had not been reviewed, 4 were ultra-orphan initial assessments (considered eligible and therefore available), and 7 were full assessments. Of the 7 full assessments by the SMC, 3 (43%) were approved, 1 (14%) was approved with restrictions, and 3 (43%) were not recommended. The main reason for non-recommendation by the SMC was a lack of justification of the treatment cost in relation to the health benefits. The economic analyses were also generally considered not sufficiently robust to gain acceptance.

CONCLUSIONS: Despite the rare nature of the conditions, there were differences in approvals between England and Scotland, primarily driven by the justification of treatment cost in relation to health benefits. Patient and clinician engagement (PACE) in Scotland has raised a potential equity issue with HSTs approved in other parts of the UK that are not accepted for use in NHS Scotland. It remains to be seen if the ultra-orphan initial assessments will follow HST approvals in the future.