POSC266 Is Ultra-Orphan Disease Treatment Access Equitable in the UK? Comparison between SMC and NICE HST Guidance

Highly specialised technology (HST) evaluations in England are recommendations by the National Institute for Health and Care Excellence (NICE) on use of new existing highly medicines very rare conditions within Service (NHS). In Scotland, treatments considered eligible ultra-orphan pathway after initial assessment available NHS; Scottish Medicines Consortium (SMC) will reassess medicine decide routine availability 3 years following full assessment. We aimed to compare SMC decisions identified NICE HST evaluation. All published guidance were from database. Corresponding advice with a comparison status decisions. England, 14 reports identified. searches Scotland these found that: had not been reviewed, 4 assessments (considered therefore available), 7 assessments. Of SMC, (43%) approved, 1 (14%) was approved restrictions, recommended. The main reason non-recommendation lack justification treatment cost relation health benefits. economic analyses also generally sufficiently robust gain acceptance. Despite nature conditions, there differences approvals between primarily driven Patient clinician engagement (PACE) has raised potential equity issue HSTs other parts UK that accepted NHS Scotland. It remains be seen if follow future.